Lunch on own, available for purchase at Outtakes (located in the BCEC) or grab a bite at a nearby restaurant.
Bring your Monday lunch voucher to any catering station located in the exhibit hall for hot/cold lunch options. Vouchers will be provided to attendees upon receiving their badge at registration.
PURPOSE: To debate the value of methods for developingendpoints based on wearable device data for use in clinical and real-world research. Methods include a patient-research-driven approach highlightingqualitative patient input, and a data-driven approach emphasizing insights from quantitative data exploration.An introduction to wearable device data and endpointswill be provided, and the strengths and weaknesses of each development approach will be examined, including how different methods may apply in registration and reimbursement contexts.
DESCRIPTION:
Wearable devices that measure various aspects of health, including sensor and platform-based applications (e.g., activity monitors, sleep mats), are increasingly used in clinical and observational research. These devices allow for collection of passive – and massive – datasets that capture elements of the participant’s daily functioning that may be uniquely important in evaluating treatment efficacy and safety. Further, HTAs often have a focus on issues like – “what will the treatment allow patients to do” for which wearable technology may provide important insights. This session will debate two approaches for creating endpoints from wearable device data: a patient-research driven approach versus a data-driven approach. Regulatory and payer contexts will be considered. 1) Dr. Garcia: Introduce panelists and issue (10min)
2) Dr. Byrom: Argue a patient-research drivenapproach that follows an established method for creating clinical outcome assessment endpoints, where endpoints are deduced from conceptual models developed from resources like patient and expert interviews (15min)
3) Dr. Gwaltney: Argue a data-driven approach that starts with a dataset and induces endpoints based on relationships between variables (15min)
4) Dr. Lloyd: Bring the payer perspective by providing commentary on both approaches in the context of adding value to HTAs, in particular how each approach develops endpoints that may help HTAs assess treatment impact on daily functioning (15min)
5) Dr. Garcia: Conclude with a real-time pollregarding audience views (5min)
Discussion Leaders
Julia Garcia, PhD, MS
AbbVie, Irvine , CA, USA
Julia K. Garcia, PhD, MS is a Director in the Patient-Centered Outcomes Research group at AbbVie, Inc. She is a subject matter expert on patient experience data particularly the development, implementation, and analysis of patient-reported outcomes and strategic communication of these data to regulatory agencies globally for new drug approvals and to the scientific community. She has worked in the pharmaceutical industry since 2014 in patient centered outcomes research and medical writing teams. She holds a PhD in Public Health from the University of California San Diego, School of Medicine, and an MS in Nutritional Sciences from San Diego State University. During her time at the University of San Diego, she worked at the UCSD Center for Wireless Communication that assessed wireless and networked technology (eg, wearables, social networks) to improve health behaviors. She is interested in understanding new applications for digital health to expand understanding of the patient experience and to make healthcare more accessible, convenient, and valuable to patients.
Discussants
Bill Byrom, B.Sc., Ph.D.
Signant Health, Nottingham, NTT, United Kingdom
Bill serves as Vice President at Signant Health, a global company providing technology solutions for pharmaceutical clinical trials. He has worked in the Pharmaceutical industry for 30+ years and is a recognised industry leader in eClinical product strategy, electronic clinical outcome assessments (eCOA), and decentralized clinical trials. Bill is an experienced scientific expert and the author of over 70 publications, and two industry textbooks on electronic patient-reported outcomes (ePRO). His recent scientific work includes the use of wearable and sensor technology, and bring-your-own-device (BYOD) eCOA in clinical trials. Bill serves on two industry expert task forces within ISPOR, is a member of the scientific leadership board of the Digital Medicine Society (DiMe), and a core contributor to the US Critical Path Institute’s eCOA Consortium. Bill is a senior visiting professor within Nottingham Trent University's Medical Engineering Design Research Group.
Chad Gwaltney, PhD
Gwaltney Consulting, Westerly, RI, USA
Chad Gwaltney, Ph.D. is President and Principal Consultant at Gwaltney Consulting. Dr. Gwaltney develops innovative methods to measure patient-centered outcomes in clinical trials. He has published over 100 peer-reviewed articles and book chapters addressing how the patient’s perspective can be examined to better understand medical product efficacy and safety. He has served on US NIH grant review committees and has co-authored industry best practice guidelines on the development and use of clinical outcome assessments. Before founding Gwaltney Consulting, Dr. Gwaltney was Chief Scientist and Regulatory Advisor, Endpoints, at ERT. He has also served as Head, Patient Experience, Hematology/Oncology, at Celgene and as a research faculty member in the Brown University School of Public Health.
Andrew Lloyd, DPhil
Acaster Lloyd Consulting Ltd, Oxford, Oxfordshire, United Kingdom
Member Group Meeting
New Special Interest Groups in Development Open Meeting: Global Access to Medical Innovation/Environmental Sustainability in HEOR
Breakouts: IP, WS, & OBS
What If Patients Are Not Acting As Expected? Methodological Options for Examining Behavioral Heterogeneity in Patient Preference Studies
Digital Conference Pass
ISSUE:
Preference elicitation methodologies require researchers to make assumptions about patients’ choice behavior, including their willingness to make trade-offs, the presence or nature of heterogeneity in preferences, and the role of the status-quo option. These assumptions guide the design of stated preference instruments as well as the analysis of collected data. However, common assumptions may not always adequately reflect respondents’ choice behaviour. Understanding the implications can help improve instrument design, and may guide the development of targeted validity tests and analytical approaches. OVERVIEW:
The panel will initially introduce fundamental assumptions made in health preference research. Panellists will then provide insights into how and why some of these assumptions may be violated and the potential implications that these have on instrument design, analysis, and data interpretation. Dr. Heidenreich will outline implications of non-trading behavior in probabilistic thresholding. He will give an example of how internal validity tests may be able to detect such behavior and provide advice on adequate analytical approaches. Dr. Boeri will discuss challenges resulting from protest behaviour and serial non-participation. He will outline the use of hurdle models to account for such choice behavior. Dr Ng will discuss the role of the status quo in choice experiments and outline how status-quo options have been handled in the literature and the common pitfalls observed. Dr Tervonen will discuss the role patient-reported outcomes can play in understanding preference heterogeneity using two case studies. The panel will close with an open discussion with the audience about implications of behavioral heterogeneity in patient preference research. Patient preference researchers and HEOR scientists benefit from attending this advanced panel.
Moderators
Tommi Tervonen, PhD
Kielo Research, Zug, ZG, Switzerland
Dr Tervonen is the Chief Scientist of Kielo Research and key opinion leader in patient preferences and benefit-risk assessment. His expertise influences decision maker opinion and he has been consulted by agencies such as the US Food and Drug Administration (FDA) and UK National Institute of Health and Care Excellence (NICE). Dr Tervonen co-chairs the ISPOR Task Force on Good Practice in Quantitative Benefit-Risk Assessment and has served as principal investigator in dozens of patient preference studies, including an IMI PREFER industry case study, and studies used in regulatory filings.
Panelists
Marco Boeri, PhD
OPEN Health, Belfast, Ireland
Marco Boeri, PhD, is a Director of preference research at Open Health and honorary professor in practice in health economics at Queen's University Belfast. Dr. Boeri has 15 years of experience in preference assessment in environmental and health economics and 2 years of experience in the private financial sector in marketing. He has extensive knowledge and expertise in experimental design, survey development, and modeling data from preference studies in health, food, and environmental economics. Dr. Boeri’s research focuses on the econometric analysis of heterogeneity in discrete-choice experiments and on comparing various stated preference analysis methodologies at the individual, household, and sample level.
Sebastian Heidenreich, MSc, PhD
Evidera, London, LON, United Kingdom
Xinyi Ng, MS, PhD
U.S. FDA, Rockville, MD, USA
Xinyi Ng, PhD is a visiting scientist with the Division of Analytics Benefit-Risk Assessment (ABRA) within the Office of Biostatistics and Pharmacovigilance, CBER, FDA. She leads patient preference information (PPI) studies within CBER and is part of the science of patient input (SPI) working group, which aims to advance the use of patient experience data, including PPI, to inform regulatory science. Prior to joining the FDA, she was a scientist in a HEOR consulting firm and have experience conducting PPI studies. She has a PhD in Pharmaceutical Health Services Research from the University of Maryland Baltimore.
Considering the Impact of the Inflation Reduction Act on Drug Development and Patients
Digital Conference Pass
ISSUE: The US pharmaceutical market is the largest in the world, resulting in high biopharmaceutical investment in research and development (R&D). As an attractive market with prices determined by private market negotiations, the US benefits from early-launches, ensuring patients currently obtain the fastest and widest access to therapies in the world. The new Inflation Reduction Act (IRA) contains measures to curb Federal spending on medicines. The impact of these measures is uncertain, but this session provides some early perspectives on their possible impact on incentives for developers, launch timings for new medicines and indications, and patient access.
OVERVIEW: We present and debate the potential implications of the IRA on drug development incentives and patient access, drawing on examples from relevant disease areas and lessons from Europe where price negotiations have been standard for many years. Steuten will moderate, setting the scene by briefly introducing the IRA and specifically its price negotiation provisions, and outlining transferable evidence from Europe, providing early evidence on the link between price/expenditure limits and access delays. Mehrotra will provide an investor’s perspective, outlining the potential impact on incentives for drug development and concomitantly the impact on innovation across therapeutic areas. Ward will provide a patient’s perspective on access to emerging therapeutics for Alzheimer’s disease and other treatments focused on older adult populations. Spangler will outline the challenges and benefits of using value assessment elements in price negotiations, balancing equitable patient access and financial sustainability. Each speaker will talk for 8 minutes, before the panel together look forward and discuss (13 minutes): How can we work together to optimise the negotiation process and the implementation of the IRA? What lessons can be leveraged from elsewhere? Steuten will encourage an open debate, seeking additional questions and perspectives from the audience (15 minutes).
Moderators
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).
Panelists
Ravi Mehrotra, PhD
5point0, New York, NY, USA
Dr. Mehrotra is the Founder of 5point0 consulting and is currently a Partner at Flagship Pioneering. Previous to investing and operational roles in biopharma, Dr. Mehrotra spent over two decades in Healthcare equity research and investment banking. He was a Senior Managing Director of the Healthcare Research Team at Evercore. a partner at MTS Health Partners, and spent 11 years at Credit Suisse as an Analyst and head of their global Biotechnology research team. During his time at Credit Suisse, Dr. Mehrotra was ranked the number one Biotechnology Analyst in both Institutional Investor and Extel surveys. Prior to Credit Suisse, Dr. Mehrotra was Head of European Biotechnology Equity Research at SG Cowen and a Pharmaceuticals Equity Analyst at Deutsche Bank.
Jason Spangler, MD, MPH, FACPM
Innovation and Value Initiative, Alexandria, VA, USA
For nearly two decades, Dr. Spangler, IVI’s Chief Executive Officer, has worked in the professional health policy and public health sector with pharmaceutical and non-profit organizations. Dr. Spangler joins IVI from his most recent role as Executive Medical Director and Director and Head of Global HTA Policy Strategy & Engagement for Amgen, Inc. Dr. Spangler earned his MD at the Pennsylvania State University College of Medicine in 1998 and his Master of Public Health from Johns Hopkins Bloomberg School of Public Health in 2002.
Michael Ward, MS
Alliance for Aging Research, Washington, DC, USA
Michael Ward, MS, is the Vice President of Public Policy and Government Relations at the Alliance for Aging Research in Washington, DC. At the Alliance, he focuses on advancing legislative and regulatory policies that support access to high-quality healthcare for older adults.
Michael previously served as Director of Payment Policy at Premier, Inc., where he led regulatory advocacy to shape emerging provider payment models and physician payment policy. He also previously served as Senior Associate Director of Policy Research at the American Hospital Association.
Medicare Coverage With Evidence Development for Drugs Approved for Alzheimer’s Disease: Which Study Designs and Data Sources Would Best Answer Medicare’s Questions?
Digital Conference Pass
ISSUE:
Several disease modifying therapies for Alzheimer’s Disease are likely to be approved by the FDA within the next 1-3 years, showing direct evidence of cognitive benefits. The Medicare program has indicated that these drugs will only be covered when patients participate in clinical studies designed to evaluate real world benefits and risks of these drugs. Panelists will debate three different approaches to generate this evidence: pragmatic clinical trials, clinical registries, and secondary analysis of routinely collected healthcare data
OVERVIEW:
In 2021, the FDA approved the first disease-modifying therapy for Alzheimer’s Disease (AD) using the accelerated approval pathway, based on the surrogate marker beta-amyloid. In 2022, CMS issued a national coverage decision that limited coverage of these FDA-approved drugs to patients enrolled in randomized controlled trials, citing the lack of direct evidence of cognitive benefit. CMS stated that future drugs approved with direct evidence of cognitive benefits would be covered under a coverage with evidence development (CED) policy. Medicare’s CED policy requires that patients receiving these therapies are included in “prospective comparative studies” as a condition of payment, allowing some flexibility in study design and data source. The participants in this issues panel will debate the pros and cons of three different approaches to evidence generation for CED studies of drugs for AD. The moderator will provide a 10 minute summary of the CMS policy, followed by the 3 panelists describing their recommended evidence generation approach (12 minutes each). The remainder of the time will be allocated to moderated dialogue with the panelists and audience members. This issues panel will appeal to those interested in understanding different approaches to studying the real world benefits and risks of novel therapies.
Moderators
Sean Tunis, MD, MSc
Tufts University, Boston, MA, USA
Sean Tunis, MD, MSc. is a Principal with Rubix Health where he consults with public and private sector organizations on health technology issues at the interface of regulatory and reimbursement policy, market access, comparative effectiveness, outcomes measurement and health technology assessment. He is also a Senior Fellow in the Tufts Center for the Evaluation of Value and Risk in Health. Dr. Tunis also serves as a Mentor-in-Residence at Johns Hopkins Tech Ventures, helping early stage companies to develop their initial reimbursement strategy and to align their clinical development programs with payer and regulatory requirements.
Panelists
Maria Glymour, ScD, MS
UCSF, San Francisco, CA, USA
Maria Glymour is a Professor in the Department of Epidemiology and Biostatistics at the University of California, San Francisco and incoming Chair of the Department of Epidemiology at the Boston University School of Public Health.
Nirosha Mahendraratnam Lederer, PhD, MSPH
Aetion, Inc, Washington, DC, USA
Nirosha Mahendraratnam Lederer, PhD is Head of US Federal Government and Senior Director of Real-World Evidence Strategy at Aetion. In this role, she leads partnership opportunities with the US federal government and advises clients on real-world evidence trends. Dr. Lederer has over 15 years of research experience using real-world data to support high-value decision-making in the US healthcare system. Before joining Aetion, she led the real-world evidence portfolio at the Duke Margolis Center for Health Policy including developing policies and strategies for increasing the usability and acceptance of RWD and RWE for regulatory and payment decision-making. She previously served as Subject Matter Expert in Patient-Focused Drug Development at the US FDA Oncology Center of Excellence where she supported the use and evaluation of novel drug development tools. Prior to FDA, Dr. Lederer worked at Avalere Health as Manager of Health Economics and Outcomes Research / Evidence-based Medicine Policy. In this capacity, she created global evidence generation programs as well as led policy development and government engagement strategies for Fortune 100 healthcare companies. Earlier, Dr. Lederer served on Capitol Hill with the House Committee on Ways and Means Subcommittee on Health during the passage of the Affordable Care Act.
She received her PhD in Health Outcomes and Policy from the UNC Eshelman School of Pharmacy. She also received her MSPH in Health Policy and Management from the Johns Hopkins Bloomberg School of Public Health and BA in Public Health from the Johns Hopkins University.
Emily Zeitler, MD, MHS
Dartmouth-Hitchcock Medical Center, Lebanon , NH, USA
Emily Zeitler is an Assistant Professor of Medicine at the Geisel School of Medicine at Dartmouth and The Dartmouth Institute as well as clinical faculty at the Dartmouth-Hitchcock Medical Center in clinical cardiac electrophysiology. Dr. Zeitler graduated from Emory Medical School with honors and received her clinical training in internal medicine at Massachusetts General Hospital. She obtained training in cardiology and electrophysiology at Duke University where she also completed training in clinical research at the Duke Clinical Research Institute where she earned a Masters in Health Sciences. Dr. Zeitler has experience in medical device regulatory policy and is engaged in device regulatory policy and general heart rhythm-related health policy. Dr. Zeitler is also engaged in cardiovascular outcomes research broadly with a particular interest in outcome disparities related to implantable medical devices and atrial fibrillation.
In Search of a Goldilocks Reference Case in the US: Could an Insured Member or Patient-Informed Reference Case Be 'Just Right?'
Digital Conference Pass
ISSUE: There is ongoing debate regarding how to develop economic evaluations that meet the needs of healthcare payors (including employers), patients and caregivers, as well as members of society who may become patients, but are also struggling with ever-increasing insurance premiums. During this issue panel, we will debate the potential merits of an “insured member unit” and “patient-informed” reference cases.
OVERVIEW: Reference cases are intended to improve consistency and comparability across cost-effectiveness analyses (CEA). Current best practices recommend presenting healthsector- and societal-perspective reference cases alongside one another. The health sector perspective is criticized as too narrow and insufficiently reflecting costs incurred by patients and their families. Although the societal reference case is praised for including many costs important to employers, patients, and their caregivers, it is also criticized as impractical because it includes costs that are hard to quantify and potentially irrelevant for informing healthcare payer decisions (e.g., criminal justice, environment). As a result, most CEAs do not take a societal perspective. Among those that do take a societal perspective, many impacts recommended by the Second Panel on CEA are omitted. This inconsistency limits comparisons across CEAs and has been criticized by healthcare stakeholders, especially members of the patient community.
Barry Liden will introduce the topic and engage the audience throughout the debate. Dr. Jon Campbell will introduce the concept of an insured member unit perspective and outline how this approach could align the priorities of US healthcare stakeholders. Dr. Julia Slejko will introduce the concept of a patient-informed reference case, differentiating it from an insured member perspective, and describing how it would align priorities of payors, society, and patients. Dr. Oehrlein will evaluate the two concepts, considering how they may address ongoing discussions about integrating novel value elements in V/HTA. Drs. Cambell, Slejko, and Oehrlein will debate the two concepts.
Moderators
Barry Liden, Juris Doctor (JD)
USC Schaeffer Center, Garrett Park, MD, USA
Barry Liden is the Director of Public Policy. He works with the Schaeffer Center leadership to amplify the Center’s impact on public policy through the development and implementation of high-impact strategies; research translation through the strengthening of existing and new methods for maximizing dissemination and uptake of our initiatives; and liaising with key stakeholders through the creation and cultivation of relationships with internal and external stakeholders.
Barry was with Edwards Lifesciences from 2002-2022, where he served in various roles, including patient engagement, government affairs and communication. Most recently, he served as the Vice President of Patient Engagement, where he pioneered the advancement of the patient engagement function in the medical device industry. Also, Barry was formerly an Associate Professor at Georgetown University’s School of Nursing and Health Studies from 2018-2022 and Adjunct Faculty at USC Annenberg from 2002-2007.
Barry is a USC Sol Price School of Public Policy alumnus and received his J.D. from Loyola Law School.
Panelists
Jon Campbell, PhD
Institute for Clinical and Economic Review, Hingham, MA, USA
Jon Campbell is Senior Vice President for Health Economics at the Institute for Clinical and Economic Review (ICER). Jon is a leader in value assessment methods and application. He is an author of over 250 manuscripts and abstracts in the field of value assessment, over twenty ICER assessments, and holds an affiliate faculty position within the Center for Evaluation of Value and Risk in Health at Tufts University School of Medicine. Jon has graduate training degrees in pharmaceutical outcomes research (PhD) and biostatistics (MS) from the University of Washington.
Elisabeth Oehrlein, PhD, MS
Applied Patient Experience, LLC, Washington, DC, USA
Julia F. Slejko, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
Julia F. Slejko, PhD is an Associate Professor of Pharmaceutical Health Services 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-lead of ISPOR’s Women in HEOR initiative, Co-Chair Elect of the ISPOR Faculty Advisor Council and is a member of ISPOR’s Health Science Policy Council.
Do We Need Advanced Modeling Methods for Equity-Informative Economic Evaluations?
Digital Conference Pass
ISSUE: There is an increased interest in incorporating health equity considerations in the value assessment of health technologies. Distributional cost-effectiveness analysis (DCEA) is an intuitively appealing extension of conventional cost-effectiveness analysis to quantify health equity impacts and facilitate potential trade-offs between improving total health and reducing health inequalities. However, there is debate about the feasibility of implementing a DCEA, especially for newer health technologies. This session will discuss whether advanced modeling methods are beneficial or even needed for DCEA, and whether these methods can help with some of the evidence challenges.
OVERVIEW: In practice, DCEA relies on mathematical modeling that integrates different sources of evidence to estimate expected outcomes and opportunity costs for the compared interventions by subgroup defined according to individual and non-individual factors such as race/ethnicity, socioeconomic status, and geography. The need to model distributional effects has implications for the model structure and simulation approach, as well as evidence requirements. After introducing the session and an opening statement on the relevance of DCEA (Avanceña, ~11 minutes), panel members will present key features of individual-level and discrete event simulation of (theoretical) relevance for DCEA (Degeling), value of information (VOI) in the context of DCEA (Kunst), and estimation of model input parameters (Jansen) (~8 minutes each). Subsequently, the panel members and audience will have a debate lead by the moderator (25 minutes). In addition to questions from the audience, the following questions will be discussed: Do we need individual-level or discrete event simulation modeling for DCEA? Are we replacing structural uncertainty with parameter uncertainty by using more complex models, and would that provide any benefit? What are the most important model parameters, and what are the implications for data collection and parameter estimation?
Moderators
Anton Avanceña, PhD
The University of Texas at Austin, Austin, TX, USA
Panelists
Koen Degeling, PhD
Lumen Value & Access – a Healthcare Consultancy Group Company, Hengelo, OV, Netherlands
Dr Koen Degeling is Scientific Director and the Decision-Analytic Modeling Lead at Lumen Value & Access, a Healthcare Consultancy Group company. He was trained as an Industrial Engineer specializing in Healthcare Technology and Management, and holds a PhD in Health Economic Modelling from the University of Twente in the Netherlands. Prior to joining Lumen Value & Access, Koen worked on real-world data-driven health economic and health services research projects at the Cancer Health Services Research department of the University of Melbourne in Australia, where he continues to be involved as an Honorary Fellow. He is an active ISPOR member and currently serves on the Editorial Advisory Board for Values & Outcomes Spotlight and ISPOR New Professionals Steering Committee, is involved in several short courses and workshops, and has served as global chair of the ISPOR Student Network.
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.
Natalia Kunst, PhD
University of Oslo, Oslo, 03, Norway
5:45 PM - 6:15 PM
Meet ISPOR's New CEO and Engage With ISPOR Leadership
Join us for a reception at the ISPOR Booth to welcome ISPOR's new CEO, Rob Abbott, and meet members of the board of directors, as well as other ISPOR senior leaders!
5:45 PM - 6:45 PM
Welcome Reception
Join us in celebrating ISPOR 2023. We’re looking forward to bringing the HEOR community together in Boston.
Sponsored by Komodo Health
Discussion Groups
Medicare Coverage With Evidence Development for Drugs Approved for Alzheimer’s Disease: Which Study Designs and Data Sources Would Best Answer Medicare’s Questions?
Moderator
Sean Tunis, MD, MSc
Tufts University, Boston, MA, USA
Sean Tunis, MD, MSc. is a Principal with Rubix Health where he consults with public and private sector organizations on health technology issues at the interface of regulatory and reimbursement policy, market access, comparative effectiveness, outcomes measurement and health technology assessment. He is also a Senior Fellow in the Tufts Center for the Evaluation of Value and Risk in Health. Dr. Tunis also serves as a Mentor-in-Residence at Johns Hopkins Tech Ventures, helping early stage companies to develop their initial reimbursement strategy and to align their clinical development programs with payer and regulatory requirements.
External Control Arms (ECA) in Phase 3 Trials: What Is Needed Now and in the Future to Enable Acceptance With Regulatory and HTA Bodies?
Moderator
Helene Karcher, PhD
Novartis Pharma AG, Basel, Switzerland
Helene is an access leader at Novartis who has built and led teams of all sizes in HEOR and Evidence Generation in pharma and consulting. She has designed numerous integrated evidence plans for access and contributed to over 70 studies, including Phase 1-3 trials and real-world studies. She is also Editor-in-Chief of the scientific journal Epidemiologic Methods.
6:15 PM - 6:45 PM
Celebrating 25 Years of Value in Health
We are celebrating the 25th Anniversary of Value in Health! Meet the editors-in-chief, Michael Drummond and C. Daniel Mullins, and other editorial advisory board members, at this reception in the ISPOR Booth.
Tue May 9
7:00 AM - 5:00 PM
Registration Hours
Sponsored by Carelon Research
7:30 AM - 8:30 AM
Morning Coffee Service
8:30 AM - 9:45 AM
Plenary Session
Second Plenary Session
Digital Conference Pass
: 200: Second Plenary: AI Wants to Chat With You: Accept or Ignore?
8:30AM - 9:45AM
Adamson B1, Sontag D2, Higashi M3, Cecchi G4, Ritchey MB5, Shreibati J6
1Flatiron Health, New York, NY, USA, 2Massachusetts Institute of Technology (MIT), Cambridge, MA, USA, 3GeneDx, Stamford, CT, USA, 4IBM Research, New York, NY, USA, 5FDA, CDRH, Silver Spring, MD, USA, 6Google Health, Menlo Park, CA, USA
During this session, key thought leaders will share insights on what to expect as AI becomes more broadly adopted in healthcare. Discussion will focus on the potential influence of AI on health economics & outcomes research. Will AI augment, disrupt, or distract our field? (or a bit of each?)
9:30 AM - 6:45 PM
Poster and Exhibit Hall Open
9:45 AM - 10:15 AM
AM Coffee Break
Located in the Exhibit Hall
9:45 AM - 1:15 PM
Poster Session 3
Live
10:00 AM - 10:45 AM
Get to Know ISPOR's Faculty Advisors
The Membership team invites you to a meet and greet with ISPOR faculty from around the globe. Faculty Advisor Council Leaders will be eager and ready to answer your questions pertaining to academia.
10:15 AM - 11:15 AM
Breakouts: IP, WS, & OBS
Beyond Burden of Illness – Using RWE for Advanced HEOR Analytics
Digital Conference Pass
Level: Intermediate
PURPOSE: With RWE playing a more central role in the drug evaluation process, we would like to discuss the applications, advantages and challenges of using RWE for advanced HEOR analytics.
DESCRIPTION: RWE has been consistently used in drug reimbursement decisions to demonstrate treatment patterns, unmet need and burden of illness. However RWE also has tremendous analytical potential beyond descriptive statistics. Although it includes the inherent messiness of real world practice and data collection, it can also provide the advantage of larger samples sizes and longer follow-up time compared to data available from clinical trials. Dr. Basu will introduce the session with an overview of potential analytic areas where RWE might be able to contribute. Dr. Adamson will then discuss the fit-for-purpose of EHR-derived RWD with strengths and weaknesses in various advanced analytics ( specific examples will include hybrid RWE-enhanced extrapolation, synthetic control arms, etc.). She will also discuss potential analytical biases and how they can be mitigated. Dr. Manson will share the manufacturer’s perspective of how these analyses can support reimbursement submissions, including a demonstration of correlating progression and survival across multiple types of RWE databases. She will also discuss how other analytical frameworks such as estimands and indirect comparisons can be considered. Dr. Campbell will then share the health technology assessment (HTA) perspective by summarizing past case studies and lessons learned. He will share ideas on how RWE analytics can reduce uncertainty and what types of RWE are most helpful through the lifecycle of HTA. The discussants will poll the audience on their perceptions of the future direction of RWE analytics, and barriers for use.
Discussion Leaders
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 Services 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 through scientific disciplines of applied economic theory, comparative and cost-effectiveness analyses, causal inference methods, program evaluation, and outcomes research. 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.
Discussants
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 Principal Scientist 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.
Jon Campbell, PhD
Institute for Clinical and Economic Review, Hingham, MA, USA
Jon Campbell is Senior Vice President for Health Economics at the Institute for Clinical and Economic Review (ICER). Jon is a leader in value assessment methods and application. He is an author of over 250 manuscripts and abstracts in the field of value assessment, over twenty ICER assessments, and holds an affiliate faculty position within the Center for Evaluation of Value and Risk in Health at Tufts University School of Medicine. Jon has graduate training degrees in pharmaceutical outcomes research (PhD) and biostatistics (MS) from the University of Washington.
Stephanie Manson, PhD
Novartis, East Hanover, NJ, USA
Is There Room for Patient-Centered Value Assessment in Medicare Negotiation and State Prescription Drug Affordability Board Processes?
Digital Conference Pass
Level: Intermediate
PURPOSE: In 2023, the federal government and state governments will begin assessing product affordability and value to regulate drug costs. Medicare will begin negotiating drug prices, and Prescription Drug Affordability Boards (PDABs) in states such as Colorado and Maryland will begin the process of setting upper payment limits. However, these processes have not been fully defined, and it is unclear the extent to which product value and patient perspectives will be reflected. The laws and regulations establishing these programs include constraints in process (e.g., short timeframes) and methodology (e.g., QALY bans) that may prevent comprehensive, patient-centered value assessments. This session will utilize real-world examples to discuss how these processes can best reflect value from the patient perspective given the policy constraints, and the risks of getting it wrong.
DESCRIPTION: Workshop attendees will obtain a working knowledge of the opportunities, tools, and constraints CMS and state governments have in assessing value and engaging patients. Dr. McQueen will provide this overview, reflecting his experience advising the Colorado PDAB, conducting ICER assessments, and piloting Multi Criteria Decision Analysis. Dr. Mattingly will present his experience engaging patients in value assessment and his work as an advisor to the Maryland PDAB. Deanna Darlington has worked with multiple patient groups responding to ICER reviews. She will highlight her work engaging patients on the value of HIV treatment and discuss the challenges patients face when contributing to value assessments. Dr. Montgomery will discuss why these new drug price regulation programs run the risk of establishing precedents for US value assessment that do not align with best practices. Workshop attendees will be presented with scenarios for how patient-centered value assessment could be implemented in these programs, asked to weigh tradeoffs between best practices and feasibility, and vote for implementation scenarios they believe best reflect patient-centered value.
Discussion Leaders
Robert McQueen, PhD
University of Colorado, Denver, CO, USA
R. Brett McQueen is an Assistant 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
Deanna Darlington, BA, PSC
Haystack Project, Washington, DC, USA
Deanna is a Principal at Innopiphany. She is also the CEO of the Haystack Project, a non-profit dedicated to ensuring patient access to treatment options for the rare and especially ultra-rare community. She is also a member of the Innovation and Value Initiative (IVI) Patient Advisory Group and President of Links2Equity.
T. Joseph Mattingly, PharmD, PhD, MBA
University of Utah College of Pharmacy, Park 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.
Russ Montgomery, PhD
Gilead Sciences, Baltimore, MD, USA
Russ is a Director of Policy at Gilead Sciences, where he focuses on value and HTA policy in the US and globally. He previously worked as a consultant on value, access, and policy for pharmaceutical and device companies. He also previously worked as a health policy analyst and advisor at the Centers for Medicare & Medicaid Services, the Office of the Assistant Secretary for Planning and Evaluation (ASPE), the Maryland Department of Health, and the Arkansas Department of Human Services. Russ holds a PhD in health services research and policy from the Johns Hopkins Bloomberg School of Public Health. He and his husband live in Baltimore, MD with their dog Simon.
Standardized Assessment Tool Designed to Assist in Evaluation of RWE on Drug Effectiveness and Safety
Digital Conference Pass
Level: Intermediate
PURPOSE: Non-randomized RWE studies are subject to multiple sources of bias. The complexity of data, study design, and analytical methods makes evaluation of such studies challenging. Building consensus on the evaluation of quality and validity of RWE studies is critical if RWE is to play an important role in decision-making. As part of the International Society for Pharmacoepidemiology’s (ISPE) effort to improve the quality of RWE evaluation, an ISPE-led group developed an assessment tool to support HTA and payer decision-makers in evaluating RWE studies of medication safety and effectiveness. The tool was further refined in collaboration with HTA agencies. This session aims to introduce the ISPOR community to the tool and solicit feedback to ensure the tool addresses the needs of HTA agencies and the broader ISPOR community.
DESCRIPTION: The discussion leader will introduce the need for a standardized assessment tool in evaluating real-world comparative effectiveness and safety studies for bias (5 mins). Dr. Girman will introduce the assessment tool and briefly discuss study relevance and data quality domain (5 mins). Dr. Bykov will walk the audience through bias-related domains (10 mins). Dr. Franklin will present analytic methods domain (10 min). Using a published RWE study as an example, the discussants will lead the attendees through real-time RWE study evaluation using the tool (20 mins). The discussants will then take questions and feedback from the audience (10 mins). While the tool was developed with HTA agencies in mind, it can be used in any other setting where RWE evaluation is needed. This interactive workshop will be valuable to HTA representatives, payers, industry analysts preparing RWE submissions to HTAs, and any ISPOR members who need to critically evaluate RWE on drug safety and effectiveness.
Discussion Leaders
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.
Discussants
Katsiaryna Bykov, PharmD, ScD
Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA
Dr.Bykov is an Assistant Professor of Medicine at Harvard Medical School and an Associated Epidemiologist in the Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women’s Hospital. She received her Doctor of Pharmacy degree from Temple University School of Pharmacy in Philadelphia and her Doctor of Science degree in Epidemiology from Harvard T.H. Chan School of Public Health.
For the past 11 years, Dr. Bykov has been using electronic healthcare data to evaluate drug utilization, impact of healthcare interventions, and medication safety and effectiveness. She has also contributed to numerous methodological investigations on conducting research in large, population-based healthcare databases. Dr. Bykov is an Associate Editor of Pharmacoepidemiology and Drug Safety and is currently leading the development of a standardized tool for the evaluation of RWE studies on drug safety and effectiveness.
Jessica Franklin, PhD
Optum, Boston, MA, USA
Dr. Franklin is currently a Principal Consultant, Epidemiology and Real-World Evidence at Optum. She has extensive experience in designing and leading studies of the effectiveness, safety and utilization of medications from large healthcare databases, including health insurance
claims and electronic health records. She led the development and application of a wide range of novel methods in pharmacoepidemiology, such as automated variable selection and model building methods for propensity score adjustment, measures for evaluating covariate balance, and methods for incorporating free-text data into model building
through natural language processing (NLP). Dr. Franklin also co-founded the FDA and NIH-funded RCT DUPLICATE project focused on producing an empirical evidence base for the
validity of real-world evidence on medications. She has authored or co-authored more than 100 articles in peer-reviewed medical, epidemiology and biostatistics journals. Dr. Franklin received a Bachelor of Science in Mathematics from the University of Georgia and a PhD in biostatistics from Johns Hopkins Bloomberg School of Public Health.
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.
Addressing Heterogeneity in Comparative Economic Analysis in the Decentralized US Setting: Are We Doing Enough to Support Patient-Centered Medical Decision-Making?
Digital Conference Pass
Level: Intermediate
PURPOSE:
Despite evidence that treatment value often varies across patient subgroups, coverage decisions in the US are usually based on evidence aggregated at the population level. Thus, patients diverging from the average risk “being denied for being different,” as cautioned by the National Pharmaceutical Council, with suboptimal patient and economic outcomes The purpose of this workshop is to promote cross-stakeholder awareness of the implications of unaddressed heterogeneity in comparative economic analysis and discuss how best to promote evidence generation capable of supporting patient-centered decision-making.
DESCRIPTION:
The workshop will begin with an overview of heterogeneity in comparative economic analyses, including a discussion of important sources relevant to the decentralized US setting (e.g., socioeconomic, geographic, insurance, and provider variability). Methods to address heterogeneity and implementation challenges will be outlined (Willis, 10 minutes). Next, the consideration of heterogeneity in the US cost-effectiveness literature will be discussed, leveraging results from two Tufts Cost-Effectiveness Analysis (CEA) Registry samples. Good practices and opportunities for improvement will be illustrated via examples from the literature (Lavelle & Willis, 10 minutes). The next discussant will highlight underappreciated and under-studied sources of heterogeneity that may be particularly important in the decentralized US setting (Chapman, 10 minutes). The last discussant will present the payer perspective, including issues with implementation in health benefit design, such as resource constraints, institutional barriers, and equity concerns (Westrich, 10 minutes). An interactive case study that includes various sources of heterogeneity will be used to facilitate audience participation. This hands-on learning exercise will highlight the importance of considering all relevant sources of heterogeneity when tailoring analyses to decision problems. Different stakeholder viewpoints, evidence requirements, and barriers to implementation will be discussed (All, 20 minutes). This workshop will be valuable to researchers, payers, patient representatives, and manufacturers interested in ensuring that comparative economic analysis supports patient-centric decision-making.
Discussion Leaders
Michael Willis, PhD
The Swedish Institute for Health Economics, Lund, Sweden
Michael Willis is a Research Director at the Swedish Institute for Health Economics, where he has been since 1997 except for a two-year pause spent at the Global Health Economics division of Janssen Global Services, LLC. Michael holds a PhD in Economics from the University of California at Santa Barbara. Michael’s leading research interests are applied quantitative analysis and current policy issues associated with resource allocation and comparative economic analysis. Michael has particular experience in the modeling of chronic and progressive diseases such as diabetes and has been a member of the executive organizing committee for the Mount Hood Diabetes Challenges and a regular speaker for more than 10 years.
Discussants
Richard Chapman, PhD, MS
Innovation and Value Initiative, Alexandria, VA, USA
Dr. Chapman is the Chief Science Officer for the Innovation and Value Initiative (IVI), a nonprofit research organization whose mission is to advance the science, practice, and use of value assessment in healthcare to make it more meaningful to those who receive, provide, and pay for care. Prior to that, Dr. Chapman was Director of Health Economics at the Institute for Clinical and Economic Review, where he led development of economic evaluations assessing the potential cost-effectiveness and budgetary impact of clinical interventions.
Tara Lavelle, PhD
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.
Kimberly Westrich, MA
Xcenda, Herndon, VA, USA
Kimberly Westrich, MA, is a Director with Xcenda’s Value and Access Strategy team. Ms. Westrich is a subject matter expert on health technology and value assessment, value-based agreements, quality measures, accountable care organizations, high-deductible health plans, and value-based insurance design. She has presented nationally for payer, patient, employer, and pharmaceutical audiences. She has authored numerous research and policy publications appearing in Health Affairs, American Journal of Managed Care, Journal of Managed Care & Specialty Pharmacy, Value in Health, and others.
Podium Session
Dynamic Simulation Modeling Methods for Health Economics
Simulation modeling methods continue to advance as we expand computing power and the availability of Big Data. This session takes a look at the latest examples of application in dynamic simulation modeling to compliment evaluations in health economics and outcomes research. Seating is limited.
Moderator
William Padula, PhD, MSc, MS
Hasbro Children's Hospital, 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.
P25: Dynamic Intervention Optimization (DIO): A Bayesian Adaptive Framework for Multicomponent Intervention Optimization Using Expected Net Benefit of Sampling
10:15AM - 10:30AM
Vanness D1, Collins LM2, Strayhorn JC2
1Pennsylvania State University, University Park, PA, USA, 2New York University, New York, NY, USA
OBJECTIVES: Multicomponent intervention science has largely relied on ad hoc assembly of individual components into treatment packages evaluated in classical 2-arm randomized controlled trials (RCTs). We demonstrate a Dynamic Intervention Optimization (DIO) framework merging the optimization and evaluation phases of the Multiphase Optimization Strategy (MOST) (Collins et al.) to identify optimized combinations of intervention components, accounting for opportunity costs of research.
METHODS: We simulated 16 interventions comprising up to 4 hypothetical components with varying main and interaction effects on a dichotomous "success" outcome in 1,000 Monte Carlo replications. We calculated maximal potential net benefit assuming the intervention with the highest true expected net benefit was assigned to a hypothetical population at day 0. Assuming $1,000,000 fixed and $500 per-participant study costs, accrual of 40 participants/month, discounted (3%) net benefit was calculated for classical treatment package RCT comparing the full 4-component treatment package to minimal treatment followed by implementing the full package for the remaining untreated population if effective vs. minimal treatment (p < 0.01). DIO used Bayesian full-factorial trials evaluated sequentially every 80 participants, stopping accrual and implementing the multi-component intervention with the highest expected net benefit if the discounted expected value of accruing a further round of participants (balancing value of information against research opportunity costs, including delayed access to potentially effective interventions for the remaining population) was negative.
RESULTS: Mean realized net benefit per-person with DIO was $361, vs. $98 for classical treatment package RCT, achieving 67% (vs. 18%) of the maximal potential net benefit of $535. DIO outperformed classical treatment package RCT in 883/1,000 replications.
CONCLUSIONS: In a proof-of-concept simulation, DIO outperformed classical treatment package RCT in identifying net-beneficial multicomponent interventions.
P27: The Impact of Incorporating Survival Heterogeneity into Modeling of Survival Outcomes for Patients with Muscle-Invasive Bladder Cancer after Radical Resection: Analyses from a Retrospective Real-World Study in France
10:45AM - 11:00AM
Chepynoga K1, De T2, Kurt M3, Colrat F4, Branchoux S5, Patel M6, Teitsson S7
1Access Consulting, Parexel, Hackensack, NJ, USA, 2Access Consulting, Parexel, Billerica, MA, USA, 3Bristol Myers Squibb, Princeton, NJ, USA, 4Bristol Myers Squibb, Rueil Malmaison, 92, France, 5Bristol Myers Squibb, Rueil-Malmaison, France, 6Bristol Myers Squibb, Summit, NJ, USA, 7Bristol Myers Squibb, Uxbridge, UK
OBJECTIVES: To examine the impact of accounting for potential long-term survivors (LTS) on the analysis and projection of survival for patients with muscle-invasive bladder cancer at high risk of recurrence after radical resection from the COBLAnCE registry cohort in France. METHODS: Publicly available disease-free survival (DFS) and overall survival (OS) data on 256 patients were digitized. Median data follow-up was 5.03 years. Mixture cure models (MCMs), standard parametric models (SPMs), and cubic-spline models (CSMs) were fitted separately to the DFS and OS data. MCMs classified patients into 2 latent subpopulations, as LTS and non-LTS, where LTS were subject only to non-disease-related mortality estimated from general population mortality rates in France, adjusted with baseline age, sex, and smoking prevalence in the cohort. Time-to-event outcomes for non-LTS were modeled via SPMs. Statistical goodness-of-fit metrics, and visual inspection of survival distributions and hazards were used to select the base-case models and their alternatives for sensitivity analyses. Restricted mean survival times (RMSTs) were estimated and compared over 6, 10, and 20 years. RESULTS: Estimated percent of LTS was 19.9% (95% CI, 11.3%-32.9%) for DFS and 25.8% (95% CI, 16.3%-38.3%) for OS. Estimated RMST by the MCM showed only marginal differences versus those by SPM and CSM over 6 and 10 years (DFS, <1.1 months; OS, <1.4 months). Twenty-year RMST projections from the MCMs were 61 months for DFS and 71.4 months for OS. These projections exceeded those from the SPM and CSM by 10.7 and 6.7 months, respectively, for DFS, and by 8.1 and 10 months, respectively, for OS. Results were not sensitive to the selected alternative models in each family. CONCLUSIONS: A considerable proportion of the cohort can be projected as LTS. Accounting for the latent survival heterogeneity in the cohort borne by these patients via MCMs can yield higher long-term survival projections.
P28: Recalibration of Risk Prediction Equations for Cardiovascular Diseases in the Taiwanese Population with Type 2 Diabetes
10:30AM - 10:45AM
Chong KS1, Yang CT2, Wang CC3, Ou HT2, Yen TC3, Kuo S4
1Institute of Clinical Pharmacy and Pharmaceutical Sciences, College of Medicine, National Cheng Kung University, Tainan, TNN, Taiwan, 2National Cheng Kung University, Tainan, TNN, Taiwan, 3Graduate Institute of Clinical Pharmacy, College of Medicine, National Taiwan University, Taipei, Taiwan, 4Division of Metabolism, Endocrinology & Diabetes, Department of Internal Medicine, University of Michigan, Ann Arbor, MI, USA
OBJECTIVES: The Chinese Hong Kong Integrated Modeling and Evaluation (CHIME) is a validated patient-level simulation model derived from a Hong Kong population with prediabetes or type 2 diabetes (T2D) to predict health outcomes. Previous analyses showed that CHIME risk equations possessed satisfactory discrimination but poor calibration for cardiovascular diseases (CVDs) among Taiwanese T2D populations. We therefore sought to recalibrate CHIME risk equations for Taiwanese T2D populations.
METHODS: T2D patients were identified from two leading medical centers in Taiwan, National Cheng Kung University Hospital (NCKUH) and National Taiwan University Hospital (NTUH), during 2013-2019. Study individuals in each hospital were randomly split into training and testing sets to generate the recalibrated risk equations and determine their predictive performance, respectively. Recalibration was implemented by adjusting the overall baseline risk of original risk equations. Further adjustment among low-, middle-, and high-CVD risk patients was performed if the performance after the adjustment of overall baseline risk was not satisfied. Predictive performance was evaluated using calibration ideal slope and Greenwood-Nam-D'Agostino (GND) test (p>0.05) indices.
RESULTS: 32,053 Taiwanese T2D patients were included. Before recalibration, a discrepancy existed between predicted and observed risks in both training sets (slopes: 0.47 and 0.38 for myocardial infarction [MI], 0.72 and 0.80 for stroke, and 0.78 and 0.37 for heart failure [HF] in NCKUH and NTUH, respectively; GND test: p<0.001). After recalibration, the performance of risk equations significantly improved in testing sets of both NCKUH (slopes: 0.98 for MI and 0.94 for HF; GND test: p>0.22) and NTUH (1.10 for MI, 0.87 for stroke, and 1.05 for HF; GND test: p>0.07), except stroke in NCKUH (slope: 1.01; GND test: p<0.01).
CONCLUSIONS: Recalibrated CHIME risk equations are more applicable to Taiwanese T2D patients for predicting CVDs than original risk equations. Other recalibration methods are needed to further improve the stroke risk equation performance.
Health-Related Quality of Life and Health State Utility Measurement
Moderator
Julia F. Slejko, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
Julia F. Slejko, PhD is an Associate Professor of Pharmaceutical Health Services 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-lead of ISPOR’s Women in HEOR initiative, Co-Chair Elect of the ISPOR Faculty Advisor Council and is a member of ISPOR’s Health Science Policy Council.
P32: Psychometric Evaluation of the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire–Core 30-Items and Myeloma 20-Item Module in Multiple Myeloma Patients
10:30AM - 10:45AM
Romero H1, Mazerolle F2, Cherepanov D3, Alin T2, Jean-Pierre P3, Regnault A2
1Takeda Development Center Americas, Inc., Bristol, RI, USA, 2Modus Outcomes, a division of THREAD, Lyon, France, 3Takeda Development Center Americas, Inc., Cambridge, MA, USA
OBJECTIVES: To confirm the fitness-for-purpose of the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30-items (EORTC-QLQ-C30) and the Myeloma 20-item module (EORTC-QLQ-MY20) scores to demonstrate clinical benefit of Multiple Myeloma (MM) therapies.
METHODS: Psychometric and meaningful within-patient change (MWPC) analysis of EORTC-QLQ-C30 and MY20 scores were conducted using pooled, blinded data from six TOURMALINE-MM clinical trials of newly diagnosed or relapsed/refractory multiple myeloma patients (N=2,972). Analyses included classical test theory (CTT: internal consistency and test-retest reliability, construct validity and ability to detect change) and Rasch measurement theory (RMT).
RESULTS: Adequate CTT properties were demonstrated: Cronbach’s alpha reliability coefficients were >0.80; correlations between EORTC-QLQ scores with other measures (Eastern Cooperative Oncology Group—Performance Status, EQ-5D, brief pain inventory-short form worst pain item) ranged from moderate (0.40≤r<0.70) to high (r≥0.70). Effect sizes for change over 4 cycles of therapy in QLQ-C30 Physical Functioning (PF) and QLQ-MY20 Disease Symptom (DS) scores were 0.35 and -0.41 in patients categorized as improved, indicating some ability to detect improvement. RMT analysis identified a meaningful hierarchy in the items comprising QLQ-C30 PF, pain and fatigue scales and the QLQ-MY20 DS scale. Gaps were identified in coverage along the measured continuums for all scales, including imperfect coverage, floor and ceiling effects. MWPC estimates for the QLQ-MY20 DS score were -11.11 points (range: -5.56 to -16.67) for improvement and +5.56 (range: 5.56 to 11.11) for deterioration.
CONCLUSIONS: The EORTC-QLQ-C30 and EORTC-QLQ-MY20 scores showed adequate measurement properties in recent MM clinical trials based on the CTT framework. RMT analyses uncovered gaps in the coverage of the measured concepts that may limit the ability of these scales to detect subtle changes when used in clinical trials, especially among MM patients with mild severity of symptoms (e.g., pain) and impacts on quality of life.
P30: Psychometric Evaluation of Two Novel Hyperphagia Questionnaires for Patients with Bardet-Biedl Syndrome (BBS)
10:45AM - 11:00AM
Mallya U1, Yang M2, Johnston C1, Greatsinger A2, Hagopian E2, Pomeroy J3, Haqq AM4
1Rhythm Pharmaceuticals, Boston, MA, USA, 2Analysis Group, Inc., Boston, MA, USA, 3Marshfield Clinic Research Institute, Marshfield, WI, USA, 4Division of Pediatric Endocrinology, University of Alberta, Edmonton, AB, Canada
OBJECTIVES: Rare genetic disorders of obesity including Bardet-Biedl Syndrome (BBS) are characterized by early onset obesity, hyperphagia, and multi-systemic complications. Symptoms of Hyperphagia (SoH) and Impacts of Hyperphagia (IoH) are novel questionnaires designed to assess severity of hyperphagia symptoms and their impact on patients and caregivers. As part of the CARE-BBS study, we evaluated the performance of these scales.
METHODS: CARE-BBS was a multi-country cross-sectional survey study of adult caregivers of patients with BBS experiencing obesity and hyperphagia. In addition to the SOH and IOH questionnaires, other instruments included Impact of Weight on Quality of Life (IWQOL)-Kids Parent Proxy, PROMIS Scale Global Health of Caregiver, Revised Impact on Family Scale (RIOFS), and Work Productivity and Activity Impairment (WPAI): BBS-Caregiver.
RESULTS: 242 eligible caregivers from Canada, Germany, UK, and US completed the survey. Exploratory factor analysis identified 1 factor each for SoH-Proxy, IoH-Proxy, and IoH-Caregiver (weighted eigenvalues: 0.83, 2.03, 2.79, respectively). Strong correlations were observed across SoH/IoH scales (r=0.558-0.798) (all p<.001). Strong internal consistency was observed for IoH-Proxy (Cronbach’s α coefficient=0.66) and IoH-Caregiver (α=0.72) and moderate for SoH-Proxy (α=0.40). All three scales correlated moderately to strongly with school days missed and all domains of IWQOL-Kids except Physical Comfort (range=0.315-0.573, all p<.001). Both Impact scales also correlated moderately with both WPAI-Caregiver total productivity and total activity impairments (range=0.417-0.448, all p<.001). Known-groups analyses indicated significantly worse SoH-proxy scores for caregivers applying more weight management approaches (6-10 vs. 5, or >10). Worse IoH scores were found when caregivers reported greater strain on RIOFS items and worse mental health on PROMIS.
CONCLUSIONS: Caregiver versions of the SoH and IoH scales demonstrated validity, reliability, and consistency for use in a real-world setting. Additional studies to further validate psychometric properties of the scales, including the IOH and SOH patient versions across other health care settings, are recommended.
P31: The Association between Patient- and Caregiver-Reported Utility Values in Duchenne Muscular Dystrophy (DMD)
11:00AM - 11:15AM
Szabo S1, Filipovic Audhya I2, Bever A1, O’Sullivan F1, Malone DC3, Feeny D4, Neumann P5, Iannaccone ST6, Jayasinghe P1, Gooch KL2
1Broadstreet Health Economics & Outcomes Research, Vancouver, BC, Canada, 2Sarepta Therapeutics, Cambridge, MA, USA, 3The University of Utah, Salt Lake City, UT, USA, 4McMaster University, Hamilton, ON, Canada, 5Tufts University, Boston, MA, USA, 6University of Texas Southwestern Medical Center, Dallas, TX, USA
OBJECTIVES: Caregivers commonly report utility on behalf of patients with DMD. As the alignment between these perspectives has not been described, the objective was to compare patient- and caregiver-derived visual analogue scale (VAS) and utility values for DMD health states.
METHODS: Thirty-one patient-caregiver dyads were recruited through a US-based patient organization to complete the EQ-5D and Health Utilities Index (HUI). Patients were classified into one of nine DMD-related health states according to level of lower/upper limb function, daytime/nighttime ventilation, and cardiomyopathy. Median (IQR) EQ-5D, VAS and HUI2 values were stratified by health state and respondent type (patient vs caregiver). Correlations were calculated for ‘ambulatory/transitional’ dyads and ‘non-ambulatory’ dyads separately. Mixed-effects regression was used to model the relationship between caregiver and patient utility values, accounting for ambulatory status and within-dyad correlation.
RESULTS: Mean (SD) patient age was 14 (5.9) years and 21 (67%) were non-ambulatory. Correlations for ambulatory/transitional dyads were r=0.97 (EQ-5D), r=0.88 (HUI2) and r=0.90 (VAS); and for non-ambulatory dyads, r=0.66 (EQ-5D), r=0.70 (HUI2), and r=0.60 (VAS). Across health states, caregivers reported lower EQ-5D (p<0.001) and VAS (p=0.04) values than patients; HUI2 utilities did not differ by respondent type. As an example, for non-ambulatory patients with mildly-impaired upper limb function, without nighttime/daytime ventilation or symptomatic cardiomyopathy, median (IQR) estimates (n=7) were: EQ-5D: 0.30 (0.24-0.35; patient-report) vs 0.15 (0.07-0.18; caregiver-report); HUI2: 0.51 (0.44-0.52; patient-report) vs. 0.47 (0.47-0.55; caregiver-report); and VAS: 84 (74-96; patient-report) vs. 80 (75-87; caregiver-report).
CONCLUSIONS: Utility and VAS values from caregiver-patient dyads were highly correlated, particularly for ‘ambulatory/transitional’ dyads. Across health states, patient-reported values were higher than caregiver-reported values for the EQ-5D and VAS; diverging more as patient status worsened. HUI2 utilities did not differ significantly between dyads. Though numeric differences should be noted, high correlations support reliance on caregivers when patient-reported data are unavailable and can inform utility instrument selection.
P29: Evaluating the Appropriateness of the EQ-5D-5L Descriptive System and the Derived Health Utility Index Scores in Adults with Transfusion-Dependent Beta-Thalassemia: A Mixed-Methods Study
10:15AM - 10:30AM
Boateng-Kuffour A1, Drahos J1, Kohli P1, Skrobanski H2, Forster K2, Acaster S2, Pakbaz Z3, Li N1, Williams K2
1Vertex Pharmaceuticals Incorporated, Boston, MA, USA, 2Acaster Lloyd Consulting, London, UK, 3University of California Irvine School of Medicine, Orange, CA, USA
OBJECTIVES: Transfusion-dependent β-thalassemia (TDT) is a severe form of β-thalassemia requiring lifelong red blood cell transfusions (RBCTs) and iron-chelation therapy. EQ-5D is commonly used to derive health utility index scores for use in cost-effectiveness analyses. However, despite the range of symptoms and functional impacts of TDT, prior studies show that EQ-5D health utility scores in TDT are often comparable to general population scores and may not adequately represent the impact of TDT. To our knowledge, this is the first study examining the appropriateness of EQ-5D-5L descriptive system (DS) for use in adults with TDT.
METHODS: Thirty adults with TDT from the US (n=14), UK (n=12), and France (n=4), who received ≥8 RBCTs/year in the past 2 years, completed EQ-5D-5L and qualitative interview. An interview guide was used to elicit participants’ experience with TDT (including EQ-5D-5L DS-dimensions: mobility, self-care, usual activities, pain/discomfort, anxiety/depression) and perceptions of the relevance of EQ-5D-5L DS-dimensions to TDT. Participants’ perceptions were analyzed using content analysis. Participants’ qualitative descriptions were compared with each EQ-5D-5L DS-dimensions score to identify any discordance.
RESULTS: Participants had a mean age of 28 years (standard deviation [SD]=5.1), most were female (73%), and received a mean of 18.4 (SD=7.7) RBCTs annually. EQ-5D-5L did not capture 8 of 16 symptoms/functional impacts elicited across all interviews (n=30), and 43% of participants (n=12/28) said it did not capture their TDT experience/expressed a neutral opinion. Reasons included that EQ-5D-5L missed important symptoms and the recall period limited their ability to appropriately capture their fluctuations in health impacts. Some discordance between participants’ qualitative descriptions and their EQ-5D-5L DS-dimension scores was observed, with qualitative descriptions indicating higher severity than dimension scores.
CONCLUSIONS: Findings suggest that EQ-5D-5L descriptive system lacks content validity and the derived health utility index score may not fully represent the burden of disease in TDT.
Breakouts: IP, WS, & OBS
ISPOR Latin America Health Policy Update
Digital Conference Pass
Level: Intermediate
Join the ISPOR Latin America Consortium for an informative session that will dive into the current state of the Latin America region. Our expert panelists will share their unique perspectives and insights on a range of topics, including innovative technologies and the policies that governments in the region can use to incorporate them into their health systems. We will also explore financing options and opportunities for health systems to navigate difficult budgetary scenarios in a post-pandemic context. Throughout the session, we will examine the roadmap that policymakers should follow to prioritize these options, and we will highlight emerging trends in the Latin America region that maximize value for money in health spending. This engaging session will conclude with a Q&A segment where attendees can ask questions and receive valuable insights from our esteemed panelists. Don't miss this opportunity to gain knowledge and insights into the healthcare landscape of the Latin America region.
Moderators
Cesar Alberto Cruz Santiago, MSc, PhD, MD
ISPOR, Mexico, Mexico
UNAM Surgeon, specialist in General Surgery by the 21st Century National Medical Center IMSS, Specialist in colon and rectal Surgery from the Adolfo López Mateos Hospital of ISSSTE, Master of Science UNAM. Doctor of Science from the University of Miami and Doctor of Hospital Administration
He has held various positions in the administration of health services, such as National Coordinator of Health Research at IMSS, Head of Specialized Medicine Services and Head of Preventive Programs Services at ISSSTE, Head of the Health Technology Assessment Unit and Pharmacoeconomics in the Sub-Directorate of Health Services of PEMEX. He currently holds the position of General Director of Scientific and Technical Assessment of the General Health Council.
Author of more than 57 articles published in various national and international journals, 6 book chapters and advisor for 21 postgraduate thesis.
Speakers
Manuel Antonio Espinoza, MD MSc PhD
Pontificia Universidad Católica de Chile, Santiago, Chile
Manuel Espinoza is Associate Professor in the Department of Public Health and Chief of the Health Technology Assessment Unit, both at Pontificia Universidad Católica de Chile. He is Editor in Chief of Value in Health Regional Issues, Chair of the HTAi Latinamerica Policy Forum and consultant retainer for the Interamerican Bank of Development. Manuel holds a medical doctor degree and Master in Epidemiology both from Pontificia Universidad Católica de Chile; a Master in Biostatistics from Universidad de Chile, and Master and PhD in Health Economics, both from University of York in the UK.
Javier Guzman, MD, MSc, MBA
Center for Global Development, Washington DC, DC, USA
Enabling Patient Access to Digital Therapeutics – The Optimal Approach?
Digital Conference Pass
ISSUE:
Current payer coverage for the available digital therapeutics has been significantly different across markets; some EU markets have developed separate evaluation and reimbursement programs, while US payers have relied on employer preference. Not only is the framework for coverage uncertain, but the value that digital therapeutics would have on healthcare resource utilization and long-term patient outcomes also remains uncertain. These challenges have led to limited covered patient access to digital therapeutics, and substantial concerns from digital therapeutics manufacturers on the financial burden borne by patients.
OVERVIEW:
The panel will bring together views from different stakeholders directly involved in Digital Therapeutics, researchers, manufacturers, and payers. Key questions are:
- What is the responsibility of an insurer vs. the manufacturer vs. the patient?
- Should all digital therapeutics be covered?
- How should insurers assess the value attribution of a digital therapeutic?
- What evidence is required to make an informed coverage decision?
The roles of the different stakeholders would entail: Dr. Marsch will share her experience conducting clinical trials with digital health technologies and will emphasize the need for a balance between the different players in the field, including the importance of patient perspective and their buy-in to the success of digital therapeutics. Mr. Parcher will present details around the development and testing processes for digital therapeutics from a manufacturer perspective, justifying the position that moving forward systems and payers should reward innovation and lower financial risk for the manufacturer. Dr. Velez will represent the payer perspective, highlighting the current and future evidence development and reimbursement models for digital therapeutics in the US market, and where the market will head as digital therapeutics become available for larger indications. Each expert will have 7.5 minutes for their presentation. After the expert’s opinions have been presented, the audience will have 10 minutes for a Q&A session.
Moderators
Monica Martin De Bustamante, AB, BE
Trinity Life Sciences, Waltham , MA, USA
Panelists
Lisa Marsch, PhD
Dartmouth College, Lebanon, NH, USA
Benjamin Parcher, PharmD, MS
Boehringer Ingelheim Pharmaceuticals, Odessa, FL, USA
Benjamin Parcher, PharmD, MS is the Global Market 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.
Fulton Velez, MD, MBA
Payer and Evidence Development Perspective, Boston, MA, USA
Dr. Velez is an experienced professional in the areas of Medical Affairs, HEOR, Market Access and RWE. He most recently worked as the Head of HEOR at Pear Therapeutics, leading their evidence development programs, which demonstrated the value of digital therapeutics in patient-relevant outcomes and healthcare resource utilization.
A Comparison of HEOR Research and Actuarial Analytics
Digital Conference Pass
ISSUE: HEOR informs the value (i.e., the benefit gained for the cost) of alternative health care treatment options. Actuaries address future financial risks and opportunities in the context of affordability (i.e., what it will cost).Together, it may be possible that the disciplines can play a highly complementary role in influencing pharmacy and medical drug coverage. Given distinct differences in professional culture, training, methods, and value drivers, the question is whether the two disciplines can indeed find opportunities to integrate, or at least produce aligned insights
OVERVIEW: Health plans, PBMs, and providers ultimately translate value-based information into Per-Member-Per-Month (PMPM) costs for the development of pharmacy and medical benefits. This practical business challenge should create a natural interface between HEOR and actuarial insights. Both disciplines answer important questions using different methodologies and perspectives. Without mutual understanding, these differences can be divisive. Without a deliberately developed bridge, benefits decision-makers may lack guidance in applying the respective insights. The three panel members have unique experience in developing and/or applying both pharmacoeconomic and actuarial insights. They will describe and compare how insights from both disciplines are used to impact benefit decisions, identify differences as well as common ground in methodology, and suggest opportunities for future and deliberate integration.
Moderators
Karl Gregor, PharmD, MS
Optum, Tucson, AZ, USA
Karl is VP of Pharmacy Advisory Services, which provides actuarially focused pharmacy training, analytics, and strategic consulting. Karl has 30 years of US and international pharmaceutical industry, consulting, and PBM experience in roles requiring the ability to deliver across different disciplines, geographies, management levels, and customer segments. Prior to his 15 years at Optum, Karl worked at Eli Lilly and Company for nearly 16 years in US, European, and Global HEOR research and leadership positions. He has BS, PharmD, and Master degrees (the latter in Pharmaceutical Economics, Policy, and Outcomes). He also completed a 2-year Health Economics Fellowship.
Panelists
David Myers, FSA, MAAA
UnitedHealthcare, Minnetonka, MN, USA
Kyle Null, PharmD, PhD
Takeda Pharmaceuticals U.S.A., Inc., Meridian, MS, USA
Kyle Null is a Senior Director of Outcomes Research & Data Science (ORDS) at Takeda Pharmaceuticals. He created the ORDS group to develop and implement value-based agreements – using a mix of actuarial and outcomes research approaches - for Takeda’s US business unit. He serves as a content expert for Takeda’s VBA Center of Excellence and has conducted workshops internationally to teach others at Takeda how to develop and implement VBAs.
Kyle has over 15 years of experience conducting health services and outcomes research primarily focused on administrative claims data. He has a Doctor of Pharmacy (PharmD) and a Doctor of Philosophy (PhD) in Pharmaceutical Sciences degrees, both from The University of Mississippi.
Case Studies
Oncology and Rare Disease Case Studies
Ranging from claims data analysis for patient pathways and economic evaluation to pricing and reimbursement issues in Latin American countries, the case studies in this session present HEOR efforts involving oncology and rare disease products.
Seating is limited.
Moderator
Sang Kyu Cho, PharmD, MPH, MS, PhD
Analysis Group, Inc., Boston, MA, USA
I am an assistant professor at the University of Houston College of Pharmacy. My training includes PhD in Health Economics and MS in Regulatory Management from the University of Southern California, MPH in Health Policy from Columbia University, and PharmD from St. John's University. I am a licensed pharmacist in New York, New Jersey, and California. I completed a two-year HEOR fellowship in oncology and hematology at AbbVie.
CS7: Using Real-World Evidence for Health Technology Assessment: Capacity Building of Parameter Calibration for Economic Evaluation of Trastuzumab Addition to Chemotherapy in Early Breast Cancer
10:45AM - 11:00AM
Nugraha R1, Suharlim C2, Tsalatsita RM3, Listiani P4, Gurning E5, Kristin E6, At-Thobari J6
1Management Science for Health (MSH), Meford, MA, USA, 2Management Sciences for Health, Medford, MA, USA, 3Center for Health Financing and Insurance Management Policy, Faculty of Medicine, Nursing, and Public Health, University of Gadjah Mada, Yogyakarta, Indonesia, 4Center for Health Financing Insurance Management Policy, Faculty of Medicine University of Gadjah Mada, Yogyakarta, Indonesia, 5Ministry of Health, Government of Indonesia, Jakarta, Indonesia, 6Faculty of Medicine, Public Health and Nursing, Universitas Gadjah Mada, Yogyakarta, Indonesia
Problem Statement: Trastuzumab has been classified as cost-effectiveness treatment for breast cancer in all stages. Indonesia through its National Health Technological Agency has concluded that in the country, Trastuzumab is denoted not cost effective for metastatic breast cancer. However, currently there is no evidence of cost-effectiveness of Trastuzumab for early breast cancer.
Description: Using the Indonesia Cancer Registries “SRIKANDI,” we generate survival parameters derived from local data of 4 cancer hospitals, namely National Cancer Dharmais, Sardjito Hospital, Surabaya Oncology Hospital, as well as Cipto Mangunkusumo Central hospital. From the database, 743 of complete early breast cancer patients’ data were collected. Kaplan-Meier survival table and curve was then generated both in general and sub-population of early breast cancers (e.g. based on cinical, treatment, and socioeconomic variables). Transitional probability parameters were gathered from systematic reviews of established studies. Using both hand calibration and Nelder Mead, all parameters were calibrated, including survival parameters (e.g., overall survival) as well as transitional probability parameters.
Lessons Learned: Capacity building for calibration method was generated by technical consultants to the Ministry of Health and researchers from local university. From the activity, we conclude that reciprocal resource- as well as data-sharing is pivotal in ensuring the successful capacity building particularly for a highly technical work. Moreover, mitigation of certain limitations such as data completion as well as unfinished parallel work need to be recognized early so that parameters driven from the capacity building can be updated.
Stakeholder perspective: This lesson learnt is described from the perspective of technical consultants working for the capacity building. The recipient of capacity building includes the team of Ministry of Health (MoH) and researcher network from local universities (or ‘research agents’).
CS5: Making More for Patients with the Same Money: Validity of a National Price Ceiling for Cancer Drugs in the Brazilian Public Health System
10:15AM - 10:30AM
Tannus G1, Etto H2, Da Silva F3, Fonseca M4
1Axia.Bio Life Sciences International, Miami, FL, USA, 2AxiaBio, São paulo, Brazil, 3Procuremed, São Paulo, SP, Brazil, 4Federal University of São Paulo, São Paulo, SP, Brazil
Problem Statement: Oncological drugs are standardized, purchased, and prescribed independently by oncology hospitals within SUS (public health system), with no intervention from individual state health secretariats or the Ministry of Health, so there is no national protocol or centralized process for negotiating acquisition prices.
Description: An economic exercise was carried out to evaluate the potential of developing a unified purchasing process, which would not necessarily require the centralization of logistics, but rather the establishment of a national ceiling for cancer drugs, where SUS units would pay only up to this limit. The Procuremed database was used to research registered public purchases of all cancer drugs in Brazil over the last six years, evaluating the quantity purchased and the amounts paid.
Lessons Learned: The results demonstrated that if each drug had been purchased at the lowest price paid in Brazil's public health system, it could have generated savings of R$1.124.877.194,30 (approximately US$216 million), a 20% reduction in spending on cancer drugs over the last six years, which could have been used, for example, to buy 2.400 mammography machines in SUS units. The exercise also revealed that the scale gain induces a price reduction of 8.5% on average, for each decade of the quantity purchased.
Another relevant finding was that the difference in the purchase price did not come only from the high-cost drugs, which varied on average 93,9%, but also from the lower-cost drugs, which had an average variation of 66,8% in the price paid by SUS unit.
Stakeholder perspective: The validity of the establishment a national price ceiling for cancer drugs in SUS, which would be a quick step in obtaining meaningful results for the financing of cancer treatment. This process combined with administrative changes and the establishment of national protocols might make a huge difference in increasing speed of access for cancer patients in Brazil
CS8: Partnering with Patient Organizations to Accelerate Research: Applying Insurance Claim Analysis to a Cohort of Rare Disease Patients from a Rare Disease Registry (PRIMARY CILIARY DYSKINESIA)
11:00AM - 11:15AM
Kauffman C1, Robicheau S2, Burns G3, Bjornson-Pennell H3, Manion M4, Worrall S5
1PCD Foundation, NIWOT, CO, USA, 2Datavant, Winchester, MA, USA, 3Chan Zuckerberg Initiative, Redwood City, CA, USA, 4PCD Foundation, San Francisco, CA, USA, 5Komodo Health, San Francisco, CA, USA
Problem Statement: Primary ciliary dyskinesia (PCD) is a rare, genetic disorder affecting the structure/function of motile cilia, with primary involvement of the upper and lower airways and increased incidence of laterality defects. PCD incidence is estimated to be ~1:10,000 and it is significantly under diagnosed.
There is currently no unique ICD10 code for PCD. This limits efforts to identify and guide affected individuals to appropriate treatment before lung damage occurs, and it hinders initiatives to better understand demographics and outcomes.
Description: This project explores the possibility of utilizing claims data to characterize the diagnostic odyssey in rare disease. The vision is a framework to better characterize their disease and the diagnostic process, providing opportunities for earlier diagnosis and improved care.
After IRB approval (Genetic Alliance), a large claims dataset with 6M+ patients was linked to the 152 de-identified PCD patients from the PCD Foundation’s patient registry in a secure environment.
Lessons Learned: Preliminary findings show it is possible to characterize patient cohorts quantitatively based on aspects of their clinical encounters from claims data. This emphasizes the value of incorporating rare disease patient registry records as ‘gold-standard’ annotated data within a much larger general dataset. This provides a powerful vehicle for exploring the utility of claims data for studying the diagnostic and care journey of rare disease patients.
Stakeholder perspective:
Patient: The registry is the core of the PCDF discovery (data) ecosystem. By investing in clinical grade data, we can efficiently link to claims data years ahead of what we thought possible.
Data scientist: Deidentified data linkage to disease registry members makes longitudinal claims and rich metadata available at large scale.
Industry: Privacy-preserving record linkages allowed rapid data collaboration–less than one month from ideation to data access–given the low risk of using de-identified data.
Member Group Meeting
Health Equity Research Special Interest Group
11:15 AM - 1:15 PM
Lunch
Bring your Tuesday lunch voucher to any catering station located in the exhibit hall for hot/cold lunch options. Vouchers will be provided to attendees upon receiving their badge at registration.
11:30 AM - 12:00 PM
Member Group Meeting
Meeting for Latin America Consortium Meet Up Meeting
11:30 AM - 12:15 PM
Poster Tours
Student Research Spotlight Poster Tour
Posters featured in this tour: EE235: Recombinant Mycobacterium Tuberculosis Fusion Protein for Diagnosis of Mycobacterium Tuberculosis Infection: A Short-Term Economic Evaluation EE297: Impact of Breathlessness Severity on Quality of Life, Healthcare Use, Productivity Loss and Economic Cost to the Australian Society EE304: Induction Cost per Responder of Biologic Therapies for Plaque Psoriasis in Canada EE428: Economic Value of a Novel Macrophage-Regulating Treatment for Diabetic Foot Ulcers from a Healthcare Sector Perspective EE253: Cost-Effectiveness of Improving Physical Activity in Older Adults with Mild Cognitive Impairment in the United States HPR89: Trends in Remote Patient Monitoring Among Medicare Beneficiaries during COVID-19: A Comparative Interrupted Time Series Analysis
Oncology Poster Tour
Posters featured in this tour: EE261: Cost-Effectiveness of Adding Darolutamide to Docetaxel and Androgen Deprivation Therapy in the Treatment of Metastatic Hormone-Sensitive Prostrate Cancer EE262: Use of Single-Arm Trials in Cadth Reviews of Oncology Drugs RWD87: Real-World Data Quality Assessment for Outcomes Research in Early-Stage Cancers EE263: First-Year Costs of Care Associated with Non-Small Cell Lung Cancer (NSCLC) Among Medicare Beneficiaries MSR53: Bayesian Parametric Mixture Survival Models in Immuno-Oncology Applications: Leveraging Control Arm Observations to Model Heterogeneous Response in the Experimental Arm EPH129: Comorbidity Patterns Among Older Women with Breast Cancer: A 10-Year Cohort Study Using an Unsupervised Machine Learning Approach
11:45 AM - 12:15 PM
Fast Facts
Building Your Social Media Presence
Level: Introductory
In this session, we will have an interactive discussion on:
- The value of social media presence on the professional level
- Understanding and engaging your network
- Keeping vs blurring the boundaries
Seating is limited.
Discussion Leader
Dalia Dawoud, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Dalia Dawoud, PhD, is Associate Director at the National Institute for Health and Care Excellence (NICE). She holds MSc in Economic Evaluation in Health Care from City University London and PhD in pharmaceutical policy and economics from King’s College London.
She has long experience in using economic evaluation in clinical guidelines development and health technology assessment (HTA), gained through working on NICE Clinical Guidelines as well as technology appraisals. Dalia’s research interests are focused on the advanced methods of evidence synthesis and use in economic models and the use of real-world evidence to inform drug development and health care decision making. Dalia currently has overall responsibility of overseeing the delivery of NICE allocated tasks on a portfolio of IMI and Horizon 2020 funded research projects including EHDEN and HTx. She is also responsible for the operational delivery of NICE HTA Innovation Lab (HTA Lab) activities. She is widely published in the field of pharmaceutical policy and pharmacoeconomics. She also serves as Associate Editor for ISPOR journal Value in Health and as Associate Editor for Pharmacoeconomics and Outcomes Research for Elsevier’s journal Research in Social and Administrative Pharmacy. Dalia also holds adjunct position as Associate Professor at the Faculty of Pharmacy, Cairo University.
Exhibit Hall Theater
The Day After Tomorrow: Evidence, Value, and Market Access After the IRA
Digital Conference Pass
The Center for Medicaid and Medicare Services (CMS) is moving forward with implementing the Inflation Reduction Act (IRA), with the first round of negotiation slated to begin this September. IRA negotiation is a significant change from the current world. Adapting to this changing ecosystem is made more challenging by the presence of many unknowns.
During this session, panelists will elucidate the latest thinking surrounding these questions. Panelists will leverage their public and private payer, HTA, evidence, and policy experience to debate and elucidate the following key issues:
- Will CMS set the negotiated price at the max statutory amount in the first few years, or will it base the pricing on a review of the evidence?
- Is the negotiation model likely to be closer to a clinical-type review or a more formal HTA?
- What is the potential role of intellectual property and research and development costs factors?
- What will be the role of subpopulations and health disparities in determining a price?
- How will CMS balance health system sustainability with supporting meaningful innovation?
- What are the downstream negotiation impacts on plans?
Seating is limited.
Sponsor: Avalere
Sponsor
Avalere
Moderators
Sarah Alwardt, PhD
Avalere Health, a member of Fishawack Health, Celebration, FL, USA
Sarah Alwardt leads Evidence & Strategy practice. A highly accomplished leader with multi-national experience in strategy, research, operations, and technology innovation, Sarah delivers data-driven insights and real-world evidence to stakeholders across the healthcare ecosystem.
Prior to joining Avalere, Sarah grew and launched Ontada, McKesson’s oncology research business providing innovative oncology evidence development strategies. She sponsored the implementation of big data technologies for natural-language processing, data integration, and visualization and lead the scientific team to produce more than 200 publications and multiple regulatory filings. She enjoyed 10 years at Humana where she held leadership roles in research, program, and process development. Sarah spent 5 years at Givaudan and 4 years at Brown-Forman. She also served 2 years as adjunct professor of chemical engineering at the University of Louisville.
Sarah holds a BS, MEng, and PhD in chemical engineering and a certificate in environmental engineering, all from the University of Louisville’s Speed School of Engineering. She is a member of the International Society for Pharmacoeconomics and Outcomes Research and the American Institute of Chemical Engineers. She is an accomplished author and speaker, having published, patented, and presented works regarding the generation and use of real-world evidence, biofuels, flavors, and fragrances.
Speakers
Jordan Banks, PhD
Avalere Health, a member of Fishawack Health, Washington, DC, USA
Jordan Banks advises clients on a range of issues, including payer coverage, pricing, the evolving landscape of innovative payment models, implications of IRA, and payer value frameworks.
Jordan was previously a director of Reporting and Analytics at Anthem, managing analytics for a large national commercial employer, leveraging claims, enrollment, and other program and individual data. She also created product evaluations for national and regional commercial employer clients across Anthem. Prior to that, Jordan spent time as a consultant in the market access space for specialty therapeutics for many conditions, including diseases affecting the immune system (e.g., rheumatoid arthritis, psoriasis, psoriatic arthritis, Crohn’s disease), multiple sclerosis, cancers, hemophilia, and other orphan diseases.
Jordan has a PhD in health services research with a focus in economics from the University of Washington. Her dissertation proposed and evaluated a potential innovative payment solution for products that are initially costly but have significant lifetime benefit for the member and payer (e.g., cell and gene therapies). She also has a Master of Public Affairs concentrating in public policy analysis and a BS in business from Indiana University.
Kim Caldwell, RPh
Texas Star Healthcare Consulting, LLC, McKinney, TX, USA
Kim A. Caldwell, RPh
3329 Drip Rock Drive, McKinney, TX 75070 | texascaldwell@yahoo.com
Skills, Experiences, and Abilities -
Pharmacy – Almost 50 years practicing pharmacy in multiple practice settings; previous owner operator of an independent pharmacy; chain pharmacy pharmacist/manager/operations; long-term care consultant; health system pharmacist; geriatric care pharmacy services; managed care pharmacy
Policy, Legislative, Regulatory – CMS Division Director helped lead creation of original Medicare Part D program rules; Texas State Board of Pharmacy member over 12 years of service including time as Board President; hundreds of face-to-face meetings with members and staff at White House, US Senate, US House, HHS, OIG, CMS, CMMI, CCIIO, FDA, FTC, CDC, and numerous state officials elected and staff; invited witness/presenter for Minority Congressional Oversight Committee Forum - PBMs
Population management, Population health –
Created and operated dedicated research business; Active participant in forward-thinking NEWDIGS’ FoCUS and LEAP projects (MIT and Tufts Medical Center); previous Brooking Institute and Duke-Margolis participant; previous guest speaker for National Health Policy Forum; regular speaker for national health conferences such as: Paying for Cures Conference – Washington, DC; National Health Council Leadership Conference – Fort Lauderdale, FL; various AMCP Spring and Fall Conferences; in-person commenter for FDA/FTC biosimilars hearing;
Leadership –
Proven leader of people, programs, processes, strategy, and outcomes; repeatedly honored by others for leadership skills within various setting; recognized mentor of talent both up and down the organization chart; frequently recruited to guide organizational strategic thinking and actions
Awards -
NEXT GENERATION LIFETIME LEADERSHIP OF THE YEAR AWARD - 2018
· Parata / Pharmacy Times
STEPHEN G. AVEY AWARD - 2018
· Academy of Managed Care Pharmacy Foundation
NABP HONORARY PRESIDENT - 2011
· National Association of Boards of Pharmacy
DISTINGUISHED SERVICE AWARD - 2010
· Academy of Managed Care Pharmacy
Post-Retirement -
PRINCIPAL | TEXAS STAR HEALTHCARE CONSULTING, LLC | APRIL 2018-PRESENT
Primary areas of service focus on Medicare Part D; Pharmacy policy including operations, networks, product coverage, contracting, reimbursement, and affordability; PBMs; US Healthcare pharmacy and pharmaceutical benefits and coverage. Recent clients include drug developers, payers, PBMs, home infusion pharmacies, trade and professional organizations, investor groups, education providers, law firms, think tanks, technical expert panels, and other consulting firms.
Michael Ciarametaro, MBA
Avalere Health, a member of Fishawack Health, Washington, DC, USA
Michael Ciarametaro advises life sciences clients on developing value and evidence strategies, drug pricing, payment reform, and the Inflation Reduction Act.
In recent projects, Mike has helped life sciences companies strategize and prepare for Medicare drug pricing negotiations under the IRA by evaluating value propositions for therapies, advising on improving those value propositions, identifying key opportunities to engage policymakers, and delivering strategic recommendations to clients.
11:45 AM - 12:45 PM
Discussion Groups
Is There Room for Patient-Centered Value Assessment in Medicare Negotiation and State Prescription Drug Affordability Board Processes?
Moderator
Robert McQueen, PhD
University of Colorado, Denver, CO, USA
R. Brett McQueen is an Assistant 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.
A Comparison of HEOR Research and Actuarial Analytics
Moderator
Karl Gregor, PharmD, MS
Optum, Tucson, AZ, USA
Karl is VP of Pharmacy Advisory Services, which provides actuarially focused pharmacy training, analytics, and strategic consulting. Karl has 30 years of US and international pharmaceutical industry, consulting, and PBM experience in roles requiring the ability to deliver across different disciplines, geographies, management levels, and customer segments. Prior to his 15 years at Optum, Karl worked at Eli Lilly and Company for nearly 16 years in US, European, and Global HEOR research and leadership positions. He has BS, PharmD, and Master degrees (the latter in Pharmaceutical Economics, Policy, and Outcomes). He also completed a 2-year Health Economics Fellowship.
Educational Symposia
Assessing the Impact of COVID-19 and the Effects of COVID-19 Vaccination on Patient-Reported Outcomes: Findings From a Nationwide Retail Pharmacy Test Database
Digital Conference Pass
Pfizer Inc engaged CVS Health Clinical Trial Services to conduct a prospective nationwide non-interventional study
1 examining symptoms, health-related quality of life and work productivity and activity impairment in symptomatic patients who tested positive for COVID-19 via reverse transcription-polymerase chain reaction (RT-PCR) at CVS Health locations in the US. Most CVS Health COVID-19 test results are reported within three days of a positive test
2, allowing for quick patient outreach and reducing potential recall bias. Subjects were enrolled in this study within three days of their positive test result, if reporting ≥1 symptom.. They were then followed over time to assess quality of life at regular intervals via validated patient-reported outcome measures (WPAI-GH, EQ-5D-5L) along with self-reported continued symptoms. Leveraging the reach of CVS Health and a streamlined e-consent process enabled the study to enroll quickly, with 537 subjects enrolled within one day of recruitment, and 77% of subjects remaining engaged at one month of follow-up.
One of the first studies of its kind, our study found that at one month of follow-up, statistically significant and clinically meaningful differences in outcomes were observed compared with baseline between vaccinated and unvaccinated groups. The EQ-5D Visual Analogue Scale (VAS) and Utility Index (UI) scores dropped in both cohorts and did not return to pre-COVID-19 levels by one month of follow-up. Individuals vaccinated with BNT162b2 reported lower prevalence and fewer symptoms both at the time of their positive test and at one month of follow-up in comparison with unvaccinated individuals. Overall, our study, published in the Journal of Patient-Reported Outcomes found that COVID-19 negatively impacted health-related quality of life (HRQoL) and work productivity among mildly symptomatic outpatient subjects3. Individuals vaccinated with BNT162b2 were less impacted by COVID-19 and recovered faster than unvaccinated individuals.
Sponsor: CVS Clinical Trial Services
Sponsor
CVS Clinical Trial Services
Moderators
Aaron Kamauu, MD, MS, MPH
Ikaika Health LLC, Bountiful, UT, USA
Dr. Aaron Kamauu MD, MS, MPH is an industry-leading advisor in the RWD space, specifically focusing on leveraging RWD to support clinical research, informatics, clinical trials, and drug development activities. Dr. Kamauu led the workstream responsible for leveraging different RWD sources into a single patient population from which a comparator cohort was created for a phase 2 pivotal study (a.k.a. external control arm); resulting in FDA drug approval. Dr. Kamauu served as VP of RWD Science at Parexel International, President & CEO of Anolinx LLC, and Head of Healthcare Data Strategy at Roche Pharmaceuticals & Genentech Inc.
Speakers
Alexandra Berk, PhD
CVS Health Clinical Trial Services, West Hartford, CT, USA
Alexandra “Alexa” Berk, PhD, is the chief scientific officer for real-world evidence (RWE) at CVS Health Clinical Trial Services. Alexa has 20 years of experience in health research, outcomes research design and large-scale global clinical trials. Alexa has co-authored several research articles including publications in the Journals of Clinical Oncology, Clinical Radiology, and Cancer Education. She is a frequent panelist discussing high-quality, patient-centric and equitable RWE, and has presented her research at conferences for the American Psychological Association, Society of Behavioral Medicine, International Society for Pharmacoeconomics and Outcomes Research, Miami Breast Cancer Conference and American Psychosocial Oncology Society. Alexa has taught and guest lectured about health disparities, patient-reported outcomes and research ethics in vulnerable populations at the New School for Social Research and Columbia University School of Social Work.
Joseph Cappelleri, MS, MPH, PhD
Pfizer Inc, Groton, CT, USA
Joseph C. Cappelleri earned his MS in statistics from the City University of New York (Baruch College), PhD in psychometrics from Cornell University, and MPH in epidemiology from Harvard University. Dr. Cappelleri is an executive director of biostatistics at Pfizer Inc. He is an elected Fellow of the American Statistical Association (ASA), elected recipient of the Long-Term Excellence Award from the Health Policy Statistics Section of the ASA, and elected recipient of the ISPOR Avedis Donabedian Outcomes Research Lifetime Achievement Award.
Manuela Di Fusco, MSc
Pfizer Inc., New York, NY, USA
Manuela Di Fusco is an Economist by training and a current PHD student in Health Economics and Policy at the Faculty of Health and Medicine of the University of Lancaster (UK). She holds a Master of Science in International Health Technology Assessment from the University of Sheffield (UK). She joined Pfizer in 2013, working in global health economics and outcomes research (HEOR) and is currently Team Lead for the mRNA Vaccines HEOR department. Manuela is passionate about working on HTA economic evaluation and RWE applied to HTA.
Xiaowu Sun, PhD
CVS Health Clinical Trial Services, Woonsocket, RI, USA
Explaining the Inflation Reduction Act
Digital Conference Pass
The Inflation Reduction Act (IRA) was signed into law in the United States in August 2022. A key aim is to lower spending on prescription drugs in Medicare. The Act is of profound relevance to many stakeholders across health care and health policy, each with their own perspectives and predictions on the potential implications of the IRA.
The IRA is complex and multi-faceted. The objective of this Educational Symposium is to provide an accessible explanation of the technical detail of the IRA. We aim to promote a common factual understanding the IRA’s provisions and to equip attendees with the information needed to support informed conversation and debate on its potential impacts. The session will be moderated by Graham Cookson, who will provide a high-level overview. Amanda Cole will then walk through the three key provisions in the IRA relevant to health policy stakeholders: the Maximum Fair Price program, inflation penalty provisions, and Medicare Part D redesign including out-of-pocket limits. Kirsten Axelsen will provide a contextual overview of the IRA, including how we arrived at the legislation. Graham will then facilitate an open discussion with the audience, and our expert speakers will aim to identify uncertainties and address common misunderstandings with the details of the Act.
Sponsor: Office of Health Economics
Sponsor
Office of Health Economics
Moderators
Graham Cookson, PhD
Office of Health Economics, London, LON, United Kingdom
Professor Graham Cookson is Chief Executive of the Office of Health Economics, the world's oldest and most prestigious health economics research organisation. He is an Honorary Professor of City University, London and a Fellow of the Royal Statistical Society. An econometrician and economist by training, Prof Cookson is an expert in the economics and policy of the life sciences sector in particular in relation to innovation. He is considered a global expert on healthcare system efficiency and productivity. Before joining OHE, Professor Cookson had a distinguished academic career including as Head of the Department of Healthcare Management & Policy at the University of Surrey (UK).
Speakers
Kirsten Axelsen, MS
DLA Piper, New York, NY, USA
Kirsten Axelsen focuses on biopharma reimbursement and health policy and has worked with biopharmaceutical and related companies for over 20 years on business strategy and public affairs in the US, EU, China and in Emerging Markets.
Kirsten is an economist who works with healthcare clients on issues including Medicare reimbursement, payer contract negotiations, designing innovative reimbursement contracts. Kirsten works with biopharma, trade associations and health technology companies on pricing, reimbursement, patient assistance, public affairs, industrial organization, and strategic matters. She has worked to developed policy platforms for CEO's and their organizations, including public positions on drug pricing, insurance coverage and regulatory reform. She has helped to establish pricing for medicines including in rare disease and oncology considering value and affordability. Kirsten has guided the development of patient financial assistance programs for biopharma. She has prepared companies and their leaders for Congressional investigations and testimony related to drug price, price increases marketing and research investment. She often uses financial models and data analytics to demonstrate how different stakeholders in the healthcare delivery chain are affected by policy and business practice to guide corporate policy and business practice recommendations and to estimate the impact of policy change for corporate financial disclosure. Kirsten is a She is a policy advisor to the DLA Piper a visiting scholar with the American Enterprise Institute and she is the founder of the non-profit Preparedness and Treatment Equity Coalition.
Amanda Cole, PhD
The Office of Health Economics, London, LON, United Kingdom
Amanda Cole is Associate Director at the Office of Health Economics. Amanda’s research interests include the economics of innovation, 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.
ISPOR Forums
Using Clinical Outcome Assessments (COAs) in Your Research Study Does Not Necessarily Make It Patient-Centric
Many sponsors claim to be patient centric, centered, or focused. However, what exactly does that mean? For example, does having clinical outcome assessments (COAs) in studies of new health technologies make them patient centric? The ISPOR COA and Patient-Centered Special Interest Groups (SIG) aim to provide leadership in alignment between patient centricity and COA development to achieve harmonization among all stakeholders of interest.
In this session, we will define patient-centricity, highlight reasons for using COA methodology in research studies, and describe how this can improve the relevance of new health technologies to patients. Members of the ISPOR community work on new health technologies at various stages of the lifecycle to meet different evidence requirements, and a common theme should be patient centeredness.
The speakers will outline different methodologies and associated regulatory guidance that pertains to COA and drive towards being patient focused to meet their needs. We will discuss how best to use qualitative, quantitative, and mixed methods for appropriately capturing the patient voice, ensuring measured outcomes are patient centered throughout the technology development journey. Case studies will be presented on how patient insights can inform COAs development. Panel members and attendees will discuss and derive a framework for understanding how COA methods can be optimized for authentic patient-centered research in the development of health technologies.
Moderators
Hoda Fotovvat, PhD
Evidera, Bethesda , MD, USA
My name is Hoda Fotovvat and I will moderate this session.
Speakers
Eleanor Perfetto, PhD, RPh, MS
University of Maryland School of Pharmacy, VENICE, FL, USA
Eleanor M. Perfetto has worked in government, industry, consulting, and academia. Most recently, Eleanor was with the leading, U.S. nonprofit organization focused on patient issues as Executive Vice President for Strategic Initiatives at the National Health Council (NHC). Currently, she holds a faculty appointment at the University of Maryland School of Pharmacy where she is Professor of Pharmaceutical Health Service Research. Her research and policy work primarily focus on patient and family engagement, including comparative effectiveness and patient centered-outcomes research; patient-focused drug and medical-product development; clinical outcome assessment, selection, and development; and patient-centered value assessment and healthcare quality measurement.
As caregiver for her late spouse who suffered from early dementia due to chronic traumatic encephalopathy, Eleanor is a recognized advocate for patients with head-trauma-related dementia and their families. In May 2007, she received the Alzheimer’s Association New York City Chapter Advocacy Leadership Award. In 2010, she received the Alzheimer’s Association National Capital Area Chapter Award for Advocacy & Awareness. In 2012, she received the Legacy Award from the Concussion Legacy Foundation, for which she is a past board president and a two-term inaugural board member. Today, she serves on the board for the Brain Injury Association of America, where she chairs the Governance Committee and serves as Board Secretary. She also is a current board member for the Dan Lewis Foundation, which is dedicated to brain regeneration research.
Prior to joining the NHC and University faculty, Dr. Perfetto was with Pfizer Inc for almost eight years, most recently as Senior Director, Federal Government Relations; past positions were in Evidence-Based Strategies and Payment Policy Analysis. Early in her career, she served in the U.S. Public Health Service serving for 6 years as a pharmacist in the Indian Health Service in Pine Ridge, SD and Talihina, OK, and as senior pharmacoepidemiologist at the Agency for Health Care Policy & Research (now Agency for Healthcare Research & Quality). Her highest rank was Commander (05).
Dr. Perfetto has a distinguished record of service to professional and other organizations. Currently, she is Chair of the Patient-Centered Special Interest Group at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and served as co-chair for the 2016 ISPOR Annual Meeting. In the past, she has served as a CMS MedCAC member; co-chair of the National Quality Forum’s Alzheimer’s Disease and Related Dementias Project and on the Patient-Reported Outcomes Expert Panel; a past Pharmacy Quality Alliance and Drug Information Association board chair; and past member of the Center for Medical Technology Policy Board and Health Industry Council Board of Advisors. She was an Assistant Editor for the Journal of Managed Care and Specialty Pharmacy and has been a peer reviewer for numerous journals, the Patient-Centered Outcomes Research Institute, government granting agencies, and pharmacy professional organizations. She has contributed book chapters and published in peer-reviewed journals. She is a frequent speaker at numerous national and international scientific conferences.
Dr. Perfetto holds BS and MS degrees in pharmacy from the University of Rhode Island and in 2009 received the Distinguished Alumni award from the College of Pharmacy. She also holds a PhD in Public Health, concentrating in health policy and epidemiology, from the University of North Carolina at Chapel Hill.
Katja Rudell, PhD, MSc
Parexel, London, United Kingdom
Katja Rudell cofounded the ISPOR Special Working Group in COA at ISPOR in 2019 and is a Senior Director of COA Science at Parexel. She holds a PhD from University of London, and has been working in Clinical Outcomes Research for 20 + years.
Kathleen Wyrwich, PhD
Bristol Myers Squibb, St. Louis, MO, USA
Kathy Wyrwich, PhD, is Senior Director in Patient-Reported Outcomes Assessment (PROA) at BMS, focused on immunology, cardiovascular, fibrosis and neuroscience diseases. Kathy earned her PhD in Health Services Research from Saint Louis University and has 25 years of experience conducting clinical research studies investigating clinical outcome assessment (COA) development, validation, and interpretation of meaningful change over time. She frequently publishes on COA research endeavors with over 17,000 citations from this work.
Using HEOR Methods for Improving the Implementation of Value-Based Healthcare
Value-based healthcare (VBHC) is about improving outcomes that matter to patients in a cost-effective way. While many HEOR projects analyse specific interventions, VBHC takes a health system level approach focusing on the breadth of interventions that target specific population segments. In this Forum we will share and discuss penultimate findings from the ISPOR VBHC special task force (STF). The STF aims to deliver an ISPOR guidance on good practices in VBHC implementation, as well as methodological guidance on how to effectively use existing HEOR methods to drive VBHC initiatives forward. These are areas of VBHC where authoritative thought leadership by credible international organizations is absent.
Lotte Steuten will open the workshop with an overview of VBHC, motivating the need for better HEOR guidance for its implementation and evaluation, and provide a status update of the Special Task Force’s progress. Ana Paula Beck da Silva Etges will report on the results of the interviews undertaken with >40 experts from the field and discuss how these contribute to a catalogue of good practices in the use of HEOR methods to assess the impact of VBHC experiments. Bill Padula will provide an outlook on the next steps for the STF to finalize their good practice guidelines for HEOR to support implementation of VBHC (eg, HEOR methods to enable continuous improvement in clinical practice and decision making). We will allow significant time to discuss comments and questions from the audience. Polls will be used throughout the session to assess perspectives and feedback from the audience on the STF approach. After this session the audience will understand the need for better HEOR guidance for the implementation and continuous evaluation of VBHC projects and have the opportunity to provide input into the evolving taskforce recommendations.
Moderators
Jens Grueger, PhD
Boston Consulting Group, Boston, MA, MA, USA
Jens Grueger is a Director and Partner at the Boston Consulting Group based in Zurich Switzerland. He provides expert advice to companies in the life science industries on pricing and market access. Since July 2019, he is an Affiliate Professor of Health Economics at the CHOICE Institute at the University of Washington School of Pharmacy in Seattle, USA. He was the 2020-2021 President for ISPOR, the leading professional society for health economics and outcomes research.
Prior to this, Jens had a distinguished career in the pharmaceutical industry. From 2011-2019, he was the Senior Vice President and Head of Global Access for F. Hoffmann-La Roche based in Basel, Switzerland. Under his leadership, Roche created rapid, broad and sustainable patient access for 12 new medicines and expanded access to essential cancer medicines in lower- and middle-income countries from 29 to 60%. Before this Jens was the Vice President and Head of Global Market Access Primary Care at Pfizer, based in New York and London (2009-2011), Head of Global Pricing & Health Economics at Novartis Pharma in Basel, Switzerland (1999-2009) and Director of Health Economics at the German affiliate of SmithKline Beecham Pharma in Munich (1994-1997). He founded Diversified Health Systems, a start-up company providing internet-based disease management services to physician networks in Europe (1997-1999).
Jens holds a MSc (Diplom) in Medical Statistics and Theoretical Medicine, and a PhD in Mathematical Statistics from the Technical University of Dortmund, Germany. He has authored more than 40 publications in biometrics, cancer epidemiology, and health economics.
Speakers
Ana Paula Etges, PhD, Eng.
Avant-garde Health, 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 Research Associate at Avant-garde Health (Boston, USA), she serves as Professor in the Graduate Program in Epidemiology at UFRGS and in the School of Technology at the Catholic University of Rio Grande do Sul(PUCRS), Senior Researcher at the National Institute for Health Technology Assessment (IATS), and a Partner from consulting firms (PEV and Way2Value, in Brazil and Portugal, respectively). 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 now is attending to the Effective Writing Certification Program at Harvard Medical School.
William Padula, PhD, MSc, MS
Hasbro Children's Hospital, 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.
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).
Assessing Real-World Data From Electronic Health Records (EHRs) for HTA
The task force will introduce the topic of assessing real-world data (RWD) from electronic health records (EHR) for health technology assessments (HTAs). While the increasing availability of EHR provides new opportunities to improve and refine HTAs, there are a number of known issues related to EHR-derived RWD that can impact their quality and suitability. Given the relatively recent availability of EHR-derived databases, there is an urgent need to provide guidance to HTA groups that review studies including such data. With an international team of experts, the task force aims to establish emerging good practices for the use and interpretation of EHR-derived RWD for HTA. The report will include recommendations and a checklist for consumers of HTAs.
During the presentation, speakers will provide a brief overview of EHR data guidelines by NICE in the UK, ICER in the US, and CADTH in Canada. They will summarize published data quality frameworks and highlight key domains that should be required for HTAs (example: representativeness, completeness and accuracy). Speakers will then discuss data provider issues, such as method transparency for extracting data and approaches for integrating structured versus unstructured data, including use of common data models.
An important deliverable of the report is a data quality checklist for use of EHR-derived data. The panel will discuss challenges to and opportunities for developing such as checklist. Finally, speakers will highlight continuing limitations of EHR data, including the lack of representativeness, privacy and confidentiality issues, consent issues, data security concerns and funding sources. The panel will conclude with a discussion on future directions as the field continues to evolve rapidly. Presented by the ISPOR Use of Electronic Health Records (EHRs) in HTA Task Force.
Moderators
Scott Ramsey, MD, PhD
Hutchinson Institute for Cancer Outcomes Research, Fred Hutchinson Cancer Center, Seattle, WA, USA
Dr. Scott Ramsey is a Professor at Fred Hutchinson Cancer Center and the University of Washington. He directs the Hutchinson Institute for Cancer Outcomes Research, a multidisciplinary team devoted to clinical and economic evaluations of cancer prevention, screening and treatment.
His research portfolio includes SEER-Medicare/Cancer Registry data linkages, patient outcomes, economic modeling of interventions, cost-effectiveness analysis, quality of life assessment, health care utilization, economic burden, pragmatic trial design, early technology assessment, and stakeholder engagement.
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 Principal Scientist 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.
Rachael Fleurence, PhD
National Institutes of Health, Bethesda, MD, USA
Seamus Kent, PhD
Flatiron Health, Amsterdam, NH, Netherlands
Seamus is a Senior Adviser in HTA at Flatiron UK. He was previous a Senior Adviser in Data & Analytics at NICE where he led the development of NICE's Real-world evidence framework.
Member Group Meeting
Biosimilars Special Interest Group Open Meeting
12:15 PM - 12:45 PM
Exhibit Hall Theater
Developing Fit-for-Purpose External Control Arms to Optimize Submissions to Regulatory and Health Technology Assessment Agencies
Digital Conference Pass
When researchers use existing data to contextualize the results of single-arm trial data, it is called an external control arm (ECA). ECAs may be appropriate when recruiting an adequate number of patients is difficult or impossible, such as for trials in rare and/or imminently life-threatening conditions. However, when using real-world data (RWD) to populate an ECA, the burden is on researchers to demonstrate the appropriateness of the data and methodology. Regulatory and health technology assessment (HTA) agencies have been critical in their feedback on previous ECA submissions, citing concerns about the validity of evidence from RWD, including confounding and selection bias. Recognizing these issues, the US Food and Drug Administration (FDA) released a draft guidance in February 2023 highlighting key agency considerations when assessing whether the real-world evidence (RWE) generated from ECAs is fit-for-purpose.
In this session, senior experts in epidemiology and biostatistics will discuss their perspectives on best practices and first-hand experiences with ECAs.
Learning Objectives:
- Describe the framework for using RWD to enable a comparison group for a single-arm clinical trial.
- Understand key regulatory and HTA agency concerns regarding the methodological challenges to ECAs, including data selection, patient identification, confounding and bias, and standardized reporting of outcomes.
- Explore the pathways to early engagement with regulatory and HTA agencies to assess the suitability of ECA methods to complement a clinical trial program in support of drug approval.
Seating is limited.
Sponsor: Genesis Research
Sponsor
Genesis Research
Speaker
Craig Parzynski, MS
Genesis Research, Hoboken, NJ, USA
Craig Parzynski is Senior Director and Head of the Biostatistics & Analytics at Genesis Research. He has 16 years of experience in research conduct and 12 years of experience in the field of statistics. He has contributed to over 40 peer-reviewed journal articles across many fields and therapeutic areas. Prior to Genesis Research, he spent over 9 years as a statistician at Yale Center for Outcomes Research and Evaluation working on statistical problems for observational and RWE studies for the Centers for Medicare & Medicaid Services, American College of Cardiology, FDA, among others. He has expertise in causal inference, external control arms, generalized linear models, generalized linear mixed models, longitudinal and repeated measurement analysis, time-to-event analysis, missing data methods, balancing methods, indirect treatment comparisons and observational studies.
Jenifer Wogen, MS
Genesis Research, Hoboken, NJ, USA
Jen Wogen is Senior Director, Evidence Strategy at Genesis Research. In her current role, Jen leads real-world evidence generation across many therapeutic areas, including studies utilizing a combination of clinical trial data and real-world data (e.g. external control arm studies). Jen has more than 20 years of experience executing both retrospective and prospective studies within epidemiology, health economics/outcomes research, and health services research. Previously, Jen has worked at roles within the pharmaceutical industry, in consulting, and in academic research and undergraduate and postgraduate teaching.
12:45 PM - 1:15 PM
Patient Synchronicity: Exploring a Novel, Patient-Centric Data Management Approach and Innovative Datasets for Integrated Evidence Generation Across the Product Lifecycle
Digital Conference Pass
Pharma companies are increasingly in need of privacy-compliant ways to leverage first- and third-party data for real-world evidence generation. Industry standards are changing, however, as more novel and specialized datasets are required by researchers. Handling this sensitive data, be it de-identified or identifiable, or both, requires high-governance technology to do so securely.
With the proper technology, consent and privacy management processes, we’ll explore how you can effectively build a registry of patients interested in participating in research, such as clinical trials or prospective observational studies. We’ll also dig deeper into innovative data solutions touching on maternal health outcomes, race, ethnicity, mortality, rare disease and more.
Learning Objectives:
- Explore the benefits of building a patient registry for interventional and prospective observational studies
- Learn the importance of accurate patient matching and identity resolution techniques
- Understand the challenges and strategies for managing privacy and consent across complex partner ecosystems
- Discover new and innovative datasets to drive real-world evidence generation for longer term outcomes and safety studies
Seating is limited.
Sponsor: Health Verity
Sponsor
Health Verity
Speaker
Reyna Klesh, MS
HealthVerity, Philadelphia, PA, USA
Reyna Klesh is a healthcare data expert with 20 years of experience analyzing various real-world datasets while meeting strict HIPAA compliance. She has been with HealthVerity for seven years and currently serves as the vice president, data insights and innovation.
Prior to joining HealthVerity, Reyna served as the senior director of global data insight and analytics for Dun & Bradstreet and held various analytics positions with a leading healthcare information services company, working her way up to director of advanced analytics. She has also co-authored and assisted with the research for five different studies related to COVID-19 research.
Reyna earned a bachelor’s degree in Mathematics from Ursinus College and a master’s degree in Statistics from Temple University.
Joe Satili, BS
HealthVerity, Philadelphia, PA, USA
Joe Satili leads the HealthVerity Sales and Account teams who are responsible for building strategic partnerships with our customers to drive company growth.
Prior to HealthVerity, Joe was Senior Vice President at IQVIA where he was responsible for leading their US Commercial Sales and Account Management teams. In this role, he led business development and transformed the go to market strategy to support the company’s growth and expansion into new capabilities. During his tenure at IQVIA, he also had responsibility for IQVIA’s information offerings and client services teams. In these roles, he drove increased customer satisfaction and turned around the positioning and growth of the information business to meet the changing market needs.
Joe holds an undergraduate degree in Electrical Engineering from New York University.
1:30 PM - 2:30 PM
Member Group Meeting
Rare Disease Special Interest Group Open Meeting
Closed Member Group Meeting
ISPOR Mexico Chapter Meeting
1:45 PM - 2:45 PM
Breakouts: IP, WS, & OBS
OPUF (Online Elicitation of Personal Utility Functions): Understanding What Brings Value to the Individual
Digital Conference Pass
Level: Introductory
PURPOSE:
Established valuation methods, such as TTO and DCE require data from large samples. They typically lead to estimates of an “average” value function and are very limited in exploring the heterogeneity in peoples values. Given an instrument with different dimensions with various levels (within the workshop the EQ-5D-5L), the personal utility approach elicits weights from each individual for the different dimensions together with values concerning the levels within dimensions. Additionally, questions are posed to enable anchoring on a 0-1 scale. The advantage is that the result is “personal”; it leads to a value function for each individual and can be used in small samples. Workshop participants will get a live demonstration of the tool using the EQ-5D. Using a real-time survey we will ask the audience to participate. As such, an “ISPOR-participant-value function” for the EQ-5D-5L will be derived. (It will be a biased estimate, but it will be fun to analyse and present.) Results will be available immediately and the “US-ISPOR-2023 value function” will be compared with those obtained from the general public. This workshop will resonate with an audience that is interested in valuation, innovation, patient-centric solutions, individualized medicine, and rare diseases. DESCRIPTION:
In this workshop, we will address the WHY-HOW-WHAT-WHEN of OPUF.
WHY (10min): Dr. Van Hout will discuss the unmet need that OPUF can fulfil in comparison to existing methods.
HOW (5min): Dr. Devlin will discuss the theoretical framework to explain what sets OPUF apart from current stated preference methods.
WHAT (20min): Dr. Schneider will provide a live demonstration based on the EQ-5D-5L with participation of the audience, and show how OPUF can be used to generate aggregate and respondent-level results.
WHEN (5min): Dr. Heisen will the discuss in which cases OPUF should be considered. Dr. Devlin will conclude the workshop and chair the Q&A session.
Discussion Leaders
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 in health care; 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 >150 original peer reviewed journal papers on a wide range of empirical and theoretical topics in health economics.
Discussants
Marieke Heisen, PhD
Open Health, Rotterdam, Netherlands
Paul Schneider, MD, MSc
University of Sheffield, Sheffield, United Kingdom
Paul Schneider is a PhD student at the Wellcome Trust funded doctoral training centre for public health, economics, and decision science at University of Sheffield, UK.
Paul's research focuses on methodological and normative issues in the valuation of health. He has a keen interest in the use of modern software and interactive user interfaces for health valuation and decision modelling.
https://bitowaqr.github.io/
Ben A Van Hout, PhD
University of Sheffield, Sheffield, United Kingdom
Ben van Hout combines a position of professor of Health Economics with that of Chief Scientific Officer in OPEN Health Evidence & Access. In 2020, he received the Donabedian Lifetime Achievement award for his contributions to the field of economic evaluations in health care. Over the last couple of years, he has taken his research to the next level by first having a myocardial infarction and second having a large motorcycle accident. Since then, he has taken more than 16,000 tablets, still counting, for prevention, pain and itch.
His current position allows him to only be involved in projects he fancies, “personal utility functions” being on the top of the list.
The Reality of Target Trial Emulation for Medical Decision-Making and HTA Recommendations - Is the Gap Between Academic and HTA Applications Too Wide?
Digital Conference Pass
Level: Intermediate
PURPOSE: Target trial emulation (TTE) is a framework for using non-randomized data to emulate randomized controlled trials (RCTs) with a clearly specified target population, intervention strategies, and outcomes. TTE was recently endorsed by the National Institute for Health and Care Excellence (NICE) and has been touted as the potential magic bullet for improving the quality and reporting of real-world evidence in HTA submissions. Because TTE is a complex topic, and because most applications illustrate its use in close to ideal data scenarios, there is considerable barrier for developers and decision-makers in being able to understand, implement and critique TTE analyses in practical scenarios. This is particularly true for those commonly submitted to HTA agencies involving small sample sizes and externally controlled single arm trials.
This workshop will cover TTE in the context of real-world data scenarios for HTA submission with examples of practical applications for TTE in the context of synthetic control arm analyses.
Kristian Thorlund will introduce the audience to the current state of TTE applications (5 min).
Joy Shi will detail the design of TTE framework, cover the nuts and bolts of causal models, and address the need for multidisciplinary teams (15 min).
Alind Gupta will detail the challenges of applying TTE on real world data sets (15 min).
Stephen Duffield will examine submissions with TTE that were deemed high versus low quality and provide guidance for moving forward (15 min).
DESCRIPTION: Participants will be introduced to the stages required for well-conducted TTEs; challenges of designing causal models and the necessity of multidisciplinary teams; how to perform valid TTE when sample sizes are small; as well as what HTA agencies and payers generally accept as good quality TTEs.
This workshop would be valuable to researchers, industry analysts, and sponsor representatives who are interested in incorporating TTE in HTA submissions.
Discussion Leaders
Kristian Thorlund, MSc, PhD
Cytel, 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 statistical methodologies that are now being used widely
Discussants
Stephen Duffield, PhD, MD
NICE, Liverpool, United Kingdom
Stephen is an Associate Director 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
Alind Gupta, PhD
Cytel, Toronto, ON, Canada
Joy Shi, PhD
Harvard T.H. Chan School of Public Health, Boston, MA, USA
Joy Shi is an Instructor of epidemiology in the CAUSALab and Department of Epidemiology at Harvard T.H. Chan School of Public Health. Her research focuses on improving causal inference methods, particularly those related to instrumental variable estimation, for answering clinically relevant questions about the treatment and prevention of chronic diseases. She teaches causal inference methodology in the Department of Epidemiology at Harvard T.H. Chan School of Public Health and is an Assistant Director for the comparative effectiveness pathway in the Master of Medical Sciences in Clinical Investigation (MMSCI) program at Harvard Medical School.
ISPOR New Professionals Session
Things They Didn't Teach You in Grad School
Graduate school does a great job of teaching technical skills. However, early career success also depends on demonstrating competencies in soft skills like influencing, creative problem-solving, leadership behaviors, critical thinking, cross-functional work, and strategic decision making. Join the members of ISPOR’s New Professional Steering Committee for this panel-style session as they highlight key soft skills and effective ways to learn and develop these skills.
Moderators
Anna Hung, PharmD, PhD
Duke University School of Medicine, Durham, NC, USA
Anna Hung, PharmD, PhD, MS is a pharmacist and health services researcher interested in payer and patient decision making related to pharmacy benefits. Her methodological research interests include health care cost evaluations, quasi-experimental study designs, and stated preference research.
Speakers
Sanket Shah, MBBS, PhD
Novo Nordisk, Inc., Gaithersburg, MD, USA
Sanket is a physician and health economist with over 7 years of experience in the biopharmaceutical industry. Currently, he leads US HEOR for the Rare Disease portfolio at Novo Nordisk. In addition to his work, he serves on the steering committee of the ISPOR New Professionals network and hosts their soft skills panel. He enjoys crafting value stories using high quality scientific evidence.
Spotlight Session
Larger, Deeper, and in Real Time: Applications of Machine Learning and Natural Language Processing on Electronic Health Records to Learn From the Patient Journey at Scale
Digital Conference Pass
Level: Intermediate
PURPOSE:
This session describes the pragmatic impact of applying machine learning (ML) and natural language processing (NLP) on Electronic Health Records (EHRs) to generate and accelerate insights on the patient journey through scale, depth, and speed.
DESCRIPTION: The emergence of novel prognostic factors (biomarkers, mutations) and associated therapies have increased appreciation and emphasis of precision medicine. To understand patient outcomes in increasingly smaller populations and to keep up with the rapidly evolving standard of care, real-world data (RWD) information systems need to be scalable, relevant, and timely. Applications to understand outcomes for patients with rare biomarkers, to identify at-risk populations, and to address inequities in care delivery and research all require large-scale and detailed RWD in order to provide precise estimates in all relevant subgroups to assist decision-making. Though conventional data sources such as claims data provide information at scale, they lack the clinical depth that unstructured EHR data (e.g., clinical notes, pathology reports) offers, such as information about biomarker results. While traditional manual curation by clinical experts provides rich information about the patient journey, ML/NLP techniques have improved upon these resource-intensive curation methods, generating deep insights at scale and in real-time.
This session will focus on case studies where ML/NLP is applied to EHRs to obtain RWD and real-world evidence (RWE) quickly and at scale. First, an overview of using ML/NLP to generate RWD/RWE is provided. Then, case studies on increasing stratification cohort sizes (scale), keeping up with standard of care (speed), and improving representation of underserved populations (depth) are discussed. Speakers will share metrics to evaluate the pragmatic impact of using ML/NLP on their applications. Speakers will pose 1-2 polling questions to guide a discussion, where the audience is invited to share their perspectives and experiences.
Signal—ISPOR’s signature program—looks beyond today’s linear thinking to explore topics that will shape healthcare decision making over the next decade. Seeking to strengthen strategic foresight and adaptive leadership capacities in the complex world of healthcare, the Signal series focuses on the “big picture,” while also addressing how health economics and outcomes research (HEOR) can best contribute to solving healthcare’s greatest challenges.
Moderators
Joe Vandigo, MBD, PhD
Applied Patient Experience, Greensburg, PA, USA
Joe Vandigo, MBA, PhD is a Principal Scientist and Director of Value at Applied Patient Experience. He oversees work related to the policy and methods of patient engagement in health technology assessment. Dr. Vandigo is a health services researcher trained in continuous patient engagement who brings more than a decade of research and policy experience around health technology assessment in the United States.
Dr. Vandigo joined Applied Patient Experience from the Market Access Innovation team at Novartis. His role there focused on developing value-based pricing approaches and highlighting pharmaceutical market distortions that impact patient access. Before Novartis, he spent five years at PhRMA, the trade organization representing innovative biopharmaceutical manufacturers. At PhRMA, his research highlighted gaps in existing methods of value assessment. His portfolio also included research and policy strategy development related to the impact of hospital consolidation and market power on pharmaceutical prices, innovative contracting approaches, and novel financing arrangements.
Speakers
Selen Bozkurt, PhD, MS
Stanford University, Palo Alto, CA, USA
Dr Selen Bozkurt is an expert in biomedical informatics, biostatistics, biomedical NLP, and RWE. She has co-authored over 100 publications, has been an invited speaker to WIDS Istanbul and PhamML 2022, and has presented her work at many academic conferences.
Ravi Parikh, MD, MPP
University of Pennsylvania, Philadelphia, PA, USA
Ravi B. Parikh, MD, MPP is an Assistant Professor of Medicine and Health Policy and Director of the Human-Algorithm Collaboration Laboratory at the University of Pennsylvania, Associate Director of the Penn Center for Cancer Care Innovation, and Staff Physician at the Corporal Michael J. Crescenz VA Medical Center. Dr. Parikh is a practicing oncologist and leads several NIH-funded trials applying AI and machine learning in clinical care workflows to improve patient outcomes.
Katherine Tan, PhD
Flatiron Health, Stamford, CT, USA
Dr Katherine Tan has published extensively in the topics of biostatistics, biomedical NLP and RWE in oncology. Her work has appeared in top academic conferences and journals such as Biostatistics, Clinical Pharmacology & Therapeutics, ISPOR, and Value of Health; and she has organized and spoken at panels (ENAR) and podcasts (ResearchX) on RWE.
Breakouts: IP, WS, & OBS
Disinvestment in Healthcare Systems: The Why, What, When, and How?
Digital Conference Pass
ISSUE:
Operating in an economic environment of increased pressures and resource scarcity, the when and how (not why) of healthcare disinvestment is increasingly discussed in academia. Disinvestment can play an important role in helping a healthcare system to meet efficiency and equity goals and hence alleviate some of these pressures. However, theory around disinvestment has not necessarily been translated to practice and there remain various efficiency and equity concerns from different stakeholders (payers, patients, public, industry and healthcare professionals). In this context, it is important to evaluate and discuss practical considerations regarding when disinvestment can be most necessary or beneficial, how it can be implemented to alleviate pressures, and influences based on the healthcare system in question.
OVERVIEW:
Naomi van Hest will introduce the topic of healthcare disinvestment (5 minutes), moderate discussion and challenge the panellists on the why, what, when and how of disinvestment for the healthcare system they’re representing. Dr André Azaredo will present on the 'tried and tested' experience of disinvestment in Brazil, and the challenges that were faced, as well as the current work on disinvestment that is in development within the United Health System (SUS) (12 minutes). Dr Rosmin Esmail will provide a perspective on the role of disinvestment within an established healthcare system (Canada, amongst others), considering it as one output of the health technology reassessment process guided by the field of knowledge translation (12 minutes). Hanin Kamaruzaman will represent a developing healthcare system perspective, presenting on the applicability of disinvestment to LMICs (e.g. Malaysia) based on her research in the field (12 minutes). Discussion time will be included (15 minutes) and audience participation encouraged throughout, with interactive voting used to gain audience input.
Moderators
Naomi van Hest, MSc, BSc
Costello Medical, London, United Kingdom
Naomi is the UK Head of Health Economics and Statistics at Costello Medical, leading the development of health economic models for reimbursement and market access, particularly in oncology. She has contributed novel research to ISPOR over the past 5 years, ranging from cost-effectiveness analyses, to survival extrapolation, reducing uncertainty and expert elicitation. Naomi has completed a Master's in health data analytics from University College London, and studied her undergraduate degrees in Australia.
Panelists
Andre Azeredo da Silva, MD, PhD
HTAnalyze Consulting, Porto Alegre, Brazil
MD by the Federal University of Health Sciences of Porto Alegre, specialized in Internal Medicine at Hospital de Clínicas de Porto Alegre. Holds a master’s degree from the Graduate Program in Medicine: Medical Sciences, Federal University of Rio Grande do Sul, and doctorate at the Graduate Program in Epidemiology at the same university. Experienced consultor for incorporation of health tecnologies in the public and in the private healthcare system in Brazil.
Rosmin Esmail, PhD, MSc, CHE
Alberta Health Services, Calgary, AB, Canada
Over the past year, Rosmin Esmail been the Assistant Scientific Director for the Diabetes Obesity and Nutrition Strategic Clinical Network (DON SCN), Alberta Health Services, Calgary, Alberta, Canada. In this capacity, she is responsible for the DON SCN Scientific Office that integrates theory, scientific evidence, and best practices on SCN research priorities and projects. She provides methodological support to various research projects in the development of knowledge translation strategies, grant writing, data support, ethics review, and preparation of a scientific impact report for the SCN. Rosmin also collaborates and engages with the diabetes, obesity and nutrition research network to embed evidence into clinical practice. She is an expert in the field of knowledge translation and implementation science. She recently completed her PhD at the University of Calgary. Her thesis focused on understanding the relationship between knowledge translation (KT) and health technology reassessment. Her thesis provides the theoretical foundation needed for the application of KT to the field of technology reassessment. This work has resulted in four publications in high impact journals. Over the past 25 years, Rosmin has worked in various clinical areas including trauma services, cancer, musculoskeletal disorders, seniors care, critical care, and more recently in diabetes, obesity, and nutrition. In these roles, Rosmin has supported teams and clinicians in the application and implementation of best practices in evidence-based research, systematic reviews, guideline development, epidemiology, evaluation, quality improvement, knowledge translation, health technology assessment, and reassessment.
Sergio Torres Rueda, BA, MSc, PhD
London School of Hygiene and Tropical Medicine (LSHTM), London, LON, United Kingdom
Inflation Reduction Act (IRA) Negotiation: Closer to Government Price Setting or Traditional HTA?
Digital Conference Pass
ISSUE: In contrast to ex-US pharmaceutical markets, the US has historically had decentralized decision-making regarding drug value. The Inflation Reduction Act (IRA) moves the US closer to centralized Health Technology Assessment (HTA) as it requires the Secretary for Health and Human Services (HHS) to negotiate maximum prices for select brand-name drugs that are covered under Medicare Part B (physician-administered drugs) and Part D (retail prescription drugs) effective plan year 2026. The Secretary must consider information about development and production (e.g., manufacturing and distribution costs, financial support from the government, patent information, R&D recoupment) and evidence about alternative treatments (e.g., therapeutic advance, prescribing information, comparative effectiveness, unmet need). In this session, panelists will explore the question of price setting or traditional HTA by examining key differences and associated ramifications.
OVERVIEW:
There are important similarities and differences in how IRA negotiation and traditional HTA reach the common goal of determining a price. Key differences reside in the evidence and methods used. How will these differences impact the outcomes? And what does it mean for the goal of patient centricity? The panel will begin with Sarah Alwardt providing a brief overview of IRA negotiation. Next, Mike Ciarametaro will highlight key evidence and method differences, including a high-level overview of potential IRA negotiation models. Then, Peter Neumann will examine the unique HTA method challenges and opportunities the government will face when implementing the negotiation process. Topics to be discussed include product life cycle timing, evidence mix, and multiple indications. Last, Emily Freeman will delve into the opportunities and challenges for IRA negotiation incorporating a patient-centered care approach. Topics will include the processes and methods for incorporating patient feedback and patient-centered outcomes to determine a negotiated price. The audience will have the opportunity to ask questions at the end.
Moderators
Sarah Alwardt, PhD
Avalere Health, a member of Fishawack Health, Celebration, FL, USA
Sarah Alwardt leads Evidence & Strategy practice. A highly accomplished leader with multi-national experience in strategy, research, operations, and technology innovation, Sarah delivers data-driven insights and real-world evidence to stakeholders across the healthcare ecosystem.
Prior to joining Avalere, Sarah grew and launched Ontada, McKesson’s oncology research business providing innovative oncology evidence development strategies. She sponsored the implementation of big data technologies for natural-language processing, data integration, and visualization and lead the scientific team to produce more than 200 publications and multiple regulatory filings. She enjoyed 10 years at Humana where she held leadership roles in research, program, and process development. Sarah spent 5 years at Givaudan and 4 years at Brown-Forman. She also served 2 years as adjunct professor of chemical engineering at the University of Louisville.
Sarah holds a BS, MEng, and PhD in chemical engineering and a certificate in environmental engineering, all from the University of Louisville’s Speed School of Engineering. She is a member of the International Society for Pharmacoeconomics and Outcomes Research and the American Institute of Chemical Engineers. She is an accomplished author and speaker, having published, patented, and presented works regarding the generation and use of real-world evidence, biofuels, flavors, and fragrances.
Panelists
Michael Ciarametaro, MBA
Avalere Health, a member of Fishawack Health, Washington, DC, USA
Michael Ciarametaro advises life sciences clients on developing value and evidence strategies, drug pricing, payment reform, and the Inflation Reduction Act.
In recent projects, Mike has helped life sciences companies strategize and prepare for Medicare drug pricing negotiations under the IRA by evaluating value propositions for therapies, advising on improving those value propositions, identifying key opportunities to engage policymakers, and delivering strategic recommendations to clients.
Emily Freeman, MSc, PhD
Gilead Sciences, Foster City, CA, USA
Global-thinking, entrepreneurial and innovative social/behavioral scientist with more than 21 years of progressive experience in health services & outcomes research (Patient Experience Data, Patient Value Frameworks), diversity, inclusion & equity in drug development, patient engagement, and FDA/EMA regulatory policy, with over a decade of that time in a biopharma environment. I believe strong user centered research helps to build more successful, patient-centered products and I have a proven track record of positively impacting products in clinical development & post-marketing and getting a stronger seat within medical affairs & clinical development for social/behavioral sciences.
Peter Neumann, ScD
Tufts University, 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.
Transparency in Health Technology Assessment: How Much Is Too Much?
Digital Conference Pass
ISSUE:
The development of new treatments has the potential to improve patient outcomes. To support this innovation, safety and efficacy data are often kept confidential during development. However, health technology assessment (HTA) agencies have a responsibility to ensure their evaluations are open and transparent. How can the global healthcare community find a balance between protecting commercially sensitive information and providing transparent information for stakeholders?
OVERVIEW:
Many HTA agencies have processes to protect confidential data that is academically- and/or commercially-sensitive. This allows companies and data owners to provide evidence to HTA agencies without jeopardising future publications or commercial agreements, because the data are redacted from published HTA documents. In 2022, the International Committee of Medical Journal Editors (ICMJE) announced it no longer considers data published by HTA agencies to be duplicate publication. This prompted HTA agencies to reconsider their policies on confidential information in their evaluations, particularly “academic-in-confidence”. Pharmaceutical companies highlighted that current policy allows for provision of clinical data in confidence earlier than public presentation or publication, and that policy changes may affect patient access. In response to this, HTA agencies have been working together to explore how best to address the issue. This is an opportunity for healthcare decision makers alongside industry to consider and debate transparency and data protection. Dan Ollendorf will present a 5-minute overview of the issue. Each panel member will then have 10 minutes to present their perspective. After the presentations, Dan will summarise and pull out the key themes before a 20-minute panel discussion with audience participation. It’s expected that all stakeholders in the HTA process would benefit from this discussion.
Moderators
Daniel Ollendorf, PhD
Tufts Medical Center, Boston, MA, USA
Dan Ollendorf, PhD, MPH is Director of Value Measurement and Global Health Initiatives at the Tufts Center for the Evaluation of Value and Risk in Health (CEVR) and Assistant Professor of Medicine. Dr. Ollendorf’s research interests include expanding the use of health technology assessment (HTA) in low- and middle-income countries, as well as refinement and use of value assessment tools in the US and other high-income settings. Prior to joining CEVR, Dr. Ollendorf was Chief Scientific Officer for the Institute for Clinical and Economic Review (ICER) for over 10 years.
Panelists
Meindert Boysen, PharmD, MSc
National Institute of Health and Care Excellence (NICE), Manchester, United Kingdom
Meindert is responsible at NICE for international partnership working, developing NICE’s role in international fora and representing NICE at key international meetings, further sustaining and developing NICE’s international reputation. Until recently, Meindert was the director at NICE responsible for delivery of the programmes that together form the centre for health technology evaluation. Including the technology appraisals, highly specialised technologies, medical technologies evaluation, diagnostic assessment and interventional procedures programmes. After qualifying as a pharmacist from Utrecht University, he worked in a hospital in the Netherlands, and held positions in health outcomes and sales in the pharmaceutical industry. Meindert completed the MSc in Health Policy Planning and Financing at the London School of Hygiene and Tropical Medicine and the London School of Economics & Political Sciences and briefly worked for the King’s Fund before starting at NICE in 2004. Meindert was a member of the Board of NICE and a director of the board of the International Society for Pharmacoeconomics and Outcomes Research (2017-2020).
Sneha Dave, BS
Generation Patient, Greenwood, IN, USA
Sneha Dave graduated from Indiana University in May 2020 where she majored in chronic illness advocacy as well as journalism. She created Generation Patient and its program the Crohn’s and Colitis Young Adults Network (CCYAN) to develop support systems for adolescents and young adults with chronic conditions across the U.S. and internationally. She is proud to work with a team composed entirely of young adults with chronic conditions and also to keep Generation Patient and CCYAN independent from the pharmaceutical and insurance industry funding. Sneha has completed an undergraduate research fellowship in health policy at Harvard T.H. Chan School of Public Health. She has also interned at numerous places such as Pfizer in health economics and outcomes research for Inflammation and Immunology. Sneha has spoken on Capitol Hill, featured nationally on C-SPAN, and is a past contributor for U.S. News and World Report. She has served on the Democratic National Committee Disability Policy Subcommittee and recently joined the Midwest Comparative Effectiveness Public Advisory Council, an independent appraisal committee of the Institute for Clinical and Economic Review. She also serves on the FDA Patient Engagement Collaborative and on a grantmaking committee with the Robert Wood Johnson Foundation. Sneha was awarded two academic fellowships with the Association of Health Care Journalists. For her work, she was selected as one of the most influential teenagers in 2018 by the We Are Family Foundation and was recognized as an American Association of People with Disabilities Emerging Leader in 2020.
Ioannis Petrakis, MPharm, MSc, MPH
Takeda International, Melrose, MA, USA
Ioannis is a passionate advocate of transformative value for patients. Such breakthroughs do not come often in the pharmaceutical industry but may be easier to achieve when involving highly skilled and real-world-oriented market access, HEOR, medical and clinical research teams early into the development phases. Ioannis is a Sr. Director and R&D Global Program Leader in Gastroenterology. He has been with the company for over seven years and has served in various HEOR, Patient Access and Medical affairs roles at affiliate, regional and global level. Prior to joining Takeda he has served in various healthcare, academic, industry and health policy roles. He holds a Pharmacy Honors degree from the University of Bradford UK, an MSc in Clinical Pharmacy from the University of Cardiff UK, an MPH specializing in health Economics from the National School of Public Health GR and is trying to complete his Phd in Pharmacoepidemiology at the School of Medicine, Democritus University of Thrace GR. He is thankful for his family’s support and is a proud father of three young ones. Connect @ https://www.linkedin.com/in/ioannis-petrakis-takeda/
Disentangling the Complex Landscape of Infectious Diseases: How Can Modelers Best Choose the Level of Complexity That Increases Both Investment in Technologies and Model Interpretability?
Digital Conference Pass
ISSUE: Understanding complexity in healthcare via disease system has the potential to reduce decision and treatment uncertainty. The complexity associated with assessment of a health technology are linked via two different processes: the first through adaptive implementation of a technology having multiple targets, and the other through dynamical heterogeneity in infection transmission for which the technology is sought for. Transmission dynamics models of infectious diseases may play a greater role in addressing these aspects. However, the tendency of many health economics models of interventions is to limit focus on the ecology of the disease system. These models are deficient by not modeling the way the system reacts to the technology. Complex models may overfit the real-world data. Conversely, complexity can be important to include when uncertain factors are central to a disease process. How to choose the right level of ecological complexity for modeling an infectious disease? Should models capture how the disease system reacts to an intervention? What is needed to evaluate the quality of such complex models?
OVERVIEW: This panel will discuss the choice of modeling complexity and broadening access to the transmission dynamics models for infectious diseases—through ecological mechanisms—considering models as R&D products developed by innovators for a payer and policymaker consumers. Dr Mubayi will moderate and provide context for the discussion, outline parallels between model development and the gaps in health technologies evaluations. Dr Lima will provide insights on key ecological drivers for modeling efforts for which evidence may be readily available and types of modeling techniques, and Dr Meltzer will argue the need to address “market failures” in modeling, drawing on the experience of the economic value projects, an important step to understand the trade-off between model accuracy and interpretability. Panelists will provide brief 8-minute presentations on their perspectives, followed by moderated discussion and audience questions.
Moderators
Anuj Mubayi, PhD
IQVIA, Tempe, AZ, USA
Dr. Anuj Mubayi is an Associate Principal at IQVIA, Distinguish IBA Fellow at the Illinois State University, Honorary Fellow at the Kalam Institute of Health Technology and Adjunct faculty at the Sri Satya Sai Institute of Higher Learning. Dr Mubayi lead a team of highly qualified researchers at the interface of data science, health economics and applied mathematics. He has extensive experience in the health decision analysis and in successful development of strong modeling and simulations programs.
Panelists
Mindy Cheng, PhD
Vir Biotechnology, Alamo, CA, USA
Mindy Cheng, PhD is Director - Evidence, Value & Access at Vir Biotechnology, a company with mission to create a world without infectious disease. Prior to Vir, Mindy has worked on HEOR/Market Access in the device, diagnostics, and consulting sectors.
Mindy earned a BS in Microbiology from the University of Michigan, a MS in Healthcare Administration from the California State University East Bay, and a PhD in Pharmaceutical Outcomes Research and Policy from the University of Washington.
When not in office, Mindy enjoys mountain biking, hiking, playing soccer and baking desserts.
Martin Meltzer, PhD
Center for Disease Control and Prevention, Atlanta, GA, USA
Lead, Health Economics and Modeling Unit (HEMU), Division of Preparedness and Emerging Infections, CDC. Led modeling teams supporting CDC’s responses to: 2009 H1N1 influenza pandemic; 2012 contaminated steroid injectable product; Ebola outbreaks in West Africa (2014-16) and the Democratic Republic of the Congo (2018-19); Zika epidemic (2016-17); Current COVID Response. Research includes: Responses to bioterrorist weapons; Economics of controlling diseases such as rabies, RSV, Japanese encephalitis, dengue, hepatitis A, meningitis, Lyme, and malaria. Published ~ 300 publications, including > 150 peer-reviewed papers + > 60 tools. Associate editor for Emerging Infectious Diseases.
Pedro Nascimento de Lima, PhD
Rand Corporation, Wake Forest, NC, USA
Pedro Nascimento de Lima is an associate engineer at the RAND Corporation. His research leverages simulation modeling, high-performance computing, and Decision Making Under Deep Uncertainty methods to inform health policy decision-analytic problems.
Prior to joining Pardee RAND, he was a lecturer at UNISINOS, where he taught simulation modeling and other quantitative courses. Both his undergraduate and master’s dissertations, focusing on wicked problems and decision under deep uncertainty, have received the Best Brazilian Dissertation in Production Engineering Prize from ABEPRO.
He has a B.S. and an M.S. in production engineering from UNISINOS University in Brazil, and a Ph.D. in policy analysis from Pardee RAND Graduate School.
Podium Session
Economic Considerations of Digital Health Technologies
Increasing use of digital health technologies has the potential to improve quality, efficiency, and access to care. However, their financial impact should be rigorously evaluated to inform value in care. This session features studies assessing economic implications of digital health technologies in applications to orthopedic and trauma care, chronic obstructive pulmonary disease, stroke rehabilitation, and chronic disease management. Seating is limited.
Moderator
Xiao Xu, PhD
Yale University, New Haven, CT, USA
Xiao Xu, PhD, is an associate professor in the Department of Obstetrics, Gynecology and Reproductive Sciences at Yale School of Medicine. As a health economist and health services researcher, her work focuses on identifying factors that influence the quality and utilization of women’s health services. Her recent studies examined hospital and regional variation in healthcare delivery, costs, and patient outcomes; comparative effectiveness and cost effectiveness of health interventions; and health and healthcare disparities. Her research has been funded by the National Institutes of Health, Agency for Healthcare Research and Quality, and various research foundations.
P35: Economic Benefit of Stroke Technology-Assisted Rehabilitation (STAR): A Scoping Review
2:00PM - 2:15PM
Samkharadze T1, Cheng HJ2, Kager S2, Chua KSG3, Lambercy O4, Wenderoth N4
1Singapore-ETH Centre, Singapore, 05, Singapore, 2Singapore-ETH Centre, Singapore, Singapore, 3Tan Tock Seng Hospital Rehabilitation Centre, Singapore, Singapore, 4ETH Zürich, Zürich, Switzerland
OBJECTIVES:
High-intensity rehabilitation is an evidence-based effective treatment for reducing disability and improving the quality of life in stroke survivors with sensorimotor impairments. Increasing healthcare costs, rising demand for rehabilitative treatment, and a shortage of healthcare professionals have motivated the development of new technologies to support high-intensity stroke rehabilitation more effectively. However, relatively little data is available regarding the economic benefits of these new approaches, including virtual reality, exoskeletons, robots, and wearable sensors. This study aimed to review existing evidence and methodologies of health-economic evaluation for stroke technology-assisted rehabilitation (STAR) in different settings (laboratory, hospital, home). METHODS:
A systematic search in seven electronic bibliographic databases (until 12 September 2022) identified 3,153 records. Following our in/exclusion criteria, 2,054 records were excluded. After removing duplicates, 1,017 entries were screened for eligibility, and 21 studies were considered for quality appraisal. Additional 18 studies were identified through the citation search. In total, 39 studies with STAR-related costs were evaluated.
RESULTS:
Frequency and type of STAR-related cost analysis differed between settings. Firstly, in laboratory settings, out of 22 studies,
only one performed a formal STAR-related cost analysis which was
prone to comparator bias. Secondly, in hospital settings, nine studies
performed cost analysis, and the majority (6) estimated STAR to be cheaper than
conventional therapy (CT). Thereof, five studies justified STAR’s implementation to save therapists’ time, and only one demonstrated the superiority of STAR in both clinical and economic outcomes. Lastly, in home settings, four studies evaluated STAR-related costs, of which two found that STAR could be economically beneficial.
CONCLUSIONS:
Available studies suggest that STAR can generate economic benefits in comparison to CT. However, the current evidence is sparse, and the applied methodologies are highly variable. To increase the comparability and generalizability of future results, more studies are needed performing early
health technology assessment and adhering to reporting guidelines.
P34: Cost Analysis of Health Care Resource Utilization Pre and Post Initiation of a Remote Physiologic Monitoring Service
1:45PM - 2:00PM
Moraveji N1, Hendricks AC1, Teresi RK1, Polsky M2, Maselli DJ3, Murray R1
1Spire Health, San Francisco, CA, USA, 2Pulmonary Associates of Richmond, Richmond, VA, USA, 3UT Health, San Antonio, TX, USA
OBJECTIVES: A remote physiologic monitoring (RPM) service that uses cardiorespiratory sensors for earlier identification of acute exacerbations of chronic obstructive pulmonary disease (COPD) has been associated with reduced healthcare resource utilization (HCRU). This economic analysis estimates the potential associated cost impact.
METHODS: In a real-world retrospective study of the RPM intervention in 126 COPD patients, HCRU from electronic medical record encounter data during the 12 months prior to RPM was compared to the 12 months post-initiation (results reported separately). Patients were included if they were enrolled on RPM for one full year and experienced at least one unplanned hospitalization or ER visit in the year prior. In this cost analysis, HCRU units were multiplied by their estimated costs, which were based on published claims data in a COPD population. Cost of the RPM intervention was based on CMS reimbursement rates. Dollar values were adjusted to 2022 values using the medical care component of the Consumer Price Index. Total and average per patient costs for the 12 months pre and post RPM intervention were compared.
RESULTS: Among the 126 COPD patients studied (age: 73.8 (9.3); 42.9% Male; FEV1: 59.5% (21.3%)), the total estimated cost of outpatient visits increased from the pre to post RPM intervention period ($69,399 vs $78,531), while the cost of ER visits ($105,774 vs $58,956) and hospitalizations ($3,141,821 vs $1,100,784) decreased. The cost of RPM in the post intervention period was $180,643 ($1,434 per patient). Total HCRU costs decreased 57.2% ($15,064 per patient) from $3,316,994 ($26,325 per patient) to $1,418,914 ($11,261 per patient).
CONCLUSIONS: Implementation of a cardiorespiratory RPM service in a real world study of COPD patients was associated with a net reduction in estimated healthcare resource expenditures.
P36: Navigating the Digital Health Landscape for Chronic Disease Management: A Value Assessment Framework for Patient-Facing Technologies
2:15PM - 2:30PM
Haig M, Main C, Chavez D, Kanavos P
London School of Economics and Political Science, London, UK
OBJECTIVES: Digital health technologies (DHTs) have the potential to revolutionize the healthcare industry by optimizing costs and improving the quality and efficiency of care. However, the rapid pace of innovation in the field and the lack of consistent evidence standards make it challenging for decision-makers to effectively evaluate these technologies. To address this challenge, we propose a value framework for assessing DHTs that are used to manage chronic diseases.
METHODS: This framework is based on a Delphi exercise eliciting the preferences of key stakeholders, including patients, physicians, industry representatives, academics and policymakers. Our methodology includes a literature review and primary data collection involving 129 participants from the United States, United Kingdom, and Germany. The data collected was analyzed using STATA to determine inter-group differences and levels of consensus.
RESULTS: The framework comprises of 33 stable indicators with consensus across six value domains, all rated ‘very important” or ‘important.’ While there was a lack of consensus among stakeholders on certain important aspects such as value-based care models, sustainable systems, and stakeholder engagement, this lack of consensus was primarily due to high rates of neutrality rather than negative evaluations. Supply-side actors and academic experts were the most unstable stakeholder groups and statistically significant disagreement was found between country groups for two indicators.
CONCLUSIONS: Participants suggested indicators that assess aspects of value for DHTs that go not only beyond traditional HTA indicators, but also beyond those included in the study countries’ frameworks. Stakeholders across country groups formed consensus around the importance of data ownership, the ability for patients to input data, and the need for clearly communicated policies. This study reveals a need for a combined regulatory and HTA policy response that updates laws to meet technological innovations, offers a pragmatic approach to evidence standards to assess DHTs, and involves stakeholders to understand and meet their needs.
Patient and Community Preferences for Decision Making in Healthcare
This session highlights discrete choice experiments conducted to elicit preferences for medical treatment and healthcare decisions around the globe. Seating is limited.
Moderator
Alejandro Amill-Rosario, PhD, MPH
University of Maryland School of Pharmacy, Baltimore, MD, USA
Dr. Amill-Rosario is a research associate at the Department of Practice, Sciences, and Health Outcomes Research (P-SHOR) and PAVE. In this role, he also serves as director of health services research, leading research projects focusing on access, quality, and cost of telehealth care and patient-level health technology assessments. Dr. Amill-Rosario received a doctoral degree in health policy and administration from The Pennsylvania State University and a master's in public health from the University of Puerto Rico. He completed a postdoctoral fellowship in patient-centered outcomes research at PAVE. Before his PhD training, he worked as a study coordinator of the Adult Spectrum of Disease project from the Centers for Disease Control and Prevention at the Universidad Central del Caribe School of Medicine and as a biostatistician of the Puerto Rico Asthma Project and the PR-BRFSS at the Puerto Rico Department of Health. As a PhD student, he worked on two major research projects at the Center for Health Care Policy Research funded by the Robert Wood Johnson Foundation (Aligning Forces for Quality Evaluation) and the Agency for Healthcare Research and Quality (RAND Center of Excellence for Health System Performance).
Dr. Amill-Rosario's research interests include health services and health policy research, health economics, patient-reported outcomes, real-world data, health information technology, and qualitative research methods. His work focuses on assessing the impact of telepsychiatry services on children and adolescents and patients' informed outcomes. His research also examines telepsychiatry, reimbursement policy, and patient value in telehealth care modalities. Dr. Amill-Rosario applies stated preference methods to elicit the patient's value of treatment and outcomes.
P48: Oncologists’ and Urologists’ Preferences for First-Line (1L) Treatment of Locally Advanced/Unresectable Metastatic Urothelial Carcinoma (AUC): A Discrete Choice Experiment (DCE) Conducted in 5 European Countries
2:00PM - 2:15PM
Panattoni L1, Kearney M2, Land N3, Flottemesch T4, Sullivan P4, Kirker M5, Bharmal M6, Hauber B5
1Precision Value and Health, Seattle, WA, USA, 2the healthcare business of Merck KGaA, Darmstadt, HE, Germany, 3Precision Value and Health, Livermore, CA, USA, 4Precision Value and Health, New York, NY, USA, 5Pfizer, New York, NY, USA, 6EMD Serono, Rockland, MA, USA
OBJECTIVES: The treatment landscape for people with aUC is currently evolving. Treatment choice involves trade-offs between efficacy, safety, and administration schedule. This study aimed to quantify preferences for 1L treatment attributes and treatment profiles among physicians who treat patients with aUC using a DCE in 5 European countries (France, Germany, Italy, Spain, and UK).
METHODS: During August-September 2022, 498 oncologists and urologists completed an online survey including 12 treatment choice tasks, each comparing 2 hypothetical therapy choices based on treatments for aUC that varied in 5 treatment attributes: grade 3/4 treatment-related adverse events (TRAEs), 1L induction and 1L maintenance administration schedule, time to disease progression, and overall survival (OS). A mixed-effects logit model was used to estimate preference weights and compute the relative importance (RI) of each attribute. Preference shares were calculated for 4 treatment profiles and stratified by country.
RESULTS: The study included 343 oncologists (69%) and 155 urologists (31%). Most were male (72%), were in practice for >10 years (69%), and treated 5-19 patients with aUC per month (58%). OS had the strongest influence on physicians’ preferences (RI=62%), followed by frequency of grade 3/4 TRAEs (RI=27%). The remaining 3 attributes had a combined RI of 11%. Among the hypothetical treatment profiles, a sequential induction + maintenance therapy (chemotherapy followed by immune checkpoint inhibitor [ICI] maintenance therapy after disease control) profile had the largest preference share (51%), followed by ICI monotherapy (29%), ICI combination therapy (ICI + chemotherapy followed by ICI after disease control; 12%), and chemotherapy alone (9%). Across the 5 countries, the preference share for a sequential induction + ICI maintenance therapy profile ranged from 38% (Italy) to 56% (UK).
CONCLUSIONS: OS was the dominant attribute driving physicians’ choice of 1L therapy, followed by TRAEs. Future 1L therapies for aUC will need to provide substantial OS benefit to influence prescribing preferences.
P45: Preferences for Long-Term Care Among Older Adults in China: A Discrete Choice Experiment
2:15PM - 2:30PM
Leng A1, Maitland E2, Nicholas S3, Wang J4
1Shandong University, Qingdao, Australia, 2University of Liverpool, Liverpool, Australia, 3University of Newcastle, Newcastle, NSW, Australia, 4Wuhan University, Wuhan, China
OBJECTIVES: We examine the preferences for long-term care among older adults in China.
METHODS: Data on 2035 over 50-year-old adults’ long-term care preferences were collected by discrete choice experiment conducted across 12 Chinese provinces selected by the stratified random sampling method in August 2022. Each scenario described four attributes: forms of long-term care, contents of long-term care, quality of life and out-of-pocket payments. Preferences were derived using a mixed logit model and latent class model.
RESULTS: All four attributes significantly influenced older adults’ decision for long-term care, with quality of life was the most important attribute, followed by the forms of long-term care. Older adults were willing to pay US$418 per month (95%CI US$389-US$450) to improve quality of life from a bad level to a good level. Compared with home-based self-directed care, older adults were less willing to pay US$68 per month (95%CI US$41-US$94) for home-based formal care, US$52 per month (95%CI US$27-US$77) for community-based formal care, US$182 per month (95%CI US$153-US$211) for nursing-institutions-based formal care and US$128 per month (95%CI US$102-US$153) for medical-institutions-based formal care. Older adults who lived in urban areas and had better knowledge of the long-term care insurance system displayed a higher probability to choose long-term care. Older adults who had more children and a stronger traditional cultural concept were less likely to prefer long-term care with formal care in institutions.
CONCLUSIONS: Quality of life obtained in long-term care is what older adults preferred. Home and basic living care provided by caregivers was still the preferred option for a majority of seniors. This suggests that we should not only strengthen the training of professional caregivers to improve the quality of basic life care services, but also strengthen the training of non-professional caregivers, such as family members.
P47: A Discrete Choice Experiment to Assess Patient-Informed Preferences for Major Depressive Disorder Treatment By Depression Severity
1:45PM - 2:00PM
Amill-Rosario A1, Huang PL1, Xie R2, Chapman R3, Slejko JF1, dosReis S1
1University of Maryland School of Pharmacy, Baltimore, MD, USA, 2Innovation and Value Initiative, Newton, MA, USA, 3Innovation and Value Initiative, Alexandria, VA, USA
OBJECTIVES: Therapeutic options for major depression disorder (MDD) carry different benefits and risks, and some may be more and others less preferred by patients. We quantified the preferences for patient-informed attributes of MDD treatment and the relative attribute importance, overall and by severity subgroups.
METHODS: Cross-sectional survey of US adults 18 and older with MDD measured preferences with a discrete-choice experiment (DCE) that included 6 experimentally-derived choice tasks. Each choice task displayed 3 profiles with 6 attributes (treatment modality, time-to-treatment effect, days hopeful, improvement of productivity, relationship with others, and out-of-pocket costs), with one of three attribute levels in each profile. Conditional logit models estimated the part-worth preference weights. We calculated relative attribute importance as the proportion each attribute contributes to the sum of attribute level min-max difference. The analysis was also replicated by severity subgroups defined by PHQ-9 (none-mild, moderate, severe).
RESULTS: Our sample (N=300) comprised 82 individuals with none-mild depression, 84 moderate, and 132 severe. Overall, about 40% were 30-49 years-old, 70% female, 60% white, 13% black, and 16% Hispanic. About 40% had lived with depression less than 10 years, 40% had been hospitalized for depression, and 80% were using medication. Overall, medicine alone (-0.2) was the least preferred treatment mode, as was 9 weeks to treatment effect (-0.9). Six days per week hopeful (0.4) was preferred to 2 days per week (-0.4). Monthly out-of-pocket costs of $30 (0.6) and $90 (0.4) were preferred to $270 (-0.8). Out-of-pocket cost (29.8%), relationships (27.0%), and days hopeful (18.0 %) explained 74% of overall relative attribute importance. Results were similar by depression severity subgroup.
CONCLUSIONS: Relationships and hopefulness are preferred attributes over productivity and treatment effects. These findings can be used to inform patient-centric elements for inclusion in economic evaluation.
2:45 PM - 3:15 PM
PM Coffee Break
Located in the Exhibit Hall
3:00 PM - 3:30 PM
Member Group Meeting
New Professionals Meet Up Meeting
3:00 PM - 4:00 PM
Precision Medicine and Advanced Therapies Special Interest Group Open Meeting
3:15 PM - 3:45 PM
Exhibit Hall Theater
Exploring Opportunities for RWE Generation Within a Hybrid Data Ecosystem
Digital Conference Pass
Healthcare claims are an important data source for health economics, outcomes, and comparative effectiveness research. Although researchers often rely on closed claims data sources (i.e., data sourced directly from a healthcare payer), open claims data sources (i.e., data sourced from clearinghouses and pharmacy systems) can provide insight and visibility into patient, provider, and payer characteristics that are not always identifiable in a closed system. Data ecosystems that include both closed and open claims data linked at the patient level create opportunities to expand the types of questions for which claims data are fit-for-purpose and mitigate limitations that come from using a closed or open claims data source alone.
In this theater presentation we will examine the strengths and limitations of closed and open claims data sources and explore RWE opportunities that exist in a hybrid claims data ecosystem.
Seating is limited.
Sponsor: Forian, Inc
Sponsor
Forian, Inc
Moderators
Paul Reuscher, MSc
Forian, Inc, Newtown, PA, USA
Paul Reuscher joined Forian as a founding team member and he currently oversees all aspects of commercial, data engineering, and product delivery functions. Paul is a subject matter expert on healthcare data, data privacy, and statistical methodology. Prior to Forian, Paul was a VP at DRG helping to lead the Data and Analytics business unit. His previous roles have included work in HEOR and RWE consulting, providing predictive analytics support for leading health networks and investment groups. Paul has a graduate education in Applied Mathematics and Economics from the University of Cincinnati.
Speakers
Seth Kuranz, PhD
Forian, Inc, Boston, MA, USA
Seth Kuranz joined Forian in 2022 to support the generation of RWE using CHRONOS, Forian’s RWD ecosystem. Before joining Forian, Seth worked in non-profit, academic, and private sectors and has over 10 years of experience applying qualitative and quantitative research methods to complex clinical and public health-related questions. Research interests include epidemiologic forecasting; health outcomes research in oncology, cardio-renal-metabolic disorders, rare and infectious diseases; and social determinants of health. Seth graduated from Boston University with a Ph.D. in epidemiology.
3:15 PM - 4:00 PM
Poster Tours
Real-World Evidence Poster Tour
Posters featured in this tour: RWD128: A Retrospective Claims Analysis Characterizing Health Care Resource Use Among Patients with Friedreich Ataxia in the United States RWD133: Clinical Trial Emulation of the Catt Using Data from the Iris® Registry (INTELLIGENT RESEARCH IN SIGHT) Using Exact Matching and Inverse Probability Weighting EPH164: Atrial Fibrillation Is Associated with Higher Incidence of Frailty in Older Adults: The Atherosclerosis Risk in Communities (ARIC) Study EPH165: Geographic Variation of Antidementia Medication Use Among U.S. Nursing Home Residents with Dementia HSD76: Investigating the Impact of Self-Help Groups on Opioid Use Disorder Treatment Retention Lift Off: From Real World Data Quality Guidelines to Actionable Assessment
Medical Devices Poster Tour
Posters featured in this tour: MT39: Incremental Costs of New Permanent Pacemaker Implantation after Transcatheter Aortic Valve Replacement (TAVR) HSD89: The Patient Journey Prior to Neuromodulation in Drug-Resistant Epilepsy, Patterns of Utilization and Cost of Healthcare Services and Pharmacotherapy Based on a Claim Database in the United States MT41: Medical Technology Guidance: A Review to Identify the Evidence Gaps and Data Needs MT42: HTA Landscape of Medical Devices in Canada EE439: Prevention of Needlestick Injuries and Blood Exposures Among Kenyan Healthcare Workers Using Safety Engineered Devices EE440: Trends in the Cost of Total Knee Arthroplasty — a US Hospital Database Analysis, 2000–2021
3:15 PM - 4:15 PM
Discussion Groups
The Reality of Target Trial Emulation for Medical Decision-Making and HTA Recommendations - Is the Gap Between Academic and HTA Applications Too Wide?
Moderator
Kristian Thorlund, MSc, PhD
Cytel, 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 statistical methodologies that are now being used widely
OPUF (Online Elicitation of Personal Utility Functions): Understanding What Brings Value to the Individual
Speaker
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 in health care; 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 >150 original peer reviewed journal papers on a wide range of empirical and theoretical topics in health economics.
Educational Symposia
Driving Changes in Obesity Care: A Multi-Stakeholder Perspective on the Value of Non-Invasive Interventions
Digital Conference Pass
Obesity is a serious chronic and complex disease that poses the largest health impact to the US society, and it is projected that half of American adults will suffer from obesity in 2030. Obesity is by far the greatest contributor to the burden of chronic diseases in the US, and by some estimates accounting to nearly half of total costs of chronic disease. Obesity is linked to various complications such as type 2 diabetes, cardiovascular disease, nonalcoholic fatty liver disease and many more and is associated with reduced life expectancy. Not only does obesity have a large direct health care burden, but individuals suffering from obesity are often young and within the working age and estimates suggests that more that the indirect costs of obesity are higher than $1 trillion in productivity losses, yet access to effective treatments are limited. The symposium aims to explore the value of weight management programs including non-invasive interventions and assess the obstacles and barriers for achieving the optimal program outcomes. With perspectives from patients, employers, payers and health systems, this session will explore the importance of establishing broad access to sustainable weight management solutions.
Sponsor: Novo Nordisk A/S
Sponsor
Novo Nordisk A/S
Moderators
Sean Sullivan, BScPharm, PhD
Choice Institute, School of Pharmacy, University of Washington, Seattle, WA, USA
Sean D. Sullivan, BScPharm, MSc, PhD, is Professor, School of Pharmacy, University of Washington and Visiting Professor, London School of Economics. He holds a joint appointment in the School of Public Health. He holds adjunct appointments in the School of Medicine and the Public Health Sciences Division at the Fred Hutchinson Cancer Research Center.
He completed training in pharmacy at Oregon State University in 1983, obtained a master’s degree at the University of Texas in 1986 and a PhD in health economics and policy at the University of California, Berkeley in 1992.
Dr. Sullivan was a member of the Medicare Evidence Development and Coverage Advisory Committee, a member of the Regence Blue Shield and Premera Blue Cross P/T Committee, and Chair of the Premera Blue Cross Value Assessment Committee. He was awarded the 2014 Stephen G. Avey Lifetime Achievement Award from the Academy of Managed Care Pharmacy (AMCP), the 2015 APhA Academy of Pharmaceutical Research Sciences (APRS) Research Achievement Award, and was elected to the National Academy of Medicine in 2020.
Speakers
Angela Fitch, MD, FACP, FOMA
knownwell, Boston, MA, USA
David Skomo, BSc Pharm
WellDyne, Pittsburgh, PA, USA
Estimating QALYs for Transformative Therapies for Severe Pediatric Rare Diseases
Digital Conference Pass
The objective of this symposium is to discuss estimation issues for QALYs in assessing the value of transformative therapies, with a particular focus on severe, pediatric rare diseases. Firstly the symposium will compare QALY estimates for transformative therapies versus conventional medicines. We will then discuss key methodological challenges in eliciting utility values for rare diseases, including use of trial-based and vignette approaches as well as sourcing values for analogous diseases. Subsequently the panelists will discuss measurement challenges associated with capturing the multidimensional aspects of treatment (e.g. cognitive benefit in additional to motor and respiratory function) from various perspectives such those of the caregiver and parents. The session will conclude with a summary of best practices, and recommendations for health technology assessment agencies and researchers.
Sponsor: Orchard Therapeutics
Sponsor
Orchard Therapeutics
Moderators
Peter Neumann, ScD
Tufts University, 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.
Speakers
Josh Cohen, PhD
Tufts Medical Center, Boston, MA, USA
Joshua Cohen, PhD, is the Deputy Director and Chief Science Officer of the Center for the Evaluation of Value and Risk in Health at the Tufts Medical Center Institute for Clinical Research and Health Policy Studies, and a Research Associate Professor of Medicine at Tufts School of Medicine. Dr. Cohen’s work focuses on the advent of “drug value frameworks”, with a special focus on the Institute for Clinical and Economic Review (ICER) framework, promotion of "open source" practices for health economic simulation models, and the potential benefits of risk-targeted disease screening.
Josie Godfrey, MA
JG Zebra Consulting, Folkestone, Kent, United Kingdom
Josie has worked in rare diseases and innovative therapies for over 12 years. She currently runs a consultancy business specialising in strategic market access, policy and stakeholder engagement. She is the Strategic Director for Duchenne UK’s Project HERCULES, an award-winning global collaboration developing evidence and tools to support HTAs for new treatments for Duchenne Muscular Dystrophy. Josie is also co-founder and joint CEO of Realise Advocacy, which supports patient involvement in drug development and access processes. She previously led work at NICE to establish the Highly Specialised Technologies programme.
Andrew Lloyd, DPhil
Acaster Lloyd Consulting Ltd, Oxford, Oxfordshire, United Kingdom
Jonathan Toft, PharmD, MBA
MedImpact, San Diego, USA
ISPOR Forums
HEOR Workforce Diversity and Health Disparity Research: An ISPOR-Led Collaboration Between Industry and Academia
This Forum will highlight the ongoing activities to build diversity, equity and inclusion (DE&I) within ISPOR with an aspiration to have a well-represented HEOR workforce in the future. This initiative involves a cross-functional collaboration between ISPOR’s Institutional Council, Student Network, Regional Chapters, and Faculty Advisor Council to leverage resources and connections to scale-up DE&I initiatives. The session will begin with ISPOR’s commitment to understand diversity within its membership. Presentations will be followed on initiatives to help increase student, early professionals, and faculty awareness regarding the HEOR field and diverse mentoring opportunities for career guidance. ISPOR’s health equity research initiatives through a newly formed Special Interest Group (SIG) will also be spotlighted. The session will end with a call to action for members to replicate initiatives within their organization and a description of future areas of focus for ISPOR’s DE&I efforts.
Moderators
Hemant Phatak, PhD
Accleron Pharma, Needham, MA, USA
Speakers
Stacey Kowal, BS, MS, MSc
Genentech, Alameda, CA, USA
Stacey Kowal currently serves as a principal researcher in Genentech’s Health Policy and Systems Research team where her work focuses on identifying and testing new methods to assess the impact of new healthcare technologies. Her background includes both US and global work to inform health technology assessment and reimbursement as well as design of public health interventions, drawing on disciplines of applied mathematics, economics, and international health policy. Stacey’s current research aims to increase focus on equity effects in health economics and outcomes research (HEOR), including integration of distributional elements into healthcare decision-making. Stacey is a Udall scholar, a Truman scholar and a Marshall scholar. She holds a BS in Mathematics from Alma College, an MSc in public health from the London School of Hygiene and Tropical Medicine and an MSc in International Health Policy and Health Economics from the London School of Economics.
Tyler Wagner, PharmD
Virginia Commonwealth University School of Pharmacy, Richmond, VA, USA
Tyler D. Wagner, Pharm.D., is a Ph.D. candidate at the Virginia Commonwealth University School of Pharmacy in the Department of Pharmacotherapy and Outcomes Science. He received his B.S. in pharmaceutical sciences from the University of South Carolina in 2017 and his Pharm.D. from the University of South Carolina College of Pharmacy in 2019. Wagner’s dissertation, funded by an American Foundation of Pharmacy Education fellowship, focuses on the development and validation of predictive risk models utilizing machine learning algorithms, the identification of disparities in primary care clinical outcomes, and pharmacoeconomic modeling of pharmacist-led services. Through this research, Wagner hopes to further expand physician-pharmacist collaborations in primary care and ultimately address outcome disparities in health care. His primary research interests pertain to health services research, primary care, economic modeling, and the role of artificial intelligence and machine learning in health care. Currently, Wagner is serving as their 2022-23 ISPOR Student Network chair. In this position, he manages the work of seven committees and provides support for over 140 international chapters, works with graduate students and HEOR leaders to communicate outcomes research and organizational happenings to students and professionals across the globe, and serves on the ISPOR HEOR Workforce Diversity Initiative and Faculty Advisory Council. Wagner is also the incoming chair for the American Pharmacists Association (APhA) Postgraduate Advisory Committee, set to serve from 2023-25. Wagner is a proponent of servant leadership and is committed to advancing pharmacy practice on a global level.
I received my Pharm.D. degree from the University of South Carolina where I had a great opportunity to work in drug information at an academic medical center and conduct research in infectious diseases, hypertension, and diabetes. Through extensive conversations with mentors, I became interested in health outcomes and the role of data within medicine. This led me to pursue my Ph.D. at VCU and work with their Center for Pharmacy Practice Innovation. My research interests include health care services — specifically evaluating the role of the pharmacist — primary care, digital health, and machine learning. My dissertation involves analyzing electronic health records from a regional health system's outpatient clinics to assess the impact of a multidisciplinary primary care model on quality measure achievement and economic outcomes and identification of patients at risk of not meeting quality measures or having high health care utilization using predictive analytics.
Annesha White, PharmD, MS, PhD
University of North Texas Health Sciences Center, Fort Worth, TX, USA
Annesha White, PharmD, MS, PhD is a Senior Associate Dean for Assessment and Associate Professor at the University of North Texas Health Science Center College of Pharmacy. Dr. White has worked on funded projects with the Florida Medicaid program including studies on COX-2 Inhibitors, Hepatitis C, and End Stage Renal Disease. She also worked at the Government Accountability Office (the investigative arm of Congress), to examine Pharmacy Benefit Managers impact on Health Plans, Enrollees, and Pharmacies. Dr. White's primary research interests include the design of studies to address issues in the health services research arena. Areas of focus include Medicare, Geriatrics, Managed Care, Pharmacoeconomics, and Outcomes Research. Her research has included a focus on a variety of disease states, such as heart disease, chronic pain, and end stage renal disease with the goal of providing care that is balanced in quality and cost. Her research involves a team approach to care examining the various aspects of the health care system and how entities can join together to enhance efforts. Dr. White has published many peer-reviewed articles, a textbook entitled Introduction to the Pharmacy Profession and serves as a referee for journals such as the Journal of Managed Care Pharmacy.
Underestimated: Identifying and Addressing Mechanisms of Bias in HEOR (An ISPOR Women in HEOR Forum)
Purpose: ISPOR’s Women in Health Economics and Outcomes Research (HEOR) Initiative will address the topic of bias in the conduct and dissemination of HEOR.
Description: A recent focus on the topic of health equity has highlighted existing inequities in the delivery of healthcare through numerous mechanisms. Some of these stem from the research that informs medical decision making, including the way data are collected and the methods used. This panel will discuss potential mechanisms for bias in HEOR and where HEOR methods are more or less susceptible to potentiating bias. This includes some of the potential shortcomings of data sources, such as real-world data with many characteristics under-represented, which may have inherent bias. Additionally, the panel will consider other mechanisms, such as whether existing clinician-reported and patient-reported outcome measures account for diversity. This includes data sources and processes but can also include the composition of research teams. The panel also will discuss opportunities to identify and address bias with a goal of improving the evidence base for healthcare decisions.
Julia Slejko will introduce the session, including the objectives of the Women in HEOR initiative. Shelby Reed will moderate a discussion among panelists who will speak to the potential mechanisms for bias in the conduct and dissemination of research and reflect on where HEOR has been more and less successful. Davene Wright will discuss appropriate techniques for communicating research results in a way that respects diverse perspectives and novel ways to disseminate research to reach diverse audiences. She will also discuss whether researchers should and how researchers can manifest their personal identities in research dissemination. Serena Guo will discuss potential mechanisms for bias in the conduct of HEOR, such as fairness and bias in machine learning algorithms and approaches to assess and mitigate bias.
Moderators
Shelby Reed, PhD, RPh
Duke Clinical Research Institute, 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 and Therapeutic Area leader for Population Health Sciences at the Duke Clinical Research Institute. She also is core faculty at the Duke-Margolis Center for Health Policy. Dr. Reed has over 20 years of experience in economic evaluation, health services research and health policy. Her research portfolio includes a broad array of trial‐based and model‐based cost‐effectiveness analyses of new and existing medical diagnostics, drugs, devices and patient‐centered interventions. For the past several years, she has increasingly dedicated her efforts to the field of stated‐preference research. In 2016, she co-founded the Preference Evaluation Research (PrefER) Group at the DCRI, and she currently serves as its director. She and the group are frequently sought to conduct stated-preference studies to inform regulatory decisions, health policy, care delivery, value assessment and clinical decision making with applied projects spanning a wide range of therapeutic areas.
Dr. Reed has published more than 200 manuscripts in peer‐reviewed journals. She was the first recipient of ISPOR's Bernie O’Brien New Investigator Award in 2005. She served on two ISPOR Task Force groups to develop recommendations for conducting economic evaluations alongside clinical trials and recommendations to address transferability of multinational economic evaluations. She recently served as a guest editor for a themed issue in Value in Health on Patient‐Focused Benefit‐Risk Analysis to Support Regulatory Decision‐Making. She served on the editorial boards for Health Services Research (2016-2020) and Value in Health (2013-2021). She served as President for ISPOR in 2017-2018, and she currently is Past-Chair of the Society’s Health Science Policy Council.
Speakers
Serena Jingchuan Guo, MD, PhD
University of Florida College of Pharmacy, Gainesville, FL, USA
Dr. Serena Jingchuan Guo is an Assistant Professor in the Department of Pharmaceutical Outcomes and Policy at the University of Florida. She conducts pharmaceutical outcomes research with a focus on improving health equity both in treatment use and outcomes of diabetes, cardiovascular disease, and neurodegenerative disorders. Her research draws on real-world data (e.g., electronic health records and insurance claims data) and AI/machine learning to 1) identify heterogeneous treatment effects (HTEs) of pharmaceuticals to promote equitable and precision patient care, and 2) develop individualized and intelligent social risk management tools to be integrated into clinical care. Her research programs and collaborations have been funded by the NIH, CDC, FDA, Veterans Affairs, and the PhRMA Foundation.
Julia F. Slejko, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
Julia F. Slejko, PhD is an Associate Professor of Pharmaceutical Health Services 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-lead of ISPOR’s Women in HEOR initiative, Co-Chair Elect of the ISPOR Faculty Advisor Council and is a member of ISPOR’s Health Science Policy Council.
Davene Wright, PhD
Harvard Pilgrim Health Care Institute and Harvard Medical School, Boston, MA, USA
Davene Wright is an Assistant Professor in the Department of Population Medicine, a research and academic partnership between the Harvard Pilgrim Health Care Institute and Harvard Medical School. There, she is the Associate Director of the Division of Child Health Research and Policy.
The overarching goal of her research is to improve the demand for and supply of effective and efficient health care for patients with chronic diseases with a focus on obesity and diabetes. She specializes in leading interdisciplinary studies that link a variety of decision sciences methods and related techniques including stated preference elicitation, economic evaluation, simulation modeling, health services research methods, and qualitative research.
In her free time, she enjoys sewing, exploring libraries, following Formula 1 racing, and competitive storytelling.
Advancing the Science, Policy, and Practice of Nutrition Economics
The Nutrition Economics Special Interest Group is dedicated to health economic evaluations of Medical Food (MF) and Food for Special Medical Purposes (FSMP) and developing scientific methodologies and approaches for studying nutrition economics more broadly. Over the past several years, the SIG has completed two collaborative papers: the first was a scoping review of medical nutrition terminology and regulations in Europe and the United States, which found extensive inconsistencies across countries in terminology used and a dearth of economic evaluations to inform the adoption of FSMP. The second paper took a broad look at coverage and reimbursement policies for FSMP across 14 countries in four continents. Despite the importance of medical nutrition for the management of malnutrition and nutrition-related disorders and conditions, substantial variation exists in the regulatory policies government reimbursement and coverage across the world. The Nutrition Economics Special Interest Group has identified four areas for research and policy: (1) inequities in reimbursement across settings, particularly in the outpatient and community settings; (2) inconsistencies in the medical conditions eligible for reimbursement across countries and settings; (3) the disconnect between clinical guidelines and reimbursement; and (4) lack of health technology assessment to inform coverage and reimbursement decisions
This Forum session will delve into the high-priority areas for research and policy and highlight the SIG’s new key project, Role of Nutrition in Older Adults: A Systematic Literature Review of Health Economic Evaluation Studies. Additionally, the Forum will discuss opportunities for future collaborations related to the economics of medical nutrition and non-medical nutrition to improve societal health. The session will wrap up with a discussion about other key SIG activities for the coming year.
Moderators
Tricia Johnson, PhD
Rush University, Oak Park, IL, USA
Tricia Johnson is Professor and Economist in the Department of Health Systems Management and co-leads the Health Equity Research Group at Rush University in Chicago, USA. She is one of the few human milk economists in the world, and her current research evaluates the short and long-term economic consequences of breastmilk and nutrition strategies for premature infants. She is Multiple Principal Investigator of a randomized controlled trial that tests an economic intervention aimed at reducing racial and ethnic gaps in the provision of mother’s own milk for very preterm infants in the neonatal intensive care unit. Dr. Johnson is Chairperson of ISPOR’s Nutrition Economics Special Interest Group.
Speakers
Amarsinh M Desai, PhD, MS, B.Pharm, D.Pharm
Nestle Health Science, Bridgewater, NJ, USA
Amarsinh Desai, PhD is an Health Economist and presently US Market Access Lead with Nestle Health Science. In his current role, he leads value demonstration, evidence generation and HEOR activities for Nestle medical nutrition products and programs.
Before joining Nestle, Dr. Desai was working as an Associate Health Economist at ICON plc, a clinical research organization, where he supported Global Health Technology Assessment – Health Economics, Reimbursement & Outcomes (GHTA – HERO) team with health economics projects.
He graduated with PhD in Health Outcomes from University of Cincinnati, Master of Science in Drug Regulatory Affairs, and has background in Pharmacy. His expertise and experience include conduct of observational studies with variety of data sources, health economic evaluations and evidence synthesis in wide variety of therapeutic areas.
Amarsinh Desai held positions such as President for International Society for Pharmacoeconomics & Outcomes Research (ISPOR) and Vice-President for International Society for Pharmacoepidemiology (ISPE) student chapters. He received several awards for distinguished services and finalist poster awards at International conferences and World Congress.
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.
3:15 PM - 6:45 PM
Poster Session 4
Live
3:45 PM - 4:15 PM
Fast Facts
HEOR Job Market and Workforce Trends: Insights From a Recruiter
Level: Introductory
Join us for an interactive discussion on the current state of the HEOR job market, factors driving growth and what is in store for the field the coming years.
Seating is limited.
Discussion Leader
Kevin Mcgrath, BA
Penfield Partners, Fairfield, CT, USA
Kevin is a shareholder with Penfield Search Partners and leads the company's HEOR & Market Access recruiting practice.
Exhibit Hall Theater
Across the Continuum of Real-World Data Use Cases in Oncology: From Early Signals to Regulatory Decision Making
Digital Conference Pass
Since The 21st Century Cures Act was signed into law in 2016, life science companies and real-world data (RWD) companies have been exploring ways in which real-world evidence (RWE) can be used for regulatory decision-making. Yet despite the number of Draft Guidance documents that the FDA has published on this topic in recent years, examples of RWE used for regulatory purposes are few and far between.
However, there are a range of study types leveraging RWD which can inform both life science companies and regulatory agencies on the use and outcomes of new therapies in real-world settings. On the continuum, use cases range from contextual information on patient characteristics, utilization, and outcomes of new medicines in real world settings, to external control arms in the situation of a rare disease where randomized clinical trials are not suitable.
In this Theater presentation, representatives from Ontada will present various uses cases of RWE in oncology decision making, including longitudinal cohorts (which can provide insights about real world use and outcomes) and considerations and challenges associated with external control arms. We will also highlight the importance of the technology and how technology can link different data sources through interoperability as well as allow access to source data for audit purposes.
Seating is limited.
Sponsor: Ontada
Sponsor
Ontada
Speaker
Amy O'Sullivan, PhD
Ontada, Las Colinas, TX, USA
Amy K. O’Sullivan, PhD, is Senior Vice President & Chief Scientific 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, Irving, TX, USA
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 Boston 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.
After being the clinical director of medical oncology at New England Medical Center (Tufts University), Dr. Robert in joined Virginia Cancer Specialists located in Fairfax, Virginia a suburb of Washington, DC. He is the former Medical Director of the Research Program of the practice. He also held positions as 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 6+ 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.
The author of over 100 publications, Dr. Robert 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.
3:45 PM - 4:45 PM
Closed Member Group Meeting
ISPOR Colombia Chapter Meeting
4:00 PM - 4:45 PM
Meet and Greet With Students and New Professionals in HEOR
The Global Networks team will provide an introduction to the ISPOR Student Network and New Professionals Network for those interested in joining or learning more.
4:30 PM - 5:30 PM
Member Group Meeting
Real-World Evidence Special Interest Group Open Meeting
4:45 PM - 5:45 PM
Breakouts: IP, WS, & OBS
Are Discrete-Choice Experiments Preferable to Time Trade-Off for Health State Valuation?
Digital Conference Pass
ISSUE: Health state valuations for quality of life measures have traditionally been conducted using methods such as time trade-off (TTO) and standard gamble (SG). More recently, discrete-choice experiments (DCEs) are increasingly used for health state valuation. Fundamentally, all three methods may elicit trade-offs between quality of life and life years, but DCEs may or may not include life years. Are DCEs or TTO better suited for health state valuation? The aim of the issue panel will be to debate the merits and limitations of DCEs versus TTO and SG for health state valuations. Do DCE methods meet the normative requirements for decision makers? DCE can avoid the cumbersome nature of TTO but at what cost? Each panelist will present their perspective (15 minutes each), followed by a discussion with panelists and the audience (15 minutes). OVERVIEW: The panelists will discuss the merits and limitations of DCEs versus traditional methods for health state valuation from an extra welfarist theoretical perspective (Nancy Devlin), practical and policy considerations (Simon Pickard), and health state valuation in rare disease (Andrew Lloyd). Each panelist will speak for 15 minutes presenting their perspective on the use of DCEs for health state valuation. This will be followed by an interactive discussion between panelists and the audience. This session may benefit anyone with an interest in methods for health valuation and applications of stated preference methods.
Moderators
Siu Hing Lo, MA MSc 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-Elect of the Health Preference Research Special Interest Group (HPR SIG).
Panelists
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 in health care; 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 >150 original peer reviewed journal papers on a wide range of empirical and theoretical topics in health economics.
Andrew Lloyd, DPhil
Acaster Lloyd Consulting Ltd, Oxford, Oxfordshire, United Kingdom
A Simon Pickard, PhD
Department of Pharmacy Systems, Outcomes and Policy, University of Illinois Chicago College of Pharmacy, Chicago, IL, USA
A. Simon Pickard, PhD, is a Professor and Director of Graduate Studies in the Department of Pharmacy Systems, Outcomes and Policymat the University of Illinois Chicago College of Pharmacy. Dr. Pickard’s research focuses on evaluating the safety, effectiveness and value of health care interventions; improving drug-related outcomes through education (particularly opioids); and on the measurement and evaluation of quality of care and health outcomes. He has extensively contributed to the EuroQol Group, and served on the Executive Committee from 2006-2020 (Chair 2014-17).
A Debate on How Best to Value and Pay for Cell and Gene Therapies: Should Cost Savings Be Shared With the Health System and Society?
Digital Conference Pass
ISSUE: Conventional cost-effectiveness methods calculate value-based prices according to both the health gains and cost-savings attributable to the treatment. These cost savings are included regardless of whether the standard of care (SOC) itself is priced in alignment with value according to opportunity costs within the US health system. Including 100% of the cost savings of potentially inefficient SOC and assigning those savings to the new intervention in the calculation of a value-based price prolongs this inefficiency for new generations of SOC. The importance of this issue is heightened with single and short-term therapies which have the potential to displace SOC costs for the remainder of a patient’s lifetime.
OVERVIEW: The panel will start with an introduction by the moderator who will highlight the issue and the subject for debate (10 min). The moderator will share the Institute for Clinical and Economic Review's (ICER) experience developing adaptations to single and short-term therapy value assessments and the existing controversy over how best to value such therapies when SOC is not priced to value. The discussion will then be opened to 3 panelists representing perspectives from health technology assessment (HTA) organizations in the US and Canada, and Industry. Each panelist will be given time (7-10min) to share their perspective on the potential impact of adapted value assessment methods, suggest alternative methods, and defend their position. The remaining 20 minutes will be a question-and-answer period with the audience. As a topic that questions traditional cost-effectiveness methods, there are implications on pricing, innovation, payment, and patient access. The panel is anticipated to be of interest to HTA practitioners, researchers, payers, manufacturers, patients, and the public.
Moderators
Steven Pearson, MD, MSc
Institute for Clinical and Economic Review, Boston, MA, USA
Steven D. Pearson, MD, MSc is the Founder and President 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
Nicole Mittmann, MSc, PhD
CADTH, Toronto, ON, Canada
Dr. Nicole Mittmann is CADTH’s Chief Scientist and Vice-President of Scientific Evidence, Methodologies and Resources.
In her Chief Scientist role, Dr. Mittmann is responsible for ensuring that CADTH actively learns, ensures rigour and quality, mobilizes evidence, and links science to strategy. In her Scientific Evidence, Methodologies and Resources role, Nicole leads CADTH’s shared science groups, including the Science and Methods, Health Economics, Research Information Services, Publishing, Early Scientific Advice and Real-World Evidence teams.
In her academic capacity, Dr. Mittmann holds an MSc and PhD in pharmacology from the University of Toronto. She holds a faculty position as an assistant professor at the University of Toronto in the Department of Pharmacology & Toxicology; and is cross-appointed to the Institute for Health Policy, Management and Evaluation. She is also an associate scientist at Sunnybrook Health Sciences Centre in Toronto, Canada. Dr. Mittmann has conducted and collaborated on notable research in the areas of economic evaluations, outcomes research, and drug/patient safety. Research methodologies include the examination of large databases, economic methodologies, and decision analysis.
She likes to link, leverage and liberate data and evidence.
Robert J Nordyke, PhD, MS
National Pharmaceutical Council, Washington, DC, USA
Bob Nordyke is VP of Research and brings extensive project and organizational leadership, health economics, and health policy experience to NPC. Over the last 20 years, Bob has held roles of increasing impact in health economics consulting and in the pharmaceutical industry. He has founded several health economics consulting practices including Beta6 Consulting Group, PriceSpective, and with Precision Medicine Group. In industry, Bob was VP and Head of Health Economics and Access at Angion Biomedica and previously was a therapeutic area head at Amgen Global Health Economics.
Dr. Nordyke has also served in senior advisory roles nationally, most recently on the steering committee for two projects under the ASN/FDA Kidney Health Initiative. He has been a lecturer at the USC Sol Price School of Public Policy and an adjunct professor at the UCLA Fielding School of Public Health. Bob received his BS in engineering from Rose-Hulman Institute of Technology, an MS in engineering from the University of Texas-Austin, and a PhD in Public Policy Analysis from the Pardee RAND Graduate School.
Marina Richardson, MSc
Institute for Clinical and Economic Review, Boston, MA, USA
Marina is a Senior Health Economist with the Institute for Clinical and Economic Review (ICER). Prior to joining ICER, Marina spent close to 7 years at CADTH (the Canadian Agency for Drugs and Technologies in Health) where she contributed to over 30 drug reimbursement review reports and recommendations. Marina has a well-rounded appreciation for Health Technology Assessment (HTA) having been involved in appraising clinical evidence and contributing to HTA-related methods and process development before shifting her efforts towards a focus on Health Economics. She is currently a PhD Candidate in Health Services Research, HTA stream at the University of Toronto in Canada.
What Is the Value of Real-World Evidence for Biosimilars?
Digital Conference Pass
ISSUE: What is the value of Real-World Evidence for Biosimilars?
OVERVIEW:
Biosimilars have been available in many countries for several years and are increasingly accepted as therapeutic options allowing sustainable and improved patient access to essential medicines. However, uptake and acceptance still vary, and the US is lagging behind other countries, although the launch of adalimumab biosimilars in 2023 is expected to be an inflection point for biosimilars adoption. Robust regulatory processes, notably by the FDA and EMA, allow to address key questions on the quality, safety and efficacy of biosimilars. However, RWE is utilized, notably by payers, healthcare providers and physicians to resolve remaining clinical uncertainties and utilization questions. The panel will explore situations where the totality of evidence, supporting regulatory approval of biosimilars, is sufficient to support confidence and adoption of biosimilars, as well as case studies in which additional questions raised by biosimilar introduction – such as real-life patient adherence and switching – can be addressed by RWE. We will discuss how to ensure robust RWE data collection, learning from the experience of the RWE on the nocebo effect with TNF-alpha inhibitor biosimilars, which delayed biosimilar adoption. The panel will also discuss the need for pragmatic data collection that can address operational questions related to the formulary listing of biosimilars – benefit of multi-dose vials for compounding, bacteriostatic diluent increasing fridge life of reconstituted vial and reducing cytotoxic waste included in pack, biosimilars addressing underdosing of filgrastim in real-life clinical practice – as well as additional biosimilar benefits linked to lower patient copay and improved adherence to chronic therapies. To conclude, the panel will recommend best practices around designing RWE studies for critical insights into biosimilar utilization and outcomes beyond clinical trials, thus addressing some of the lingering clinical barriers to biosimilars adoption.
Moderators
Delphine Courmier, PhD, MBA
Organon, Jersey City, NJ, USA
Panelists
Tijana Fazlagic, MSc, PharmD
BC Ministry of Health, Victoria, BC, Canada
Dr. Tijana Fazlagic is the Executive Director of Therapeutic Assessment & Access, part of the Pharmaceutical, Laboratory & Blood Services Division at the British Columbia Ministry of Health. Dr. Fazlagic’s responsibilities include leadership of the formulary management, specialty medicines, precision medicine and genetic services and the business team. Dr. Fazlagic represents BC on various committees including the Canadian Agency for Drugs and Technologies in Health and pan-Canadian Pharmaceutical Alliance. Dr. Fazlagic co-led the Biosimilars Initiative in BC. Dr. Fazlagic holds a Doctor of Pharmacy degree from the University of Florida, a Master of Science and a Bachelor of Science degrees in Pharmacy from the University of Saskatchewan.
Steven Simoens, PhD
KU Leuven, Leuven, VBR, Belgium
Steven is a senior health economist with expertise in economic evaluation, budget impact analysis, market access policy and incentives. He has a particular interest in oncology drugs, orphan drugs, biosimilars and generics, vaccines, antibiotics, cell and gene therapies.
Phani Veeranki, MD, DrPH
Optum Life Sciences, CYPRESS, TX, USA
Dr. Phani Veeranki is a physician-scientist with more than 10 years of experience in clinical epidemiology, policy analysis and economic valuations to demonstrate the value of health care interventions.
Dr. Veeranki has served as an advisor and investigator for numerous clinical trials, several cohort studies and cross-sectional surveys, along with hospital-based quality improvement projects.
He has deep knowledge and expertise in methodological approaches to address client needs in multiple therapeutic areas including respiratory, oncology, cardiovascular, neurology, psychiatry and rare/genetic diseases.
Additionally, he is an expert in retrospective and prospective cohort studies, clinical trials, medical chart review studies, preference-elicitation methods, expert-elicitation methods and health care economic models.
He has authored or coauthored more than 70 peer-reviewed publications and over 100 conference abstracts or presentations. His work has been published in leading journals including Journal of the American Medical Association, CHEST, BMJ and American Journal of Public Health.
In his current role with Optum life sciences, Dr. Veeranki leads a team of researchers in conducting retrospective cohort studies using administrative claims data and electronic health record data.
Before joining Optum, he held research positions in both academia and the life sciences industry. He holds both a doctorate and a Doctor of Public Health in epidemiology, and completed a fellowship in health outcomes research from Vanderbilt University Medical Center.
Why Is Specialty Drug Coverage Becoming More Restrictive?
Digital Conference Pass
ISSUE:
Health plans’ drug coverage policies play a fundamental role in guiding patients’ access to specialty therapies. Research has found that health plans are imposing coverage restrictions (e.g., step therapy protocols, patient subgroup restrictions, etc.) in their specialty drug coverage policies more often. This panel will provide a forum to discuss the potential reasons for this trend, and its consequences for patients and other health care stakeholders.
OVERVIEW:
Rising costs mean that health plans face a challenge to provide their enrollees with appropriate access to specialty drugs. James Chambers will moderate the panel and will frame the issue by summarizing new research that has found that among US commercial health plans’ specialty drug coverage has become increasingly restrictive. This empirical research, which tracked roughly 5,000 plan coverage policies for 200 specialty therapies over a five-year period, found that the percentage of coverage policies with utilization management criteria increased from 39% in 2017 to 52% in 2021. Plans’ greater use of step therapy protocols is one factor driving this trend. C. Bernie Good will provide a health plan perspective, describing the challenges encountered by health plans, and the various approaches plans are taking to rise to these challenges, including the use of innovative value based contracts for pharmaceuticals. Rochelle Henderson will provide an industry perspective and will give an overview of how product manufacturers interact with health plans and their evidence generation practices. Panelists’ presentations will be limited to three PowerPoint slides (a maximum of 5 minutes), after which the moderator will encourage debate through Q&A and audience participation.
Moderators
James Chambers, PhD
Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, MA, USA
James Chambers is an investigator at the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center, and an Associate Professor of Medicine at Tufts University School of Medicine. His research has highlighted variation in patients’ access to specialty and orphan medications and in the evidence base that health plans cite in their policies. He graduated from Queens University in Belfast with an MPharm degree and previously worked as a pharmacist in the UK and Ireland. James also obtained an MSc from the University of York and PhD from Brunel University, both in Health Economics.
Panelists
Chester Good, MD, MPH
UPMC Centers for High-Value Health Care and Value-Based Pharmacy Initiatives, Pittsburgh, PA, USA
Chester Good, MD, MPH, is professor of medicine and pharmacy, University of Pittsburgh School of Medicine and School of Pharmacy. He is also the senior medical director, UPMC Health Plan Insurance Division, as well as the director for the Center for Value-Based Pharmacy Initiatives.
Rochelle Henderson, PhD
National Pharmaceutical Council, Washington, DC, USA
Dr. Henderson has been a regular speaker on drug coverage issues. She has published on various related topics, including on patient centered formulary and benefit design, evidence for decision-making and biopharmaceutical innovation. Dr. Henderson is highly knowledgeable about the subject area and will be an entertaining and energetic speaker. Her experience at Express Scripts, in combination with her current role at NPC, means that she is an ideal contributor to this panel.
Case Studies
Regional Case Studies
Management of drug-related schemes can involve many real-world considerations, such as spatial access, feasibility of managed access, financial sustainability, and patient engagement in HTA. These case studies provide interesting examples of such considerations across 4 different countries.
Seating is limited.
Moderator
George Papadopoulos, BSc(Hons), GradDipEpi.
Lucid Health Consulting, McMahons Point, NSW, Australia
George has more than 25 years experience in the pharmaceutical industry including clinical research, health economics, reimbursement, pricing and business development. He has worked in both the local operating company and global levels in increasing levels of responsibility. He has worked for leading pharmaceutical companies in Australia, Belgium and the United States including Pharmacia, Johnson & Johnson, Schering-Plough and Merck. George recently returned to Australia and established Emerald Corporate Group, a strategic consultancy providing services to assist healthcare companies with the market access for their technologies. These services include health economics, health technology assessment, and pricing and reimbursement strategy and event management. George’s strengths are his ability to develop health economic and reimbursement strategic plans, strategic pricing initiatives, and the early commercial assessment of opportunities. George has put many of these strategies into practice around the world, including the successful reimbursement and timely market access for healthcare technologies, publication in peer reviewed journals and strategic stakeholder meetings. This is combined with his expertise and understanding of US, Canadian, European, Asia Pacific and Japanese market access issues developed through his global network of colleagues in government, academia and the healthcare industry.
CS9: The Use of Evidence in Benefit Package Reviews to Ensure Financial Sustainability - A Case of the National Health Insurance Scheme of Ghana
4:45PM - 5:00PM
Mensah Annan R
National Health Insurance Authority, Accra, AA, Ghana
Problem Statement: The inclusion of new services onto health benefit packages without ascertaining the implications of the additional costs, can affect the financial sustainability of health financing institutions.
Description: The benefit package (BP) of the National Health Insurance Scheme (NHIS) of Ghana covers about 95% of disease conditions occurring in the country. It operates with an explicit exclusion list but often receives requests for coverage of these excluded services. A review of the BP was however approached with caution in the interest of sustainability. In 2018, the National Health Insurance Authority (NHIA) in collaboration with its partners, built a new actuarial model that allowed for projections of the costs of adding new benefits to the package to be made. The new model provided a granular assessment of the future sustainability of the NHIS by modelling the financial impact of additional services that were being proposed, such as family planning, childhood leukemia, prostate cancer, breast and cervical cancer screening and sickle cell treatment using hydroxyurea. The actuarial model utilised NHIS claims and membership data, information from Global Burden of Disease Studies, national population data, budget forecasts of the Ministry of Finance and NHIS income and expenditure data to model projections. The results of the study indicated that the NHIS would remain sustainable with its current financial inflows if clinical family planning services, four childhood cancers (Acute Lymphoblastic Leukemia, Burkitt’s Lymphoma, Retinoblastoma and Wilms Tumour) and sickle cell treatment with hydroxyurea were included in the BP. Following on this, the NHIA added these services to the BP for reimbursement under the scheme.
Lessons Learned: The use of a robust actuarial model to assess available funding and project the capability of a health insurance scheme to support further BP inclusions is a prudent strategy to ensure financial sustainability.
Stakeholder perspective: Payer.
CS12: Suitability of Managed Access in England to Resolve Uncertainties in the Cost-Effectiveness of Tixagevimab–Cilgavimab for the Prevention of COVID-19
5:30PM - 5:45PM
Austin C1, Lawrence T2, Norton S2, Strong T2
1National Institute for Health and Care Excellence, Manchester, LAN, UK, 2National Institute for Health and Care Excellence, Manchester, UK
Problem Statement: The Innovative Medicines Fund is a Managed Access Fund in England designed to allow patient access to clinically uncertain promising medicines while further evidence is collected to address uncertainty. The NICE Managed Access Team assesses the suitability of new technologies for managed access by producing a feasibility assessment and discussing suitability with stakeholders. The Managed Access Team has recently considered the suitability of tixagevimab–cilgavimab for managed access.
Description: Several factors emerged indicating that managed access would not feasibly resolve uncertainty. While there are uncertainties in the clinical evidence and further evidence generation will likely be relevant, it is unfeasible that these would be resolved by data collection through managed access. Unpredictable landscape changes including changes to baseline rates of infections and hospitalisations and emergence of new variants would not be resolved through further pre-specified data collection. Monitoring these changes would require constant surveillance and reactive guidance updates. Discussing real-world evidence with stakeholders identified several barriers. To generate evidence in managed access, widespread testing for COVID-19 infection and genomic analysis would be required. Additionally, a new national dataset would be required, or multiple diverse datasets linked: no single unified dataset exists that could track patients through the multiple services relevant to delivering tixagevimab-cilgavimab and subsequent care and testing. There is no relevant primary care data collection that has national coverage, and varied data collection practices exist between primary and secondary care. Therefore, effective collection would require significant amendments to existing, large data sources. Furthermore, existing patient consent and ethical approval would not allow data sharing between sources.
Lessons Learned: Tixagevimab–cilgavimab is currently not considered suitable for managed access by the NICE Managed Access Team. Barriers to managed access identified for this topic are likely to be common across infectious disease prevention topics.
Stakeholder perspective: Government-affiliated
CS11: Spatial Access to Healthcare Facilities: A Case Study of Gandhinagar District Hospital
5:15PM - 5:30PM
Kumar V, Tripathi T
Central University of Gujarat, Gandhinagar, GJ, India
Problem Statement: This study set out to investigate the spatial accessibility to healthcare facility in Gandhinagar district. It study establishes the connection between geographical determinants, neighborhood impact, and infrastructure that supports health. In order to comprehend the ease and difficulty of access to healthcare services from Gandhinagar district hospital and create index of access to healthcare services, this research will also contain a spatial component.
Description: One of the most important variables in the preservation and promotion of societal health is the availability of healthcare facilities and services. The process of getting access to health care involves a number of variables, including the availability and cost of health care, the area and ease of access to the healthcare facilities, and supporting infrastructure like pharmacies, communication networks, among others. So far, acceptability and cost have been used as glasses through which to view access to health treatments. However, there are situations when a health center's location and service area can play a significant role in determining how easy it to receive healthcare.
Lessons Learned: The index will provide straightforward figure to monitor healthcare accessibility over time. Appropriate descriptive tools along with percentages, graphs, and tables will be used to substances the study objective. All the analysis will be run in QGIS and STATA Software. The study found healthcare accessibility is poor for more than 30 minutes and 50 km away from district hospital. There was gap in access to the health district hospital because time and distance are not same for all patients to the health facility. Health resources should be allocated depending on the degree of development and the priorities should be established.
Stakeholder perspective: This study will offer significant policy inputs about the equitable and ideal distribution of healthcare facilities on the basis of distance, time, and supporting infrastructure.
Keywords: Spatial-Access, Healthcare-Facilities, GIS, Healthcare Access-Index
Breakouts: IP, WS, & OBS
ISPOR Asia Pacific Health Policy Update
Digital Conference Pass
Level: Intermediate
Hosted by ISPOR Asia Consortium, this session will feature two presentations highlighting cases of important health policy trends for the Asia Pacific region. The first presentation will cover the topic “evidence-based healthcare policy initiatives - an Indian perspective,” presented by an expert familiar with the research, development, and uptake of critical medical technologies in India. The second presentation will cover the topic “Korean reimbursement for Digital Health,” presented by a leading decision-maker in a large health technology assessment body in South Korea. The presentations will be followed with an opportunity for Q&A by the audience.
Moderators
Joe Caputo, MSc
Vista Health Pte Ltd, Singapore, Singapore
Joe’s background and experience spans industry and consulting roles across multiple continents. He worked for 14 years with GlaxoSmithKline where his roles included drug development, sales & marketing and UK & global health outcomes. As a Principal at IMS Health, Joe moved to Singapore in 2012 to set up and manage the Real-World Evidence & HEOR team for the Asia-Pacific region, before founding Vista Health in 2016. An expert in RWE & HEOR strategy development, HTA, market access, patient level data analysis and value communications, Joe is currently the President of ISPOR Singapore Chapter.
Joe is also the former Associate Director for ESSEC Business School Institute of Health Economics and Management Asia-Pacific, and guest lecturer at ESSEC; he is a frequent speaker at international conferences, having attended many client and subject matter meetings as an industry expert; In 2012 he was invited to present an overview of HTA to the China FDA and in 2017 presented on HTA methodology to VSS and MOH in Vietnam.
Speakers
Young Ae Jeong, ,
Health Insurance Review & Assessment Service (HIRA), Wonju, South Korea
Kavita Kachroo, BDS, MHA
Kalam Institute of Health Technology, Andhra Pradesh MedTech Zone, Andhra Pradesh, India
Dr Kavita Kachroo is the Deputy Director at Kalam Institute of Health Technology (KIHT) one of the leading organizations in India. Kalam Institute of Health Technology (KIHT) is under the Department of Biotechnology Government of India, which aims to facilitate focused research on critical components pertaining to medical devices and drugs by supporting institutions involved with R&D, industry, policy makers and knowledge repositories. It is also a WHO-Collaborating Centre for Health Innovation.
Dr Kavita Kachroo is an accomplished health research professional with over 15 years of experience in the field. With a passion for improving public health outcomes through evidence-based research, she has made significant contributions to the development and implementation of research projects that have had a positive impact on health policy and practice. Her contributions to the field are well-documented through her many publications, which have been widely recognized as influential in research translation. She brings a wealth of knowledge and leadership skills to the role, along with a proven track record of successful management of research teams and complex projects nationally and internationally.
Prior to joining Kalam Institute of Health Technology (KIHT), Dr. Kavita has served as a consultant with WHO Collaborating Centre for Policy Medical Devices & Health Technology Policy Division of Healthcare Technology & Innovations, National Health Systems Resource Center, New Delhi under, Ministry of Health & Family Welfare, Govt. of India and other reputed organizations.
Accounting for Equity in Health Technology Assessment
Digital Conference Pass
Level: Intermediate
PURPOSE:
To present extensions of the conventional cost-effectiveness framework, including multiple criteria decision analysis (MCDA) and distributional cost-effectiveness analysis (DCEA), that are capable of quantitatively assessing the impact of a healthcare treatment on equity. For each method, the implementation complexity, data requirements, integration with cost-effectiveness and budget impact estimates, and the ability to measure change in the inequality distribution will be presented.
DESCRIPTION:
Traditional methods for economic evaluation are criticized because they are focused on efficiency and affordability, while other factors such as equity are important attributes to decision making. Equity is traditionally incorporated in health technology assessments (HTA) through committee deliberations, but this approach has also been criticized as lacking rigor and transparency. Melanie Whittington (10 minutes) will introduce the workshop by presenting the results of the Institute for Clinical and Economic Review’s recent health equity initiative, including findings from a literature review and interviews with experts related to equity-informative economic evaluation. Brett McQueen (15 minutes) will provide an overview of how MCDA can be used to incorporate equity into a value assessment, with an in-depth discussion of the potential use of MCDA to inform structured deliberation. He will include an interactive exercise to demonstrate how additional criteria can be explicitly included in value assessment deliberations. Stacey Kowal (15 minutes) will provide an overview of how DCEA can be used to incorporate equity in a value assessment, with an in-depth discussion of data and stakeholder-alignment needed to support DCEA. Her session will include audience polling about the range of use cases for DCEA, including informing the equity impact of funding decisions and decision-making about patient access. Adequate time (20 minutes) for audience questions and comments will be reserved. This workshop will be relevant for a range of stakeholders, including health economists, HTA groups, life science companies, patients, and decision makers.
Discussion Leaders
Melanie Whittington, PhD, MS
Institute for Clinical and Economic Review, Boston, MA, USA
Dr. Whittington is the Director of Health Economics at the Institute for Clinical and Economic Review (ICER). In her role, she leads the cost-effectiveness analyses and potential budget impact analyses within ICER reviews, directs the content within the Interactive Modeler™, and advances the field of health economics by developing innovative methods with applications for value assessment.
Discussants
Stacey Kowal, BS, MS, MSc
Genentech, Alameda, CA, USA
Stacey Kowal currently serves as a principal researcher in Genentech’s Health Policy and Systems Research team where her work focuses on identifying and testing new methods to assess the impact of new healthcare technologies. Her background includes both US and global work to inform health technology assessment and reimbursement as well as design of public health interventions, drawing on disciplines of applied mathematics, economics, and international health policy. Stacey’s current research aims to increase focus on equity effects in health economics and outcomes research (HEOR), including integration of distributional elements into healthcare decision-making. Stacey is a Udall scholar, a Truman scholar and a Marshall scholar. She holds a BS in Mathematics from Alma College, an MSc in public health from the London School of Hygiene and Tropical Medicine and an MSc in International Health Policy and Health Economics from the London School of Economics.
Robert McQueen, PhD
University of Colorado, Denver, CO, USA
R. Brett McQueen is an Assistant 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.
Valuing Innovations - Opportunities and Challenges to Better Define and Measure Innovative Properties of Health Technologies
Digital Conference Pass
Level: Intermediate
PURPOSE:
- Discuss the strengths and weaknesses in accounting for innovations in existing approaches of health technology assessments (HTA),
- Identify prioritized areas for methods and data research to better define and measure innovations,
- Outline practical steps that different stakeholders can take to advance the methods and practice to better account for innovations in HTA.
DESCRIPTION:
Over the past decades, innovations in health technologies have been a major contributor to improved life expectancy and quality of life. However, as biomedical and digital innovations accelerate, the methods and practice to define and measure innovations in HTA are not keeping pace. IVI convened a multi-stakeholder expert roundtable to identify prioritized areas that need further research in methods and data to better define and measure innovations. This workshop brings together cross-stakeholder participants to highlight these prioritized areas, and outline action steps that stakeholders can take to better account for innovations in HTA. In this workshop, Dr. Xie will first discuss the importance of considering innovations in HTA, and briefly introduce the Expert Roundtable held by IVI to identify prioritized areas for research. (8 minutes) Ms. Laing will provide an overview of how innovative properties of health technologies are being considered in current approaches to HTA, and discuss their strengths and limitations. (8 minutes) Based on the results from the roundtable held by IVI and live-polling with the audience, Dr. Campbell will discuss prioritized areas for additional research, and how novel methods and data can be better integrated into existing HTA frameworks. (8 minutes) Dr. Krieger will discuss the steps that stakeholders can take to better account for innovations in HTA, and the likely impacts on patients’ well-being and long-term dynamic efficiency. (8 minutes) The workshop will end with a facilitated dialogue and live Q&A with the audience.
Discussion Leaders
Richard Xie, PhD
The Innovation and Value Initiative, Newton, MA, USA
Richard Xie is a health economist and serves as Director of Research at the Innovation and Value Initiative. He has a track record of conducting HEOR studies to inform health policy design and valuation of pharmaceutical therapies. Formerly he was a consultant at the Analysis Group, managing a portfolio of studies to demonstrate the value of novel therapies for pharmaceutical clients. Xie was also a health policy researcher at the Leonard D. Schaeffer Center for Health Policy and Economics at the University of Southern California, examining policy questions in medication non-adherence, health disparities, and healthcare innovation evaluation. His work has been published in journals including Journals of the American College of Cardiology, Journal of General Internal Medicine, PharmacoEconomics, and PLOS One. Xie earned his doctorate in economics from the University of Southern California.
Discussants
Jon Campbell, PhD
Institute for Clinical and Economic Review, Hingham, MA, USA
Jon Campbell is Senior Vice President for Health Economics at the Institute for Clinical and Economic Review (ICER). Jon is a leader in value assessment methods and application. He is an author of over 250 manuscripts and abstracts in the field of value assessment, over twenty ICER assessments, and holds an affiliate faculty position within the Center for Evaluation of Value and Risk in Health at Tufts University School of Medicine. Jon has graduate training degrees in pharmaceutical outcomes research (PhD) and biostatistics (MS) from the University of Washington.
Joshua Krieger, PhD
Harvard Business School, Newton, MA, USA
Josh Krieger is an assistant professor of business administration in the Entrepreneurial Management Unit. Josh’s research focuses on R&D strategy and the economics of innovation. His work examines project portfolio selection, R&D competition, and how firms and research organizations adjust their R&D efforts in response to new information and resources. He has taught the first year course, The Entrepreneurial Manager, and currently teaches a second year elective course: Tough Tech Ventures, which examines the development, commercialization and financing of cutting-edge science and technology.
Maia Z. Laing, MBA
Optum, Washington, DC, USA
Maia is a seasoned healthcare professional with over 20 years of experience in healthcare care systems, policy, and quality improvement. She is a thought leader in improving access to quality care through human centered, data informed - innovation. Maia has demonstrated ability to drive outcomes across various healthcare sectors including non-profit healthcare delivery, government, and private industry. In the past, Maia worked for Brigham and Women’s Hospital, ranked top 20 hospital by U.S. News and World Report. She led innovation for the U.S Department of Health and Human Service and most recently joined Optum.
Maia’s most celebrated accomplishments include leading the strategic roadmap for the Indian Health Service Health IT modernization (Link) and the development of Health+ (Link) a process that unleashes the power of data and technology to advance new solutions for underserved communities. She is passionate about improving the quality of life and treatment for people living with Sickle Cell Disease.
Podium Session
Patient Experience Data in Regulatory Decision Making and Value Assessment
Research presented in this session addresses patient experience data in the context of regulatory decisions and value assessment, in the United States and Europe. Seating is limited.
Moderator
Tamas Agh, MSc, PhD, MD
University of Pécs & Syreon Research Institute, Budapest, Hungary
Tamás Ágh MD MSc PhD DrHabil is a principal researcher of Syreon Research Institute and a research associate professor at the Center for Health Technology Assessment and Pharmacoeconomic Research, University of Pecs. With around 15 years of experience in health economics and outcome research, he developed expertise in the fields of medication adherence, patient reported outcomes, health technology assessment, and evidence synthesis. Dr. Ágh is a core group member of the ISPOR Medication Adherence and Persistence Special Interest Group and the ENABLE (European Network to Advance Best practices & technoLogy on medication adherencE) Cost Action.
P40: How Did FDA Consider Patient Experience Data in Regulatory Decisions Among Approved Drugs in 2022?
5:00PM - 5:15PM
Patil D1, Oehrlein E2
1University of Maryland School of Pharmacy, Baltimore, MD, USA, 2Applied Patient Experience, LLC, Washington, DC, USA
OBJECTIVES: This research aims to identify trends regarding types of patient experience data (PED) submitted to FDA and insights about how FDA considers PED.
METHODS: We identified all new molecular entities and new therapeutic biological products approved by the FDA’s Center for Drug Evaluation and Research in 2022 and reviewed the corresponding new drug application/biologics license application review documents to determine whether or not a PED Table was included. Next, we extracted information about 1) which types of PED were submitted by the sponsor; 2) additional information not submitted by the sponsor but identified by FDA staff; and 3) qualitative information about how PED guided decisions. All data was reviewed by two researchers.
RESULTS: Among the 37 drugs approved by CDER in 2022, clinical/multidisciplinary reviews were available for 32 at the time of this analysis. Of these, the vast majority included a PED Table (n=30). The tables documented that 21 drugs included clinical outcome assessments (COAs) as the type of PED submitted, with patient-reported outcomes (PRO) the most common type of COA. Several qualitative studies, patient-focused drug development meeting summary reports, natural history studies and patient preference studies were also considered. We observed significant differences across reviews in the level of detail included in the “Patient Experience Data” section. This ranged from checkmarks with little context to a detailed description of how qualitative interviews and other interactions with a patient organization informed FDA staff’s overall benefit-risk determination.
CONCLUSIONS: Among our sample of approved products, COAs, especially PROs were the most common type of PED submitted. We also identified other types of PED including data from qualitative interviews and patient-focused drug development meetings contributing to regulatory decisions. There is an opportunity to improve consistency in how PED Tables are filled in by FDA staff and to provide additional information about how PED guides decisions.
P38: Current Practices and Challenges When Submitting Patient Experience Data for US Regulatory Decision-Making: An Industry Survey
4:45PM - 5:00PM
Pinto CA1, Tervonen T2, Jimenez-Moreno C3, Levitan B4, Soriano Gabarro M5, Girman C6, Norquist J7, Hauber B8
1Merck & Co., Inc, rahway, NJ, USA, 2Kielo Research, Zug, ZG, Switzerland, 3Evidera, London, LON, UK, 4Johnson & Johnson, Titusville, NJ, USA, 5Bayer AG, Berlin, Germany, 6CERobs Consulting, LLC, Wrightsville Beach, NC, USA, 7Merck & Co., Inc, Rahway, NJ, USA, 8Pfizer Inc, New York, NY, USA
OBJECTIVES: To describe industry perspectives related to the collection and use of patient experience data (PED) in Food and Drug Administration (FDA) regulatory submissions.
METHODS: An online survey of experiences (part 1) and best-worst exercise for prioritizing PED advancement efforts (part 2) was completed by industry and contract research organization (CRO) members with ≥2 years of recent experience with patient reported outcome (PRO), natural history study (NHS), and patient preference (PP) data; and direct experience with FDA filings including PED. The survey was pilot tested by 10 industry members, with the final survey disseminated by special interest groups within seven professional societies and consortia. Descriptive summaries were generated.
RESULTS: With 657 views of the survey, 50 eligible participants (84% industry, 16% CRO) completed part 1, among which 46 participants completed part 2. Respondents had PRO (86%), PP (50%), and NHS (18%) experience, most with ≥5 years of experience; and experience meeting with FDA to discuss PED use (72%). All respondents indicated that FDA meetings should have a standing agenda item to discuss PED use in decision-making, and most (78%) reported that FDA meetings to discuss PED use should occur before pivotal trials. Moreover, most responded that the FDA and Sponsor should co-develop the PED Table in the FDA clinical review (74%), and the table should report PED use (90%) or reason(s) for not using PED (96%) in regulatory decision-making. The most important efforts to advance the use of PED in decision-making were a dedicated meeting pathway and expanded FDA guidance (both ranked first in 51% of choices containing this item), followed by a roadmap (26%), collaborative initiatives (19%), and public inventory of acceptable methods (17%).
CONCLUSIONS: While the FDA has policy targets extending PED use, challenges still exist regarding pathways for submission and transparency regarding PED use in regulatory decision-making.
P37: Review of Institute for Clinical and Economic Review (ICER) Reports: Use and Accessibility of Surveys to Incorporate the Patient Perspective
5:30PM - 5:45PM
Majercak K1, Perfetto E2, Slejko JF1, Mullins CD1
1University of Maryland School of Pharmacy, Baltimore, MD, USA, 2University of Maryland School of Pharmacy, VENICE, FL, USA
OBJECTIVES: Patient-experience survey data collected by patient organizations can be leveraged to inform patient-centered value and health technology assessment (V/HTA). While patient engagement in V/HTA has increased over time, it is unknown whether the use of patient-survey data in V/HTA reports has increased as well. Our objective was to describe use of patient-experience surveys in Institute for Clinical and Economic Review (ICER) reports.
METHODS: ICER VA reports were reviewed for patient-experience survey-data use/reporting within the patient and caregiver perspectives section of the report. All of ICER’s publicly available final reports from 2017-2022 (n=56) were accessed and reviewed. Descriptive attributes were abstracted, including: condition, report date, report title, survey used, survey publicly available, report update and condition revisited. A survey was deemed publicly available if the full instrument was accessible from the ICER report (appendix or via citation) and partially available if examples of items were accessible from the report.
RESULTS: From 2017-2022, less than half of ICER assessments referenced patient-input data via patient surveys collected by patient organizations (n=16). The frequency of survey use did not increase over time across reports (4/8 (50%); 4/12 (33.3%); 2/9 (22.2%); 1/11 (9.1%) 3/10 (30%); 2/6 (33.3%) for 2017-2022 respectively). For reports referencing survey data, instruments were not always publicly available; only 4/16 survey instruments were publicly accessible, and 5/16 surveys were partially available beyond survey result summaries.
CONCLUSIONS: Use of patient-experience survey data did not increase across ICER reports from 2017-2022. Survey data may not exist or other methods of data collection for patient-input may have been used as an alternative to surveys. When used, however, survey instruments were not always publicly available. This suggests a need for transparency and accessibility of patient-experience surveys used to inform V/HTA, which may enhance understanding of which/how data have been collected, and gaps in available data.
P39: Patient-Reported Outcomes As Endpoint in Oncology Trials: An Analysis of FDA and EMA Claims
5:15PM - 5:30PM
Desvignes-Gleizes C, Kraft NG, Desruol-Allardin S, Bothorel S, Sherafat R
Mapi Research Trust, Lyon, 69, France
OBJECTIVES: Patient-Reported Outcomes (PROs) capture patient’s subjective perception of the effects of a disease or treatments. Previous reports have indicated that regulators have different approaches in inclusion of PROs as endpoint measures in the drug approval process. We aim at highlighting such differences by investigating the current use of PROs, as endpoints in oncology trials and label claims granted by the FDA in comparison with the EMA.
METHODS: Number of drugs with a PRO claim, PRO endpoint positioning, the underlying concepts and instrument names were extracted from PROLABELS™ database. The findings were contrasted and compared between the two regulatory environments.
RESULTS: EMA and FDA both granted PROs in drugs labels over the past two decades. During 2015-2022, 60% of EMA-approved drugs (78% of oncology drugs) included PROs in claims. For the FDA, these rates were 47% and 39%, respectively.
For oncology drugs, PROs mainly measured “
Symptoms” (including pain) as primary endpoint and
“Quality of Life” (QOL) or “
Symptoms” as secondary endpoint under EMA-approved labels, while for the FDA,
“Symptoms” were the main primary and secondary endpoints. Out of 69 EMA-approved drugs in oncology, 52 included “
QOL” measures (EORTC-C30, EQ-5D, FACT-P, FACT-Lym, Fact-O, EORTC-BR23) and 32 included “
Symptoms” measures (EORTC-LC13, Lung Cancer Symptom Scale, FACT-KSI-Disease related Symptoms). Among 37 FDA-approved drugs in oncology, 26 measured “
Symptoms” (Brief Pain Inventory, study specific PRO assessing pain or flushing) and 6 drugs included “
QOL” measures (EORTC-C30 and FACT-L).
CONCLUSIONS: Despite growing recognition of their value in the drug development process, PROs inclusion in claims remains limited in number and narrower in concept of interest in FDA-approved cancer therapies. EMA, on the other hand, takes broader concepts, such as quality of life, under consideration.
Health Policy and Pricing
New approach and appraisal of framework and models for health policies and pricing policies will be presented in this session. Different models (value-based pricing, Accelerated Approval, drug reform payment method, HTA guided decision making) will be discussed from different countries. Seating is limited.
Moderator
Christopher Blanchette, PhD, MBA
Novo Nordisk Inc, Plainsboro, NJ, USA
P41: Uncertainty Management in Regulatory and Health Technology Assessment Decision-Making: Guidance of the HTAi-DIA Working Group
5:15PM - 5:30PM
Hogervorst M1, Heikkinen I2, Vreman RA3, Oortwijn W4
1Utrecht University, Utrecht, UT, Netherlands, 2MSD, Copenhagen, 84, Denmark, 3Utrecht University, Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht, UT, Netherlands, 4Radboud university medical centre, Nijmegen, Netherlands
OBJECTIVES: Uncertainty is a fundamental component of healthcare decision-making. Framing uncertainty depends on the scientific discipline and is highly context specific. Therefore, its interpretation and the corresponding risks are a major challenge to decision-makers. Following the 2021 HTAi Global Policy Forum, an HTAi-DIA Working Group (WG) was initiated to develop guidance to facilitate uncertainty management. This guidance aims to support stakeholder dialogue on the identification and mitigation strategies for perceived uncertainties in the interface of regulatory and Health Technology Assessment (HTA)-regulatory decision-making.
METHODS: The WG consisted of 26 members with different backgrounds, and expertise relevant to the topic including the academic, regulatory, HTA, payer, developer, and patient perspective. The guidance was established through: i) six online discussions among the WG between December 2021 and September 2022 using insights from a scoping literature review and two literature case studies (larotrectinib and lumacaftor/ivacaftor), ii) a survey disseminated across the WG members and iii) applying the guidance to an ongoing case study.
RESULTS: The WG identified key terms and concepts, codified into twelve building blocks that together were perceived to define uncertainty, i.e., ‘unavailable’, ‘biased, ‘conflicting’, ‘incomprehensible’, ‘random variation’, ‘information’, ‘prediction’, ‘impact’, ‘risk’, ‘judgement’, ‘context’, ‘relevance’. The building blocks were then translated into a checklist to be able to define whether any issue constitutes a relevant uncertainty. Additionally, a regulatory-HTA-relevant taxonomy, including domains in which uncertainty may exist, was developed to facilitate the categorization of the relevant uncertainties. Functioning of the guidance and how it may be applied in dialogues on mitigation strategies was demonstrated using a case study.
CONCLUSIONS: The systematic approach for the identification of uncertainties in this guidance has the potential to ensure mutual understanding between stakeholders through improved consistency and transparency. To further support uncertainty management and stakeholder dialogue, a future step is to systematically align uncertainty identification with suitable mitigation strategies.
P43: Critical Appraisal of Drug Pricing for Advanced Breast Cancer Medications Approvals in Brazil (2007 - 2022) and Comparison with Major National Economic Indicators
4:45PM - 5:00PM
Goncalves V1, Stefani S2
1Hospital Sao Lucas da PUCRS, Porto Alegre, RS, Brazil, 2Grupo Oncoclínicas, Porto Alegre, Brazil
OBJECTIVES: To provide a critical appraisal of drug pricing for advanced breast cancer medications approved in Brazil in the last 15 years (2007 - 2022) and establish a comparison with major economic indicators of the country during the same time period.
METHODS: By using the Brazilian ANVISA database we extracted the pricing of each new medication at the time of the approval for the treatment of advanced breast cancer in Brazil in the last 15 years, with posterior adjustments in these prices for inflation and foreign exchange rates variation with the US Dollar. After that, we correlated the monthly cost of each treatment with major economic data for Brazil during the same time period (economic growth and inflation).
RESULTS: There was a sharp increase in the monthly cost of medications approved by ANVISA for the treatment of advanced breast cancer in Brazil during the last 15 years, jumping from 9.754 BRL in 2007 to over 85.316 BRL in 2021 (+774%). This increase is maintained, although less pronounced, when adjusted for inflation (+ 296%) and exchange rate variation with the US Dollar (+176%) but highly superior to the national inflation index (+139,7%) and increment in GDP per Capita (adjusted by purchasing power parity) observed during the same time period in Brazil (+28%).
CONCLUSIONS: This study adds on the topic of affordability of cancer care, especially in middle income countries, and the necessity to establish better drug pricing and national willingness to pay for each cancer drug approval that consider not only the international pricing but other important local economic metrics such as inflation and GDP per capita adjusted by purchasing power parity in order to avoid restrictions in access to important cancer medications for the patients in the future.
P42: Value-Based Pricing for Drugs with Accelerated Approval Incorporating Uncertainty and Risk Aversion
5:30PM - 5:45PM
Jiao B, Hsieh Y(, Verguet S
Harvard T.H. Chan School of Public Health, Boston, MA, USA
OBJECTIVES: The U.S. Food and Drug Administration accelerated approval (AA) program allows novel therapies to reach patients based on preliminary clinical evidence, in spite of potentially large uncertainties remaining with respect to their efficacy. Cost-effectiveness analyses (CEAs) could be conducted to inform the value-based price (VBP) of AA drugs, for which the incremental net monetary benefit (iNMB) compared with the standard of care (SoC) is set to zero. Such CEA approaches implicitly assume that the payers are risk neutral, even though they are likely risk averse. We propose a methodology to determine the VBP of AA drugs that explicitly incorporates risk aversion.
METHODS: We prescribe a payer’s utility by using a constant relative risk aversion (CRRA) utility function for the iNMB of an AA drug (relative to SoC); and this CRRA utility function is combined with the expected value of perfect information metric. The risk-averse VBP of the AA drug is then computed when the expected utility of the iNMB of an AA drug with perfect evidence (on drug efficacy) equals the iNMB of that drug with preliminary evidence. We illustrate our approach with the case study of a hypothetical AA drug for patients with relapsed or refractory multiple myeloma, using a range of input parameters for quantifying uncertainties and the CRRA coefficients.
RESULTS: We observe substantial changes when incorporating risk aversion into the VBP of AA drugs. As a case in point, in our hypothetical myeloma case study, the risk-neutral VBP of the AA drug would be $9,000 per month under a willingness to pay of $100,000 per quality-adjusted life year; while it could decrease to as low as $5,000 per month for a risk-averse payer.
CONCLUSIONS: Incorporating risk aversion and associated utility into value of information frameworks enables the explicit valuation of uncertainty into the determination of the VBP for risk-averse payers.
P44: Moral Hazard Effects of Payment Methods Reform in China
5:00PM - 5:15PM
Bai J1, Chen N2, Tan J2, Yan P2, Wang J2, Ren M2, Kong S3
1Wuhan University, WuHan, China, 2Wuhan University, Wuhan, 42, China, 3China National Institute of Hospital Administration, Beijing, China
OBJECTIVES: With the development of economy, the intensification of healthy aging and the gradual expansion of health insurance coverage, the rapidly increasing medical expenses have become one of the main challenges facing the reform of China's medical and health care system. The key to effectively controlling the excessive rise of medical expenditures and ensuring the sustainability of medical insurance funds lies in whether the reform of medical insurance payment methods can be effectively promoted. China started a pilot project to modify the DRG payment reform in 2017, and intends to implement a multifaceted and complex medical insurance payment method based on payment by diagnosis-related groups or disease type in 2024. Diagnosis-related group payment system is frequently seen as helpful in containing the development of healthcare costs. However, it also causes moral hazard issues on the supply side, including higher rates of readmission to hospitals and cost shifting. This research aims to explore the impact of diagnosis-related group payment reform on supply-side moral hazard in China.
METHODS: The model in this study was built using interrupted time series analysis, and basic medical insurance claims data for 10% of patients from all secondary and tertiary health care institutions in City A during 2019–2021 were randomly selected. We selected several diseases.
RESULTS: The diagnosis-related group payment reform increased the two-week and one-month readmission rates for patients and dramatically decreased total medical costs and out-of-pocket expenses for inpatients.
CONCLUSIONS: This shows that medical professionals may alter treatment programs and fragment hospital stays in response to the reform.The core goal of the diagnosis-related group reform is to place more financial pressure on healthcare providers and encourage them to take charge of cost management, enhancing the effectiveness of healthcare resource utilization. Medical professionals, however, have the potential to lower expenses while having a negative impact on the standard of service.
5:45 PM - 6:45 PM
Networking Reception
We’re so excited for our international conference that we’re continuing the celebration into night 2. Network with your peers, visit exhibitors, and enjoy spending time with the largest HEOR community.
Women in HEOR: Networking and Meet the Speakers Event
Meet the Women in HEOR session speakers and mingle with other women in HEOR and "cool guy allies" at a reception in the ISPOR Booth!
Discussion Groups
A Debate on How Best to Value and Pay for Cell and Gene Therapies: Should Cost Savings Be Shared With the Health System and Society?
Accounting for Equity in Health Technology Assessment
Moderator
Melanie Whittington, PhD, MS
Institute for Clinical and Economic Review, Boston, MA, USA
Dr. Whittington is the Director of Health Economics at the Institute for Clinical and Economic Review (ICER). In her role, she leads the cost-effectiveness analyses and potential budget impact analyses within ICER reviews, directs the content within the Interactive Modeler™, and advances the field of health economics by developing innovative methods with applications for value assessment.
Wed May 10
7:00 AM - 8:00 AM
Morning Coffee Service
7:00 AM - 1:00 PM
Registration Hours
Sponsored by Carelon Research
8:00 AM - 9:00 AM
Breakouts: IP, WS, & OBS
Quantitative Bias Analysis in Practice: Exploring the Nuts and Bolts of Applications to Comparative Effectiveness Studies
Digital Conference Pass
Level: Intermediate
Purpose: The objective of this session is to dig deeper than the common high-level presentations of quantitative bias analysis and explore the nuts and bolts of how they are conducted in practice. Description: Quantitative bias analysis (QBA) comprises a collection of approaches for modeling the magnitude and direction of systematic errors in data which cannot be addressed with conventional statistical adjustment. QBA is useful when working with imperfect real-world data where uncertainty about bias often arises due to substantial missing covariate data, limited synthetic control arm data, or incomplete outcome data. While QBA is gaining in popularity, there has been little guidance on the nuts and bolts of applying QBA to real-world data. The session will start with a brief introduction to QBA and its potential role for medical decision-making (Thorlund, 5 minutes). The nuts and bolts of QBA applied to unknown population-level confounders will be illustrated using two examples in advanced non-small cell lung cancer with substantial missing covariate data that is ‘missing not at random’, and synthetic control arms with sample sizes too small for statistical adjustments. (15 minutes, Hsu). The nuts and bolts of applying QBA for extrapolation of long-term benefits from surrogate outcomes will be illustrated across oncology examples in a Bayesian framework, further discussing how to best incorporate information from external sources such as expert opinion or related literature (Thorlund, 15 minutes). Finally, reporting requirements for applied QBA as well as perspectives on which approaches would work in practice for HTA and medical decision-making will be covered. (Duffield, 15 minutes). Throughout, the audience will be prompted with questions about the likely impact of bias that QBA will reveal as well as the pros and cons of applying simple versus complex approaches. The session will conclude with a Q&A (10 minutes).
Speakers
Stephen Duffield, PhD, MD
NICE, Liverpool, United Kingdom
Stephen is an Associate Director 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
Grace Hsu, MSc
Cytel Canada Health Inc, Waltham, MA, USA
Grace is an Associate Director & Principal Statistician in Real World Evidence at Cytel with 9 years of experience in consulting and guiding project strategies. She has performed clinical trial simulations using Bayesian methods with adaptive designs. At Cytel, Grace provides statistical consulting and strategy generation regarding the curation of data and the application of advanced analytics to clinical and real-world data. Examples include comparative effectiveness analyses via synthetic control arms and other methods of indirect comparison, both for pharmaceutical research and for regulatory submissions. Grace also develops statistical communication for non-statisticians. Prior to joining Cytel, Grace was a Senior Statistician at MTEK Sciences (acquired by Cytel in 2019), a boutique analytics firm where she consulted with the Bill and Melinda Gates Foundation (BMGF) and provided simulations and adaptive trial planning services across large-scale BMGF-funded clinical trials and analysed a national-level registry.
Discussion Leaders
Kristian Thorlund, MSc, PhD
Cytel, 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 statistical methodologies that are now being used widely
Incorporating External Evidence Into Extrapolation: Making Sense of Seemingly Endless Options
Digital Conference Pass
Level: Intermediate
PURPOSE:
Producing plausible lifetime extrapolations is fundamental to appropriate decision-making within health technology assessment (HTA). However, continuing development of novel therapies and reliance on surrogate outcomes lead to increasingly immature data, complicating this essential task. Real-world evidence (RWE) and expert elicitation have been proposed as potential solutions to incorporate external evidence, with both ICER in the U.S. and NICE in the UK seeking to increase their use. But how exactly should external evidence be used? Whilst guidance provided so far by HTA bodies and in the literature is welcome; practical guidance to inform selection of methods is sorely lacking. Existing guidance includes frameworks which provide rationale for the intention behind using external evidence more widely, how to source such data (such as the NICE RWE framework), and the use of individual patient-level data from clinical trials for extrapolation (such as NICE TSD21). However, no guidance considers how external evidence should be incorporated into survival extrapolations for HTA. More research is needed to determine the best solutions; however, there is an urgent need for guidance on how to manage the seemingly endless options available to analysts in the interim.
DESCRIPTION:
This workshop will take the first steps towards development of practical guidance. Bart Heeg will demonstrate how the DIVE framework can be used to transparently document developer considerations and how this fits with existing HTA guidance. Ash Bullement will provide an overview of methodological approaches in use and pros/cons based upon the results of a systematic review of methods literature. Brett McQueen will provide his insights on the use of RWE in a recent multiple myeloma appraisal in collaboration with ICER. Dawn Lee will seek audience input on a draft framework for decision-making based on her insights from attempted application of NICE guidance to a multiple technology appraisal in RCC.
Discussion Leaders
Robert McQueen, PhD
University of Colorado, Denver, CO, USA
R. Brett McQueen is an Assistant 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
Ash Bullement, MSc
ScHARR, University of Sheffield, Nottingham, NTT, United Kingdom
Ash is an Analyst at Delta Hat, a UK-based HEOR consultancy, and is also a part-time PhD student at ScHARR, University of Sheffield. Ash's PhD is focused on methods for survival extrapolation that make use of external evidence, particularly in the context of cancer treatments
Bart Heeg, MSc, PhD
Cytel, Rotterdam, ZH, Netherlands
Bart is Vice President HEOR at CYTEL. Bart has a PhD in health economics. He has over 20 years of experience in heath economic and outcomes consulting. He focusses on indirect treatment comparisons, surrogacy analyses and health economic modelling.
What Do Patients Consider Meaningful Change? Use of Qualitative Methods to Inform Meaningful Within Patient Change
Digital Conference Pass
Level: Intermediate
PURPOSE: Clinical outcome assessment (COA) endpoints in clinical trials can incorporate patient perspectives into drug development. However, it can be challenging to understand whether changes in these endpoints are meaningful to patients. This workshop focuses on qualitative methods to address this challenge.
DESCRIPTION: Workshop attendees will obtain knowledge of qualitative methods to inform meaningful within-patient change (MWPC). The workshop will review a) how to leverage patient input to inform MWPC, and b) how to choose and apply qualitative methods to interpret MWPC. Dr. Weinfurt (chair) will present challenges and current methods for interpreting COA endpoints (15 min). Dr. Daniels will provide the regulatory perspective regarding the role of qualitative methods and their use to inform MWPC (15 min), Dr. Burgess will describe different qualitative methods that can be considered to elicit patient input on MWPC (15 min), and Mr. Stokes will share key learnings from an industry perspective, inclusive of approaches to integrate and communicate multiple sources of evidence to support regulatory submissions (15 min).
Discussion Leaders
Kevin Weinfurt, PhD
Duke University, Durham, NC, USA
Kevin Weinfurt, Ph.D., is the James B. Duke Professor and Vice Chair of Research in the Department of Population Health Sciences at Duke University Medical Center and a faculty member of the Duke Clinical Research Institute. Dr. Weinfurt co-directs the Center for Health Measurement in the Duke University School of Medicine. He works part-time as a Special Governmental Employee with the U.S. Food and Drug Administration, developing guidance for the Patient-Focused Drug Development initiative. Dr. Weinfurt received his PhD in psychology at Georgetown University and did graduate work in the history of science and philosophy of mind at Linacre College, Oxford.
Discussants
Somali Burgess, PhD
Lumanity, Long Beach, CA, USA
Somali Misra Burgess, PhD, is a Senior Research Scientist on the Patient-Centered Research team at Evidera, a PPD business. Dr. Burgess draws from her over 19 years of consulting experience and three years of pharmaceutical industry experience to provide clients with the appropriate COA-related solutions. Prior to joining Evidera, Dr. Burgess was a senior director and PRO thought leader at Xcenda. Other positions Dr. Burgess has held include CEO and research director at Strategic Outcomes Services, a boutique consulting company that specialized in the development, validation, and evaluation of COAs and regulatory strategy; vice president in the endpoint development and outcomes assessment group at Adelphi Values; and, director in the GHEOR division at Allergan heading up the products/indications in Allergan Medical, Health, and Medical Dermatology. Dr. Burgess has extensive experience developing and demonstrating value arguments for new and existing products/indications, COA evaluations, questionnaire development and validation, endpoint development, patient-centric research, and global regulatory strategies and submissions. Her therapeutic areas of expertise include, but are not limited to, aesthetic indications, medical dermatology, gastrointestinal conditions, urology, ophthalmology, and respiratory conditions. Dr. Burgess has experience developing regulatory strategies and supporting submissions to Asia-Pacific regulatory agencies. Dr. Burgess has a PhD in pharmacoeconomics and outcomes research from the College of Pharmacy, University of Georgia. She is a member of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and the International Society for Quality of Life Research (ISOQOL).
Selena Daniels, PharmD, PhD
US Food and Drug Administration, Silver Spring, MD, USA
Dr. Selena Daniels serves as a Team Leader in the Division of Clinical Outcome Assessment at the FDA. She leads 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 for almost five years, 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.
Jonathan Stokes, MBA
Abbvie, Madison, NJ, USA
Jonathan Stokes
Senior Director
Patient-Centered Outcomes Research
AbbVie, United States
jonathan.t.stokes@abbvie.com
Jonathan Stokes, MBA, is a Senior Director in Patient-Centered Outcomes Research overseeing neurology, ophthalmology, and specialty therapeutic areas at AbbVie. With 18 years of experience conducting patient-centered research and generating patient experience data, Jonathan has a devoted interest in understanding and integrating the patient voice in drug development. He has extensive experience in designing, evaluating, and implementing clinical outcome assessments (COA) to assess treatment benefit and has made significant contributions to major product approvals and launches, as well as the development of multiple novel patient-reported outcome (PRO) instruments for use in clinical trials and real-world practice. He also serves as a volunteer researcher for the Barth Syndrome Foundation. Jonathan holds a MBA from Northeastern University.
Routing RWE Sources in HTA Submissions When Standard of Care Is Not Established: How to Robustly Validate Your Uncertainties
Digital Conference Pass
Level: Intermediate
PURPOSE:
Health technology assessment (HTA) bodies make decisions on the added benefit of new products compared to standard of care (SoC). However, in some rare diseases and highly selected populations, SoC may be not established. Although the value of real-world evidence (RWE) for HTAs is well discussed, the selection of appropriate RWE sources and its modelling assumptions when no SoC is established, and how it may impact HTA decisions, is unclear. Recent RWE frameworks can inform some of these steps, but decision uncertainty remains.
DESCRIPTION:
Dr. Dawoud will introduce the HTA challenges when direct comparative clinical data are lacking due to absence of established SoC and how RWE may address this evidence gap; two recent NICE submissions in the same rare disease and target population (10 minutes). Dr. Kent will present the RWE sources submitted by the companies and their justifications. The audience will be asked to take the decision-making perspective and hypothesize HTA critiques on the submitted RWE evidence and its quality (15 minutes). Dr. Hernandez will present the challenges from the industry perspective, the HTA critiques in these case studies, and the recommendations; the audience will be asked to take the industry perspective and consider if and how to address the challenges and HTA suggestions to resolve RWE-related uncertainties (15 minutes). Lastly, Dr. Sarri will present how current RWE frameworks could guide RWE decisions, and will prompt questions to the audience on areas such as unbiased selection, data provenance, suitability, and robust analysis that may increase trust in RWE estimates for SoC (15 minutes). The workshop will conclude with an audience discussion on opportunities and challenges of using RWE frameworks in HTA submissions when SoC is not defined (5 minutes). This workshop will benefit statisticians, epidemiologists, payers and industry.
Discussion Leaders
Dalia Dawoud, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Dalia Dawoud, PhD, is Associate Director at the National Institute for Health and Care Excellence (NICE). She holds MSc in Economic Evaluation in Health Care from City University London and PhD in pharmaceutical policy and economics from King’s College London.
She has long experience in using economic evaluation in clinical guidelines development and health technology assessment (HTA), gained through working on NICE Clinical Guidelines as well as technology appraisals. Dalia’s research interests are focused on the advanced methods of evidence synthesis and use in economic models and the use of real-world evidence to inform drug development and health care decision making. Dalia currently has overall responsibility of overseeing the delivery of NICE allocated tasks on a portfolio of IMI and Horizon 2020 funded research projects including EHDEN and HTx. She is also responsible for the operational delivery of NICE HTA Innovation Lab (HTA Lab) activities. She is widely published in the field of pharmaceutical policy and pharmacoeconomics. She also serves as Associate Editor for ISPOR journal Value in Health and as Associate Editor for Pharmacoeconomics and Outcomes Research for Elsevier’s journal Research in Social and Administrative Pharmacy. Dalia also holds adjunct position as Associate Professor at the Faculty of Pharmacy, Cairo University.
Discussants
Luis G. Hernandez, PhD MPH MSc
Takeda Pharmaceuticals America, Inc., Westford, MA, USA
Luis Hernandez, PhD MPH MSc, is the Head of Global Health Economics at Takeda Oncology. His team is responsible for developing the health economics strategy and evidence to demonstrate the value of Takeda innovative and transformative oncology medicines for patients, payers and society. Also, to develop valuable partnerships across Industry, and with patient organizations, payers, and academia, to shape the environment in relevant health economics and outcomes research topics.
Dr. Hernandez has extensive experience in health economics and outcomes research, evidence generation planning, market access, and cross functional team leadership and management.
Prior to joining Takeda, Dr. Hernandez spent 13 years at Evidera (a business within Pharmaceutical Product Development, LLC, [PPD] a leading global contract research organization [CRO]) where he spearheaded health economics evidence generation and dissemination to support the optimal value proposition for pipeline, launch, and lifecycle assets. During his tenure, Dr. Hernandez held several leadership roles including Senior Director and Senior Scientist heading Evidera’s health economics modeling and simulation in Waltham, Massachusetts, USA.
Between 2009 and 2010, Dr. Hernandez was a full time professor of Probability, Statistics, Discrete Event Simulation, and Decision Analysis for undergraduate and graduate students at Universidad de los Andes (Bogota, Colombia) for the Industrial Engineering Department and the Executive MBA program.
Dr. Hernandez holds a PhD in Health Economics from the University of Groningen (the Netherlands), a Master’s in Public Health from Tufts University (Boston, Massachusetts, USA), and Master’s and Bachelor’s degrees in Industrial Engineering from Universidad de los Andes (Bogota, Colombia).
Seamus Kent, PhD
Flatiron Health, Amsterdam, NH, Netherlands
Seamus is a Senior Adviser in HTA at Flatiron UK. He was previous a Senior Adviser in Data & Analytics at NICE where he led the development of NICE's Real-world evidence framework.
Grammati Sarri, PhD, MSc, DiDS
Cytel, London, United Kingdom
Grammati has 15+ years’ experience in epidemiology studies, evidence synthesis and decision making from her prior roles in academia, health technology assessment (NICE, UK) and private health consultancies. Grammati has strong methodological experience in generating impactful evidence from different sources (clinical trials, real-world evidence, economic studies), clearly communicating findings in an impactful way and using complex analytical methods for health care-decision making. Grammati currently holds the position of Chair of the ISPE comparative effectiveness research special interest group and is a topic co-editor for the Journal of Frontiers in Pharmacology.
From Concept to Practice: How to Tactically Design a Feasible Outcomes-Based Contract in Oncology?
Digital Conference Pass
Level: Intermediate
PURPOSE:
The purpose of this workshop is to drill down on specific components intrinsic to the tactical design of a successful outcomes-based contract in oncology, address payer uncertainties, and accelerate patient access to innovative medicines.
DESCRIPTION:
The concepts of value-based contracting (VBC) have been made clear in the literature, however there is a gap between these concepts and executional realities. The discussion leader (Mark Trusheim) will start by introducing the concept of VBCs, narrowing quickly to focus on outcomes-based contracts (OBCs) in oncology, including definitions, pros and cons, and examples of how they have been used. (7 mins) The industry lead (Jamie Foley) will provide their perspective, including rationale and practical guidance for building OBCs, including internal and external barriers and best practices. (7 mins) Finally, the payer leads (Ann Nguyen and Ron Potts) will provide their perspective, including preferred engagement, considerations for trust and transparency, and common challenges that limit acceptance and execution. (7 mins) Next the discussion leader will provide a mock launch scenario of an oncology medicine with details to support the exploration of key tactical considerations for building an OBC. (30 mins) In real-time, the audience will be engaged through various polling questions on three key topics: 1. Contract metric selection and eligible population 2. Data collection challenges 3. Contract reconciliation and adjudication, e.g., aligning contractual expectations with the reality of imperfect data collection, etc. After each question, the presenters will provide their respective perspective and comments on the audience’s responses. The workshop will conclude with practical considerations from each presenter, including how industry and payers can work better together to design feasible OBCs. (9 mins) Stakeholders that would benefit from this session: Payers, industry, researchers and consultants who are interested in learning more about the feasibility aspects and execution/implementation challenges of VBCs, payment innovation and outcomes tracking.
Discussion Leaders
Mark Trusheim, MS, BS
Massachusetts Institute of Technology, Cambridge, MA, USA
Mark Trusheim is Strategic Director, NEWDIGS at Tufts Medical Center where he also co-leads the Financing and reimbursement of Cures in the US (FoCUS) Project.
Mark’s research focuses on the economics of biomedical innovation, especially precision financing for patient access, precision medicine, adaptive pathways, platform trials, biosimilars, and digital health advances. Mark has served as a Special Government Employee for the FDA’s Office of the Commissioner.
Mark is also President of Co-Bio Consulting, LLC. His career has spanned policy as the President of the Massachusetts Biotechnology Council, diagnostics as founder of Cantata Labs, genomics as President of Cereon Genomics, eHealth as Vice President of Monsanto Health Solutions, managed care marketing at Searle Pharmaceuticals, and big data at Kenan Systems.
He holds degrees in Chemistry from Stanford University and Management from MIT.
Discussants
Jamie Foley, MA, Economics
Takeda, Cambridge, MA, USA
LinkedIn profile: https://www.linkedin.com/in/jamie-foley-5304a142/
Ann Nguyen, PharmD
UnitedHealthcare, West Hills, CA, USA
Ron Potts, MD
6 Degrees Health, Portland, OR, USA
Who Should Pay for Medication Adherence Enhancing Interventions and What Are They Paying for?
Digital Conference Pass
ISSUE:
There are potentially two payers for medication adherence enhancing interventions, the payer and the pharmaceutical manufacturer. While the improved health outcome is of utmost interest to the payer, increased adherence is of significance to the pharmaceutical manufacturer. Though both payer and manufacturer have a stake in improved medication adherence, it is yet unclear who would or should be responsible for paying MAEIs.
OVERVIEW:
The responsibility of medication adherence falls on patients (Brown, 2011) with the providers often being held accountable for lack of adherence (Cutler, 2010). Research has shown that MAEIs can improve adherence (DeFulio, 2012) and both Defulio and Bosworth (2011) call for research on the long-term effectiveness of the patient pay-for-performance model. Embedded in this is the need to determine who should pay for MAEIs and whether they should be incentivized. Kardas (2022) suggests that it may be reasonable for healthcare systems to “assign special funds” to assist in improving adherence. Others suggest that the pharmaceutical manufacturers should pay for these interventions (Healthprize, 2022). The other question is, what is being paid for? The DeFulio (2012) review suggests MAEIs are worthwhile, but Barankay (2020) indicates that while adherence improves, clinical outcomes may not. This panel will examine the question of what MAEIs should be paid for and who should be paying for them from three different perspectives: the payer, the provider, and the manufacturer. The payer will discuss why they should/should not be responsible for paying for MAEIs as well as what they would be paying for. Similarly, the manufacturer discuss what they would consider a reasonable outcome to pay for, should they be paying for it. Finally, the provider will offer their insights into how either payment model would impact their practice and how effective they think each approach to adherence would be for their patients.
Moderators
Bijan Borah, PhD
Mayo Clinic College of Medicine and Science, Rochester, MN, USA
Dr. Bijan Borah is a Professor of Health Services Research at the Mayo Clinic College of Medicine and Science. He is also the Endowed Scientific Director of the Clinical and Economic Evaluation Program within the Kern Center for the Science of Health Care Delivery at Mayo Clinic. He is also a leadership team member in the Special Interest Group for Medication Adherence and Persistence within ISPOR.
Panelists
Tamas Agh, MSc, PhD, MD
University of Pécs & Syreon Research Institute, Budapest, Hungary
Tamás Ágh MD MSc PhD DrHabil is a principal researcher of Syreon Research Institute and a research associate professor at the Center for Health Technology Assessment and Pharmacoeconomic Research, University of Pecs. With around 15 years of experience in health economics and outcome research, he developed expertise in the fields of medication adherence, patient reported outcomes, health technology assessment, and evidence synthesis. Dr. Ágh is a core group member of the ISPOR Medication Adherence and Persistence Special Interest Group and the ENABLE (European Network to Advance Best practices & technoLogy on medication adherencE) Cost Action.
Przemyslaw Kardas, MD, PhD
Medical University of Lodz, Lodz, Poland
Prof. Przemyslaw Kardas, MD, PhD is a founder and Director of Medication Adherence Research Center at the Medical University of Lodz, Poland. He is also Professor of Family Medicine and the Head of the Department of Family Medicine at the Medical University of Lodz, Poland. His professional activities focus on patient adherence to medication, management of chronic conditions, active and healthy ageing, Big Data and eHealth. He is the author of a number of peer-reviewed scientific publications and conference presentations, as well as four monographs. From 2020, he is a Vice-chair of Work Group 3 ‘Sustainable implementation of adherence enhancing technologies’ of the COST Action ENABLE.
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
8:30 AM - 10:00 AM
Breakfast Break With Exhibitors
Meet in the Exhibit Hall for a final networking opportunity with exhibitors. Enjoy a breakfast break provided by ISPOR with coffee and hot/cold food options.
8:30 AM - 11:30 AM
Poster and Exhibit Hall Open
Poster Session 5
Live
8:45 AM - 9:45 AM
Podium Session
PROs and Patient Experiences in Oncology and Rare Diseases
This session explores subjects and methodology that are wide-ranging, but that all are looking at the patient experience of disease and treatment beyond survival, which is often the focus of data collection and analysis. The studies explore treatment barriers, surrogate endpoints, patient preferences, and biomarkers. Seating is limited.
Moderator
Mark Larkin, PhD, MA
Vitaccess Ltd, Oxford, United Kingdom
Mark is the chief executive office & founder of Vitaccess. His background is HEOR and market access, and most recently at Vitaccess has specialized in the design and implementation of prospective digital RWE studies. Mark founded Vitaccess to combine patient-centred research expertise with the company's Vitaccess Real(tm) data capture platform (clinical data as well as patient- and caregiver-reported data). Studies co-created with PAGs and KOLs offer real-time insights, globally and rich publication opportunities.
P52: Barriers to Reducing Time to Diagnosis and Treatment in Rare Disease
8:45AM - 9:00AM
Everett W1, Tatroe S2, Feng L3, Strumph P4, James J3, McNeil-Posey K3, Mungin B3, Rowe C3, Kielhorn A5
1Atlas Clarity, San Francisco, CA, USA, 2TwelveStone Health, Murfreesboro, TN, USA, 3Alexion, AstraZeneca Rare Disease, Boston, MA, USA, 4Seraxis, Germantown, MD, USA, 5Alexion, AstraZeneca Rare Disease, Rockland, MA, USA
OBJECTIVES:
For patients with rare diseases, it can take years to receive an accurate diagnosis and subsequent treatment. In underserved communities, clinical, social, and economic factors may further lengthen these time horizons. The goal of the ASPIRE study was to identify, evaluate, and better understand the barriers to timely diagnosis and treatment among patients with generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD).
METHODS:
As part of a landscape assessment for ASPIRE, 10 one hour-long, semi-structured interviews were conducted via telephone or videoconference with representatives from patient advocacy organizations (n=2), policy makers (n=2), payers (n=4), and providers (n=2) from April to June 2022. The interviews aimed to elicit expert knowledge and experience about diagnosing and treating gMG and NMOSD from different perspectives.
RESULTS:
Multiple barriers to diagnosis and treatment were identified. Patients reported to patient advocacy organizations barriers that included lack of access to a specialist, inadequate insurance coverage for visits, limited or no access to telemedicine (lack of internet access), and difficulties getting time off work. Policy makers and payers highlighted a lack of priority for rare diseases relative to other diseases, long wait times to access clinical centers of excellence, and lack of adequate insurance. For providers, key challenges included limited clinical knowledge of gMG and NMOSD, a shortage of trained specialists, and implicit gender, racial, and ethnic biases. Improving the referral process, availability of diagnostic algorithms, access to care within underserved communities, education, and policy changes that link reimbursement to equity of care were highlighted as ways to reduce these barriers in treating gMG and NMOSD.
CONCLUSIONS:
This study identified multi-dimensional barriers that may prolong the time to diagnosis and treatment for patients with gMG and NMOSD. The contribution of each of these barriers to delaying diagnosis will be further elucidated in future studies.
P49: Evaluation of Surrogate Endpoints (SES) for Previously Untreated Unresectable or Metastatic Melanoma (MMEL): Analyses from a Longitudinal Electronic Health Record Database in the United States (US)
9:15AM - 9:30AM
Leung L1, Mohr P2, Serafini P1, Kanters S1, Pourrahmat MM1, Moshyk A3, Srinivasan S3, Kurt M3
1Evidinno Outcomes Research Inc., Vancouver, BC, Canada, 2Elbe Klinikum Buxtehude, Buxtehude, NY, Germany, 3Bristol Myers Squibb, Lawrenceville, NJ, USA
OBJECTIVES: To evaluate progression-free survival (PFS), time-to-progression (TTP), time-to-next-treatment-or-death (TNTD), and time-to-treatment discontinuation (TTD) as SEs for overall survival (OS) in previously untreated unresectable or mMel in real-world settings.
METHODS: Individual patient data was acquired from the US Flatiron Health database comprising >265 cancer clinics. Individual-level correlation between each SE and OS was estimated using Spearman’s rank correlation. To calculate treatment effect correlations, in absence of randomized settings, patients were allocated to 12 non-overlapping clusters with synthetic treatment and control arms using propensity score matching to balance baseline age, race, sex, ECOG, and BRAF mutation status. Pearson’s correlation coefficient, estimated by weighted linear regression (WLR), was used to measure the treatment-effect correlation for each SE. Predictive performance of WLR was assessed via cross-validation. Correlation measures are reported as (estimate; 95% CI).
RESULTS: 489 patients were eligible for the individual-level analysis and 428 for the cluster-level analysis, with a median of 36 patients per cluster (range: 18-46). Patients were all diagnosed post-2011, mostly stage IV (58.5%) and BRAF wild-type (53.2%). Among patients assigned to clusters, most received either single or combinations of immunotherapy, or BRAF-MEK inhibitors (84.8%). Individual-level correlations were estimated as (0.62; 0.54-0.72) for PFS, (0.67; 0.56-0.76) for TNTD, (0.60; 0.43-0.74) for TTD, and (0.47; 0.36-0.58) for TTP. Correlations between treatment-effects were (0.78; 0.53-0.97) for PFS, (0.77; 0.36-0.98) for TNTD, (0.87; 0.69-0.97) for TTD and (0.67; 0.36-0.97) for TTP. In cross validation, the 95% prediction intervals of OS hazard ratios included their reported counterparts in >83% of the clusters for each SE.
CONCLUSIONS: Correlations between the candidate SEs and OS were moderate in this study. Predictions from the derived surrogacy equations may enable earlier assessment of real-world treatment effect on OS for previously untreated unresectable or mMel; however, classification of real-world data in small clusters may result in wide uncertainty around estimates.
P51: Evaluating the Patient Experience with CD55 Deficiency with Hyperactivation of Complement, Angiopathic Thrombosis, and Protein-Losing Enteropathy (CHAPLE) Disease: A Deadly Ultra-Rare Pediatric Disease
9:00AM - 9:15AM
Litcher-Kelly L1, Ollis S1, Yaworsky A1, Baris Feldman H2, Harari O3, Ozen A4, Jalbert J5
1Adelphi Values, Boston, MA, USA, 2Tel Aviv Sourasky Medical Center, and Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel, 3Regeneron Pharmaceuticals, Inc., Tarrytown, NY, USA, 4Marmara University, Istanbul, Turkey, 5Regeneron Pharmaceuticals, Inc., Sleepy Hollow, NY, USA
OBJECTIVES: To identify key disease-related signs and symptoms (s/s) of CHAPLE disease, an ultra-rare, newly discovered genetic disease associated with severe complications causing premature death.
METHODS: Sixty-minute within-trial qualitative interviews were conducted with patients who have CHAPLE disease and/or their primary caregivers at the screening and week 24 visit of a trial of pozelimab treatment (NCT04209634). Patients ≥8 years old were the interview’s primary respondent; the primary respondent for patients <8 years old and those with cognitive impairment was the caregiver. If both the patient and their caregiver participated in the interview, the unit of analysis was the interview. Semi-structured interview guides were used by trained interviewers to explore the s/s of CHAPLE disease and, at screening, to identify the patient’s most bothersome s/s (MBS). Interviews were preferentially conducted in person. All were conducted in the dyad’s native language and were audio-recorded, transcribed, anonymized, and translated into English for coding and analysis.
RESULTS: Ten patients were enrolled in the trial (median age at screening: 8.5 years [range: 3–19], 60% female, 70% interviewed in Turkey); 50% of interviews were conducted as a dyad at screening and 60% were conducted as a dyad at week 24. Thirty-one CHAPLE-related s/s were reported during the interviews. The core s/s of CHAPLE were (% having experienced s/s): abdominal pain (100%), facial edema (100%), peripheral edema (100%), diarrhea (100%), vomiting (100%), and nausea (90%). The MBS reported were abdominal pain (n=9), and facial edema (n=1); with n=7/10 and n=2/10 patients identifying these s/s as most important to improve with treatment, respectively. Other s/s experienced by ≥3 patients included fever, bowel incontinence, and headaches.
CONCLUSIONS: Patients with CHAPLE disease experience a core set of six s/s with the most critical aspect being abdominal pain, suggesting a relatively homogeneous experience of disease.
P50: Using Biomarker Change and Treatment Adherence to Predict Risk of Relapse Among Chronic Myeloid Leukemia Patients Who Are in Remission
9:30AM - 9:45AM
Montano-Campos JF1, Haupt EC2, Hahn EE2, Radich J3, Bansal A1
1University of Washington, Seattle, WA, USA, 2Southern California Permanente Medical Group, Pasadena, CA, USA, 3Fred Hutchinson Cancer Research Center, SEATTLE, WA, USA
OBJECTIVES:
Decision-making in chronic myeloid leukemia (CML) is based on risk scores that incorporate information collected at diagnosis. However, there is evidence that the rate of change in tumor biomarker levels after treatment initiation and adherence to treatment are two key factors that predict the risk of relapse among patients in remission. We developed a risk prediction model incorporating these two longitudinal features that may be key prognostic factors.
METHODS:
We used EHR data from an integrated health system on 443 patients who were diagnosed with CML between 2007-2019 and monitored for relapse after remission. We fit a Cox model with time to relapse as the outcome. The predictors included log biomarker change between the first two collections (~3 months apart) after treatment initiation, medication adherence in the first year of treatment, race, sex, BMI, age, and smoking behavior. Medication adherence was measured approximately as the proportion of days in which a patient had access to medication in the first year, and dichotomized into high (>80%) versus low (<=80%) adherence. We evaluated the accuracy of our model using the area under the ROC curve (AUC).
RESULTS:
Compared to patients who experienced no biomarker change, the risk of relapse was significantly higher in patients with increased biomarker levels and significantly lower in patients with decreased biomarker levels (HR=4.91, 95% CI (1.74,13.85); HR=0.45, 95% CI (0.22,0.91), respectively). Furthermore, the risk of relapse for patients with high medication adherence was 62% lower than patients with low adherence (HR: 0.38, 95% CI (0.20,0.72)). Our model was highly accurate at predicting relapse within 6 months post-remission, with an AUC of 0.80 (95% CI: 0.73-0.99).
CONCLUSIONS:
We incorporated key longitudinal features to develop a high-performing risk prediction model in CML. Upon further external validation, this model may be used in clinical practice for identifying high-risk patients for targeted intervention.
9:15 AM - 9:45 AM
Exhibit Hall Theater
Bench to Practice: Exploring Real-World Evidence of COVID-19 Vaccines
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Description to be provided.
Seating is limited.
Sponsor: Veradigm
Sponsor
Veradigm
Speaker
Mac Bonafede, PhD, MPH
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.
James Mansi, PhD
Moderna, Cambridge, MA, USA
Nital Patel, MS, MBA
Veradigm, Chicago, IL, USA
10:00 AM - 11:00 AM
Breakouts: IP, WS, & OBS
Improving Coordination Between the FDA and CMS: Exploring Potential Policy Solutions to Improve Patient Access to Drugs Granted Accelerated Approval
Digital Conference Pass
ISSUE: Recent events, including controversies around the approval of aducanumab (Aduhelm), the monoclonal antibody for early Alzheimer’s disease, highlight the problems that can arise because the FDA and CMS lack mechanisms to coordinate their decision making, particularly around promising technologies for serious illness. For drugs approved through expedited pathways, there is a growing evidence gap between FDA and CMS decisions, highlighting the limited policy tools that are currently available to either FDA or CMS to responsibly navigate that space. We will discuss ways in which the FDA and CMS might better coordinate their policy mechanism for promising technologies in order to produce more transparent, predictable, evidence- and outcomes-based processes for new medicines. We will debate the merits of different policy strategies, including discussion of intended and unintended consequences and barriers to implementation.
OVERVIEW: The moderator will give a 10 minute presentation on background information for this issue, including a brief description of the aducanumab case study. The panel will offer their perspectives on different policy solutions that could be used to achieve coordination, including the potential benefits and harms of each. Tunis, formerly chief medical officer at CMS will address options including: improved communication between the Agencies; early advice to product developers from CMS; use of real world evidence; reform of coverage with evidence development; innovative payment mechanisms; and others. Kaltenboeck, who formerly worked on the Senate Finance Committee, will discuss potential legislative solutions. Health policy makers and researchers will be the best audience for this panel because it will apply directly to their work, but the panel will also be of interest to individuals working in or with the drug industry since they navigate both the drug approval and reimbursement processes.
Moderators
Peter Neumann, ScD
Tufts University, 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.
Panelists
Gregory Daniel, PHD, MPH
Eli Lilly and Company, Washington, DC, USA
Anna Kaltenboeck, MA
ATI Advisory, New York, NY, USA
Anna Kaltenboeck is Head of the Prescription Drug Reimbursement Practice at ATI Advisory.
Prior to joining ATI, she was Senior Health Advisor on the Senate Committee on Finance, where she led on drug pricing issues, including Medicare negotiation. In her previous role as Program Director for the Drug Pricing Lab at Memorial Sloan Kettering Cancer Center, she studied how reimbursement policies shape the market for prescription drugs.
Ms. Kaltenboeck received her undergraduate and master’s degrees in Economics from Tufts University, and her MBA from Yale University.
Sean Tunis, MD, MSc
Tufts University, Boston, MA, USA
Sean Tunis, MD, MSc. is a Principal with Rubix Health where he consults with public and private sector organizations on health technology issues at the interface of regulatory and reimbursement policy, market access, comparative effectiveness, outcomes measurement and health technology assessment. He is also a Senior Fellow in the Tufts Center for the Evaluation of Value and Risk in Health. Dr. Tunis also serves as a Mentor-in-Residence at Johns Hopkins Tech Ventures, helping early stage companies to develop their initial reimbursement strategy and to align their clinical development programs with payer and regulatory requirements.
Are There Clear Criteria for Whether Real-World Data Sources Are Regulatory-Grade?
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ISSUE:
Are there clear criteria for whether real-world data sources are regulatory-grade?
OVERVIEW:
Marc Berger will open the session. (5 minutes) Jacqueline-Corrigan Curay will discuss how the FDA views RWD quality as it considers using RWE in its decision making with reference to the several relevant FDA guidance documents. (12.5 minutes) Jesper Kjaer will discuss the EMA approach to the issue with reference to the Eu Data Quality Framework and Good Practice Guide for the use of Metadata in the Catalogue of Real-World Data Sources. (12.5 minutes) William Crown will review efforts to harmonize RWD quality criteria as well as provide his thoughts about standards to assess whether a particular RWD source may be fit-for-purpose. (10 minutes) Panel Discussion (20 minutes)
Moderators
Marc Berger, MD
Marc L. Berger, LLC, New York, NY, USA
Marc L. Berger, MD, is a semi-retired, part-time consultant and scientific advisor. Until July 2017, he was Vice President, Real World Data and Analytics at Pfizer, Inc. Marc has held senior-level positions in industry including Executive Vice President and Senior Scientist at OptumInsight; Vice President, Global Health Outcomes at Eli Lilly and Company; and Vice President, Outcomes Research and Management at Merck & Co., Inc. He was a temporary employee of CMS from July-December 2022.
Marc is an ISPOR Special Advisor for Real World Evidence and a member of the Duke-Margolis Center for Health Policy Real-World Evidence Collaborative. He has written or co-written more than 130 peer-reviewed articles, book chapters, and other publications on a range of topics including health services research, outcomes research, health economics, and health policy. He received the Donabedian Lifetime Achievement Award from ISPOR in 2019.
Panelists
Jacqueline Corrigan-Curay, J.D., M.D
U.S. Food and Drug Administration, Washington DC, DC, USA
William Crown, PhD
Brandeis University, Waltham, MA, USA
Dr.Crown is a Distinguished Research Scientist in the Heller School of Social Policy and Management, Brandeis University. He is an internationally recognized expert in real world data analysis, focusing upon research designs and statistical methods for drawing causal inferences from transactional health care datasets such as medical claims and electronic health records. Dr. Crown was 2013-14 President of ISPOR and currently co-chairs the ISPOR Task Force on Machine Learning. He is particularly interested in the intersection of machine learning and causal inference methods, as well as transparency in the conduct and reporting of empirical health care research.
Jesper Kjær, MSc
HMA-EMA, København S, Denmark
Director of The Data Analytics Centre at the Danish Medicines Agency and co-chair of HMA / EMA Big Data Steering Group
Boosting Caregivers’ Role in Measuring the Value of New Treatments in Rare Diseases
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ISSUE:
Caregiver engagement in valuing new treatments in rare diseases is limited, and few HTA agencies incorporate caregiver burden when evaluating new treatments. Is the role of the caregiver considered a vital resource when measuring the value of new treatments for rare diseases? What specific ways can caregivers inform rare disease drug development and approval?
OVERVIEW:
Olivier Chassany will introduce the path to acknowledgment of the value of clinical outcome assessments (COAs) as study endpoints. Studies highlight the caregiver burden, but this burden in all physical, mental, emotional, social, and productivity domains is like an “invisible labor that is not captured”. It is time to value the caregiver’s perspective of health products in HTA. Selena Daniels will discuss leveraging the use of caregiver input in rare diseases to inform the COA measurement strategy in medical product development and regulatory decision-making. Terry Jo Bichell will talk about the potential to approve a drug based on the improvement in quality of life (QoL) of the family and whether there is proof that the QoL of the patient has improved. When it is difficult to measure improvements in patients, measuring the indirect improvements on the family could be an alternative way to evaluate biomarkers in the patient. Laurie Lambert will discuss CADTH’s Learning by Doing Period on optimizing the integration of RWE into decision-making in rare diseases. Exploring how the perspectives of a variety of stakeholders, including caregivers, can be used to assess the value of both drugs and health technologies. CADTH is establishing guidance for the reporting of RWE that will be submitted for the purposes of regulatory and HTA submissions and the generation of RWE. She will discuss the lessons learned from a recently published multistakeholder report concerning the treatment of pediatric cancer. The audience will interact throughout the session.
Moderators
Olivier Chassany, MD, PhD
Patient-Centered Outcomes (PROQOL), University Paris Cité, Paris, 75, France
Professor of Therapeutics (Health Economics Clinical Trial Unit, AP-HP Paris hospitals, France), specialist in gastroenterology, with a long experience in developing Patient-Reported Outcomes (PRO) questionnaires. Involved for more than 20 years in the expertise of dossiers for EMA and French Drug Agency and for more than 30 years in Ethics Committees. Co-author of the EMA Reflection Paper on Health-Related Quality of Life. Deputy director of an academic research team on epidemiology and PRO (Université Paris Cité, Inserm). Past chair of the ISPOR SIG Clinical Outcomes Assessment (COA).
Panelists
Terry Jo Bichell, PhD
COMBINEDBrain, Brentwood, TN, USA
Terry Jo Bichell is the Founder/Director of COMBINEDBrain, a patient-advocacy led organization representing 65 member organizations, which was formed in 2019 with a mission to identify outcome measures and biomarkers for neurodevelopmental disorders. Dr. Bichell is the mother of an adult son with Angelman syndrome. She earned a PhD in neuroscience from Vanderbilt University in 2016. Dr. Bichell has published studies on clinical aspects of Angelman syndrome, Gabrb3 mutation, Ube3a gene expression, circadian rhythms, and conceptual models of disease for STXBP1 and SLC6A1-related disorders.
Selena Daniels, PharmD, PhD
US Food and Drug Administration, Silver Spring, MD, USA
Dr. Selena Daniels serves as a Team Leader in the Division of Clinical Outcome Assessment at the FDA. She leads 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 for almost five years, 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.
Laurie Lambert, PhD
Canadian Agency for Drugs and Technologies in Health, Ottawa, ON, Canada
Laurie Lambert completed a Master of Public Health in the epidemiology and biostatistics stream at the University of Western Australia. She then received a four-year Australian National Health and Medical Research Training scholarship to pursue her PhD. From 2004 to 2020, Laurie was the scientific coordinator of the Cardiovascular Evaluation Unit at INESSS where the focus was on evaluations of new technologies in the real-world context. In August, 2020 Laurie began a new chapter in her career as Lead of Real World Evidence at CADTH.
Podium Session
Impact of Adherence and Intervention on Health Outcomes and Cost
This session will highlight the impact of medication adherence intervention programs on patient health outcomes and healthcare costs with a focus of health service patients with chronic illness and or cardiovascular disorders. Seating is limited.
Moderator
Manjiri Pawaskar, PhD
Merck & Co., Inc., Norht Wales, PA, USA
P57: The Role of Anticoagulants Clinics in Anticoagulant Therapy Initiation for Incident Atrial Fibrillation
10:45AM - 11:00AM
Ndai A1, Smith S2, Svensson M2, Vouri SM3, Reise R2
1University of Florida, Gainesville , FL, USA, 2University of Florida, Gainesville, FL, USA, 3University of Florida, College of Pharmacy, Gainesville, FL, USA
OBJECTIVES:
Although direct oral anticoagulants (DOACs) are more effective and safer than warfarin, for patients in the US, depending on the prescription drug coverage, the out-of-pocket costs with DOACs are significantly higher, which could impact the choice between warfarin and DOACs. We hypothesized that clinics with specialized pharmacists in anticoagulation clinics (ACs) who assist patients in providing information on prescription coverage and reviewing the cost implications of various anticoagulants are more likely to initiate patients on warfarin compared to clinics without ACs.
METHODS:
Using electronic medical records, we conducted a cohort study in patients with incident AF and without a mechanical heart valve and initiated on anticoagulation therapy between January 1, 2019, and October 31, 2022, at the University of Florida Health Physicians (UFHP) Practice Sites. Exposures: Patients newly diagnosed with AF in clinics with ACs versus similar patients in clinics without ACs. Outcomes: Within 360 days of an index AF diagnosis and started on anticoagulation therapy (AT), the outcome of interest is the relative risk of being initiated on warfarin compared to DOAC.
RESULTS:
Among 2150 patients newly diagnosed with AF and initiated on anticoagulant 250 (11.6%) were initiated on warfarin, and 1900 (88.4%) were initiated on DOACs. Of the 250 patients newly initiated on warfarin, 60 (24.0%) were in clinics with AC, and 190 (76.0%) were in clinics without ACs. After adjusting for the CHA
2DS
2-VASc score, insurance type, and year of AF diagnosis, patients in clinics with ACs have a 74% higher risk of being initiated on warfarin instead of DOACs (RR 1.74 [95% CI, 1.34 – 2.27]) compared to patients in clinics without ACs.
CONCLUSIONS:
The findings suggest that services such as routine patient management and reviewing the out-of-pocket costs provided by specialized pharmacists significantly favor the choice of warfarin over DOACs from patients' perspectives.
P59: Medication Adherence and Subsequent Year Payer Medical Costs Among Medicare Advantage Beneficiaries: Differences across Quality Measures
10:30AM - 10:45AM
Parikh M1, Nsiah I2, Campbell P3, Ramachandran S4, Karmarkar T5, Castora-Brinkley M6, Black H5, Bentley J4
1Pharmacy Quality Alliance, Kendall Park, NJ, USA, 2Department of Pharmacy Administration, School of Pharmacy, University of Mississippi, University, MS, USA, 3Merck & Co., Inc., North Wales, PA, USA, 4Department of Pharmacy Administration and Center for Pharmaceutical Marketing and Management, School of Pharmacy, University of Mississippi, University, MS, USA, 5Merck & Co., Inc., Rahway, NJ, USA, 6Pharmacy Quality Alliance, Alexandria, VA, USA
OBJECTIVES: To assess differences in the relationship between medication adherence and healthcare costs across three quality measures in a Medicare Advantage (MA) sample.
METHODS: An observational study was conducted among cohorts of MA beneficiaries using the 2018-19 Optum Clinformatics Data Mart. Pharmacy Quality Alliance’s adherence measures for 1) oral antidiabetics; 2) RASAs; and 3) statins were used to determine cohort eligibility. Cohort assignment was based on mutually exclusive inclusion in one of the adherence measures or inclusion in all three measures. Medication adherence, assessed as Proportion of Days Covered (PDC), was measured in year 1 and payer medical costs were measured in year 2. Generalized linear modeling (log link) with interaction terms was used to assess relationships between adherence and subsequent payer medical costs, and to evaluate differences in this relationship across the three measures. Separate analyses were conducted using Poisson and Gamma distributions. Analyses were adjusted for sociodemographic, clinical, prescription-related, and insurance-related variables.
RESULTS: The study cohort included 962,480 MA beneficiaries, with 272,371 beneficiaries included in all three PDC measures. There was a negative relationship (p<0.0001) between adherence in year 1 and payer medical costs in year 2 for all medication classes. These associations were stronger for diabetes medications, followed by RASA and statin medications. The findings were consistent for patients included in one of the three medication adherence cohorts and patients using all three medication classes. Additionally, results were consistent when comparing Gamma and Poisson models.
CONCLUSIONS: Better adherence was associated with lower payer medical costs across several therapeutic areas, providing additional evidence of the importance of adherence in managing health care costs. The relationship between adherence and future medical costs appears stronger for oral anti-diabetic medications relative to RASAs or statins. Further research may explore interventions to increase adherence to oral anti-diabetic medication to improve diabetes management.
P60: Cost-Related Medical Care Use Reduction and Mediation Nonadherence in US Adults with Multiple Chronic Conditions: A Comparison of High Deductible Health Plans and Traditional Insurance Enrollees
10:15AM - 10:30AM
Davis-Ajami ML1, St. Hilaire M1, Kokkotos F1, Lu ZK2
1Merrimack College, North Andover, MA, USA, 2University of South Carolina, Columbia, SC, USA
OBJECTIVES: To examine cost-related forgone and delayed medical care and medication nonadherence in privately insured high deductible health plan (HDHP) vs. traditional insurance plan enrollees with multiple chronic conditions (MCC) in the US.
METHODS: This study identified privately insured adults (18-64 years) with MCC enrolled in HDHP or traditional plans from the 2019 US National Health Interview Survey (NHIS) Sample Adult file. Primary outcomes included forgone and delayed medical care due to cost and cost-related medication nonadherence defined as skipped medication doses, taking less medication, delayed filling a prescription or not getting needed medications due to cost in the past 12 months. Logistic regression assessed the association between HDHP (vs. traditional plans) and primary outcomes. Subgroup analysis focused on HDHP enrollees to identify demographic and health characteristics associated with primary outcomes.
RESULTS: Among the 4025 privately insured adults with MCC included in the study, 42% were covered by a HDHP. Compared with traditional insurance enrollees, HDHP enrollees were more likely to report forgone (14.1% vs. 7.4%, p<0.001; OR=2.29, 95% CI: 1.75, 3.00) and delayed medical care (15.5% vs. 8.4%, p<0.001; OR=2.15, 95% CI: 1.67, 2.78) and cost-related medication nonadherence (18.7% vs. 13.8%, p=0.001; OR=1.52, 95% CI: 1.20, 1.93). Among HDHP enrollees, females, reporting worse health status, or having more than two chronic conditions, were more likely to reduce medical care and medication use due to cost (p<0.001).
CONCLUSIONS: Significant cost-related reductions in medical care and medication use exist in HDHP enrollees with MCC compared to traditional insurance plans. Disparities in reduced medical care and medication use due to cost occurred by sex, general health status, and the number of chronic conditions.
Budget Impact Analysis of Health Interventions
Budget impact analysis is increasingly used as a tool to inform financial consequences of new health interventions. This session features budget impact analysis of interventions for the management of chronic obstructive pulmonary disease, Hemophilia B, and COVID-19 vaccine, as well as methodological considerations for incorporating time-to-event outcomes in budget impact analysis. Seating is limited.
Moderator
Austin Williams, PhD
Centers for Disease Control and Prevention, Atlanta, GA, USA
Austin Williams is an economist in the Division of STD Prevention at the Centers for Disease Control and Prevention. Previously, Austin worked as a research economist at RTI International and served as a Prevention Effectiveness Fellow at the CDC. He received a PhD in Agricultural and Applied Economics from the University of Wisconsin, Madison.
Austin’s research interests include economic evaluation, quasi-experimental designs, and health equity. His research has been published in the American Journal of Preventative Medicine, Proceedings of the National Academy of Science, Environmental and Resource Economics, MMWR, and other peer reviewed journals.
P54: Budget Impact Analysis (BIA) of the Use of Nonacog Beta Pegol in Patients with Haemophilia B in the Colombian Health System
10:00AM - 10:15AM
Casas-Ramírez D1, Moreno N1, Butron-Granados NC1, Hernández F2, Moreno-Calderon A1
1Novo Nordisk, Bogota, Colombia, 2IQVIA, Bogota, Colombia
OBJECTIVES: To estimate the annual impact of the inclusion of nonacog beta pegol in the Colombian health system for the prophylaxis treatment of previously treated patients with a confirmed diagnosis of haemophilia B.
METHODS: An Excel BIA model was built to estimate the economic impact of the progressive inclusion of nonacog beta pegol in the Colombian institutional health market for the prophylactic treatment of haemophilia B. The model was developed and populated with local data from The National Administrative Department of Statistics (DANE) and epidemiological data from the public and official records of the High-Cost Account (CAC) of 113 patients with haemophilia B in 2021; Clinical information including dosage, treatment schemes and annual bleeding rates were obtained from the published clinical trial of nanocog beta pegol and nonacog alfa. The costs were calculated and expressed in US dollars (USD) for a 3-year horizon, according to the Medicine Price Information System (SISMED) and the fee manual of a private hospital. A deterministic sensitivity analysis was implemented.
RESULTS: The BIA showed that the inclusion of nonacog beta pegol resulted in savings of USD $604,842, USD $582,607 and USD $561,154 for the first, second and third year, respectively. As a complementary analysis, the number of additional potential patients that could be treated per year due to savings was calculated: year1: 5 patients; year2: 4.8 patients and year3: 4.6 patients.
CONCLUSIONS: The inclusion of nonacog beta pegol in the Colombian health system for the treatment of patients with haemophilia B in prophylaxis can result in savings of up to USD1,748,603 over a 3-year horizon.
P55: Budget Impact Analysis of COVID-19 Vaccines on U.S. Medicare and Commercial Health Plans after Depletion of the Federally-Purchased Supply
10:30AM - 10:45AM
Vu H, Nguyen K, Hendricks J
MedImpact Healthcare Systems, Inc, San Diego, CA, USA
OBJECTIVES:
To analyze the budget impact (BI) of Covid-19 vaccines from a mixed U.S commercial and Medicare payer perspective after depletion of the Federally-Purchased Supply (FPS)
METHODS:
BI analyses were conducted in a hypothetical one-million member health plan with a mixed commercial (55%) and Medicare (45%) population over a one-year time horizon based on the current (January 2023) Covid-19 vaccine recommendations from the Centers for Disease Control and Prevention (CDC). The two scenarios in the model include 1) the health plan does not pay for Covid-19 vaccines, and 2) after the depletion of FPS, the health plan must cover all costs for Covid-19 vaccines. Model inputs include the market shares of available Covid-19 vaccines in the US as of December 2022, Covid-19 vaccine utilization trends stratified into age groups (<12, 12-17, 18-24, 25-49, 50-64, ≥65 years old) between commercial and Medicare populations, and predicted Covid-19 vaccine costs. Model inputs were based on the CDC publicly available data, real world evidence, published literature, and expert opinions. Sensitivity analyses (SA) were conducted to test uncertainties arising from the input values in the model.
RESULTS:
The number of members receiving one primary dose, completed Covid-19 vaccine series, one booster dose and two booster doses was estimated at 9,253, 49,720, 594,933 and 29,387, respectively. The incremental Covid-19 vaccine cost per member per month over one year after depletion of the FPS was $5.92 for the commercial population, $8.93 for the Medicare population, and $7.27 for the total population in the health plan. In the SA, the largest effect was observed for the scenario which varied the percentage of population ≥65 years old receiving one booster dose.
CONCLUSIONS:
The model results indicate that there will be a high budget impact from a mixed U.S commercial and Medicare perspective after depletion of the FPS of Covid-19 vaccines.
P53: Budget Impact Analysis of Adopting Primary Care-Based COPD Case Detection in the Canadian General Population
10:15AM - 10:30AM
Mountain R1, Johnson K2
1Lancaster University, Lancaster, LAN, UK, 2University of British Columbia, Vancouver, BC, Canada
OBJECTIVES:
An estimated 70% of Canadians with chronic obstructive pulmonary disease (COPD) remain undiagnosed, representing a critical barrier to early intervention to improve disease outcomes. Emerging evidence suggests that opportunistic primary-care based case detection for COPD is a cost-effective solution. We built on a previous cost-effectiveness analysis by evaluating the budget impact of adopting a case detection programme in the general Canadian population.
METHODS:
This study accords with ISPOR best practice guidelines for budget impact analysis. We used a validated whole disease microsimulation model of COPD in the general Canadian population to evaluate eight case detection strategies implemented during routine primary care visits, varying in their patient eligibility criteria and testing technology. We assessed COPD-related healthcare costs from the healthcare payer perspective over a five-year time horizon (2022-2026) with gradual programme uptake from 5% to 25% by 2026. Costs were determined from Canadian studies and updated to 2021 Canadian dollars. Key parameters were varied in one-way sensitivity analysis.
RESULTS:
Compared to no case detection, all strategies resulted in substantial budget expansion. In the most cost-effective scenario at a willingness-to-pay of $50,000/QALY (questionnaire-based testing for all patients
≥40 years), total additional costs were $427 million over five years, with 86% of costs attributed to administering case detection and subsequent diagnostic testing. Furthermore, there were 4.6 million referrals to diagnostic spirometry, 96% of which were false positives. The proportion of individuals with COPD who were diagnosed increased from 30.4% to 37.8% by 2026. Results were most sensitive to case detection uptake in primary care.
CONCLUSIONS:
A national primary care-based COPD detection programme will require prioritisation by budget holders and significant additional investment in the availability of diagnostic spirometry. Case detection could be effective for reducing the burden of undiagnosed COPD but will depend on successful uptake of the programme in primary care.
P56: Comparison of Methods for Incorporating Time-to-Event Outcomes in Budget Impact Analyses
10:45AM - 11:00AM
Law-Koune Q1, Hopmans Galofré G2, Genestier V3, Clayson M2, Cawston H1, Borget I4
1Amaris Consulting, Paris, France, 2Amaris Counsulting, Barcelona, B, Spain, 3Amaris Consulting, Toronto, ON, Canada, 4Gustave Roussy Cancer Centre and University Paris-Saclay, Villejuif, 92, France
OBJECTIVES:
Systematic review of guidelines for conducting budget impact analyses (BIA) revealed a lack of recommendations for the incorporation of time-to-event (TTE) outcomes (e.g., treatment duration, disease progression, death). Our objective was to compare three methods of incorporating TTE outcomes and assess their impact on total costs (TC).
METHODS:
A 5-year BIA was developed using a three-state partitioned survival model (PSM), estimating the proportion of yearly incident cohorts (IC) in either progression-free, progressed, or death health states using survival equations. For each IC, the overall means method (OMM) applied 5-year mean TTE outcomes. The incremental means method (IMM) adjusted the mean TTE outcomes to the time remaining in the model. The restricted means method (RMM) considered yearly restricted means for each model year.
Two interventions were included, with equal market shares in the ‘New Environment’. Pre- and post-progression acquisition costs, disease management, and end-of-life costs were included. Scenario analyses were conducted to assess when TTE method most affected results.
RESULTS: The RMM and IMM had equal TCs while the OMM consistently estimated higher TCs. The RMM distributed TCs most similarly to TTE curves compared to IMM, with lower TCs in Years 1-3 and higher TCs in Years 4-5 versus IMM. Scenario analyses indicated that TC distributions between RMM and IMM differed most when (1) increasing overall and progression-free survival for both interventions, (2) increasing disease management costs, and (3) increasing overall survival for the new intervention; IMM estimated higher TCs by 27%, 32%, and 28% in the first year and lower TCs by 37%, 31%, and 27% in the fifth year, respectively.
CONCLUSIONS:
Alternative methods to incorporate TTE outcomes in BIAs produce important yearly differences in results. This study highlighted the need for
clear methodological guidance to ensure consistency across manufacturer submissions, improve accuracy of BIA estimates, and ultimately, facilitate more informed resource allocation.
Breakouts: IP, WS, & OBS
Comorbidity Modeling in HTA for Long-Term Health Conditions: A Missing Piece of the Puzzle?
Digital Conference Pass
Level: Intermediate
PURPOSE:
Our objective is to outline the challenges when modelling and evaluating pharmaceutical therapies for conditions with comorbidities, and present possible solutions. Comorbidities are common in older patients with complex long-term health conditions such as type-2 diabetes, chronic kidney disease, and cardiovascular diseases. Pharmaceutical therapies that are effective for one condition may have benefits for comorbidities due to the clinical correlations between these conditions and comorbidities. This could be through direct treatment effects or indirectly by improving overall health of the patient. Such factors are challenging for health technology assessment (HTA) as modelling all comorbidities would require multifaceted models with high data demands. It also presents a policy challenge as therapy benefit is typically valued on one rather than multiple indications.
DESCRIPTION:
MV will outline the challenge of, and seek audience views on, comorbidities in HTA (8 minutes). She will draw on her consulting experience for how Payers consider comorbidities, such as with subgroup analyses and covariate models. AA will describe the risk equations approach to comorbidities in diabetes and cardiovascular modelling (12 minutes). In diabetes these model longitudinal A1C trajectory and its effect on stroke, myocardial infarction, foot ulcer and other comorbidities. He will describe challenges from Payers about insufficient trial data on patients with multiple conditions. NL will give an academic and charities perspective on using an American Diabetes Association sponsored cost-effectiveness model in type-2 diabetes (12 minutes). She will explain how choice of comorbidities to model was made and how they were modelled. HT will give an academic and assessor’s perspective on comorbidities in HTA models submitted to the UK National Institute for Health and Care Excellence (NICE) (12 minutes). He will also use an academic example for routine monitoring in type-2 diabetes, chronic kidney disease and hypertension to indicate future research directions in multimorbidity modelling for HTA.
Discussion Leaders
Meena Venkatachalam, MSc
Precision HEOR, London, United Kingdom
Discussants
Adnan Alsumali, MS, PhD
Merck & Co., Inc., Rahway, NJ, USA
Neda Laiteerapong, MD, MPH
The University of Chicago, Chicago, IL, USA
Howard Thom, MSc, PhD
University of Bristol, Bristol, SOM, United Kingdom
Howard Thom is a Senior Lecturer in Health Economics at the University of Bristol and Senior Director at the consultancy Clifton Insight. His research interests are value of information, uncertainty in economic models, network meta-analysis, and R for health economics.
Does Geography Affect Health? Insights Derived From Integration of Geography-Based Social Determinants of Health With Patient-Level Real-World Data
Digital Conference Pass
Level: Intermediate
PURPOSE:
Social determinants are an increasingly important lens for understanding patient health, through which patient- and geography-based disparities in access to care and clinical outcomes are pronounced despite advances in medicine and technology. Patient-level social determinants of health (SDoH) are widely unavailable from many real-world databases. In the absence of individual-level SDoH data, an opportunity in drawing meaningful insights arises by integrating patient-level health data with geography-level SDoH. Furthermore, FDA’s draft guidance for the industry provided recommendations to sponsors on the development of a Diversity Plan to enroll representative participants from underrepresented racial and ethnic populations in clinical trials. This workshop aims to provide appropriate methods for statistical analysis, and industry examples of using SDoH data to understand disparity and improve diversity, equality, and inclusion (DE&I).
DESCRIPTION:
This workshop has wide application across healthcare research stakeholders, addressing whether location, i.e., community resource availability and demographic composition, influences patient health outcomes. Four analytical methods for generating rich insights will be highlighted across research objectives. They are (1) running separate models for patient-level data in different geographies, where results are interpreted qualitatively by geography-level SDoH, (2) aggregating patient-level data to the geography-level so the unit of analysis is common, where results are at the geography-level, (3) ascribing geography-level SDoH to each patient living in that geography, such that interpretation of results is, e.g., “patients in communities with higher rates of unemployment have greater odds for heart disease,” and (4) mixed-effects modeling with geography as the random effect, where results are interpretated the same as the third method, but modeling accounts for the dependent nature of health and geography. Subsequently, similarities and differences between methods will be discussed, along with examples using RWD with SDoH data to improve DE&I in clinical studies. Finally, the workshop will conclude with challenges / opportunities for integrating these methods.
Discussion Leaders
Won Lee, PhD
Axtria, Inc., San Francisco, CA, USA
Discussants
Jennifer Ken-Opurum, PhD
Axtria, Inc., Berkeley Heights, NJ, USA
Dr. Ken-Opurum is an experienced consultant and Real-World Evidence (RWE) researcher, providing solutions to key business problems for clients in the pharma and biotech domain. She has conducted investigative research on topics across therapeutic areas including rare disease, oncology, dermatology, HIV, migraine, vaccination, neurodegenerative disease, mental health, autoimmune disorders, metabolic syndrome, diabetes, and cardiovascular disease. Dr. Ken-Opurum has been responsible for providing strategic insights for study design, project implementation, and quality of deliverables, leading teams of researchers, analysts, and data scientists on secondary database analyses, economic modeling, primary patient/stakeholder surveys, patient chart reviews, prospective cohort studies, HRQoL/PRO studies, abstract and manuscript publications, and systematic literature reviews. She is a trained epidemiologist with subject matter expertise on health behaviors and outcomes and has provided trainings to external researchers and data users of national health surveys.
Keran Moll, PhD
Regeneron Pharmaceuticals, Inc., Tarrytown, NY, USA
Keran Moll, Ph.D., is a Director Health Economics & Outcomes Research at Regeneron Pharmaceuticals, Inc. As part of Regeneron’s Real World Data & Analytics Research Team, Keran leads the fit-for-purpose evaluation of real-world data and tools, supports epidemiologic studies using RWD across multiple therapeutic areas, and oversees the analytical activities in RWE generation. Keran also leads several other internal initiatives, such as real-world data standardization and the integration of SDoH data. Before joining Regeneron, while at IBM Watson Health, Keran served as principal investigator in multiple FDA CBER-sponsored post-market epidemiologic studies under the Biologics Effectiveness and Safety (BEST) initiative; and led common data model conversion efforts to support both CBER and CDER.
Ron Preblick, PharmD, MPH
Sanofi, Bridgewater, NJ, USA
10:00 AM - 11:15 AM
Spotlight Session
Apples and Oranges in the Context of Anchored Indirect Treatment Comparisons – Is There More to It Than Effect Modifiers?
Digital Conference Pass
Level: Intermediate
PURPOSE:
There is debate about whether marginal or conditional treatment effect estimates are of interest in the context of health technology assessment (HTA). Traditionally, the question about the “estimand” of interest has been overlooked, ignored, or implicitly assumed in HTA. In answering this question, it is important to understand the difference between marginal and conditional treatment effects. This becomes pertinent with the rapid development of methods for indirect treatment comparisons and network meta-analysis that make use of individual participant level data and not just published aggregated level data. In this session, relevant terminology will be summarized, methods to obtain marginal and conditional treatment effect estimates presented, and potential implications to the practice of indirect treatment comparisons and network meta-analysis in the context of HTA will be discussed.
DESCRIPTION:
In Part 1, Ms. Cope will introduce the terminology, explain challenges arising with non-collapsible effect measures, and define target estimands using a simple example. In Part 2, Dr. Campbell will illustrate that marginal and conditional treatment effect estimates do not necessarily equate to unadjusted and adjusted analyses and will show how to ‘marginalize’ adjusted estimates in a single study for odds ratios, carrying forward the earlier example. In Part 3, Dr. Remiro-Azócar will provide an overview of methods for population adjustment in the context of indirect comparisons and network meta-analysis with a specific emphasis on the “marginal versus conditional” issue. Dr. Jansen will provide a summary of the discussion and will facilitate a debate among the panelists regarding the implications for indirect treatment comparisons and network meta-analysis in the context of health technology assessment. Attendees will have the opportunity to pose questions after this discussion.
Moderators
Hwee-Lin Wee, PhD
National University of Singapore, Singapore, Singapore
A/P WEE Hwee Lin is joint Associate Professor at the Saw Swee Hock School of Public Health and Department of Pharmacy, Faculty of Science at the National University of Singapore. She is a member of the International Society for Pharmacoeconomics and Outcomes Research Health Sciences Policy Council, member of the International Editorial Advisory Board for Journal of Patient Reported Outcomes, and member of the National Advisory Committee on Cancer in Singapore. A/P Wee’s current work focuses on the use of real world evidence in health technology assessments.
Speakers
Harlan Campbell, PhD
University of British Columbia, Rossland, BC, Canada
Harlan Campbell is a statistician and Postdoctoral Research Fellow at the University of British Columbia. His research interests include Bayesian hypothesis testing, causal inference, evidence synthesis and applications in ecology, epidemiology and clinical trials.
Shannon Cope, MSc
PRECISIONheor, VANCOUVER, BC, Canada
Shannon Cope is a Vice President with PRECISIONheor with fifteen years of experience consulting for biopharmaceutical industry. Shannon leads evidence synthesis projects involving (network) meta-analyses, population-adjusted indirect comparisons, multi-level network meta-regressions, and economic models. Her publications relate to application and extension of evidence synthesis methods related to (network) meta-analysis, the feasibility assessment process, time-to-event outcomes, and the integration of individual patient data and expert opinion. She has an MSc in Health Administration from the University of Toronto, Canada.
Antonio Remiro-Azócar, PhD
Bayer, London, LON, United Kingdom
Antonio is a Lead Medical Affairs Statistician at Bayer, specializing in health technology assessment (HTA), health economics and outcomes research (HEOR), and observational studies. At Bayer, Antonio leads cross-functional teams in providing inputs to life-cycle management strategies, publication plans, reimbursement requirements, HTA studies and analyses for payers, across a number of therapeutic areas.
Prior to his current role, Antonio provided support on the statistical aspects of evidence synthesis, HTA and HEOR 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.
Discussants
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.
11:00 AM - 11:30 AM
Break
Located in the Exhibit Hall
11:30 AM - 12:45 PM
Plenary Session
Closing Plenary Session
Digital Conference Pass
: 316: Closing Plenary: Issues and Solutions When Estimating Treatment Effects Using US Electronic Health Record Data
11:30AM - 12:45PM
Schneeweiss S1, Rassen J2, Hubbard R3, Weberpals J1, Wyss R4
1Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA, 2Aetion, Inc., New York, NY, USA, 3Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA, 4Harvard Medical School , Brigham and Women’s Hospital and Sentinel Innovation Center, Boston, MA, USA
The use of electronic health record (EHR) data has great promise to augment claims-based RWE studies to estimate causal treatment effects. Success stories regarding regulatory and payer decision making cluster in disease areas with structured and intense care, like oncology, post-transplant care, or hemophilia. Given the detailed clinical information available in structured and free text form, an abundance of features can be derived for improved confounding adjustment and outcome definition.
This third plenary summarizes several cutting-edge research approaches to overcome typical issues encountered with EHR data and improve causal inference to inform decision-making, drawing from a group of researchers especially focused in this key methodological area. This plenary session highlights 4 recurrent issues:
- How to reduce an ultra-high-dimensional covariates space for optimal confounding adjustment?
- How to deal with differential information content that results from physicians ordering tests in light of clinical need, e.g., pre-surgical medical work-up and its consequences for studying the cardiovascular effects of bariatric surgery.
- How selective observability in EHR data will influence inference in an example of missing BMI measurements in pediatric patients and what to do about it.
- Missing data are common and biostatistics has provided tools to reduce biases arising from it. How can we efficiently learn about the missingness processes in EHR data and pick the adequate tool for a given situation?
Four experts briefly explain the issues and sketch out pathways to practical solutions. In the discussion section we will ask the audience to share their own experiences and add to the issues raised and solutions presented.
