Don’t miss the start to the day with the Plenary Session. Enjoy your morning coffee as you listen to dynamic presentations intended to inspire and empower. Provided by ISPOR.
Head to the exhibit hall to refuel, recharge and connect with fellow attendees and exhibitors over a steaming cup of coffee. Provided by ISPOR.
As you enjoy your lunch in the Exhibit Hall, seize the opportunity to engage in meaningful conversations with fellow attendees. Take this time to exchange ideas, forge new partnerships, or simply enjoy causal conversation. Provided by ISPOR.
PURPOSE:
Cell and gene therapies (CGTs) can offer considerable life extension and quality of life improvements for patients with previously untreatable conditions. The European Medicines Agency and the Food and Drug Administration have each approved over 20 CGTs to date. Post approval, manufacturers face reimbursement challenges given many CGTs are up-front high-priced technologies combined with uncertainties around the durability of benefit. Associated financial risk as a function of these uncertainties have led to proposed alternative payment approaches that may improve access while creating predictable budget scenarios. However, reimbursement negotiations may be limited by using short-term regulatory approval evidence alone. The value of real-world evidence (RWE) combined with conventional simulation modeling methods can reduce the aforementioned uncertainties. This session will use case examples from different country-specific settings to discuss how RWE combined with simulation modeling approaches can help reimbursement agencies understand trade-offs of paying for CGTs.
DESCRIPTION:
Workshop attendees will obtain a working knowledge of how RWE and simulation modeling can inform payment strategies for up-front high-cost CGTs. Brett McQueen will provide an overview of global reimbursement strategies and examples of novel payment approaches for CGTs. Julia Slejko will present examples of how payment approaches have been implemented for CGTs in mainland Europe and the role of RWE. Antal Zemplenyi will present a case study illustrating the utilization of real-world data to investigate the association between eligibility criteria, the proportion of eligible patients, and the financial impact for the United States public Medicaid insurance program paying for gene therapies in hemophilia A and B. Finally, Andrew York will provide a policy perspective on the use of RWE to complement clinical trial data when considering different payment models for CGTs. Workshop participants will be asked about their own experience using RWE for payment approaches using ISPOR polling software.
Discussion Leaders
Robert McQueen, PhD
University of Colorado Anschutz Medical Campus, Denver, CO, USA
R. Brett McQueen is an Associate Professor at the University of Colorado (CU) Skaggs School of Pharmacy and Pharmaceutical Sciences, and member in the Center for Pharmaceutical Outcomes Research. His research interests include decision-analytic modeling applications and methodology, applied microeconometrics in health, and novel value assessment methods. Brett has current funding in micro-costing health interventions, evaluating performance-based risk sharing agreements, estimating patient and payer preferences for various pharmaceuticals, and novel value assessment methods. He is the course director for “Pharmaceutical Economics and Policy Analysis” in the Pharmaceutical Outcomes Research PhD program at CU.
Discussants
Julia F. Slejko, PhD
University of Maryland Baltimore, Baltimore, MD, USA
Julia F. Slejko, PhD is an Associate Professor of Practice, Sciences, and Health Outcomes Research at the University of Maryland School of Pharmacy and is Co-Director of the Patient-Driven Values in Healthcare Evaluation (PAVE) Center. Dr Slejko’s research is focused on innovative approaches for decision-analytic modeling for economic and health outcomes assessments. She holds a BA in Molecular, Cellular, and Developmental Biology from the University of Colorado Boulder. During her PhD training, she focused on pharmacoeconomics at the University of Colorado School of Pharmacy Center for Pharmaceutical Outcomes Research (CePOR). Her postdoctoral training was completed at the Pharmaceutical Outcomes Research and Policy Program in the University of Washington School of Pharmacy. Prior to her PhD training, she had a 7-year career in drug discovery at Array BioPharma. Dr Slejko serves as a Value in Health Associate Editor, Co-Chair of the ISPOR Faculty Advisor Council, co-lead of ISPOR’s Women in HEOR initiative, and is a member of ISPOR’s Health Science Policy Council.
Andrew York, PharmD, JD
Maryland Prescription Drug Affordability Board, Bowie, MD, USA
Dr. Andrew York is Executive Director of the Prescription Drug Affordability Board for the State of Maryland, and a pharmacist and attorney. He previously served in the Patient Care Models Group (PCMG) at the Center for Medicare and Medicaid Innovation (CMMI). Andrew received his PharmD from the University of Maryland School of Pharmacy and his JD from the University of Maryland Francis King Carey School of Law.
Antal Zemplenyi, PhD
University of Colorado Skaggs School of Pharmacy and Pharmaceutical Sciences, Aurora, CO, USA
Antal Zemplényi, PhD, is a health economist with more than 5 years of experience in academia specializing in HTA, health economics and outcome research and over 10 years of experience in the financial management of hospitals. He is the past president of the Hungarian Health Economics Association and the Hungary Chapter of ISPOR. He is the head of the Health Technology Assessment Centre at the University of Pécs and a senior health economist at the Syreon Research Institute. He is currently a Fulbright scholar based at the University of Colorado.
In or Out? Challenges in Determining Sample Selection Criteria and Recruitment Approaches for Patient Preference Studies
Digital Conference Pass
Level: Experienced
ISSUE:
Patient preference information is increasingly integral to decision-making along the medical product lifecycle (MPLC). As described in the ISPOR report the impact of patient-preference information on decision-making is enhanced by improving the acceptability of the results to decision makers. This involves thoughtful consideration of recruitment strategies that yield acceptable sample properties and response quality. While data quality has long been a key aspect of generating evidence on patient preferences, increased use of this evidence along MPLC decision-making makes it imperative to establish fit-for-purpose approaches for identifying, reaching, and surveying the right study participants for the various types of questions practitioners seek to address.
OVERVIEW:
The aim of this Issue Panel is to invite dialogue with the audience about existing recruitment challenges in health-preference research, and potential strategies to improve the appropriateness of the study sample and quality of the data collected from respondents. The audience will be polled to get their insights on potential recruitment approaches, importance of certain inclusion/exclusion criteria, and biggest barriers to obtaining high-quality, fit-for-purpose samples.
Moderators
Ellen Janssen, PhD
Janssen R&D, Baltimore, MD, USA
Dr. Janssen is a Director of Benefit-Risk assessment at Johnson & Johnson. There she leads patient-focused benefit-risk assessments and patient preference studies across a variety of therapeutic areas. Ellen is passionate about ensuring that the patient voice is reflected in medical decision making. Her work has focused on the application of good research practices for the conduct of stated-preference methods to ensure that patient preferences are transparently measured and accurately reflected. Ellen is involved as a preference expert in MDIC, and BIO. She previously participated in IMI PREFER and on the ISPOR Taskforce on using patient preferences to inform decision making.
Panelists
David Gebben, PhD
U.S. Food and Drug Administration, Silver Spring, MD, USA
David Gebben, PhD is a research economist focusing on stated preference methods applied within a health care context. Specifically, how stated preference methods can advance regulatory science to inform benefit risk evaluations, clinical trial endpoints, and inform ordering of patient priorities. David has worked at the FDA since June 2018.
Juan Gonzalez, PhD
Duke University, Cary, NC, USA
Dr. Gonzalez is an Associate Professor in the Department of Population Health Sciences at the Duke University School of Medicine. He is an expert in the design of stated-preference survey instruments and the use of advanced statistical tools to analyze stated-preference data. His research has focused on two main areas: 1) transparency in benefit-risk evaluations of medical interventions, and 2) elicitation of health preferences from multiple stakeholders to support shared decision making.
Dr. Gonzalez Co-led the first FDA-sponsored preference study. The study was highlighted in FDA’s recent precedent-setting guidance for submitting patient-preference evidence to inform regulatory benefit-risk evaluations of new medical devices. More recently, Dr. Gonzalez collaborated with the Medical Devices Innovation Consortium (MDIC) to prepare the first catalog of preference-elicitation methods suitable for benefit-risk assessments of medical devices. The catalog was part of the Patient-Centered Benefit-Risk Assessment Framework developed by MDIC. As a core group member of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Conjoint Analysis Task Force, Dr. Gonzalez helped draft good-practice recommendations for statistical analysis, interpretation, and reporting of health preference data. Dr. Gonzalez is the new Chair of the International Academy of Health Preference Research (IAHPR) and past Chair of the ISPOR special interest group on stated-preference research.
Jennifer Whitty, PhD
Evidera, part of PPD, London, United Kingdom
Dr Jenny Whitty, PhD, is a Senior Research Scientist with Evidera. As a health economics and outcomes researcher, she focusses on leading health preference studies and on their application to inform decisions across the product lifecycle, including regulatory and health technology assessment or payer decision-making. Dr Whitty has co-authored more than 130 peer-reviewed journal articles (including over 40 health preference studies), is a Board member for the International Academy of Health Preference Research (IAHPR) and holds Honorary appointments at the Professorial level with the Universities of East Anglia, UK and Queensland, Australia.
Rethinking Value Assessment of Preventive Health Interventions: The Health ROI Assessor Framework
Digital Conference Pass
Level: Intermediate
ISSUE:
Health investments drive global life expectancy, quality of life and economic growth. However, there is limited progress in increasing national budget allocation to the health sector. This narrows the room for decision-making inside the sector. Health Technology Assessment (HTA) enhances efficiency in healthcare by focusing on economic burden and costs-benefit evaluation of new health interventions. Nevertheless, understanding mechanisms how health investments yield economic gains remains incomplete. Furthermore, HTA focuses on curative care, which limits the applicability to preventive measures. Urgently, new outcome measures reflecting the health sector's economic contribution are needed. This workshop describes a novel approach designed to evaluate health investments from a social and macroeconomic perspective: The Health ROI Assessor framework provides a robust metric to assess economic returns on investment in specific interventions and thus complements HTA. We aim to debate and understand the applicability to different contexts, guiding budget allocation to and within the health sector.
OVERVIEW:
Professor Ostwald will discuss systematic measurement of value creation through health investments and introduce the approach. He will share recent experience on standardized health metrics. Professor Atun will cover new approaches of understanding preventive health interventions from a global health management and strategic health investment’s perspective. He will share his expertise in analysing health systems performance and the implementation of health reforms and medical innovations. Murugan Varnee will discuss challenges of infectious diseases and NCDs on global health and the economy. Addressing economic and social benefits of their prevention and treatment, she will emphasize the importance of value-based health care and strong partnerships between governments and private sectors. Carolina Goic will conduct her presentation from the perspective of a former senator and architect of the National Cancer Law in Chile. She will share her experience of policy and law making and the political processes related to investments in health.
Moderators
Dennis Ostwald, Prof., Dr.
WifOR Institute, Darmstadt, HE, Germany
Prof. Dr. Dennis A. Ostwald is CEO and co-founder of the independent research institute WifOR. Prof. Ostwald aims to instigate a paradigm shift towards recognizing health as an investment – into societal well-being, prosperity, and ultimately: better health for all. In recent years, Prof. Ostwald has spoken at the EU Parliament and the German Bundestag, as well as at events including the UN General Assembly, G20 Health & Development Partnership’s H20 Summit, and the CEO Summit of the Americas. He has a PhD from the University of Darmstadt and is a professor at the SIBE University.
Panelists
Rifat Atun, Prof., PhD
Harvard University, Cambridge, MA, USA
Rifat Atun leads the Health Systems Innovation Lab at Harvard University, where he is professor of global health systems. His research focuses on health-system performance, health-system transformation and innovation globally. Dr Atun has advised governments around the world on health-system reforms and is a consultant and adviser to the World Bank, World Health Organisation and leading health and technology companies. In 2019-20 he was a senior adviser to the G20 presidency, and in 2021-22 he co-chaired its T20 Task Force on Global Health Security and Covid-19. From 2008 to 2012 he was director of strategy, performance and evaluation at the Global Fund to Fight AIDS, Tuberculosis and Malaria.
Carolina Goic, MSc
Center for cancer prevention and control Medicine School Pontificia Universidad Catolica de Chile, Santiago, Santiago, Chile
Carolina Goic is a Chilenean politician and former two-time senator, now she is Executive director
Center for cancer prevention and control
Medicine School
Pontificia Universidad Catolica de Chile
Varnee Murugan, Master of Public Health
U.S. Chamber of Commerce, Bethesda, MD, USA
Varnee Murugan joined the U.S. Chamber of Commerce as executive director of the Global Initiative on Health and the Economy (GIHE). The GIHE champions public health, health financing, and regulatory policies that enable companies in the health care sector to deliver solutions that benefit people around the world. Murugan highlights the unique value of the private sector to drive sustainable public health outcomes at scale and employers that value a healthy workforce and its positive impact on productivity and economic growth.
Murugan’s career spans well over a decade across both U.S. and global health systems. Before joining the Chamber, she was a principal consultant at Kaiser Permanente’s (KP’s) national headquarters in California. There she led the development of enterprise-wide strategies to improve care delivery, access, and public health, working closely with the C-suite and other senior leaders across the organization.
Prior to KP, Murugan directed the Global Health Practice at Development Finance International where she managed a team across the U.S., India, and the Philippines. She supported leading health care companies in accelerating growth and gaining market access through policy advocacy and collaborative partnerships with government leaders, NGOs, and donors in international markets.
Earlier, Murugan worked at Avalere Health, a consultancy focused at the intersection of U.S. health policy and business strategy; the World Health Organization in Geneva; and Cetero Research in clinical data management. She has co-authored publications on health systems strengthening and care coordination outcomes.
Murugan holds a Master of Public Health from Yale University and a Bachelor of Science from Queen’s University in Ontario, Canada. She resides in Bethesda, Maryland, with her husband and young daughter.
2:30 PM - 3:15 PM
Student Networking Reception
Sponsor: National Pharmaceutical Council
2:45 PM - 3:15 PM
Coffee and Connect
Head to the exhibit hall to refuel, recharge and connect with fellow attendees and exhibitors over a steaming cup of coffee. Provided by ISPOR.
2:45 PM - 3:45 PM
Member Group Meetings
ISPOR Korea Chapter Meeting
The ISPOR Korea Chapter extends a warm welcome to anyone interested in connecting with us. Established in 2010, the chapter currently boast a great number of members. We invite you to join and discover what’s happening in the HEOR field in Korea.
3:00 PM - 3:45 PM
Special Onsite Join / Renewal Event for ISPOR Membership
Special Onsite Promo! Stop by the ISPOR Booth to join or renew your ISPOR membership! We might just have a surprise for you...
3:15 PM - 3:45 PM
Exhibit Hall Theater
Payer Perceptions of Health Inequalities in Reimbursement Decision Making: Implications for the Pharmaceutical Industry
Digital Conference Pass
Level: Introductory
Addressing health inequalities will become more important in future reimbursement and coverage decisions. However, there remains a need for a clear framework to be in place, a better understanding of how to measure inequality, and shared knowledge of how these factors will be considered relative to clinical outcomes. For example, future use of health technology assessment (HTA) tools such as cost-effectiveness may need to factor in unmet need for particular sub-populations of patients.
This HEOR Theater presentation will highlight recent recommendations from the Institute for Clinical and Economic Review (ICER) and from National Institute for Health and Care Excellence (NICE) related to HTA methods and health inequality. Results from a qualitative payer survey on their perceptions of health equity, its current and future role in decision-making will provide direct insight into current stakeholder views on this important issue.
Audience Objectives:
- Understand how health inequality is currently considered in the HTA and payer coverage & reimbursement decisions
- Find out what aspects of health inequality are prioritized by payers and HTAs
- Learn how HTA tools and needs may evolve to decrease health inequality and key challenges in achieving those goals
Sponsor
Genesis Research Group
Speaker
Tijana Ignjatovic, PhD
Genesis Research Group, Newcastle upon Tyne, England, United Kingdom
Tijana Ignjatovic is an Executive Director within the Access and Pricing Team and has over 15-years of consultancy experience in market access. In her time both in analysis and consultancy roles, Tijana has worked on multiple research methodologies and applied this experience, along with her market access knowledge, to lead and deliver numerous payer research projects in a broad range of therapeutic areas, including oncology, cardiovascular, infectious diseases, neurology, rheumatology, and many others. With a keen eye for detail, Tijana leverages her deep understanding of the industry and market access to quickly visualize and make connections on complex projects and provide strategic input to optimize clients’ market access strategy and value messaging across different payer requirements.
Kuldeep Singh, MIH, MBA
Genesis Research Group, Newcastle upon Tyne, England, United Kingdom
3:15 PM - 4:15 PM
ISPOR Forums
The Role for Health Economic Research in the Evolving US Healthcare Landscape: Perspectives From ISPOR and ASHEcon
Digital Conference Pass
The US healthcare system is changing rapidly. Horizontal consolidation within hospital, insurer, and provider industries; as well as vertical integration across industries is increasing. The role and influence of private payers in government programs, i.e., Medicare and Medicaid, is expanding. Moreover, the composition of stakeholders is changing as private equity’s positions in healthcare businesses grow and large tech companies like Amazon, Google, Apple, Uber, etc. move into healthcare. This dynamic environment presents myriad opportunities for HEOR contributions. The objective of this forum is to describe opportunities for ISPOR, its members, and the HEOR discipline to address issues of access, affordability, and quality at a healthcare system level. Given that the field of health economics, distinct from HEOR, has long focused on studying healthcare markets, financing, and policy, the session will feature American Society of Health Economists (ASHEcon) in addition to ISPOR representation. The forum will begin with the ISPOR Chief Science Officer providing context for ISPORs vision of broadening value at the healthcare system level and the forthcoming ISPOR strategic plan update. The remaining panelists will summarize US healthcare system trends and related research. Featured topics will include insurance coverage and its effect on patient access, outcomes, and provider behavior; consolidation, integration, and healthcare financing and the implications for costs and quality; and the HEOR research of interest to large health systems and large tech companies. Each panelist will identify priority research topics, discuss differences in HEOR and health economic methodological approaches to research, and discuss areas for potential collaboration between ISPOR and ASHEcon. A moderated discussion will follow where the panelists will describe their respective organizational perspectives, resources available to all researchers, and potential areas for member development. The panel will conclude with time for audience Q&A.
Moderators
Eric Barrette, PhD
Medtronic, Washington, DC, USA
Dr. Barrette is Senior Director, Global Health Economics and Outcomes Research at Medtronic. Prior to his current position, he has worked as a consultant, researcher, and University instructor. His research areas of interest include technology adoption, application of causal methods to real-world data, and competition in the health care industry and its effects on spending, utilization and outcomes.
Speakers
Stephen T Parente, PhD
University of Minnesota, Minneapolis, MN, USA
STEPHEN T. PARENTE, PhD, MPH, MS Stephen T Parente is the Minnesota Insurance Industry Chair of Health Finance in Carlson School of Management at the University of Minnesota and Associate Dean of the Carlson Global Institute.
As a finance professor, he specializes in health economics, information technology, and health insurance. He has been the principal investigator on large government and foundation funded studies regarding consumer-directed health plans, health information technology and health policy micro-simulation. He is the founding director of the Medical Valuation Laboratory, a nine-college interdisciplinary effort to accelerate medical innovation from scientists, clinicians, and entrepreneurs. Parente previously directed the AASCB award winning Medical Industry Leadership Institute at the University of Minnesota from 2006 to 2017.
Parente has been published in peer-reviewed journals on health reform, medical technology assessment, and consumer choices in health and wealth management, including JAMA, Inquiry, Health Services Research, the RAND Journal of Economics, Health Economics, the Journal of Health Economics, and Medical Care. He has been quoted and interviewed by the New York Times, Washington Post, National Public Radio, the PBS News Hour, USA Today, and The Wall Street Journal.
In Washington DC, he served in government as Chief Economist for Health Policy on the Council of Economic Advisers at the White House, Senior Adviser to the Secretary for Health Economics in the Department of Health and Human Services and adviser to the Congressional Budget Office. He is President-Elect of the American Society of Health Economists. Parente also served as Chair of the Health Care Cost Institute, health policy advisor for the McCain 2008 and Legislative Fellow in the office of Senator John D. Rockefeller IV (D-WV). He has a PhD in health finance and organization from Johns Hopkins University, and an MPH in health economics and an MS in public policy analysis, both from the University of Rochester in New York.
Laura Pizzi, PharmD, MPH
ISPOR, Lawrenceville, NJ, USA
Dr. Laura Pizzi is Chief Science Officer for ISPOR and Research Professor at Rutgers University. For nearly 25 years, she has led interdisciplinary teams of methodologists, statisticians, and clinicians to design and conduct economic and outcomes analyses on healthcare interventions and is a frequent author, speaker, and mentor on the topic.
At ISPOR, she leads the organization’s scientific strategy and initiatives in collaboration with ISPOR's global members and stakeholders.
Kosali Simon, PhD
Indiana University, Bloomington, IN, USA
Kai Yeung, PharmD, PhD
Amazon, Seattle, WA, USA
Exploring the Multifaceted Roles of Patients and Caregivers in Health Preference and Patient-Reported Outcomes Research
Digital Conference Pass
The forum aims to provide insight into the diverse roles patients and caregivers have and provides examples of these roles in patient-centered research. Case studies will be drawn from health preference and patient-reported outcome (PRO) research to enrich the discussion.
Siu Hing Lo will provide a broad overview of the roles patients and caregivers have as engaged research partners versus research participants in patient-centered research. She will then discuss how the form of patient/caregiver engagement could differ depending on the study objectives, existing evidence, practical considerations, and the intended use of the study findings. She will also highlight similarities and differences in patient/caregiver roles between health preference and PRO studies.
Holly Peay will discuss case studies in which patients and caregivers participated in a community engaged process for the development, interpretation, and dissemination of qualitative and quantitative HPR research. She will summarize barriers and facilitators to engagement and approaches to match engagement to stated preference research objectives.
Ryan Fischer will discuss his experience collaborating with academic and industry partners for advocacy-led patient preference and experience studies, and how advocacy organizations use the results generated from such studies in their strategic planning, advocacy, education, and research funding.
Fraser Bocell will discuss the roles of patients and the use of patient engagement in selecting, modifying, and adapting meaningful PRO instruments, from a regulator’s perspective.
Speakers will use polling questions to engage the audience in discussion.
This forum is a collaboration presented by the Health Preference Research and the Patient-Centered Special Interest Groups.
Moderators
Siu Hing Lo, PhD
Acaster Lloyd Consulting Ltd, London, LON, United Kingdom
Siu Hing is an Associate Director at Acaster Lloyd Consulting Ltd, London, United Kingdom. She specialises in patient-centered research, including preference surveys, utility studies and COA development and analysis. She is the Chair of the Health Preference Research Special Interest Group (HPR SIG).
Speakers
Fraser Bocell, PhD
U.S. Food and Drug Administration, Silver Spring, MD, USA
Fraser D. Bocell, MEd, PhD is the Patient-Science Team Lead and a Psychometrician with the Division for Patient Centered Development in the Center for Devices and Radiological Health at the US FDA. At CDRH he provides expertise and training, leads Patient-Centered research, and helps develop policy on the evaluation and use of COAs in regulatory decision-making. His work helps bring patient voices to regulatory decision making.
Ryan Fischer, BA
Foundation for Angelman Syndrome Therapeutics, Marble Falls, TX, USA
Ryan serves as Chief Operating Officer for the Foundation for Angelman Syndrome Therapeutics or FAST. Ryan works in collaboration with FAST’s President to establish, direct, and achieve the organization’s short and long-term strategic goals and objectives across all operational areas, including general administration, programs, grants, and external relations. He maintains and builds effective relationships with key stakeholders, including Congress, regulators, pharmaceutical companies, other foundation leaders, and the broader rare disease advocacy community. Prior to joining FAST, Ryan served as Chief Advocacy Officer for Parent Project Muscular Dystrophy (PPMD). Ryan held various roles within PPMD over 18 years helping to grow the organization into one of the leading rare diseases advocacy nonprofits globally. In 2012, Ryan was a member of the core team (in partnership with Johns Hopkins School of Public Health) that developed and produced a novel patient preference study on caregiver benefit-risk preferences and priorities for emerging treatments in Duchenne. Since then, Ryan has co-led several additional preference studies and advocates for advancing the science of patient input within Patient-Focused Drug Development.
Holly Peay, PhD
RTI International, Research Triangle Park, NC, USA
Holly Peay is a Senior Research Scientist at RTI International. She conducts research on the ethical application of new technologies in research, clinical, and public health settings. Her research includes patient and stakeholder preference studies, decision support interventions, and engagement science.
ISPOR Latin America Health Update Forum: Tackling NCDs, Multimorbidity, and Medical Innovations’ Social Impact
Digital Conference Pass
Hosted by the ISPOR Latin America Consortium, the forum will present research conducted in Latin America on the social impact of Non-Communicable diseases (NCDs) from the WifOR Institute and on Multimorbidity from the World Bank’s Global Health Unit.
Chronic or non-communicable diseases (NCDs) pose a global health challenge, particularly affecting the poor and vulnerable due to their higher exposure to risks, reduced access to health services, and health promotion and prevention programs. The impact of NCDs is further amplified by the fact that many patients have multiple chronic conditions simultaneously. Multimorbidity (defined as the presence of two or more chronic conditions in the same person) is increasingly recognized as a challenge for individuals, caregivers, families, community, and health systems, due to its social, economic, and public health impact.
Professor Dennis Ostwald (WifOR) will present a novel approach to evaluating the social impact of medical innovations, focusing on informed healthcare decision-making and sustainable development. Roberto Iunes (World Bank) will discuss the prevalence and economic burden of multimorbidity and NCDs in Latin America.
Finally, a moderated panel and Q&A session will be offered at the end of presentations for interaction and engagement with attendees.
Moderators
Diego Guarin, MD MPH
ISPOR Latin America Consortium, Rahway , NJ, USA
Dr. Diego Guarin is the Regional Market Access Lead for Latin America at Merck (known as MSD outside USA and Canada), where he focuses on improving patient access to health innovations. Since 2008, he has led Regional Market Access and Evidence and Value Development (HEOR) teams at top global biopharmaceutical companies. In addition to his industry experience, he has worked as a consultant for multilateral organizations (PAHO, GFATM) and in a health policy think-tank.
Dr. Guarin is a founding member of the ISPOR Colombia chapter and has served as chair of the ISPOR Latin American Consortium Industry Committees and Advisory Board, as well as the first representative of Latin America at the ISPOR Health Science Policy Council. In 2023, he was appointed chair of the ISPOR Latin America Consortium Executive Committee. For the past 10 years, he has been a company representative at FIFARMA, the Latin American Federation of the Pharmaceutical Industry, and served as co-Chair of two working groups (Value and Access to Innovation and Health Policies).
Dr. Guarin graduated as a Medical Doctor from Universidad del Rosario-1653 (Colombia) and holds master’s degrees in public health in Developing Countries from the London School of Hygiene and Tropical Medicine (UK), in Health Economics and Pharmacoeconomics from University Pompeu Fabra (Spain), and in Health Economics Policy and Management from the London School of Economics and Political Science (UK).
Speakers
Roberto Iunes, PhD
The World Bank, Washington, DC, USA
Roberto Iunes is a Senior Economist at the World Bank’s Global Health Unit. He coordinates the SaluDerecho Initiative, supporting nine Latin American countries in progressively realizing the right to health. In Latin America, he has led the Bank’s work in the health sector in Colombia, El Salvador, and Mexico, and is currently also working with Brazil.
Before joining the World Bank, he worked for over thirteen years at the Inter-American Development Bank, designing and implementing health projects, and at the Independent Office of Evaluation and Oversight.
He was also a professor and researcher in health economics and nutrition at the Faculty of Public Health at the University of São Paulo, and a Takemi Fellow and Research Fellow in the Health Financing Program, both at Harvard University.
In the Brazilian public sector, he worked for the São Paulo state and municipal health secretariats. He has published several articles in books and academic journals.
Dennis Ostwald, Prof., Dr.
WifOR, Darmstadt, HE, Germany
Prof. Dr. Dennis A. Ostwald is CEO and co-founder of the independent research institute WifOR. Prof. Ostwald aims to instigate a paradigm shift towards recognizing health as an investment – into societal well-being, prosperity, and ultimately: better health for all. In recent years, Prof. Ostwald has spoken at the EU Parliament and the German Bundestag, as well as at events including the UN General Assembly, G20 Health & Development Partnership’s H20 Summit, and the CEO Summit of the Americas. He has a PhD from the University of Darmstadt and is a professor at the SIBE University.
Educational Symposia
Harnessing the Power of AI: Revolutionizing HEOR and Market Access
Digital Conference Pass
The application of generative and predictive AI in Health Economics and Outcomes Research and Market Access is changing the way we approach evidence generation and healthcare decision-making. This growing interest is exemplified by the recent increase in literature. A targeted search of PubMed and the ISPOR database identified 600+ publications on the application of AI in HEOR methods since 2018 and 200+ poster/panel presentations at ISPOR on AI in 2023 alone. While there is significant promise in AI for HEOR and Market Access, it remains a nascent field, and questions remain on how and where to best apply AI and how to drive adoption.
Our goal is to explore approaches to leverage AI in HEOR and Market Access and relate real-world learnings. Our experts will relate their experiences on the application of AI by exploring tangible examples in HEOR and Market Access. They will share their experiences including challenges faced and best practices learned.
The symposium will explore the potential of AI to improve specific aspects of HEOR research, including methodologies and or areas where AI can be applied to improve efficiency while maintaining quality and rigor. Predictive and generative AI has the potential to transform the way pharma conducts HEOR and Market Access research through its ability to analyze and summarize large amounts of data, extract insights, and provide institutional memory to new team members. Specific applications developing content for dossiers and product value messaging, summarizing and synthesizing HTA decisions, developing access rationales and predicting access outcomes.
Panelists will discuss the importance of setting clear goals/objectives, cross-functional alignment and a multi-stakeholder approach to fully realize the potential of AI in HEOR and Market Access. Our goal is to cut through the noise and provide real-world examples of what has worked and what has not and what the future holds.
Sponsor
Trinity Life Sciences
Panelist
James Creeden, MD, PhD
CGC Consulting, Basel, Switzerland
Over 20 years of experience in medical strategy and leadership in Pharma, Diagnostics and Decision Support industries in the US, Europe and Asia. Expertise in precision oncology, companion diagnostics and cancer genomics. Former Chief Medical Officer at Roche Diagnostics, Country Medical Director for Roche Pharma China, and Global Medical Director at Foundation Medicine. In 2023, co-founded an on-demand cancer genomics report interpretation service and developed an explainable AI-based LLM for semi-structured data classification and complex report synthesis using reinforcement learning.
Board member at Fromtestingtotargetedtreatments.org, a patient advocacy consortium.
Francesco De Solda, PharmD, MBA
Janssen Global Services LLC, Raritan, NJ, USA
Francesco De Solda is a seasoned pharmaceutical professional with a rich background in Market Access, Health Economics and Outcomes Research, and R&D. With extensive leadership experience in the Pharmaceutical Industry in different functions and geographies, and a solid academic foundation - including a Doctorate in Pharmacy, a Master in Business Administration, a post-graduate degree in Clinical Research, and Executive Certificates in Health Economics, HTA, Market Access and Government Affairs - Francesco excels in driving global market access strategies for innovative healthcare solutions. Passionate about Oncology, he brings a dynamic blend of expertise and strategic vision.
Staying Ahead of the Curve: Navigating Policy Changes and Ensuring Patient Access in the Era of IRA and EU HTA
Digital Conference Pass
In this session, we will delve into the complex landscape of policy changes affecting patient access to healthcare. We will explore two key areas: Inflation Reduction Act (IRA) in the United States and Health Technology Assessment (HTA) in Europe.
Our expert speakers will provide a comprehensive understanding of these policy changes, their objectives, implementation timelines, and associated challenges.
Key learnings:
- Understanding policy changes and timelines: Overview of IRA in the US and EU HTA in Europe, key objectives and implementation timelines, key challenges
- Considerations for stakeholders, including biopharma companies, healthcare providers, payers, and patients: Ensuring alignment and collaboration, leveraging data-driven insights for informed decision-making, strategies at national level, tools and resources to accelerate patient access
- Q&A with the audience
Join us for an engaging session where you will gain valuable insights into navigating policy changes and discover strategies to ensure patient access amidst the evolving landscape of IRA and EU HTA.
Sponsor
Cencora
Moderators
Corey Ford, MHA
Cencora, Conshohocken, PA, USA
Panelists
Michael Drummond, PhD
University of York, Lichfield, United Kingdom
Michael Drummond is Professor Emeritus of Health Economics and former Director of the Centre for Health Economics at the University of York. His particular field of interest is in the economic evaluation of health care treatments and programmes. He has undertaken evaluations in a wide range of medical fields including care of the elderly, neonatal intensive care, immunization programmes, services for people with AIDS, eye health care and pharmaceuticals. He is the author of two major textbooks and more than 750 scientific papers, and has acted as a consultant to the World Health Organization and the European Union. He has been President of the International Society of Technology Assessment in Health Care, and the International Society for Pharmacoeconomics and Outcomes Research. In October 2010 he was made a member of the National Academy of Medicine in the USA. He has advised several governments on the assessment of health technologies and chaired one of the Guideline Review Panels for the National Institute for Health and Care Excellence (NICE) in the UK. Until recently he was Co-Editor-in-Chief of Value in Health and has been awarded 3 honorary doctorates, from City University (London), Erasmus University (Rotterdam) and the University of Lisbon.
Casper Paardekooper, MSc
Vintura, part of Cencora, Utrecht, Netherlands
Casper Paardekooper is Partner at strategy consultancy Vintura, part of Cencora, which focuses on transforming healthcare, making it more accessible and beneficial for all. In Vintura he is head of the Life Sciences practice, and he also leads Vintura's Value, Access & Policy Center of Excellence. In that capacity, he focuses on access policy, market access strategy, value demonstration and value-based healthcare. He is also part of Cencora’s EU HTA Center of Excellence, where he particularly focuses on organizational readiness of his clients. Casper is a trusted advisor to many large biopharma companies and brings more than 15 years of strategy consultancy in life sciences and healthcare.
Kimberly Westrich, MA
National Pharmaceutical Council, Herndon, VA, USA
Kimberly Westrich, MA, is the Chief Strategy Officer of the National Pharmaceutical Council (NPC), which sponsors and conducts research on health policy issues related to the development and use of innovative biopharmaceuticals to improve the health of patients. Ms. Westrich leads several research initiatives across NPC’s portfolio, including employer-sponsored insurance. She has published extensively on issues related to value assessment frameworks, patient-centered formulary and benefit design, value-based contracts, quality performance measurement, and accountable care organizations.
3:30 PM - 4:15 PM
Poster Tours
AI in HEOR Poster Tour
Posters Featured in this Tour:
PT13: Claims-Based Machine Learning Algorithms to Predict ECOG Performance Status (ECOG) for Pan-Cancer (Prostate, Breast, Colorectal, Gastric, Non-small Cell Lung (NSCLC), and Pancreatic Cancer) Patients
PT14: The Effectiveness of Bisphosphonates on Recurrent Osteoporotic Fractures Post-Denosumab Discontinuation Using Causal Machine Learning
PT15: Machine Learning Vs Traditional Statistics: Developing a Novel Proxy for HPV-Associated LA SCCHN
PT16: A Machine Learning-Based Approach to Predicting Acute Kidney Injury and Associated Medication Regimen Use in Critically Ill Adults
PT17: Addressing Gaps in Real-World Data Sources through Automated Extraction of Psychiatric Comorbidities and Medication Side Effects from Clinical Notes
PT18: Predicting Cost-Related Medication Adherence in Patients with Cognitive Impairment: A Machine Learning Approach
Moderator
Kyeryoung Lee, PhD
IMO health, Ardsley, NY, USA
Methodology Research in HEOR Poster Tour
Posters Featured in this Tour: PT19: Developing a Bespoke Neural Network Model for Diagnosing Alzheimer's Dementia: A Fit-for-Purpose Machine Learning Study PT20: Gleaning Novel Insights from Real World Data: A Machine-Learning Guided Analytical Framework PT21: A Generalized Framework for Eliciting Unreported Subgroup-Specific Events and Survival Outcomes from Aggregate Level Data: Analyses & Insights from Adjuvant & Advanced Stage Gastrointestinal Cancers PT22: Consistency between Comparative Efficacy Estimates from Target Trial Emulations and Their Origin Target Trials - a Meta-Epidemiological Study PT23: Is a Large Language Model (LLM) More Accurate Than Human Researchers in Correctly Identifying Diseases in Biomedical Abstracts? a Pilot Study PT24: Quality Appraisal of Randomized Controlled Trials Using Robins 2.0 Tool: A Case Study on Comparing the Performance of CHATGPT4.0 with a Human Reviewer
Moderator
Surrey M Walton, PhD
Institute for Clinical and Economic Review, Boston, MA, USA
3:30 PM - 6:30 PM
Poster Session 2
Live
3:45 PM - 4:15 PM
Exhibit Hall Theater
Accelerating Therapy Adoption: The Power of Regulatory-Grade EHR Data and Clinical Expert-Led AI
Digital Conference Pass
The potential of real-world data to advance research and speed therapy adoption has been recognized for some time, but fragmentation and data inaccessibility challenges have slowed progress. Truveta’s deep health system partnerships enable timely data access, and our clinical expert-led AI capabilities facilitate normalization of massive volumes of healthcare data at scale. Learn how high-quality, research-ready data from EHRs is being used to fuel novel and efficient research on safety, comparative effectiveness, label expansion, and closing care gaps.
Sponsor
Truveta
Speaker
Ryan Ahern, MD
Truveta, Bellevue, WA, USA
Dr. Ryan Ahern is Co-founder and Chief Medical Officer of Truveta and has more than a decade of experience in healthcare data and clinical research. At Truveta, Ryan leads the Life Science Partner team which focuses on developing new partnerships and ensuring those partners are successful in their research. He is currently a Clinical Assistant Professor of Medicine at the University of Washington School of Medicine and practices at Harborview Medical Center, Seattle’s large county hospital. Prior to this, he trained in internal medicine at the Massachusetts General Hospital and has served as a Clinical Assistant Professor of Medicine at Weill Cornell Medical Center and New York Presbyterian; attending physician at Massachusetts General Hospital; and instructor at Harvard Medical School.
Anita Loughlin, PhD, MS
Moderna Therapeutics Inc, Cambridge, MA, USA
Anita M. Loughlin, PhD
Director Epidemiology, Rare Disease and Therapeutics, at Moderna Therapeutics Inc; where she is the Lead Epidemiologist studying Ornithine Transcarbamylase Deficiency (OTCD), the most common urea cycle disorder. Anita has had a distinguished career in clinical research working both nationally and internationally, and is an experienced pharmacoepidemiologist. Formerly, she was the Senior Director of Epidemiology at CorEvitas, an autoimmune registries company, and prior to this she was a Senior Epidemiologist at Optum Epidemiology. She has a depth of experience designing and conducting research using RWD, specifically claims, Electronic Medical Record (EMR), and registries and other linked data. In addition, she was an Assistant Professor at both Boston University School of Medicine (Dept of Pediatric Infectious Disease) and School of Public Health (Dept of Epi), and over saw both vaccine trials as well as post licensure safety and effectiveness research. Anita obtained her PhD in Epidemiology from Johns Hopkins Bloomberg School of Public Health, with specialties in Infectious Diseases and Clinical Trials, and a MS in Epi from University of Massachusetts Amherst.
At Moderna, Anita brings an understanding of strengths and weaknesses of epidemiologic, outcomes research and RWE to meet advance Moderna’s capabilities and needs in Therapeutics.
3:45 PM - 4:45 PM
ISPOR Strategic Plan Session
ISPOR’s New 2030 Strategy – Raising the Global Profile and Impact of HEOR
Level: Introductory
Join us for an exclusive Connection Zone session led by ISPOR executive leadership as we take a deep dive into ISPOR’s new Strategic Plan 2030. During this session, attendees will learn about ISPOR’s bold new vision, mission, and core values, as well as the overarching goals and objectives we will be pursuing over the next 5-6 years. Attendees will be encouraged to engage in an open dialogue and share their ideas about how to implement the plan.
Moderators
Brian O'Rourke, PharmD
ISPOR, Lawrenceville, NJ, USA
Dr. Brian O’Rourke is the President of ISPOR for the period from July 2023 to June 2024. He served as the President and Chief Executive Officer of CADTH from 2009-2020. He joined CADTH following a distinguished career as a Pharmacist and Health Care Executive with the Canadian military. With over 40 years of experience in health care, Brian is a leading expert in the science and practice of health technology assessment (HTA) and served as the Board Chair for the International Network of Agencies for Health Technology Assessment from 2014 to 2018. He has a Bachelor of Science in Pharmacy from Dalhousie University and a Doctor of Pharmacy from the University of Toronto. Dr. O’Rourke continues to play an active role in the global HTA community. He is Chair of the Health Technology Assessment Steering Committee and a member of the Scientific Advisory Council at the Centre for Innovation in Regulatory Science (CIIRS). In November 2020, Dr. O’Rourke was appointed Colonel Commandant (Honorary) of the Royal Canadian Medical Service.
Speakers
Rob Abbott
ISPOR, Lawrenceville, NJ, USA
Mr Abbott is a highly accomplished, visionary leader with more than 20 years’ experience providing executive-level strategic and operational leadership in businesses, nongovernmental organizations (NGOs), government environments, as well as professional societies and associations. Most recently, he served as executive director and CEO of Health Technology Assessment International, a global nonprofit society focused on health technology assessment (HTA). He is recognized for his knowledge in HTA, strategy, and stakeholder relations. He has successfully guided a number of organizations through their transformation and growth. He is also a pioneering social responsibility thinker and advisor who has authored 2 books and numerous articles on the nature of business and society. Mr Abbott holds an undergraduate honors degree from the University of Victoria and graduate degrees from the University of Toronto. Additionally, he holds professional designations as both a management consultant and an ICF-accredited coach.
Mitchell Higashi, PhD
ISPOR, Lawrenceville, NJ, USA
Mitch Higashi, PhD, is ISPOR's Associate Chief Science Officer. In this role, Dr Higashi contributes to the development and deployment of ISPOR’s science and content strategies. He also collaborates with the Society’s members, groups, stakeholders, and staff to advance initiatives that lead to increased awareness, understanding, and application of HEOR around the world.
Dr Higashi has more than 20 years’ experience in the field of HEOR. Most recently he served as Senior Vice President, HEOR at GeneDx. Previously he was Vice President, HEOR at Bristol-Myers Squibb and has worked in related roles at GE Healthcare and GlaxoSmithKline. Dr Higashi earned his PhD in health economics and outcomes research at the University of Washington. He has been an active member at ISPOR during his career, serving on the Society’s Health Science Policy Council from 2012 – 2017 and most recently as an ISPOR 2023 conference committee co-chair.
Eberechukwu Onukwugha, BA, MSc, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
Eberechukwu Onukwugha, PhD is a Professor in the Department of Practice, Sciences, and Health Outcomes Research and is the Executive Director of Pharmaceutical Research Computing at the University of Maryland School of Pharmacy. She received a Master of Science in agricultural and applied economics as well as a Doctor of Philosophy in economics (concentration: econometrics) from Virginia Tech. Dr. Onukwugha has approximately 20 years of experience conducting health economics and outcomes research. She examines the costs and health outcomes associated with health-related decisions as well as the institutional and environmental context framing these decisions.
Laura Pizzi, PharmD, MPH
ISPOR, Lawrenceville, NJ, USA
Dr. Laura Pizzi is Chief Science Officer for ISPOR and Research Professor at Rutgers University. For nearly 25 years, she has led interdisciplinary teams of methodologists, statisticians, and clinicians to design and conduct economic and outcomes analyses on healthcare interventions and is a frequent author, speaker, and mentor on the topic.
At ISPOR, she leads the organization’s scientific strategy and initiatives in collaboration with ISPOR's global members and stakeholders.
4:00 PM - 4:45 PM
Student & Faculty Meet and Greet
Come meet the student and faculty leaders and converse with your peers about all things pertaining to ISPOR Student Chapters! Faculty will have the opportunity to meet with other members from the Faculty Advisor Council, and Student Leaders will also be able to meet with their peers and faculty.
4:15 PM - 5:30 PM
Member Group Meetings
ISPOR Boston Chapter Meeting
This meeting welcomes all those with an interested in the ISPOR Boston chapter. It’s a fantastic opportunity to dive deeper into the chapter’s activities and discover how to actively participate.
ISPOR Patient-Centered Research Special Interest Group Joint Open Meeting
This meeting aims to serve as a dynamic platform where the Patient-Centered, Clinical Outcome Assessment, and Health Preference Research Special Interest Groups showcase their ongoing initiatives and present their upcoming projects. In addition, this Open Meeting fosters invaluable networking opportunities, facilitating connections and collaborations to advance the field of patient-centered research.
5:00 PM - 6:00 PM
Podium Session
Addressing Confounding in Real-World Evidence Studies
Digital Conference Pass
Moderator
Boris Gorsh, PharmD
Daiichi Sankyo, Inc., Yardley, PA, USA
Boris is an experienced Health Economics and Outcomes researcher with a Doctor of Pharmacy degree from University of Rhode Island. He has previously completed a fellowship in outcomes research with UT Austin, Novartis and Scott & White health plan.
Currently, he is a Senior Director and leads the global oncology HEOR & RWE team for pipeline assets at Daiichi Sankyo within the Global Oncology Value Access & Pricing (GOVAP) team. His prior experience includes similar roles at GSK as Director and Value Evidence and Outcomes (VEO) lead for several oncology products, Pfizer and BMS.
Boris is passionate about drug development and the incorporation of value and the patient experience into evidence generation planning. He has extensive experience in real-world evidence, patient reported outcomes, economic model development and strategic planning to support regulatory and access decision-making. Boris has participated in key regulatory decision making processes including FDA advisory committee interactions, regulatory submission document development for FDA/EMA filings, orphan drug designation evaluations, HTA/ICER interactions and has led numerous studies for scientific communication.
P17: The Cardiovascular Effect of Abiraterone and Enzalutamide Among Veterans Affairs’ Metastatic Castration-Resistant Prostate Cancer Patients
5:00PM - 5:15PM
Baser O1, Baser E2, Samayoa G3
1City University of New York, New York, NY, USA, 2Columbia Data Analytics, New York, UNITED STATES, 3Columbia Data Analytics, New York, NY, USA
OBJECTIVES: There is growing concern about the adverse metabolic and cardiovascular effects of two standard hormonal therapies for prostate cancer, abiraterone acetate (AA) and enzalutamide (ENZ). We analyzed the risk of cardiovascular adverse events among patients treated with AA and ENZ. This retrospective cohort study aims to analyze the risk of developing cardiovascular adverse events during treatment with AA and ENZ among the veterans’ prostate cancer population.
METHODS: Using Veterans Administration data from January 2019–October 2022, we identified patients with prostate cancer and divided them into two cohorts: patients treated with AA (NDC code 71921-0178) or ENZ (NDC code 0469-0625). Index date was defined by >1 pharmacy claims for AA and ENZ during January 2020–December 2021. We then defined cardiovascular event presentation by a composite of several cardiovascular diseases. Descriptive and multivariate analysis were conducted using SAS. Propensity matching technique was used for risk adjustment.
RESULTS: We analyzed 972 and 3537 patients in the AA and ENZ cohorts, respectively. After controlling for age, region, and comorbidity differences, propensity score matching yielded 956 matched patients. Follow-up cardiovascular events—hypertension (46.03% vs 45.40%), ischemic heart disease (16.84% vs 17.26%), myocardial infarction (1.88% vs 2.30%), heart failure (10.77% vs 9.62%), ventricular arrhythmias (5.02% vs 5.33%), cerebral infarction (2.09% vs 2.51%), peripheral vascular diseases (3.77% vs 4.08%), pulmonary heart diseases (0.94% vs 1.36%), atrial fibrillation (13.18% vs. 11.92%), paroxysmal tachycardia (1.88% vs 1.67%), cardiomyopathy (2.62% vs 3.14%), pulmonary embolism (2.51% vs 1.78%), and aortic aneurysm (2.41% vs 2.20%)—were similar between AA users and ENZ users.
CONCLUSIONS: This study provides real-world evidence of the cardiovascular risk of AA and ENZ that may not appear in clinical trial settings. Adjusting for age, baseline comorbidities, and socioeconomic status, the likelihood of a cardiovascular event did not differ between treatment groups.
P19: Negative Control Outcomes (NCOs) to Assess Comparability of Psoriasis (PsO) and Psoriatic Arthritis (PsA) Treatments
5:30PM - 5:45PM
Horner ME1, Ogdie AR2, Kent ST3, Tran KT3, Deignan C3, Cordey M3, Orroth KK3, Brookhart MA4
1Dermatology Consultants of Sacramento, Sacramento, CA, USA, 2Hospital of the University of Pennsylvania, Philadelphia, Pennsylivania, PA, USA, 3Amgen Inc, Thousand Oaks, CA, USA, 4Duke University, Durham, NC, USA
OBJECTIVES: Treatment selection is often associated with patient prognosis, leading to confounding when comparing effectiveness between treatments in clinical practice. We used NCO to assess residual confounding when comparing apremilast versus other PsO/PsA treatments.
METHODS: Adults ≥18 years old, with PsO/PsA, initiating apremilast, topicals, methotrexate, tumor necrosis factor inhibitor (TNFi), interleukin (IL)-12/23i, or IL-17i (index date) since 9/23/2016 from the Optum Clinformatics® DataMart database were included. Follow-up ended at treatment switch/discontinuation, NCO, end of enrolment or 9/30/2022. NCOs were assessed for frailty (accidents, decubitus ulcer, fractures); healthy user behavior (wellness visit, herpes zoster vaccine, colon cancer screening, pelvic screening); and treatment channeling for methotrexate (substance abuse, transfusions). NCO relative risks (RR) and 95% confidence intervals (CIs) for apremilast versus other treatments were estimated using inverse probability of treatment and censoring weights.
RESULTS: Treatment cohorts for PsO included 111,070, 5,105, 5,572, 7,779 and 2,359 new users of topicals, methotrexate, TNFi, IL-12/23i and IL-17i, respectively; the apremilast PsO cohort ranged from 6,663–7,264 depending on comparison treatment. RRs of most NCOs were comparable across treatments. Wellness visits and pelvic examinations were less likely among apremilast versus topicals users, RR (95% CI), 0.84 (0.71, 0.97) and 0.82 (0.66, 0.99), respectively. The difference in wellness visits was attenuated in individuals receiving ≥1 topical before index (0.89 [0.76, 1.03]). No significant differences were observed between apremilast versus methotrexate or IL-12/23i. Colon cancer screening was more likely in apremilast versus IL-17i users (1.43 [1.07, 1.79]), suggesting healthy user bias. NCO events for frailty were low and not informative. Results from the PsA cohort showed no residual confounding between apremilast and other treatments.
CONCLUSIONS: Eligibility criteria and weighting reduced residual confounding when comparing real-world effectiveness of apremilast with other treatments. NCOs should be integrated into comparative effectiveness/safety studies of PsO/PsA treatments to detect potential residual confounding and ensure robust results.
P20: Cardioprotective Effectiveness of Sodium-Glucose Cotransporter-2 Inhibitors in Early Breast Cancer Following Anthracycline- or Trastuzumab-Based Chemotherapy
5:45PM - 6:00PM
Liu YS, Barner JC, Lawson K, Liu Y, Park C
The University of Texas at Austin, Austin, TX, USA
OBJECTIVES: Given the growing body of evidence supporting the cardioprotective benefits of sodium-glucose cotransporter-2 inhibitors (SGLT2is) in type 2 diabetes, this study aims to examine whether these benefits extend to older women with early-stage breast cancer (BC) following anthracycline- or trastuzumab-based therapies.
METHODS: This retrospective cohort study used the 2011-2019 Surveillance, Epidemiology, and End Results (SEER)-Medicare database. We identified women over 65.5 years and diagnosed with stage I-III BC who received anthracycline or trastuzumab and subsequently initiated anti-diabetic medications. Propensity scores were used to match one new-user episode of SGLT2is with four new-user episodes of other anti-diabetic medications (OAMs). The primary outcome was a composite endpoint consisting of heart failure (HF), stroke, myocardial infarction, and arrhythmia. Secondary outcomes included HF hospitalization, and HF or cardiomyopathy incidence. Cause-specific hazard ratios (csHR) between the SGLT2i and OAM groups were assessed for each outcome, with all-cause death treated as a competing event.
RESULTS: A total of 1,777 new-user episodes were identified from 1,195 women. After 1:4 matching, there were 131 episodes in the SGLT2is and 469 episodes in the OAMs. Patient characteristics including sociodemographic, cancer-related factors, comorbidities, and co-medications were well-balanced between the two groups. Although the point estimates favored the initiation of SGLT2is over OAMs, no significant differences were observed in the primary outcome (csHR=0.71; 95% CI: 0.44-1.15), HF hospitalization (csHR=0.92; 95% CI: 0.10-8.27), and HF or cardiomyopathy incidence (csHR=0.66; 95% CI: 0.24-1.82). The directions of the results were consistent across individual SGLT2i and patient subgroups, including those with and without established cardiovascular diseases.
CONCLUSIONS: No significant differences in cardiovascular risks were found between women with early BC who initiated SGLT2is versus OAMs after anthracycline or trastuzumab treatments, which might be due to the limited sample size. However, our study could inform future studies on SGLT2is’ cardioprotective benefits in these patients.
P18: Assessing the Value of Revascularization Strategies for Chronic Limb Threatening Ischemia in Patients with Diabetic Foot Ulcers: A Propensity Score-Weighted Evaluation
5:15PM - 5:30PM
Zheng H1, Tan TW1, Armstrong DG2, Padula W1
1University of Southern California, Los Angeles, CA, USA, 2Keck School of Medicine, University of Southern California, Los Angeles, CA, USA
OBJECTIVES:
To compare the real-world outcomes of open and endovascular revascularization approaches in chronic limb-threatening ischemia (CLTI) patients with diabetic foot ulcers (DFUs).
METHODS:
We conducted a retrospective cohort study using the OPTUM’s de-identified Clinformatics® Data Mart (2010-2021), focusing on patients who underwent revascularization after the initial diagnosis of DFUs. Outcomes of the open-first and endovascular-first revascularization strategies included major amputation-free survival, major adverse limb events (MALE, defined as major amputation or reintervention), and reintervention. Unadjusted and adjusted multivariate regression models were used to assess the association between the revascularization strategy and outcomes. Propensity score weighting was applied to balance the cohorts.
RESULTS:
Among the 19,802 patients included, 85.3% underwent endovascular revascularization and 14.7% underwent open bypass. Patient characteristics were comparable after propensity score weighting. The 12-months major amputation rate was higher with open revascularization (10.9% vs. 8.1%). In the unadjusted KM survival analysis, although the overall mortality and major amputation-free survival were similar, the open-first group had a higher amputation rate (p<0.0001) but a lower MALE rate (p<0.0001), primarily driven by fewer reinterventions. In the Cox model, the open-first approach was associated with a higher likelihood of major amputation (Hazard Ratio [HR] 1.18, 95% Confidence Interval [CI] 1.07-1.30; propensity score weighted cohort: HR 1.28, 95% CI 1.16-1.44) than endovascular-first revascularization. In logistic regressions, open-first revascularization was associated with a lower probability of MALE (34.2% vs. 41%; Odds Ratio (OR) 0.80, 95% CI 0.75-0.85) and reintervention (28.5% vs. 37.3%; OR 0.65, 95% CI 0.6-0.71) at 12 months.
CONCLUSIONS:
We found that an open revascularization approach in patients with DFUs was associated with an increased risk of major amputation, despite similar amputation-free survival rates compared to endovascular revascularization. The lower MALE rate associated with open revascularization was primarily driven by the reduced need for reintervention.
Advancing the Use of Patient Reported Outcomes and Patient Preferences
Digital Conference Pass
Moderator
Sarah Daugherty, PhD MPH
Carelon Research, Wilmington , DE, USA
Sarah Daugherty, PhD MPH, is a Principal Scientist at Carelon Research with expertise in epidemiology, health outcomes research, and patient-centered research. Dr. Daugherty provides senior leadership and oversight for patient-centered research studies using qualitative and quantitative methods and stakeholder engagement. Prior to joining Carelon Research, she led prospective and retrospective studies in the Division of Cancer Epidemiology and Genetics at the National Cancer Institute, oversaw comparative effectiveness research at the Patient-Centered Outcomes Research Institute (PCORI), and served as a Principal Consultant at Optum Genomics, United Health Group.
P24: Real-World Impact of Treated Hereditary Angioedema Attacks on Patients’ Employment and Work Productivity
5:45PM - 6:00PM
Craig TJ1, Busse P2, Christiansen S3, O'Connor M4, Radojicic C5, Ulloa J6, Danese S6, Andriotti T7, Audhya P7, Desai V7
1The Pennsylvania State University, Hershey, PA, USA, 2The Mount Sinai Hospital, New York, NY, USA, 3University of California San Diego, La Jolla, CA, USA, 44Allergy, Asthma, & Immunology Relief of Charlotte, Charlotte, NC, USA, 5Duke University School of Medicine, Cary, NC, USA, 6Outcomes Insights, Agoura Hills, CA, USA, 7KalVista Pharmaceuticals, Inc., Cambridge, MA, USA
OBJECTIVES: Hereditary angioedema (HAE) is a rare genetic disease associated with unpredictable attacks of tissue swelling. We examined the impact of the patients’ last treated attack on their ability to work and whether this was diminished among those having attacks while receiving long-term prophylaxis (LTP).
METHODS: Patients with type I/II HAE completed an online survey. Participants ≥12yrs old treated ≥1 HAE attack with an on-demand (OD) therapy in the prior 3 months. The Work Productivity and Activity Impairment Questionnaire: General Health assessed the impact of the last treated attack on participants’ ability to work during 7 days following attack onset.
RESULTS: Respondents included 80 adults and 14 adolescents, of which 42 patients self-reported as employed at the time of their last treated attack. Of those, 24 (57%) managed HAE with OD only, while 18 (43%) were receiving LTP. Sixty-seven (72%) rated their attack severity as moderate to very severe (72% OD; 71% LTP). Median (interquartile range) time from attack recognition to OD treatment was 2hrs (1-4hrs). Twenty (48%) patients were moderately to completely unable to do their job due to their last attack (46% OD; 50% LTP). Average impairment for overall ability to work was 39% (36% OD; 43% LTP), and the average absenteeism was 15% (13% OD; 17% LTP). Forty patients worked ≥1hr in the 7 days following the attack. Of those, 14 patients (35%) indicated that their last treated attack modestly to severely impacted their productivity (35% OD; 41% LTP); mean impairment (presenteeism) at work was 35% (33% OD; 37% LTP).
CONCLUSIONS: Despite treatment with OD therapy, HAE attacks impacted the work lives of employed patients resulting in impairments in their ability to work, substantial absenteeism, reduced productivity, and presenteeism among those who were able to work. The impact was similar among those managed with OD treatment only and those receiving LTP.
P21: A Framework and Set of Tools for Graphical Comparison of the Sensitivity of Different Instruments for Health Utilities and Other Preference Based Values
5:00PM - 5:15PM
Estévez-Carrillo A1, Jiang R2, Pickard AS3, Rand K4
1Maths in Health B.V., La Matanza de Acentejo, TF, Spain, 2Merck, Kenilworth, NJ, USA, 3Maths in Health B.V., Klimmen, Netherlands, 4Maths in Health B.V., Fjerdingby, 02, Norway
OBJECTIVES: The growing variety of Health-Related Quality of Life (HRQoL) instruments and value sets calls for methods and tools to assess which alternative is most sensitive to change in a particular target population. We introduce novel methodology and an R package 'valueSetCompare', for graphical and statistical comparison of the sensitivity of different instruments and different value sets and demonstrate its usefulness by comparing the US EQ-5D-5L, SF-6D, and HUI3 value sets using data from the Multi Instrument Comparison (MIC) study and Visual Analogue Scale as a severity proxy.
METHODS: The graphical comparison relies on cross-sectional data incorporating utility values from two or more different instruments, and an independent variable correlated with severity. A large number of simulated datasets are generated using resampling where the likelihood of sampling is a function of the severity proxy score. Mean scores and confidence bands along the severity for each instrument are presented in a ribbon plot, where the slope represents sensitivity. Discriminative ability is also investigated using the ratio of F-statistics estimated from analysis of variance (ANOVA).
RESULTS: Graphical comparison indicates that EQ-5D-5L and HUI3 had greater sensitivity to severity changes than SF-6D. No statistically significant differences were observed using the F-statistic approach. Theoretical comparison revealed that EQ-5D-5L had the widest scale range (1.57) compared to HUI3 (1.35) and SF-6D (0.98). In EQ-5D-5L, 20% of health states were rated below 0, compared to none in SF-6D and 77.5% in HUI3.
CONCLUSIONS: The novel framework and the ‘valueSetCompare’ R package allow for intuitive graphical representation of the sensitivity of different instruments, as well as standardized statistical tests. Comparison of sensitivity could be used to determine which instrument is best suited to capture treatment effects in populations of interest.
P23: Validity of the EQ-HWB-S in United States-Based Caregivers
5:30PM - 5:45PM
Chamoun Nasser S1, Kuharic M2, Pickard AS2
1Lebanese American University, Byblos, BA, Lebanon, 2Department of Pharmacy Systems, Outcomes and Policy, University of Illinois Chicago College of Pharmacy, Chicago, IL, USA
OBJECTIVES: EQ-Health and Wellbeing short form (EQ-HWB) is designed to evaluate the impact of health and social care interventions not only in patients but also caregivers. The short form version (EQ-HWB-S) provides a system for value sets to facilitate quality-adjusted life-years. While its validity is predominantly established in patient populations, this study aims to assess the EQ-HWB-S's validity in caregivers, specifically examining its convergent validity in relation to the EQ-5D-5L, CarerQoL, and ASCOTCarer.
METHODS: A secondary analysis of cross-sectional data collected online via the Qualtrics platform in the US between August 2022-March 2023, focused on caregivers 18 years or older providing unpaid care at least one hour of caregiving per week. Convergent validity of the four instruments was examined using Spearman rank-order correlation. Correlations were interpreted as strong (rs>0.50), moderate (rs=0.31-0.50), weak (rs=0.11-0.30), or trivial/none (rs<0.10).
RESULTS: The study included 504 caregivers, with the most common relationships being with spouses/partners (34.5%) and parents (29.8%). Analysis revealed strong correlations between EQ-HWB-S Mobility and EQ-5D Mobility (rs=0.57); EQ-HWB-S Daily activities and EQ-5D Usual activities (rs=0.55). EQ-HWB-S Anxiety exhibited strong correlation with EQ-5D Anxiety/Depression (rs=0.66) and moderate with CarerQol Mental Health (rs=0.48); while EQ-HWB-S Sadness/Depression exhibited strong correlation with EQ-5D Anxiety/Depression (rs=0.69) and CarerQol Mental Health (rs=0.55). EQ-HWB-S Control was strongly correlated with two ASCOTCarer, Spent time (rs=0.51) and Control (rs=0.51). EQ-HWB-S Loneliness had strong correlated with EQ-5D Anxiety/Depression (rs=0.57) and moderately with both CarerQol Mental Health (rs=0.44) and ASCOTCarer Spent time (rs=0.44). EQ-HWB-S Pain had a strong correlation with EQ-5D Pain/Discomfort (rs=0.68), and a moderate with CarerQoL Physical Health (rs=0.38).
CONCLUSIONS: Support for the construct validity of the EQ-HWB-S in US caregivers was evidenced by moderate to strong correlations across key related dimensions with established measures of health and well-being that span a broad range of constructs relevant to caregivers.
P22: Measuring Health-Related Quality of Life during Pregnancy: Exploring the Psychometric Properties of PROMIS to Assess Health-Related Quality of Life
5:15PM - 5:30PM
Raad J, Hanmer J
University of Pittsburgh, Pittsburgh, PA, USA
OBJECTIVES: Pregnancy is associated with significant changes in health-related quality of life (HRQoL). However, prior research has not assessed HRQoL using the Patient-reported Outcome Measurement Information System (PROMIS). This study describes HRQoL from months five to post-partum and provides evidence of convergent validity of PROMIS item banks with the Health Utilities Index (HUI) and EuroQol-5D (EQ-5D) measures.
METHODS: Participants were community adults with a pregnancy of at least 20 weeks of gestation at the time of enrollment. Participants completed the EQ5D, HUI, and seven PROMIS short forms (i.e., the four-item Depression, Anxiety, Physical Function, Fatigue, Sleep Disturbance, Ability to Participate in Social Roles, and Pain Interference scales). Participants also provided their age, race, ethnicity, and week of gestation. Surveys were repeated monthly. Descriptive statistics and bivariate correlations were used to evaluate scale properties.
RESULTS: 135 participants completed up to four monthly assessments, with 61 participants completing a post-partum assessment, resulting in a total of 458 assessments. Descriptive analysis of PROMIS items banks demonstrates important changes over time with decrements in PROMIS mean scores for Physical Function from months five (49.8) to nine (43.0); Pain Interference at months five (48.8) to nine (55.1) and Sleep Disturbance at months five (50.4) to nine (54.7) demonstrating the greatest change over time. Bivariate correlations between PROMIS, HUI, and EQ5D depression (r=.59, r=.70), PROMIS, HUI, and EQ5D Pain Interference (r=.84, r=.85), PROMIS Anxiety, HUI Emotion, and EQ5D Anxiety (r=.61, r=.70); and PROMIS Physical Function, HUI Ambulation, and EQ5D Usual Activities (r=.61, r=-.74) were significantly correlated with moderate to strong linear relationships.
CONCLUSIONS: This study is the first to demonstrate patterns of change during pregnancy using PROMIS items banks as well as evidence demonstrating the convergent validity between PROMIS, HUI, and EQ5D measures of HRQoL in pregnancy.
Breakouts: IP, WS, & OBS
Global Harmonization for Patient Focused Drug Development (PFDD): What Are the Arguable Challenges for Pragmatic Guideline Uptake and Implementation and How Should We Negotiate and Navigate Alignment?
Digital Conference Pass
Level: Intermediate
ISSUE:
There is growing consensus that patient experience data (PED) and patient engagement (PE) is important to consider by regulatory authorities during drug development. However, there are a multitude of guidelines and frameworks published and emerging for patient focused drug development (PFDD) and ongoing challenges for harmonization. Further, an initial response to the guidelines by regulated industry challenges the clarity on how and/or when PE and PED collected during drug development will be considered by regulators as sufficiently robust for the purpose of benefit-risk decision making and/or inclusion in the regulatory documents and product information.
OVERVIEW:
Rebecca will open discussion with the imperative, benefits and state-of-the-art of PFDD. The panel will present their multi-stakeholder perspectives on the importance to ensure that the chief complaints of patients are explicitly factored into drug development especially for outcome measures and endpoints used in a clinical trial to establish safety and efficacy of a medical product. Robyn will examine some tangible examples of PE and collecting PED in product development. Challenges and barriers for PE and collecting PED during drug development such as “Time constraints”, “Lack of budgetary commitment”, “Challenges in finding/engaging patients to collect patient centric data” and “Lack of expertise and resources” will be argued by the Yvette and Rebecca. Robyn will present perspectives from regulatory authorities for
opportunities for a
globally harmonized approach to PFDD
that is methodologically sound and sustainable and for successful partnerships with regulated industry to support PFDD. Yvette will highlight the key role of the PFDD message being driven by regulatory bodies to mitigate skepticism and provoke the knock-on impacts for value demonstration from a HTA/payer evidence needs perspective through to implementation science and to long-term overall improvement of standards of healthcare and health outcomes.
An interactive discussion (panelists, moderator, audience) for questions and comments will follow.
Moderators
Elise Berliner, PhD
Oracle Life Sciences, Kansas City, MO, USA
Panelists
Robyn Bent, MS
Center for Drug Evaluation and Research Patient Focused Drug Development, FDA, Silver Spring, MD, USA
Robyn Bent is the director of the Patient-Focused Drug Development (PFDD) Program in the Center for Drug Evaluation and Research (CDER). PFDD is an effort to systematically obtain patient input and facilitate the incorporation of meaningful patient input into drug development and regulatory decision making.
Prior to joining FDA, Robyn held several positions at the National Institutes of Health. Robyn has a background in pediatric oncology nursing and extensive experience in clinical trial design, conduct, and oversight. Robyn earned her Bachelor of Science in Nursing from The Catholic University of America and her Master of Science degree from the George Washington University.
Rebecca Edelmayer, PhD
The Alzheimer’s Association®, Chicago, IL, USA
As senior director, scientific engagement for the Alzheimer’s Association®, Dr. Edelmayer leads
efforts to accelerate the organization’s scientific agenda through the creation and delivery of
ongoing research education. She engages with chapters across the country, ensuring that
communities are informed of the global progress of dementia science and the organization’s
crucial role in advancing research to improve the lives of those facing Alzheimer’s and all other
dementia.
Dr. Edelmayer provides leadership on several of the Association’s national and international
initiatives in dementia science uniting researchers and clinicians on topics related to biomarker
testing, drug discovery and therapeutic development. Dr. Edelmayer is a study investigator for
the Association’s Alzheimer’s Network for Treatment and Diagnostics (ALZ-NET), an initiative to
gain insight on novel FDA-approved Alzheimer’s therapies. She also serves on steering
committees and as an external advisory board member for various cutting-edge scientific
consortia established by the National Institute on Aging to advance treatment development,
including the Accelerating Medicines Partnership-Alzheimer’s Disease, MODEL-AD and
TREAT-AD.
Dr. Edelmayer has more than 20 years of experience as a scientist and educator, spending
more than six years as a pharmacologist in the Neuroscience and Immunology Divisions at
Abbott and AbbVie. She has lectured, published and led collaborations in areas of
neurodegenerative disease, neurophysiology, and pain neurobiology. She completed her Ph.D.
and postdoctoral training in medical pharmacology at the University of Arizona College of
Medicine. Dr. Edelmayer holds a bachelor’s degree in neuroscience from the University of
Pittsburgh, where she also completed a National Institute of Mental Health research fellowship.
T. Joseph Mattingly, PharmD, MBA, PhD
University of Utah College of Pharmacy, Salt Lake City, UT, USA
Dr. Joey Mattingly has been in pharmacy for over twenty years, with pharmacy operations experience as an entry-level technician, staff pharmacist, pharmacy manager, district manager for a major pharmacy chain, and private equity start-up director for a long-term care pharmacy operation serving multiple states. Dr. Mattingly left the private sector in 2014 to pursue an academic career as an Assistant Professor at the University of Maryland School of Pharmacy while simultaneously earning his Doctor of Philosophy in Health Services Research with a specific focus on pharmacoeconomics.
Since 2014, Dr. Mattingly has developed a research portfolio that primarily focuses on drug pricing policy where he has engaged policymakers at local, state, and federal levels, including serving as Speaker of the House of Delegates and Trustee for the American Pharmacists Association (APhA) from 2019-2021. From November 2022 to April 2023, Dr. Mattingly served as an advisor to the Center of Medicare and Medicaid Services (CMS) to aid in the implementation of the new “Drug Price Negotiation Program” authorized by Congress through the Inflation Reduction Act. Most recently, he was named a Research Fellow for the National Association of Insurance Commissioners (NAIC) to help inform policy on pharmacy benefit manager practices.
In addition to drug pricing, Dr. Mattingly has led or co-led multiple federal research proposals aimed at improving patient outcomes through a health equities research lens (U01FD007563 and U01MD017437). Additionally, he led a subaward to a large cooperative agreement with the Food & Drug Administration (U01FD005946) focused on improving diversity in patient engagement activities. This experience with health disparities research was intended to better understand policy implications that may exacerbate disparities in multiple priority populations to ensure our cost-effectiveness methods incorporate health equity impacts.
Dr. Mattingly has previously served as an advisor on Effectiveness and Decision Science for the Patient-Centered Outcomes Research Institute, the Maryland Department of Health Prescription Drug Monitoring Program, and the Maryland Prescription Drug Affordability Stakeholder Council. He also serves as an Editorial Advisory Board member for the American Journal of Pharmaceutical Education and PharmacoEconomics - Open.
VBAs and Warranties in an Uncertain CMS World: Opportunities or Challenges?
Digital Conference Pass
Level: Intermediate
ISSUE: CMS implementation of IRA will likely challenge pharmaceutical developers to re-assess pipeline priorities and commercialization strategies, potentially impacting development of future medicines, as well as go-to-market tactics. As a byproduct of IRA, CMS is simultaneously developing a pilot to re-imagine value frameworks through its Cell and Gene Therapy Access Model. With the goal to launch in 2026, the CMS CGT Access Model will explore payment and performance models as it will begin negotiation on behalf of states, including a role for CMS as centralized outcomes monitor for therapies utilized through the pilot. Further spillover into commercial payers will increase market uncertainty.
OVERVIEW: This panel will discuss the impact IRA and CMS pilots might have on value-based agreements (VBAs), as well as emerging performance frameworks in the face of rising public and private payer demands for stronger recourse if therapies are not effective. Attendees will be challenged to reimagine how value can be quantified and defended amidst a changing landscape, specifically using drug warranties aimed at true end payers and next generation VBAs. Panelist will discuss trends with innovative access agreements in the oncology space as drug developers seek to distinguish their drugs with providers and patients. Furthermore the role of evolving value frameworks for CGTs and how they can be applied to support VBAs for next-generation products will be discussed. Finally the role drug warranties can play to mitigate uncertainty for cell, gene and even specialty therapies will be reviewed. Overall how could VBAs and Warranty programs be applied to assets in response to payer pressure in the form of IRA and the CMS CGT Access Model will be debated. Attendees will learn about these and other trends and ways HEOR and market access teams can assist their organizations adapt and respond to the implications of the IRA.
Moderators
Ambarish J. Ambegaonkar, PhD
APPERTURE LLC, Marlboro, NJ, USA
Ambi is an accomplished healthcare executive, passionate and focused on delivering Value. Over his 25+ year career, he has excelled in multiple functions of HEOR, Market Access, & Marketing across several therapeutic areas. He led the development of Pricing, Contracting and Value communication strategies for Pfizer’s signature brands. Having worked as a Payor (WV Medicaid), his unique insight into Global Payor needs guides successful generation and communication of HEOR evidence. APPERTURE was founded by him with a mission to be Focused on Amplifying Value in Healthcare.
Panelists
Lou Garrison, PhD
CHOICE Institute, School of Pharmacy, University of Washington, Seattle, WA, USA
Lou Garrison, PhD, is Professor Emeritus in The Comparative Health Outcomes, Policy, and Economics Institute in the School of Pharmacy at the University of Washington, where he joined the faculty in 2004.
Dr. Garrison has worked in non-profit, industry, and academic settings. He received a PhD in Economics from Stanford University and has more than 200 publications in peer-reviewed journals.
Dr. Garrison was elected as ISPOR President for 2016-2017, and currently serves as co-chair of ISPOR’s Policy Outlook Committee for the Health Science Policy Council. In September 2022, he was announced as the recipient of the 2022 Avedis Donabedian Outcomes Research Lifetime Achievement Award from ISPOR.
Emad Samad, BA
Octaviant Financial, Inc, New Brunswick, NJ, USA
Mr. Samad is a co-founder of Octaviant Financial and currently serves as the firm’s President & Chief Executive Officer.
In 2010, Mr. Samad founded Octaviant LLC, a merchant bank and specialized holding company dedicated to the life sciences and healthcare. Mr. Samad focused his practice at Octaviant LLC on strategic and financial advisory, earning the trust of the c-suite executives he supported and their investors.
During his tenure as an investment banker, Mr. Samad worked extensively on business transactions for life science and medical device companies, focused on structuring private and public equity financings and M&A transactions. Mr. Samad has advised immunotherapy, infectious disease, biodefense, gene and cell therapy, and regenerative medicine companies.
It was during his work as an advisor to gene therapy companies that Mr. Samad began to focus on the market access challenges facing gene and cell therapies, primarily, their affordability, their potential lack of durability, and the transferability of payments between different payers as patients move from plan to plan.
A consensus builder, Mr. Samad began to work with an ever-growing coalition of like-minded, mission-driven people and firms in the healthcare, finance and insurance sectors to build on the ideas that led to the formation of Octaviant Financial. Together, they look toward the finance and commercial insurance industries for ways to reimagine healthcare delivery with the goal to make transformational therapies accessible to patients and their plan sponsors, while providing new avenues for drug manufacturers to commercialize breakthrough innovation.
Mr. Samad is a regular commentator on topics concerning healthcare finance and biotech, and has been published in Bloomberg, STAT News, and ABC News, and peer reviewed journals including Nature, and the Journal of Biosecurity and Bioterrorism.
Gergana Zlateva, BA, MPA, PhD
Pfizer Inc., New York, NY, USA
Gergana Zlateva is VP, Global Access Strategy & Pricing for the Oncology Division at Pfizer. Gergana is proud to have helped increase patient access to medicines across the globe throughout her 20-year career with Pfizer and to have worked across multiple therapeutic areas. Gergana holds a PhD in Economics from Fordham University, NY and a BA and MPA from Southern Illinois University, IL. Gergana’s CV includes more than 60 peer-reviewed manuscripts.
Case Studies
Oncology and Rare Disease
Moderator
Sorrel Wolowacz, PhD
RTI Health Solutions, Manchester, United Kingdom
Sorrel Wolowacz, PhD, is Head of European Health Economics at RTI-HS, with 22 years of experience in health economics research and consulting. Her research focuses primarily on economic modelling, health utility estimation, observational studies, and health technology appraisal submissions. Dr. Wolowacz is a member of the editorial board for the Journal of Comparative Effectiveness Research and was co-chair of the ISPOR Good Research Practices Task Force addressing Measurement of Health State Utility Values for Economic Models in Clinical Studies and is a member of the ISPOR Oncology Special Interest Group.
CS5: Patient Voice of Autosomal Recessive Polycystic Kidney Disease through an Externally-Led Patient-Focused Drug Development Meeting
5:20PM - 5:40PM
Hoover E1, Bauer L2, Dell K3, Guay-Woodford L4, Hartung E4, Liebau M5, Oberdhan D6, Palaty C7, Valentine J2, Vanasco W8, Fedeles S8
1PKD Foundation, Kansas City, MO, USA, 2Hyman, Phelps & McNamara, Washington, DC, USA, 3Cleveland Clinic, Cleveland, OH, USA, 4Children's Hospital of Philadelphia, Philadelphia, PA, USA, 5University of Cologne, Cologne, Germany, 6Otsuka Pharmaceutical Development & Commercialization, Inc., Alexandria, VA, USA, 7Metaphase Health Research Consulting, Vancouver, BC, Canada, 8Critical Path Institute, Tucson, AZ, USA
Problem Statement: Patient-focused drug development is key to creating disease-specific therapies that target outcomes important to patients and their families. An estimated 1,500 children and young adults in the United States are living with autosomal recessive polycystic kidney disease (ARPKD), a rare genetic disease diagnosed
in utero or early childhood with considerable variability in disease manifestations and severity. Treatment at the few existing clinical expert centers is symptomatic and focused on kidney replacement therapy which comes with considerable burden for patients and their families. Insights to the patient and caregiver lived experience, preferences, and unmet needs are needed to inform medical research.
Description: A virtual Externally Led-Patient Focused Drug Development (EL-PFDD) meeting was convened for ARPKD with participants from the patient community, industry, and regulators. The virtual meeting format allowed many ARPKD community members to participate through live online polls, telephone call-ins, and by providing written comments through an online portal.
Lessons Learned: An EL-PFDD meeting is an excellent way to engage rare disease patient populations and expert clinicians to highlight patient and family caregiver experience with the disease, current standard of care, and unmet needs.
Stakeholder Perspective: The meeting produced insights into the disease journeys of ARPKD. Reported health concerns included high blood pressure (experienced by 80%), fatigue (65%), gastrointestinal issues (60%), growth failure/small stature (55%), enlarged spleen (55%), anxiety/depression (45%), kidney failure (45%), and liver problems (45%). An ideal treatment for ARPKD should: prevent kidney disease progression (95%), prevent liver disease progression (81%), and delay time to transplant or avoid dialysis (62%). Additionally, a facilitated discussion gave participants an opportunity to share their lived experiences on these topics and provide a rich set of qualitative information to contextualize ARPKD and its unmet medical need.
CS4: The Psychometric Properties of the EQ-5D-5L Among Ethiopian Cervical Cancer Patients: A Longitudinal Study
5:00PM - 5:20PM
Gebremariam G
Addis Ababa University, Addis Ababa, Ethiopia
Problem Statement: The responsiveness and minimal clinically important difference (MCID) of the instrument in patients with cervical cancer in Ethiopia has not been evaluated. This study aimed to evaluate the validity, reliability, responsiveness, MCID, and minimal detectable change (MDC) among patients with cervical cancer patients in Ethiopia.
Description: A longitudinal study was conducted from March 2022 to July 2023 among 422 patients with cervical cancer. Test re-test reliability of EQ-5D-5L dimensions by Gwet’s AC2 while the index and EQ-VAS scores were calculated by intra-class correlation coefficients (ICC). Convergent validity with the EORTC QLQ-C30 was estimated using the Spearman correlation coefficient. Kruskal Wallis test was used to assess its discriminate ability of varied levels of patient health status among patient subgroups. The anchor-based, distribution-based, and instrument-based methods were used to calculate the MCID estimates. The MCID estimates to MDC ratios were computed at the individual and group levels at a 95% CI. All statistical analysis was performed using R software.
Lessons Learned: Three hundred seventy-one patients with cervical cancer had completed the survey at baseline and follow-up with a median age of 50 (41, 56) years. The index value and EQ-VAS scores of EQ-5D-5L improved by 0.04 and 7.0 post-treatment compared with baseline respectively. The Gwet’s AC2 ranged from 0.73 to 0.97 for EQ-5D-5L dimensions while the ICC for index value and EQ-VAS were 0.71 and 0.79. The effect size ranged between -0.12 to 0.60 for the index value and -0.12 to 1.16 for the EQ-VAS scores. Moreover, the average (range) MCID value of the EQ-5D index score was 0.10-0.15.
Stakeholder Perspective: The EQ-5D-5L instrument was able to detect improvements and deteriorations in health and could discriminate between patients with different levels of health. The MCIDs estimated could be used at group-level health assessment however, health changes at the individual level needed to be considered in future research.
Breakouts: IP, WS, & OBS
How to Navigate the Digital Health Landscape: Global Value Frameworks and Payment Pathways
Digital Conference Pass
Level: Introductory
PURPOSE:
The objectives of this workshop are to introduce the digital health landscape, share real-world case studies, discuss challenges in value and access for digital innovations, and evaluate emerging assessment frameworks across US, UK and EU-4.
DESCRIPTION:
Digital health encompasses many innovations, most that are not funded by payers. Market access pathways are rapidly evolving in this space. This workshop will kick off with an interactive poll to assess audience familiarity with foundational concepts such as "digital medicines, digital diagnostics, and digital therapeutics". Discussant lead Betsy Lahue will provide definitions for the workshop. Next, John Hernandez from Google will share emerging regulatory and payment pathways for digital health applications in key global markets (15 minutes). Speaker Arthi Chandran from Abbott will narrow the topic and share a case study example on a remote monitoring technology (10 minutes). Discussant leader Betsy Lahue will ask the speakers to compare funding and HTA frameworks for digital innovation in North American versus European markets (15 mins). During this time, the audience will learn about key Digital Health stakeholders, such as ICER and NICE digital health guidance, the Digital Therapeutics Alliance, the Digital Medicine Society, and other authoritative groups. Next, the audience will be engaged in an interactive component to prioritize gaps in current digital value frameworks (15 minutes). The session will conclude with a walk-through of key resources available in multiple local languages for patients, clinicians, policy-makers and payers seeking to navigate the global digital health access landscape (5 mins). This session will cover value concepts and resources on emerging market access frameworks in digital health. HEOR professionals interested in understanding the current regulatory and policy issues relevant to digital health innovations will benefit from attending.
Discussion Leaders
Betsy J. Lahue, MPH
Alkemi, Manchester Center, VT, USA
Betsy Lahue leads Alkemi's HEOR and market access strategy practice. Ms. Lahue has over 25 years experience in evidence planning, outcomes endpoints and funding processes for rare disease therapies, digital innovations and other novel healthcare technologies. Her early career experiences included social and behavioral outcomes research at Boston University, coordinating grants and trials in academic medical centers and conducting program evaluation for the Massachusetts State Public Health Agency. Her prior industry roles include executive leadership and forming new functional capabilities to support health economics, outcomes research, reimbursement, and medical/regulatory strategy in both U.S. and international markets at Boston Scientific, Genzyme, Vertex and Becton Dickinson. Ms. Lahue earned her MPH at Boston University and her BA at Amherst College. She currently advises graduate programs, medtech and pharmaceutical companies. Ms. Lahue current serves on the board of EvoEndo.
Discussants
Arthi Chandran, MS, MPH, DPH
Abbott, Santa Clara, CA, USA
Dr. Arthi Chandran is the Divisional Vice President HEOR and Reimbursement leadership role at Abbott. She has been recognized for championing a cross-functional approach to innovation and market shaping with a focus on strategic evidence generation to enable broader adoption of health care technologies. She has successfully brought the practice of value integration into early stages of medical device product development, resulting in a portfolio of technologies with stronger value demonstration opportunities. Dr. Chandran began her HEOR career at Pfizer Inc. where she was dedicated to supporting both the pain and women’s health therapeutic areas. She has been an HEOR leader in both US and Global roles and has focused on supporting growth and demonstrating value through strategic health economics and outcomes research programs to highlight differentiation in highly generic markets.
Dr. Chandran is an active board member of 1000 Dreams Fund and serves on the board of the Healthcare Businesswomen’s Association’s Northern NJ Chapter. She earned a Masters in Public Health in Chronic Disease Epidemiology from Yale University, a Masters in Science in Quality Assurance and Regulatory Affairs from Temple University and a Doctorate in Health Policy and Management from the City University of New York.
John Hernandez, PhD
Google, Mountain View, CA, USA
Dr. John Hernandez is Head of Clinical Research, Health Economics and Outcomes Research at Google, which is leveraging information technology and artificial intelligence to assist in promoting healthy lifestyles, diagnosing cancer, preventing blindness and much more. He joined Alphabet in 2016 where he has built and led teams at Google and Verily Life Sciences leveraging artificial intelligence to conduct research and launch health products using digital platforms, smartphones, apps, wearables, connected biosensors and interoperable health data to help people lead healthier lives. Dr. Hernandez serves on the board of directors for ResMed and Carmat, which are publicly traded medical device companies. Prior to Alphabet, John worked for 15 years in executive roles at Boston Scientific and Abbott where he led global clinical research, health economics and outcomes research initiatives. John has served in research and consulting roles at the RAND Corporation, Quintiles (now IQVIA), PwC and the American Society of Internal Medicine (now ACP). John obtained his PhD and Masters in Health Policy at the RAND Graduate School in Santa Monica, CA and his BA from the University of North Carolina at Chapel Hill.
Prior Knowledge Is Power: Bayesian Methods to Optimize Trial Design in Clinical Development
Digital Conference Pass
Level: Introductory
PURPOSE: While Bayesian methods have grown in popularity in the last few decades, applications in clinical development have only recently gained momentum. Bayesian methods may increase trial efficiency by leveraging prior information such as historical or real-world data to reduce the number of enrolled study participants. This workshop will provide a gentle introduction to the principles of Bayesian statistics including Bayes’ theorem, prior distributions, likelihoods, and posterior distributions. Attendees will learn about current opportunities for use in clinical development with a focus on practical considerations when implementing these methods. Common methods, their advantages and limitations, and use cases will be highlighted.
DESCRIPTION: Workshop attendees will receive a gentle introduction to Bayesian principles and understand opportunities for application in clinical development. Mr. Parzynski will introduce Bayesian thinking and how it differs from traditional statistical methods. Focus will be placed on the core components of Bayesian methodology: Bayes’ theorem, prior distributions, likelihoods, and posterior distributions (~15 minutes). Dr. Clark will then introduce clinical development settings in which Bayesian applications have been considered including early drug/biological product development, non-inferiority designs, adaptive designs, rare diseases, and pediatric studies. These will be presented alongside current challenges to their use. (~15 minutes). Next, Dr. Zeldow will introduce commonly used methods for increasing power for clinical trials using historical or real-world data (~15 minutes). The session will end with Dr. Saranadasa walking through primary considerations when pursuing these approaches in clinical development, highlighting limitations and decision hurdles with a particular emphasis on power borrowing methods (~15 minutes).
Discussion Leaders
Craig Parzynski, MS
Genesis Research Group, Hoboken, NJ, USA
Craig Parzynski is Executive Director and Head of Biostatistics & Analytics at Genesis Research Group. He obtained his Masters of Science in Biostatistics from the University of Pittsburgh in 2011. He is a methodologist and biostatistician with expertise in real-world evidence, non-randomized studies, and causal inference.
Discussants
Jennifer Clark, PhD
U.S. Food and Drug Administration, Silver Spring, MD, USA
Jennifer Clark is a lead mathematical statistician at the Center for Drug Evaluation and Research at the FDA. She is the lead for the cardiology and nephrology statistical review team and has an interest in looking into understanding Bayesian studies and designs.
Madhawa Saranadasa, PhD
J & J Innovative Medicine, Bridgewater, NJ, USA
Bret Zeldow, PhD
Genesis Research Group, Waterville, ME, USA
Bret Zeldow has been a Principal Scientist of Biostatistics at Genesis Research Group since January 2023. He obtained a PhD in biostatistics from the University of Pennsylvania in 2017 and is an expert in Bayesian statistics, causal inference, and in the analysis of observational data.
Real-World Data Integration for Causal Inference: Benefits, Costs, and Case Studies
Digital Conference Pass
Level: Intermediate
PURPOSE: To guide participants in using integrated real-world data for causal inference, highlighting the advantages and challenges. Several case studies will illustrate practical applications and lessons learned. Participants will interactively share their experiences and participate in open discussion.
DESCRIPTION: Causal inference from observational data is growing in importance, driven by the need for generalizable and rapidly delivered real-world evidence (RWE) to inform regulatory, payer, and patient/provider decision-making. Integrating two or more individual sources of real-world data (e.g., administrative health claims, electronic medical records, registries, and publicly available social determinants of health data) can provide deeper insights into the patient's health journey. Using integrated data can lower methodological and resource barriers to comparative effectiveness and long-term safety assessments. However, integrating data often requires trade-offs regarding variable consistency, available sample size, and selection bias.
Workshop participants with intermediate-level subject knowledge of integrated data or causal inference will be familiarized with crucial considerations when using these data for causal study design. Dr. Grabner will initiate the workshop by discussing the basics of integrated data, causal inference, and their interplay and solicit participant feedback on their experiences using integrated data for causal inference. Afterward, Dr. Yu will present the first case study, which used an external control arm to enable causal inference in a study of patients with relapsed refractory multiple myeloma and resulted in regulatory approval of a new therapy. Next, Dr. Dixon will present the second case study, where directed acyclic graphs were developed to understand causal pathways for treating metastatic colorectal cancer and to illustrate their application to a large, integrated data set. Dr. Lloyd will conclude by providing an FDA case study on generating RWE to study COVID-19 vaccines in the
Biologics Effectiveness and Safety Initiative. There will be real-time polling and time for audience questions to support interactivity.
Discussion Leaders
Michael Grabner, PhD
Carelon Research, Wilmington, DE, USA
Dr. Grabner has fifteen years experience conducting HEOR using a variety of study designs, data sources, and statistical methods. As a Principal Scientist at Carelon Research, he is responsible for developing research solutions for internal and external clients, ensuring the scientific appropriateness and integrity of study goals, and for the dissemination of research findings. His research focuses on the comparative effectiveness and cost effectiveness of medical treatments with an emphasis in diabetes, autoimmune disease, and oncology.
Discussants
Ruth Dixon, PhD
Carelon Research, Athens, GA, USA
Ruth Wangia Dixon, Ph.D., is a senior researcher at Carelon Research, specializing in real-world evidence generation in oncology. Ruth harnesses the power of real-world data to address healthcare’s most complex challenges. She possesses notable expertise in health economics and outcomes, comparative/cost-effectiveness analysis, and causal inference research. In addition, Ruth leads the development, validation, and dissemination of novel causal inference research that leverage real-world healthcare data assets. Powered by her expansive experience in healthcare research, she is excited to serve as an insightful contributor during the 2024 ISPOR conference.
Patricia Lloyd, PhD, ScM
U.S. Food and Drug Administration, Silver Spring, MD, USA
Edward Yu, ScD
Bristol Myers Squibb, Summit, NJ, USA
How Artificial Intelligence Can Be Used to Develop Clinical Outcome Assessments?
Digital Conference Pass
Level: Introductory
PURPOSE: There is a need for the development of valid and reliable clinical outcome assessments to support earlier disease detection and better evaluation of patients’ symptoms, experience, and burden. With the support of emerging methods using Artificial Intelligence (AI), we are able to analyze large amounts of patient generated health data in order to develop these clinical outcome assessments. In this session, we will explore how Dr. Pirge, Dr. Kwon and Dr. Mckay are using AI in their field of research to develop predictive algorithms and reliable assessments of clinical outcomes and how it could improve precision of clinical trials and access to care. DESCRIPTION: Lysel Brignoli will open the workshop by talking about the need for the development of valid and reliable clinical outcome assessments and the issues the industry is facing when developing these assessments. Dr. Pirge will discuss how he accesses clinical notes via Natural Language Processing (NLP) and Large Language Models (LLM) to assess clinical outcomes. He will address how to best combine NLP and LLM models. He will describe examples that John Snow Labs has delivered in the areas of mental health and Oncology. Dr. Kwon and Dr. Mckay will describe an example that applied state-of-the-art deep learning approaches to score freezing of gait (FOG) in people with Parkinson’s disease (PD). Currently, FOG is a poorly understood heterogeneous gait disorder which is measured subjectively by movement disorders specialists or through patient completed questionnaires that are unsuitable for use in clinical trials. The discussants will facilitate a discussion with the audience about their specific needs for patient centered clinical outcome assessments, and also the expertise and resources needed to use AI in this field and the potential use in the future.
Discussion Leaders
Lysel Brignoli, MS
Oracle Life Sciences, Paris, 75, France
Lysel has over a decade of expertise in Real-World Evidence (RWE) and epidemiologic studies. Currently dedicated to designing and overseeing RWE studies, she also focuses on optimizing Oracle Life Sciences' RWE offerings, with a particular emphasis on registries and innovative approaches.
Her extensive background includes leading international non-interventional studies spanning diverse disease areas such as dermatology and rare diseases. Specializing in developing direct-to-stakeholders studies, Lysel possesses deep experience in incorporating Patient-Reported Outcomes (PROs) into research initiatives. Her proficiency extends to the publication of research findings in congresses and peer-reviewed journals.
Lysel holds a master’s degree in management and biotechnology.
Discussants
Hyeokhyen Kwon, Ph.D., M.Sc
Emory University, Decatur, GA, USA
Hyeokhyen Kwon, or Hyeok, is an Assistant Professor at Emory Biomedical Informatics. His research focuses on designing efficient, secure, and privacy-preserving machine learning systems using distributed on- and off-body sensors to tackle brain health challenges. His applications lie at the intersection of computer vision, speech analytics, ubiquitous computing, and human activity recognition. He has published in top-tiered journals and conferences, such as CVPR, ICCV, Ubicomp/ISWC, IMWUT, and Sensors. His Ph.D. research received a Samsung Ph.D. Fellowship and an Oracle for Research Award, and he was named a finalist in the Gaetano Borriello Outstanding Student Award at Ubicomp/ISWC 2021, as well as a finalist in Oracle for Research Fellow. During his PostDoc at Emory BMI, he also received the Oracle for Research Award.
J McKay, Ph.D., M.S.C.R.
Emory University, Atlanta, GA, USA
Dr. McKay is an Associate Professor at Emory University School of Medicine. He maintains one of the largest repositories of full-body behavioral testing data of movement disorders patients in the world. He has authored numerous peer-reviewed journal papers and conference papers, contributing significantly to the fields of neuroengineering, rehabilitation, and the basic understanding of Parkinson's disease. Dr. McKay's work emphasizes interdisciplinary collaboration and bridging the gap between engineering and clinical research in Parkinson's disease and related disorders.
Gursev Pirge, PhD
John Snow Labs, Lewes, DE, USA
Dr. Gursev Pirge, PhD, is a senior data scientist specializing in healthcare natural language processing (NLP). With extensive experience in training Named Entity Recognition (NER) and text classification models, he has made contributions to projects aimed at improving healthcare data analysis. At John Snow Labs, Dr. Pirge worked on the development of NER models in multiple languages using Spark NLP annotators. He also played a role in training text classification models, including patient email intention classifiers, supporting real-time email classification for healthcare professionals.
Additionally, Dr. Pirge served as a consultant and developer on several high-profile projects at John Snow Labs, collaborating with esteemed organizations.
Actually, Me Too Drugs Are Awesome: Multi-Stakeholder Perspectives on the Value of Me Too Drugs
Digital Conference Pass
Level: Introductory
PURPOSE: The Inflation Reduction Act has implications for drug development priorities and will likely result in fewer me too drugs. Some have argued that having fewer me too drugs is not a bad thing as similar treatments already exist for that condition and thus they only offer marginal health benefits. However, that perspective ignores the benefits that me too drugs have on the market and for patient access. This workshop will provide multi-stakeholder perspectives on the value of me too drugs that are not accounted for in conventional value assessment.
DESCRIPTION: Melanie Whittington will introduce the workshop by defining a me too drug, presenting both sides of the debate regarding the relative importance of me too drugs, and discussing if value assessors adapt their methods when evaluating a me too drug. (10 minutes) Peter Kolchinsky will provide the investor perspective by presenting a 15-year timeline of innovation in Hepatitis C, that started with low-efficacy interferons marketed in 2004 and led to multiple efficacious cures in 2018. (10 minutes) Joel Barrish will provide the manufacturer perspective on developing a me too drug for the treatment of Hepatitis C, focusing on the unique considerations and reasons to innovate in a class with an existing market leader. (10 minutes) Danny Dike will provide the payer perspective on negotiating practices used for drugs in a class with numerous therapeutic options and the ability for me too drugs to result in more options for patients. (10 minutes) Polling questions will be used before and after the panelists’ presentations to assess audience opinions on the value of me too drugs and if their opinions were swayed after hearing the multi-stakeholder perspectives. Twenty minutes will be reserved for audience questions. This workshop will be relevant for health economists, HTA bodies, manufacturers, payers, and patient advocacy organizations.
Discussion Leaders
Melanie Whittington, PhD, MS
Tufts Medical Center (CEVR), Boston, MA, USA
Dr. Melanie Whittington is a Senior Fellow at the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center and has a decade of experience in health economics and outcomes research. Most recently, she was the Director of Health Economics at the Institute for Clinical and Economic Review (ICER) where she led the cost-effectiveness analyses and budget impact analyses within ICER reviews. She was the 2022 recipient of the Bernie J. O’Brien New Investigator Award.
Discussants
Joel Barrish, PhD
Jnana Therapeutics, Boston, MA, USA
Joel was co-founder, President and Chief Scientific Officer at Jnana Therapeutics, a platform company focused on unlocking hard-to-drug targets. Joel’s first role in Biotech was as Chief Scientific Officer at Achillion Pharmaceuticals, where he led discovery efforts on Factor D inhibitors for the treatment of Paroxysmal Nocturnal Hemoglobinuria and C3 Glomerulopathy. Joel is currently a Venture Partner at RA Ventures focusing on new company creation.
Danny Dike, Bachelor of Science, Management Information Systems
Express Scripts, St. Louis, MO, USA
Peter Kolchinsky, PhD
RA Capital Management, Boston, MA, USA
Peter Kolchinsky is Founder and Managing Partner at RA Capital, investing in companies developing drugs, devices, and diagnostics. He serves as a board member for various public and private companies. Peter served on the Board of Global Science and Technology for the National Academy of Sciences and is the author of “The Great American Drug Deal” and “The Entrepreneur’s Guide to a Biotech Startup.” He founded the nonprofit No Patient Left Behind to advocate for healthcare reform and promote innovation. Peter holds a BA from Cornell University and a PhD in Virology from Harvard University.
5:30 PM - 6:00 PM
Get to Know ISPOR's Science & Health Policy Leadership
Come by the ISPOR Booth to meet and talk with ISPOR's Science and Health Policy leadership. Learn more about ISPOR's science priorities and how to get involved in the scientific member groups.
6:00 PM - 7:00 PM
Welcome Reception
Join us for a lively Welcome Reception, a perfect kick-off to connect, unwind, and gear up for what's ahead! Sponsored by Komodo Health in Booth 519.
Tue May 7
7:00 AM - 9:00 AM
Coffee and Connect
Don’t miss the start to the day with the Plenary Session. Enjoy your morning coffee as you listen to dynamic presentations intended to inspire and empower. Provided by ISPOR.
7:00 AM - 5:00 PM
Registration Hours
8:30 AM - 9:45 AM
Plenary Session
Missing Link for HEOR: A Path Forward for HEOR Data Integration
Digital Conference Pass
The integration and linkage of real-world data allows assembly of the whole picture of how patients feel by supporting incorporation of patient-generated health data with clinical data from electronic medical records and study-specific case report forms, and payer information through linkage of pharmacy and other health insurance claims. Data linkage may even extend beyond health care facilitates to include information about school attendance and other community data. In addition to incorporating patients’ experience to understand medical product safety, tolerability and effectiveness, linkage to health systems facilitates long term follow-up of medical products like cell and gene therapies and can also be used to evaluate delayed safety and risks. However, the process of linkage and integration is far from straight forward. Privacy constraints limit what and how much data can be shared, and the linkage process itself involves many assumptions to determine accurate matches from often-incomplete personal identifier information. We are about to learn a lot more about how well these linkages work since the FDA has asked to receive information establishing the accuracy of any linkage before linked data can be used for regulatory submissions. Come join this lively discussion with seasoned experts about the vision, key issues and reasons for optimism.
Moderators
Nancy Dreyer, MPH, PhD, FISPE
Dreyer Strategies LLC; University of North Carolina at Chapel Hill; Picnic Health, Newton, MA, USA
Nancy Dreyer is Adjunct Professor of Epidemiology at the University of North Carolina at Chapel Hill, Chief Scientific Advisor to Picnic Health, and a Trustee of Brandeis University. Prior to her switch from full-time employment to consultancy, she served as Chief Scientific Officer at IQVIA Real World Solutions, Global Chief of Scientific Affairs at Outcome Sciences Inc and Quintiles, and CEO of Epidemiology Resources Inc. She maintains an active career, teaching for the International Society of Pharmacoepidemiology (ISPE), participating in the ISPOR RWE leadership team, and publishing, and is a Fellow of both ISPE and the Drug Information Association.
One of her primary interests is advancing global use of real-world evidence by medical product regulators, clinicians and payers. Her current interests also include the role of patient-generated health data as well as data linkage and integration.
Speakers
Niall Brennan, MPP
CDC, Atlanta, GA, USA
Senior Advisor for Data Modernization, Centers for Disease Control and Prevention (CDC). Niall Brennan is the Senior Advisor for Data Strategy to the CDC Director. In this role, he provides guidance to the CDC Director on the multi-billion dollar CDC Data Modernization Initiative (DMI), as well as working directly with CDC Centers and Offices to accelerate implementation of the DMI. Prior to coming to CDC, Brennan was Chief Analytics and Privacy Officer at Clarify Health, where he led Clarify’s analytics and informatics strategy. He joined Clarify following his role as president and CEO of the Health Care Cost Institute, from 2017–2022, and previously served as the Chief Data Officer at the Centers for Medicare & Medicaid, from 2010–2017, where he led the Obama administration’s healthcare transparency efforts, built an advanced analytics enterprise, and played a key role in implementing numerous Affordable Care Act provisions, including the Qualified Entity Program and the Physician Payments Sunshine Act. Brennan is a graduate of Georgetown University’s MPP program and has an undergraduate degree from University College Dublin. He is married to Myra and has two children, Niamh (22) and Eoghan (17), who he inflicted difficult-to-pronounce Irish names upon. He is proud to be a native of Ireland, an immigrant, and a naturalized US Citizen.
Joe Franklin, JD, PhD
Verily, Washington, DC, USA
Joe Franklin leads the Strategic Affairs team at Verily and focuses on evidence generation strategy, including regulatory policy and health data privacy, at the intersection of clinical research and care. Before joining Verily in 2021, Joe held a variety of positions at FDA, including as senior advisor on data and evidence initiatives. Joe led the biosimilars policy staff in the Office of New Drugs and served as an attorney in the chief counsel’s office for multiple periods during his career at FDA. Joe has a PhD in cell biology from his early career as a bench scientist.
Keri Monda, PhD
Amgen Inc, Thousand Oaks, CA, USA
Keri Monda, PhD, is an Executive Director of Observational Research, and Clinical Development Epidemiology Head of the Data & Analytics Center within the Center for Observational Research (CfOR) at Amgen. In her role, she leads a team of epidemiologists and data scientists responsible for generating real-world evidence in support of programs from discovery through early and late development until launch, building and maintaining capabilities that make use of a large, integrated real-world data and analytics ecosystem. Prior to her time at Amgen, Keri was a genetic epidemiologist on faculty in the Department of Epidemiology at the University of North Carolina, Chapel Hill.
9:30 AM - 7:00 PM
Exhibit Hall Open
9:45 AM - 10:15 AM
Coffee and Connect
Head to the exhibit hall to refuel, recharge and connect with fellow attendees and exhibitors over a steaming cup of coffee. Provided by ISPOR.
10:15 AM - 11:15 AM
Breakouts: IP, WS, & OBS
Developing Preference-Based Health-Related Quality-of-Life Instruments for Young Children and Infants: What’s the Best Way to Proceed?
Digital Conference Pass
Level: Intermediate
ISSUE: The value of the universal quality-adjusted life year (QALY) has long been recognized, but cost-utility analysts face significant challenges in measuring and valuing utility in children. These include an inability to directly elicit utilities, reliance on proxies, disagreement regarding whether a child or adult perspective or preference is most relevant for valuing child health, and a lack of preference-based health-related quality of life (HRQoL) measures specifically for young children and infants. This international panel will examine and debate different approaches to measuring and valuing health in very young children and infants.
OVERVIEW: Scott Grosse will moderate and provide historical context. Tara Lavelle will share findings on using a visual analog scale to capture infant utility and how parents’ perspectives affected reporting. Kim Dalziel will present the comparative psychometric performance of the EQ-5D-Y, adapted from the adult EQ-5D, and the PedsQL in preschool children, and will describe work to develop the EQ-TIPs for infants. Wendy Ungar will discuss qualitative research identifying attributes relevant to infant HRQoL and alternative approaches. Those include restructuring and adding new attributes to existing adult tools (HUI -> HuPS), mapping non-preference-based but age-appropriate child HRQoL instruments to derive utility weights (PedsQL ->PedUtil), and developing child-centric preference-based tools (e.g., CHU-9D) and expanding their use to younger children. There is an inherent tension between conserving attributes and levels used in adult tools versus developing ones more reflective of infant or young child HRQoL. While the former enables easier pooling of data across pediatric and adult age groups for lifetime modeling, it may lack construct validity in young children. After the presentations, the moderator will pose questions to panelists on measurement and valuation challenges to spark debate and each speaker will be asked to suggest ways forward. The session will end with an audience Q&A.
Moderators
Scott D Grosse, PhD
Centers for Disease Control and Prevention, Atlanta, GA, USA
Scott Grosse, Ph.D., is senior health economist in the National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention (CDC), Atlanta, Georgia. He joined CDC in 1996 after completing doctoral studies in economics and public health at the University of Michigan. He conducts health services research, economic evaluations, and policy analyses on childhood-onset rare disorders and neurodevelopmental disorders and associated risk factors and public health policies such as newborn screening and genetic testing. He has published more than 250 peer-reviewed papers, including analyses of multiple healthcare databases.
Panelists
Kim Dalziel, PhD
The University of Melbourne, Carlton, VIC, Australia
Professor Kim Dalziel is Head of the Melbourne Health Economics Group at the University of Melbourne. She is also a Group Leader of Health Services and Economics within the Health Services Group at Murdoch Children's Research Institute (MCRI) and collaborates with the Royal Children's Hospital, The Women's Hospital and the Peter MacCallum Cancer Centre.
She leads a program of research in child health and has specialist expertise in economic evaluation and modelling, health-related quality of life measurement and valuation, child health policy, vulnerability/equity and use of health services and economic evaluation alongside clinical trials. She actively supervises Masters of Public Health, clinical advanced trainee research projects and PhD students.
Tara Lavelle, PhD
Tufts University School of Medicine and Tufts Medical Center, Boston, MA, USA
Tara Lavelle is an Assistant Professor at the Tufts Medical Center Institute for Clinical Research and Health Policy Studies and an Investigator at the Center for the Evaluation of Value and Risk in Health. Her research addresses a range of topics related to the cost and value of health care services delivered in the United States. Dr. Lavelle is particularly interested in advancing the use of comparative and cost-effectiveness research in vulnerable populations, including children. Previous work includes the development of novel methods for evaluating health and economic outcomes of childhood illness, and assessing preference-based quality of life outcomes in caregivers.
Dr. Lavelle earned her PhD in Health Policy with a concentration in Decision Science from Harvard University and was previously a postdoctoral research fellow in the Child Health Evaluation and Research Unit in the Division of General Pediatrics at the University of Michigan.
Wendy Ungar, MSc, PhD
The Hospital for Sick Children Research Institute, Toronto, ON, Canada
Wendy Ungar is a Senior Scientist, SickKids Research Institute, and Professor, University of Toronto. She holds the Canada Research Chair in Economic Evaluation and Technology Assessment in Child Health and directs Technology Assessment at Sick Kids (TASK) where she applies economic methods to child health. Her team maintains the PEDE database, a searchable online database of pediatric economic evaluations used by global HTA agencies Dr. Ungar chairs the Ontario Genetic Testing Advisory Committee which makes funding recommendations on emerging genetic testing technologies for Ontario, Canada. Her book, Economic Evaluation in Child Health was published by Oxford University Press in 2010.
The Market Access Conundrum in Digital Health: Balancing Rapid, Robust Validation With Integrated Evidence Plans
Digital Conference Pass
Level: Intermediate
ISSUE: The widespread adoption of digital health products (DHPs) is hindered by a lack of evidence supporting their clinical value, which is essential for their broad acceptance, commercial success, and improved patient care. However, while product developers have successfully pioneered new regulatory pathways, securing FDA acceptance, market access and the evidentiary needs of downstream decision-makers have been largely opaque. This results in early and substantial investments in evidence generation being overfitted to regulatory decision-makers, missing the opportunity for more efficient and effective studies that address the requirements of all decision-makers. Consequently, this increases the costs of evidence generation, delays broad adoption, frustrates potential buyers and end-users, and can even lead to commercial failures, as seen in some cases this year.
OVERVIEW: In the dynamic arena of digital health, a pivotal debate focuses on the optimal strategy for addressing the evidentiary needs of a diverse range of stakeholders and decision-makers — including payers, purchasers, clinicians, and patients — in the development and deployment of DHPs for broad acceptance. The proliferation of FDA-authorized DHPs has not resulted in broad patient access to their benefits nor commercial viability for many of their developers. The challenge lies in the fact that despite FDA authorization, many DHPs fail to achieve broad patient access or commercial viability. Regulatory acceptance, while necessary, is not sufficient for the success of DHP innovation. It does not guarantee that the benefits of healthcare digitization will impact health, healthcare, and health economics at a large scale. This panel will center around finding the balance with the right strategies, optimized commercial model, and case examples for finding fit-for-purpose strategies for making progress for broad access and acceptance of DHPs.
Moderators
Terri Kim, MBA, CHIE
Lunit, Inc., Atlanta, GA, USA
Terri is a breast cancer survivor dedicated to supporting Lunit’s mission to conquer cancer. Lunit is a medtech AI company with global leading technology in analyzing radiology and pathology images for cancer screening and treatment solutions. She is currently focused on developing and executing organic and inorganic growth opportunities for Lunit. Prior to joining Lunit, Terri was an executive at Anthem (now Elevance Health), one of the largest US commercial payers, focusing on Innovation. She has also worked for several years as a management consultant at McKinsey & Company where she worked on strategy, marketing, finance and operations projects for world leading institutions like the World Health Organization. Terri holds an MBA from Stanford University and a BS in Computer Science from KAIST.
Panelists
Jeff Gombala, BA
ZS, Suwanee, GA, USA
Jeff Gombala is Head of Digital Health Platforms and Products at ZS. His focus is shaping and bringing to market ZS’s suite of platform and products offerings to innovate the patient journey through analytics & insights, patient engagement, digital medicine & SaMD, and evidence generation. Jeff has over a decade of experience in supporting healthcare and life science companies in designing, developing, and deploying digital health solutions.
Benjamin Parcher, PharmD, MS
Boehringer Ingelheim Pharmaceuticals, Odessa, FL, USA
Benjamin Parcher, PharmD, MS is the Digital Product Access Lead for Boehringer Ingelheim’s CNS digital therapeutics in schizophrenia.
He has over 20 years of US and global experience in the healthcare industry across multiple therapeutic areas. In his current role, he is responsible for driving access strategy in Schizophrenia disease through payer-based, collaborative research and impactful value communications. He has also authored over a dozen studies in the digital space, including key initiatives with the Digital Therapeutic Alliance and the Academy of Managed Care Pharmacy. He holds a Doctor of Pharmacy from the University of California, San Francisco School of Pharmacy and a Master of Science in Applied Pharmacoeconomics from the University of Florida.
Benjamin currently lives in Tampa, Florida with his wife and son. In his free time, he enjoys deep sea fishing and spending time with his family.
Jordan Silberman, MD PhD
Elevance Health, Sunnyvale, CA, USA
Jordan Silberman, MD, PhD is a Director of Clinical Analytics and Research, Digital Care Delivery, Anthem, Inc. Jordan co-founded Anthem's Digital Health Evidence Committee. He leads scientific and statistical strategy for digital health initiatives including the Asthma Digital Study (tinyurl.com/5cfnkwxy), a randomized controlled trial. A behavioral scientist and experienced data analyst, Jordan has more than a decade of experience in digital health. Jordan earned an MD (honors in research), a PhD (psychology), and a certificate in statistics from the University of Rochester, as well as a master's degree in psychology from the University of Pennsylvania.
Cookbooks Versus Blenders: Paradigms for Integrating Factors Relevant to the CMS Drug Price Negotiation Process
Digital Conference Pass
Level: Intermediate
ISSUE: The first phase of Medicare drug price negotiation is under way, but how the Centers for Medicare and Medicaid Services (CMS) will put all the pieces of information together in its initial offer prices and subsequent negotiation remains a critical unknown for stakeholders and the academic community. Since CMS has suggested that they will evolve this process as they learn more and their capabilities grow, this panel will present key options for how the disparate types of information, including qualitative and quantitative elements, can be integrated and translated into the negotiation process, highlighting key pros and cons of different approaches, and exploring the role that external input can best support CMS in this effort.
OVERVIEW: Steve Pearson (Moderator) (10 minutes) will set the stage by outlining the elements that the inflation reduction act (IRA) sets out as relevant for determining initial price offers by CMS. Marina Richardson (12 minutes) will share elements of ICER’s submission to CMS, which included a comparative clinical and cost-effectiveness assessment of apixaban and rivaroxaban, both of which are on the initial negotiation list. Brett McQueen (12 minutes) will discuss how a structured deliberation could be used by CMS to aggregate and prioritize evidence in a way that acknowledges the relative importance of factors being considered in the determination of an initial offer price. Kyle Hvidsten (12 minutes) will react to both presentations and share the views and concerns of industry on how information should be integrated to guide CMS actions. The floor will then be open to interactive polling and Q&A with the audience, and discussions about how the research and policy community should focus their efforts to inform CMS processes.
Moderators
Steven Pearson, MSc, MD
Institute for Clinical and Economic Review, Boston, MA, USA
Steven D. Pearson, MD, MSc is the Founder and Special Advisor of the Institute for Clinical and Economic Review (ICER), an independent non-profit organization that evaluates the evidence on the value of medical tests, treatments, and delivery system innovations to encourage collaborative efforts to improve patient care and control costs. Dr. Pearson is also a Lecturer in the Department of Population Medicine at Harvard Medical School. He received his medical degree from UCSF, completed an internal medicine residency and research fellowship at Brigham and Women’s Hospital, and obtained a Master of Science Degree in Health Policy and Management at the Harvard School of Public Health.
Panelists
Kyle Hvidsten, MPH
Sanofi, Cambridge, MA, USA
Kyle Hvidsten is Head of the Global Specialty Care Health Economics and Value Assessment department. In this role, Kyle oversees input into clinical trial design and the development of economic models and RWE studies to support scientific exchanges with health technology assessment agencies and healthcare payer organizations across the globe. Prior to joining Sanofi, Kyle held several positions in US and Global health economics and outcomes research with increasing levels of responsibility over the past 20+ years. He holds a MPH degree from Columbia University and a BA from Emory University.
Robert McQueen, PhD
University of Colorado, Denver, CO, USA
R. Brett McQueen is an Associate Professor at the University of Colorado (CU) Skaggs School of Pharmacy and Pharmaceutical Sciences, and member in the Center for Pharmaceutical Outcomes Research. His research interests include decision-analytic modeling applications and methodology, applied microeconometrics in health, and novel value assessment methods. Brett has current funding in micro-costing health interventions, evaluating performance-based risk sharing agreements, estimating patient and payer preferences for various pharmaceuticals, and novel value assessment methods. He is the course director for “Pharmaceutical Economics and Policy Analysis” in the Pharmaceutical Outcomes Research PhD program at CU.
Marina Richardson, PhD
Institute for Clinical and Economic Review, Boston, MA, USA
Marina is an Associate Director, HTA Methods and Health Economics with the Institute for Clinical and Economic Review (ICER). Prior to joining ICER, Marina spent 7 years at the Canadian Agency for Drugs and Technologies in Health (CADTH) where she contributed to over 30 drug reimbursement review reports and recommendations. Marina has a PhD in Health Services Research from the University of Toronto and is an active member of the Health Technology Assessment (HTA) community including as a deputy editor for the International Journal of Technology Assessment in Health Care (IJTAHC).
Implementing Comprehensive, Integrated, Whole Person Care Models Using Real-World Data
Digital Conference Pass
Level: Intermediate
ISSUE:
The staggering rise in health care expenditure across many health systems without a commensurate improvement in health outcomes benefiting patients relative to cost has signaled a need for models that aim to deliver improved value for patient populations. Translating this concept into practice requires a substantial rethink around how the business in healthcare is done, starting with the way care model are built and how the data is collected and shared among stakeholders to ensure continual improvement and sustainability of such models. This panel will describe real world outcomes and cost data from applied projects implementing IPU-based models of care across 3 countries with the same purpose: to improve population health outcomes relative to costs for delivering outcomes. From the United States, a musculoskeletal IPU delivering comprehensive, integrated will be demonstrated, including the barriers, facilitators, opportunities, and achievements to date. In a similar approach, the Asthma project from Brazil will share with the audience how a step-by-step method is being followed to transform asthma care in the country, from a decentralized and volume-based to an integrated and data-driven services. Finally, the project in development in Portugal will be used to demonstrate how the technology is serving as an essential element to make feasible the implementation of IPU for bariatric surgery. OVERVIEW:
The panel will be started by the moderator and, in sequence, each one of the speakers will have 10 minutes to share their experiences from each one of the countries. The last 10 minutes will be dedicated to the discussion. It is expected that the audience leave the session motivated to start practical initiatives for different care pathways, understanding; which type of technology is required to make it happen; which stakeholders need to be involved, and how it is possible to follow a step-by-step method to make happen.
Moderators
Ana Paula Etges, PhD, Eng.
PEV Healthcare Consulting, Porto Alegre, RS, Brazil
Ana is passionate for inspiring people for scientific education and works for a more effective and sustainable healthcare system. In addition to her position as Partner at PEV Healthcare Consulting, she serves as Professor in the Graduate Program in Epidemiology at UFRGS and Insper and Senior Researcher at the National Institute for Health Technology Assessment (IATS) and at the Dream Big Health. Ana founded the TDABC in Healthcare Consortium and has performed dozens of TDABC and healthcare strategy management consulting projects in Brazil, Portugal, and USA. Ana is Ph.D in industrial engineer at UFRGS, in collaboration with Stanford Medicine, and completed her post-doctoral fellowship in Epidemiology at UFRGS in collaboration with the Brigham Women’s Hospital. She also attended to the VBHC Seminary at Harvard Business School, and attended to the Effective Writing Certification Program at Harvard Medical School (Class 2023).
Panelists
Anne Geubelle, MBA
Prologica, Lisboa, 11, Portugal
With more than 25 years of CFO and management experiences in multicultural and international technology environment, with a successful track record in acquisition and fusion operations, I decided in 2015 to use my experience in technology, health and management to create a new business unit at Prologica focused exclusively on developing solutions that allow the use of health data for real time clinical and management support decisions.
Today, Prologica’s core business is centered at Healthcare and with a strong and committed team with the same vision we unlock the power of data in hospitals and related healthcare organizations in order to accelerate the necessary digital transformation of the healthcare sector around the world! Since 2018, Value-Based Healthcare has also become part of my path, focus and expertise.
After get the VBHC certified Green Belt and successfully completed the program Train the Trainer – VBHC Green Belt Track, I was invited by Dr. Fred van Eenennaam and VBHC Center Europe to be a VBHC Center Europe Ambassador on the topic Data & IT.
As an Ambassador of VBHC, I am convinced that the adoption of VBHC methodologies at a larger scale is highly depending on the implementation of flexible and user-friendly information system designed to better evaluate patient outcomes, costs (TDABC), and, as a consequence, empower better decision making!
Prakash Jayakumar, MD PhD
Dell Medical School, The University of Texas Health Austin, Austin, TX, USA
Carisi Anne Polanczyk, ScD, MD
PEV Healthcare Consulting, Porto Alegre, Rio Grande do Sul, Brazil
She is an Associate Professor of Medicine at the Federal University of Rio Grande do Sul (UFRGS) and a senior researcher from the Brazilian National Agency (CNPq). Chief of the Cardiology Service at Hospital Moinhos de Vento, since 2013, and an attending physician at the Cardiology Service at Hospital de Clínicas de Porto Alegre. She is the coordinator of the Institute for Health Technology Assessment (IATS/CNPq) and dedicated the last decades to studies on health economics, technology assessment, and the use of real-world data, with an emphasis on cost-effectiveness, management, and incorporation of technology. Lately, Carisi has been working on VBHC implementation and research initiatives, focusing on applying ICHOM standard sets and TDABC methodology. She is a co-founder of PEV consulting firm conducting projects with a scientific framework to increase efficiency and value for the health system. Additional studies focus on coronary artery disease and perioperative care, with national and international collaboration. Also, a consultant to the Ministry of Health, a Brazilian HTA Network (REBRATS) member, and the Brazilian Society of Cardiology.
Case Studies
US Health Policy
Moderator
Laura Housman, MPH, MBA, DrPH(c)
Avalere, Washington, DC, USA
Laura T. Housman, DrPHc (ABD), MBA, MPH is Senior Vice President and Practice Director of Evidence and Strategy within Avalere Health, bringing broad and extensive expertise in strategy, evidence generation, reimbursement, patient engagement, population health and personalized medicine to drive access to innovation across healthcare. Laura received her Bachelor of Arts degree in Economics from the College of Arts and Sciences and a Minor in Business Administration from the School of Management from Boston University; her Master of Public Health from the Boston University School of Public Health; and her Master of Business Administration with honors from Boston University’s Questrom Graduate School of Management. Laura is currently ABD as a Candidate in the Doctor of Public Health (DrPH) program at Boston University School of Public Health.
CS7: Actuarial Approach to Evaluating the Stakeholder Impact from the Inflation Reduction Act
10:15AM - 10:35AM
George S1, Hayes M2
1Milliman, tampa, FL, USA, 2Milliman, Lutz, FL, USA
Problem Statement: Addressing the reimbursement differences between the existing and Inflation Reduction Act (IRA) is critically important for Part D stakeholders (patients, payers, pharmaceutical manufacturers, providers, CMS). Understanding how the drug prices, reimbursement, and the financing of the Part D benefit will influence pharmaceutical manufacturers pipeline development is important to policy makers and Part D stakeholders.
Description: Stakeholders have significant cost shifting between the Part D drug reimbursement methods (current vs IRA). Government negotiation of drug prices, Part D benefit redesign, and inflation rebate penalties influence the pharmaceutical industry decisions to bring pipeline drugs to the market. This session will use an actuarial Part D drug pricing model to assess stakeholder liabilities.
Lessons Learned: Actuarial process with modeling Part D stakeholder drug financial liabilities. Payment factors influencing drug pipeline development.
Stakeholder Perspective: Part D patients, plan sponsors, pharmaceuticals manufactures, CMS are addressed with the modeling.
CS8: Department of Veterans Affairs (VA) National Real-Time Medication Use Evaluation (MUE) on Lecanemab-Irmb
10:35AM - 10:55AM
Kima C1, Smith T2, Burk ML2, Shumunov A2, O’Donnell AJ3, Fuller MA4, Martin JL2, Cunningham F2
1Veterans Health Administration Center for Medication Safety (VA MedSAFE), Pharmacy Benefit Services, Pittsburgh, PA, USA, 2Veterans Health Administration Center for Medication Safety (VA MedSAFE), Pharmacy Benefit Services, Hines, IL, USA, 3VAPHS Geriatric Research Education and Clinical Center, Pittsburgh, PA, USA, 4Veterans Health Administration Center for Medication Safety (VA MedSAFE), Washington, DC, USA
Problem Statement: Lecanemab is an IgG1 monoclonal antibody, which targets soluble aggregated amyloid beta (aβ) and works by removing amyloid plaques in the brain. While lecanemab effectively removes beta-amyloid and has full FDA approval as of July 6, 2023, the clinical benefits remain uncertain. Adverse drug events, particularly amyloid-related imaging abnormalities with hemorrhage (ARIA-H) and edema (ARIA-E), present significant safety concerns for this medication.
Description: Nearly half a million US Veterans have Alzheimer’s disease, and the prevalence is expected to rise, particularly with the increasing incidence of traumatic brain injury and posttraumatic stress disorder. The objective of the real-time medication use evaluation (MUE) is to assess the safe and appropriate use of lecanemab in the Veterans Health Administration, confirm that patients meet inclusion and exclusion criteria as outlined in the VA Pharmacy Benefits Management (PBM) Lecanemab Criteria for Use (CFU), assess and characterize any adverse drug events (ADE) experienced during the course of treatment as well as the management of ADEs, and assess reasons for discontinuation. The MUE is designed to collect information on the safe and appropriate use of lecanemab in real-time during, immediately following, and between infusion appointments. Local VA pre-screening of patient-specific inclusion and exclusion criteria are conducted per the CFU, and these data are submitted to the VA Center for Medication Safety via a web-based data collection form. Lecanemab dosing, administration, MRI screening, and adverse event reporting and management are documented on the form at each treatment infusion visit.
Lessons Learned: This web-based real-time MUE tool will enable the VA to collect lecanemab safety and appropriateness of use data to optimize Veteran care, assist decision-makers on continued guidance on use of the agent in VA, and identify potential areas for future research.
Stakeholder Perspective: National government health care system.
CS9: The Impact of the Inflation Reduction Act on Early-Stage Biomedical Investment Decisions - An Illustrative Real-World Case Example
10:55AM - 11:15AM
Xie R1, Cameron T2, Kolchinsky P2
1RA Capital Management, Newton, MA, USA, 2RA Capital Management, Boston, MA, USA
Problem Statement: How early-stage biomedical R&D efforts will vary by reward size is one of the most debated topics in modeling the societal impacts of the Inflation Reduction Act (IRA). Existing studies typically relied on aggregate data to estimate the elasticity of innovation by examining the relationship between global revenues with the number of drugs launched/in development.
Since ultimately all medicines come to market from investors deciding to fund their development, a micro-level analysis of how investors make their decisions can inform the key assumptions for simulations and generate more realistic insights for policymaking.
Description: Using a real-world example of a marketed drug indicated for cardiovascular diseases (CD), we constructed a net present value (NPV) model to examine how the discounted value and investment decisions would vary in pre- and post-IRA scenarios. Data inputs on revenue and profit over time were derived from historical data. Key specifications included: a patented period of 14 years, a weighted average cost of capital of 11%, and a risk-adjusted internal rate of return of 12%. The post-IRA scenario assumed that US profits after year 9 after launch is zero in the base case. Sensitivity analyses were performed. Outcomes included NPV and investment decisions at six different R&D stages including preclinical, phases 1-3, filing, and launch.
Lessons Learned: IRA would lead to a 40% reduction in the NPV at the time of launch. The reduction was greater in earlier stages of development due to expected dilution and discounting, resulting in discontinuing this project at earlier decision points. Incorporating discounted profits into empirical analyses reflects real-world investment decision-making, and reveals a basis for potentially greater elasticity of innovation in response to government price-setting in existing literature.
Stakeholder Perspective: The analysis is conducted from the perspective of investors and can be useful to all stakeholders seeking to understand the overall impact of the IRA.
Spotlight Session
Harmonizing Real-World Data Networks: Bridging Regulatory, Patient, and Research Perspectives
Digital Conference Pass
In the rapidly evolving landscape of real-world evidence, the integration and harmonization of real-world data (RWD) networks have emerged as a pivotal frontier. This panel session delves into the intricate dynamics of merging RWD networks, with a focus on the collaborative efforts involving regulatory, patient and research organizations.
Moderators
Keith Marsolo, PhD
Duke University School of Medicine, Durham, NC, USA
Dr. Marsolo is a Professor in the Department of Population Health Sciences (DPHS) and a member of the Duke Clinical Research Institute (DCRI). His current research focuses on infrastructure to support the use of electronic health records (EHRs) and other real-world data sources in observational and comparative effectiveness research and public health surveillance, as well as standards and architectures for multi-center learning health systems. He is co-Principal Investigator of the Common Data Model service of the Coordinating Center for the National Patient-Centered Clinical Research Network (PCORnet), a co-lead of the Data Infrastructure core of the FDA-funded Sentinel Innovation Center, and co-lead of the electronic health record (EHR) core of the Coordinating Center of the NIH Pragmatic Clinical Trials Collaboratory. Dr. Marsolo received his PhD in Computer Science from The Ohio State University, with a dissertation on data mining, specifically the modeling and classification of biomedical data.
Panelists
Daniel Leaver Morales
EMA, Amsterdam, Netherlands
Dr Daniel Morales, is a senior clinical epidemiologist within the Data Analytics Taskforce at the European Medicines Agency (EMA). Until recently he was a European Commission appointed Independent Scientific Expert to the EMA Pharmacovigilance Risk Assessment Committee. Dr Morales background is in clinical general practice and academia.
Donna R Rivera, PharmD, MSc
US Food and Drug Administration, Washington, D.C., MD, USA
James Wiggins
Amazon Web Services, unknown, DC, USA
James leads AWS’ global solutions architect team for public sector healthcare (non-profit, academic, government) and the global healthcare technical community across all of AWS. This is a team composed of specialists in electronic health record systems, bioinformatics, genomics, health AI/ML, clinical and research medical imaging, mobile medical applications, open source research tools, and many other topics. James’ team supports providers, payors, researchers, and regulators as they advance healthcare around the world by helping them apply cloud technology. James provides guidance to healthcare executives and industry analysts to help them understand what healthcare services AWS and their partners offer, the capabilities of the global healthcare tech team, and how customers are succeeding using AWS. James began work with the OHDSI community in 2018 and won the OHDSI Titan Award for Community Support in 2019.
Podium Session
Market Access on Availability and Use of Drugs
Digital Conference Pass
Moderator
Soumana Chamoun Nasser, PharmD, MHEPE
Lebanese American University, Byblos, BA, Lebanon
Soumana Chamoun Nasser earned her BS degree in pharmacy from Massachusetts College of Pharmacy and Allied Sciences in 1998; her PharmD degree from Rhode Island University; and a Master in Health Economics & Pharmacoeconomics from University of Pompeu Fabra, Barcelona. She worked as pharmacist at New England Medical Center, then at Massachusetts General Hospital, in Boston. In 2003, she joined the School of pharmacy at the Lebanese American University in Lebanon. She is a Clinical Associate Professor, and former president of ISPOR Lebanon chapter. Her teaching and research are in the area of pharmacy practice and health economics. She is currently a visiting scholar at University of Illinois Chicago.
P27: Uptake and Costs of Rituximab Biosimilars Among the Medicare and Medicaid Populations in 2019-2021 in the United States
10:35AM - 10:55AM
Qian J
Auburn University Harrison College of Pharmacy, Auburn, AL, USA
OBJECTIVES: Rituximab (Rituxan®) is a monoclonal antibody medication used to treat certain autoimmune diseases and types of cancer. Three rituximab biosimilars have been approved by the U.S. FDA since 2018 but real word evaluations in their uptake and costs in nationally representative populations are limited. This exploratory study evaluated the uptake and costs of three biosimilars rituximab-abbs (Truxima®), rituximab-pvvr (Ruxience®), and rituximab-arrx (Riabni®) among the Medicare and Medicaid populations in 2019-2021.
METHODS: Using the annually aggregated, product-level (using brand and generic drug names, and Part B HCPCS codes) utilization and cost data of biologic and biosimilar rituximab products in 2019-2021 from the Centers for Medicare and Medicaid (CMS) drug spending data, total number of claims and costs for all four rituximab products were identified and extracted for Medicare Part B, Part D, and Medicaid reimbursement. Annual average cost per dosage unit (ACPDU) of individual rituximab products was also extracted and their annual growth rates in 2021 (vs. 2020) were calculated. Descriptive data analyses were performed using Microsoft Excel 2016.
RESULTS: After 3 years of entering the U.S. market, use of rituximab biosimilars increased from 0-7% in 2019 to 50%, 27%, and 48% of all rituximab claims paid by Medicare Part B, Part D, and Medicaid, respectively in 2021. Total cost for rituximab biosimilars also reached 41%, 21%, and 38% of all rituximab products paid by Medicare Parts B and D and Medicaid, respectively. ACPDU of biologic rituximab increased 2% in Medicare Part D in 2021 (vs. 2020) but decreased by 5% and 3% in Part B and Medicaid, respectively. ACPDU of rituximab biosimilars (rituximab-abbs and rituximab-pvvr) decreased by 2-17% in 2021 in different Medicare and Medicaid programs compared to 2020.
CONCLUSIONS: Significant uptake of rituximab biosimilars in Medicare and Medicaid programs occurred within the first 3 years of marketing in the U.S.
P28: Impact of FDA Expedited Review Programs on New Drug Approval Time
10:55AM - 11:15AM
Ronnebaum S1, Smith D2, Andrawes M2
1Evidera, Inc, Bethesda, MD, USA, 2Evidera, Inc, Waltham, MA, USA
OBJECTIVES: To examine the impact of FDA expedited review programs on new drug approval times and identify key factors associated with expedited approval.
METHODS: All New Drug and Biologic License Applications approved from January 2015 to December 2022 were analyzed. Review times associated with priority review and other expedited programs were compared to standard review with no expedited programs. The relative risk (RR) was calculated for comparisons of interest.
RESULTS: The 360 approvals included priority only (n=13%), priority and ≥1 expedited pathway (52%), standard only (n=30%), and standard and ≥1 expedited pathway (n=5%). Therapies under priority review had a shorter mean (median) review time of 9.4 (8.0) months compared to 18.6 (12.0) months for therapies under standard review only, equating to 9.2 (4.0) months saved. Individually, fast track, breakthrough, and accelerated approval designations had little impact on review time for therapies under priority review, but the combination of priority review, accelerated approval, and breakthrough therapy designations provided the shortest overall mean (median) time to approval: 6.9 (5.9) months. Furthermore, therapies were significantly more likely to be approved during the first review cycle if they underwent priority review (RR=2.44), breakthrough therapy designation (RR=3.43), or fast-track designation (RR=1.96), compared to therapies without the respective designation.
The likelihood of priority review increased for therapies with any expedited designation, with accelerated approval producing the largest impact (RR=32.18) and fast-track designation the smallest (RR=4.02). First-in-class therapies were also significantly more likely than non-first-in-class therapies to receive a priority review (RR=1.39), breakthrough therapy designation (RR=2.01), or fast-track designation (RR=1.34).
CONCLUSIONS: FDA review is shorter and more likely to result in first-round approval if an expedited program designation and/or priority review are applied. The combination of accelerated approval and breakthrough therapy designations was most likely to enable priority review status and facilitate rapid approval.
P26: Analyzing Drug Shortages and Pricing: Lessons from Canada
10:15AM - 10:35AM
Gaudette E1, Rizzardo S1, Tadrous M2, Pothier K1
1PMPRB, Ottawa, ON, Canada, 2University of Toronto, Toronto, ON, Canada
OBJECTIVES: International supply chains could favor countries with higher prices when there are production disruptions, leading to more shortages in countries with lower prices. This research aimed to assess whether drug shortages reported by Canadian manufacturers were associated with lower drug prices in Canada than in other countries.
METHODS: Using international drug sales data from IQVIA’s MIDAS database and drug shortage reports from the Drug Shortages Canada website, logistic and linear probability regression models were used to estimate the likelihood of oral solid drugs being reported as in shortage in Canada between 2017 and 2022. Model specifications included drug-level, ingredient-level and company characteristics, year effects, and international price ratio variables to assess associations between the relative price of Canadian drugs and shortages.
RESULTS: N = 31,651 drug-year observations and N = 14,750 shortage reports were included in the analysis. Among the N=13,564 drug-year observations for which international price comparisons were available, N=3,704 (27%) were reported in shortage. Price ratios were not significantly associated with the likelihood of shortages (p=0.087 in the logistic model; p=0.117 in the linear probability model), while therapeutic class, generic status, number of countries with sales, and number of manufacturers were (p<0.000 to p=0.041). In sub-analyses, the absence of a significant association between price ratios and shortages was found to be robust in both the non-patented and the patented market segments.
CONCLUSIONS: There was no significant association between the relative price of medicines and shortages in Canada during the study period, which did not support the hypothesis of lower drug prices increasing the probability of a drug being in shortage.
Modeling for HTA
Digital Conference Pass
Moderator
William Herring, PhD
RTI Health Solutions, Research Triangle Park, NC, USA
Will Herring, PhD, is an Executive Director in the Health Economics group at RTI Health Solutions with expertise in cost-effectiveness and value-based pricing modeling. His primary research focus is economic modeling for chronic progressive diseases (Alzheimer’s disease [AD], multiple sclerosis, nonalcoholic steatohepatitis, and diabetic retinopathy) and for conditions and interventions affecting older adults (AD, age-related macular degeneration, herpes zoster vaccination, and respiratory syncytial virus vaccination). He is Affiliated to Research at the Karolinska Institutet and is a member of the ISPOR Open-Source Modeling Special Interest Group.
P31: Improving Long-Term Survival Assessment in Cost-Effectiveness Analyses: Examining Excess Hazard Methods and Cure Models in Non-Small Cell Lung Cancer Study
10:45AM - 11:00AM
Pandey S1, Bajaj P2, Sharma A3, Singh B4, Kaur S2
1Pharmacoevidence, SAS Nagar, Mohali, PB, India, 2Heorlytics, SAS Nagar, Mohali, India, 3Pharmacoevidence, SAS Nagar, Mohali, India, 4Pharmacoevidence, SAS Nagar Mohali, PB, India
OBJECTIVES: Assessing the influence of different survival extrapolation techniques on cost-effectiveness analyses is crucial. This is particularly significant in situations where the need for projections extends beyond clinical trial durations, given the constraints of limited follow-up. This research aims to recognize and overcome obstacles related to parametric survival models by incorporating general population mortality rates directly into the modelling process through an excess hazard (EH) model.
METHODS: To incorporate the excess hazard (EH) directly into the modeling process, a clinical trial assessing a treatment with potential curative properties in non-small cell lung cancer (NSCLC) was selected. Seven standard parametric models were fitted to overall survival and progression-free survival data. The same seven models were subsequently applied within the EH framework, both with and without a cure parameter, integrating general population mortality rates to prevent unrealistic projections. The EH model separates mortality rates into background (expected) and excess rates, enabling more realistic long-term projections. Model fit evaluation was assessed by the Akaike information criterion (AIC) and visual inspection. The fitting of models and extrapolations was conducted using the "flexsurv" and "flexsurvcure" packages in R.
RESULTS: Based on the AIC and visual inspection, the Log-normal model exhibited the best fit in both frameworks-standard parametric model (AIC = 799.8) and EH framework (AIC = 765.4). The introduction of a cure assumption in EH framework led to an overall improvement in the goodness of fit results for most parametric models, except for the Exponential and Gompertz. Additionally, comparisons of root mean squared prediction error (RMSPE) further affirmed the improved fit of EH models for specific parametric distributions.
CONCLUSIONS: As excess hazard may decrease over time, extrapolating excess hazard beyond trial follow-up is easier than all-cause hazards, especially in oncology trials. EH cure models enhance stability and reliability, particularly when cure is plausible.
P32: Effective Data Visualization Techniques for Presenting Scenario Analysis Results in Health Economic Models
11:00AM - 11:15AM
Srivastava T1, Mitra S2, Purkayastha P2, Hriday K2, Swami S1
1ConnectHEOR, London, UK, 2ConnectHEOR, Delhi, India
OBJECTIVES: Probabilistic sensitivity analysis (PSA), deterministic sensitivity analysis (DSA), and scenario analysis are typically conducted to evaluate parameter and methodological uncertainties in health economic models. While PSA and DSA commonly use data visualization techniques like scatter plots and tornado charts to display outcomes, a standard/recommended method for effectively visualizing scenario analysis data remains undeveloped. This abstract explores various potential visualization techniques to effectively analyze data from multiple scenario analyses.
METHODS: A diverse range of data visualization techniques were examined, spanning categories such as comparison, correlation, part-to-whole and hierarchical, temporal, distribution, etc. For scenario analysis results, net monetary benefit (NMB) was chosen as preferred outcome measure over incremental cost-effectiveness ratio (ICER), as ICER can include a combination of numerical and textual values ("Dominant", "Dominated"). Scenarios with NMB>0 were classified as favorable, while those with NMB<0 were deemed unfavorable. For evaluating these visualization methods, a hypothetical model comprising 100 scenarios was used.
RESULTS: Our analysis identified two-part (diverging) bar charts and radial column charts as the most suitable data visualization techniques, given their ease of implementation in MS-Excel - a widely used tool in HTA modeling. In two-part bar charts, columns are arranged from highest to lowest based on the NMB values for each scenario, with favorable scenarios color-coded in green and unfavorable ones in red. Radial column charts are also effective featuring two distinct charts for favorable and unfavorable scenarios, sorted by NMB values. Additionally, for specific scenario categories, such as varying drug pricing or multiple time horizons, heat maps were identified as another viable option.
CONCLUSIONS: Effective data visualization for scenario analysis offers stakeholders a clear and comprehensive understanding of various scenarios, aiding in informed decision-making. Additionally, use of more advanced software for data visualization can further enhance this process, adding depth and clarity to the analysis.
P30: Semi-Markov Model (sMM) Versus Trial-Level Surrogacy to Predict Overall Survival (OS) for Adjuvant Treatment of Muscle-Invasive Urothelial Carcinoma (MIUC) with Chemotherapy
10:30AM - 10:45AM
Berardi A1, Kurt M2, Cope S3, van Beurden-Tan C3, Patel M4, Teitsson S5
1PRECISIONheor, London, LON, UK, 2Bristol Myers Squibb, Lawrenceville, NJ, USA, 3PRECISIONheor, VANCOUVER, BC, Canada, 4Bristol Myers Squibb, Princeton, NJ, USA, 5Bristol Myers Squibb, Uxbridge, UK
OBJECTIVES: Given longer time to maturity for OS in adjuvant MIUC trials, this study compared the accuracy of OS predictions for the EORTC-30994 trial (immediate [imm-chemo] versus deferred chemotherapy [def-chemo]) using two approaches: Method-1) a four state sMM, Method-2) an established surrogacy relationship between disease-free survival (DFS) and OS hazard ratios (HRs).
METHODS: Method-1: sMM included disease-free (DF), local recurrent (LR), distant recurrent (DR), and death states. DFS data for imm-chemo were modelled independently using parametric distributions. Transitions were estimated: from the DF by the modelled DFS and distribution of first recurrence events; from LR based on the median LR survival; from DR based on OS-HRs from published first-line metastatic indirect comparison and distribution of treatments received upon DR from def-chemo. Method-2: The predicted OS-HR (derived from surrogacy relationship using trial DFS-HR), was applied to the modelled OS of def-chemo. DF-patients at 5 years were assumed to be cured. OS predictions from each method were compared to the reported and extrapolated OS for imm-chemo using restricted mean survival time (RMST).
RESULTS: 10-year OS rates were reported to be 43.6%, and predicted to be 42.7% (Method-1) and 52.3% (Method-2). Method-1 performed slightly better than Method-2 when comparing RMST predictions (in months) (observed: 71.2; Method-1: 73.3; Method-2: 77.5). At 30-years, predicted OS rates were 4.7% (best fit), 4.0% (Method-1), and 5.9% (Method-2). 30-year RMST predictions (in months) were more precise for Method-1 over Method-2 when compared with the RMST obtained from the best-fit (best fit: 131.2; Method-1: 129.8; Method-2: 150.2).
CONCLUSIONS: The sMM may provide a more flexible and accurate approach than the surrogacy-based framework to predict long-term OS for adjuvant treatment of MIUC with chemotherapy by accounting for the disease process and impact of subsequent treatments on OS (not feasible for surrogacy method) while avoiding the proportional hazards assumption (required for surrogacy method).
P29: Calibration of a Patient-Level Simulation Model for Diabetes in the Absence of Patient-Level Data: A Proposed Method and Health Economic Implications
10:15AM - 10:30AM
Tran-Duy A1, Palmer A2, Dakin H3, Leal J3, Clarke P3
1University of Melbourne, Melbourne, VIC, Australia, 2University of Tasmania, Hobart, TAS, Australia, 3University of Oxford, Oxford, UK
OBJECTIVES: Many patient-level models for diabetes used old data for model development and therefore need calibrating for use in a new setting. In the absence of patient-level data for calibration, we proposed a method for adapting the UKPDS Outcomes Model version 2 to the Australian setting using published standardised mortality rate ratios (SMRs), and examined the health economic implications of this approach.
METHODS: We introduced new parameters representing annual percent reductions in the risks of major events, and applied a grid search of parameter values in the calibration. A synthetic cohort of 1,000 individuals aged 60 years was used as model input and the model run until all individuals had died. The simulated SMRs were calculated as the ratios of the predicted deaths to the expected deaths and compared with the observed SMRs. Sum of square errors (SSEs) was used to measure goodness-of-fit. The original model and best calibrated model were then used to assess the cost-effectiveness of SGLT2 inhibitors compared to sulfonylureas.
RESULTS: Infinitesimal SSEs were achieved with the grid search from < 600 combinations of parameter values. The smallest SSE (< 0.01) was obtained when annual risks of mortality, myocardial infarction, stroke, congestive heart failure and ischaemic heart disease were reduced by 36%, 32%, 20%, 28% and 30% for men and 44%, 42%, 34% 42% and 42% for women. The calibrated models produced greater healthcare costs and QALYs compared to the original model. The incremental costs per QALY gained predicted by the calibrated and original models were A$29,939 and $$26,997, respectively for men, and A$27,316 and A$24,396, respectively, for women.
CONCLUSIONS: Our study suggested that SMRs could be used to calibrate a patient-level model to predict mortality rates with a high accuracy. Without calibration, a model may produce biased estimate of the incremental cost-effectiveness ratio.
Breakouts: IP, WS, & OBS
The Future of Data-Driven HEOR Decision-Making Powered by Generative AI: How Soon is Now?
Digital Conference Pass
Level: Intermediate
PURPOSE:
This session will present the findings of the rapidly advancing generative AI, particularly Large Language Models (LLMs) across clinical development ranging from systematic literature review, in depth RWE, social media listening to RWD-informed clinical trials for decision making. Furthermore, it will highlight the unique impact of AI-enabled insight generation from different perspectives including health systems, pharmaceutical and technology industries. DESCRIPTION:
Everyday billions of data points are collected in diverse health data platforms. Expanding health data and innovation in AI modeling are coinciding with health challenges of a scale we’ve never seen — creating urgency for true and rich insights for decision making. In this session, Dr. Xiaoyan Wang from IMO will delve into the advancements of generative AI and LLMs, focusing specifically on her recent research in AI-enabled evidence synthesis from literature and clinical RWD for oncology clinical trials (12’). Dr. Hua Xu from Yale University will provide an overview of the Observational Health Data Sciences and Informatics (OHDSI) consortium he leads on AI methods to scale the use of clinical notes in RWE studies of mental health (12’). Dr. Abeed Sarker from Emory University will present on how AI has powered ”social listening” on self-reported outcomes and the formation of auto-growing chronic pain cohorts from social media platforms for clinical development of treatment strategies (12’). Dr. Ergin Soysal from Eli Lilly will share his insights from a pharmaceutical industry perspective on integrating AI into clinical trials, and whether AI and LLMs can reduce costs and accelerate the scientific process in HEOR (12’) We will review the opportunities and challenges of AI-powered insight generation, assessing its reliability for adequate, well-controlled investigations and confirmatory evidence for payers and regulatory decision-making (all panelists). Attendees are invited to engage with the panelists through a digital poll and Q&A during this session (12’).
Speakers
Abeed Sarker, PhD
Emory University School of Medicine, Atlanta, GA, USA
Abeed Sarker, PhD, is associate professor in the Department of Biomedical Informatics at Emory University School of Medicine. Dr. Sarker holds secondary appointments as associate professor in the Department of Computer Science of the College of Arts and Sciences at Emory University, and associate professor in the Department of Biomedical Engineering at Georgia Institute of Technology and Emory University.
Dr. Sarker is a member of the Cancer Prevention and Control Research Program at Winship Cancer Institute.
Ergin Soysal, MD PhD
Eli Lilly and Company, Indianapolis, IN, USA
Hua Xu, PhD
Yale University School of Medicine, New Haven, CT, USA
Dr. Hua Xu is Robert T. McCluskey Professor and Vice Chair for Research and Development, Section of Biomedical Informatics and Data Science at Yale School of Medicine (YSM), as well as Assistant Dean for Biomedical Informatics at YSM. He received his Ph.D. in Biomedical Informatics from Columbia University. His primary research interests include biomedical natural language processing (NLP) and data mining, as well as their applications in secondary use of electronic health records data for clinical and translational research. His research is funded by multiple agencies (i.e., NLM, NCI, NIGMS, NIA, AHA, and CPRIT), and methods/tools developed in his lab have been top ranked in a number of biomedical NLP shared tasks and widely used to support diverse biomedical applications. He served as the Chair of American Medical Informatics Association (AMIA) NLP Working Group and now leads the Observational Health Data Sciences and Informatics (OHDSI) NLP Working Group. Dr. Xu is a fellow of both the American College of Medical Informatics (ACMI) and the International Academy of Health Sciences Informatics (IAHSI).
Discussion Leaders
Xiaoyan Wang, PhD
Intelligent Medical Objects, Rosemont, IL, USA
Dr. Xiaoyan Wang serves as IMO’s Chief Scientist. She focuses on translating scientific insights and health intelligence into partnerships and programs that improve healthcare outcomes for patients, providers, and researchers.
Dr. Wang has spent two decades committed to driving innovations in healthcare AI modeling and transforming health care with data intelligence. She published hundreds of papers in NLP, AI modeling and data intelligence in health care and life sciences. She was most recently VP of Healthcare Analytics and Informatics and VP of Biopharma Solutions at GeneDx Mount Sinai Genomics Inc, where she led the development of clinical evidence generation platforms and clinical research in oncology, immunology, cardiovascular, respiratory, and rare diseases. Before joining the biopharma industry, she was a faculty member and principal investigator at the University of Connecticut, UConn Health Center, and Mount Sinai Health Systems bridging research, health services, and teaching.
She holds a BA in Biochemistry from Shandong University, a MA in Genetics from the University of Kansas, and a Ph.D. in Biomedical Informatics and NLP from Columbia University School of Medicine.
Instrumental Variables: Revolutionizing Evidence Generation Using Real-World Data
Digital Conference Pass
Level: Intermediate
PURPOSE:
RWD is pivotal in generating evidence for drug discovery, regulatory, and post-authorisation purposes. However, inherent biases can compromise the validity of RWD. While instrumental variable methods are widely utilised in economics and epidemiology research in academia, their adoption in the pharmaceutical industry has been limited until recently. In this discussion, we present use-cases of instrumental variables within the UK Biobank and CPRD, illustrating their role in generating evidence pertaining to drug discovery, post-authorisation effectiveness, safety evaluations, and facilitating market access.
DESCRIPTION:
Case 1: IVs in the Clinical Practice Research Database (CPRD) were used to examine real-world safety and effectiveness in specific populations, unexplored outcomes, and long-term follow-ups (1–5 years) for smoking cessation medicines. Notably, our studies provided precise estimates overcoming underpowered analyses in the largest comparable trials to-date. Case 2: Assessed the safety and effectiveness of two type-2 diabetes treatments in older adults (+70 years) using IVs and hospital-linked UK primary care data. Current guidelines recommend individualized treatment decisions for this demographic, but limited evidence exists due to their frequent exclusion from trials. Our approach addressed this gap, offering insights based on larger sample sizes and overcoming issues related to residual confounding in previous observational studies. Case 3: In large real world cohort studies like the UK Biobank, participants undergo a comprehensive baseline assessment to capture clinical, genetic and omics measurements. This has made it an indispensable tool in contemporary drug development using a genetic IV analyses termed 'Mendelian randomization' (MR). We describe how MR provides key lines of evidence to inform early drug target discovery as well as post-licensing expansion of an existing therapy to new indications. In conclusion, we argue that IVs provide an attractive alternative to other adjustment methods in the evolving landscape of evidence generation, particularly compared to propensity scores and multivariable regression analysis.
Speakers
Jack Bowden, PhD
Novo Nordisk, Oxford, Oxfordshire, United Kingdom
Jack Bowden is a Professor of Biomedical Data Science within the diabetes group at the University of Exeter and a Senior Scientific Director at Novo Nordisk Oxford. He obtained his PhD from the University of Leicester in 2007, before completing post-doctoral research positions at the MRC Biostatistics unit in Cambridge and the MRC Clinical Trials Unit in London. From 2015-2019 he led a research programme around Mendelian randomization at the MRC Integrative Epidemiology Unit in Bristol. Since 2019 at Exeter, his research has focused on exploiting genetics to understand the causes of multimorbidity and treatment effect heterogeneity, as well as using routine data for precision medicine in diabetes. Within the Genetics Centre of Excellence at Novo Nordisk Research Centre Oxford (NNRCO), he oversees the use of Mendelian randomization methods to support target discovery and the broader use of genetics within clinical trials. He has published over 100 scientific papers in the areas of statistical genetics, causal inference, clinical trials methodology and meta-analysis, and has been listed as a Clarivate Highly Cited Researcher each year since 2019.
Laura Güdemann, PhD
University of Exeter, Exeter, DEV, United Kingdom
Laura Güdemann is a Biostatistician and Research Associate in the Medical School at University of Exeter. Her research focuses on the development and application of causal inference methodology for the assessment of safety and effectiveness of Type 2 Diabetes treatments. Her expertise lies in causal treatment effect estimation using Instrumental Variable approaches and health care provider prescription preference as instrument to mitigate the risk of unmeasured confounding bias in observational studies.
Discussion Leaders
Gemma Taylor, PhD
Health Economics and Outcomes Research Ltd, Cardiff, United Kingdom
Dr. Gemma Taylor is an award-winning statistical consultant, academic, and epidemiologist with over 15 years of experience spanning the NHS, pharmaceutical, and academic sectors. Holding a dual appointment, she serves as the Director of Data Science and Analytics in the pharmaceutical consulting sector. In this role, her team specializes in generating evidence to support clients with market access, focusing on bespoke analysis, real-world data (RWD), meta-analysis, and clinical trial analysis.
Simultaneously, Dr. Taylor holds a position as a Reader in the academic sector, where she teaches statistics and research methods. Additionally, she leads an independent research portfolio centered on real-world data (RWD) and clinical trials.
Dr. Taylor is widely recognized for her pioneering and internationally renowned work in tobacco epidemiology. Her research has been instrumental in demonstrating the mental health benefits of smoking cessation and implementing effective interventions in psychology services.
10:30 AM - 11:15 AM
Job Seeker or Hiring Manager? Explore ISPOR’s HEOR Career Center
Looking to take a new step in your career or fill an open position? Learn how ISPOR can support your career development and hiring needs through its HEOR Career Center. Bonus: Stop by the ISPOR Booth and get your job posting discount code!
10:30 AM - 1:30 PM
Poster Session 3
Live
11:15 AM - 1:15 PM
Lunch
As you enjoy your lunch in the Exhibit Hall, seize the opportunity to engage in meaningful conversations with fellow attendees. Take this time to exchange ideas, forge new partnerships, or simply enjoy causal conversation. Provided by ISPOR.
11:30 AM - 12:00 PM
Fast Facts
We Need to Talk: Using the Academy of Managed Care Pharmacy Format for Formulary Submissions 5.0 to Facilitate More Effective Evidence-Based Discussions with US Payer Audiences
Level: Introductory
The need for effective, evidence-based dialogue between manufacturers and payers has never been greater. In this session, experts will review the recently released AMCP Format for Formulary Submissions, v. 5.0, highlighting how it can support effective, bidirectional communication between manufacturers and payers. Join us to learn more about the updated Format, and gain insights on best practices to support early and ongoing scientific exchange.
Discussion Leader
Diana Brixner, RPh, PhD, FAMCP
University of Utah College of Pharmacy, Salt Lake City, UT, USA
Dr. Diana Brixner is currently a Professor in the Department of Pharmacotherapy and is a Founder of the Pharmacotherapy Outcomes Research Center ( PORC)at the University of Utah in Salt Lake City, UT. She is a member of both the Huntsman Cancer Institute Cancer Control and Population Sciences and the Center for Genomic Medicine. Prior to her academic appointment in 2002 she worked both in the biotech (NeoRx) and pharmaceutical (SmithKline Beecham, Novartis) industry focused on value assessment of their technologies in the early years of the field. Dr. Brixner was a Past President of both the Academy of Managed Care Pharmacy (AMCP) and International Society of Pharmacoeconomics and Outcomes Research (ISPOR). She has served as a one-year Scholar-in-Residence for the Academy of Managed Care Pharmacy, recently served on the scientific advisory board of Lumanity and is a member of the Executive Board of the International Market Access Society.
Jeff Lee, PharmD, FCCP
Lumanity, Franklin, TN, USA
Jeff Lee, PharmD, FCCP is Vice President, Value Demonstration at Lumanity, where he leads a global team of HEOR strategists and researchers focused on value evidence creation, optimization, and translation to a wide range of payer audiences. Jeff is a career HEOR leader in a variety of pharma and academic roles, most notably as Head of the global HEOR and US payer medical affairs teams at Allergan, International Director of HEOR at Glaxo, and Associate Dean of Academic Affairs at Lipscomb University College of Pharmacy.
As Past Chair of the AMCP Format Executive Committee, he led the development of the AMCP Format for Formulary Submissions, Version 4.0. Jeff remains on the committee and recently led the subgroup on preapproval information exchange (PIE) for AMCP Format 5.0. Jeff is a frequent moderator for AMCP Partnership Forums on payer evidence considerations, formulary decision-making, and rare diseases, has provided expert testimony for US Congressional briefings on PIE, and led development of ACCP’s fellowship training guidelines in pharmacoeconomics.
11:30 AM - 12:15 PM
Poster Tours
Student Research Poster Tour
Posters Featured in this Tour: PT31: Self-Rated Health: Predictors of Poor Health Status and Associations with 5-Year Mortality Among UK Adults PT32: Assessing the Effect of Risk of Bias Assessments in Randomized Controlled Trials of Pharmacological Interventions with Different Types of Outcomes and Comparators – a Bayesian Meta-Epidemiological Study PT33: Cost-Effectiveness of Cemiplimab Plus Chemotherapy Versus Pembrolizumab Plus Chemotherapy As the First-Line Treatment for Advanced Non-Small Cell Lung Cancer: A Preliminary Study PT34: Assessment of Direct, Indirect, and Intangible Costs of Rheumatoid Arthritis in Patients Receiving Biological Therapy in Morocco: Ecoram Study PT35: Cost-Effectiveness Analysis of Nivolumab Versus Docetaxel for the Treatment of Advanced Nonsquamous NSCLC Including Pd-L1 Testing in the United States PT36: Use and Discontinuation Rates of Long-Acting Injectable Antipsychotics between Race/Ethnicity in Older Adults Using Medicaid Claims Data
Moderator
Zeba Khan, RPh, MS, PhD
Center for Health Outcomes, Policy & Economics, Ernest Mario School of Pharmacy, School of Public Health, Rutgers University, Piscataway, NJ, USA
Dr. Zeba Khan is currently the Founder and CEO of Zebgene LLC, and the Interim Program Director for Rutgers University Center for Health Outcomes, Policy, and Economics (HOPE) at the Ernest Mario School of Pharmacy. She has more than 25 years of experience establishing and leading multi-disciplinary corporate functions for biopharmaceutical companies including health economics and outcomes research, market access, pricing and reimbursement, public policy, corporate responsibility, and global health. Zeba holds a PhD and MS in Health Economics and Health Outcomes from The University of Texas at Austin. Zeba graduated with a Pharmacy degree from the University of Utah.
Oncology Poster Tour
Posters Featured in this Tour: PT25: The Economic Burden Associated with Treating Resected Non-Small Cell Lung Cancer in the US PT26: Health Economic Impact of Non-Persistence with Tamoxifen Adjuvant Endocrine Therapy in Pre-Menopausal Women with Early-Stage Breast Cancer: A Simulation Study PT27: Changes in the Economic Burden of Breast Cancer Neoplasms in Colombia after the Cancer Control Model PT28: Exploring the Potential of Real-World Data Sources in Oncology and Rare Diseases: A North American Perspective PT29: A Methodologic Solution to Missing Deauville Scores Using Imaging Report Data to Classify Lymphoma Treatment Response in Real-World Data PT30: Real-World Cost-Effectiveness of Publicly Reimbursed Multi-Gene Panel Sequencing to Inform Therapeutic Decisions for Advanced Non-Small Cell Lung Cancer in British Columbia, Canada
Moderator
Elizabeth S. Mearns, PharmD
Genentech, South San Francisco, CA, USA
11:45 AM - 12:15 PM
Exhibit Hall Theater
Rare Cancers, More Common Than You Think: Addressing the Challenges, Opportunities and Methodologies in Studying Small Populations
Digital Conference Pass
The rapid development of multiple new actionable biomarkers in oncology has transformed how we treat cancer patients. In the last 5 years, more than 60% of oncology drugs have been approved based on specific biomarkers, requiring biomarker testing to determine the best treatment options, and meaning that many cancer types that were previously common are now rare (defined by specific biomarker status). What are the implications of this for real-world research?
In this presentation, we will discuss what constitutes a rare cancer, the challenges and opportunities associated with studying small patient populations, and the ways in which analytic approaches must be adapted to generate robust real-world evidence and insights and make reliable inferences.
Sponsor
McKesson/Ontada
Speaker
Amy O'Sullivan, PhD
Ontada, Boston, MA, USA
Amy K. O’Sullivan, PhD, is Chief Research Officer at Ontada, where she leads the Real World Research team. Amy is a health economist with over 25 years of experience conducting health economics and outcomes research (HEOR) on medical interventions throughout the product lifecycle. Prior to joining Ontada, Amy held leadership positions at Alkermes, Inc., Vertex Pharmaceuticals, IMS Health, and OptumInsight. She holds a BA in Economics and English from Boston College and PhD in Health Economics from the Johns Hopkins University Bloomberg School of Public Health.
Nicholas Robert, MD
Ontada, Boston, MA, USA
Dr. Nicholas J. Robert is the Chief Medical Officer at Ontada, a McKesson company). A graduate of the McGill Medical School, Dr. Robert completed an Internship and Residency in Internal Medicine at the Royal Victoria Hospital in Montreal. Following a residency in Anatomic Pathology at Massachusetts General Hospital where he was Chief Resident, he continued his training in Hematology at Brigham and Women’s Hospital and in Medical Oncology at Dana Farber Cancer Institute. He was a Fellow at Harvard Medical School.
Other former positions include Clinical Director of Medical Oncology at New England Medical Center; Medical Director of Research program at Virginia Cancer Specialists; Chief of the Section of Hematology/Oncology and Chairman of the Cancer Committee of the Inova Fairfax Hospital and Chairman of the Cancer Committee as well as the Chairman of the ECOG Breast Committee and Co-chair of the Breast Committee of The US Oncology Research Network. He was a faculty member of ASCO University.
In the last 7+ years he has been involved with McKesson HEOR program and in 2022 became the Chief Medical Officer at Ontada, a McKesson company involved with data, analytics, and technology in oncology.
He has authored over 100 publications and is a reviewer for several medical publications including the Journal of Clinical Oncology-Clinical Cancer Informatics, as well as the recipient of numerous research grants. He is also a member of several organizations including the American Society of Clinical Oncology (ASCO) and former member of the Cancer Research Committee and past chair of the Research Community Forum. He was a member of the Eastern Cooperative Oncology Group and National Surgical Adjuvant Breast and Bowel Project. He is board certified in Internal Medicine, Anatomic Pathology, Medical Oncology, and Hematology.
Zhaohui Su, PhD
Ontada, Chestnut Hill, MA, USA
Zhaohui Su, PhD is VP of Biostatistics at Ontada, and is a scientific leader with 25 years of experience in applying statistical, epidemiological and machine learning methods to Real-World studies. He was VP of Real-World Evidence (RWE) Analytics at OM1, Inc., and Sr. Director of Biostatistics at Quintiles, Inc., responsible for conducting Real-World Data and RWE analytics. Dr. Su has been a Statistical Editorial Board member and a scientific reviewer for Patient-Centered Outcomes Research Institute since 2015.
11:45 AM - 12:45 PM
Educational Symposia
Advancing HEOR and RWE Research With Generative AI (GenAI)
Digital Conference Pass
Level: Intermediate
GenAI is revolutionizing HEOR and RWE by offering innovative methods to process and analyze vast datasets, model complex health economics phenomena, and enhance decision-making processes. GenAI tools powered by LLMs facilitate the development of deeper and more comprehensive understanding of diseases and treatment outcomes and significantly improve the extraction of insights from diverse data sources, including electronic health records, claims data, and scientific literature. By harnessing the power of GenAI, researchers and practitioners in the field are unlocking new possibilities for advancing health care innovation and improving patient care. Participants in this symposium will explore the frontiers in GenAI, discuss key developments and challenges, and present examples of GenAI’s applications in HEOR and real-world research. Among these examples is Analysis Group’s own proprietary GenAI platform, which excels in text classification, research summarization, and automated data analysis. The introduction of this platform and similar GenAI platforms is set to further empower the health care sector, offering more effective and efficient tools to researchers for streamlining complex analyses, enhancing research accuracy, facilitating evidence-based decision making, and deepening overall understanding of complex diseases and treatments, ultimately leading to more effective health care solutions and policymaking.
Sponsor
Analysis Group
Moderators
Eric Q. Wu, PhD
Analysis Group, Boston, MA, USA
Dr. Wu is a health economist with expertise in health economics and outcomes research (HEOR), market access,
real-world evidence (RWE) generation, AI applications, and scientific evidence strategy. He has conducted
research in more than 30 countries and contributed to over 300 publications. Dr. Wu focuses a significant portion
of his practice on developing new scientific methods to address challenges in medical big data, RWE, AI, and
innovative comparative-effectiveness research methodologies.
Speakers
Rajeev Ayyagari, PhD
Analysis Group, Inc., Boston, MA, USA
Jimmy Royer, PhD
Analysis Group, Inc., Boston, MA, USA
Dr. Royer applies a broad range of AI and GenAI tools to address client needs in data science and HEOR. His recent work includes using AI models to predict treatment resistance and the potential future onset of rare or undiagnosed conditions. In addition, Dr. Royer has conducted extensive academic research and coauthored books and papers on using neural networks to predict treatment resistance in tuberculosis, using new AI advances in HEOR, using machine learning algorithms in propensity score models, and determining the impact of hypertension therapies on mortality, among other topics.
Hua Xu, PhD
Yale University School of Medicine, New Haven, CT, USA
Dr. Hua Xu is Robert T. McCluskey Professor and Vice Chair for Research and Development, Section of Biomedical Informatics and Data Science at Yale School of Medicine (YSM), as well as Assistant Dean for Biomedical Informatics at YSM. He received his Ph.D. in Biomedical Informatics from Columbia University. His primary research interests include biomedical natural language processing (NLP) and data mining, as well as their applications in secondary use of electronic health records data for clinical and translational research. His research is funded by multiple agencies (i.e., NLM, NCI, NIGMS, NIA, AHA, and CPRIT), and methods/tools developed in his lab have been top ranked in a number of biomedical NLP shared tasks and widely used to support diverse biomedical applications. He served as the Chair of American Medical Informatics Association (AMIA) NLP Working Group and now leads the Observational Health Data Sciences and Informatics (OHDSI) NLP Working Group. Dr. Xu is a fellow of both the American College of Medical Informatics (ACMI) and the International Academy of Health Sciences Informatics (IAHSI).
Methodological and Policy Issues Related to the Cost-Effectiveness of Newborn Screening in Severe Pediatric Genetic Disorders
Digital Conference Pass
Revising newborn screening guidelines in the United States and other countries is slow and arduous, with the implementation of newborn screening measures often taking 4-5 years. HEOR provides a systematic framework for evaluating proposed revisions and offers the hope of speeding adoption of worthwhile technologies for patients. The objective of this educational symposium is to explore the challenges involved in using HEOR to evaluate potential revisions and make recommendations regarding elements of best practice for the adoption of new diseases in newborn screening panels, with a focus on severe genetic diseases such as for example, metachromatic leukodystrophy (MLD).
The symposium will begin with an overview by a health economist of the potential methodological issues that could be encountered when framing and populating a cost-effectiveness analysis of newborn screening. These will be complemented with perspectives from a clinician, patient advocate and payor. The session will conclude with policy, procedural and methodological recommendations for best practice and future research.
Sponsor
Orchard Therapeutics
Moderator
Peter Neumann, ScD
Tufts Medical Center, Boston, MA, USA
Peter J. Neumann, Sc.D., is Director of the Center for the Evaluation of Value and Risk in Health (CEVR) at the Institute for Clinical Research and Health Policy Studies at Tufts Medical Center, and Professor of Medicine at Tufts University School of Medicine. He is the founder and director of the Cost-Effectiveness Registry, a comprehensive database of cost-effectiveness analyses in health care. Dr. Neumann has written widely on the role of clinical and economic evidence in pharmaceutical decision making and on regulatory and reimbursement issues in health care. He served as co-chair of the 2nd Panel on Cost-Effectiveness in Health and Medicine. He is the author or co-author of over 300 papers in the medical literature, and the author or co-author of 3 books: Using Cost-Effectiveness Analysis to Improve Health Care (Oxford University Press, 2005) ; Cost-Effectiveness in Health and Medicine, 2nd Edition (Oxford University Press, 2017); The Right Price: A Value-Based Prescription for Drug Costs (Oxford University Press, 2021). Dr. Neumann has served as President of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). He is a member of the editorial advisory board of Health Affairs and the health policy advisory board for the Congressional Budget Office. He has also held several policy positions in Washington, including Special Assistant to the Administrator at the Health Care Financing Administration. He received his doctorate in health policy and management from Harvard University.
ISPOR Forums
Incorporating Health Equity in HTA Systems and Research: A Global Perspective and Future Outlook
Digital Conference Pass
Health disparities and equity in HTA systems and research are pivotal issues that influence the accessibility, affordability, and equality of healthcare. The data connectivity between healthcare and social security, and the varying levels of awareness further complicate these issues. The session aims to address these complexities by fostering a holistic discussion on the landscape of health equity, and how to promote relevant research and incorporate them into healthcare policy for improved patient access.
Ota will open the session by describing the importance of accounting for equity within HTA from the industry perspective and then will invite panellists representing diverse geographical regions and professional backgrounds to share the current state of health equity research within HTA. Then, each speaker will describe how health equity is currently considered within HTA in their region based on their experience in their professional role (Dr Igarashi-academic, Dr Pearson-Stuttard-regulatory, and Dr Whittington-value assessment). Each panellist will present an agenda for the next three to five years that will allow for the better incorporation of health equity considerations within HTA decision-making and research activities. The role of each stakeholder group (i.e., industry, academic, regulatory, value assessment) in promoting the consideration of health equity will be addressed, reflecting upon panellists’ diversified professional roles. Using the polling software, we will ask participants about the outlook of health equity at the beginning and the end of the workshop. Twenty minutes will be reserved for audience comments.
Moderators
Riku Ota, MPH
Novo Nordisk Pharma Ltd., Nerima, 13, Japan
Riku Ota is a dynamic and dedicated public health professional, with a comprehensive career spanning the pharmaceutical sector and consultancy across the Asia Pacific and Europe. His journey began with a focus on the clinical development of cutting-edge treatments for inflammatory bowel diseases (IBD), laying a solid foundation for his expertise in health outcomes and evidence generation.
In his pivotal role at Novo Nordisk's Japanese affiliates, Riku served as a Health Economics and Outcomes Research (HEOR) manager, where he played a critical role in expanding patient access to healthcare by highlighting the clinical and economic benefits of novel health technologies. Progressing within Novo Nordisk A/S in Denmark, he now excels as the Global Payer Evidence Lead, overseeing the MASH/NASH therapeutic area. This position underlines his commitment to integrating clinical value with strategic market access initiatives on a global scale.
An engaged member of the Health Equity Special Interest Group (SIG) within ISPOR for the Asia-Pacific region, Riku contributes to advancing health equity discussions and initiatives. His dedication to the field is further evidenced by his selection as a member of the New Professional Steering Committee for ISPOR for the 2023-2024 term, where he plays a significant role in steering the organization's future direction and initiatives.
Riku has also led the Health Technology Assessment (HTA) Taskforce for the EFPIA Japan Access Committee, demonstrating his leadership in one of the pharmaceutical industry's most influential bodies. His academic foundation is robust, with a Bachelor of Science in Statistics from the University of Otago, New Zealand, and a Master of Public Health from King's College London, UK. These qualifications underscore his analytical prowess and understanding of global health landscapes.
Currently, Riku is enhancing his strategic and leadership skills through a hybrid part-time Master of Business Administration program at IE Business School, Spain. His primary interests lie in global public health and addressing health disparities, particularly those arising from socioeconomic factors. Riku Ota's multifaceted career and ongoing academic pursuits highlight his commitment to making a significant impact on public health and healthcare accessibility worldwide.
Speakers
Ataru Igarashi, Ph.D.
Yokohama City University School of Medicine, Yokohama, Kanagawa, Japan
Jonathan Pearson-Stuttard, FRSPH, FFPH, MAPHA
Health Analytics, Lane Clark & Peacock LLP, London, LON, United Kingdom
Jonathan is Head of Health Analytics at LCP, leading a multi-disciplinary team developing innovative analytics and technology approaches to address the biggest health challenges today. As a Public Health Physician and Epidemiologist, he is also Chair-elect of the Royal Society for Public Health, Chair of the Health Inequalities Programme Board at Northumbria Healthcare NHS Foundation Trust, and Honorary Clinical Senior Lecturer at Imperial College London. Jonny co-authored the book– Whose Health is it, anyway? – with Dame Sally Davies where they make the case to reposition health as our most untapped opportunity for prosperity and happiness.
Melanie Whittington, PhD, MS
Tufts Medical Center (CEVR), Boston, MA, USA
Dr. Melanie Whittington is a Senior Fellow at the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center and has a decade of experience in health economics and outcomes research. Most recently, she was the Director of Health Economics at the Institute for Clinical and Economic Review (ICER) where she led the cost-effectiveness analyses and budget impact analyses within ICER reviews. She was the 2022 recipient of the Bernie J. O’Brien New Investigator Award.
ISPOR Good Practice Task Force Recommendations on Valuing HRQoL of Children & Adolescents in Economic Evaluation (Pediatric Utilities)
Digital Conference Pass
ISPOR launched a Good Practice Task Force on pediatric utilities to address the gaps in both methods research and guidance from decision makers about pediatric HRQoL utilities for use in economic evaluation. There is a lack guidance specific to child HRQoL utilities, or the implicit assumption that what is recommended for the valuation of adult HRQoL is also appropriate for children and adolescents. Yet there are a number of methods issues specific to the valuation of HRQoL in children that do
not arise in the valuation of adult HRQoL. Moreover, the methods used to preference-weight pediatric HRQoL instruments differ in important ways that decision makers may not be aware of.
This forum will highlight the principal methods issues concerning:
- Whose stated preferences are relevant to the preference weighting of pediatric HRQoL states?
- What perspective respondents should be asked to adopt in imagining and valuing pediatric HRQoL?
- What preference elicitation methods should be used? and
- To what extent is the comparability and consistency of health state utilities for adult and child HRQoL important for their use in QALY estimation, economic evaluation and decision making?
The task force will present their recommendations and seek audience feedback on the overall approach taken. Pediatric utilities inevitably involves both HEOR science and normative considerations. Given the pivotal role of value judgements in methods choices in pediatric utilities, consultation with local HTA bodies is essential both to ensure that utilities are ‘fit for purpose’, and to ensure that decision makers are well informed about the role and implications of value judgements when establishing methods guidelines. The session will conclude with a question and answer session.
Moderators
Nancy Devlin, PhD
School of Population and Global Health, University of Melbourne, Melbourne, Australia
Nancy is Professor of Health Economics at the University of Melbourne and Senior Fellow at the Office of Health Economics, London. She is Chair of the Board of the EuroQol Research Foundation and served as President of ISPOR (2019-2020). Her principal areas of research expertise are the measurement and valuation of patient reported health outcomes; the cost effectiveness thresholds used in making judgments about value for money; and priority setting in health care. She is currently leading a large programme of research to improve measurement and valuation of quality of life in children (QUOKKA), funded by the Medical Research Future Fund (Australia). Nancy has published >180 original peer reviewed journal papers on a wide range of empirical and theoretical topics in health economics.
Speakers
Fleur Chandler, MSc
Sanofi and Patient Advisory Board Lead, Duchenne UK, Reading, Berkshire, Great Britain
Fleur is Head of Market Access UK & Ireland at Sanofi. Fleur is a health economist with a wealth of outcomes research and health technology assessment experience, with over 30 years leading both global and local teams in the pharmaceutical industry. Fleur is especially driven by rare disease, a parent of a teenager with Duchenne Muscular Dystrophy, she is on the advisory board of patient organisation Duchenne UK. Bringing her professional and personal experience together, she conceptualised and leads Project HERCULES, a global collaboration generating gold standard disease level evidence for HTA in Duchenne.
Ernest Law, PharmD, PhD
Pfizer, New York, NY, USA
Ernest is a Senior Director, HTA, Value & Evidence (HV&E) Strategy team at Pfizer. His research interests include health preferences, patient-reported outcomes, patient-centered and comparative effectiveness research, and shared decision-making. Ernest is involved in conducting early- and late-phase clinical trials, real-world studies, and economic evaluations, currently focusing on autoimmune diseases in both adult and adolescent populations. He is a member of the Euroqol Group, an international network of scientists dedicated to the science of health measurement, valuation, and evolving the EQ-5D family of instruments. Ernest holds a Doctor of Pharmacy (PharmD) from the University of British Columbia and obtained his Ph.D. from the University of Illinois at Chicago.
Louis Matza, PhD
Patient-Centered Research, Evidera, Bethesda, MD, USA
Member Group Meetings
ISPOR Mexico Chapter Meeting
This meeting is open to everyone interested in connecting with the ISPOR Mexico chapter. It offers a valuable opportunity to learn more about the chapter’s initiatives and explore how to participate in the chapter.
ISPOR Women in HEOR Session
Economic EmpowerHERment: Unveiling Insights Into Public Health and Economic Research
Digital Conference Pass
The objective of the Women in HEOR initiative is to support the growth, development, and contribution of women in HEOR, serve as a catalyst for leadership and a platform for networking, collaboration and mentorship. This session will highlight a historical perspective of economic evaluation across federal government to support policy decision making. Keynote speaker and renowned expert, Dr Phaedra Corso, will offer insights on the intersection of public health and economic considerations with an emphasis on specific health disparities and challenges faced by women. The agenda for the session will include: 1) an introduction to the initiative and its objectives and activities 2) keynote address by Dr Phaedra Corso, and 3) a moderated discussion of the topic, addressing the priorities of the Women in HEOR initiative. Attendees will gain valuable perspectives, considering both public health priorities and the economic impact of interventions. This session is open to all conference attendees.
Moderators
Shelby Reed, PhD, RPh
Duke University’s School of Medicine, Durham, NC, USA
Shelby D. Reed, PhD, is Professor in the Departments of Population Health Sciences and Medicine at Duke University’s School of Medicine. She is the director of the Center for Informing Health Decisions at the Duke Clinical Research Institute. She also is core faculty at the Duke-Margolis Center for Health Policy and an affiliate member at the Duke Cancer Institute. Shelby has 25 years of experience in health economics, health preferences and health policy research, with more than 200 published manuscripts. Shelby has been an active member of ISPOR for over 20 years. She was the first recipient of ISPOR's Bernie O’Brien New Investigator Award in 2005. She was elected to ISPOR’s Board of Directors in 2009. She served as President of ISPOR in 2017-2018 when she helped to establish ISPOR’s Women in HEOR Initiative. Most recently, she served as Chair of the Health Sciences Policy Council from 2019 to 2021.
Speakers
Phaedra Corso, PhD, MPA
Indiana University, Indianapolis, IN, USA
Julia F. Slejko, PhD
University of Maryland Baltimore, Baltimore, MD, USA
Julia F. Slejko, PhD is an Associate Professor of Practice, Sciences, and Health Outcomes Research at the University of Maryland School of Pharmacy and is Co-Director of the Patient-Driven Values in Healthcare Evaluation (PAVE) Center. Dr Slejko’s research is focused on innovative approaches for decision-analytic modeling for economic and health outcomes assessments. She holds a BA in Molecular, Cellular, and Developmental Biology from the University of Colorado Boulder. During her PhD training, she focused on pharmacoeconomics at the University of Colorado School of Pharmacy Center for Pharmaceutical Outcomes Research (CePOR). Her postdoctoral training was completed at the Pharmaceutical Outcomes Research and Policy Program in the University of Washington School of Pharmacy. Prior to her PhD training, she had a 7-year career in drug discovery at Array BioPharma. Dr Slejko serves as a Value in Health Associate Editor, Co-Chair of the ISPOR Faculty Advisor Council, co-lead of ISPOR’s Women in HEOR initiative, and is a member of ISPOR’s Health Science Policy Council.
12:15 PM - 12:45 PM
Exhibit Hall Theater
Innovative Strategies for Fit-for-Purpose RWE Research: Maximizing Data Completeness and Accuracy
Digital Conference Pass
Traditional real-world datasets often present challenges due to missing or inaccurate data. We will explore innovative strategies for real-world evidence research that leverage deep patient-level clinical datasets abstracted by treating physicians. Through our own research examples, we will showcase the impact of our unique methodology, highlighting the value of data completeness and accuracy in fit-for-purpose RWE.
Learning objectives
- Discuss common dataset challenges
- Explore our novel physician-led chart abstraction methodology
- Review successful RWE examples of data completeness and accuracy
Sponsor
Cardinal Health
Speaker
Danielle Gentile, PhD
Real-World Evidence and Insights, Cardinal Health, Dublin, OH, USA
Danielle Gentile, PhD is a Senior Scientist on the Real-World Evidence and Insights team at Cardinal Health. She investigates the health outcomes of patients treated in real-world settings outside of clinical trials. Before joining Cardinal Health, she served as a Health Services Researcher in the Department of Supportive Oncology at Levine Cancer Institute, Atrium Health in Charlotte, NC where she resides. She earned her PhD in Health Promotion, Education, and Behavior from the Arnold School of Public Health at the University of South Carolina.
Richard Swain, PhD, MPH
Cardinal Health, Dublin, OH, USA
Director of Regulatory Science and Real-world Evidence with Cardinal Health. Primary duties include developing strategies to incorporate alternative study design methods, such as inclusion of real-world evidence, to increase efficiency of clients' regulatory submissions. Previously, as Lead Epidemiologist in the FDA/CDER Division of Epidemiology, led the epidemiology team overseeing real-world evidence safety and efficacy issues for oncology, hematology, and medical imaging products. Over a decade of experience with epidemiological study design, protocol and statistical analysis plan development, performing systematic literature reviews, and conducting research using large, multi-level electronic healthcare datasets in regulatory, consulting, and academic settings. Publication history includes abstracts and presentations for scientific conferences and manuscripts in peer-reviewed journals. Earned a PhD in Epidemiology, and a Certificate in Pharmacoepidemiology, from Johns Hopkins University and an MPH in Epidemiology, with a concentration infectious disease, at the George Washington University. Additional areas of expertise include real-world evidence in oncology, type 1 diabetes, self-harm and suicide, and clinical trials.
12:30 PM - 1:15 PM
Special Onsite Join / Renewal Event for ISPOR Membership
Special Onsite Promo! Stop by the ISPOR Booth to join or renew your ISPOR membership! We might just have a surprise for you...
12:45 PM - 1:15 PM
Exhibit Hall Theater
AI in RWE: Key Drivers for Accelerating Clinical Development and Patient Access
Digital Conference Pass
The evolution of the healthcare ecosystem over the past decade has resulted in the increasing availability of big health data – i.e., real-world data (RWD) for patients. As a corollary, real-world evidence (RWE) derived from the analysis of RWD - including claims data, trial data, and meta-analyses - is becoming a critical component throughout the product development lifecycle, especially in support of patient access via health technology assessment (HTA) and reimbursement discussions. The volume and complexity of RWD make this a prime area for the application of artificial intelligence (AI) to generate RWE for clinical development and patient access.
The speakers will focus on how to leverage RWE and AI for accelerated clinical development and patient access:
Matthew Gordon will provide an overview of RWE and a summary of current regulatory guidelines for its use across the product development lifecycle.
Jackie Vanderpuye-Orgle will present the applications of RWE in clinical development and patient and the use of AI to generate RWE, focusing on methods including large language models (LLMs), machine learning (LM) and the concept of digital twins.
The presentation will provide the audience with concepts/tools to inform future evidence-generation activities in health economics and outcomes research and foster evidence-based decision-making.
Following the presentations, there will be an interactive Q&A session where the speakers will address the questions from the audience and discuss the challenges and opportunities of RWE in the healthcare sector. The session will conclude with some key takeaways and recommendations for practice in this field.
Sponsor
Parexel
Speaker
Matthew Gordon, BA
Parexel International, Glenview, IL, USA
Matthew has over 20 years' experience designing global real-world evidence solutions to support the development and commercialization of pharmaceutical/biotechnology products and devices. His experience covers a wide-range of therapeutic areas, as well as strategic objectives from natural history programs, external control arm studies for regulatory approval, and global safety-surveillance mandate. His technical expertise includes program strategy and operational structure in disease and product registries, prospective pharmacoeconomic studies, and systematic reviews of scientific literature.
Work Experience
Prior to joining Parexel, Matthew served as Vice President, Real-World Evidence at Worldwide Clinical Trials, where he led a global team responsible for all observational research programs. Matthew had also held senior management roles at inVentiv Health Clinical, Outcome Sciences, ICON Clinical Research (formerly Ovation Research Group), and MetWorks, Inc.
Education
Matthew holds a Bachelor of Arts in Sociology from Boston University.
Jackie Vanderpuye-Orgle, PhD
Parexel International, Billerica, MA, USA
Jackie is a health economist with over 20 years of progressive experience in the application of econometric principles and statistical analysis to both clinical and real-world data. In her role at Parexel, Jackie is responsible for providing technical expertise and oversight on network meta-analysis, advanced parametric modelling, predictive analytics, AI, and general statistical analysis workstreams. She focuses on identifying fit-for-purpose analytic solutions to leverage both clinical trial and real-world data to bring the right treatment to the right patient at the right time.
1:15 PM - 2:15 PM
Meditation Session
Meditation for Mental Clarity
Level: Introductory
This workshop is open to both novice and advanced practitioners where we integrate meditation with scientific insights, tailored for attendees of our scientific conference. Participants will acquire practical, evidence-based strategies for stress management and the enhancement of mental clarity. This session will highlight the fascinating connection between the breath and emotions and how we can leverage the former for managing the latter. Attendees will also experience guided meditation, specifically designed to induce mental focus. The workshop will conclude with a Q&A session, encouraging an exchange of questions and personal reflections on the meditative practice.
Leaders: The session will be led by Dr Jag Chhatwal and Dr. Rachael Fleurence. Dr. Chhatwal is an associate professor at Harvard Medical School and director of the Institute for Technology Assessment at Massachusetts General Hospital. He has been practicing meditation for two decades and is a certified meditation instructor with the Art of Living with 1000+ hours of training. Dr Fleurence is a senior advisor at the National Institutes of Health. She previously served at the White House on the launch of a national elimination plan for Hepatitis C. She has been practicing meditation for over 15 years.
Member Group Meetings
ISPOR Statistical Methods in HEOR Special Interest Group Open Meeting
Leadership members from the Statistical Methods in HEOR Special Interest Group will present upcoming activities, volunteer opportunities, and lead an open discussion with attendees who will share their suggestions, ideas, and feedback for future projects.
1:30 PM - 2:30 PM
ISPOR New Professional Session
Charting Your Career Journey – Navigating Transitions and Setting Goals
For new HEOR professionals starting out in their careers, traversing a new landscape could seem quite daunting. The successful HEOR professional will need to be dynamic, skilled in navigating transitions and adapting to change, all the while maintaining a vision for their career end goals. This session will feature a diverse panel of stakeholders who will share experiences on how to successfully chart out a new course for their careers, adapting to the changing HEOR landscape and setting and managing career goals.
Moderators
Irwin Tran, PharmD, MS, ACC
NextLeader Coaching, Alameda, CA, USA
Speakers
Blythe Adamson, PhD, MPH
Flatiron Health, New York, NY, USA
Blythe Adamson is an epidemiologist and economist accelerating the development of valuable medicines and health policy. She serves as Head of Outcomes Research and Evidence Generation at Flatiron Health in New York. Dr. Adamson advances artificial intelligence applications of natural language processing and machine learning in the curation of electronic health records for real world evidence with a team of engineers, scientists, and clinicians. She designs treatment comparative-effectiveness studies, codes mathematical models of disease, and delivers research for global health technology assessment. Dr. Adamson served as a former member of the White House COVID Task Force on Healthcare Resilience while lead pandemic data scientist in the West Wing. She is an Affiliate Professor at the University of Washington in the Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute. Dr. Adamson has held roles at the Bill and Melinda Gates Foundation Institute for Disease Modeling, Infectious Economics, the NIAID HIV Vaccine Trials Network, and Fred Hutch Cancer Center. She continues to serve as a leader in the International Society for Health Economics and Outcomes Research (ISPOR). Dr. Adamson holds degrees in pharmaceutical economics, public health epidemiology, and microbiology. She enjoys writing about the economics of infectious diseases at www.blytheadamson.com.
Sanket Shah, MBBS, PhD
Jazz Pharmaceuticals, Gaithersburg, MD, USA
Daniel Simmons, PharmD, MS, RPh
AstraZeneca, Frederick , MD, USA
1:45 PM - 2:45 PM
Breakouts: IP, WS, & OBS
A Workshop on Systematically Collecting, Integrating, and Measuring the Impact of Patient Engagement Across Research
Digital Conference Pass
Level: Introductory
PURPOSE: ISPOR's patient-centered special interest group defines 'patient engagement in research' as “the active, meaningful, and collaborative interaction between patients and researchers across all stages of the research process, where research decision-making is guided by patients’ contributions as partners, recognizing their specific experiences, values, and expertise.” In the past, this definition was largely aspirational. This workshop will introduce participants to real-world examples of how global pharmaceutical and research organizations operationalize meaningful patient engagement and measure its' impact across research portfolios. Topics covered include strategies for collecting high-quality patient experience data (PED), partnering with cross-functional teams to integrate PED across key decisions, and measuring the impact of patient engagement across drug and core outcome set development.
DESCRIPTION: Elisabeth Oehrlein will moderate the session, introduce key terminology, and engage the audience to consider how the processes, tools, and lessons learned from the case studies can apply to their work. Beyza Klein will describe the Novartis approach of collaborating with the patient community; building a thorough understanding of patients' life experiences to guide decision-making; and establishing standardized methods to measure the impact of patient engagement efforts throughout a product's lifecycle. She will outline how pharmaceutical companies and other stakeholders can contribute to creating a patient-centered health ecosystem by adopting systematic approaches to measure and communicate the outcomes of their patient engagement initiatives. Stacie Cavallaro will describe how BioMarin adapted the National Health Council's Patient Experience Mapping Toolkit to standardize a process for capturing and utilizing patient and caregiver insights starting early in clinical development. Jennifer Bright will describe the International Consortium for Health Outcomes Measurement’s (ICHOM) approach to engaging patient representatives to identify the outcomes that matter most to patients, develop core outcome sets, and drive adoption and reporting of these measures worldwide to drive value-based healthcare.
Discussion Leaders
Elisabeth Oehrlein, PhD, MS
Applied Patient Experience, LLC, Washington, DC, USA
Discussants
Jennifer Bright, MPA
ICHOM, Alexandria, VA, USA
Jennifer Bright, MPA (she/her) is President, International Consortium for Health Outcomes Measurement (ICHOM), a nonprofit that works with patients and clinical experts worldwide to define and deploy standardized tools to measure outcomes that matter to patients. Prior, she held executive roles at the Innovation and Value Initiative (IVI), focusing on advancing patient-centricity, transparency and equity in value assessment.
Jennifer is President, Momentum Health Strategies®; Board Chair, Mental Health America; and editor, American Journal of Accountable Care and the Journal of Patient Experience. She holds degrees in political science and public administration from Trinity University and George Washington University.
Stacie Cavallaro, BS
BioMarin Pharmaceutical, Alpharetta, GA, USA
Beyza Klein, MA
Novartis, Riehen, BS, Switzerland
Beyza serves as Global Patient Engagement Director at Novartis. She is experienced patient advocacy lead with a demonstrated history of working in the pharmaceuticals industry on many levels; country, region and global. Skilled in External Stakeholder Management, Product Lifecycle Strategy, Analytical Skills, Patient Engagement and Insights, Market Research and Patient Centric Drug Development, External Communication, Crisis Management. Leadership level strong patient advocacy professional with a MA focused in Sociology & Social Policy from University of Sydney.
Valuing Obesity Treatments: An Application of Generalized Cost-Effectiveness Analysis (GCEA)
Digital Conference Pass
Level: Intermediate
PURPOSE:
The obesity treatment landscape is rapidly evolving with recent approvals of highly effective therapies (e.g., glucagon-like peptide-1 receptor agonists) and a robust pipeline advancing products testing different mechanisms of action. These current and future treatments have the potential to alleviate the significant societal burden of obesity, improving quality of life for patients and generating long-term savings. However, insurance coverage for these innovative therapies remains limited currently. Understanding the value of these treatments from different stakeholders is essential to informing healthcare decision-making in obesity. This workshop will discuss the value of obesity treatments from the perspectives of different stakeholders in the healthcare system, identify similarities and differences in value elements across stakeholders, and then demonstrate how to incorporate stakeholder priorities using generalized cost-effectiveness analysis (GCEA) to assess the value of obesity treatments from a societal perspective.
DESCRIPTION:
Tess Cameron will first provide an overview of the treatment landscape, describe the potential benefits, and highlight the importance of understanding the societal value of obesity treatments (10 minutes). Tom Hubbard will outline the key value elements from the perspectives of patients and employers identified from extensive stakeholder engagement and research conducted by NEHI (10 minutes). Mike Sherman, former Chief Medical Officer with Point32Health, a major payor, will discuss the value elements from a payor perspective, and key considerations and challenges in coverage and reimbursement decision-making (10 minutes). Built on the value elements from different stakeholders, Alison Sexton Ward will illustrate how to apply GCEA to incorporate stakeholder priorities, and discuss implications for decision-making (10 minutes). The workshop will end with Q&A with the audience (20 minutes). Polling questions will be used throughout to elicit key challenges in modeling obesity treatments and prioritized approaches in modeling. This workshop will be relevant for a wide range of stakeholders including patients, employers, payors, modelers, and value assessors.
Discussion Leaders
Tess Cameron, BA
RA Capital Management, Boston, MA, USA
Tess Cameron is a Principal on the Investment Team at RA Capital Management. Tess works on both public and private investments and serves on several company boards. Previously, Tess held finance leadership roles at Foghorn Therapeutics, Wave Life Sciences, and Biogen. Prior to joining biotech/pharma, she was a specialist in the corporate finance team at McKinsey & Co, focused on corporate transactions. Tess has a BA with a double major in Economics and Peace & Conflict studies from the University of Toronto, Canada.
Discussants
Thomas Hubbard, MPP
Network for Excellence in Health Innovation, Westborough, MA, USA
Tom Hubbard, Senior Vice President
NEHI-Network for Excellence in Health Innovation
Boston, MA
Tom Hubbard manages projects on pharmaceuticals, vaccines, diagnostics and related policy at NEHI, a non-profit and non-partisan think tank organized around the shared interests of stakeholders across key sectors of U.S. health care. NEHI's mission is to advance adoption of innovations that improve health, enhance the quality of health care and achieve greater value for money spent.
His most recent NEHI reports focus on innovations in Alzheimer's disease diagnostics, value-based payment for obesity treatment, Medicare vaccination policy, multi-cancer early detection tests, and patient-payer communication over coverage of novel therapies.
He previously served as Vice President at the Massachusetts Technology Collaborative, as an economic development aide to Governor Michael Dukakis in Massachusetts and then to U.S. Senator John Kerry, and as community development director for the City of Gardner MA. He is a graduate of Harvard College and received his MPP from the Harvard Kennedy School of Government.
Alison Sexton Ward, PhD
USC Schaeffer Center, EL DORADO HILLS, CA, USA
Alison Sexton Ward is an economist with extensive experience on healthcare policy and pharmaceuticals. Her research has focused on economic valuations of various therapies and drug classes, and the economic implications of drug pricing policies. Alison has spent most of her career working with pharmaceutical manufacturers, government agencies, and non-profit health organizations on topics ranging from drug pricing policies, social value of new treatments, and long-term survival prediction. Prior to joining USC, she worked as a senior associate with the Brattle Group where she designed and led economic analysis in support of litigation on health-related issues. Alison joins the Value of Life Science Innovation (VLSI) team led by Karen Van Nuys.
She holds a Ph.D. in Applied Economics from the University of Minnesota, a M.S in Ag and Resource Economics from the University California Davis, and a B.S. in Managerial Economics from the University of California Davis.
Michael Sherman, MD, MBA, MS
RA Ventures, Boston, MA, USA
Dr. Michael Sherman serves as a Venture Partner at RA Ventures and on the faculty of the Dept of Population Medicine at Harvard Medical School. Prior to joining RAVen, he served as EVP and chief medical officer at Point32Health, formed by the merger of Harvard Pilgrim Health Care and Tufts Health Plan, two leading not-for-profit health plans insuring over 2 million members. Prior to the merger, Dr. Sherman served as chief medical officer of Harvard Pilgrim Health Care, nationally recognized for both innovation and quality. He has been widely recognized as a pioneer in bringing advances in transformational therapeutics, precision medicine, and digital therapeutics to Point32Health’s members by launching structured pilots that balance access and affordability and also generate real world evidence. Dr. Sherman holds board appointments with the Personalized Medicine Coalition, the Harvard Business School Healthcare Initiative, and NEHI (Network for Excellence in Health Innovation). Prior to joining Harvard Pilgrim in 2011, Dr. Sherman held executive roles at Humana, UnitedHealth Group, Thomson Medstat (now part of IBM Watson Health), and Da Vita, as well as several early stage companies. Before pursuing an M.B.A. at Harvard Business School, Dr. Sherman received his M.D. from Yale and practiced as a cardiac anesthesiologist. He also holds a B.A. in Anthropology and Natural Sciences and an M.S. in Biomedical Anthropology from the University of Pennsylvania.
“Whole Health” Value Assessment: Universal Survey Framework for Integrating Patient Experience Data in Health Technology Assessment
Digital Conference Pass
Level: Introductory
PURPOSE:
Value and health technology assessment (V/HTA) bodies have established opportunities for patients and other stakeholders to contribute to assessments. These opportunities have revealed gaps in data and evidence to support patient-centered, “whole health” V/HTA.
To overcome gaps, patient groups, individually or in partnership with V/HTA bodies, have conducted patient surveys to inform reviews, providing information about patient experiences with treatment not collected in clinical trials or claims data, such as indirect costs or caregiver burden. These surveys often represent the best available data, reduce data gaps, and advance V/HTA centered on patients' experiences, perspectives, and needs. However, there is often discordance between the information submitted to health economic modelers and the information collected in patient experience surveys. There can also be a lack of understanding of how survey data impacts model outputs.
This workshop will present a disease-agnostic survey template, highlight the merits of a universal survey template, consider relevance to specific economic modeling decisions, and explore modifications to drive meaningful adoption and use in real-world settings.
DESCRIPTION:
- Kimberly Westrich will moderate the session, engage the audience with interactive polls and feedback opportunities, and frame the policy context through a real-world case example using published reviews in multiple sclerosis.
- Kayleigh Majercak will introduce a disease-agnostic survey template that has been co-developed with the patient community, guided by V/HTA stakeholder input. She will describe the development and testing among people with asthma, multiple sclerosis, and colorectal cancer to inform the standardized, patient-centric template.
- Joe Vandigo will highlight how the template can contribute to V/HTA by quantifying insights on concepts important to patients and can inform the selection of the value elements and structure of the assessments.
- Brett McQueen will discuss the relevance of the survey template in the context of specific modeling decisions and suggest modifications.
Discussion Leaders
Kimberly Westrich, MA
National Pharmaceutical Council, Herndon, VA, USA
Kimberly Westrich, MA, is the Chief Strategy Officer of the National Pharmaceutical Council (NPC), which sponsors and conducts research on health policy issues related to the development and use of innovative biopharmaceuticals to improve the health of patients. Ms. Westrich leads several research initiatives across NPC’s portfolio, including employer-sponsored insurance. She has published extensively on issues related to value assessment frameworks, patient-centered formulary and benefit design, value-based contracts, quality performance measurement, and accountable care organizations.
Discussants
Kayleigh Majercak, MS
University of Maryland School of Pharmacy, Baltimore, MD, USA
Kayleigh Majercak is a researcher within the Pharmaceutical Health Services Research department at the University of Maryland Baltimore (UMB). Her research interests include patient-centered outcomes research with a focus on patient-reported outcomes and patient experience data as well as mixed-methods research. In her previous role, Kayleigh worked as a data analytics consultant at Humana, Inc. Using claims data, her work focused on informing market leaders with population-level analytics to support Humana’s Bold Goal Communities’ initiative towards improving members’ health. At UMB, her research and the focus of her PhD dissertation was co-developing a disease-agnostic survey template to inform a standardized approach to fill patient-centered evidence gaps in US value assessment. Her project was supported by the National Health Council as part of the Pre-Doctoral Fellowship. Kayleigh graduated with a Master of Science degree in pharmaceutical evaluation and policy from the University of Arkansas for Medical Sciences and earned her PhD in Pharmaceutical Health Services Research from UMB.
Robert McQueen, PhD
University of Colorado, Denver, CO, USA
R. Brett McQueen is an Associate Professor at the University of Colorado (CU) Skaggs School of Pharmacy and Pharmaceutical Sciences, and member in the Center for Pharmaceutical Outcomes Research. His research interests include decision-analytic modeling applications and methodology, applied microeconometrics in health, and novel value assessment methods. Brett has current funding in micro-costing health interventions, evaluating performance-based risk sharing agreements, estimating patient and payer preferences for various pharmaceuticals, and novel value assessment methods. He is the course director for “Pharmaceutical Economics and Policy Analysis” in the Pharmaceutical Outcomes Research PhD program at CU.
Joe Vandigo, MBA, PhD
Applied Patient Experience, LLC, Greensburg, PA, USA
Revolutionizing Regulatory Pathways: Unleashing the Power of Real-World Evidence, Adaptive Trials, and Synergistic Collaboration for Expedited FDA Device Approval, Breakthrough Designation, and CMS Reimbursement
Digital Conference Pass
Level: Intermediate
PURPOSE:
This panel will explore the use of real-world evidence (RWE), adaptive trials, and enhanced collaboration between the US Food and Drug Administration (FDA) and the US Centers for Medicare and Medicaid Services (CMS) to expedite FDA device clearance, breakthrough designation, and subsequent reimbursement decisions.
DESCRIPTION:
The proposed Transitional Coverage for Emerging Technologies (TCET) pathway at CMS for Breakthrough Devices supports both improved patient care and innovation by providing a clear, transparent, and consistent coverage process while maintaining robust safeguards for the Medicare population. US National Institutes of Health [NIH] (Dr. Arnold), FDA (Dr. Kelly), CMS (Dr. Farmer), and academic policy (Dr. Neumann) experts will share insights on leveraging these approaches to accelerate patient access to innovative medical devices. Dr. Arnold will discuss some of the patient cohort concerns of using RWE in fit-for-purpose trials to enable inferences about healthcare resource utilization in support of healthcare decision-making. Dr. Kelly will highlight key features of the FDA Breakthrough Devices Program. Dr. Farmer will provide a history of the CMS TCET pathway to reimbursement. Dr. Neumann will argue that CMS, in light of its growing role in advancing TCET, Coverage with Evidence Development (CED) approaches and Medicare drug price negotiations under the Inflation Reduction Act, is inescapably assuming a more active role as a health technology assessment (HTA) agency. The panel will address the opportunities and challenges in enhancing collaborations between the FDA, CMS, and HEOR professionals to expedite access to innovative devices while ensuring cost-effectiveness. Pre-program knowledge polling and post-program Q&A will be featured.
Discussion Leaders
Renée Arnold, PharmD, RPh
National Institutes of Health, Miami Beach, FL, USA
Renée JG Arnold is currently Entrepreneur-in-Residence (EIR), HEOR, NHLBI (NIH) and EIR, Biohealth Innovation, Inc, New York, NY; Adjunct Full Professor, Master of Public Health Program, Department of Environmental Medicine and Public Health, Icahn School of Medicine at Mount Sinai, New York, NY, USA, where she has developed and teaches the pharmacoeconomics coursework. She is also president & CEO of Arnold Consultancy & Technology, LLC where she oversees outcomes research and develops affiliated software for pharmaceutical and federal government programs. Her special interest in evidence-based health derives from her research that deals with use of technology to collect and/or model real-world data for use in rational decision making by healthcare practitioners and policy makers. Dr. Arnold completed her undergraduate training at the University of Maryland and received her Doctor of Pharmacy degree from the University of Southern California in Los Angeles. She also completed a one-year post-doctoral residency at University Hospital in San Diego/University of California at San Francisco School of Pharmacy. Dr. Arnold was previously vice president of HEOR at Quorum Consulting, Inc./Navigant Consulting; principal of IMS Health (IQVIA); and president and co-founder of Pharmacon International, Inc. Center for Health Outcomes Excellence.
Dr. Arnold is a founding member of ISPOR and is the head of both the ISPOR Distance Learning Program, as well as the former head of the Open Source Models Special Interest Group. She is an author/co-author of numerous articles, book chapters, and books in the areas of pharmacology, pharmacoeconomics, and cost-containment strategies.
Discussants
Steven Farmer, MD, PhD
Centers for Medicare & Medicaid Services, Baltimore, MD, USA
Dr. Steven Farmer serves as Chief Strategy Officer in the Coverage and Analysis Group (CAG) at the Centers for Medicare & Medicaid Services (CMS). He joined CMS in 2017 as a Senior Advisor in the Center for Medicare and Medicaid Innovation where he assisted with development and refinement of value-based payment programs, with a particular emphasis on the Bundled Payments for Care Improvement Advanced model. He moved to the Coverage and Analysis group in 2019 where he leads an effort to streamline and accelerate evidence-based coverage policy development. He is a practicing non-invasive cardiologist.
Douglas Kelly, MD
Food and Drug Administration, Silver Spring, MD, USA
Peter Neumann, ScD
Tufts Medical Center, Boston, MA, USA
Peter J. Neumann, Sc.D., is Director of the Center for the Evaluation of Value and Risk in Health (CEVR) at the Institute for Clinical Research and Health Policy Studies at Tufts Medical Center, and Professor of Medicine at Tufts University School of Medicine. He is the founder and director of the Cost-Effectiveness Registry, a comprehensive database of cost-effectiveness analyses in health care. Dr. Neumann has written widely on the role of clinical and economic evidence in pharmaceutical decision making and on regulatory and reimbursement issues in health care. He served as co-chair of the 2nd Panel on Cost-Effectiveness in Health and Medicine. He is the author or co-author of over 300 papers in the medical literature, and the author or co-author of 3 books: Using Cost-Effectiveness Analysis to Improve Health Care (Oxford University Press, 2005) ; Cost-Effectiveness in Health and Medicine, 2nd Edition (Oxford University Press, 2017); The Right Price: A Value-Based Prescription for Drug Costs (Oxford University Press, 2021). Dr. Neumann has served as President of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). He is a member of the editorial advisory board of Health Affairs and the health policy advisory board for the Congressional Budget Office. He has also held several policy positions in Washington, including Special Assistant to the Administrator at the Health Care Financing Administration. He received his doctorate in health policy and management from Harvard University.
Bridging Real-World Data and Regulatory Decision-Making: The Role of AI in External Control Arm Development
Digital Conference Pass
Level: Intermediate
PURPOSE:
The workshop aims to critically examine recent guidelines on external control arms (ECA), with a specific focus on integrating AI to enhance the utilization and efficacy of Real-World Data (RWD) in ECA development. The discussion intends to offer insightful perspectives and practical applications to advance the methodologies in clinical research settings. DESCRIPTION:
NICE has pioneered among European HTA organizations by introducing guidelines for implementing ECAs within its 2022 RWE framework. In early 2023, The US FDA released guidance for the utilization of ECA, emphasizing the use of patient-level data. Concurrently, China's CDE unveiled a draft outlining the design and framework for real-world drug research, with extensive discussions in ECA development. In parallel, the rapidly advancing generative AI, epitomized by Large language models (LMMs) and ChatGPT, has initiated a paradigm shift in the healthcare landscape. In this workshop, Dr. Arora will provide an overview of the guidelines, address common challenges of utilizing RWD for ECA in the US, and review a case study highlighting potential benefits of AI in ECAs (15’). Dr. Yang will explore the guidelines and challenges associated with utilizing RWE for ECA in China, including a case study in rare diseases (15’). Dr. Wang will delve into the advancements in generative AI and LMMs, discussing her recent research on ECAs created from case reports using generative AI in the fields of rare disease and oncology (15’). All panelists will analyze the areas of alignment and divergence based on the audience poll, steering the discussion of opportunities, challenges and future trajectories of integrating AI and RWD for regulatory submissions (15’) The workshop is designed to be particularly relevant for professionals in RWE research, clinical development, and regulatory sectors. It aims to elucidate the significant potential contribution of AI in enhancing the generation and application of RWE.
Discussion Leaders
Paul Arora, PhD
University of Toronto, Toronto, ON, Canada
Paul is an epidemiologist and status assistant professor at the University of Toronto in the Dalla Lana School of Public Health. He has over 15 years of experience in conducting and leading evaluations in public health and clinical epidemiology. He holds PhD in epidemiology and has a strong background in biostatistics and scientific writing, which he applies to generate real-world evidence for health outcomes and policy decisions. He was a co-founder and CEO of Lighthouse Outcomes, a consulting firm that specialized in advanced analytics for clinical epidemiology research, which was acquired by Cytel in 2020. At Cytel, he served as a vice president and led the advanced analytics group in the real-world evidence division, where he worked on the application of cutting-edge epidemiologic methods and machine learning to health outcomes problems. He also continues to teach and mentor students and researchers in the Division of Epidemiology, where he has published more than 50 peer-reviewed scientific articles and developed software for Bayesian network meta-analysis of clinical trials. He is passionate about advancing the field of epidemiology and improving health outcomes through rigorous and innovative research.
Discussants
Xiaoyan Wang, PhD
Intelligent Medical Objects, Westport, CT, USA
Dr. Xiaoyan Wang serves as IMO’s Chief Scientist. She focuses on translating scientific insights and health intelligence into partnerships and programs that improve healthcare outcomes for patients, providers, and researchers.
Dr. Wang has spent two decades committed to driving innovations in healthcare AI modeling and transforming health care with data intelligence. She published hundreds of papers in NLP, AI modeling and data intelligence in health care and life sciences. She was most recently VP of Healthcare Analytics and Informatics and VP of Biopharma Solutions at GeneDx Mount Sinai Genomics Inc, where she led the development of clinical evidence generation platforms and clinical research in oncology, immunology, cardiovascular, respiratory, and rare diseases. Before joining the biopharma industry, she was a faculty member and principal investigator at the University of Connecticut, UConn Health Center, and Mount Sinai Health Systems bridging research, health services, and teaching.
She holds a BA in Biochemistry from Shandong University, a MA in Genetics from the University of Kansas, and a Ph.D. in Biomedical Informatics and NLP from Columbia University School of Medicine.
Mei Yang, Ph.D.
Happy Life Technology, Short Hills, NJ, USA
Dr. Mei Yang is now the VP of iHS Global from Happy Life Technology, an affiliate of Yidu Tech Inc, which provides leading data science and real-world evidence generation for life science companies.
Mei received her PhD in Biostatistics from Boston University. She started her industry career in AbbVie, where she worked as the Global Health Economics and Outcomes Research (GHEOR) lead to support the launch of Humira in treating patients with inflammatory bowel disease. Subsequently, Mei worked in Daiichi Sankyo in the pain area, where she gained plenty experience on Patient Reported Outcomes (PROs) and Phase III clinical development. She joined Merck in 2015 as a Director of GHEOR, leading a cross-functional team to support late phase clinical development and drug launch in cardiovascular and diabetes area. She has developed a robust understanding of physicians, patients, payers, and their decision making systems; implemented innovative approaches to healthcare research and analytics; and became a results-oriented strategist and an expert in providing scientific insights and improving business impact through the utilization of value-based evidence and access strategies.
Since 2009, Mei has been focusing on real world evidence (RWE), health economics, and market access in the pharmaceutical industry and became a lead with deep insights into evolving health care delivery and reimbursement systems and the evidentiary needs of diverse customer groups across major global markets. Mei has published about 40 articles in high tier medical journals in various disease areas and is continuously publishing new researches. With over 11 years of experience in HEOR and RWE, Mei is devoted to this globally evolving environment and has broad interest in Big Data, AI technology, healthcare policy, and market access.
Podium Session
Pricing and Regulatory Policies: Implications for Pharmaceutical Manufacturers
Digital Conference Pass
Moderator
Maja Kuharic, MPharm, PhD, Msc, MPharm
Northwestern University Feinberg School of Medicine, Evanston, IL, USA
Maja Kuharic, MPharm, MSc is a Research Associate at the Department of Medical Social Sciences at Northwestern University Feinberg School of Medicine. Her research interests involve measurement and evaluation of health-related quality of life and health outcomes in patients and caregivers and the evaluation of the safety, effectiveness and value of health care interventions. Maja received her master’s degree in Health Economics and Pharmacoeconomics at Pompeu Fabra University in Barcelona, Spain, her master’s degree in Clinical Pharmacy, and a bachelor’s degree in Pharmacy from the University of Zagreb, Croatia.
P35: Are Proposals for a Scheme to Stimulate Investment in Critically Needed New Antimicrobials Fit for Purpose?
2:15PM - 2:30PM
Hofer M, Hampson G
The Office of Health Economics, London, UK
OBJECTIVES: Antibiotics are essential for modern medicine, but antimicrobial resistance is reducing their effectiveness, causing millions of deaths globally. New antibiotics are urgently needed, but their development is hindered by market failure. A pull incentive in the form of a volume-delinked subscription approach has been suggested to overcome this market failure, providing compensation regardless of sales volume.
In 2023, NHS England published proposals for the implementation of this type of subscription model, specifying how antibiotics will be scored to determine the size of the subscription payment. The proposals are of international significance as they are the first of their kind, providing decision-makers globally with insight into how effective incentives could be designed. This research examines the likely success of the proposals.
METHODS: We interviewed (potential) investors (n=9) in the antibiotics space (investors are key stakeholders as their buy-in is critical to unlocking the funds for the development of antibiotics) and tested how new antibiotics are likely to be valued under the new system, including examining through interviews whether expert clinicians consider the scoring system to be appropriate.
RESULTS: We found that realistic dummy products can be constructed from the pipeline that would achieve annual payments of £5-15m. The highest-scoring dummy product, an innovative antibiotic targeting gram-negative pathogens and resistance mechanisms, with both oral and IV formulations, just missed the top (£20m) value band. All investors agreed that the UK proposals are hugely significant. However, they reported the scheme as proposed is not sufficiently predictable and does not offer sufficient financial incentive to stimulate research in this area.
CONCLUSIONS: We recommend changes to the proposals that will ensure critically needed new antimicrobials will be adequately incentivised. The recommendations will be critical for international researchers and policymakers in designing local models, in pursuit of a global pull incentive sufficient to stimulate much-needed investment in novel antimicrobials.
P34: National Costs of Prescription Drug Market Exclusivity Extensions
2:00PM - 2:15PM
Hong D1, Tu S2, Beall R3, Russo M4, Rome B5, Kesselheim AS1, Sarpatwari A1
1Brigham and Women's Hospital/Harvard Medical School, Boston, MA, USA, 2West Virginia University, Morgantown, WV, USA, 3University of Calgary, Calgary, AB, Canada, 4the Ohio State University, Columbus, OH, USA, 5Brigham and Women's Hospital, Boston, MA, USA
OBJECTIVES: Brand-name drugs in the US are sold at high prices during market exclusivity periods defined by their patents, which block entry of competing generics. Drug manufacturers routinely attempt to extend market exclusivity periods using strategies such as obtaining numerous additional patents on aspects of the drugs other than their active ingredients. We estimated excess US spending associated with market exclusivity extensions.
METHODS: We focused on four drugs from distinct disease areas that experienced generic entry between 2014-2018 as case studies: imatinib (cancer), glatiramer (multiple sclerosis), celecoxib (arthritis), and bimatoprost (glaucoma). Cost data was retrieved from Merative MarketScan (commercial plans), Medicare fee-for-service, and Medicaid, and adjusted for rebates estimated in the SSR Health Net Price Database. The extended market exclusivity was calculated as the time between expiration of the key composition of matter patent and first generic marketing. We used segmented linear regression analyses to compare estimated spending under the status quo with estimated spending in the absence of market exclusivity extensions. Analytic weights were used to estimate national spending.
RESULTS: The market exclusivity length of the drugs ranged from 14 (bimatoprost) to 18 (glatiramer) years, including an extended market exclusivity of 7 (celecoxib/imatinib) to 13 (glatiramer) months. In the absence of extended market exclusivity, net spending would have decreased by $715 million411 million in commercial plans and $304 million in Medicare—including $11 million for bimatoprost, $33 million for glatiramer acetate, $270 million for imatinib, and $401 million for celecoxib. Because of substantial statutorily required rebates on these drugs under the Medicaid Drug Rebate Programs, spending in Medicaid would not have decreased.
CONCLUSIONS: Policies to curb strategies to promote timely generic availability and avoid extending market exclusivity beyond the patent on a drug’s active ingredient can result in substantial savings for commercial plans and Medicare.
P36: Unsupported Policy Information? The Institute for Clinical and Economic Review's Unsupported Price Increase Reports
2:30PM - 2:45PM
Ha J1, Westrich K2
1Cencora, Conshohocken, PA, USA, 2National Pharmaceutical Council, Herndon, VA, USA
OBJECTIVES: The Institute for Clinical and Economic Review (ICER) positions its Unsupported Price Increase (UPI) reports as relevant policy tools. We aimed to identify trends in manufacturer evidence submissions and ICER’s rejection/acceptance decisions.
METHODS: A codebook was developed to compile and categorize manufacturer-submitted evidence and ICER’s reasons for rejecting or accepting evidence across 5 national UPI reports published from 2019 to 2023. We identified submission and rejection trends, as well as study characteristics for accepted evidence.
RESULTS: Manufacturers submitted evidence for 42 of the 57 drugs reviewed across the 5 reports, totaling 1,237 pieces of evidence and averaging 29 pieces per drug. The total evidence per UPI report and average pieces of evidence per drug declined over time, ranging from n=67 pieces per drug in 2019 to n=12 in 2023. Overall, 96% of evidence submissions were rejected by ICER, with a slight downward trend, ranging from 99% in 2019 to 91% in 2023.
Only 21 distinct randomized controlled trials were accepted by ICER, spanning phases 2-4, with the majority double-blinded. In 2019, ICER described the accepted evidence using a single category: longer-term data with improved outcomes (n=5). In subsequent years, ICER continued to introduce new descriptive categories—including evidence that supported Food and Drug Administration (FDA) label expansion for a new (n=5) or existing (n=4) indication, extended the evidence base to new populations or outcomes excluded in previous trials (n=3), supported FDA accelerated approval (n=2), supported FDA approval requirements for longer-term safety data (n=1), and strengthened the existing evidence base and guideline recommendations (n=1).
CONCLUSIONS: Manufacturers appeared to become increasingly selective over time with evidence they submitted to ICER’s UPI reports, while ICER accepted slightly more evidence with each UPI report. However, our findings demonstrate that ICER continues to reject the majority of UPI evidence submissions (96%), calling their policy-relevance into question.
P33: Inflation Reduction Act Data Collection Provisions
1:45PM - 2:00PM
Vossen C1, Mahony KE2, Bianic F3, Guelfucci F4, McCaslin T5
1Syneos Health, Amsterdam, NH, Netherlands, 2Syneos Health, New York, NY, USA, 3Syneos Health, Paris, Ile-de-France, France, 4Syneos Health, Montrouge, Ile-de-France, France, 5Syneos Health, La Plata, MD, USA
OBJECTIVES: The enactment of the Inflation Reduction Act (IRA) in 2022 empowers Medicare (CMS) to negotiate directly with pharmaceutical companies for high-expenditure, sole-source medications lacking generic or biosimilar alternatives. Non-compliance with the IRA’s stringent data collection mandates can lead to significant penalties, including civil fines and a substantial excise tax (65%-95% of US sales).
METHODS: We meticulously analyzed IRA guidelines and processes, focusing on the specifics of data collection (including elements and sources), submission procedures (utilizing designated portals and templates), audit details (timing and particularities), implications of noncompliance, confidentiality issues, and associated costs of compliance.
RESULTS: Manufacturers of selected drugs are required to electronically submit comprehensive data to CMS in standardized formats, encompassing: 1. Research and development (R&D) expenditures; 2. Unit production and distribution costs; 3. Budget impact analyses; 4. Comparative clinical effectiveness (including real-world evidence) and therapeutic value assessments, both general and specific to Medicare subgroups. Internal data (points 1-3) undergo independent audit verification, whereas clinical data (point 4) derive from manufacturer-conducted studies. The Department of Health and Human Services (HHS) will verify data for accuracy, completeness (for 1-3), and relevance (for 4), reserving the right to request supplemental details. Public disclosure will include R&D costs (detailed by phase and funding source); unit costs (broken down by region and dosage form/strength); list prices; and clinical effectiveness data. However, cost-effectiveness and budget impact data will remain confidential. Estimated compliance costs per drug are approximately $100,000 for R&D, $50,000 for production/distribution, and $35,000-$500,000 for clinical data. One estimate suggests price reporting requirements for the US market alone may cost ~$5 million per manufacturer.
CONCLUSIONS: In light of these intricate data requirements and the substantial cost of compliance, proactive strategic planning is crucial for manufacturers, starting as early as the potential selection phase, to ensure full and efficient compliance.
The Patient and Caregiver Perspective for Healthcare Decision-Making
Digital Conference Pass
Moderator
Tamas Agh, MSc, PhD, MD
Syreon Research Institute, Budapest, PE, Hungary
Tamás Ágh MD MSc PhD DrHabil graduated from the Semmelweis Medical University (MD, 2006), the Corvinus University of Budapest (MSc Physician-Economist, 2010), the Semmelweis University School of PhD Studies (PhD in Pharmacoeconomics, 2013) and the University of Pecs Doctoral School of Health Sciences (DrHabil in Health Sciences, 2020). He specialized in family medicine in 2010. Dr. Ágh is a principal researcher at the Syreon Research Institute and has been practicing as a medical doctor since 2006. He is also a research associate professor and the head of the Medication Adherence Research Group at the Center for Health Technology Assessment and Pharmacoeconomic Research at the University of Pécs. With a professional background spanning over 15 years, his areas of specialization in health economics and outcome research include medication adherence, patient-reported outcomes measures, health technology assessment, and evidence synthesis. He serves as the chair of the ISPOR Medication Adherence and Persistence Special Interest Group, chair-elect of the ESPACOMP International Society of Medication Adherence, and holds a leadership position in the European Network to Advance Best practices & technoLogy on medication adherencE (ENABLE) Cost Action. Dr. Ágh has co-authored about 50 peer-reviewed articles.
P37: Patients’ Perspectives on Muscular Dystrophy: Insights from Social Media Listening
1:45PM - 2:00PM
Dubey A1, Poddar S2, Shakira A3, Kumar J4, Shaikh J5, Hood D6
1Axtria, Bangalore, KA, India, 2Axtria, Berkley Heights, NJ, USA, 3Axtria, Hyderabad, Telangana, India, 4Axtria, Gurugram, Haryana, India, 5Axtria, Hyderabad, AP, India, 6Axtria, Gales Ferry, CT, USA
OBJECTIVES: A comprehensive understanding of the real-world issues with muscular dystrophy (MD) is essential to improve patient care. There is paucity of data on the patients’ perspective of how MD impacts daily living. This study aimed to understand the patients’ perspective through social media listening.
METHODS: This retrospective study analyzed publicly available data from the r/Muscular Dystrophy subreddit on Reddit spanning January 2018 – November 2023. Natural Language Processing models were used to classify anonymized dataset into relevant posts pertaining to journey of MD patients. The processed data was categorized for further analysis into patient journey information such as symptoms, diagnosis, treatment, and quality of life (QoL).
RESULTS: A total of 802 posts from 511 members were identified on the MD subreddit. Gender information was discerned in 30% of the posts, with male/female distributions reported in 69%/31% of the posts, respectively. Commonly mentioned symptoms were muscle weakness/degeneration (30%), inability/difficulty to walk (25%), and chronic pain (16%) which affected the activities of daily living. Diagnosis was discussed in 28% of posts; of which 6% indicated individuals seeking diagnostic options. Treatment options were mentioned in nearly 18% of posts; out of which 45% discussed therapeutic interventions and 55% mentioned the use of physical therapies/adaptive equipment in conjunction with everyday activities for managing symptoms. Approximately half (53%) of the identified posts highlighted considerable impact on the patients’ emotional and physical well-being. Lack of disease awareness, accurate and timely diagnosis, and limited treatment options were identified as key unmet needs.
CONCLUSIONS: Insights from this study reported patients’ experiences and concerns of living with MD. Assessing patient-generated content revealed that MD imposes a substantial burden of impaired QoL that spans multiple aspects of daily living. Hence, it is critical to integrate patients' insights and address unmet needs when considering disease management including drug development.
P39: Quantifying Patient Preferences and Healthy-Time-Equivalents for Eosinophilic Esophagitis Treatments: A Stated-Preference Study
2:15PM - 2:30PM
Sutphin J1, Johnson R2, Strobel MJ3, Leiman D4
1Duke Clinical Research Institute, Mooresville, NC, USA, 2Duke Clinical Research Institute, Durham, NC, USA, 3American Partnership for Eosinophilic Disorders (APFED), Atlanta, GA, USA, 4Duke University School of Medicine, Durham, ND, USA
OBJECTIVES: Eosinophilic esophagitis (EoE) is a chronic allergic, inflammatory condition of the esophagus that can lead to scarring. Successful treatment involves resolution of symptoms and inflammation; however, treatments that resolve inflammation aren’t always accompanied by symptom improvement. Additionally, most treatments lack regulatory approval for EoE, and treatment mode and side effects vary widely, leading to complex treatment decisions. We conducted a stated-preference study to quantify patient preferences for features of EoE treatments in both utility space and healthy-time space. Healthy-time equivalents are a patient-centric construct based on time-tradeoff data that measures patients’ valuation of treatment features, as well as clinical outcomes of health interventions using the same metric.
METHODS: Preferences were elicited from adults with EoE using an online discrete-choice experiment (DCE). Attributes included months without symptoms: 10, 5 or 2 months, chance of no inflammation after one year: 90, 60, or 40%, regulatory approval for EoE, and treatment type: proton pump inhibitor, swallowed topical steroid, or biologic. Data were analyzed with mixed-logit models in utility space to elicit relative importance weights (RI) and healthy-time space to elicit equivalent values in months without symptoms and associated 95% confidence intervals (CI).
RESULTS: On average, patients (N=212, mean age: 46.6 years (SD 14.5), 56.7% female) highly valued improving the chance of eliminating inflammation from 40% to 90% (RI:4.6, CI:3.5-5.7) and extending symptom-free time from 2 to 10 months (RI:2.7, CI:1.9-3.5). Regulatory approval (RI:0.6, CI:0.3-0.9) and treatment type (RI:0.8, CI:0.2-1.4) were less important. In terms of healthy time, 90% chance of no inflammation equated to 15.1 months without symptoms (CI:12.7-17.6) while regulatory approval and treatment type were equivalent to 2.0 (CI:0.9-3.0) and 2.3 (CI:0.6-4.1) months, respectively.
CONCLUSIONS: Mixed-logit models of DCE data estimated in utility and healthy-time space demonstrated the value to patients of EoE therapies that address both symptoms and inflammation.
P38: Sources of Financial Support to Households Caring for Individuals with Duchenne Muscular Dystrophy: Results from a Caregiver Survey
2:00PM - 2:15PM
Innis B1, Jarvis J2, Renteria T3, Patel S1, Audhya IF1
1Sarepta Therapeutics, Inc., Cambridge, MA, USA, 2Medicus Economics, LLC, Culver City, CA, USA, 3Medicus Economics, LLC, Milton, MA, USA
OBJECTIVES: Duchenne muscular dystrophy, an X-linked neuromuscular disease, results in progressive muscle weakness, including loss of ambulation. Duchenne requires considerable indirect non-medical expenditures, particularly as the disease progresses across health states based on ambulatory status. Sources of financial support for Duchenne households have not been well described. This study sought to characterize financial benefits received by Duchenne households.
METHODS: Caregivers completed an online survey that gathered information on financial, social, and other aspects influenced by Duchenne. Caregivers were required to have provided care to a household member with Duchenne for >12 months and to have incurred a Duchenne-related expense within the past 5 years to accommodate disease progression. Potential sources of financial support to fund direct non-medical costs included 1) social program benefit payments; 2) compensation for caregiving; and 3) financial support for Duchenne-related home/vehicle, medical equipment, professional caregiving, and supportive therapy.
RESULTS: Caregivers (n=90) representing 106 individuals with Duchenne completed the survey. Benefit payments from social programs were received by 34% of households, primarily from Supplemental Security Income (68%), Social Security Disability Insurance (19%), and Social Security (10%), with an average total benefit of $8504 (SD: $6902) in the past year. Additionally, 26% of caregivers were compensated for caregiving, largely through Medicaid (48%) or In-Home Supportive Services (17%). Households with non-ambulatory individuals (n=60) were more likely to receive benefit payments (43%) or compensation for caregiving (32%) compared to households with individuals with greater ambulatory function (n=30; 17% and 13%, respectively). Across home and vehicle expenditures, households received an average of 24% of financial support from the US government, charitable foundations, community groups, family or friends, and independently raised funds.
CONCLUSIONS: Although the majority of direct non-medical costs are covered by households and most caregiving is informal, social resources play a crucial role in providing assistance to individuals with Duchenne and their families.
P40: Caregivers’ Stated and Revealed Preferences for Antipsychotic Medication Use Among Youth: A Discrete Choice Experiment of Benefit-Risk Trade-Offs
2:30PM - 2:45PM
Fu YH1, Amill-Rosario A1, Illoh O2, dosReis S1
1University of Maryland School of Pharmacy, Baltimore, MD, USA, 2FDA/University of Maryland Baltimore, Silver Spring, MD, USA
OBJECTIVES: Antipsychotic medication, commonly prescribed to manage aggressive behavior in youth, poses significant metabolic risks. Caregivers must make important benefit-risk decisions when considering use for their child. This study aimed to evaluate caregivers' benefit-risk preferences for antipsychotic medication use in their child and to assess congruence between stated and revealed preferences.
METHODS: A cross-sectional survey recruited 26 caregivers of a youth <18 years-old who had used or ever considered antipsychotic medication for their child from parent support groups in Maryland. We piloted a discrete-choice experiment comprising 9 choice tasks with 3 profiles. Each profile displayed 6 attributes (weight gain, diabetes risk, cholesterol risk, self-harm episodes per year, days without aggressive behavior, and hospitalizations per year) and one of 3 attribute levels. Participants selected the profile most acceptable to them and indicated willingness to use it. Conditional logit models estimated part-worth preference weights and relative attribute importance measured the contribution of each attribute to the overall preference. Finally, we calculated the proportion of the sample that would use the selected profile and estimated the total preference utility of the profile.
RESULTS: Caregiver participants (mean age 44 years-old) were mostly biological mothers (76%). Nearly half of youth (46%) had prior mental health hospitalization. The relative attribute importance revealed that participants prioritized benefits (i.e., hospitalizations (34%), days without aggressive behaviors (22%), and self-harm episodes (15%)) over the potential harms (i.e., diabetes risk (14%), weight gain (11%), and high cholesterol (4%)). Participants would trade off 2.21 hospitalizations per year to offset diabetes risk reduction from 4 to 2. Caregivers would use profiles yielding higher part-worth utility. Sixty-five percent would use the profile with the highest part-worth utility, featuring 4 days without aggression weekly, 2 self-harm episodes, and 2 hospitalizations annually.
CONCLUSIONS: Caregivers of youth requiring antipsychotics prioritized benefits over the potential harms, which aligned with their revealed preferences.
Breakouts: IP, WS, & OBS
I Have a Better QALY Than You: How to Make Sense of Alternatives to the QALY for Research and Policymaking
Digital Conference Pass
Level: Intermediate
ISSUE:
The quality-adjusted life year (QALY) has been a global mainstay for summarizing health gains from treatment and consideration of cost-effectiveness. But the QALY has been criticized by the academic community for its multiplicative nature, proportional tradeoffs between quality and length of life, and misalignment with stated preferences. In the policy sphere, critics argue that QALYs penalize those with severe or disabling diseases, and the QALY has been banned from use for decision-making by US government legislation. Researchers and policymakers have developed alternative measures in response, including the equal-value life year (evLY), healthy years in total (HYT), and the generalized, risk-adjusted QALY (GRA-QALY). Developers have published their rationale, postulated and/or summarized the effects of these measures in practice, and critiqued their competitors. To date, however, they have not been together to debate the merits of these measures and discuss how to move cost-effectiveness policy forward.
OVERVIEW:
Dan Ollendorf (moderator) (10 minutes) will set the stage by reviewing the history of anti-QALY sentiment and providing an overview of the current landscape for use of cost-effectiveness in American decision-making. Each panelist (10 minutes each) will discuss the evolution of their measure (Whittington: evLY; Lakdawalla: GRA-QALY; Basu: HYT), the novel or unique properties each possesses, advantages and disadvantages, and examples of postulated and/or actual use. Each panelist will also offer their critique of the other measures. In the remaining 20 minutes, the moderator will lead a debate among the panelists on the merits of these approaches for research, identification of key gaps in each approach as well as possible refinements to the measures, and ways in which policymakers, payers, and others in the US can usefully employ these measures for technology adoption and resource allocation decisions. The panel will conclude with a Q&A session that includes interactive polling to gauge audience preferences.
Moderators
Dan Ollendorf, PhD
Tufts Medical Center, Boston, MA, USA
Dan Ollendorf is Chief Scientific Officer and Director of Health Technology Assessment (HTA) Methods and Engagement at the Institute for Clinical and Economic Review (ICER), with responsibility for managing the organization’s internal health economics capacity and program of external collaboration, as well as collaboration with international HTA bodies and other stakeholders on key process and methods topics.
From 2007-2018, Dr. Ollendorf was Chief Scientific Officer for ICER, where he oversaw the broader HTA process and managed relationships with multiple stakeholders and research collaborators. From 2018-2023, Dr. Ollendorf served as the Director of Value Measurement & Global Health Initiatives at the Center for the Evaluation of Value and Risk in Health (CEVR) at Tufts Medical Center, where he focused on value assessment methods, drug pricing policy, and building international capacity for and interest in HTA. In addition to his current role at ICER, Dr. Ollendorf maintains a faculty appointment at CEVR and continues to teach in the Center’s graduate certificate program as well as participate in ongoing research.
Dr. Ollendorf holds a PhD in clinical epidemiology from the University of Amsterdam and an MPH from Boston University. He is the current Chair of the Health Technology Assessment International Global Policy Forum as well as a non-resident Fellow at the Center for Global Development, and served on the US Medicare Evidence Development and Coverage Advisory Committee (MEDCAC) from 2015-2019. Dr. Ollendorf has authored over 125 articles in peer-review journals and is co-author of The Right Price: A Value-Based Prescription for Drug Costs (Oxford University Press).
Panelists
Anirban Basu, PhD, MS
University of Washington, Seattle, WA, USA
Anirban Basu is a Professor of Health Economics and the Stergachis Family Endowed Director of The CHOICE Institute at the University of Washington, Seattle. He holds joint appointments with the Departments of Health Systems & Population Health and Economics at UW, is a Faculty Research Fellow at the US National Bureau of Economic Research, and an elected Fellow of the American Statistical Association. His work sits at the intersection of microeconomics, statistics, and health policy. His research focuses on understanding the economic value of health care, generating causal evidence, and, lately, on the potential for discrimination with machine learning and artificial intelligence algorithms. He served on the Second Panel on Cost-effectiveness Analysis in Health and Medicine and serves on the Editorial Advisory Board for Value in Health Journal. He received his master's in Biostatistics from UNC-Chapel Hill and a PhD in Public Policy Studies from the University of Chicago.
Darius Lakdawalla, PhD
USC Leonard D. Schaeffer Center for Health Policy and Economics, Los Angeles, CA, USA
Darius Lakdawalla is a widely published, award-winning researcher and leading authority on health economics and health policy. He holds the Quintiles Chair in Pharmaceutical Development and Regulatory Innovation at the University of Southern California, where he sits on the faculties of the School of Pharmacy and Sol Price School of Public Policy. He is also the Director of Research at the USC Leonard D. Schaeffer Center for Health Policy and Economics, one of the nation’s premier health policy research centers. His research has focused primarily on the economics of risks to health, the value and determinants of medical innovation, the economics of health insurance markets, and the industrial organization of healthcare markets.
Melanie Whittington, PhD, MS
Tufts Medical Center (CEVR), Boston, MA, USA
Dr. Melanie Whittington is a Senior Fellow at the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center and has a decade of experience in health economics and outcomes research. Most recently, she was the Director of Health Economics at the Institute for Clinical and Economic Review (ICER) where she led the cost-effectiveness analyses and budget impact analyses within ICER reviews. She was the 2022 recipient of the Bernie J. O’Brien New Investigator Award.
Value Assessment and Reimbursement of Early Treatment for Prevention in Chronic Progressive Diseases: Are Traditional Approaches Up to the Task?
Digital Conference Pass
Level: Intermediate
ISSUE:
Early treatment represents an opportunity for prevention of the long-term complications and economic burden associated with many chronic progressive diseases. Estimating the full societal value of early preventive treatment is essential to ensuring patient access and incentivizing innovation. Treatments with high upfront costs, large target populations, and/or significant lags before benefit accrual also may require innovative payment models to ensure affordability. Recent commentaries on value assessment and reimbursement challenges in conditions such as Alzheimer’s disease and for one-time curative gene therapies suggest that traditional economic evaluation approaches and payment models may not be up to the task. In this issue panel, we propose that these challenges can be generalized to other early preventive treatments for chronic progressive diseases and debate the appropriateness of traditional value assessment and reimbursement approaches.
OVERVIEW:
Dr. Herring will introduce the topic and conduct an audience poll on initial perceptions (9 minutes). Dr. Cole will consider the payer’s perspective and reflect on methodologies employed by the National Institute for Health and Care Excellence in the United Kingdom, their appropriateness, and what evolution in payment models may be required to support and incentivize innovation (12 minutes). Dr. Phelps will advocate for the use of the Generalized Risk-Adjusted Cost-Effectiveness (GRACE) approach, explicitly addressing applications of GRACE when severity increases with time and presenting on the incorporation of caregiver impacts in GRACE (12 minutes). Dr. Jiao will present and review emerging methods for incorporating broader, non-health benefits into value assessment, focusing on potential productivity, financial risk protection, and equity benefits, illustrated by recent evaluations of gene therapy for sickle cell disease (12 minutes). We will conclude with a discussion among the panelists and the audience, including a final audience poll (15 minutes). This issue panel may benefit payers, economic modelers, and other stakeholders involved in value assessment and reimbursement decisions.
Moderators
William Herring, PhD
RTI Health Solutions, Research Triangle Park, NC, USA
Will Herring, PhD, is an Executive Director in the Health Economics group at RTI Health Solutions with expertise in cost-effectiveness and value-based pricing modeling. His primary research focus is economic modeling for chronic progressive diseases (Alzheimer’s disease [AD], multiple sclerosis, nonalcoholic steatohepatitis, and diabetic retinopathy) and for conditions and interventions affecting older adults (AD, age-related macular degeneration, herpes zoster vaccination, and respiratory syncytial virus vaccination). He is Affiliated to Research at the Karolinska Institutet and is a member of the ISPOR Open-Source Modeling Special Interest Group.
Panelists
Amanda Cole, PhD
Office of Health Economics, London, LON, United Kingdom
Amanda Cole is Associate Director at the Office of Health Economics, and Honorary Professor of Practice at University College London. Amanda’s research interests include the economics of innovation, the use of real-world evidence to support HTA and product development; novel pricing and reimbursement mechanisms for pharmaceuticals, and the interaction between HTA policy and optimal R&D decisions by industry. Before joining OHE in 2014, Amanda was a research fellow at the University of Birmingham where she focused on the HTA of medical devices and embedding health economic evaluation early in product development. Amanda holds a PhD in health economics from the University of Birmingham.
Boshen Jiao, PhD, MPH
Harvard University, Boston, MA, USA
Boshen Jiao, PhD, MPH, is a postdoctoral research fellow at the Harvard T.H. Chan School of Public Health. His research focuses on comparative effectiveness and cost-effectiveness analysis, health outcomes research, and value-based pricing and insurance design. Dr. Jiao's work aims to support medical decision-making and guide health policies in various disease areas through health economics and outcomes research (HEOR). He has a notable publication record in health economics and clinical journals, and his work has been featured in the media on multiple occasions. He has developed a novel method for incorporating productivity into cost-effectiveness analysis in the absence of direct data. This method has been adopted by the Institute for Clinical and Economic Review for their value assessments. Dr. Jiao has received several academic awards, including the ISPOR Best Podium Presentation Award and the SMDM Lee Lusted Prize. He completed his PhD in HEOR at the University of Washington School of Pharmacy.
Charles Phelps, PhD
University of Rochester, Pittsford, NY, USA
Charles E Phelps, PhD, a health economist, has developed key models of cost-effectiveness analysis that provide the intellectual foundations for its practice. He was given the Victor R Fuchs Award for Lifetime Achievement in the Field of Health Economics in 2019, In 2023, he received ISPOR's Avedis Donabedian Lifetime Acheivement Award. He has been a member of the National Academy of Medicine since 1991. His leading textbook, Health Economics is now in its 6th Edition. His recent interests include the use of multi-criteria decision analysis (MCDA), particularly in its proper use when the “decision-maker” is a group, and development of the Generalized Risk Adjusted Cost-Effectiveness (GRACE) methodology for valuing health gains.
2:45 PM - 3:15 PM
Coffee and Connect
Head to the exhibit hall to refuel, recharge and connect with fellow attendees and exhibitors over a steaming cup of coffee. Provided by ISPOR.
Fast Facts
How to Get Published and Influence People!
Level: Introductory
In this session the Editors-in-Chief of Value in Health will talk about writing for impact, offering tips on what to do and not to do when preparing and submitting your papers. This includes how to write the “highlight” points, which we now require and publish. The session will also address what Value in Health can do to draw attention to articles we publish and what you can do as authors to help us maximize the impact of your work.
Discussion Leader
Nancy Devlin, PhD
University of Melbourne, Melbourne, VIC, Australia
Nancy is Professor of Health Economics at the University of Melbourne and Senior Fellow at the Office of Health Economics, London. She is Chair of the Board of the EuroQol Research Foundation and served as President of ISPOR (2019-2020). Her principal areas of research expertise are the measurement and valuation of patient reported health outcomes; the cost effectiveness thresholds used in making judgments about value for money; and priority setting in health care. She is currently leading a large programme of research to improve measurement and valuation of quality of life in children (QUOKKA), funded by the Medical Research Future Fund (Australia). Nancy has published >180 original peer reviewed journal papers on a wide range of empirical and theoretical topics in health economics.
C. Daniel Mullins, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
C. Daniel Mullins, PhD is a professor at the University of Maryland School of Pharmacy. He is founder and executive director of the University of Maryland PATient-centered Involvement in Evaluating effectiveNess of TreatmentS (PATIENTS) Program. He received his BS in Economics from M.I.T. and his PhD in Economics from Duke University. His research and teaching focus on comparative effectiveness research (CER) and patient-centered outcomes research (PCOR). Dr. Mullins has received funding as a Principal Investigator from AHRQ, FDA, NHLBI, NIA, NIMHD, Patient-Centered Outcomes Research Institute (PCORI) and various patient advocacy organizations and pharmaceutical companies. He is the lead for the Community & Collaboration (C&C) Core of the University of Maryland Institute for Clinical and Translational Research (ICTR) and co-lead of the C&C Core for Johns Hopkins’ CTSA.
Professor Mullins is 1 of 2 editors-in-chief for Value in Health and is author of over 325 peer-reviewed articles and book chapters. At the University of Maryland Baltimore (UMB), he received the Dr. Patricia Sokolove Outstanding Mentor Award and the Dr. Martin Luther King Jr. Faculty Diversity Award. He was named Researcher of the Year at UMB and was awarded a University System of Maryland Wilson H. Elkins Professorship. He is a past recipient of the Dr. Daniel D. Savage Memorial Science Award, the Association of Black Cardiologists’ most prestigious annual award, and the ISPOR Marilyn Dix Smith Leadership Award.
2:45 PM - 3:45 PM
Member Group Meetings
ISPOR Global Access to Medical Innovation Special Interest Group Open Meeting
Leadership members from the Global Access to Medical Innovation Special Interest Group will present the group's mission and goals and will lead an open discussion on topics and ideas for future activities. Members will also have an opportunity to express their areas of interest and engage in breakout discussion groups.
2:45 PM - 4:15 PM
ISPOR Brazil Chapter Meeting
3:15 PM - 3:45 PM
Exhibit Hall Theater
The Next Generation of Observational Research: How Technology and AI Enable Research on “Whole Health” and a Better Patient Experience
Digital Conference Pass
Level: Intermediate
Traditional approaches to observational research often capture only a fraction of a patient’s healthcare journey and give little attention to patients’ experiences with therapeutics. Such a narrow view can diminish the ability of real-world evidence to provide insights to support decision-making by patients, providers, payers, and regulators.
With new advancements in technology and AI, researchers can now easily consent patients into a study and quickly tap into their past clinical encounters to create an on-going inflow of data as medical encounters occur. This means insights can be generated from all of patients’ structured and unstructured provider records, labs, claims, and long-term PRO responses – without tokens, data gaps, or loss-to-follow-up – for radically simplified observational research. These same tools can be used to collect data directly from patients prospectively while at the same time providing direct value to them with an information tool at hand, a technique that has demonstrated increased study retention and engagement.
In this presentation, participants will learn:
- How technology and AI can enhance the depth and breadth of real-world data and provide value back to patients
- How engaging patients in studies enables researchers to collect more information from them directly, leading to more robust and clinically-informative research
- How these advanced technologies have been leveraged in real world research to engage patients and generate novel insights
Sponsor
PicnicHealth
Speaker
Nancy Dreyer, MPH, PhD, FISPE
PicnicHealth, Newton, MA, USA
Nancy Dreyer is Adjunct Professor of Epidemiology at the University of North Carolina at Chapel Hill, Chief Scientific Advisor to Picnic Health, and a Trustee of Brandeis University. Prior to her switch from full-time employment to consultancy, she served as Chief Scientific Officer at IQVIA Real World Solutions, Global Chief of Scientific Affairs at Outcome Sciences Inc and Quintiles, and CEO of Epidemiology Resources Inc. She maintains an active career, teaching for the International Society of Pharmacoepidemiology (ISPE), participating in the ISPOR RWE leadership team, and publishing, and is a Fellow of both ISPE and the Drug Information Association.
One of her primary interests is advancing global use of real-world evidence by medical product regulators, clinicians and payers. Her current interests also include the role of patient-generated health data as well as data linkage and integration.
Kristen Hahn, MPH, PhD
PicnicHealth, San Francisco, CA, USA
Kristen Hahn, PhD, MPH is the Head of RWE Research at PicnicHealth and an epidemiologist passionate about delivering creative, fit for purpose and scalable research to stakeholders. She thrives in advancing innovative solutions from concept through delivery and is passionate about transforming the experience of participating in studies to ensure that it is a more inclusive process and results in scientifically valid inferences. Kristen is an established leader with 15+ years of experience as an epidemiologist who has demonstrated proficiency in aligning teams with diverse backgrounds from science and technology across complex organizations. Her experience spans many indications and data collection modalities including direct to patient, patient-mediated, national registries, claims, electronic health records, and site-based data collection.
3:15 PM - 4:00 PM
Network, Collaborate, and Get Involved in ISPOR's Global Groups
Are you interested in getting involved in the development of HEOR globally in support of ISPOR's mission? Join us to learn more about ISPOR's global member engagement activities and how you be part of the Society's global groups.
3:15 PM - 4:15 PM
Educational Symposium
From Hype to Reality: Applications of Generative AI in HEOR and Market Access
Digital Conference Pass
The rapid evolution of generative artificial intelligence (AI) from a burgeoning concept to a transformative technology heralds a new era for Health Economics and Outcomes Research (HEOR) and Market Access. This educational symposium aims to demystify the applications and real-world use cases of generative AI, bridging the gap between theoretical potential and practical utility. Join us as leading experts offer insights into how generative AI can transform the landscape of HEOR and Market Access.
Dr. Chhatwal will moderate the session, setting the stage with an insightful introduction into the current landscape and potential of generative AI in healthcare, aiming to provide a foundational understanding for all attendees. The symposium will delve deeper into the intricacies of generative AI with Dr. Ayer exploring the current state of technology and addressing the challenges posed by the limitations of commonly available large language models (LLMs). Dr. Ayer will also highlight the critical need for developing niche-LLMs tailored for the unique demands of HEOR and market access. Next, Dr. Ozer-Stillman will share examples where LLMs are transforming HEOR practice. Dr. Shreay will showcase practical examples where generative AI could be a boon for Market Access, illustrating the technology’s capacity to enhance research outcomes and decision-making processes. Dr. Fleurence will discuss the integration of Real-World Evidence (RWE) and Real-World Data (RWD) with generative AI, offering insights into how these collaborations can yield novel insights and drive HEOR field forward. Lastly, Dr. De Solda will highlight how generative AI complements traditional AI in HEOR and market access, thereby offering strategic advantages.
Through these expert-led sessions, the symposium aims to equip attendees with a comprehensive understanding of generative AI’s role in HEOR and market access, fostering a future where healthcare and technology synergize to improve outcomes and accessibility.
Sponsor
ValueGen.AI
Moderators
Jag Chhatwal, PhD
Massachusetts General Hospital, Harvard Medical School, Wilmington, MA, USA
Dr. Chhatwal is an associate professor at Harvard Medical School and director of the Institute for Technology Assessment at Massachusetts General Hospital. His research is centered in data/decision analytics, AI, and health economics. Dr. Chhatwal has co-authored over 120 original research articles and editorials in peer-reviewed journals. His work has been cited in leading media outlets, including CNN, Forbes, National Public Radio, New York Times, and Wall Street Journal.
Since 2011, Dr. Chhatwal has taught several workshops and short courses on decision modeling and AI at the ISPOR. He also serves as an associate editor of Value in Health and recently served as the guest editor for special issues on the opioid crisis and COVID-19 published in Value in Health. He is a co-chair of 2024 SMDM meeting, whose theme is "The future of medical decision making in the era of generative AI".
Panelists
Turgay Ayer, PhD
Value Analytics Labs, Boston, MA, USA
Turgay Ayer, PhD, stands at the forefront of innovative healthcare solutions as the Chief Scientific Officer at Value Analytics Labs. Under Dr. Ayer's guidance, Value Analytics Labs has developed ValueGen.AI, a revolutionary generative AI-based platform designed to transform market access and Health Economics and Outcomes Research (HEOR). This platform exemplifies Dr. Ayer's commitment to leveraging advanced data science and machine learning techniques to drive real-world evidence and health economics modeling.
Beyond his role as a Chief Scientific Officer, Dr. Ayer is a distinguished academic, serving as a professor and the research director for Healthcare Analytics and Business Intelligence in the Center for Health & Humanitarian Systems at Georgia Tech. He also holds a courtesy appointment at Emory Medical School and is a senior scientist at the CDC. His academic work, deeply rooted in health economics, real-world evidence, data science, and machine learning, has led to the publication of over 75 journal papers and more than 250 conference abstracts, particularly enriching the field of oncology. Dr. Ayer's contributions have not only been recognized in top-tier academic journals but have also captured the attention of major media outlets such as the Wall Street Journal, Washington Post, U.S. News, and NPR, highlighting his impact on both academic and practical aspects of healthcare analytics.
Francesco De Solda, PharmD, MBA
Janssen Global Services LLC, Raritan, NJ, USA
Francesco De Solda is a seasoned pharmaceutical professional with a rich background in Market Access, Health Economics and Outcomes Research, and R&D. With extensive leadership experience in the Pharmaceutical Industry in different functions and geographies, and a solid academic foundation - including a Doctorate in Pharmacy, a Master in Business Administration, a post-graduate degree in Clinical Research, and Executive Certificates in Health Economics, HTA, Market Access and Government Affairs - Francesco excels in driving global market access strategies for innovative healthcare solutions. Passionate about Oncology, he brings a dynamic blend of expertise and strategic vision.
Rachael Fleurence, PhD
National Institutes of Health, Bethesda, MD, USA
Dr Fleurence is a Senior Advisor at the National Institutes of Health where she is working on launching a national initiative to eliminate Hepatitis C in the United-States. Dr Fleurence is also affiliated with the National Institute of Biomedical Imaging and Bioengineering where she works on advances in Artificial Intelligence and Machine Learning. Dr Fleurence currently co-leads the ISPOR Task Force on the suitability of EHR data for Health Technology Assessments. Previously, Dr Fleurence served as a senior health advisor in the Biden-Harris White House, Dr. Fleurence received a BA from Cambridge University (United Kingdom), an MA in business management from ESSEC-Paris (France), and an MSc and PhD in health economics from the University of York (UK).
Ipek Ozer Stillman, MS, MBA
Takeda Pharmaceuticals, Boston, MA, USA
Ipek is a team leader at a global biotechnology company, responsible for articulation and coordination of strategy and health economics activities across various functional teams to identify, assess and communicate value of new interventions.
• More than 15 years of experience in research, consulting and data analysis with > 9 years of direct management experience, demonstrating exceptional individual contribution and effective team leadership.
• Successful track record in data and analytics, evidence generation strategy and execution, health economics and outcomes research.
Sanatan Shreay, PhD, MS
Cytokinetics, South San Francisco, CA, USA
Sanatan Shreay, a seasoned leader in value and access, brings over 15 years of expertise in enhancing patient access to innovative treatments. With extensive experience in launching products globally across therapeutic domains like Oncology, Cardiology, HCV, HIV, and others, he excels in optimizing clinical trial designs and demonstrating product value through HEOR. Currently at Cytokinetics, he oversees teams driving the communication of product value for muscle-directed therapies. Prior roles include leadership positions at Gilead, Nektar, TG Therapeutics, and Amgen.
ISPOR Forums
Uncovering the Impact of HTA on Medical Device and Diagnostic Procurement Process and Outcomes: Insights and Discussion From the ISPOR Medical Devices and Diagnostics SIG Key Project Research
Digital Conference Pass
Health Technology Assessments (HTAs) for MedTech often do not lead to a funding or reimbursement mandate despite being a very robust manner of technology evaluation. Initiatives such as value-based procurement, where the procurement decisions are made on clinical improvement and reduced total costs suggest HTA could be a very valuable resource for procurement decision making. The ISPOR Medical Devices and Diagnostics Special Interest Group (MD&D SIG) working group has undertaken and published a systematic review of literature describing how HTA is influencing and linked to procurement decisions. This project identified limited evidence that HTAs inform medical device procurement. Procurement decisions were often considered standard HTA factors but lacked transparency. The study advocates for increased transparency, research, and collaboration to better link HTA findings with procurement decisions, potentially leading to improved healthcare savings and accessibility. We will discuss these potential policy implications and the ongoing efforts of the ISPOR MD&D SIG working group to survey HTA and procurement stakeholders to understand in more depth the links between these groups and identify opportunities for closer collaboration. The panel will discuss the linkage between HTA and procurement of MedTech devices and diagnostics and policy implications to (1) support and inform ongoing ISPOR MD&D SIG working group actively surveying of HTA and procurement stakeholders and (2) extend an opportunity for ISPOR members to participate in this working group survey activity and (3) inform the survey questions.
Moderators
Michael Cangelosi, MA, MPH
Boston Scientific, Natick, MA, USA
Mr. Cangelosi is an Associate Director of Health Economics and Market Access at Boston Scientific Corp. His work uses health economic methods to analyze real-world data to support the commercial and evidentiary goals of the gastrointestinal-endoscopy business. Prior to Boston Scientific, Mr. Cangelosi was a Research Associate with the CEVR at Tufts Medical Center and has degrees in economics and public health from Tufts University. He is currently a member of the leadership team and key-project leader of ISPOR’s Medical Device and Diagnostics Special Interest Group.
Speakers
Meike Bomhof, MA
Avania, Valencia, V, Spain
Meike Bomhof is Vice President Europe for Avania, a global end-to-end CRO with dedicated expertise in medical devices, diagnostics and combination products.
Meike joined Avania as part of the acquisition in 2023 of HullAssociates, a market access strategy firm specialized in supporting and implementing global MedTech market access and reimbursement strategies. Prior to co-founding Hull Associates in 2007, Meike served 15 years in management roles related to market access, business development and finance in the Medtech Industry in companies such as Baxter and Edwards Lifesciences.
In addition, Meike actively collaborates with Medtech venture builders and start-up accelerators such as Biocat, EIThealth, Techleap, NLC and CIMIT through market access and reimbursement workshops and mentoring activities.
Arturo Cabra, MSc
GE Healthcare, Miami, FL, USA
Patricia Synnott, MS, MALD
Tufts Medical Center, Boston, MA, USA
Establishing the Criteria for the Value Assessment for Medication Adherence-Enhancing Interventions
Digital Conference Pass
PURPOSE: To discuss criteria for the value assessment of medication adherence enhancing interventions (MAEIs). Audience will be asked to participate in identifying and ranking pre-determined criteria from most to least important and discussing their rationale for their choices.
DESCRIPTION: Several studies demonstrated that MAEIs (e.g., patient counselling, home telemonitoring, support groups) may improve adherence outcomes but existing evidence on the effectiveness of these MAEIs is of poor quality. This project aims to identify criteria for the value assessment of MAEIs. Values may include elements to measure health/non-health benefits for the patients or their family/caregiver and also benefits for societal health and the health care system. A systematic review of the literature was performed to identify important value domains and criteria for the assessment of MAEIs. Further, to critically evaluate these criteria and identify any potential new criteria, focus groups involving researchers/academia, payers, and healthcare practitioners were conducted. These focus group participants in addition to identifying new criteria, also ranked their top 10 criteria from their perspective. The workshop will consist of a 15-minute presentation on the project, followed by a review of the three sets of criteria. Employing a think-pair-share approach, the audience will be asked to review these criteria and identify to which one they most agree with. They will discuss this with their colleagues at their table/seat, then, through online polling, the audience members will choose one of the three as their preferred. The presenters will discuss the results and ask the audience to then identify, from an alphabetical listing of the criteria, their top 5 criteria. The audience will share this list with colleagues at their table/seat, discuss the rationale for their ranking and submit their individual rankings, through online polling, so a final list of the top 5 criteria from all audience members can be generated.
Moderators
Dweeti Nayak, MS
Precision Medicine Group, Jersey City, NJ, USA
My name is Dweeti Nayak. I am a Research Scientist at a consulting firm called Precision Medicine Group with over 5 years of experience in conducting and managing a wide range of HEOR projects including evidence synthesis, gap analysis, and value assessments for major pharmaceutical and life sciences companies. I have a stronger focus on evidence synthesis projects like systematic literature reviews and feasibility assessments to support network-meta analysis and other quantitative synthesis. I am also a member engagement co-chair for the Medication Adherence and Persistence Special Interest Group, involved in conducting various research projects, journal clubs, and webinars on topics related to Medication Adherence and Persistence.
Speakers
Tamas Agh, MSc, PhD, MD
Syreon Research Institute, Budapest, PE, Hungary
Tamás Ágh MD MSc PhD DrHabil graduated from the Semmelweis Medical University (MD, 2006), the Corvinus University of Budapest (MSc Physician-Economist, 2010), the Semmelweis University School of PhD Studies (PhD in Pharmacoeconomics, 2013) and the University of Pecs Doctoral School of Health Sciences (DrHabil in Health Sciences, 2020). He specialized in family medicine in 2010. Dr. Ágh is a principal researcher at the Syreon Research Institute and has been practicing as a medical doctor since 2006. He is also a research associate professor and the head of the Medication Adherence Research Group at the Center for Health Technology Assessment and Pharmacoeconomic Research at the University of Pécs. With a professional background spanning over 15 years, his areas of specialization in health economics and outcome research include medication adherence, patient-reported outcomes measures, health technology assessment, and evidence synthesis. He serves as the chair of the ISPOR Medication Adherence and Persistence Special Interest Group, chair-elect of the ESPACOMP International Society of Medication Adherence, and holds a leadership position in the European Network to Advance Best practices & technoLogy on medication adherencE (ENABLE) Cost Action. Dr. Ágh has co-authored about 50 peer-reviewed articles.
Lina Eliasson, PhD, CPsychol
Sprout Health Solutions, Pinner, LON, United Kingdom
I specialise in understanding, measuring and improving treatment adherence and in identification and validation of COAs for clinical trials. Following the completion of my PhD from UCL School of Pharmacy on adherence to oral oncology drugs, I worked as a post-doc at Imperial College London. I then joined an agency that specialised in treatment adherence where I oversaw the development of patient support programs. Before founding Sprout in 2017, I led the European COA team for one of the world’s largest CROs. Sprout provides patient engagement services throughout the drug lifecycle.
Adina Turcu-Stiolica, PhD.
University of Medicine and Pharmacy of Craiova, Craiova, DJ, Romania
Professor of Pharmacoeconomics at the University of Medicine and Pharmacy of Craiova, Romania.
Her educational background is Faculty of Pharmacy, but also in Applied data science and artificial intelligence (MIT, Massachusettes, USA) and Applied methods on cost-effectiveness analysis (University of Oxford, Nuffield Department of Population Health, UK).
She is member of ISPOR from 2010 and, now, she is the Chair of the ISPOR Medication Adherence and Persistence Special Interest Group.
She is the Faculty Advisor of the ISPOR Romania Student Chapter.
She is an author of over 100 peer-reviewed articles, including those on cost-effectiveness, MCDA, health-related quality of life, medication adherence and interventional/observatory studies.
https://www.researchgate.net/profile/Adina-Turcu-Stiolica
Economic Insights Into Nutrition: Unlocking the Value in Chronic Disease Management
Digital Conference Pass
The food we eat has a powerful impact on risk factors and severity of the most prevalent and costly chronic diseases, including obesity, cancer, diabetes, heart disease, and cerebrovascular disease. While breakthrough pharmaceuticals such as statins, ACE inhibitors, calcium channel blockers, and glucagon-like peptide 1 agonists have revolutionized chronic disease care, nutrition plays an important role in the effective prevention, management, and treatment of hypertension, diabetes, cancer, and other chronic diseases. Community-based interventions, addressing health behaviors and food environments, are promising strategies to improve community health and reduce health inequities. This forum will examine the burden of diet-related chronic diseases and the state of the science on the effectiveness of community-based interventions to improve nutrition and reduce the risk of chronic disease. It will explore the value health economic evaluations of nutrition in health care and the challenges and opportunities in these evaluations in the special case of nutrition interventions. Speakers will highlight the need for economic evaluations of food and nutrition security-related interventions and why incorporating health equity and social determinants of health considerations into these evaluations is important.
Elizabeth Lynch will discuss the state of the science on community-based nutrition interventions for individuals with hypertension and strategies for community-partnered research aimed at improving nutrition and reducing chronic disease. Mark Nuijten will examine the results of the experience of applying health economics evaluation for nutrition interventions in malnutrition, including malnutrition related to obesity and to oncology. Jason Shafrin will discuss the opportunities and challenges of economic evaluation of nutrition interventions and compare economic analysis of nutrition interventions with analysis of pharmaceutical interventions.
Moderators
Kirk Kerr, PhD
Abbott Nutrition, Columbus, OH, USA
Kirk W. Kerr joined the Abbott Nutrition’s Health Economics and Outcomes Research team in June 2019. While at Abbott, Kirk has researched the impact of nutrition interventions in improving patient outcomes in inpatient, outpatient, ICU, wound care and surgical care settings. Prior to joining Abbott, Kirk worked in the FDA’s Center for Drug Evaluation and Research researching the impact of FDA policies on drug development, drug prices, and patient access to medicines and provided economic support for FDA’s User Fee negotiating teams. Kirk has a PhD and MA in Economics from Ohio State University, and BS and MA in Economics from the University of Missouri.
Speakers
Elizabeth Lynch, PhD
Rush University, CHICAGO, IL, USA
Dr. Lynch is a Professor of Family and Preventive Medicine at Rush University Medical Center. She is a health equity researcher focused on development and implementation of effective community partnerships to conduct behavioral intervention research to promote health equity across a wide range of health conditions. She is a founder and the Research Director of the Alive Faith Network (AFN), a partnership between African American churches and researchers to address health inequities in African American communities. She has served as PI on numerous studies testing community based interventions to promote health equity and is the co-PI of the Chicago Chronic Conditions Equity Network (C3EN), which is an NIMHD-funded P50 center at RUSH and University of Chicago to develop community-partnered interventions to address health inequity in chronic conditions in the Chicago region.
Mark Nuijten, MBA, PhD, MD
A2M, Amsterdam, Netherlands
Mark Nuijten is a medical doctor, health economist, valuation economist, and healthcare publicist. He is a visiting professor at Ben-Gurion University in Israel, setting up the department on Clinical and Economic Valuation of Medical Innovation. He has become a leading health policy and economics expert over the last 2 decades, reflected in more than 200 publications and leading positions in scientific societies and editorial boards. Dr. Nuijten was board director of ISPOR (2002-2004) and chair of the Management Board of Value in Health (2002-2004). He was a member of the Editorial Advisory Board of Value in Health. He obtained his PhD in health economics (2003) on the thesis “In search for more confidence in health economic modelling” at the Erasmus University, Rotterdam.
Mark Nuijten is founder of A2M (Ars Accessus Medica) and founding partner of the Minerva International Health Economic Network. He was trained as a physician and worked in clinical research before obtaining his international MBA from Erasmus University, Rotterdam, where he later was a senior staff member. Prior to setting up Ars Accessus Medica, Dr. Nuijten was the founding managing director of the IQVIA Quintiles office in the Netherlands, which included European responsibility for the policy and health economic division.
He is a pioneer in the field of healthcare innovation in biotechnology and has been the first classical health economist successfully applying and developing Discounted Cash Flow methodologies for valuation of biotechnology innovation (eg, a pricing model to assess prices of expensive orphan drugs from an investor’s perspective—published in a Nature journal). He also developed an integrated valuation model, an interactive dynamic tool for the economic valuation of R&D projects, which can be used to optimize the initial clinical program (eg, indication, comparator, outcomes, and study design), and the associated pricing and market access pricing strategy.
Jason Shafrin, PhD
Center for Healthcare Economics and Policy, FTI Consulting, Los Angeles, CA, USA
Jason Shafrin, Ph.D. is a Senior Managing Director at FTI Consulting's Center for Healthcare Economics and Policy. Dr. Shafrin has over 20 years of health economics research experience serving as trusted advisor and expert to a wide variety of healthcare and life sciences companies, governments and non-governmental organizations (NGOs). Dr. Shafrin is an Adjunct Professor at the University of Southern California, the former Director of Research at the Innovation and Value Initiative and the Founder and Editor of the Healthcare Economist blog.
3:30 PM - 4:15 PM
Poster Tours
Real-World Evidence Poster Tour
Posters featured in this tour: PT43: How Has Real-World Evidence Been Leveraged in Non-Oncology Submissions in the UK, France, and Germany? PT44: Prevalence of Comorbidities and Hospitalization in the 65 and over SLE (SYSTEMIC LUPUS ERYTHEMATOSUS) Patient Population: A Comparative Analysis of the FORWARD Lupus Registry and Medicare Data in the US (2017-2021) PT45: Patient Perspective in Endometriosis and Clinical Trial Barriers PT46: Off-Label Use of Semaglutide in the United States: Increasing Prevalence, Variability in Prescribing Provider Specialty, and Patient Characteristics PT47: Prevalence of Systemic Lupus Erythematosus By Social Determinants of Health: Nationally Representative Estimates for 2017-2021 PT48: Using a Database Analysis to Determine Alignment between Physician-Reported and Objectively Derived Fibrosis Scores for US Patients with Metabolic Dysfunction-Associated Steatohepatitis (MASH)
Moderator
Ashley Jaksa, MPH
Aetion, Inc, Boston, MA, USA
Ashley Jaksa is Market Access Scientific Strategy Lead at Aetion. She focuses on collaborating with decision-makers to enhance their use of RWE and setting methodological guidance for RWE generation. She also works with Aetion’s biopharma clients on incorporating RWE in their global market access strategy. She has presented her research at numerous international conferences including ISPOR, HTAi, and AMCP, and published her findings in various peer-reviewed publications. She holds a bachelor's degree from the University of Michigan-Ann Arbor and a MPH from Yale University.
Medical Devices Poster Tour
Posters Featured in this Tour: PT37: The Clinical Outcome of Image-Guided System in Endoscopic Sinus Surgery for Chronic Rhinosinusitis: A Meta-Analysis PT38: Early and Mid-Term Outcome of Valve Replacement with Porcine Bioprosthesis in the Aortic Positions PT39: Catheter Ablation of Atrial Fibrillation Followed By Left Atrial Appendage Closure: A Retrospective Analysis of Adverse Events Occurring between Procedures PT40: National Readmission Rates Post Surgical Heart Valve Replacement: A Comparative Analysis of Tissue and Mechanical Valves PT41: Comparative Analysis of Reintervention Rates in Mesh Versus No Mesh Hernia Repair Using Electronic Health Records PT42: A Systematic Literature Review and Meta-Analysis Comparing Robotic-Assisted Surgery Using the Da Vinci Surgical Systems for Benign Gynecological Procedures to Laparoscopic or Open Approach
Moderator
Julian Shepelev, MSc
Baxter Healthcare, London, LON, United Kingdom
3:30 PM - 6:30 PM
Poster Session 4
Live
3:45 PM - 4:15 PM
Exhibit Hall Theater
Ahead of the Curve: Navigating Early Planning Strategies for External Control Arms in HTA and Regulatory Submissions
Digital Conference Pass
Existing guidance on the design and conduct of externally controlled trials places emphasis on early engagement with regulatory and HTA bodies to justify the rationale for using an external control arm (ECA), and to discuss the preliminary study design and statistical analyses. With the increasing use of ECAs in both regulatory and HTA submissions, the acceptable use cases for ECAs (e.g., single arm trials in rare indications, undefined control/standard of care) and important design and analytic considerations are becoming clearer. However, key questions remain for manufacturers:
1) When is the most optimal timing in the development process to plan for an ECA?
2) When and how should a company best prepare for early interactions to address differing regulatory and HTA perspectives?
In this session, we will discuss these questions, as well as differences in how regulatory vs HTA stakeholders appraise ECAs, the most common key issues from each perspective, and proposed solutions. We will also cover early planning strategies that should be considered by clinical development/regulatory and HEOR teams to optimize both regulatory and market access pathways. Finally, we will conclude with recommendations for early ECA feasibility assessments and demonstrate how to successfully navigate early stakeholder interactions through an example case study.
Sponsor
Cytel
Moderators
Jason Simeone, PhD
Cytel Inc, Waltham, MA, USA
Jason has over 15 years of experience in pharmacoepidemiology and database analytics, and his global research has focused on medication safety, effectiveness, burden of illness, and treatment patterns in a wide range of therapeutic areas, with experience conducting retrospective PASS studies and multi-country studies. He is particularly interested in the application of RWE for regulatory decision-making and has served as a member of several Duke-Margolis Real-World Evidence Collaborative working groups. He has an MS and PhD from the University of Rhode Island in pharmacoepidemiology and pharmacoeconomics.
Speakers
Grace Hsu, MSc
Cytel Inc., Waltham, MA, USA
Grace is Director of Real-World Evidence at Cytel with 9 years of experience in consulting and guiding project strategies. She holds a Master's degree in Statistics, providing statistical consulting and strategy development for data curation, and the application of advanced analytics to clinical and RWD. Examples of her peer-reviewed publications include work on COVID-19, synthetic/external control arm comparative effectiveness analysis, quantitative bias analysis, Bayesian borrowing and other methods of indirect comparison for both pharmaceutical research and HTA/regulatory submissions.
Evie Merinopoulou, MSc
Cytel Inc., London, England, United Kingdom
Evie Merinopoulou is a Health Economist and Real-World Data Scientist working on applications of Real-World Evidence in support of regulatory and HTA decision making. Ms Merinopoulou has worked in the healthcare consulting industry for over 10 years. She currently serves as a Senior Director, Real-World Evidence at Cytel, based in London, UK. She leads the design and execution of observational research projects using global real-world data. Ms Merinopoulou particularly focuses on studies involving real-world external comparator arms, quantitative bias analysis, head-to-head comparisons using target trial emulation, and transportability analysis.
4:00 PM - 4:30 PM
Fast Facts
Can Generative Artificial Intelligence (GenAI) Deliver Robust Research and Shorten the Healthcare Innovation Cycle?
Level: Introductory
Evidence requirements burden industry and delay patient access to healthcare innovation. In this session, experts will share how Generative Artificial Intelligence (GenAI) applications such as literature reviews and data synthesis can significantly alleviate that burden. Join to discuss how a systems approach to GenAI including 'experts-in-the-loop' can increase accuracy and provide transparency.
Discussion Leader
Lana Feng, PhD
Huma.AI, Palo Alto, CA, USA
Dr. Feng is the CEO and cofounder of Huma.AI, a leading generative AI platform for life sciences. The platform automates data curation for Pharma and med device companies thereby accelerating the development of new products and saving patient lives. Via its collaboration with OpenAI since May 2022, Huma.AI’s GenAI platform removes hallucinations and adds citations, providing much needed accuracy and transparency. Technology Thought Leader Gartner has named Huma.AI as a leading GenAI vendor in 20 of its Hype Cycle Reports, including those for AI, emerging technologies, and life sciences. Dr. Feng spent over 20 years in precision medicine. Before founding Huma.AI, She worked at Novartis' Oncology Business Unit, joining via its acquisition of Genoptix. Dr. Feng was hired by Genoptix to build a BioPharma division, growing sales from zero to $45M in five years and was instrumental to its acquisition by Novartis. Dr. Feng holds a Ph.D. in Genetics, Cellular and Developmental Biology from Indiana University. She did her postdoctoral research at University of California, San Diego.
John Hernandez, PhD
Google, Mountain View, CA, USA
Dr. John Hernandez is Head of Clinical Research, Health Economics and Outcomes Research at Google, which is leveraging information technology and artificial intelligence to assist in promoting healthy lifestyles, diagnosing cancer, preventing blindness and much more. He joined Alphabet in 2016 where he has built and led teams at Google and Verily Life Sciences leveraging artificial intelligence to conduct research and launch health products using digital platforms, smartphones, apps, wearables, connected biosensors and interoperable health data to help people lead healthier lives. Dr. Hernandez serves on the board of directors for ResMed and Carmat, which are publicly traded medical device companies. Prior to Alphabet, John worked for 15 years in executive roles at Boston Scientific and Abbott where he led global clinical research, health economics and outcomes research initiatives. John has served in research and consulting roles at the RAND Corporation, Quintiles (now IQVIA), PwC and the American Society of Internal Medicine (now ACP). John obtained his PhD and Masters in Health Policy at the RAND Graduate School in Santa Monica, CA and his BA from the University of North Carolina at Chapel Hill.
4:30 PM - 6:00 PM
Member Group Meetings
ISPOR RWE SIG Open Meeting and Discussion of CMS Proposed Access to Medicare Data
Leadership members from the Real World Evidence Special Interest Group will present recent accomplishments, upcoming activities and volunteer opportunities, ongoing projects, and lead breakout groups with attendees who will share their suggestions, ideas, and feedback for future projects.
5:00 PM - 6:00 PM
Breakouts: IP, WS, & OBS
How Elastic Is Biopharmaceutical Innovation With Respect to Expected Reward? What Is Missing From Existing Literature on Elasticity of Innovation?
Digital Conference Pass
Level: Intermediate
ISSUE: The impact on biopharmaceutical R&D from reduced expected reward size has been a recurring theme in the ongoing debates on the Inflation Reduction Act (IRA) in the US. There is currently no consensus from the HEOR community on the scale and magnitude of the elasticity of innovation to expected reward. Existing empirical evidence differed in their methods, data sources, and estimates. A more thorough understanding of the scale of elasticity of innovation is essential to inform policy making, ensuring the balance between affordability and innovation. The purpose of this session is to review the strengths and weaknesses of existing evidence, identify areas for further research, and discuss the implications of the findings on policy making and social welfare.
OVERVIEW: Gunnar Esiason will first describe the importance of pharmaceutical innovation for patients based on his lived experience with cystic fibrosis, and underscore the need to understand the true scale of the elasticity of innovation (10 minutes). Peter Kolchinsky will demonstrate the impacts of proposed policy changes from IRA using net present value modeling, and show how regulatory changes can impact R&D decisions from an investor perspective (10 minutes). Amanda Cole will present a select set of existing empirical estimates from the literature, and discuss the strengths and limitations of each approach (10 minutes). Darius Lakdawalla will highlight areas that should be prioritized for further research, and the implications for more informed policy making and welfare of current and future patients. The session will end with Q&A with the audience (20 minutes). Polling questions will be used throughout to identify consensus on the range of empirical estimates of innovation and priorities of areas for further research. This session will be relevant for a wide range of stakeholders including patients, employers, payors, modelers, policymakers, and value assessors.
Moderators
Gunnar Esiason, MBA, MPH
Boomer Esiason Foundation, New Canaan, CT, USA
Gunnar Esiason is a cystic fibrosis and rare disease patient leader. He is the head of patient engagement at RA Capital's venture group, RA Ventures.
Gunnar has been the face fundraising efforts for the Boomer Esiason Foundation, which has yielded close to $200 million raised for the fight against cystic fibrosis since 1993. He was a founding advisor of the non-profit No Patient Left Behind, and previously led patient operations at a leading clinical trial technology startup.
Gunnar has been a leading voice on rare disease market access issues with health policies opinions featured in The Hill, Wall Street Journal and STAT News.
Gunnar holds an MBA and MPH from Dartmouth College and a BA from Boston College.
Panelists
Amanda Cole, PhD
Office of Health Economics, London, LON, United Kingdom
Amanda Cole is Associate Director at the Office of Health Economics, and Honorary Professor of Practice at University College London. Amanda’s research interests include the economics of innovation, the use of real-world evidence to support HTA and product development; novel pricing and reimbursement mechanisms for pharmaceuticals, and the interaction between HTA policy and optimal R&D decisions by industry. Before joining OHE in 2014, Amanda was a research fellow at the University of Birmingham where she focused on the HTA of medical devices and embedding health economic evaluation early in product development. Amanda holds a PhD in health economics from the University of Birmingham.
Peter Kolchinsky, PhD
RA Capital Management, Boston, MA, USA
Peter Kolchinsky is Founder and Managing Partner at RA Capital, investing in companies developing drugs, devices, and diagnostics. He serves as a board member for various public and private companies. Peter served on the Board of Global Science and Technology for the National Academy of Sciences and is the author of “The Great American Drug Deal” and “The Entrepreneur’s Guide to a Biotech Startup.” He founded the nonprofit No Patient Left Behind to advocate for healthcare reform and promote innovation. Peter holds a BA from Cornell University and a PhD in Virology from Harvard University.
Darius Lakdawalla, PhD
USC Leonard D. Schaeffer Center for Health Policy and Economics, Los Angeles, CA, USA
Darius Lakdawalla is a widely published, award-winning researcher and leading authority on health economics and health policy. He holds the Quintiles Chair in Pharmaceutical Development and Regulatory Innovation at the University of Southern California, where he sits on the faculties of the School of Pharmacy and Sol Price School of Public Policy. He is also the Director of Research at the USC Leonard D. Schaeffer Center for Health Policy and Economics, one of the nation’s premier health policy research centers. His research has focused primarily on the economics of risks to health, the value and determinants of medical innovation, the economics of health insurance markets, and the industrial organization of healthcare markets.
Is the Employer Perspective Sufficiently Reflected in Common Value Frameworks?
Digital Conference Pass
Level: Intermediate
ISSUE:
Employers, particularly large employers self-sponsoring health insurance, are consequential decision-makers for healthcare coverage and access in the U.S. However, employers have a distinct perspective and objectives compared to publicly financed health insurance programs, private insurers who sell products to employers, and health technology assessment organizations. Given that the employer perspective is rarely explicitly addressed in research or health technology assessments, this panel will discuss whether common value frameworks such as the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Value Flower and the Institute for Clinical and Economic Review (ICER) Value Assessment Framework can sufficiently reflect the employer’s perspective and evidence needs.
OVERVIEW:
In recent years, there have been efforts to coalesce evidence generation to common value frameworks. Importantly, these frameworks have explicitly expanded value dimensions beyond quality-adjusted life years (QALYs) and direct healthcare costs. In the case of the ISPOR Value Flower, value dimensions include elements such as health equity, real option value, and scientific spillovers. In the case of the ICER Value Assessment Framework, short-term affordability, productivity, and caregiver burden are considered quantitatively, with other qualitative components of the deliberative process. However, whether these value frameworks do or do not successfully meet the needs of employers, who collectively make health benefit decisions for over half of the U.S. population, has not been sufficiently discussed. The panel will start with a 10-minute overview by the moderator, Dr. Lofgren, on the unique considerations of employers. The panel will then discuss how the ICER and ISPOR value frameworks do and do not address the employer perspective anchored on 1-2 case studies. In our discussion experts will speak from their specific perspectives as an institutional leader (Dr. Ollendorf), academic researcher (Dr. Doshi) and employer benefits strategist (Mr. Prettol). We will conclude with recommendations for creating evidence more inclusive of the employer perspective.
Moderators
Katherine Lofgren, PhD, MPH
Abett, Inc., Denver, CO, USA
Dr. Katherine Lofgren is the Director of Health Economics and Outcomes Research at Abett, a software platform supporting the data and evidence needs of large self-insured employers. Dr. Lofgren has previously held research positions at Foundation Medicine, the World Bank, and the Institute for Health Metrics and Evaluation. She received her PhD in Health Policy from Harvard University and an MPH in Global Health from the University of Washington, Seattle.
Panelists
Meng Li, PhD, ScM
Tufts University, Boston, MA, USA
Meng Li is an Assistant Professor at the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center and a nonresident fellow at the Leonard D. Schaeffer Center for Health Policy & Economics at the University of Southern California. Her research focuses on defining and measuring novel elements of value for medical technologies, as well as healthcare innovation and pricing.
Prior to joining CEVR, she was an Assistant Professor at the Department of Health Services Research at the University of Texas MD Anderson Cancer Center. She completed her PhD in Pharmaceutical Outcomes Research & Policy at the University of Washington.
Dan Ollendorf, PhD
Institute for Clinical and Economic Review, Boston, MA, USA
Dan Ollendorf is Chief Scientific Officer and Director of Health Technology Assessment (HTA) Methods and Engagement at the Institute for Clinical and Economic Review (ICER), with responsibility for managing the organization’s internal health economics capacity and program of external collaboration, as well as collaboration with international HTA bodies and other stakeholders on key process and methods topics.
From 2007-2018, Dr. Ollendorf was Chief Scientific Officer for ICER, where he oversaw the broader HTA process and managed relationships with multiple stakeholders and research collaborators. From 2018-2023, Dr. Ollendorf served as the Director of Value Measurement & Global Health Initiatives at the Center for the Evaluation of Value and Risk in Health (CEVR) at Tufts Medical Center, where he focused on value assessment methods, drug pricing policy, and building international capacity for and interest in HTA. In addition to his current role at ICER, Dr. Ollendorf maintains a faculty appointment at CEVR and continues to teach in the Center’s graduate certificate program as well as participate in ongoing research.
Dr. Ollendorf holds a PhD in clinical epidemiology from the University of Amsterdam and an MPH from Boston University. He is the current Chair of the Health Technology Assessment International Global Policy Forum as well as a non-resident Fellow at the Center for Global Development, and served on the US Medicare Evidence Development and Coverage Advisory Committee (MEDCAC) from 2015-2019. Dr. Ollendorf has authored over 125 articles in peer-review journals and is co-author of The Right Price: A Value-Based Prescription for Drug Costs (Oxford University Press).
Luke Prettol, BA
AT&T, Spring, TX, USA
Luke Prettol is the Principal Benefits Strategist at AT&T, where he plays a pivotal role in shaping the strategy and design of the company's active and retiree health and welfare benefits. Throughout his 15+ years leading benefits strategy at companies of various sizes, he has been a passionate advocate for delivering equitable access to patient-centered healthcare solutions. Luke holds a BBA in Human Resource Management from Baylor University and a Certified Employee Benefits Specialist designation from the International Foundation of Employee Benefit Plans.
Can Distributional Cost Effectiveness Analysis (DCEA) Actually Inform and Improve Health Equity in Oncology? Perspectives From the Methodologist, Physician, and Payer
Digital Conference Pass
Level: Intermediate
ISSUE: The World Health Organization (WHO) defines health equity as the absence of unfair, avoidable, or remediable differences in health among population groups defined socially, economically, demographically, or geographically. According to this definition, an increase in distributional fairness in health outcomes across these groups implies a positive health equity impact. Attention and commitment to improving health equity has grown, as evidenced by initiatives like the Centers of Medicare and Medicaid Services (CMS) updating the basis of 2027 Star Ratings to “Health Equity Index” (HEI) from the current “Reward Factor”. Consequently, methods like DCEA, have evolved from conventional CEA to also quantify health equity impact. This session will feature a debate among a methodologist, physician, and payer on whether and how DCEA can inform and improve health equity, with an oncology focus.
OVERVIEW: DCEA is an intuitively appealing extension of conventional CEA to quantify health inequality impact of a medical intervention. The purpose of this panel is to posit the question, how far can DCEA go in informing health equity-related decision-making in oncology from multi-dimensional perspectives including a methodologist, clinician, and payer. After a session introduction and opening statement on the oncology context of DCEA (Grossman ~5 minutes), panel members will discuss its methodological considerations (Jansen), clinical relevance (Trinh), and payer value (Lopes) (~10 minutes each). Subsequently, panel members will have a debate led by the moderator (25 minutes). In addition to audience questions, the following will be addressed: Is DCEA credible, useful, implementable, impactful, or even the right measure for health equity? How does DCEA fit in with existing frameworks that address health equity such as CMS AHEAD Model, Enhancing Oncology Model (EOM), and National Committee for Quality Assurance (NCQA) Health Equity Accreditation for health plans? How can methodologists best work with physicians and payers to operationalize and optimize DCEA in practice?
Moderators
Jamie Grossman, PhD, MBA
Bayer Pharmaceuticals, Westerville, OH, USA
Jamie Partridge Grossman is a health economist who works at Bayer Pharmaceuticals in global market access.
Panelists
Jeroen Jansen, PhD
University of California – San Francisco, San Francisco, CA, USA
Jeroen P Jansen PhD is a methodologist working at the intersection of evidence synthesis, biostatistics, and health economics.
He is an Associate Professor in the Department of Clinical Pharmacy in the School of Pharmacy at the University of California, San Francisco and Chief Scientist − Health Economics & Outcomes Research at the Precision Medicine Group.
For the past 15 years, Dr. Jansen has worked on research to understand the clinical and economic value of healthcare interventions. His research has frequently been conducted in the context of health technology assessment (HTA) with a focus on comparative effectiveness and cost-effectiveness. Prompted by the challenges encountered in applied research projects, he has performed methodological research. Notable contributions are the development of novel statistical methods to overcome the typical challenges in model-based cost-effectiveness evaluations characterized by gaps in the evidence base and complex evidence structures. Furthermore, Dr. Jansen led initiatives to develop guidance for consumers and producers of network meta-analysis studies. He has promoted a more transparent and credible approach to model-based health economic evaluations and led the development of open-source simulation models to illustrate its feasibility. Furthermore, Dr. Jansen has been involved in the ongoing development of an R software package to develop simulation models for health economic evaluations.
His current research interests are the clinical and economic value of precision medicine, incorporating health disparities in health economic modeling studies, and statistical methods for evidence synthesis.
He has published extensively in his areas of expertise and is widely cited. He is co-author of a textbook on network meta-analysis for decision-making and was associate editor for the Journal for Research Synthesis Methods. Dr. Jansen has a PhD in epidemiology from the Erasmus University in the Netherlands.
Maria Lopes, MD, MS
MMDLOPES, LLC, Cresskill, NJ, USA
Dr. Lopes has been in the managed care industry for over 20 years. She has served as Chief Medical Officer of CDMI/Magellan, Chief Medical Officer GHI/EmblemHealth, Chief Medical Director of Horizon BCBS NJ and CMO of AMCHealth. Dr. Lopes is an Obstetrician and Gynecologist. She has a BA in Biochemistry from Wesleyan University, received her Medical Degree from the University of Connecticut and has a Masters Degree in Administrative Medicine from the University of Wisconsin.
Quoc-Dien Trinh, MD, MBA
Brigham and Women’s Faulkner Hospital, Chestnut Hill, MA, USA
Quoc-Dien Trinh, MD, MBA, is the Section Chief of Urology at Brigham and Women’s Faulkner Hospital, an Associate Professor of Surgery at Harvard Medical School, and Co-Director of the Dana-Farber/Brigham and Women's Prostate Cancer Center. Dr. Trinh is the 2021 recipient of the Outstanding Citizenship Award of Brigham and Women’s Hospital and the 2022 American Urological Association Young Urologist of the Year. He is a Core Faculty and member of the Internal Advisory Board at the Center for Surgery and Public Health, a joint program of Harvard Medical School and the Harvard School of Public Health. Dr. Trinh’s research focuses primarily on inequity and outcomes of cancer care delivery. He co-founded the Mass General Brigham Prostate Cancer Outreach Clinic, a United Against Racism initiative. His publications include over 500 peer-reviewed articles, book chapters, and videos. His research has been funded by the American Cancer Society, the Conquer Cancer Foundation, the Department of Defense, and the Henry M. Jackson Foundation for the Advancement of Military Medicine. Dr. Trinh received his medical degree from the Université de Montréal in Montreal, Canada, where he also completed his residency training in Urology. He completed his fellowship in minimally invasive urologic oncology at the Vattikuti Urology Institute in Detroit, Michigan.
Podium Session
Patient Impact of Rare Diseases
Digital Conference Pass
Moderator
Nabin Poudel, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
P52: Burden of the Untreated Attacks and Its Impact on Social, Mental and Physical Health
5:45PM - 6:00PM
Radojicic C1, Busse P2, O'Connor M3, Danese S4, Ulloa J4, Desai V5, Andriotti T6, Audhya P6, Christiansen S7
1Duke University School of Medicine, Cary, NC, USA, 2The Mount Sinai Hospital, New York, NY, USA, 34Allergy, Asthma, & Immunology Relief of Charlotte, Charlotte, NC, USA, 4Outcomes Insights, Agoura Hills, CA, USA, 5University of California, Los Angeles, Santa Monica, CA, USA, 6KalVista Pharmaceuticals, Inc., Cambridge, MA, USA, 7University of California San Diego, La Jolla, CA, USA
OBJECTIVES: HAE Management Guidelines recommend all attacks be considered for treatment to prevent progression and optimize outcomes. Despite availability of on-demand therapies, patients do not universally treat attacks. We examined the burden of untreated attacks and its impact on social, mental, and physical health.
METHODS: Individuals with HAE-C1-INH (target n=20) recruited by the US HAE Association completed a 20-minute self-reported online survey. Participants >18yrs old whose last attack (≤3 months prior) was not treated with on-demand treatment, were eligible. The impact of the last attack was assessed using a modified Hereditary Angioedema Quality of Life Questionnaire.
RESULTS: Twenty respondents (80% Type 1 HAE-C1-INH; mean age 39yrs; 75% female; 11 [55%] non-androgen long-term prophylaxis [LTP] users; 9 [45%] on-demand treatment only [OD] users) reported having an average of 10 attacks over the past year, of which only 21% were treated. Fourteen respondents (70%) described their last untreated attack as mild at onset and of these, 7 (50%) progressed to moderate/severe. Five attacks (25%) spread to other locations, including 1 to the larynx and 1 to the face. Fourteen (70%) patients indicated that their last untreated attack had an impact on their energy levels (medium/severe impact indicated by 36% OD [3/9] and 36% LTP [4/11] patients). Twenty-two percent OD (2/9) and 36% LTP (4/11) patients felt socially isolated, 22% OD (2/9) and 55% LTP (6/11) felt reluctant to go out in public, and 5 respondents (25%) felt like a burden to others because they needed help during their last untreated attack.
CONCLUSIONS: Patients (including those taking LTP) reported that untreated attacks often progressed in severity, migrated to other locations, and were associated with social isolation and impact on physical/mental health. Results emphasize the need for greater education on the implications of not treating HAE attacks and support guidelines that all attacks should be considered for treatment.
P50: Increasingly Stringent Clinical Responses and the Association with Improvements in Pain, HRQoL, and Work Productivity in Patients with Hidradenitis Suppurativa: 16-Week Results from Phase 3 Studies BE HEARD I&II
5:15PM - 5:30PM
Gottlieb AB1, Mørup M2, Davis L3, Lukowski B4, Lambert J5, Horvath B6
1The Icahn School of Medicine at Mount Sinai, New York, NY, USA, 2UCB Pharma, Copenhagen S, 84, Denmark, 3UCB Pharma, Raleigh, NC, USA, 4UCB Pharma, Brussels, Belgium, 5UCB Pharma, Colombes, France, 6University Medical Center Groningen, Groningen, Netherlands
OBJECTIVES: Hidradenitis suppurativa (HS), a chronic inflammatory skin disease, is associated with reduced quality of life (QoL) and work productivity.1,2 The association between increasingly stringent efficacy responses and changes in patient-reported pain, health‑related QoL (HRQoL), and work productivity was examined in patients with moderate to severe HS.
METHODS: This post hoc analysis used pooled data from the phase 3 studies BE HEARD I&II.3,4 Patients were pooled regardless of treatment (placebo, bimekizumab 320mg every 2 or 4 weeks), and grouped by achievement of mutually exclusive HS Clinical Response (HiSCR) levels (<50% improvement from baseline [<HiSCR50]; 50–<75% improvement [HiSCR50–<75]; 75–<90% improvement [HiSCR75–<90]; 90–100% improvement [HiSCR90–100]) at Week 16.
Associations between level of increasingly stringent efficacy response and improvement in patient-reported outcomes (HS Symptom Daily Diary Worst Skin Pain, EQ-5D Utility, HS QOL [HiSQOL] scores, and Work Productivity and Activity Impairment [WPAI] Percent Overall Work Impairment) to Week 16 were assessed. Observed case data are reported.
RESULTS: In BE HEARD I&II, 1,014 patients were randomized to placebo or bimekizumab; 915 (90.2%) completed Week 16.
At Week 16, patients achieving increasingly stringent HiSCR responses demonstrated sequentially numerically greater mean (95% confidence intervals) improvements from baseline in Worst Skin Pain score (<HiSCR50: −0.9[−1.2,−0.7]; HiSCR50–<75: −1.6[−2.0,−1.2]; HiSCR75–<90: −2.4[−2.9,−1.9]; HiSCR90–100: −2.8[−3.3,−2.3]). Similar improvements were seen for EQ-5D Utility and HiSQOL scores. Increasingly stringent HiSCR response was associated with numerically greater improvements in WPAI Percent Overall Work Impairment (<HiSCR50: −7.6[−11.1,−4.2]; HiSCR50–<75: −14.3[−19.8,−8.8]; HiSCR75–<90: −17.9[−23.1, −12.6]; HiSCR90–100: −21.3[−26.8,−15.9]). CONCLUSIONS: Patients with HS achieving increasingly stringent clinical response outcomes had greater improvements in pain, HRQoL, and work productivity. Pain and work productivity improvements were up to ~3x greater in HiSCR90–100 responders vs <HiSCR50 responders.
1. Zouboulis CC. Dermatology 2015;231:184
–90;
2. Chernyshov PV, et al. J Eur Acad Dermatol Venereol. 2023;37:954
–64;
3. BE HEARD I: www.clinicaltrials.gov/study/NCT04242446; 4. BE HEARD II: www.clinicaltrials.gov/study/NCT04242498.
P49: Examination of Age-Related Medical Complications Among Pediatric, Adolescent, and Young Adult Patients with Sickle Cell Disease in Texas Medicaid
5:00PM - 5:15PM
Odonkor G1, Barner JC2, Kang HA2, Lawson K2, Fasipe T3
1The University of Texas at Austin, Austin , TX, USA, 2The University of Texas at Austin, Austin, TX, USA, 3Baylor College of Medicine/Texas Children's Hospital, Houston, TX, USA
OBJECTIVES: Patients with sickle cell disease (SCD) experience several severe and life-threatening complications across their lifespans. Thus, information on the clinical course of the disease is essential in developing interventions to improve the care of these patients. This study aims to investigate the number, type, and duration of SCD-related complications by age group among pediatric, adolescent, and young adult patients with SCD in Texas Medicaid.
METHODS: Texas Medicaid prescription and medical claims from 03/01/2012 to 05/31/2021 were retrospectively analyzed. Subjects were included if they were aged 2-26 years, had ≥ 3 hospitalizations or outpatient visits associated with an SCD diagnosis, and were continuously enrolled in Texas Medicaid for ≥ 12 months after the index date. Descriptive and inferential (ANOVA, Chi-square, and logistic regression) statistics were used to address the study objectives.
RESULTS: Of the 3,606 patients (mean age=13.8 [±7.4]) included, 66.5% were female and subjects were categorized into six age groups: 2-4 (15.3%), 5-9 (18.4%), 10-14 (16.5%), 15-18 (15.8%), 19-21 (13.5%), and 22-26 (20.6%) years. Overall, the number, type, and duration of complications differed significantly (all p<0.0001) by age group. Patients aged 10-14 years had the highest mean number of encounters for complications, organ-systems affected, and chronic complications, while those aged 22-26 years had the lowest mean number of encounters for complications. Patients aged 5-9 years had a 23% significantly lower likelihood of experiencing vaso-occlusive crises, respiratory infections, chronic anemia, or acute chest syndrome, compared to those aged 2-4 years (Odds Ratio [OR]=0.774, 95% CI=0.604-0.993, p=0.0441), while controlling for other covariates.
CONCLUSIONS: Patterns of complications in children, adolescents, and young adults with SCD differ significantly by age group, especially for those aged 10-14 years and 22-26 years. Better access to health care beyond the transition period and the recommended use of preventive therapy are needed to improve the health outcomes of this population.
P51: Characterization of Misdiagnosed Opsoclonus Myoclonus Ataxia Syndrome (OMAS)
5:30PM - 5:45PM
Michaelis M1, Milligan S2
1OMSLife Foundation, Cypress, TX, USA, 2Trio Health Analytics, Belfast, ME, USA
OBJECTIVES: As with other rare diseases, challenges to Opsoclonus Myoclonus Ataxia Syndrome (OMAS) care include misdiagnosis and lack of provider knowledge. Here, we leverage information from the OMAS Natural History Registry to characterize a misdiagnosed population.
METHODS: The OMAS Natural History Registry was developed by OMSLife Foundation and the National Organization for Rare Disorders with support from the FDA. The registry contains demographics and family history, symptoms, diagnosis and disease severity, therapies (behavioral, occupational, physical, speech), and medication details input from the patient and/or caregiver. Study Population: 200 patients with completed demographic, symptoms, and diagnosis surveys. Univariate comparisons: two-sided independent sample t-test (continuous), Pearson χ2 or Fisher’s exact tests (categorical) with subsequent z-test of column proportions. Variables approaching significance (p≤0.1) in univariate analyses or of clinical interest were further assessed by forward stepwise logistic regression.
RESULTS: Of 200 study patients, 113 (57%) were initially misdiagnosed; the remaining 87 (43%) were correctly diagnosed and/or not aware of misdiagnosis. Twenty different misdiagnosed conditions were recorded with Cerebellar Ataxia most common (72/113, 64%). In univariate analyses, the misdiagnosed group had a higher proportion with US residence (91% v. 80%, p=0.028) and autoimmune disease in immediate family (40% v. 20%, p=0.025) and lower proportion of opsoclonus at symptom onset (52% v. 70%, p=0.010). These variables were retained as significant in multivariate analysis. An assessment of conditions necessary for OMAS diagnosis at symptom onset (3 of 4 conditions: opsoclonus, ataxia or myoclonus, behavior change or sleep disturbance, and/or tumor) did not find significant differences between groups in proportions of patients with 1, 2, 3, or 4 of these criteria. In the subset with sufficient conditions (≥3) for diagnosis (47%, 93/200), 52% (48/93) were misdiagnosed.
CONCLUSIONS: Greater OMAS awareness is needed as even when sufficient diagnostic criteria are present, misdiagnosis occurs in half of the cases.
AI and Machine Learning in HEOR
Digital Conference Pass
Sponsor
Moderator
Erkki Soini, MSc (health econ)
ESiOR Oy, Kuopio, 15, Finland
Erkki Soini is CEO and a health economist at ESiOR Oy, Finland (LinkedIn: erkkisoini). ESiOR provides HEOR, evidence generation, and market access services (https://esior.fi/en) and secure processing environment platform SPESiOR (https://esior.fi/en/spesior/). Erkki has won an ISPOR best poster award twice and co-chaired scientific review committee twice. His key interests are in predictive decision analytical modelling, real-world evidence, cost-effectiveness, and value of information. He has been a permanent advisor of Joint Action Towards the European Health Data Space (TEHDAS) and chairs the board of directors at Kuopio Health.
P48: Automating Economic Modelling: Potential of Generative AI for Updating Excel-Based Cost-Effectiveness Models
5:40PM - 6:00PM
Rawlinson W1, Klijn S2, Teitsson S3, Malcolm B3, Gimblett A1, Reason T4
1Estima Scientific Ltd, London, UK, 2Bristol-Myers Squibb, Utrecht, ZH, Netherlands, 3Bristol Myers Squibb, Uxbridge, UK, 4Estima Scientific Ltd, South Ruislip, LON, UK
OBJECTIVES: Using large language models (LLMs) such as Generative Pre-trained Transformer 4 (GPT-4) to edit Microsoft Excel files could revolutionize the way we interact with health economic models. The aim of this study was to assess the accuracy and capability of GPT-4 in automating the adjustment of an HTA-ready Excel cost-effectiveness model (CEM) for muscle-invasive urothelial carcinoma (MIUC) from the setting of one country to another.
METHODS: This adaptation, conducted by humans, was submitted to HTA authorities globally who deemed the model appropriate for decision making. For this case study, GPT-4 was used to adapt the MIUC model from a UK base case to a Czech Republic perspective. Prior to conducting the study, the model received minor updates to improve its interpretability, such as clarifying vague descriptive text. GPT-4 was then provided with natural language instructions and tabular data that described adaptations in a human-oriented manner (without the use of cell references). Based on this, GPT-4 automatically updated input values in the Excel model without human intervention. All edits made by GPT-4 were highlighted, enhancing subsequent review by a health economist. Accuracy was measured by a human checking whether all required adaptations had been performed and whether all updates performed by GPT-4 were correct.
RESULTS: The AI-generated adaptations were performed in 245 seconds. GPT-4 performed 62/64 required updates, and 100% of these updates were performed correctly. This resulted in an overall accuracy score of 97% (adverse event costs, 100% [7/7]; model settings, 100% [2/2]; drug acquisition and administration costs, 82% [9/11]; resource costs, 100% [32/32]; subsequent treatment proportions 100% [12/12]).
CONCLUSIONS: This study demonstrates the technical feasibility of using LLMs to automate the editing of Excel-based CEMs. Given that models are set up clearly, this is a promising early indication that highly accurate edits of input values can be achieved.
P46: Can Large Language Models Simulate HTA Committee Discussions? Findings and Challenges from a Case Study in Neoadjuvant Treatment of Resectable Non-Small Cell Lung Cancer
5:00PM - 5:20PM
Reason T1, Klijn S2, Gimblett A3, Malcolm B4
1Estima Scientific Ltd, South Ruislip, LON, UK, 2Bristol-Myers Squibb, Utrecht, ZH, Netherlands, 3Estima Scientific Ltd, London, UK, 4Bristol Myers Squibb, Middlesex, LON, UK
OBJECTIVES: Health Technology Assessment (HTA) committees play a crucial role in evaluating reimbursement dossiers for healthcare interventions for the routine use of emerging technologies and interventions. These committees comprise members with vast amounts of expertise whose knowledge is not readily available to pharmaceutical manufacturers.
METHODS: We developed a Large Language Model (LLM) based simulation in Python using GPT-4 Turbo to replicate an HTA committee discussion, using a real Economic Assessment Group (EAG) report in non small cell lung cancer (NSCLC) as a reference document. The virtual committee comprised a fixed number of members with varying categorical attributes, including Health Economics and Outcomes Research (HEOR) knowledge, attitudes towards the pharmaceutical industry, occupations and personal perspectives. These attributes were programmatically modified to generate a range of virtual personalities. The LLM facilitated the committee discussion, with each member contributing and continuing the discussion based on their predefined characteristics. Finally, a chair simulated by the LLM (deterministically), summarised the discussions and formulated a final recommendation on the healthcare intervention under review.
RESULTS: The LLM demonstrated capability in generating realistic and coherent committee discussions. Virtual members maintained distinct and consistent personalities, contributing perspectives aligned with their assigned attributes. However it was difficult to sustain seeds of disagreement between members who tended to converge on consensus towards recommending products. The virtual committee chair effectively summarised discussions and made recommendations that were coherent with the rest of the virtual discussion.
CONCLUSIONS: This study highlights the potential and limitations of using LLMs to simulate HTA committee discussions. While LLMs show promise in replicating realistic committee dynamics and maintaining diversity in accordance with distinct member characteristics, further refinement is needed to enhance focus specificity. This approach paves the way for future research in AI applications for training, policy analysis, and exploring decision-making processes requiring committee approval in healthcare settings.
P47: GPT: The Next Frontier in Health Economic Modeling?
5:20PM - 5:40PM
Srivastava T
ConnectHEOR, London, UK
OBJECTIVES: This study aims to explore the application of Generative Pre-trained Transformers (GPT) in developing early health economic models and predicting cost-effectiveness in the context of early-stage Health Technology Assessment (HTA) submission planning and discussions. The focus is on assessing the feasibility and accuracy of GPT for generating foundational data, crucial in informing early pricing strategies and go/no-go decisions before proceeding to comprehensive HTA-compliant models.
METHODS: GPT's advanced natural language processing capabilities using Open AI’s GPT 4.0 were employed to analyze and synthesize data from a diverse range of sources, including clinical trials published report, statistical analysis plan, epidemiological research, and existing economic evaluations. The study primarily used GPT to construct an early health economic model, focusing on identifying cost-effectiveness scenarios, key economic indicators and evidence gaps. Comparative analysis was conducted between GPT-generated early-stage health economic models with a published model to evaluate the consistency and reliability of the AI-driven approach.
RESULTS: GPT-enhanced models exhibited an increased capacity to process diverse datasets, yielding more nuanced early-stage economic assessments. The scenarios identified by GPT closely mirrored those obtained from subsequent, detailed HTA-compliant models. Notably, GPT was effective in pinpointing critical variables impacting cost-effectiveness and providing potential evidence gap that need to be filled prior to HTA submission.
CONCLUSIONS: The study indicates that GPT could significantly contribute to early health economic modeling, enhancing the precision and efficiency of initial cost-effectiveness analyses. This is especially valuable for early decision-making and resource allocation in healthcare technology development. Despite its potential, the study underscores the need for expert oversight in interpreting and applying GPT outputs, ensuring their applicability and relevance to real-world healthcare scenarios. Future research should focus on further integrating GPT into health economic modeling workflows, improving its interpretability, and optimizing its application in the dynamic field of HTA.
Student Research in Economic Evaluation
Moderator
Priti Jhingran, PhD
Genesis Research Group, Hoboken, NJ, USA
With over two decades in the pharma industry, Priti Jhingran has focused on understanding evidence needs and delivering tools/solutions for access decision makers/HTAs. She has led multiple enterprise level initiatives; launched 15+ products; and developed diverse teams of scientists dedicated to the generation, dissemination, and communication of evidence.
P44: Cost-Effectiveness of Eliglustat Tartrate for the Treatment of Type 1 Gaucher Disease in China Using a Semi-Markov Model
5:45PM - 6:00PM
Xin Y1, He X2
1School of Pharmaceutical Science and Technology, Tianjin University, Tianjin, 12, China, 2School of Pharmaceutical Science and Technology, Tianjin University, Tianjin, China
OBJECTIVES: Type 1 Gaucher Disease (GD-1) is an inherited lysosomal storage disorder with severe clinical and socioeconomic impact. We presented an analysis to simulate the long-term cost-effectiveness of Eliglustat Tartrate which was added to the China’s National Reimbursement Drug List (NRDL) in 2023 for the treatment of GD-1.
METHODS: A Semi-Markov model with ten health states from the perspective of Chinese healthcare system was constructed to compare Eliglustat Tartrate versus standard care without Eliglustat Tartrate. Efficacy data and costs were based on published studies, supplemented by expert opinion. Utilities were derived from a nationwide social survey of 85 adult GD-1 patients in 2023. Costs and effects were discounted at 5% per annum and costs were measured in 2023 Chinese Yuan (CN¥). Lifetime costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs) were estimated. Deterministic and probabilistic sensitivity analyses were conducted to test the robustness of the model. In addition, two scenario analysis were conducted in this study: (1) a 3% discount rate; and (2) a societal perspective.
RESULTS: Over the lifetime horizon (70 years), compared to standard care without Eliglustat Tartrate, Eliglustat Tartrate led to incremental costs of CN¥1,202,670 (CN¥ 4,045,103 and CN¥ 2,842,433, respectively) and incremental QALYs of 9.5 (14.9 and 5.4, respectively), with a base-case ICER of CN¥126,613/QALY. Sensitivity analyses showed the results to be robust. After discounting costs and effects by 3% and under a societal perspective, ICERs could decrease to CN¥106,430/QALY and CN¥55,863/QALY respectively.
CONCLUSIONS: Using the threshold of 2 times per capita GDP (2022, CN¥171,396), Eliglustat Tartrate was predicted to improve outcomes and be a cost-effective treatment option compared with standard care without Eliglustat Tartrate for GD-1 patients in China. Considering that Eliglustat Tartrate was added to the NRDL in 2023, the introduction of Eliglustat Tartrate would benefit more GD-1 patients.
P43: Cost-Effectiveness of Talquetamab-tgvs Vs Idecabtagene Vicleucel for Triple-Class Exposed Relapsed or Refractory Multiple Myeloma
5:30PM - 5:45PM
Rong R, Tang T, Shi L
Tulane University, New Orleans, LA, USA
OBJECTIVES: Chimeric Antigen Receptor (CAR) T-cell and bispecific antibodies are emerging treatments for triple-class exposed relapsed/refractory multiple myeloma (TCE RRMM), with improved clinical outcomes at high costs. This study aims to evaluate the cost-effectiveness of Talquetamab-tgvs (Tal) compared to Idecabtagene Vicleucel (Ide-cel) for TCE RRMM from the US payer perspective.
METHODS: A Markov model with a decision tree was developed to assess three regimens: Tal 800μg/kg Q2W (Tal800), Tal 400μg/kg QW (Tal400), and Ide-cel. The model was based on clinical data from the MonumenTAL-1 and KarMMa trials, and assumptions regarding median overall survival of Tal and the long-term survival post treatment. Drug costs were collected from the average wholesale prices on RED BOOK. Other associated healthcare cost and utility data were derived from the literature. A 3% annual discount rate was applied to the costs and utilities. Evaluation metrics included costs, quality-adjusted life years (QALYs), and the incremental cost-effectiveness ratio (ICER) with a willingness-to-pay threshold of $150,000/QALY. Probabilistic and one-way sensitivity analyses were performed.
RESULTS: In the base-case analysis, the Tal800 regimen, compared to Ide-cel, yielded an additional 1.29 QALYs at an incremental cost of $215,322, resulting in an ICER of $167,081 per QALY gained. The Tal400 regimen was associated with incremental QALYs of 0.13 and an incremental cost of $46,537, leading to an ICER of $370,385 per QALY gained. Ide-cel had a 57.8% probability of being cost-effective, while Tal800 had a 36.4% probability. The results of Tal800 were most sensitive to the median overall survival. For Tal400, drug acquisition cost had the greatest impact. Other assumptions, including the age of starting treatments and post-treatment survival projection, also substantially influenced the results.
CONCLUSIONS: Although Tal800 showed an improved effectiveness on cancer outcomes, Talquetamab-tgvs was not a cost-effective option for triple-class exposed relapsed/refractory multiple myeloma at a willingness-to-pay of $150,000/QALY, compared to Idecabtagene Vicleucel.
P41: Cost-Effectiveness of Technologies for the Treatment of Spinal Muscular Atrophy: A Systematic Review of Economic Studies
5:00PM - 5:15PM
Motta-Santos AS1, Noronha KVMDS2, Reis CDB2, Freitas DA3, Carvalho L4, Andrade MV2
1University of Southern Queensland, Toowoomba, QLD, Australia, 2Universidade Federal de Minas Gerais, Belo Horizonte, Brazil, 3Universidade José do Rosário Vellano (UNIFENAS-BH), Belo Horizonte, Minas Gerais, Brazil, 4Hospital das Clínicas da Universidade Federal de Minas Gerais, Belo Horizonte, Brazil
OBJECTIVES: This systematic review aims to systematically collect data on cost-effectiveness analyses that assess technologies for spinal muscular atrophy (SMA) and evaluate their recommendations.
METHODS: A structured electronic search was conducted in four databases. Additionally, a complementary manual search was conducted. Complete economic studies that evaluated nusinersen, risdiplam, onasemnogene abeparvovec (OA), and the best support therapy (BST) from the health system’s perspective were selected. The incremental cost-effectiveness ratios were compared to various thresholds for the analysis. The review was registered a priori in PROSPERO (CRD42022365391).
RESULTS: Twenty studies were included in the analyses. They were all published between 2017 and 2022 and represent the recommendations in eight countries. Most studies adopted 5, 6, or 10-state Markov models. Some authors took part in multiple studies. OA, risdiplam, and nusinersen were considered inefficient compared to the BST. Risdiplam and OA were generally regarded as cost-effective when compared to nusinersen. Since nusinersen is an inefficient drug, no recommendation can be derived from this result. Risdiplam and OA were compared in two studies that presented opposite results.
CONCLUSIONS: Nusinersen, risdiplam, and OA are being adopted worldwide as a treatment for SMA. Despite that, the cost-effectiveness analysis shows that the technologies are inefficient compared to the BST. The lack of controlled studies for risdiplam and OA hamper conclusions about their face-to-face safety and efficiency.
Breakouts: IP, WS, & OBS
Quantify to Qualify: A Quantitative Bias Analysis Workshop to Bust Bias and Drive Trustworthy Data-Driven Decision Making in Real-World Evidence
Digital Conference Pass
Level: Introductory
PURPOSE:
Attendees will gain solutions for the challenge of bias with applications of quantitative bias analysis (QBA) in real-world data (RWD) studies. Participants will walk away with a list of methods for applying QBA to inform real-world evidence (RWE) use cases including early drug development decision making and high-quality submissions to HTA bodies. DESCRIPTION:
RWD/RWE is increasingly used to inform decision making in pharmacoeconomic and health outcomes research. However, RWD studies often involve risk from multiple sources of bias (selection bias, measurement errors, and unmeasured confounding), which may lead to erroneous conclusions if unaccounted for. QBA provides a principled approach to quantify the effects of these biases. However, QBA is not routinely implemented in RWD studies, partially due to limited knowledge or accessibility of the methods. Thus, it is essential for researchers to understand how to implement QBA for high-quality RWE studies. This will be useful for researchers working across therapeutic areas answering questions ranging from natural history to comparative-effectiveness. Speakers will share pragmatic use cases for QBA at various stages of drug development and decision making. Based on his experience as a NICE scientist, Dr. Duffield will discuss the potential value and challenges of using different QBA results in HTA decision making. Dr. Wang will provide insights into systematically quantifying measurement errors that can impact a RWD study’s conclusion. Ms. Hsu will provide examples using QBA methods to address challenges with real-world synthetic control arms in assessing comparative effectiveness. Dr. Calip will present applied QBA use cases supporting decision-making in oncology early development, including bias analyses for contextual RWE in FDA diversity plans and orphan drug designation planning. Throughout the workshop, live-time polling and in-person and online questions-and-answers will be used for interactive discussions with the audience. The workshop will be relevant to industry and academic researchers, decision makers and methodologists.
Discussion Leaders
Stephen Duffield, PhD, MD
National Institute for Health and Care Excellence, Liverpool, LAN, United Kingdom
Stephen is Associate Director of Real-world methods at NICE. His role involves the continuing development of NICE’s real-world evidence (RWE) framework, collaboration on RWE demonstration projects, and helping to transform NICE’s use of real-world data across guidance products. He is also involved with upskilling individuals within and externally to the organisation, contributing to training workshops and technical forums. Stephen has a degree in medicine and a PhD in public health. Previously, he worked as a clinical doctor and a guideline developer in NICE Centre for Guidelines
Discussants
Gregory Calip, PharmD, MPH, PhD
AbbVie Inc., Chicago, IL, USA
Gregory Calip is an experienced cancer epidemiologist with over a decade of experience in academia and the healthtech industry. After completing his PharmD and MPH in Biostatistics, he received his PhD in Epidemiology from the University of Washington and completed postdoctoral training at the Fred Hutchinson Cancer Center in Seattle. He was previously Associate Professor at the University of Illinois Chicago in the Center for Pharmacoepidemiology and Pharmacoeconomic Research where his NIH-funded research focused primarily on health inequities in multiple myeloma and cancer treatment-related cardiotoxicity. More recently, at Flatiron Health, Greg was Quantitative Sciences Head of Academic Health Systems and Principal Quantitative Scientist in the Flatiron Health Research Unit, where he conducted research focused on the impacts of COVID-19 on oncology real-world evidence, propensity score calibration methods for missing data, and the representativeness of oncology clinical trials. Greg is currently Director of Global Epidemiology (Oncology) at AbbVie and adjunct faculty at the USC Alfred E. Mann School of Pharmacy and Pharmaceutical Sciences in the Program on Medicines and Public Health.
Grace Hsu, MSc
Cytel Inc., Waltham, MA, USA
Grace is Director of Real-World Evidence at Cytel with 9 years of experience in consulting and guiding project strategies. She holds a Master's degree in Statistics, providing statistical consulting and strategy development for data curation, and the application of advanced analytics to clinical and RWD. Examples of her peer-reviewed publications include work on COVID-19, synthetic/external control arm comparative effectiveness analysis, quantitative bias analysis, Bayesian borrowing and other methods of indirect comparison for both pharmaceutical research and HTA/regulatory submissions.
Xiaoliang Wang, PhD, MPH
Flatiron Health, Inc., New York, NY, USA
Dr. Xiaoliang (Wendy) Wang received her PhD in Epidemiology at the University of Washington and MPH in Epidemiology at the Columbia University. Her research interests include cancer epidemiology, risk prediction and statistical genetics. As a Senior Research Scientist at Flatiron Health, she leads research collaborations on various topics, including treatment patterns, health outcomes, quality of care and health equity research. She has also led several studies by leveraging real-world data to enhance survival extrapolation, evaluate the impact of outside scan documents and prospective validation of ML prediction models. Her work has been published in JCO, JAMA Oncology, JAMA Network Open, Cancer, CEBP, JCO CCI and Value in Health.
Outside Help: Can Bayesian Borrowing Help Supplement Limited Sample Sizes in Pediatric and Rare Disease Trials While Mitigating Risk of Bias?
Digital Conference Pass
Level: Intermediate
PURPOSE: Bayesian borrowing methods are seeing growing uptake in regulatory settings for supplementing limited sample sizes in rare indications–particularly in pediatric populations. These methods can allow for the amount of information borrowing to depend on the degree of heterogeneity across data sources, with greater borrowing from the external data when outcomes are compatible across data sources and down-weighting of the external data when outcomes are incompatible. This dynamic borrowing approach has also been used to partially borrow information across different subpopulations in basket trials, to supplement limited sample sizes in pediatric trials by borrowing from adult trial populations, and to augment small concurrent control arms in randomized controlled trials. The goal of the workshop will be to introduce these methods to a health economics and outcome research (HEOR) audience and outline some recent applications. Lastly, potential for application of these methods to support future health technology assessments (HTA) will be discussed.
DESCRIPTION: Dr. Arora will provide a brief introduction to the topic (5 minutes). Dr. Springford will outline the challenges faced when evaluating novel therapies targeting rare indications, including challenges of recruiting enough patients to power trials, survival data immaturity, and potential barriers to conducting randomized controlled trials, and will provide an introduction to Bayesian borrowing methods, including static and dynamic borrowing approaches and use of Bayesian priors. (15 minutes) Ms. Mackay will provide an introduction and overview of several prior-based and hierarchical modelling approaches that have seen recent application in regulatory and HTA settings. (15 minutes) Dr. Dayimu will then discuss an application to economic evaluation in a basket trial setting. (15 minutes). The session will conclude with a Q&A / panel discussion on the potential for Bayesian borrowing methods in future HTA applications. An interactive smartphone-based polling feature will be used to encourage audience participation.
Discussion Leaders
Paul Arora, PhD
University of Toronto, Toronto, ON, Canada
Paul is an epidemiologist and status assistant professor at the University of Toronto in the Dalla Lana School of Public Health. He has over 15 years of experience in conducting and leading evaluations in public health and clinical epidemiology. He holds PhD in epidemiology and has a strong background in biostatistics and scientific writing, which he applies to generate real-world evidence for health outcomes and policy decisions. He was a co-founder and CEO of Lighthouse Outcomes, a consulting firm that specialized in advanced analytics for clinical epidemiology research, which was acquired by Cytel in 2020. At Cytel, he served as a vice president and led the advanced analytics group in the real-world evidence division, where he worked on the application of cutting-edge epidemiologic methods and machine learning to health outcomes problems. He also continues to teach and mentor students and researchers in the Division of Epidemiology, where he has published more than 50 peer-reviewed scientific articles and developed software for Bayesian network meta-analysis of clinical trials. He is passionate about advancing the field of epidemiology and improving health outcomes through rigorous and innovative research.
Discussants
Alimu Dayimu, PhD
University of Cambridge, Cambridge, CAM, United Kingdom
Emma Mackay, MA, MSc
Inka Health, Toronto, ON, Canada
Emma Mackay is a Scientific Advisor in Statistics at Inka Health, specializing in Bayesian methods. Her recent work has focused on the application of Bayesian borrowing to address challenges of evaluating efficacy/effectiveness in rare disease settings. She has extensive experience in HEOR analytics with expertise in meta-analysis, survival modelling, synthetic control arms, and population-adjusted indirect comparisons.
Aaron Springford, PhD
Cytel, Mississauga, ON, Canada
Aaron Springford is an accredited Statistician with experience collaborating with scientists and businesses across a variety of domains in academia and industry. He is currently a Research Principal (Statistics) at Cytel. He holds a PhD in Statistics from Queen’s University at Kingston, specializing in time series analysis and Bayesian methodology, and a Master of Resource Management degree from Simon Fraser University, specializing in spatio-temporal analysis, numerical simulation, and management strategy evaluation. Aaron has been a statistical consultant on projects ranging from the development of the Canadian Air Quality Health Index to management of endangered species and carbon offset crediting, and worked previously as a Statistician for Weyerhaeuser in Seattle, WA.
Assessing Medication Adherence With Group-Based Trajectory Modeling
Digital Conference Pass
Level: Intermediate
PURPOSE: Medication adherence is an important determinant of patient outcomes, their quality of life, and overall healthcare utilization. Identifying prevalent adherence patterns can assist decision-makers in targeting these populations and tailoring interventions to enhance medication adherence; however, current evaluation of adherence is limited. For instance, the Pharmacy Quality Alliance (PQA) organization has endorsed the use of the proportion of days covered (PDC) as the primary method of measuring adherence. While PDC is easy to calculate, there are several drawbacks to using it as the primary measure of medication adherence. As an alternative, group-based trajectory modelling (GBTM) that can determine and cluster groups of patients based on their dynamic adherence patterns may be better suited to study the dynamic nature of medication adherence behaviours. In this workshop, we aim to introduce concept of GBTM and provide a step-by-step overview on how to utilize these models to study medication adherence. At the end of this workshop, participants will gain a better understanding of how to conduct GBTM with R.
DESCRIPTION: Workshop participants with intermediate-level subject knowledge will be familiarized with key considerations when assessing medication adherence using GBTM. Dr. Park will begin the workshop by introducing the concept of medication adherence and the limitations of PDC (10minutes). Next Dr. Thorlund will introduce the concept of GBTM and other latent trajectory analyses (10-minutes). Ms. Berringer will demonstrate how GBTM can be used to study medication adherence using a case study and pre-population R codes (20 minutes). Dr. Metcalfe will conclude by discussing the Reporting Guidelines for GBTM and other latent trajectory analyses and ways to avoid common pitfalls of GBTM (10-minutes). In addition to conducting real-time polling to assess the audience's experience with medication adherence studies, we will allocate time for questions (10 minutes).
Discussion Leaders
Jay Park, PhD
McMaster University, Hamilton, ON, Canada
Jay Park is the Founder and Scientific Lead of Core Clinical Sciences (CCS) and an Assistant Professor at McMaster University. Jay specializes in innovative trial designs, decision analytics, and evidence synthesis methods. At CCS, Jay helps clients explore and apply appropriate study designs and address difficult clinical development and reimbursement problems. Jay has over 40 publications and the lead author of the book “Introduction to Adaptive Trial Designs and Master Protocols” with the Cambridge University Press. Prior to his work with CCS, he has worked with larger consultancies. At McMaster University, Jay has taught graduate-level courses on adaptive trial designs and master protocols.
Discussants
Heather Berringer, MSc
Core Clinical Sciences, saanich, BC, Canada
Heather Berringer, MSc PhD(c), is a Statistician on the Core Clinical Sciences team. She currently is working on projects and methodological development for simulation-guided designs, real-world analytics, indirect treatment comparison methods, and decision analytics for multi-criteria decision making. At Core Clinical Sciences, Heather is leading our Multi-Criteria Augmented Decision Analysis methodological development that can combine multiple benefit and risk parameters from various evidence sources for comprehensive drug benefit-risk assessment.
Prior to joining Core Clinical Sciences, Heather has worked at Cytel’s Advanced Analytics Division as a Statistician. Heather has trained in population dynamics, clinical trial design, causal inference, population dynamics, survival analysis, and Bayesian statistics. With a pure mathematics undergraduate degree from the University of Calgary and a MSc in Mathematical Statistics from the University of Cambridge, Heather brings wide-ranging theoretical understanding and statistical experience to developing novel statistical methods in clinical settings.
Rebecca Metcalfe, PhD
Core Clinical Sciences, Calgary, BC, Canada
Rebecca Metcalfe, PhD is a Senior Scientist, Advanced Epidemiology on our team with more than 15 years of experience in health research. A mixed-methods researcher, she blends techniques from psychology, epidemiology, and health economics to explore stakeholder priorities and generate actionable insights.
Focusing on decision science, Rebecca has worked with stakeholders and patients in diverse health contexts including: oncology; neurological disease; maternal health; mental health; and general well-being. Much of her work has focused on patient and stakeholder preferences, and implementing user experience (UX) principles to design digital health applications. Rebecca’s approach to health research is shaped by her applied training in clinical psychology and her work designing clinical care pathways at the Center for Addiction and Mental Health in Toronto. Her experience reinforced the importance of patient voices in health research and she has given numerous presentations and workshops on this topic.
Prior to joining CCS, Rebecca was responsible for developing and implementing the patient engagement strategy for a national health initiative. She holds an MA in Clinical Psychology, a PhD in Population and Public Health, and completed a postdoctoral fellowship at the University of British Columbia.
Kristian Thorlund, MSc, PhD
McMaster University, Hamilton, ON, Canada
Kristian Thorlund, PhD, is a trained statistician with specialization in Bayesian statistics and complex models for evidence synthesis and health care decision-making. He has published extensively and led the development of several methodologies and software platforms in data sciences and biomedical research that are now being used widely
Combatting Truth-Telling Disincentives in Stated-Preference and Value-Set Surveys
Digital Conference Pass
Level: Introductory
PURPOSE:
This workshop will provide participants with tools to identify causes of uninformative or misleading responses in stated-preference and value-set surveys and guides participants through alternative strategies to encourage truthful responses. Participants become familiar with relevant findings on incentive-compatible study designs from market research and environmental economics and evaluate how to apply these findings in health applications.
DESCRIPTION:
When asked the question “What would you do if you had to make this choice?” a natural response could be: “This researcher is asking me to think hard about a speculative, emotionally uncomfortable situation with no reward for my effort.” This problem generally is known as “incentive incompatibility” in survey research. At best, noisy data from incentive-incompatible surveys produce imprecise parameter estimates. At worst, data from such surveys can be systematically biased from satisficing response strategies that economize on time and effort required to complete the surveys. Workshop presenters will take 15 minutes each to discuss important considerations when designing incentive-compatible preference studies with methods such as “Bayesian truth serum” and game-theoretic “cheap talk.” Dr. Gebben will provide an overview of research on truth-telling in survey research, including the extensive research on improving veracity in nonmarket-valuation studies. Dr. Johnson will discuss implications for designing choice-experiment and threshold-method surveys for benefit-risk, stated-adherence, and value-set studies. Dr. Gonzalez will share results from surveys on Olympic-athlete doping decisions, preferences for novel weight-loss devices, and stated adherence to cystic-fibrosis antibiotic treatment.
Each audience member will respond to two versions of a survey to simulate the effects of incentive-incompatible and incentive-compatible survey designs. Panel members will lead a discussion comparing the results of the two designs. (15 minutes) This workshop will be of considerable value to industry and regulatory users of preference data, as well as both less-experienced and more-experienced researchers.
Discussion Leaders
Juan Gonzalez, PhD
Duke Clinical Research Institute, Durham, NC, USA
Dr. Gonzalez is an Associate Professor in the Department of Population Health Sciences at the Duke University School of Medicine. He is an expert in the design of stated-preference survey instruments and the use of advanced statistical tools to analyze stated-preference data. His research has focused on two main areas: 1) transparency in benefit-risk evaluations of medical interventions, and 2) elicitation of health preferences from multiple stakeholders to support shared decision making.
Dr. Gonzalez Co-led the first FDA-sponsored preference study. The study was highlighted in FDA’s recent precedent-setting guidance for submitting patient-preference evidence to inform regulatory benefit-risk evaluations of new medical devices. More recently, Dr. Gonzalez collaborated with the Medical Devices Innovation Consortium (MDIC) to prepare the first catalog of preference-elicitation methods suitable for benefit-risk assessments of medical devices. The catalog was part of the Patient-Centered Benefit-Risk Assessment Framework developed by MDIC. As a core group member of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Conjoint Analysis Task Force, Dr. Gonzalez helped draft good-practice recommendations for statistical analysis, interpretation, and reporting of health preference data. Dr. Gonzalez is the new Chair of the International Academy of Health Preference Research (IAHPR) and past Chair of the ISPOR special interest group on stated-preference research.
Discussants
David Gebben, PhD
U.S. Food and Drug Administration, Silver Spring, MD, USA
David Gebben, PhD is a research economist focusing on stated preference methods applied within a health care context. Specifically, how stated preference methods can advance regulatory science to inform benefit risk evaluations, clinical trial endpoints, and inform ordering of patient priorities. David has worked at the FDA since June 2018.
Reed Johnson, PhD
Department of Population Health Sciences, Durham, NC, USA
F. Reed Johnson, PhD, has 50 years of academic and research experience in health and environmental economics. He currently is Professor in the Departments of Population Health Sciences and Medicine, Duke School of Medicine, and the Duke Clinical Research Institute. He led the first FDA-sponsored study to quantify patients’ willingness to accept benefit-risk tradeoffs for new health technologies. In 2018 the International Society for Pharmacoeconomics and Outcomes Research awarded him the Donabedian Outcomes Research Lifetime Achievement Award.
6:00 PM - 7:00 PM
ISPOR Women in HEOR Networking Event
Stop by the ISPOR Booth to continue the conversation from today’s Women in HEOR session and network with other supporters of this important ISPOR initiative! All conference attendees are welcome!
Networking Reception
End the second day of the conference at the Networking Reception where connections are made, ideas are sparked, and collaborations begin to flourish sponsored by Precision Value & Health.
Wed May 8
7:00 AM - 8:00 AM
Coffee and Connect
It may be the last day, but there is still so much to learn! Head to the breakout rooms for the final session banks and an opportunity to share your thoughts with fellow attendees. Provided by ISPOR.
7:00 AM - 1:00 PM
Registration Hours
8:00 AM - 9:00 AM
Breakouts: IP, WS, & OBS
Gaps in Clinical Outcomes Assessment Measurement in Real-World Studies: Solutions and Applications
Digital Conference Pass
Level: Intermediate
PURPOSE:
Administrative claims data are the conventional source of generating real-world-evidence (RWE) for patient journey evaluation. Significant gaps in understanding patient outcomes exist using this data. Integrating COA’s in real-world-data (RWD) is critical for health care and pharmaceutical organizations to better understand and address patients’ needs, including electronic health records, medical charts, patient/provider surveys. Claims identified samples for RWE data collection enhances study validity. Applications of these findings have meaningful real-world implications for treatments and patient care. Audience participation includes polling questions engaging the audience (e.g., how many audience members have worked with administrative claims data that includes COA's?)
DESCRIPTION:
Dr. Bennett will provide an overview (8min) defining COA’s and associated gaps within the context of RWD: a)Patient reported outcomes (PRO’s) –patient self-report surveys/interviews, b)Observer Reported Outcomes (ObsRO) –observer outcomes, caregiver/parent report c)Clinician reported outcomes (ClinRo) –provider surveys or clinical notes d)Performance outcome (PerfO) assessments on standardized tasks. Dr. Taylor will examine how does EHR data (structured/unstructured) enhance the use of RWD (12 min). Includes the significance of data such as tests ordered, screenings/assessments, AE’s, HCU visits, and clinical notes. Discussion on pros/cons for this strategy. Dr. Tkatch will discuss the use of self-report data collection generating RWE (12min). Assessments measuring provider/patient/caregiver experience. How are claims and surveys used concurrently to generate valuable RWD? Use of claims for patient identification and linking assessments back to claims for HCRU and clinical outcomes analysis. Dr. Rosenblatt will explore applications of COA generated RWE (12min). How is data used to benefit the patient experience? Can RWE COA data impact the development of treatments or the expansion of current treatments? Can RWE help physicians in treatment decisions, and can it be used to inform treatment guidelines? How can RWE be utilized in regulatory applications or decision-making around drug development and approval? Audience participation (20min)
Panelists
Lisa Rosenblatt, MD, MPH
Bristol Myers Squibb, East Brunswick, NJ, USA
Dr. Rosenblatt is a Senior Director of US HEOR at Bristol Myers Squibb. She is responsible of a team of HEOR researchers working on solid tumor therapies in Oncology. She joined BMS in 2006 and has held several roles with increasing responsibility within the US HEOR team across Immunology, Neuroscience and Virology. She also worked in Medical Affairs in the Cardiovascular therapeutic area.
Prior to BMS, Dr. Rosenblatt worked as an HEOR consultant for 4 years with Analytica in NYC.
Dr. Rosenblatt holds an MD from McGill University and an MPH from Columbia University Mailman School of Public Health.
Michael Taylor, PhD
Genenentech, Redwood City, CA, USA
Michael Taylor is Vice President and Global Head of Real World Data Science within the Product Development Data Sciences organization at Genentech/Roche. In this role, he leads a team of quantitative scientists (e.g., epidemiologists, statisticians, outcomes researchers, mathematicians, etc.) focused on developing RWD-based insights to support and improve decision-making for R&D, regulatory, access/reimbursement, and healthcare providers.
Previously, he was the Global Head of RWD Collaborations at Genentech/Roche. In that role he led Roche’s RWD collaborations with Flatiron, Foundation Medicine, and PicnicHealth. During his 18 years with Genentech/Roche he has supported the development of multiple molecules currently marketed or in development for oncology indications. He has participated on panels focused on Real World Data at Friends of Cancer Research meetings and currently serves on the Duke-Margolis Real World Evidence Collaborative Advisory Group.
Michael holds a Ph.D. in Pharmaceutical Sciences (2005) and a Doctor of Pharmacy (2001) from the University of Florida.
Rifky Tkatch, PhD
Optum, Oak Park, MI, USA
Rifky Tkatch, PhD
Director, HEOR OPTUM
Rifky Tkatch is the Director of the Primary Data Collection (PDC) Team on HEOR. She provides oversight of project development and supports the business development activities for the PDC team. Dr. Tkatch has been at Optum since 2015. She is trained as a social and health psychologist with an expertise in research and program design and evaluation, survey development, and quantitative and qualitative analysis. She received her PhD in Psychology from Wayne State University where she also completed an NIH funded pre- and post-doctoral training program at the Institute of Gerontology. Rifky completed her post-doctoral fellowship at the Karmanos Cancer Institute and the Department of Oncology at Wayne State University in Communication and Behavioral Oncology.
Discussion Leaders
Bryan Bennett, PhD, Cpsychol
Jazz Pharmaceuticals, Oxford, NBL, United Kingdom
Dr Bryan Bennett is currently employed by Bristol Myers Squibb as Asset/Indication Lead in the Patient-reported Outcomes Assessment team. He has been involved in Clinical Outcomes Assessments for more than 20 years in clinical practice, academia, consulting and industry.
International Fallout: How the Inflation Reduction Act (IRA) in the US and Joint Clinical Assessment (JCA) and Revision of the EU Pharmaceutical Strategy in Europe Will Have Global Consequences
Digital Conference Pass
Level: Intermediate
PURPOSE:
The session will seek to highlight the consequences of the Inflation Reduction Act (IRA) and Joint Clinical Assessment (JCA) and revision of the EU Pharmaceutical Legislation and the intra- and inter- impact on healthcare systems and R&D.
DESCRIPTION:
IRA (15 minutes, Brixner) Whilst the primary aim of the IRA is to reduce spending for Medicare – its impact will be broader. There will be spillovers to other US markets via the Best Price for the Medicaid Rebate Program, the 340B ceiling price, and the calculation of Average Sales Price. It is likely to affect competitive dynamics in Medicare Part D, with competing products in impacted therapeutic classes facing pressure to match the government-set price, further reducing industry revenue. Will this impact global drug pipelines and thereby influence access across the rest of the world?
JCA and EU legislation (15 minutes, Teale) Is there the potential in Europe for a "perfect storm": JCA with "challenging" PICOs, Value judgements lying outside the scope of JCA, JCA “insufficient” for National HTA, and price driven by affordability and reference pricing "disconnected" from JCA and National HTA. How will JCA impact access to medicines in US and Europe?
What is a pharmaceutical company’s view? (15 minutes, Mettam) Recent substantial landscape changes on both sides of the Atlantic with JCA and the IRA have increased uncertainty for manufacturers. These changes, when brought together with increasing payer pressure in general have made designing evidence generation both more difficult and more important. What are the impacts for manufacturers, and how does it change evidence planning for full lifecycle global access success?
Moderated discussion (15 minutes, Alderson) What will the major areas of global impact of all the above be? What should be done today and tomorrow to ensure these developments lead to a win-win for all stakeholders?
Speakers
Diana Brixner, RPh, PhD, FAMCP
University of Utah College of Pharmacy, Salt Lake City, UT, USA
Dr. Diana Brixner is currently a Professor in the Department of Pharmacotherapy and is a Founder of the Pharmacotherapy Outcomes Research Center ( PORC)at the University of Utah in Salt Lake City, UT. She is a member of both the Huntsman Cancer Institute Cancer Control and Population Sciences and the Center for Genomic Medicine. Prior to her academic appointment in 2002 she worked both in the biotech (NeoRx) and pharmaceutical (SmithKline Beecham, Novartis) industry focused on value assessment of their technologies in the early years of the field. Dr. Brixner was a Past President of both the Academy of Managed Care Pharmacy (AMCP) and International Society of Pharmacoeconomics and Outcomes Research (ISPOR). She has served as a one-year Scholar-in-Residence for the Academy of Managed Care Pharmacy, recently served on the scientific advisory board of Lumanity and is a member of the Executive Board of the International Market Access Society.
Sam Mettam, MSc
Jazz Pharmaceuticals, Bramley, SRY, United Kingdom
Christopher Teale, BSc MSIB MORS
TealeHealth, Chesterfield, Derbyshire, United Kingdom
Chris worked for 25 years within the pharmaceutical industry as Director of Global Pricing and Market Access (Oncology) at Astra Zeneca, Director Health Economics, Pricing, Reimbursement and Access at Allergan, Commercial Planning Manager at Fisons, and Portfolio Planning Manager at Novartis. For 12 years he worked in Consultancy (Bridgehead International, GfK, and Ipsos). In 2023 he founded TealeHealth, a healthcare consultancy offering expert advisory, access, and analytic services across a broad spectrum of Advanced, Genomic, Digital, and Traditional Therapies to leading pricing and market access consultancies and directly to clients.
Discussion Leaders
David Alderson, MBA, MEng
Cogentia Healthcare Consulting, Cambridge, United Kingdom
David is Director leading the market access team at Cogentia. His interest is in problem solving and developing integrated, evidence-based solutions to new and perennial access challenges. He brings a wealth of experience from over 30 years in the industry, within many different multi-disciplinary teams and companies, spanning both industry and consulting, and global and national roles. Prior to Cogentia he worked for Evidera, GfK (now IPSOS), Bridgehead International, AZ/Medimmune, Novartis and GSK, and has been fortunate enough to be involved in a number of merger integrations. David’s focus is on market access strategy, pricing, due diligence and opportunity assessment, evidence development, value communication and messaging, ensuring that access is integrated into early development and portfolio decisions, and facilitating access for innovation.
David has an MBA from Cranfield, a post graduate diploma in marketing, and is a Chartered Engineer.
Skin in the Game or Financial Overdose? Navigating the Future of Patient Cost-Sharing on Prescription Drugs
Digital Conference Pass
Level: Intermediate
ISSUE:
Prescription drug affordability remains a critical challenge for patients in the U.S.; 39% of insured adults aged 19-64 were unable to fill their prescriptions due to cost in 2020 (Commonwealth Fund). Particularly in the commercial market, cost-related affordability issues appear to disproportionately burden sicker patients from lower-income and historically marginalized communities. Escalating financial pressure on patients can lead to decreased medication adherence, adverse health outcomes, and excess healthcare utilization. Barriers in insurance design reduce the real-world value of medical innovation to society. The panel will explore these issues and offer varied perspectives on patient cost-sharing in insurance design and its intersection with the valuation of medical innovation.
OVERVIEW:
Economic moral hazard theory posits that insurance can cause pharmaceutical overuse by lowering costs for the insured. This has spurred the rise of increased patient cost sharing, such as in High-Deductible Health Plans in the U.S. Yet, the shift away from copays towards coinsurance and deductibles – culminating in a record $82Bn patient out-of-pocket costs in 2022 (IQVIA) – remains underexplored in empirical economic research. Since price elasticities have been found to vary considerably across therapy areas and medication classes, uniform levels of coinsurance lack clinical nuance. Additionally, without adjustments for income, people with limited financial resources continue to bear a disproportionate burden. The pressing need to reduce healthcare inequities and to optimize pharmacy benefits in the context of these challenges underscores the imperative for an interdisciplinary dialogue on value: Without innovation in benefit design, even the most transformative treatments will not deliver value to patients. Darius Lakdawalla will discuss moral hazard and economic incentives in US insurance designs, A. Mark Fendrick will talk about dynamic cost exposure and designing value-based insurance. Annette James will discuss equity-enhancing benefits in employer-based insurance. Kimberly M. Hooks will share her experience as a chronic disease patient.
Moderators
Ulrich Neumann, MBA, MSC, MA
Johnson & Johnson, Titusville, NJ, USA
Ulrich Neumann, MBA MSc MA BSc BA FRSA currently serves as Director for Market Access Scientific and External Strategy at Johnson & Johnson Scientific Affairs. At J&J, he is part of a senior research team developing models and evidence for health economic policy evaluations, focusing on domains such as value assessment methodologies, healthcare financing and value-based insurance design. Ulrich previously led the US market access team at the drug development platform Certara in New York and was a Senior Director at the population health intelligence consultancy Analytica Laser. He also founded Pharmvivo Ventures LLC in 2016. Ulrich holds an MBA from Imperial College London, an MSc from the London School of Economics and an MA from the University of Southern California, Annenberg School and Marshall School of Business. His undergraduate degrees are in Public Administration (BSc) and Multi-level Governance (BA). He has published various articles, whitepapers, research reports, and posters on critical pharmaceutical policy and managed care topics. Ulrich is frequently speaking at conferences such World Congress, Reuters or CBI and was the opening speaker at 'eyeforpharma' in 2016. In 2018, Ulrich launched the “Market Access Radar”, a broadcast and global intelligence product on current pharma policy and reimbursement topics. As time allows he enjoys sharing his work via lectures and graduate seminars, such as one he recently led on clinical trial innovation at Rutgers University Irvine Center. A member of AMCP, AMBA, DIA, he sat on the Health Economics Interest Group at Academy Health from 2021-2023 and is a nominated Fellow of the Royal Society of Arts and Commerce since 2014. He lives in New York City.
Panelists
A. Mark Fendrick, MD
University of Michigan, Ann Arbor, MI, USA
A. Mark Fendrick is the Director of the Center for Value-Based Insurance Design (V-BID) and a Professor of Internal Medicine in the School of Medicine and a Professor of Health Management and Policy in the School of Public Health at the University of Michigan. Dr. Fendrick conceptualized and coined the term V-BID and currently directs the V-BID Center at the University of Michigan [www.vbidcenter.org], the leading advocate for development, implementation, and evaluation of innovative health benefit plans. His research focuses on how clinician payment and consumer engagement initiatives impact access to care, quality of care, health care disparities, and health care costs. Dr. Fendrick is an elected member of National Academy of Medicine (formerly IOM), serves on the Medicare Coverage Advisory Committee, and has been invited to present testimony before the U.S. Senate Committee on Health, Education, Labor and Pensions, the U.S. House of Representatives Ways and Means Subcommittee on Health, and the U.S. Senate Committee on Armed Services Subcommittee on Personnel. Dr. Fendrick is the co-editor in chief of the American Journal of Managed Care and is an editorial board member for 3 additional peer-reviewed publications. He remains clinically active in the practice of general internal medicine.
Kimberly Hooks
Patient Advocate, Atlanta, GA, USA
Kimberly Hooks, an Atlanta-based IBD patient advocate, is dedicated to enhancing community health
through advocacy. Diagnosed with Ulcerative Colitis in 2011 and undergoing a proctocolectomy in May
2020, Kimberly has been navigating her IBD journey. Recognizing disparities within minority
communities, she found her life's purpose in sharing her testimony to help others and advocate for
health equity. Actively engaged with the American Gastroenterological Association and Color of
Gastrointestinal Illnesses, Kimberly amplifies the patient's voice in GI care. Serving on multiple patient
advisory boards, she ensures patient perspectives are prioritized. Kimberly's blog, kimberlymhooks.com,
serves as a valuable resource, empowering and educating those living with chronic illness.
Annette James, MAAA, FSA, FCA
American Academy of Actuaries, Reno, NV, USA
Annette V. James is a health actuary and an active volunteer in the actuarial community. She holds several leadership positions with the American Academy of Actuaries. She chairs the Academy’s Health Equity Committee as well as the Actuarial Standards Board’s Health Committee. James is a regular director of the Academy and was a 2021 recipient of an Academy Outstanding Volunteerism Award. She is president & CEO of Ibis Actuarial Consulting, LLC and is a former regulatory actuary specializing in health insurance and healthcare analysis, with a particular focus on financial solvency, financial reporting, healthcare policy and healthcare reform.
Darius Lakdawalla, PhD
USC Leonard D. Schaeffer Center for Health Policy and Economics, Los Angeles, CA, USA
Darius Lakdawalla is a widely published, award-winning researcher and leading authority on health economics and health policy. He holds the Quintiles Chair in Pharmaceutical Development and Regulatory Innovation at the University of Southern California, where he sits on the faculties of the School of Pharmacy and Sol Price School of Public Policy. He is also the Director of Research at the USC Leonard D. Schaeffer Center for Health Policy and Economics, one of the nation’s premier health policy research centers. His research has focused primarily on the economics of risks to health, the value and determinants of medical innovation, the economics of health insurance markets, and the industrial organization of healthcare markets.
Real-World Evidence for Crossover Adjustment: Challenges, Opportunities, and Newly Proposed Methods
Digital Conference Pass
Level: Intermediate
PURPOSE:
1 – Oncology trials often allow those receiving the control treatment to crossover to the experimental treatment at disease progression. Since participants who switch often have a different prognosis than non-switchers, estimating the treatment effect with regards to overall survival can be challenging. Specialized statistical methods (e.g., rank-preserving structural failure time models (RPSFT), inverse probability of censoring weighting (IPCW), two-stage estimation (TSE)) are often recommended for “crossover adjustment”, but these can fall short when sample sizes are small, and the degree of crossover is high.
2 – Given that real-world evidence (RWE) is increasingly used to support applications for regulatory and HTA approvals, the potential of RWE for crossover adjustment is certainly worth considering.
3 – We review examples of how RWE has been used for crossover adjustment in a number of recent studies (e.g., to validate different crossover adjustment methods (Hawkins et al. 2022); to create an external control arm without any crossover contamination (Morschhauser et al. 2023)).
4 – We discuss newly proposed methods to supplement RCT data with RWE for efficient and unbiased crossover adjustment.
DESCRIPTION:
Workshop attendees will develop an understanding of methods for cross-over adjustment and the potential of RWE. The audience will be asked (real-time polling) to consider if RWE has potential to improve cross-over adjustment and if potential biases can be addressed. Ms. Cope will introduce the main ideas and summarize the specialized methods that are often recommended for cross-over adjustment (Part 1 - 10 min). Dr. Jansen will review how RWE has been used in different applications across HTA (Part 2 - 15 min). Prof. Hawkins will then review specific examples of how RWE has been considered for cross-over adjustment (Part 3 - 15 min) and Dr. Campbell will discuss newly proposed statistical methods (15 min). Ms. Cope will conclude leading an audience discussion (5 min).
Discussion Leaders
Shannon Cope, MSc
PRECISIONheor, VANCOUVER, BC, Canada
Shannon Cope, MSc, is a Senior Vice President with PRECISIONheor. Shannon has over ten years of experience consulting for biopharmaceutical industry regarding evidence synthesis and economic evaluations to assess the value of new interventions.
Discussants
Harlan Campbell, PhD
University of British Columbia, Rossland, BC, Canada
In addition to serving as a Principal Scientist/Senior Methodologist at PrecisionHEOR, Dr. Campbell is an Adjunct Professor in the University of British Columbia Department of Statistics. He completed his PhD in the field of Statistics at UBC, researching statistical methods for hypothesis testing and causal inference. His applied research focusses on applications in evidence synthesis, epidemiology and clinical trials.
Neil Hawkins, MSc, PhD, MBA, CStat
University of Glasgow, Oxford, United Kingdom
Neil Hawkins, MSc, MBA, PhD, CStat is a Professor of Health Technology Assessment at the University of Glasgow, Scotland, UK. Previously, he was Vice President leading the global Health Economics practice at ICON PLC and a member of the board of directors at Oxford Outcomes Ltd. He has post-graduate degrees in pharmacology, health economics and applied statistics. He has worked in the pharma, academic, and consultancy sectors in roles spanning basic pharmacological research, clinical development, epidemiology, and health technology assessment. His current research and consultancy focuses on: evidence synthesis and decision-analytic modeling; precision medicine, and estimation of subgroup effects; the use of health technology assessment analyses to inform technology investment and development; and the efficient allocation of capital in pharmaceutical development. He has taught ISPOR short courses since 2008, is a member of the Statistical Methods Special Interest Group, and was part of the Indirect Comparisons Task Force.
Jeroen Jansen, PhD
Department of Clinical Pharmacy, School of Pharmacy, University of California – San Francisco, USA; PrecisionHEOR, Oakland, CA, USA
Jeroen P Jansen PhD is a methodologist working at the intersection of evidence synthesis, biostatistics, and health economics.
He is an Associate Professor in the Department of Clinical Pharmacy in the School of Pharmacy at the University of California, San Francisco and Chief Scientist − Health Economics & Outcomes Research at the Precision Medicine Group.
For the past 15 years, Dr. Jansen has worked on research to understand the clinical and economic value of healthcare interventions. His research has frequently been conducted in the context of health technology assessment (HTA) with a focus on comparative effectiveness and cost-effectiveness. Prompted by the challenges encountered in applied research projects, he has performed methodological research. Notable contributions are the development of novel statistical methods to overcome the typical challenges in model-based cost-effectiveness evaluations characterized by gaps in the evidence base and complex evidence structures. Furthermore, Dr. Jansen led initiatives to develop guidance for consumers and producers of network meta-analysis studies. He has promoted a more transparent and credible approach to model-based health economic evaluations and led the development of open-source simulation models to illustrate its feasibility. Furthermore, Dr. Jansen has been involved in the ongoing development of an R software package to develop simulation models for health economic evaluations.
His current research interests are the clinical and economic value of precision medicine, incorporating health disparities in health economic modeling studies, and statistical methods for evidence synthesis.
He has published extensively in his areas of expertise and is widely cited. He is co-author of a textbook on network meta-analysis for decision-making and was associate editor for the Journal for Research Synthesis Methods. Dr. Jansen has a PhD in epidemiology from the Erasmus University in the Netherlands.
Target Trial Emulation in Practice: An Introduction and Exploration of Application Through Case Studies
Digital Conference Pass
Level: Introductory
PURPOSE: Target Trial Emulation (TTE) represents an advancement in HEOR by offering a rigorous approach to establish treatment effectiveness from observational data, thus overcoming the limitations of traditional real-world evidence (RWE) studies. This workshop will delve into how TTE addresses the critical challenge of bridging the gap between randomized controlled trials and RWE, enhancing the accuracy and applicability of healthcare interventions in diverse patient populations, including case studies involving target trial emulation applications in practice. Topics include (1) how to benchmark studies, (2) per-protocol estimation and what adherence means, (3) external comparators from RWE, and (4) data limitations, including unmeasured confounders and missing data problems
DESCRIPTION: Participants will gain insights into crucial TTE aspects, including benchmarking studies, per-protocol estimation and understanding adherence, leveraging real-world external comparators, and addressing missing data. Winson Cheung will introduce TTE, emphasizing its significance in bridging the gap between observational studies and randomized controlled trials. The discussion leaders will guide participants through real-world case studies illuminating the practical application of TTE. Manuel Gomes will describe the nuances of benchmarking in TTE and its role in ensuring robust study design and interpretation. He will also speak about the intricacies of per-protocol estimation and challenges in determining treatment adherence, with practical insights from relevant case studies. Alind Gupta will describe the use of external comparators in the context of target trial emulation, focusing on their calibration, application and interpretation. Dr Gupta will also address challenges related to data limitations, including unmeasured confounders and missing data. Audience participation will be encouraged through interactive discussions, where attendees can share their perspectives and experiences. We will tailor this workshop for researchers, statisticians, and practitioners interested in gaining practical insights into applying TTE in real-world scenarios.
Discussion Leaders
Alind Gupta, PhD
University of Toronto, Toronto, ON, Canada
Alind Gupta is a researcher and consultant at Cytel in Toronto, Canada and a adjunct lecturer at the University of Toronto. His work focuses on applied methods for observational data analysis in health outcomes research. He has served as an analyst and technical advisor on projects with public and private institutions involving causal inference, quantitative bias analysis, generalizability, treatment policy optimization and risk prediction, and public health interventions for COVID-19 in low- and middle-income countries. He is also leading research initiatives for improving scientific rigour, trust, and transparency in real-world evidence by policy-makers.
Discussants
Winson Cheung, MD, MPH
University of Calgary, Calgary, AB, Canada
Manuel Gomes, PhD
University College London, London, United Kingdom
Manuel Gomes is an Associate Professor of Health Economics at the Department of Applied Health Research, UCL. He holds a MSc in Economics from the University of Porto (Portugal), a MSc in Health Economics from the University of York (UK), and a PhD in Health Economics from the University of London (LSHTM). Before joining UCL, he was an Assistant Professor at the Health Economics, Policy and Technology Assessment group at LSHTM. Manuel has been a Visiting Scholar at the Centre for Health Economics, University of York, and at the Department of Health Care Policy, Harvard University.
He is a member of the International Society for Health Economics and Outcomes Research (ISPOR) expert group on statistical methods for health economics and outcomes research. Additionally, he serves as an Associate Editor for Value in Health and is a member of the Editorial Board for Medical Decision Making.
How Can Grace and Other Novel Methods Estimate ISPOR Value Flower Components? A User Guide to Generalized Cost Effectiveness Analysis (GCEA)
Digital Conference Pass
Level: Experienced
PURPOSE:
The ISPOR Value Flower continues to have a major impact on the assessment of value for healthcare technologies. The petals within the ISPOR Value Flower have become a mainstay in the health economics and outcomes research community, yet the novel methods to quantify each petal are not regularly implemented in value assessment today. This workshop provides a user guide on the novel methods to incorporate the petals within value assessment using a framework referred to as generalized cost effectiveness analysis (GCEA).
DESCRIPTION:
The workshop will begin with an overview of the GCEA framework. Dr. Shafrin will describe how value petals within this framework correspond to four value assessment themes: uncertainty, dynamics, value based on beneficiary identity, and other factors. Dr. Steuten will next discuss how the UK has tried to address these issues using decision modifiers around disease severity and other factors. Then, Professor Towse will explain how Generalized Risk-Adjusted Cost Effectiveness (GRACE) can be incorporated into GCEA to vary treatment value depending on disease severity. Next, Dr. Whittington will explain how stacked cohort models can be used to estimate long-run pricing dynamics, by incorporating genericization and pricing trends over time to more accurately capture the opportunity costs of healthcare interventions. Finally, Dr. Garrison will provide considerations for estimating value from the societal perspective in general and—more specifically—best practices for quantifying real option value. All three Discussants will provide applied examples of these methodologies. By the end of the session, the audience will understand the GCEA framework, which petals correspond to which broader value themes, and the fundamental steps and relevant references for estimating key GCEA petals. Real-time polling will be used to assess the attendees’ knowledge of which petals map to which themes and the methodological step ordering for the key GCEA value petals presented.
Discussion Leaders
Jason Shafrin, PhD
Center for Healthcare Economics and Policy, FTI Consulting, Los Angeles, CA, USA
Jason Shafrin, Ph.D. is a Senior Managing Director at FTI Consulting's Center for Healthcare Economics and Policy. Dr. Shafrin has over 20 years of health economics research experience serving as trusted advisor and expert to a wide variety of healthcare and life sciences companies, governments and non-governmental organizations (NGOs). Dr. Shafrin is an Adjunct Professor at the University of Southern California, the former Director of Research at the Innovation and Value Initiative and the Founder and Editor of the Healthcare Economist blog.
Discussants
Lou Garrison, PhD
The Comparative Health Outcomes, Policy, and Economics Institute, School of Pharmacy, University of Washington, Seattle, WA, USA
Lou Garrison, PhD, is Professor Emeritus in The Comparative Health Outcomes, Policy, and Economics Institute in the School of Pharmacy at the University of Washington, where he joined the faculty in 2004.
Dr. Garrison has worked in non-profit, industry, and academic settings. He received a PhD in Economics from Stanford University and has more than 200 publications in peer-reviewed journals.
Dr. Garrison was elected as ISPOR President for 2016-2017, and currently serves as co-chair of ISPOR’s Policy Outlook Committee for the Health Science Policy Council. In September 2022, he was announced as the recipient of the 2022 Avedis Donabedian Outcomes Research Lifetime Achievement Award from ISPOR.
Lotte Steuten, PhD
Office of Health Economics, London, United Kingdom
Lotte Steuten, PhD, is Deputy Chief Executive of the not-for-profit Office of Health Economics (OHE) and the scientific and business lead for its international research-led work program—maintaining its reputation for objective, innovative, and high-quality research, and meeting its charitable goals. She is also an Honorary Visiting Professor at City, University of London (UK) contributing to its teaching programmes in health economics.
For more than 20 years, Lotte has worked in academic, research, and consulting HEOR roles with one focus — improving healthcare decision-making through high-quality research and analysis. To achieve that, she has collaborated with HTA authorities, pharmaceutical and medical device companies, patients, payers, policymakers, academic researchers, clinical specialists, and venture capitalists. She has gained broad HEOR expertise of various technologies: precision medicine, oncology, vaccines, antibiotics, diagnostics, digital health, and medical devices.
Lotte’s career has been based in the United States, the Netherlands, and the UK. Collaborations with HEOR experts in Southeast Asia, Africa, and Latin America have provided her with deep insights into the differences and commonalities between the role of HEOR in different healthcare systems, cultures, and societies. With this background, she has: led diverse international teams; developed strategies and delivered on program and organisational missions, values, and objectives; acquired research funding and managed budgets; and been responsible for executive decision-making as well as legal and fiduciary matters.
As an active ISPOR member since 2004, Lotte has served on various Taskforces and Committees, delivered Short Courses, was co-Chair of ISPOR EU 2021, and is currently a member of the Board of Directors, and an Associate Editor for Value in Health. She aims to be a role model for diversity and played an active role in the 'Women in HEOR' initiative.
Before joining OHE, Lotte worked in Seattle (US) as an Associate Professor at the University of Washington and at the Fred Hutch Cancer Research Center. She graduated cum laude with her PhD from Maastricht University (NL) and then worked at Brunel University (UK) and Twente University (NL).
Melanie Whittington, PhD, MS
Tufts Medical Center (CEVR), Boston, MA, USA
Dr. Melanie Whittington is a Senior Fellow at the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center and has a decade of experience in health economics and outcomes research. Most recently, she was the Director of Health Economics at the Institute for Clinical and Economic Review (ICER) where she led the cost-effectiveness analyses and budget impact analyses within ICER reviews. She was the 2022 recipient of the Bernie J. O’Brien New Investigator Award.
Silence Is Not Always Golden: The Value of Engagement for Collection of Patient Experience Data in Drug Development
Digital Conference Pass
Level: Intermediate
PURPOSE:
The workshop aims to discuss practical aspects of investing in and generating patient experience data (PED) for drug development. Experts will discuss challenges, opportunities, and best practices related to engaging regulators in PED generation throughout the drug life cycle. Through interactive discussions, the goal is to inspire collaborative ideas and insights to enhance PED generation in drug development.
DESCRIPTION:
In the patient-focused drug development era, clinical trials are evolving to actively incorporate the collection of PED (information related to patients’ experience of their disease/condition). Various data sources, such as Clinical Outcome Assessments (COAs), Digital Health Technologies (DHTs), and Patient Preference Information, can collect this data. Although PED has the potential to expedite drug development, pharma and biotech firms may struggle with resource allocation to prioritize PED generation and stakeholder engagement at early stages, which can lead to insufficient data or data that fails to meet regulatory requirements. This workshop seeks to highlight the need for early investment in PED measurement strategy development. Discussions will focus on complexities, opportunities, and engagement points with regulators for input on PED collection tools, fostering a more direct path to reliable and valid PED generation. Dr. Yeh (chair) will present an overview of PED in drug development, highlighting common challenges and opportunities. Dr. Dashiell-Aje will introduce a recently developed Biotechnology Innovation Organization (BIO) FDA-sponsor COA engagement framework, elucidating its purpose, development process, and its practical utility for industry sponsors. Dr. Engmann will share strategies and challenges for early involvement in generating PED from a small biotech perspective. Dr. Daniels will provide the regulatory perspective regarding how regulatory input can be leveraged at critical timepoints in drug development to inform a PED measurement strategy. The workshop employs live polling for audience input on PED in drug development, leading to an interactive discussion to conclude the session.
Discussion Leaders
Danny Yeh, PhD
AESARA, Burlingame , CA, USA
Danny Yeh is the Head of the Center of Excellence within AESARA's Value and Evidence Team. With over a decade of experience in the pharmaceutical industry, Danny brings a wealth of knowledge to his role. Most recently, he held the position of Healthcare System Solution Cluster Lead, effectively serving as the Head of Government Affairs at Roche Taiwan.
Prior to his tenure at Roche, Danny spearheaded the establishment and leadership of the Health Policy and Systems Research team and Technical Institute at Genentech, which served as a distinguished center of excellence for Real-World Data (RWD) analytics and economic modeling capabilities.
Danny's professional journey also includes significant contributions within the Health Economics and Outcomes Research (HEOR) function at Biogen and Allergan. Throughout his career, Danny has played pivotal roles in supporting assets at various stages, from early phases to launch, across diverse disease areas. His expertise spans regions including the US, Asia Pacific, and Taiwan, where he has cultivated firsthand collaborations with key stakeholders such as clinical experts, regulatory bodies, payers, academia, policymakers, hospitals, and patient advocacy groups.
Discussants
Selena Daniels, PharmD, PhD
US Food and Drug Administration, Silver Spring, MD, USA
Dr. Selena Daniels serves as the Deputy Division Director in the Division of Clinical Outcome Assessment at the FDA. She provides direction, oversight, and leadership to a team of expert analysts who provide consultation and advice on clinical outcome assessment (COA) endpoint development and validation, including considerations for clinical trial design, conduct, analysis, interpretation, and reporting for regulatory determinations of medical product benefit.
Prior to joining the FDA in 2015, Dr. Daniels worked in the Health Economic and Outcomes Research (HEOR) group at Allergan, Inc (now Abbvie), where she developed and executed HEOR strategies, as well as developed and implemented innovative COA strategies and endpoints for clinical trials.
Dr. Daniels received her Doctor of Philosophy degree in Education at Nova Southeastern University and Doctor of Pharmacy degree at Loma Linda University.
Ebony Dashiell-Aje, PhD
BioMarin Pharmaceutical, San Rafael, CA, USA
Ebony Dashiell-Aje, PhD, is the Executive Director and Head of Patient Centered Outcomes Science at BioMarin Pharmaceutical Inc. and is a leading expert in clinical outcome assessment (COA) design and implementation, advising on COAs, digital health technology strategy, study endpoint issues, and regulatory science. As a health outcomes researcher and methodologist, Dr. Dashiell-Aje is driven by her passion for public health and promoting patient-focused medical product development. Dr. Dashiell-Aje has notably contributed her research expertise in academic, consulting and regulatory environments to arrive at evidence-based solutions, and to shape health policy.
Natalie Engmann, PhD
Denali Therapeutics, San Francisco, CA, USA
Natalie Engmann is an Associate Director at Denali Therapeutics where she leads the clinical outcomes team. Prior to Denali, Natalie was a health economist at Genentech where she led real world evidence generation for multiple molecules in the neuroscience and rare disease space. Natalie has over 15 years of health outcomes research experience across industry and academic settings, and in oncology, neuroscience and rare disease therapeutic areas. Natalie specializes in clinical and economic evidence generation to support regulatory and access decision-making. Natalie has a PhD in Epidemiology from UCSF and a MSc from the London School of Hygiene and Tropical Medicine.
Steps to Derive and Justify Novel Outcomes in Real World Evidence Studies Using Claims or Electronic Health Records
Level: Introductory
PURPOSE:
Regulators and payers are amenable to use of novel algorithmic or composite real world evidence (RWE) outcomes for clinical endpoints. However, the evidentiary requirements to justify the adequacy of these novel endpoints are not clearly defined. This workshop will review potential approaches to develop and justify measurement properties of novel RWE outcomes for use as clinical endpoints within studies.
DESCRIPTION:
Attendees will obtain a working knowledge of methods for RWE outcome validation as well as for developing health measurements, drawing from the paradigm of clinical outcome assessments (COAs).
Dr. Ritchey will introduce the importance of rigorous development and validation of outcome measurements in studies using RWE (15 min.). Ms. Norquist will discuss traditional approaches to developing health measurements, including COAs beginning with identifying relevant and feasible concept of interest and derivation of measures relevant to patients, and she will review what constitutes good measurement properties and interpretation for COAs that are meaningful to patients (15 min.). Dr. Girman will propose steps for development and justification to support use of outcomes that are fit-for-purpose for specific RWE study objectives (15 min).
The audience will participate in discussion of a case example (15 min) which will include digital handouts. This interactive workshop will be valuable to researchers studying novel outcomes in RWE studies.
Discussion Leaders
Mary E Ritchey, PhD, MSPH, FISPE
CERobs Consulting, LLC, Philadelphia, PA, USA
Mary Beth Ritchey, MSPH, PhD, FISPE has spent her career dedicated to better understanding medical product safety and effectiveness in clinical practice and has held positions within government, academics, and industry. In her current role as the Chief Scientific Officer for CERobs Consulting, Dr. Ritchey provides scientific and operational leadership for real-world evidence strategy development and execution along with leading evidence generation to inform decision-makers. In addition, she teaches and mentors as an Associate Research Professor in the Center for Pharmacoepidemiology and Treatment Sciences at Rutgers University.
Before re-joining CERobs in 2023, Dr. Ritchey was the Chief Epidemiologist for the US FDA Center for Devices and Radiological Health (CDRH), providing guidance and oversight related to clinical evidence and real-world evidence policies and programs. In April 2021, she released the book Pragmatic Randomized Clinical Trials: Using Primary Data Collection and Electronic Health Records, co-edited with Cynthia Girman, DrPH. Dr. Ritchey is also the current President-Elect and Fellow of the International Society for Pharmacoepidemiology (ISPE).
Discussants
Cynthia Girman, DrPH, FISPE
CERobs Consulting, LLC, Wrightsville Beach, NC, USA
Cynthia J Girman, DrPH, FISPE has forty years of epidemiology and biostatistical experience in the pharmaceutical industry and is founder and President of CERobs Consulting LLC (www.cerobs.com), which provides consulting on study design and methodology for real world evidence (RWE), observational non-interventional studies as well as external control arms for clinical trials. CERobs also provides services for endpoint strategies for clinical trials, including clinical outcome assessments and patient reported outcomes and their justification to regulatory bodies and payers. Previously, Cindy served as Executive Director and Head of Data Analytics & Observational Methods in the Center for Observational & Real World Evidence at Merck Research Laboratories, where she founded the Center of Excellence for Comparative & Outcomes Evidence and one for the Development, Validation, Standardization and Implementation of Endpoints (DEVISE) for clinical trials. She has worked extensively since the late 1990s in designing and implementing epidemiologic studies and methods in administrative claims and since the early 1990s in the development and validation of trial endpoints. She is an adjunct Professor in Epidemiology at the University of North Carolina (UNC) GIllings School of Global Public Health. In 2014, she became the first industry representative to serve as a member of PCORI’s Methodology Committee, on which she actively continues along with liaising with PCORI’s Clinical Trials Advisory Panel. She is an active fellow of the International Society for Pharmacoepidemiology, having served on the Board of Directors and as co-lead of the RWE and Regulatory Decisions task force. Dr. Girman received her DrPH in biostatistics with emphasis in epidemiology from UNC Chapel Hill, and her MS in applied statistics/computer science from Villanova University.
Josephine Norquist, MS
Merck & Co., Inc, West Point, PA, USA
Josephine Norquist, Exec Dir-Head of Patient-Centered Endpoints & Strategy at Merck & Co., Inc. leads SMEs on Clinical Outcome Assessments (COAs) strategies for studies across therapeutic areas to support labeling claims & reimbursement. She is the current President of ISOQOL (International Society of Quality of Life) and member of the C-PATH PRO Consortium.
She has co-authored many peer-reviewed journal articles, represented Merck at COA/PRO-related regulatory and scientific meetings and has developed several regulatory endpoint dossiers.
She has a B.S. in Statistics & Economics (Universita’ degli Studi di Palermo, Italy) and M.S. in Statistics (Northwestern University IL, USA).
8:30 AM - 10:00 AM
Breakfast Bites and Networking Delights
Kick off the final day of the conference in the Exhibit Hall with a light breakfast – use this time to connect with exhibitors and ignite conversations. Provided by ISPOR.
8:30 AM - 11:30 AM
Exhibit Hall Open
8:45 AM - 9:45 AM
Podium Session
Novel Outcomes Research Data Methods
Digital Conference Pass
Moderator
Lisa Hess, PhD
Eli Lilly and Company, Indianapolis, IN, USA
Lisa M. Hess, PhD, is Associate Vice President of Value, Evidence, and Outcomes research at Eli Lilly and Company and adjunct professor of Public Health at Indiana University, USA and at UMIT TIROL-University for Health Sciences and Technology, Austria. After more than 20 years in an academic career, she joined Eli Lilly in 2012, where her work focuses on modeling, observational research, and innovative approaches to real-world database analysis in oncology. Her interests involve developing and applying research methods to analyses that lead to improved cancer decision making, as well as the development of scientific talent in the broader field of health outcomes research.
P54: Evaluating ChatGPT's Efficacy and Efficiency in the Risk of Bias Assessment in Health Economic Evaluations: A Comparative Analysis Using the Drummond Checklist
9:00AM - 9:15AM
Ohra S1, C RR1, Siddiqui MT1, Gupta J2, Siddiqui MK3
1EBM Health Consultants, New Delhi, DL, India, 2EBM Health, Cleckheaton, West Yorkshire, UK, 3EBM Health, New Delhi, DL, India
OBJECTIVES: Drummond checklist is a detailed tool for evaluating the methodological rigor of health economic (HE) studies, requiring a time-consuming review process. The use of large language models (LLMs) such as ChatGPT4.0 can significantly reduce the efforts required by a human reviewer. This study aimed at evaluating the performance of ChatGPT4.0 versus a trained human reviewer in assessing the quality of HE studies using the Drummond checklist.
METHODS: A list of HE studies was randomly selected from an existing review and each study was anonymized to ensure an unbiased evaluation by ChatGPT4.0 and the human reviewer. We developed standardized prompts using an iterative process by providing explicit instructions on the Drummond checklist, which encompasses 36 questions across study design and data collection methods, measurement and evaluation of outcomes, analysis and interpretation of results. These prompts included an appraisal guidance document, a response template and the study documents to be assessed for quality appraisal. The primary outcome of the analysis was the level of agreement assessed by Kappa statistics between ChatGPT4.0 and the human reviewer.
RESULTS: We piloted the prompt using 10 studies and post-standardization further expanded the assessment to another 27 studies. After standardization of the prompt, meantime in minutes to complete the checklist was significantly shorter with ChatGPT4.0 compared to the human reviewer (8 mins/study vs 25 mins/study, a 68% reduction). Our assessment indicated that the level of agreement between ChatGPT4.0 and the expert reviewer across the three domains ranged between 2.7% to 94.5%. Differences were noted in specific areas such as the generalizability of the study results which are more subjective and require human judgement.
CONCLUSIONS: LLMs such as ChatGPT4.0 can expedite the quality appraisal of health economic studies, but it should be utilized only as an adjunct to human expertise rather than a standalone reviewer, particularly for domains requiring subjective evaluation.
P56: Extraction of Inflammatory Bowel Disease Severity Scores from Unstructured Clinical Notes Using a Retriever-Reader Architecture
9:30AM - 9:45AM
Kumar V1, Rasouliyan L1, Althoff A1, Black D2, Mummert A1, Chang S1, Long S1
1OMNY Health, Atlanta, GA, USA, 2OMNY Health, Washington, DC, USA
OBJECTIVES: Our objective was to extract disease severity scores for inflammatory bowel disease (IBD) patients from electronic health record unstructured clinical notes using a transformer-based retriever-reader architecture and assess the accuracy.
METHODS: Deidentified clinical note paragraphs of IBD patients (International Classification of Diseases, Tenth Revision: K50*, K51*) from two hospital systems (2017-2022) in the OMNY Health real-world data platform were written to a document store. To retrieve paragraphs relevant to the severity scores of interest, queries were composed inquiring about four severity scores: 9-point Mayo score, 6-point Mayo score, Endoscopic Mayo score, and short Crohn’s Disease Activity Index (CDAI). Relevant paragraphs were retrieved using the Best Match 25 algorithm. A Robustly Optimized Bidirectional Encoder Representations from Transformers Pre-training Approach (RoBERTa)-based question-answering model was used to identify the tokens in each paragraph corresponding to the severity scores. Results were sorted and manually examined to select an optimal confidence score cutoff for each query, and 25% of the records scoring above the cutoffs were randomly sampled and manually annotated for accuracy.
RESULTS: 39,338 IBD patients were included; 3.36 million paragraphs were written to the document store. For severity scores listed above, score cutoffs of 0.810, 0.650, 0.490, and 0.694 were selected, respectively, yielding overall score (unique patient) counts of 428 (167), 425 (170), 317 (146), and 402 (161), respectively. Accuracy for the four scores was 100%, 100%, 96%, and 100%, respectively.
CONCLUSIONS: The results show that a retriever-reader architecture extracts severity scores from clinical notes of IBD patients with >95% accuracy. Improvements in accuracy and discrimination between several scores in the same paragraph were observed in this pipeline compared to previously published pipelines. This model demonstrates that key clinical outcomes may be extracted at scale. Further development and deployment of the model would increase patient yields and isolate Endoscopic Mayo scores for various intestinal regions.
P55: Treatment and Outcomes in Metastatic Colorectal Cancer: A Causal Study Design Framework
9:15AM - 9:30AM
Dixon R1, Guzman M1, Hopkins K1, Lanes S1, Grabner M1, Hill N2, Dixon M2
1Carelon Research, Wilmington, DE, USA, 2Bristol Myers Squibb, Lawrenceville, NJ, USA
OBJECTIVES: Directed Acyclic Graphs (DAGs) are structured diagrams describing the assumed underlying causal relationships between treatments, outcomes, and covariates. As evidence generation using real-world data (RWD) continues to expand, DAGs can help identify biasing paths, inform selection of covariates, and generate a more valid causal effect estimate. This study systematically built DAGs to elucidate causal pathways between first-line (1L) treatment (immune-oncology (IO) versus non-IO therapy) and survival among patients with metastatic colorectal cancer (mCRC).
METHODS: The study design comprised three components: 1) two targeted literature searches to identify relevant variables related to treatment (IO [nivolumab, ipilimumab, or pembrolizumab] versus non-IO [standard of care chemotherapy]) and outcomes (progression-free/overall survival); 2) plausibility assessments of each proposed relationship to be captured in the DAG, and 3) a case study utilizing Carelon Research’s Healthcare Integrated Research Database (HIRD). Using US integrated claims and clinical data from 2014-2023, we differentiated measured from unmeasured variables and estimated bivariate relationships between each covariate, treatment, and overall survival via logistic regression to refine the proposed relationships.
RESULTS: The two targeted literature searches identified 94 RCTs and 22 RWD studies, from which 28 variables were extracted. These potential confounders (e.g., tumor characteristics, performance status, health care access) or colliders (e.g., data collection methods) relative to the treatment-outcome relationship were built into the DAG. We identified 9,046 patients with mCRC from the HIRD, of whom 213 patients initiated IO therapy (mean age 60 years, 47% female, 80% Non-Hispanic White). Age, performance status, prior chemotherapy, and BRAF mutation status demonstrated large effect sizes and statistically significant associations with both treatment (IO versus non-IO) and overall survival.
CONCLUSIONS: Creating DAGs in a systematic and efficient manner, informed by existing literature and plausibility assessments, provides transparency when estimating causal effects from RWD and can reduce bias in the chosen statistical model.
P53: Validation of AI-Generated Synthetic Data for Outcomes Research: A Clinico-Genomics Case Study
8:45AM - 9:00AM
Theodorou B1, Das A2, Zhang NJ3, Sun J1, Yay Donderici E4
1Medisyn Inc., Las Vegas, NV, USA, 2Guardant Health, Cambridge, MA, USA, 3Guardant Health, Palo Alto, CA, USA, 4Guardant Health, Burlingame, CA, USA
OBJECTIVES: The burgeoning field of healthcare data analysis is continually confronted with the dual challenges of data privacy and the need for high-quality datasets with which to perform cutting edge data analysis and research. We aim to use a synthetic data generation pipeline to bridge the gap between these challenges by producing synthetic patient data capable of performing real research.
METHODS: We utilize GuardantINFORM, which includes anonymized genomic and structured payer claims data, to generate lung adenocarcinoma (LUAD) patient cohorts. We process this data into a tabular format before decomposing it into an internal sequential format that is able to leverage advanced transformer-based large language models. This innovation allows for the creation of synthetic datasets that not only retain the statistical properties of the original data but also preserve the privacy of underlying data points.
RESULTS: We show that there is a 0.993 R2 correlation between real and synthetic variable probabilities, demonstrating the robustness of the synthetic data in mimicking real-world data distributions. We then use a membership inference attack test, with a resulting accuracy of 49% or roughly a random guess, to underscore the effectiveness in preserving patient privacy while generating that data. Finally, we replicated a real study using the synthetic data by showing that LUAD patients with the KRAS G12C mutation were more susceptible to earlier loss of life in the face of a cancer diagnosis and that this problem compounded upon co-occurring STK11 mutations. This not only validates the practical utility of synthetic data but also highlights its potential in facilitating groundbreaking research.
CONCLUSIONS: We present a promising solution for the healthcare research community. It enables the sharing of privatized, synthetic data that retains the integrity and utility required for significant scientific discoveries, paving the way for more liberalized, responsible, and effective data sharing practices in healthcare research.
9:00 AM - 11:30 AM
Poster Session 5
Live
9:15 AM - 9:45 AM
Exhibit Hall Theater
Amplifying HEOR Studies Using Real-World Electronic Health Record Data
Digital Conference Pass
Level: Intermediate
Since the adoption of electronic health records (EHRs), healthcare institutions have collected a massive amount of broad, real-world clinical data. This data has the potential to power retrospective HEOR studies of unprecedented scale and depth, but it can be challenging to harness.
This presentation will showcase how nference leverages language models and AI-assisted curation to de-identify and harmonize EHR data from leading health systems. nference's nSights platform makes the complete patient journey readily accessible for research, enabling breakthrough discoveries and collaborations as well as the creation of new venture companies that harness various data modalities.
nference will highlight specific HEOR studies that were successfully executed using the nSights platform. Examples will include the quantification of healthcare resource utilization (HCRU), determination of risk factors for high HCRU, and stratified outcomes analysis based on disease stage, each derived from broad EHR data (diagnosis, lab data, AI-analyzed pathology reports, and biopsy data).
Sponsor
nference
Speaker
Michiel Niesen, PhD
nference, San Diego, CA, USA
10:00 AM - 11:00 AM
Breakouts: IP, WS, & OBS
Capturing Family Member Spillover Health Effects in Economic Evaluation of Pediatric Interventions: Avoiding the Landmines
Level: Intermediate
ISSUE: While it’s clear that family members can be profoundly affected by the ill health of a child, how to capture family member spillover health effects in pediatric economic evaluation is fraught with controversy. In the case of life-extending interventions, the “Carer-QALY trap” suggests that whereas small quality-adjusted life year (QALY) gains may be experienced by the patient, the extensive QALY losses experienced by family members due to prolonged caregiving may paradoxically make treatment appear less favourable than death. Inclusion of caregiving effects is also subject to bias associated with the number of caregivers available, with treatments demonstrating better outcomes in families with larger caregiving circles. The inclusion of family member spillover health effects is increasing, but a wide range of study designs, preference-based health-related quality-of-life instruments and modeling approaches are being used, revealing a lack of consensus on the optimal design, tool and modelling approach. This international panel will examine and debate different approaches to capturing family member/caregiver health effects in pediatric economic evaluation.
OVERVIEW: Mick Tilford will moderate and provide context. Ramesh Lamsal will address the appropriateness of including spillover health effects and will contrast the different approaches used, such as measuring parent and child health state utilities in a single or separate models, deriving joint health states, summing QALY gains across family members, or deriving a household/family utility function. Wendy Ungar will illustrate the issue using economic evaluation of pediatric genetic testing, where testing is often directed at families (through cascade testing or trio sequencing), and present how family member health effects and resource use can be included in the reference case analysis. After the presentations, the moderator will pose questions to panelists to spark debate and each speaker will be asked to suggest ways forward. The session will end with an audience Q&A.
Moderators
J. Mick Tilford, PhD
University of Arkansas for Medical Sciences, Little Rock, AR, USA
John “Mick” Tilford currently serves as a Professor and Chair of Health Policy and Management, at the University of Arkansas for Medical Sciences. He has a secondary appointment in the Division of Pharmaceutical Evaluation and Policy in the College of Pharmacy. His research program focuses on methods for the economic evaluation of health services. He has studied the cost-effectiveness of improving outcomes in children with traumatic brain injuries, quality of care associated with intensive care units, and quality-adjusted life years in children with chronic conditions, especially children with autism. Recent areas of interest include the development of methods for incorporating family effects in economic evaluations.
Panelists
Ramesh Lamsal, Msc, PhD
Centers for Disease Control and Prevention, Atlanta , GA, USA
Wendy Ungar, MSc, PhD
The Hospital for Sick Children Research Institute, Toronto, ON, Canada
Wendy Ungar is a Senior Scientist, SickKids Research Institute, and Professor, University of Toronto. She holds the Canada Research Chair in Economic Evaluation and Technology Assessment in Child Health and directs Technology Assessment at Sick Kids (TASK) where she applies economic methods to child health. Her team maintains the PEDE database, a searchable online database of pediatric economic evaluations used by global HTA agencies Dr. Ungar chairs the Ontario Genetic Testing Advisory Committee which makes funding recommendations on emerging genetic testing technologies for Ontario, Canada. Her book, Economic Evaluation in Child Health was published by Oxford University Press in 2010.
The Invisible Costs in Rare Disease Management: Who Bears the Expense?
Digital Conference Pass
Level: Intermediate
ISSUE:
Ten thousand rare diseases (RD) affect ~400 million people worldwide. In US the total healthcare expenditure of RDs (2019) was $966 billion but the true costs of RDs are unknown. The caregivers complement the healthcare and social security systems although its underappreciated and very little recognized. Evidence relating to cost and burden of disease suggests significant costs, both economic and social, and ranging far beyond the healthcare expenditure for the individual patient. Patients and their families face substantial (‘hidden’) intangible costs associated with disease management. Who bears the cost: patients and their families or payers?
OVERVIEW:
Nicole will provide an overview of healthcare costs: direct, indirect, and intangible. Intangible costs will be the focus of the discussion, such as loss of independence, productivity impacts, and psychosocial effects. These costs are not appropriately considered in the evaluation of treatments and greatly impact patients and their families. Michael will review the issues in the estimation of patient and family costs, and quality of life impacts, within the current HTA environment(s) and suggest ways of capturing these items within the evaluation of new treatments. Fleur will give an industry perspective, additionally drawing on examples from HERCULES and as a caregiver in DMD, discussing the need, desire, and complexities in capturing these impacts and the challenges in articulating these items to decision makers so the full value of treatments can be demonstrated. Daniel will discuss the impact of acid sphingomyelinase deficiency (ASMD) in both pediatric and adult patients. He will dive deeper and unpack the social and psychological impacts/costs experienced by patients both pediatric and adult. These costs affect more than just the patients, impacting the larger support unit (siblings, children, and spouses). He will provide a unique perspective into these ‘hidden’ costs on siblings, family/parents/carers to understand the economic, social, and psychological impact.
Moderators
Nicole Lyn, MPH
Sanofi, Cambridge, MA, USA
Nicole Lyn is Global Health Economics and Value Assessment Business Partner at Sanofi, where she is responsible for the strategic planning and execution of clinical and economic studies in Sanofi’s rare disease portfolio.
Nicole started her career in consulting where she developed and validated COAs across various therapeutic areas before moving into industry, where she focuses on generating evidence to demonstrate the value of new therapies. Nicole holds a BA in Sociology from Boston University, and an MPH in Health Policy & Law and Epidemiology & Biostatistics from Boston University.
Panelists
Fleur Chandler, MSc
Sanofi and Patient Advisory Board Lead, Duchenne UK, Reading, Berkshire, Great Britain
Fleur is Head of Market Access UK & Ireland at Sanofi. Fleur is a health economist with a wealth of outcomes research and health technology assessment experience, with over 30 years leading both global and local teams in the pharmaceutical industry. Fleur is especially driven by rare disease, a parent of a teenager with Duchenne Muscular Dystrophy, she is on the advisory board of patient organisation Duchenne UK. Bringing her professional and personal experience together, she conceptualised and leads Project HERCULES, a global collaboration generating gold standard disease level evidence for HTA in Duchenne.
Daniel de Vicente, BS
ASMD España, Madrid, Spain
Pharmacist with more than 20 years of experience in community pharmacy. Postgraduate in Public Health. Postgraduate in management and strategic planning for non-profit entities. Health Consultant and Project Manager specialist in rare diseases, PROMs PREMs, RWE, RWD and expert patient programmes. President of the Association of Patients ASMD Spain. Member of FEDER (Rare Diseases Spain) BoD. Member of Eurordis BoD. Research Liaison Officer INPDA (International Niemann-Pick Alliance)
Michael Drummond, PhD
University of York, Lichfield, United Kingdom
Michael Drummond is Professor Emeritus of Health Economics and former Director of the Centre for Health Economics at the University of York. His particular field of interest is in the economic evaluation of health care treatments and programmes. He has undertaken evaluations in a wide range of medical fields including care of the elderly, neonatal intensive care, immunization programmes, services for people with AIDS, eye health care and pharmaceuticals. He is the author of two major textbooks and more than 750 scientific papers, and has acted as a consultant to the World Health Organization and the European Union. He has been President of the International Society of Technology Assessment in Health Care, and the International Society for Pharmacoeconomics and Outcomes Research. In October 2010 he was made a member of the National Academy of Medicine in the USA. He has advised several governments on the assessment of health technologies and chaired one of the Guideline Review Panels for the National Institute for Health and Care Excellence (NICE) in the UK. Until recently he was Co-Editor-in-Chief of Value in Health and has been awarded 3 honorary doctorates, from City University (London), Erasmus University (Rotterdam) and the University of Lisbon.
Value of Diagnostic Tests When Treatment Is Not Optimal?
Digital Conference Pass
Level: Introductory
ISSUE: The cost-effectiveness of diagnostic tests hinges on their ability to correctly diagnose a condition and guide appropriate treatment, which is expected to improve clinical outcomes. How might diagnostic tests be valued for conditions that lack optimal treatment? Many infectious diseases provide good examples – e.g. norovirus, rhinovirus, bacterial vaginosis. There are currently no treatment options for norovirus and rhinovirus. Despite antibiotics being available for bacterial vaginosis, symptom persistence/recurrence rates are high. For these conditions where improvements in clinical outcomes may not be achieved through treatment, how is the value of diagnostic information (VODI) perceived? How does the valuation criteria differ for diagnostics intended for conditions with good treatment options? Does disease severity affect the perceived value? The aims of this issue panel are: 1) to use infectious disease examples to explore more complex relationships between treatment effectiveness and diagnostic value across multiple perspectives; 2) explore different value parameters for diagnostics and how these may be captured in cost-effectiveness analysis.
OVERVIEW: The moderator will present a 10-minute overview of the issue. Each panel member will then have 10 minutes to present their perspective. This will include the concept of VODI and how VODI supports broader value assessment of diagnostics beyond treatment outcomes; clinician perspective on the real-world clinical and economic benefits of molecular diagnostics for infectious diseases; and payer perspective on current priorities and considerations for diagnostic reimbursement. After the presentations, the moderator will summarise and pull out the key themes and consider wider applicability to additional disease areas before a 20-minute panel discussion with audience participation. It’s expected that researchers and manufacturers in the diagnostic tests field and payers would benefit from this discussion.
Moderators
Karissa Johnston, PhD
Broadstreet Health Economics & Outcomes Research, Vancouver, BC, Canada
Karissa Johnston’s academic training is in statistics (MSc) and epidemiology (PhD), and she’s worked for more than 15 years in HEOR and epidemiology across oncology, cardiovascular disease, HIV/AIDS and rare disease. In addition to being a Broadstreet HEOR co-founder, Karissa is an adjunct professor in the School of Pharmacy, Memorial University (Newfoundland).
Panelists
Lon Castle, MD
eviCore healthcare, Richmond Hill, GA, USA
Dr. Castle is responsible for the clinical performance of the Laboratory, Specialty Drug and Gene Therapy programs at EviCore healthcare, a wholly-owned subsidiary of Cigna. In addition to his decade-long experience with specialty drugs, Dr. Castle has been working in the genomic and molecular diagnostic field since 2007, both designing initiatives that support the advancement of precision medicine as well as managing programs that ensure these tests are used appropriately.
Lou Garrison, PhD
CHOICE Institute, School of Pharmacy, University of Washington, Seattle, WA, USA
Lou Garrison, PhD, is Professor Emeritus in The Comparative Health Outcomes, Policy, and Economics Institute in the School of Pharmacy at the University of Washington, where he joined the faculty in 2004.
Dr. Garrison has worked in non-profit, industry, and academic settings. He received a PhD in Economics from Stanford University and has more than 200 publications in peer-reviewed journals.
Dr. Garrison was elected as ISPOR President for 2016-2017, and currently serves as co-chair of ISPOR’s Policy Outlook Committee for the Health Science Policy Council. In September 2022, he was announced as the recipient of the 2022 Avedis Donabedian Outcomes Research Lifetime Achievement Award from ISPOR.
RIchard Rothman, MD, PhD
Department of Emergency Medicine, Johns Hopkins University, Cockeysville, MD, USA
Dr. Richard Rothman, MD, PhD, FACEP is a Professor of Emergency Medicine at The Johns Hopkins University and a practicing emergency physician at Johns Hopkins Hospital, where he has worked for the past 3 decades. He serves as Executive Vice Chair and Vice Chair for Research for Emergency Medicine, and holds a Joint Appointment in The Division of Infectious Diseases. His research focus is on advancing tools for early diagnosis and management of varied acute care conditions and he has led multiple federal funded center grants/contracts studying diagnostic approaches to infectious disease conditions in emergency care settings. At Johns Hopkins, he directs a team of interdisciplinary investigators from emergency medicine, microbiology, infectious disease, and public health.
Addressing Key Challenges in the Setting of Tumor-Agnostic Drugs: Meeting an Unmet Need?
Digital Conference Pass
Level: Intermediate
PURPOSE:
Tumor-agnostic or histology independent therapies are cancer therapies that target specific genetic or molecular alterations, regardless of tumor histology. Often evaluated using ´basket trials´, the assessment of tumor-agnostic therapies presents stakeholders with many challenges, including small sample sizes due to rare conditions, lack of a counterfactual, immature endpoints at the time of drug approval, and heterogeneity in treatment effect across histologies. This workshop will focus on how to leverage established biostatistical and economic modeling methods to assess novel therapies to inform reimbursement decisions.
DESCRIPTION:
Workshop attendees will obtain a working knowledge of existing methods for evaluating tumor-agnostic therapies. The workshop will review (a) current challenges faced by health technology assessment (HTA) agencies when considering tumor-agnostic therapies by context and across jurisdictions: (b) how biostatistical and economic modeling tools can be leveraged to address these challenges; (c) perspectives of the National Institute for Health and Care Excellence Technology Appraisal Committees and the Institute for Clinical and Economic Review (ICER). Dr. Devine will chair the session and introduce the topic in the context of existing frameworks and approaches taken by different countries (5min.). Ms. Mackay will show how Bayesian hierarchical models can handle small sample sizes and heterogeneity in basket trials (15min.). Dr. Chen will describe potential approaches to addressing challenges in cost-effectiveness analysis, providing a case example using pembrolizumab in 8 MSI-H/dMMR metastatic cancers in the US (15min.). Dr. Ollendorf will offer an ICER perspective and discuss the policy implications of these methods for assessment of product value, fair pricing, and reimbursement decisions in the US (15min.). Each speaker will incorporate real-time audience polling to highlight learning opportunities and focus on take-away messages (10min.). This interactive and informative workshop will be valuable to HTA policy makers, payers, and industry analysts who are considering tumor-agnostic indications in healthcare decision-making.
Discussion Leaders
Beth Devine, PhD, PharmD, MBA
University of Washington, Seattle, WA, USA
With 25 years of experience in the field of HEOR, Dr. Devine has expertise in evidence synthesis, precision medicine, real world evidence, health technology assessment, preference assessment and patient-centered outcomes research. She is currently a member of the ISPOR Board of Directors and an Associate Editor for Value in Health. She is the Associate Director of the CHOICE Institute, University of Washington.
Discussants
Yilin Chen, MPH, PhD
Curta, Inc., Seattle, WA, USA
Yilin Chen is currently a Senior Research Associate at Curta Inc., a consultancy specializing in health economics and outcomes research (HEOR). She completed her Ph.D. in HEOR in the Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute at the University of Washington. Her areas of expertise include real-world evidence, economic modeling, discrete choice experiment, and health technology assessment, with experience in oncology, immunology, diabetes, HIV/AIDS, and women’s health. She has worked with various partners including the Institute for Clinical and Economic Review (ICER), Fred Hutchinson Cancer Center, the Gates Foundation, and Kaiser Permanente.
Emma Mackay, MA, MSc
Inka Health, Toronto, ON, Canada
Emma Mackay is a Scientific Advisor in Statistics at Inka Health, specializing in Bayesian methods. Her recent work has focused on the application of Bayesian borrowing to address challenges of evaluating efficacy/effectiveness in rare disease settings. She has extensive experience in HEOR analytics with expertise in meta-analysis, survival modelling, synthetic control arms, and population-adjusted indirect comparisons.
Dan Ollendorf, PhD
Institute for Clinical and Economic Review; Tufts University School of Medicine and Tufts Medical Center, Boston, MA, USA
Dan Ollendorf is Chief Scientific Officer and Director of Health Technology Assessment (HTA) Methods and Engagement at the Institute for Clinical and Economic Review (ICER), with responsibility for managing the organization’s internal health economics capacity and program of external collaboration, as well as collaboration with international HTA bodies and other stakeholders on key process and methods topics.
From 2007-2018, Dr. Ollendorf was Chief Scientific Officer for ICER, where he oversaw the broader HTA process and managed relationships with multiple stakeholders and research collaborators. From 2018-2023, Dr. Ollendorf served as the Director of Value Measurement & Global Health Initiatives at the Center for the Evaluation of Value and Risk in Health (CEVR) at Tufts Medical Center, where he focused on value assessment methods, drug pricing policy, and building international capacity for and interest in HTA. In addition to his current role at ICER, Dr. Ollendorf maintains a faculty appointment at CEVR and continues to teach in the Center’s graduate certificate program as well as participate in ongoing research.
Dr. Ollendorf holds a PhD in clinical epidemiology from the University of Amsterdam and an MPH from Boston University. He is the current Chair of the Health Technology Assessment International Global Policy Forum as well as a non-resident Fellow at the Center for Global Development, and served on the US Medicare Evidence Development and Coverage Advisory Committee (MEDCAC) from 2015-2019. Dr. Ollendorf has authored over 125 articles in peer-review journals and is co-author of The Right Price: A Value-Based Prescription for Drug Costs (Oxford University Press).
How to Adjust Economic Models for Health Equity in the Conduct of Generalized Cost-Effectiveness Analysis (GCEA)
Level: Experienced
PURPOSE: The ISPOR value flower has provided the field with a list of ‘what’ novel value elements should be incorporated into cost-effectiveness analysis (CEA), among them the value of health equity. Health equity, while important to consider, does not have an established system of measurement and weighting on economic evaluation. Recent measurement and methodological advancements that consider the importance of health equity on decision-making can play into approaches for generalized cost-effectiveness analysis (GCEA).
DESCRIPTION: Padula will provide an overview GCEA and the relative importance of adjusting economic models for health equity. Lakdawalla will introduce the generalized risk-adjusted cost-effectiveness (GRACE) framework as a way of overcoming discriminatory decision-making by adjusting for baseline disability and concerns about health equity. Basu will introduce a new measurement system to health utility, Healthy Years in Total (HYT) as an alternative to QALYs that addressed health equity concerns. Vasco Pontinha will introduce a novel method to encapsulate health equity based on budgetary constraints for mainstream health technologies. At the end of the workshop, the discussants will poll the audience about which method(s) may be able to gain traction to make U.S. health technology assessment processes more equitable.
Incorporating Patient-Reported Outcomes in Drug Pricing and Reimbursement Decision-Making: Development and Validation of Chrome System in China
Digital Conference Pass
Level: Intermediate
PURPOSE: Since 2018, China has adopted a centralized drug review and price negotiation process to inform national coverage decision making. Quality-adjusted life year (QALY) is a recommended outcome measure for this process. However, all existing patient-reported outcome measures (PROMs) were developed in western countries. There was a need for PROMs that measure important aspects of health perceived by Chinese populations. The China Health-Related Health Outcome Measures (CHROME) were developed for this purpose. CHROME is a system of preference-based PROMs that consists of a core module for generic use and a series of disease-specific modules for measuring disease or condition-specific impact. This system has a novel design in which both generic and disease specific measurement needs are integrated throughout the conceptualization, validation, and valuation. Therefore, they can be used in clinical and economic evaluations with consistency and transitivity, while maintaining independence. The primary goal of this workshop is to introduce the development and validation of the CHROME system and its application to support drug price negotiation in China.
DESCRIPTION:
This workshop will start with an introduction of health technology assessment (HTA) and drug price negotiation process in China (10 minutes, by Dr. Kun Zhao). Dr. Feng Xie will provide an overview of the design, concept and structure of the CHROME system (15 minutes). Dr. Xue Li will present the development of CHROME instruments and their validations using both classic testing theory and item response theory in large representative samples (n=2,929) in China (20 minutes). The workshop will be concluded by discussion on the implications of the CHROME systems for HTA and drug price negotiation in China (10 mins by Dr. Qiang Fu), followed by a Q&A session (5 minutes). This workshop may benefit the audience who is interested in the PROMs development and validation for HTA and drug coverage decision making in China.
Discussion Leaders
Shitong Xie, PhD
School of Pharmaceutical Science and Technology, Tianjin University, Tianjin, 12, China
Discussants
Xue Li, PhD
China National Health Development Research Center, Beijing, China
Xue (Michelle) Li, PhD. She is the associate director in Department of Health Technology Assessment, China National Health Development Research Center. She is the adjunct professor in Western University, Canada. She shows her research interests in Health Technology Assessment, Health Economics Evaluation, Comprehensive Drug Evaluation, Real-world Data Evaluation, Development, Validation and Valuation of Patient Reported Outcomes.
Guo Wudong, PhD
China National Health Development Research Center, Beijing, Beijing, China
Feng Xie, PhD
McMaster University, Hamilton, ON, ON, Canada
Dr. Feng Xie is Professor in the Centre for Health Economics and Policy Analysis (CHEPA) at McMaster University. His research interests include health technology assessment (HTA), economic evaluations using models and clinical trial data, patient-reported outcome and preference measures, and health utility measures. He is vice chair of the EuroQol Executive Committee and the lead of China Health Related Outcomes Measures (CHROME) project.
Advanced Methods for Matching-Adjusted Indirect Comparison (MAIC)
Digital Conference Pass
Level: Intermediate
PURPOSE: Anchored covariate-adjusted indirect treatment comparisons (ITCs) inform reimbursement decisions when (1) there are no head-to-head trials between the treatments of interest, (2) there is a common comparator arm shared by two studies, and (3) there are patient-level data limitations. Matching-adjusted indirect comparison (MAIC) is currently the most widely used method in HTA but is not always possible. We begin with an overview of newly proposed MAIC weighting schemes and approaches for assessing the numerical feasibility of conducting an MAIC. We then consider the "two-stage MAIC", an extension to MAIC which has been recently proposed and shown to yield improved precision and efficiency, while maintaining similarly low levels of bias when assumptions are met. We review the two-stage MAIC and how it has recently been applied to achieve greater precision. Finally, certain approaches in causal inference are known to be “doubly-robust” meaning that they will provide unbiased estimates so long as either the trial allocation model or the outcome model is correctly specified. However, doubly-robust methods for ITCs have not yet been proposed. Here we consider the potential of “augmented” MAIC for doubly-robust estimation in anchored ITCs.
DESCRIPTION: Workshop attendees will review the main advantages and limitations of MAIC and be introduced to newly proposed methods. The audience will be asked (real-time polling) to consider if these “advanced” methods should be more widely considered in HTA . Ms. Cope will chair the session and introduce the main issues involved with existing MAIC methods and summarize recent work on alternative weighting schemes and feasibility assessment (20 min). Dr. Remiro-Azócar will summarize his recent work on the two-stage MAIC method (15 min) and Dr. Campbell will consider the potential of “augmented” methods for doubly-robust estimation (15 min). To conclude, an audience discussion will consider the potential and priorities for MAIC methods research (10 min).
Discussion Leaders
Shannon Cope, MSc
PRECISIONheor, VANCOUVER, BC, Canada
Shannon Cope, MSc, is a Senior Vice President with PRECISIONheor. Shannon has over ten years of experience consulting for biopharmaceutical industry regarding evidence synthesis and economic evaluations to assess the value of new interventions.
Discussants
Harlan Campbell, PhD
University of British Columbia, Rossland, BC, Canada
In addition to serving as a Principal Scientist/Senior Methodologist at PrecisionHEOR, Dr. Campbell is an Adjunct Professor in the University of British Columbia Department of Statistics. He completed his PhD in the field of Statistics at UBC, researching statistical methods for hypothesis testing and causal inference. His applied research focusses on applications in evidence synthesis, epidemiology and clinical trials.
Antonio Remiro-Azócar, PhD
Novo Nordisk, Madrid, Spain
Antonio is a statistical methodologist within the Biostatistics, Methods and Outreach department at Novo Nordisk. His expertise lies in quantitative evidence synthesis, HTA, HEOR and observational science. Antonio is currently developing and implementing innovative statistical methods for the transportability and generalizability of research data, and for the enrichment of randomized clinical trials with real-world data sources. Prior to his current role, Antonio was Lead Medical Affairs Statistician at Bayer Pharmaceuticals, and provided statistical support to contract research organizations such as IQVIA and ICON plc and public bodies such as SickKids. Antonio holds a PhD in Statistical Science and master's degrees in Machine Learning and Financial Computing from University College London.
Getting Real About Real-World Data From Electronic Health Records
Digital Conference Pass
Level: Introductory
PURPOSE: Real world evidence (RWE) is only trusted by regulatory bodies, health technology assessment (HTA) organizations, payers, clinicians and patients if the underlying real-world data (RWD) is demonstrated to be high quality and fit-for-purpose. Electronic health records (EHRs) are an important source of real-world data: they are the primary record of the patient symptoms, diagnoses, treatments, outcomes, and in many cases, preferences and experiences. However, EHRs were not designed for research, and clinician engagement and interaction with EHRs varies widely from scope of practice to health care organization type. Clinician documentation in EHRs can often be limited by time pressure and administrative burden. Moreover, much information is captured in unstructured formats within the EHR, and coding systems and other factors often vary between health system EHRs. Patient records may also be split between multiple health systems and private practice EHRs. In this workshop, we will explore how all of these factors impact RWE generated from EHR data, and how these challenges can be overcome to improve trust and transparency around uses and the reliability of EHR data for research and regulatory purposes.
DESCRIPTION: In this workshop, Rachele Hendricks-Sturrup will describe health system practices in generating RWE to support learning health system goals, and Duke-Margolis’ current focus on operationalizing EHR data relevance, reliability, and quality to build trust and transparency among health system stakeholders. Mac Bonafede, Elise Berliner and Louis Ehwerhemuepha will take some of those examples and describe the strengths and weaknesses of EHR data for the specific questions and how they are working to improve the validity and quality of EHR data to answer these questions. The panel will take questions on specific challenges audience members are facing using EHR data and other RWD, and brainstorm solutions with panel and audience members.
Discussion Leaders
Elise Berliner, PhD
Oracle Life Sciences, Kansas City, MO, USA
Discussants
Mac Bonafede, PhD
Veradigm, Brentwood, NH, USA
Mac is Vice President of Research Consulting at Veradigm where he leads their team of data scientists, statisticians, and epidemiologists in conducting and guiding real world health economics and outcomes research studies. Mac’s team is adept at using varied real world data sources to generate real world evidence findings in a range of therapeutic areas.
Before joining Veradigm, Dr. Bonafede was with IBM Watson Health (previously Truven Health Analytics and Thomson Reuters Healthcare). Prior to that, Dr. Bonafede served on the faculty of the Albany College of Pharmacy, where he taught courses covering research and statistical methods, total quality management, and evidence-based medicine.
Dr. Bonafede continues to teach in the University of New Hampshire Department of Health Management and Policy’s Masters Degree in Health Data Science. He holds a PhD in health services research and policy and a master’s degree in public health, both from the University of Rochester.
Louis Ehwerhemuepha, PhD
Children's Hospital of Orange County, Orange, CA, USA
Louis Ehwerhemuepha, PhD is the Director of Computational Research at the Children's Hospital of Orange County (CHOC) where he works with a diverse team to develop and manage a pediatric research data science program that supports CHOC's mission to nurture, advance, and protect the health and well-being of children. He leads a team of data scientists and analysts who leverage machine learning, explainable AI, and statistical methods to generate insights and solutions for various clinical and operational challenges.
Rachele Hendricks-Sturrup, DHSc, MSc, MA
Duke-Margolis Center for Health Policy, Washington, DC, USA
Dr. Rachele Hendricks-Sturrup is the Research Director of Real-World Evidence (RWE) at the Duke-Margolis Institute for Health Policy in Washington, DC, strategically leading and managing the Institute's RWE Collaborative.
Podium Session
Healthcare Simulation Modeling in HEOR
Digital Conference Pass
Moderator
Antal Zemplenyi, PhD
University of Colorado Skaggs School of Pharmacy and Pharmaceutical Sciences, Aurora, CO, USA
Antal Zemplényi, PhD, is a health economist with more than 5 years of experience in academia specializing in HTA, health economics and outcome research and over 10 years of experience in the financial management of hospitals. He is the past president of the Hungarian Health Economics Association and the Hungary Chapter of ISPOR. He is the head of the Health Technology Assessment Centre at the University of Pécs and a senior health economist at the Syreon Research Institute. He is currently a Fulbright scholar based at the University of Colorado.
P60: Evaluating Dynamic Treatment Using a Target Trial: The Real-World Effectiveness of Adding Oral Selexipag to a Double Oral Therapy for the Treatment of Patients With Pulmonary Arterial Hypertension
10:45AM - 11:00AM
Tang W1, Panjabi S2, Burger CD3
1Janssen Scientific Affairs, LLC, a Johnson & Johnson company, Philadelphia, PA, USA, 2Janssen Scientific Affairs, LLC, a Johnson & Johnson company, Titusville, NJ, USA, 3Mayo Clinic, Jacksonville, FL, USA
OBJECTIVES: For patients with pulmonary arterial hypertension (PAH), selexipag is often added several months from initiating double oral therapy (DOT) with an endothelin receptor antagonist (ERA) and phosphodiesterase type 5 inhibitor (PDE5i). To appropriately assess the effectiveness of escalation to triple oral therapy (TOT) compared with DOT, it is critical to account for the DOT duration prior to selexipag initiation. This study compared the effectiveness of adding selexipag within 3, 6, and 12 months to DOT versus DOT alone for risk of hospitalization and PAH-related disease progression using a target trial design.
METHODS: A hypothetical, pragmatic trial protocol was developed to analyze real-world claims data from Komodo. Eligible study patients with PAH on DOT for 60 days between July 2015–June 2022 were cloned to both treatment groups and artificially censored when their observed treatment deviated from their assigned treatment strategy. Randomization was emulated using inverse probability treatment weight, and censoring-induced selection bias was accounted for using inverse probability censoring weight. A pooled logistic model was used to estimate the per-protocol effect between the treatment arms. Time to all-cause hospitalization, PAH-related hospitalization, and PAH-related disease progression were compared via adjusted hazard ratios (aHRs).
RESULTS: The risk of all-cause hospitalization (aHR=0.82;95%CI:0.72–0.94), PAH-related hospitalization (aHR=0.81;95%CI:0.70–0.95), and PAH-related disease progression (aHR=0.82;95%CI:0.70–0.95) were significantly lower among patients who added selexipag within 6 months of DOT compared with DOT alone. More pronounced treatment effects were observed when patients added oral selexipag early (3-months), or when continuous treatment was defined using a shorter gap in treatment (45 versus 90-days).
CONCLUSIONS: Adding selexipag as early as 3 months from initiation of DOT of ERA and PDE5i significantly reduced the risk of hospitalization among patients with PAH. Delays in selexipag initiation (≥12 months) and treatment gaps may contribute to suboptimal outcomes.
P57: Clinical Outcomes of Low-Risk Stage II Colon Cancer Survivors Adhering to Guidelines: A Microsimulation Model
10:00AM - 10:15AM
Gursel E1, Samur S2, Gu NY3, Palomares M3, Sahinkoc M4, Erdogan ZM5, Cuyun Carter G3, Ayer T6, Chhatwal J4, Neugut AI7, Parikh A4
1Value Analytics Labs, Boston, MA, USA, 2Value Analytics Labs, Chantilly, VA, USA, 3Exact Sciences, Madison, WI, USA, 4Harvard Medical School, Boston, MA, USA, 5Istanbul Technical University, Istanbul, Turkey, 6Georgia Institute of Technology, Atlanta, GA, USA, 7Columbia University, New York, NY, USA
OBJECTIVES: Colon cancer (CC) is one of the leading cancers in the US and worldwide. Current guidelines recommend that low-risk Stage II CC patients undergo surveillance to monitor potential recurrences post-curative surgery. Despite being classified as low risk, as defined by clinicopathologic factors, these patients are still at risk of recurrence. Long-term clinical outcomes under the current practice in this population remain unclear. This study aimed to develop a lifetime comprehensive microsimulation model to capture the disease prognoses, treatments, and surveillance of these patients, offering a better evaluation of current guideline recommendations.
METHODS:
A Markov-based model was developed to simulate the lifetime natural history of low-risk Stage II CC survivors starting at age 65. Published data were used to inform the disease progression rates, performance characteristics of screening modalities, and real-world screening adherence patterns. The model integrated surveillance algorithms and post-recurrence treatment options as recommended in the current guidelines. We also validated our model with the National Cancer Database records following ISPOR validation guidelines.
RESULTS: The model predicted a 12.1% chance of recurrence over 10 years in low-risk Stage II CC survivors, with 10-year overall survival of 73.2% and recurrence-free survival of 68.0%. The average life expectancy for a low-risk Stage II CC survivor was 16.7 years. The projected recurrences per 10,000 survivors in 10 years was 1,210 (380 locoregional and 830 distant). Current surveillance modalities detect only 530 of these cases, leaving 56% undetected.
CONCLUSIONS: Our microsimulation model offers predictions for clinical outcomes for low-risk Stage II colon cancer survivors, highlighting the suboptimal recurrence detection rates with current guidelines. The model is a valuable tool to identify unmet needs for future studies, such as the use of molecular residual disease testing that could better follow patients’ clinical outcomes post-curative surgery.
P59: A Microsimulation Model for High-Risk Stage II and Stage III Colon Cancer Survivors Following the Current Guidelines
10:30AM - 10:45AM
Samur S1, Gursel E2, Gu NY3, Palomares M3, Sahinkoc M4, Hoban O2, Cuyun Carter G3, Ayer T5, Chhatwal J4, Parikh A4, Neugut AI6
1Value Analytics Labs, Chantilly, VA, USA, 2Value Analytics Labs, Boston, MA, USA, 3Exact Sciences, Madison, WI, USA, 4Harvard Medical School, Boston, MA, USA, 5Georgia Institute of Technology, Atlanta, GA, USA, 6Columbia University, New York, NY, USA
OBJECTIVES: Colon cancer (CC) constitutes approximately 6% of global cancer incidence. Post-curative surgery, current guidelines recommend adjuvant chemotherapy for high-risk patients, as defined by clinicopathologic factors, followed by routine surveillance and monitoring. However, some patients face ongoing risk of recurrence. We developed a novel comprehensive microsimulation model for this population within the framework of current guidelines, providing a nuanced understanding of CC management.
METHODS: A microsimulation model, replicating disease progression and guideline recommendations, was developed using a lifetime horizon with monthly cycles. The starting age of the cohort was 65 years. Treatment decisions post-curative-surgery, post-recurrence, and surveillance algorithms were incorporated into the model following the current guidelines. Disease progression parameters and performance characteristics of the recommended testing modalities were derived from the published literature. The model was validated using the National Cancer Database records following ISPOR validation guidelines.
RESULTS: Our model predicted a 10-year recurrence-free survival of 71.5% for high-risk Stage II and 62.8% for Stage III patients. The 10-year overall survival rate was 73.7% for high-risk Stage II and 68% for Stage III survivors, with post-surgery average life expectancy of 17.1 years and 16.0 years, respectively. The model predicted 810 recurrences per 10,000 high-risk Stage II survivors (250 locoregional and 560 distant) and, 1,820 recurrences for Stage III survivors (570 locoregional and 1,250 distant) over 10 years. However, only 510 (63%) and 1,370 (75%) of recurrences were detected by the current surveillance modalities.
CONCLUSIONS: Our clinically validated model enables the evaluation of long-term clinical outcomes in high-risk Stage II and Stage III CC survivors. Model predictions highlighted suboptimal recurrence detection rates under the current screening modalities. Given the newer emerging molecular residual disease tests, this model could serve as a foundational tool for evaluating both current clinical practices and the potential of these new tests to improve early detection.
P58: Clinical Impact of Replacing 13-Valent With a 15-Valent Pneumococcal Conjugate Vaccine in Costa Rican National Immunization Program
10:15AM - 10:30AM
Rendon AM1, Herrera-Solano W2, Hirata L3, White M4, Huang M4, Weaver J4, Chhabra N5, Singla P6, Kumari S5, Parellada C3
1MSD Central America, Panamá City, Panamá, Panama, 2Hospital México, Caja Costarricense de Seguro Social, San José, San José, Costa Rica, 3MSD Brazil, São Paulo, SP, Brazil, 4Merck & Co., Inc., Rahway, NJ, USA, Rahway, NJ, USA, 5CHEORS, North Wales, PA, USA, 6CHEORS, Sonipat, HR, India
OBJECTIVES: This study aimed to assess clinical impact of replacing PCV13 with PCV15 in the national immunization program (NIP) for infants in Costa Rica.
METHODS: A state-transition Markov model was utilized to simulate the occurrence of cases and deaths associated with pneumococcal disease (PD) in Costa Rica population. The model incorporated three health states: no PD, post-meningitis sequelae (PMS), and death; and tracked the occurrence of acute PD events, including acute otitis media (AOM), non-bacteremic pneumococcal pneumonia (NBPP), and invasive pneumococcal disease (IPD). Health outcomes, including life-years (LYs), were reported. The initial birth cohort in 2024, consisting of 69,654 newborns, and the subsequent 100 cohorts were eligible to receive either PCV13 or PCV15 according to the 2+1 schedule, administered at 2, 4, and 12 months. Costa Rican-specific data from official sources/literature were used, with data from other Latin America countries as proxy, when necessary. A life table was included to account for all-cause mortality among the cohorts. The same vaccine-type efficacy and waning profile was assumed for both vaccines. For the two additional PCV15 serotypes (22F and 33F), the average overall PCV13 vaccine efficacy was applied, including indirect effect for the first five years following PCV15 introduction.
RESULTS: Replacing PCV13 with PCV15 in the Costa Rican NIP was projected to avoid additional 740 IPD cases (53 meningitis, 158 bacteremia without focus, and 528 bacteremic pneumonia), 4,260 NBPP cases (1,865 inpatient and 2,395 outpatient NBPP), 3,297 AOM cases and 16 PMS cases over a 100-year time horizon. Additionally, PCV15 was estimated to avoid 84 IPD/NBPP deaths compared to PCV13, resulting in gains of 1,606 LYs over the 100-year time horizon.
CONCLUSIONS: PCV15 is projected to prevent more PD-related cases and deaths compared with PCV13. These findings highlight the additional impact of PCV15 in preventing PD mortality and morbidity in Costa Rica.
HTA Decisions and Deliberative Processes
Digital Conference Pass
Moderator
Jay Park, PhD
Core Clinical Sciences, Vancouver, BC, Canada
Jay Park is the Founder and Scientific Lead of Core Clinical Sciences (CCS) and an Assistant Professor at McMaster University. Jay specializes in innovative trial designs, decision analytics, and evidence synthesis methods. At CCS, Jay helps clients explore and apply appropriate study designs and address difficult clinical development and reimbursement problems. Jay has over 40 publications and the lead author of the book “Introduction to Adaptive Trial Designs and Master Protocols” with the Cambridge University Press. Prior to his work with CCS, he has worked with larger consultancies. At McMaster University, Jay has taught graduate-level courses on adaptive trial designs and master protocols.
P61: Payer Willingness to Pay for Novel Value Attributes: The Impact of Higher WTP Thresholds on ICER’s Cost-Effectiveness Determinations
10:00AM - 10:15AM
Penley B1, Downen S2, Zheng C3, Westrich K4
1Cencora, Durham, NC, USA, 2Cencora, Conshohocken, PA, USA, 3Cencora, WESTLAKE, OH, USA, 4Cencora, Herndon, VA, USA
OBJECTIVES: To investigate (1) whether payers have higher willingness to pay (WTP) for interventions that meet novel value criteria: groundbreaking durable/curative, disease severity, and health equity; and (2) for relevant interventions reviewed by ICER from 2021‒2023, how many additional treatments would be deemed cost-effective at higher WTP thresholds.
METHODS: A double-blinded web-based survey of US healthcare payers was fielded in July 2023 through Cencora’s research panel, the Managed Care Network, to assess payer WTP for interventions meeting novel value criteria. Identified ICER reports assessing pharmaceutical interventions published between Mar 2021 and Nov 2023 that included an appraisal committee (AC) meeting (N=54). Determined whether an intervention met novel value criteria based on surrogate measures: (1) groundbreaking durable/curative: use of single and short-term therapies (SST) framework (n=6); (2) disease severity: ≥50% AC voted disease had high acuity of need (n=7); and (3) health equity: ≥50% AC voted intervention would have positive impact on health inequities (n=5).
RESULTS: A total of 48 payers participated in the survey. Most payers strongly agreed/agreed they would pay more per unit of health gained for groundbreaking durable/curative treatments (75%). Fewer strongly agreed/agreed they would pay more per unit of health gained for interventions that treated higher-severity diseases (40%) or reduced health inequities (33%). At a WTP threshold of $100,000, ICER found 3 “groundbreaking durable/curative” treatments cost-effective (50%), 1 “disease severity” treatment cost-effective (14%), and 1 “health equity” treatment cost-effective (20%). At WTP threshold of $200,000, the respective numbers rose to: 6 (100%), 4 (80%), and 2 (29%). At $300,000, the numbers rose to: 6 (100%), 5 (100%), and 3 (43%).
CONCLUSIONS: Some payers are willing to pay more for interventions meeting novel value criteria. With increased WTP thresholds, more interventions meeting novel value criteria would be considered cost-effective, with potential implications for formulary decision-making and patient access.
P62: Targeted Literature Review of the Use of Real-World Evidence From Single Real-World Data Sources and in Meta-Analyses: Methodologies and Recommendations From Health Technology Assessment Bodies
10:15AM - 10:30AM
Martin C1, Diawara K2, Jaiswal AK3, Gamburg R4, Miller J2, Park C2, Ranjan N5, Mehta AM2, Tamminina D6, Shah T3, Schmerold L2, Hood D7
1Axtria, Hamilton, ON, Canada, 2Axtria, Berkeley Heights, NJ, USA, 3Axtria, Hyderabad, AP, India, 4Axtria, Boston, MA, USA, 5Axtria, Noida, UP, India, 6Axtria, Gurugram, HR, India, 7Axtria, Gales Ferry, CT, USA
OBJECTIVES:
Real-world evidence (RWE) can mitigate challenges surrounding clinical evidence generation, and is therefore growing popular in health technology assessments (HTAs). Building upon this is the novel process of meta-analysis (MA) of RWE. This study aims to characterize the current role of RWE and MA of RWE in decision-making by HTA and regulatory bodies.
METHODS:
A targeted literature review of PubMed and Google Scholar explored methodologies used to generate and apply RWE and MA of RWE to HTA submissions between 2022-2023. Guidance documents from the Food & Drug Administration (FDA) and National Institute for Health and Care Excellence (NICE) websites were reviewed and summarized.
RESULTS:
Screened search results included 25 studies of (MA of) RWE in HTA submissions and 6 guidance documents. Methodologies used in RWE studies included external comparators (ECs) for single-arm clinical trials (SATs), pragmatic trials, target trial emulations, inferential models, and machine learning. In a study of 433 HTA submissions with SATs between 2011-2019, 52% employed ECs. Submissions with RWD ECs had a higher approval rate (59%) compared to those with no EC data (43%). MA of RWE studies integrated evidence from multiple sources through confidence profile methods, naive data synthesis, cross design synthesis, and three-level hierarchical models. Guidance on RWE use varied, with FDA guidance focusing on the fit of RWE and the quality of evidence, and NICE focusing on the three tenets of planning, conduct, and reporting. However, neither provided guidance on the use of MA of RWE in HTA submissions.
CONCLUSIONS:
HTA bodies provide little guidance regarding the use of MA of RWE. To facilitate the use of these evidence sources, HTA bodies must establish reporting/quality standards and standard frameworks for analyses. Future studies should evaluate the impact of the use of RWE and MA of RWE studies on recent regulatory submissions.
P64: Which Healthcare-System Factors Influence Reimbursement Success Rates? A Multi-Country Analysis of Reimbursement Decisions in 2023
10:45AM - 11:00AM
Piper R1, Shaw A2, Wantling J1, Darlington O3
1Initiate Consultancy, London, London, UK, 2Initiate Consultancy, London, UK, 3Initiate Consultancy, NA, UK
OBJECTIVES:
Health technology assessment (HTA) is used internationally to establish whether new interventions represent an efficient allocation of scarce healthcare resources. However, reimbursement decisions are often inconsistent between countries likely due to differences in HTA frameworks adopted and wider political and socio-economic factors. The objective of this study was to investigate the relationships between different health-system factors and the likelihood of a successful HTA outcome across several countries.
METHODS:
HTA recommendations published in 2023 across 10 countries (England, Scotland, France, Denmark, Spain, Ireland, Netherlands, Sweden, Australia, and Canada) were reviewed, and data were collated describing the total number of appraisals, and the number resulting in positive reimbursement recommendations. Health-systems data was also collected for each country, including healthcare expenditure (as a proportion of GDP), healthcare funding source (primarily social insurance or tax-financed), pharmaceutical pricing policy, and HTA processes such as cost-effectiveness and patient-group involvement. Univariate meta-regression models were used to examine associations between each of the health-systems factors and positive reimbursement recommendations.
RESULTS:
In total, 783 HTA appraisals were conducted across the 10 countries in 2023, of which 527 (67%) received a positive reimbursement decision. Substantial differences in reimbursement decisions were observed between countries, with Ireland having the lowest proportion of positive reimbursement recommendations (7%) and France having the highest (88%). The meta-regression analysis indicated that healthcare expenditure was a statistically significant predictor of reimbursement success rate (p<0.001), whereby countries with greater healthcare expenditure are more likely to reimburse new products. Countries where healthcare is predominantly funded through taxation, whose HTA framework includes cost-effectiveness assessment, and who do not operate a free pricing model were nominally less likely to issue positive recommendations.
CONCLUSIONS:
Significant heterogeneity was observed in the reimbursement success rates between the countries included in the analysis. In general, countries with higher healthcare expenditure more frequently recommend new products for reimbursement.
P63: Comparative Analysis of Health Technology Assessment Agency Frameworks for Digital Health Technologies
10:30AM - 10:45AM
Blackmore L, Cribbs K, Lahue BJ
Alkemi LLC, Manchester Center, VT, USA
OBJECTIVES: Regulatory and payment pathway guidance are emerging related to digital health technologies (DHTs). As DHT applications are commercialized, healthcare technology assessment (HTA) agencies will play an increasingly important role in assessing their potential value. The goal of this study was to identify and review available DHT value assessment frameworks.
METHODS: We conducted a targeted review using PubMed and HTA sites to identify DHT assessment frameworks from HTA authorities in North America and Europe. We prioritized DHT frameworks published in English from January 1, 2018-November 10, 2023. Frameworks limited to DHT subcategories (e.g., mobile health apps, personalized medicine solutions) were excluded. Twelve EUnetHTA HTA Core Model domains (Health problem and current use of technology; Technical technology characteristics; Safety; Clinical effectiveness; Patient and social aspects; Economics; Legality; Ethics; Organizational impact; Usability; Data security; Interoperability) informed descriptive analysis.
RESULTS: Three DHT frameworks met inclusion criteria from the US (ICER), the UK (NICE), and Finland (Digi-HTA). All 3 frameworks covered 5/12 domains: ‘safety,’ ‘clinical effectiveness,’ ‘economics,’ ‘usability,’ and ‘data security.’ ‘Technological characteristics’ (ICER, Digi-HTA), ‘patient and social aspects’ (NICE, ICER), and ‘interoperability’ (NICE, Digi-HTA) were addressed by 2/3 frameworks. ‘Health problem and current use of technology’ was covered in ICER's framework. NICE addressed both ‘legal’ and ‘ethical’ guidance. No framework covered ‘organizational impact.’ NICE's framework encompassed the most domains (9/12), followed by ICER (8/12) and Digi-HTA (7/12). Related to the ‘economics’ category, budget-impact analysis was recommended by ICER and NICE, and cost-effectiveness analysis was suggested by NICE (for costly interventions) and Digi-HTA.
CONCLUSIONS: Few HTA agencies have published broad DHT assessment frameworks. Among existing frameworks, assessment criteria vary, and domains salient to DHT evaluation—including technological characteristics, interoperability, and ethics—are not universally considered. Establishment of clear, explicit evidence standards is needed to facilitate robust DHT value assessment.
10:00 AM - 11:15 AM
Spotlight Session
How to Adjust Economic Models for Health Equity in the Conduct of Generalized Cost-Effectiveness Analysis (GCEA)
Digital Conference Pass
Level: Experienced
The ISPOR value flower has provided the field with a list of ‘what’ novel value elements should be incorporated into cost-effectiveness analysis (CEA), among them the value of health equity. Health equity, while important to consider, does not have an established system of measurement and weighting on economic evaluation. Recent measurement and methodological advancements that consider the importance of health equity on decision-making can play into approaches for generalized cost-effectiveness analysis (GCEA).
Padula will provide an overview GCEA and the relative importance of adjusting economic models for health equity. Lakdawalla will introduce the generalized risk-adjusted cost-effectiveness (GRACE) framework as a way of overcoming discriminatory decision-making by adjusting for baseline disability and concerns about health equity. Basu will introduce a new measurement system to health utility, Healthy Years in Total (HYT) as an alternative to QALYs that addressed health equity concerns. Vasco Pontinha will introduce a novel method to encapsulate health equity based on budgetary constraints for mainstream health technologies. At the end of the workshop, the discussants will poll the audience about which method(s) may be able to gain traction to make U.S. health technology assessment processes more equitable.
Discussion Leaders
William Padula, PhD, MSc, MS
University of Southern California, Los Angeles, CA, USA
William Padula, PhD is Assistant Professor of Pharmaceutical & Health Economics at the University of Southern California School of Pharmacy, and a Fellow in the Leonard D. Schaeffer Center for Health Policy & Economics. He is a Co-Founder & Principal at Stage Analytics. His research focuses on the theoretical foundations of medical cost-effectiveness analysis and applications of machine learning. He was the 2021 recipient of ISPOR’s Bernie O’Brien New Investigator Award, Co-Chair of the ISPOR Machine Learning Task Force, and is an Associate Editor for Value in Health.
Discussants
Anirban Basu, PhD, MS
University of Washington, Seattle, WA, USA
Anirban Basu is a Professor of Health Economics and the Stergachis Family Endowed Director of The CHOICE Institute at the University of Washington, Seattle. He holds joint appointments with the Departments of Health Systems & Population Health and Economics at UW, is a Faculty Research Fellow at the US National Bureau of Economic Research, and an elected Fellow of the American Statistical Association. His work sits at the intersection of microeconomics, statistics, and health policy. His research focuses on understanding the economic value of health care, generating causal evidence, and, lately, on the potential for discrimination with machine learning and artificial intelligence algorithms. He served on the Second Panel on Cost-effectiveness Analysis in Health and Medicine and serves on the Editorial Advisory Board for Value in Health Journal. He received his master's in Biostatistics from UNC-Chapel Hill and a PhD in Public Policy Studies from the University of Chicago.
Darius Lakdawalla, PhD
USC Leonard D. Schaeffer Center for Health Policy and Economics, Los Angeles, CA, USA
Darius Lakdawalla is a widely published, award-winning researcher and leading authority on health economics and health policy. He holds the Quintiles Chair in Pharmaceutical Development and Regulatory Innovation at the University of Southern California, where he sits on the faculties of the School of Pharmacy and Sol Price School of Public Policy. He is also the Director of Research at the USC Leonard D. Schaeffer Center for Health Policy and Economics, one of the nation’s premier health policy research centers. His research has focused primarily on the economics of risks to health, the value and determinants of medical innovation, the economics of health insurance markets, and the industrial organization of healthcare markets.
Vasco Pontinha, PhD, MPharm, MBA
Virginia Commonwealth University School of Pharmacy, Richmond, VA, USA
Vasco Pontinha is a health economist specialized in healthcare quality, medication use, and economic evaluation of pharmaceutical and non-pharmaceutical interventions in healthcare. He received his PhD from the Virginia Commonwealth University School of Pharmacy where he developed group-based trajectory models to identify patterns of long-term medication adherence and implemented machine learning algorithms to evaluate value-based health care policy. He was also able to attract extramural funding to pursue his doctoral dissertation. Prior to his doctoral training, he received his pharmacy degree from the University of Lisbon and his Master in Management from the Católica-Lisbon School of Business and Economics, and worked extensively for pharmaceutical companies and in consulting.
11:00 AM - 11:30 AM
Break
11:30 AM - 12:45 PM
Plenary Session
AI Enabling Whole Health: Opportunities and Challenges for HEOR and HTA
Digital Conference Pass
Artificial intelligence (AI) is transforming the life sciences and healthcare sectors, offering unprecedented possibilities for innovation, efficiency, and quality. How can we harness the power of AI while ensuring its ethical, reliable, and fair use? How can we leverage AI to generate and communicate evidence that supports health technology assessment (HTA) and health economic and outcomes research (HEOR)? And how can we prepare ourselves for the future of AI and its impact on Whole Health? Join us in this plenary session where we will explore these questions and more from different perspectives and domains. You will learn about:
- The basics of AI and generative AI (GenAI), and how they differ from traditional data analysis and modelling techniques
- The current and emerging applications of AI in life sciences and healthcare, and how they are driving innovation and value across
- The challenges and opportunities of working with unstructured data sources, such as natural language, images, and real world evidence in the field of HEOR and HTA
- The technical and methodological aspects of developing, validating, and interpreting AI models and outputs, and how to ensure their transparency, reproducibility, and accountability
The session will feature presentations from experts in AI, HEOR, and HTA, followed by an interactive panel discussion.
Moderators
Thomas Senderovitz, MD
NovoNordisk, Copenhagen, Denmark
Thomas Senderovitz is an M.D. from University of Copenhagen. He has more than 30 yrs experience from healthcare, pharmaceutical industry, biotech, CRO and regulatory authorities / governmental administration.
Since 2021 Senior Vice President Data Science at Novo Nordisk leading app 1100 data scientists across various domains and functions incl. Clinical Data Operations & Insights, Biostatistics/Statistical Programming, AI/ML & Real World Evidence, Pharmacometrics, Data Orchestration, External Affairs and Strategic and Digital Development. He recently joined the Board of Directors of Instem.
Previously CEO of the Danish Medicines Agency, Chair of Heads of Medicines Agencies Management Group and member of EMA Management Board. Member of executive management of PAREXEL, Grünenthal and VP Global Exploratory Development of UCB, VP Clinical R&D Ferring Pharmaceuticals a.o.
Speakers
Anirban Basu, PhD, MS
University of Seattle, Seattle, WA, USA
Anirban Basu is a Professor of Health Economics and the Stergachis Family Endowed Director of The CHOICE Institute at the University of Washington, Seattle. He holds joint appointments with the Departments of Health Systems & Population Health and Economics at UW, is a Faculty Research Fellow at the US National Bureau of Economic Research, and an elected Fellow of the American Statistical Association. His work sits at the intersection of microeconomics, statistics, and health policy. His research focuses on understanding the economic value of health care, generating causal evidence, and, lately, on the potential for discrimination with machine learning and artificial intelligence algorithms. He served on the Second Panel on Cost-effectiveness Analysis in Health and Medicine and serves on the Editorial Advisory Board for Value in Health Journal. He received his master's in Biostatistics from UNC-Chapel Hill and a PhD in Public Policy Studies from the University of Chicago.
Alexandra Berk, PhD
CVS Healthspire Life Sciences Solutions, Woonsocket, RI, USA
Alexa Berk leads Real World Evidence (RWE) for CVS Health®, serving as General Manager, CVS Healthspire™ Life Sciences Solutions - RWE. Alexa has 20 years of experience in observational and clinical research, starting her career at Memorial Sloan Kettering Cancer Center and moving into research leadership positions in life sciences, consulting, and startup environments. Alexa was most recently Medical Director for Digital Health and Data at Invitae, prior to joining CVS Health® in 2022 as Chief Scientific Officer for RWE. Alexa has co-authored manuscripts and abstracts in the research literature across indications, with a particular focus on oncology and rare diseases, and is a frequent speaker on the use of high-quality, patient-centric, and equitable research approaches. She is a graduate of Barnard College and the New School for Social Research.
Rachael Fleurence, PhD
NIH, Washington, DC, USA
Dr Fleurence is a Senior Advisor at the National Institutes of Health where she is working on launching a national initiative to eliminate Hepatitis C in the United-States. Dr Fleurence is also affiliated with the National Institute of Biomedical Imaging and Bioengineering where she works on advances in Artificial Intelligence and Machine Learning. Dr Fleurence currently co-leads the ISPOR Task Force on the suitability of EHR data for Health Technology Assessments. Previously, Dr Fleurence served as a senior health advisor in the Biden-Harris White House, Dr. Fleurence received a BA from Cambridge University (United Kingdom), an MA in business management from ESSEC-Paris (France), and an MSc and PhD in health economics from the University of York (UK).
