* Program subject to change
Track: Patient-Centered Research
Rebecca Crawford, MA
RTI Health Solutions, Manchester, United Kingdom
Lynda Doward, MRes
Arinesalingam Gnanasakthy, MSc, MBA
RTI Health Solutions, Succasunna, NJ, USA
Shanshan Qin, PhD
RTI Health Solutions, Durham, NC, USA
Nicholas J. Rockwood, PhD
RTI Health Solutions, Bend, OR, USA
Track: Methodological & Statistical Research
Devin Incerti, PhD
EntityRisk, Inc., San Francisco, CA, USA
Jeroen Jansen, PhD
PRECISIONheor, Oakland, CA, USA
Track: Health Technology Assessment
Lou Garrison, PhD
CHOICE Institute, School of Pharmacy, University of Washington, Seattle, WA, USA
Charles Phelps, PhD
University of Rochester, Rochester, NY, USA
Richard Willke, PhD
ISPOR, Lawrenceville, NJ, USA
Wei-Hsuan Jenny Lo-Ciganic, PhD, MSPharm, MS
Health Research Scientist Geriatric Research Education and Clinical Center (GRECC), Gainesville, FL, USA
William Padula, PhD, MSc, MS
University of Southern California, Los Angeles, CA, USA
Uwe Siebert, MD, MPH, MSc, ScD
UMIT - University for Health Sciences Medical Informatics and Technology Hall in Tirol, Austria and Harvard Chan School of Public Health Harvard University, Boston, MA, USA
Elisabeth Fenwick, PhD
OPEN Health Evidence & Access, Oxford, United Kingdom
Natalia Kunst, PhD
University of York, York, United Kingdom
Lotte Steuten, PhD
Office of Health Economics, London, United Kingdom
Track: Economic Evaluation
Emma Hawe, MSc
Andrew Lloyd, PhD
Acaster Lloyd Consulting Ltd, Oxford, OXF, United Kingdom
Sorrel Wolowacz, PhD
Track: Real World Data & Information Systems
Douglas E. Faries, PhD
Consulting Services, Alma, AR, USA
Darius Lakdawalla, PhD
USC Leonard D. Schaeffer Center for Health Policy and Economics, Los Angeles, CA, USA
Track: Health Policy & Regulatory
Marlene Gyldmark, MPhil
Idorsia Pharmaceuticals, Allschwill, Switzerland
Mark Nuijten, MBA, PhD, MD
A2M, Amsterdam, Netherlands
Jeremy Rassen, ScD
Aetion, Inc., New York, NY, USA
Sebastian Schneeweiss, MD, ScD
Brigham and Women’s Hospital, Harvard Medical School, Boston, MA, USA
Shirley Wang, PhD, MSc
Jag Chhatwal, PhD
Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA
Turgay Ayer, PhD
Georgia Institute of Technology, Boston, MA, USA
Dalia Dawoud, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Baris Deniz, MSc
GSK, Durham, NC, USA
Jamie Sullivan, MPH
EveryLife Foundation for Rare Diseases, Washington, DC, DC, USA
Jessica Daw, PharmD
Sentara Health Plans, Virginia Beach, VA, USA
Ryan Fischer, BA
Foundation for Angelman Syndrome Therapeutics, Austin, TX, USA
Colin M Young, PhD
Tufts Medical Center, Boston, MA, USA
ISSUE: It generally is assumed that patient involvement in clinical decisions increases the likelihood that treatments align with patient preferences, leading to improved health outcomes and patient well-being. While it seems logical that better health outcomes will improve well-being, health is only one component of perceived well-being and could compete with non-health priorities. To avoid undesirable impacts on well-being, patients may be willing to forgo the full benefits of treatment. Consequently, patients could reasonably be expected to use treatments in a way that optimizes overall well-being, but not necessarily overall health. Thus, incorporating patient preferences in patient-centered care potentially conflicts with the goal of cost-effectiveness analysis, which is to maximize gains in health-related quality-adjusted survival within a budget constraint. Why should personal non-health priorities be considered in clinical and reimbursement decision making, and what are the challenges in assessing these non-health priorities?
OVERVIEW: The panel will discuss challenges and opportunities of accounting for non-health determinants of health behaviors in evaluating the full impact of medical technologies. Potential challenges include our limited ability to identify and measure patient preferences for non-health factors that influence health behaviors. Recent developments in the use of patient-preference methods to assess individual-level preferences and frameworks to quantify their role in health-behaviors offer potential avenues to address these challenges. Finally, the panelists will provide an assessment of the potential value of a more holistic evaluation of patient needs and wants in product development, clinical decision-making and market access decisions. Each panelist will take 10 minutes to discuss their perspectives. The moderator will facilitate audience discussion and debate. This discussion will be relevant for a wide range of healthcare stakeholders, including industry, clinical, and reimbursement decision makers.
Shelby Reed, PhD, RPh
Duke University’s School of Medicine, Durham, NC, USA
Marco Boeri, PhD
Open Health, New York, NY, USA
Juan Gonzalez, PhD
Duke Clinical Research Institute, Durham, NC, USA
Brett Hauber, PhD
Pfizer, New York, NY, USA
Sreeram Ramagopalan, PhD
Lane Clark & Peacock, London, LON, United Kingdom
Alejandra Castanon, MD, MSc, PhD
Lane Clark and Peacock, London, United Kingdom
Stephen Duffield, PhD, MD
National Institute for Health and Care Excellence, Liverpool, United Kingdom
Rob Reynolds, PhD
GSK, New York, NY, USA
Meghana Chalasani, MHA
FDA, Silver Spring, MD, USA
Robyn Bent, MS
Food and Drug Administration, Silver Spring, MD, USA
Kevin Bugin, PhD
PURPOSE: Growing interest in using real-world evidence (RWE) to inform regulatory decision making has given rise to significant refinements in analytic methods to elevate the quality and reliability of RWE. Many of the advances have addressed bias and confounding in analyses of secondary real-world data (RWD) sources (predominantly claims & electronic health records [EHRs]). However, the importance of selecting the right patients, classifying them into the right treatment groups, and assessing the right outcome measures in RWD cannot be overstated. This is the domain of computable operational definitions, or “phenotypes”, which are prominent in the field of clinical informatics but less well established in health economics & outcomes research (HEOR). The objective of this workshop is to evaluate the criticality of computable phenotypes in the development of valid and reliable RWE.
DESCRIPTION: A computable phenotype is a machine-executable algorithm to identify patient cohorts, exposures, and outcomes from variables in RWD. Guidance issued by the US Food & Drug Administration in 2021 for analyses of EHRs and claims data to support regulatory submissions describes the importance of computable phenotypes as a means of standardizing key design elements across different analyses. However, challenges associated with implementation remain a concern. This workshop will address these challenges head on. It will begin with an introduction to computable phenotypes based on examples from different disease states and types of RWD. The next segment will present a case study quantifying efficiency gains associated with use of a technology platform to standardize computable phenotypes across alternative data sources. This will be followed by a description of the use of computable phenotypes in analyses of EHR data at the US Department of Veterans Affairs Health System. The final segment will provide an FDA perspective on the topic. Real-time polling will be used to solicit audience feedback on the issues discussed.
David Thompson, PhD
Rubidoux Research LLC, Manchester, MA, USA
Marie Bradley, PhD
US FDA, Silver Spring, MD, USA
Scott DuVall, PhD
VA Informatics and Computing Infrastructure, VA Salt Lake City Health Care System, Salt Lake City, UT, USA
Aaron Kamauu, MD MS MPH
Navidence LLC, Bountiful, UT, USA
Tess Cameron, BA
RA Capital Management, Boston, MA, USA
Mikkel Oestergaard, PhD
MSD Innovation & Development GmbH, Zurich, Switzerland
Siu Hing Lo will provide a broad overview of the roles patients and caregivers have as engaged research partners versus research participants in patient-centered research. She will then discuss how the form of patient/caregiver engagement could differ depending on the study objectives, existing evidence, practical considerations, and the intended use of the study findings. She will also highlight similarities and differences in patient/caregiver roles between health preference and PRO studies.
Holly Peay will discuss case studies in which patients and caregivers participated in a community engaged process for the development, interpretation, and dissemination of qualitative and quantitative HPR research. She will summarize barriers and facilitators to engagement and approaches to match engagement to stated preference research objectives.
Ryan Fischer will discuss his experience collaborating with academic and industry partners for advocacy-led patient preference and experience studies, and how advocacy organizations use the results generated from such studies in their strategic planning, advocacy, education, and research funding.
Fraser Bocell will discuss the roles of patients and the use of patient engagement in selecting, modifying, and adapting meaningful PRO instruments, from a regulator’s perspective.
Speakers will use polling questions to engage the audience in discussion.
This forum is a collaboration presented by the Health Preference Research and the Patient-Centered Special Interest Groups.
Siu Hing Lo, PhD
Acaster Lloyd Consulting Ltd, London, LON, United Kingdom
Fraser Bocell, PhD
U.S. Food and Drug Administration, Silver Springs, MD, USA
Holly Peay, PhD
RTI International, Research Triangle Park, NC, USA
Larry Liu, MD, PhD
Merck & Co., Inc., Rahway, NJ, USA
Viva Ma, PhD, MBA
Becton, Dickinson and Company, Singapore, Singapore
Jianwei Xuan, MD, PhD
Sun Yat-sen University, Guangzhou, Guangdong, China
Michael Cangelosi, MA, MPH
Boston Scientific, Natick, MA, USA
Arturo Cabra, MSc
GE Healthcare, Miami, FL, USA
Arthi Chandran, MS, MPH, DrPH
Abbott, Santa Clara, CA, USA
Belinda Mohr, Ph.D.
Philips, Phoenix, AZ, USA
Patricia Synnott, MS, MALD
Kanya Shah, PharmD, MBA, MS
University of Illinois Chicago College of Pharmacy, Chicago, IL, USA
Piaopiao Li, Master of Science, Ph.D. candidate
University of Florida, Gainesville, FL, USA
After decades of debate, the U.S. Congress passed legislation in 2022 authorizing Medicare to negotiate prescription drug prices. The first ten drugs subject to negotiation were selected in 2023. Evidence of comparative effectiveness, net prices, and unmet need alongside industry R/D and productions costs have been submitted to CMS for consideration in the negotiation. The negotiated Maximum Fair Price (MFP) will be published for the ten drugs by September 2024. This session will provide an update on implementation and future steps with viewpoints from CMS, the pharmaceutical industry, and a drug pricing policy expert. Dr. Sullivan will serve as moderator and provide a brief overview of the Medicare Prescription Drug Price Negotiation Program (DPNP). He will introduce the three panelists. Ms. Martin will discuss implementation, progress and challenges of the DPNP from the perspective of CMS. She will comment on the initial product selection, the evidence submission process, the patient and stakeholder listening sessions and the formal negotiation process. What has CMS learned, what is working well, and what might CMS do differently in subsequent rounds of negotiation? Dr. Blanchette will discuss how companies like Novo Nordisk have organized internally to respond to the initial round of the DPNP. He will discuss lessons learned for HEOR departments on how best to prepare for future engagements with CMS. Finally, Dr. Hernandez will conclude by discussing sources of savings associated with negotiation, and implications of the DPNP for formulary coverage and pricing within Medicare Part D and private health insurance. We will finish with a moderated Q/A session.
Sean Sullivan, BScPharm, PhD
The CHOICE Institute School of Pharmacy, University of Washington, Seattle, WA, USA
Christopher Blanchette, PhD, MBA
NovoNordisk, Princeton, NJ, USA
Inmaculada Hernandez, PharmD, PhD
University of California San Diego, La Jolla, CA, USA
Kristi Martin, MPA
Center for Medicare and Medicaid Services, Washington, DC, USA
PURPOSE: With the proliferation of digital health interventions (DHIs) there is an increasing need for a structure to guide health economic and outcomes research (HEOR) and health technology assessments (HTA) by defining these technologies. In this session, an audience with an interest in HEOR and HTA related to patient-facing Digital Health will learn about and apply a proposed descriptive framework for assessment of DHIs.
DESCRIPTION: The PICOTS-ComTeC (population, intervention, comparator, outcome, timing, setting, communication, technology, context) framework has been developed to overcome the issue that many terms used for DHIs are not sufficiently precise enough to support HEOR best practices. The ISPOR Digital Health SIG researched the current use of digital health terms. Based upon the shortcomings identified, a Delphi panel was used to gain consensus on the most relevant domains/subcategories to be specified in definitions of patient-facing DHIs for evidence summaries. The resulting PICOTS-ComTeC framework, an extension of the well-established PICOTS, specifically addresses the variability of DHIs.
Annette Champion, MBA
Healthcare Research Insights, Inc, Lake Forest, IL, USA
Carl Asche, PhD
University of Utah, Salt Lake City, UT, USA
Artem Boltyenkov, PhD, MBA
Siemens Healthcare Diagnostics Inc., Hellertown, PA, USA
Katelyn Keyloun, PharmD, MS, BS
AbbVie, Irvine, CA, USA
Bill Byrom, B.Sc., Ph.D.
Signant Health, Nottingham, NTT, United Kingdom
Julia Garcia, PhD, MS
PURPOSE: This workshop will aim to discuss the application of novel methods of estimating causal inference using real world data.
Suzanne McMullen, MHA
Medlior Health Outcomes Research, Calgary, AB, Canada
John Paul Ekwaru, MSc, PhD
Medlior Health Outcomes Research, Edmonton, AB, Canada
Mark van der Laan, PhD
University of California Berkeley, Berkeley, CA, USA
Robert McQueen, PhD
University of Colorado Anschutz Medical Campus, Denver, CO, USA
Renske Ten Ham, PhD, PharmD, MSc
University Medical Center Utrecht, Utrecht, Utrecht, Netherlands
Andrew York, PharmD, JD
Maryland Prescription Drug Affordability Board, Bowie, MD, USA
Antal Zemplenyi, PhD
University of Colorado Skaggs School of Pharmacy and Pharmaceutical Sciences, Aurora, CO, USA
Ellen Janssen, PhD
Janssen R&D, Baltimore, MD, USA
David Gebben, PhD
U.S. Food and Drug Administration, Silver Spring, MD, USA
Jennifer Whitty, PhD
Evidera, part of PPD, London, United Kingdom
Dennis A. Ostwald, Prof., Dr.
WifOR Institute, Darmstadt, Hessen, Germany
Rifat Atun, Prof., PhD
Harvard University, Cambridge, MA, USA
Carolina Goic, MSc
Center for cancer prevention and control Medicine School Pontificia Universidad Catolica de Chile, Santiago, Santiago, Chile
Varnee Murugan, Master of Public Health
U.S. Chamber of Commerce, Bethesda, MD, USA
Dweeti Nayak, MS
Precision Medicine Group, Jersey City, NJ, USA
Tamas Agh, MSc, PhD, MD
Syreon Research Institute, Budapest, PE, Hungary
Adina Turcu-Stiolica, PhD.
University of Medicine and Pharmacy of Craiova, Craiova, DJ, Romania
In this forum, we will explore innovative approaches to surmounting these barriers associated with OSMs, offering three distinct viewpoints. Dr. Bob Beck will represent the academic/consultant perspectives, considering these individuals are often employed, via their affiliated institutions, to provide many of the models designated by the pharmaceutical industry. Dr. Daniel Ollendorf, representing the de facto health technology assessment (HTA) authority in the United States, will elaborate on the HTA perspective regarding OSMs. Dr. Rob Boer will provide insights into the pharmaceutical industry's standpoint on OSMs. The discussion will be enriched with an interactive Q&A session, fostering dynamic engagement and knowledge exchange.
Renée Arnold, PharmD, RPh
National Institutes of Health/NHLBI and BioHealth Innovation, Inc., Bethesda, MD, USA
J. Robert Beck, MD
Fox Chase Cancer Center, Philadelphia, PA, USA
Rob M. Boer, PhD
Abbvie, Irvine, CA, USA
Daniel Ollendorf, PhD
Institute for Clinical and Economic Review; Tufts University School of Medicine and Tufts Medical Center, Boston, MA, USA
Eric Barrette, PhD
Medtronic, Washington, DC, USA
Stephen T Parente, PhD
University of Minnesota, Minneapolis, MN, USA
Laura Pizzi, PharmD, MPH
The International Society for Pharmacoeconomics and Outcomes Research, Lawrenceville, NJ, USA
Kosali Simon, PhD
Indiana University, Bloomington, IN, USA
Kai Yeung, PharmD, PhD
Amazon, Seattle, WA, USA
Chronic or non-communicable diseases (NCDs) pose a global health challenge, particularly affecting the poor and vulnerable due to their higher exposure to risks, reduced access to health services, and health promotion and prevention programs. The impact of NCDs is further amplified by the fact that many patients have multiple chronic conditions simultaneously. Multimorbidity (defined as the presence of two or more chronic conditions in the same person) is increasingly recognized as a challenge for individuals, caregivers, families, community, and health systems, due to its social, economic, and public health impact.
Professor Dennis Ostwald (WifOR) will present a novel approach to evaluating the social impact of medical innovations, focusing on informed healthcare decision-making and sustainable development. Roberto Iunes (World Bank) will discuss the prevalence and economic burden of multimorbidity and NCDs in Latin America.
Finally, a moderated panel and Q&A session will be offered at the end of presentations for interaction and engagement with attendees.
Diego Guarin, MD MPH
ISPOR Latin America Consortium, Rahway, NJ, USA
Roberto Iunes, PhD
The World Bank, Washington, DC, USA
This forum will highlight the principal methods issues concerning:
The task force will present their recommendations and seek audience feedback on the overall approach taken. Pediatric utilities inevitably involves both HEOR science and normative considerations. Given the pivotal role of value judgements in methods choices in pediatric utilities, consultation with local HTA bodies is essential both to ensure that utilities are ‘fit for purpose’, and to ensure that decision makers are well informed about the role and implications of value judgements when establishing methods guidelines. The session will conclude with a question and answer session.
Nancy Devlin, PhD
School of Population and Global Health, University of Melbourne, Melbourne, Australia
Fleur Chandler, MSc
Sanofi and Patient Advisory Board Lead, Duchenne UK, Reading, Berkshire, Great Britain
Ernest Law, PharmD, PhD
Louis Matza, PhD
Patient-Centered Research, Evidera, Bethesda, MD, USA
Nivantha Subiron-Naidoo, MBChB, DipPEC, MPH, MSc CER
Oracle Life Sciences, Le Perreux-sur-Marne, 94, France
Rebecca Edelmayer, PhD
The Alzheimer’s Association®, Chicago, IL, USA
Yvette Venable, BA
Astra Zeneca, Cambridge, United Kingdom
ISSUE: CMS implementation of IRA will likely challenge pharmaceutical developers to re-assess pipeline priorities and commercialization strategies, potentially impacting development of future medicines, as well as go-to-market tactics. As a byproduct of IRA, CMS is simultaneously developing a pilot to re-imagine value frameworks through its Cell and Gene Therapy Access Model. With the goal to launch in 2026, the CMS CGT Access Model will explore payment and performance models as it will begin negotiation on behalf of states, including a role for CMS as centralized outcomes monitor for therapies utilized through the pilot. Further spillover into commercial payers will increase market uncertainty.
OVERVIEW: This panel will discuss the impact IRA and CMS pilots might have on value-based agreements (VBAs), as well as emerging performance frameworks in the face of rising public and private payer demands for stronger recourse if therapies are not effective. Attendees will be challenged to reimagine how value can be quantified and defended amidst a changing landscape, specifically using drug warranties aimed at true end payers and next generation VBAs. Panelist will discuss trends with innovative access agreements in the oncology space as drug developers seek to distinguish their drugs with providers and patients. Furthermore the role of evolving value frameworks for CGTs and how they can be applied to support VBAs for next-generation products will be discussed. Finally the role drug warranties can play to mitigate uncertainty for cell, gene and even specialty therapies will be reviewed. Overall how could VBAs and Warranty programs be applied to assets in response to payer pressure in the form of IRA and the CMS CGT Access Model will be debated. Attendees will learn about these and other trends and ways HEOR and market access teams can assist their organizations adapt and respond to the implications of the IRA.
Ambarish J. Ambegaonkar, PhD
APPERTURE LLC, Marlboro, NJ, USA
Emad Samad, BA
Octaviant Financial, Inc, New Brunswick, NJ, USA
Gergana Zlateva, BA, MPA, PhD
Pfizer Inc., New York, NY, USA
Betsy Lahue, MPH
Alkemi, Manchester Center, VT, USA
Arthi Chandran, MS, MPH, DPH
John Hernandez, PhD
Google, Mountain View, CA, USA
PURPOSE: While Bayesian methods have grown in popularity in the last few decades, applications in clinical development have only recently gained momentum. Bayesian methods may increase trial efficiency by leveraging prior information such as historical or real-world data to reduce the number of enrolled study participants. This workshop will provide a gentle introduction to the principles of Bayesian statistics including Bayes’ theorem, prior distributions, likelihoods, and posterior distributions. Attendees will learn about current opportunities for use in clinical development with a focus on practical considerations when implementing these methods. Common methods, their advantages and limitations, and use cases will be highlighted.
DESCRIPTION: Workshop attendees will receive a gentle introduction to Bayesian principles and understand opportunities for application in clinical development. Mr. Parzynski will introduce Bayesian thinking and how it differs from traditional statistical methods. Focus will be placed on the core components of Bayesian methodology: Bayes’ theorem, prior distributions, likelihoods, and posterior distributions (~15 minutes). Dr. Clark will then introduce clinical development settings in which Bayesian applications have been considered including early drug/biological product development, non-inferiority designs, adaptive designs, rare diseases, and pediatric studies. These will be presented alongside current challenges to their use. (~15 minutes). Next, Dr. Zeldow will introduce commonly used methods for increasing power for clinical trials using historical or real-world data (~15 minutes). The session will end with Dr. Saranadasa walking through primary considerations when pursuing these approaches in clinical development, highlighting limitations and decision hurdles with a particular emphasis on power borrowing methods (~15 minutes).
Craig Parzynski, MS
Genesis Research Group, Hoboken, NJ, USA
Jennifer Clark, PhD
Madhawa Saranadasa, PhD
J & J Innovative Medicine, Titusville, NJ, USA
Bret Zeldow, PhD
PURPOSE: To guide participants in using integrated real-world data for causal inference, highlighting the advantages and challenges. Several case studies will illustrate practical applications and lessons learned. Participants will interactively share their experiences and participate in open discussion.
DESCRIPTION: Causal inference from observational data is growing in importance, driven by the need for generalizable and rapidly delivered real-world evidence (RWE) to inform regulatory, payer, and patient/provider decision-making. Integrating two or more individual sources of real-world data (e.g., administrative health claims, electronic medical records, registries, and publicly available social determinants of health data) can provide deeper insights into the patient's health journey. Using integrated data can lower methodological and resource barriers to comparative effectiveness and long-term safety assessments. However, integrating data often requires trade-offs regarding variable consistency, available sample size, and selection bias.
Michael Grabner, PhD
Carelon Research, Wilmington, DE, USA
Ruth Dixon, PhD
Carelon Research, Athens, GA, USA
Patricia Lloyd, PhD, ScM
Edward Yu, ScD
Bristol Myers Squibb, Summit, NJ, USA
Lysel Brignoli, MS
Oracle Life Sciences, Paris, 75, France
Hyeokhyen Kwon, Ph.D., M.Sc
Emory University, Decatur, GA, USA
J. Lucas Mckay, Ph.D., M.S.C.R.
Emory University, Atlanta, GA, USA
Sarah Ostadabbas, PhD
Northeastern University, Boston, MA, USA
PURPOSE: The Inflation Reduction Act has implications for drug development priorities and will likely result in fewer me too drugs. Some have argued that having fewer me too drugs is not a bad thing as similar treatments already exist for that condition and thus they only offer marginal health benefits. However, that perspective ignores the benefits that me too drugs have on the market and for patient access. This workshop will provide multi-stakeholder perspectives on the value of me too drugs that are not accounted for in conventional value assessment.
DESCRIPTION: Melanie Whittington will introduce the workshop by defining a me too drug, presenting both sides of the debate regarding the relative importance of me too drugs, and discussing if value assessors adapt their methods when evaluating a me too drug. (10 minutes) Peter Kolchinsky will provide the investor perspective by presenting a 15-year timeline of innovation in Hepatitis C, that started with low-efficacy interferons marketed in 2004 and led to multiple efficacious cures in 2018. (10 minutes) Joel Barrish will provide the manufacturer perspective on developing a me too drug for the treatment of Hepatitis C, focusing on the unique considerations and reasons to innovate in a class with an existing market leader. (10 minutes) Everett Neville will provide the payer perspective on negotiating practices used for drugs in a class with numerous therapeutic options and the ability for me too drugs to result in more options for patients. (10 minutes) Polling questions will be used before and after the panelists’ presentations to assess audience opinions on the value of me too drugs and if their opinions were swayed after hearing the multi-stakeholder perspectives. Twenty minutes will be reserved for audience questions. This workshop will be relevant for health economists, HTA bodies, manufacturers, payers, and patient advocacy organizations.
Melanie Whittington, PhD, MS
Tufts Medical Center (CEVR), Boston, MA, USA
Joel Barrish, PhD
Jnana Therapeutics, Boston, MA, USA
Peter Kolchinsky, PhD
Everett Neville, MBA
The Cigna Group, Kamas, UT, USA
ISSUE: The value of the universal quality-adjusted life year (QALY) has long been recognized, but cost-utility analysts face significant challenges in measuring and valuing utility in children. These include an inability to directly elicit utilities, reliance on proxies, disagreement regarding whether a child or adult perspective or preference is most relevant for valuing child health, and a lack of preference-based health-related quality of life (HRQoL) measures specifically for young children and infants. This international panel will examine and debate different approaches to measuring and valuing health in very young children and infants.
OVERVIEW: Scott Grosse will moderate and provide historical context. Tara Lavelle will share findings on using a visual analog scale to capture infant utility and how parents’ perspectives affected reporting. Kim Dalziel will present the comparative psychometric performance of the EQ-5D-Y, adapted from the adult EQ-5D, and the PedsQL in preschool children, and will describe work to develop the EQ-TIPs for infants. Wendy Ungar will discuss qualitative research identifying attributes relevant to infant HRQoL and alternative approaches. Those include restructuring and adding new attributes to existing adult tools (HUI -> HuPS), mapping non-preference-based but age-appropriate child HRQoL instruments to derive utility weights (PedsQL ->PedUtil), and developing child-centric preference-based tools (e.g., CHU-9D) and expanding their use to younger children. There is an inherent tension between conserving attributes and levels used in adult tools versus developing ones more reflective of infant or young child HRQoL. While the former enables easier pooling of data across pediatric and adult age groups for lifetime modeling, it may lack construct validity in young children. After the presentations, the moderator will pose questions to panelists on measurement and valuation challenges to spark debate and each speaker will be asked to suggest ways forward. The session will end with an audience Q&A.
Scott D Grosse, PhD
Centers for Disease Control and Prevention, Atlanta, GA, USA
Kim Dalziel, PhD
The University of Melbourne, Carlton, VIC, Australia
Tara Lavelle, PhD
Tufts University School of Medicine and Tufts Medical Center, Boston, MA, USA
Wendy Ungar, MSc, PhD
The Hospital for Sick Children Research Institute, Toronto, ON, Canada
ISSUE: The widespread adoption of digital health products (DHPs) is hindered by a lack of evidence supporting their clinical value, which is essential for their broad acceptance, commercial success, and improved patient care. However, while product developers have successfully pioneered new regulatory pathways, securing FDA acceptance, market access and the evidentiary needs of downstream decision-makers have been largely opaque. This results in early and substantial investments in evidence generation being overfitted to regulatory decision-makers, missing the opportunity for more efficient and effective studies that address the requirements of all decision-makers. Consequently, this increases the costs of evidence generation, delays broad adoption, frustrates potential buyers and end-users, and can even lead to commercial failures, as seen in some cases this year.
OVERVIEW: In the dynamic arena of digital health, a pivotal debate focuses on the optimal strategy for addressing the evidentiary needs of a diverse range of stakeholders and decision-makers — including payers, purchasers, clinicians, and patients — in the development and deployment of DHPs for broad acceptance. The proliferation of FDA-authorized DHPs has not resulted in broad patient access to their benefits nor commercial viability for many of their developers. The challenge lies in the fact that despite FDA authorization, many DHPs fail to achieve broad patient access or commercial viability. Regulatory acceptance, while necessary, is not sufficient for the success of DHP innovation. It does not guarantee that the benefits of healthcare digitization will impact health, healthcare, and health economics at a large scale. This panel will center around finding the balance with the right strategies, optimized commercial model, and case examples for finding fit-for-purpose strategies for making progress for broad access and acceptance of DHPs.
Megan Coder, PharmD, MBA
Digital Medicine Society, Arlington, VA, USA
Jeff Gombala, BA
ZS, Atlanta, GA, USA
Benjamin Parcher, PharmD, MS
Boehringer Ingelheim Pharmaceuticals, Odessa, FL, USA
Jordan Silberman, MD PhD
Elevance Health, San francisco, CA, USA
ISSUE: The first phase of Medicare drug price negotiation is under way, but how the Centers for Medicare and Medicaid Services (CMS) will put all the pieces of information together in its initial offer prices and subsequent negotiation remains a critical unknown for stakeholders and the academic community. Since CMS has suggested that they will evolve this process as they learn more and their capabilities grow, this panel will present key options for how the disparate types of information, including qualitative and quantitative elements, can be integrated and translated into the negotiation process, highlighting key pros and cons of different approaches, and exploring the role that external input can best support CMS in this effort.
OVERVIEW: Steve Pearson (Moderator) (10 minutes) will set the stage by outlining the elements that the inflation reduction act (IRA) sets out as relevant for determining initial price offers by CMS. Marina Richardson (12 minutes) will share elements of ICER’s submission to CMS, which included a comparative clinical and cost-effectiveness assessment of apixaban and rivaroxaban, both of which are on the initial negotiation list. Brett McQueen (12 minutes) will discuss how a structured deliberation could be used by CMS to aggregate and prioritize evidence in a way that acknowledges the relative importance of factors being considered in the determination of an initial offer price. Kyle Hvidsten (12 minutes) will react to both presentations and share the views and concerns of industry on how information should be integrated to guide CMS actions. The floor will then be open to interactive polling and Q&A with the audience, and discussions about how the research and policy community should focus their efforts to inform CMS processes.
Steven Pearson, MSc, MD
Institute for Clinical and Economic Review, Boston, MA, USA
Kyle Hvidsten, MPH
Sanofi, Cambridge, MA, USA
Marina Richardson, PhD
Ana Paula Etges, PhD, Eng.
PEV Healthcare Consulting, Porto Alegre, RS, Brazil
Anne Geubelle, MBA
Prologica, Lisboa, NA, Portugal
Prakash Jayakumar, MD PhD
Dell Medical School, The University of Texas Health Austin, Austin, TX, USA
Carisi Anne Polanczyk, ScD, MD
PEV Healthcare Consulting, Porto Alegre, Rio Grande do Sul, Brazil
Abeed Sarker, PhD
Emory University School of Medicine, Atlanta, GA, USA
Ergin Soysal, MD PhD
Eli Lilly and Company, Indianapolis, IN, USA
Hua Xu, PhD
Yale University School of Medicine, New Haven, CT, USA
Xiaoyan Wang, PhD
University of Connectict, Westport, CT, USA
Jack Bowden, PhD
Novo Nordisk, Oxford, Oxfordshire, United Kingdom
Laura Güdemann, PhD
University of Exeter, Exeter, Devon, United Kingdom
Gemma Taylor, PhD
Health Economics and Outcomes Research Ltd, Cardiff, United Kingdom
Ota will open the session by describing the importance of accounting for equity within HTA from the industry perspective and then will invite panellists representing diverse geographical regions and professional backgrounds to share the current state of health equity research within HTA. Then, each speaker will describe how health equity is currently considered within HTA in their region based on their experience in their professional role (Dr Igarashi-academic, Dr Piniazhko-regulatory, and Dr Whittington-value assessment). Each panellist will present an agenda for the next three to five years that will allow for the better incorporation of health equity considerations within HTA decision-making and research activities. The role of each stakeholder group (i.e., industry, academic, regulatory, value assessment) in promoting the consideration of health equity will be addressed, reflecting upon panellists’ diversified professional roles. Using the polling software, we will ask participants about the outlook of health equity at the beginning and the end of the workshop. Twenty minutes will be reserved for audience comments.
Riku Ota, MPH
Novo Nordisk Pharma Ltd., Nerima, 13, Japan
Ataru Igarashi, Ph.D.
Yokohama City University School of Medicine, Yokohama, Kanagawa, Japan
Oresta Piniazhko, PhD
State Expert Centre of the Ministry of Health of Ukraine, Kyiv, Ukraine, Kyiv, Ukraine
This forum will focus on three areas of innovations that have the potential to make a paradigm shift in how RWD/RWE is used:
The forum will start with a discussion on NLP, which is now an accepted way to mine text data. The experience of NLP can provide a roadmap for faster adoption of other innovations. In addition, new NLP techniques will be explored. The forum will also discuss the role that synthetic data can play in RWE generation and propose ideas on how to overcome the current challenges to adoption. Finally, the forum will discuss using RWD to identify novel drug signals, and how this can improve efficiency in drug development. The speakers will present case studies and outline how these innovations can improve healthcare decision-making.
Sandipan Bhattacharjee, MS, PhD
Bayer US LLC, Belle Mead, NJ, USA
Jiang Bian, PhD
Lucy Mosquera, BSc, MSc
Replica Analytics, Ottawa, ON, Canada
Elizabeth Lynch will discuss the state of the science on community-based nutrition interventions for individuals with hypertension and strategies for community-partnered research aimed at improving nutrition and reducing chronic disease. Mark Nuijten will examine the results of the experience of applying health economics evaluation for nutrition interventions in malnutrition, including malnutrition related to obesity and to oncology. Jason Shafrin will discuss the opportunities and challenges of economic evaluation of nutrition interventions and compare economic analysis of nutrition interventions with analysis of pharmaceutical interventions.
Kirk Kerr, PhD
Abbott Nutrition, Columbus, OH, USA
Elizabeth Lynch, PhD
Rush University, Chicago, IL, USA
Jason Shafrin, PhD
Center for Healthcare Economics and Policy, FTI Consulting, Los Angeles, CA, USA
Yan Zhi Tan, MSc (Health Economics and Management), BSc (Pharm) (Hons)
Monitor Deloitte, Brussels, VWV, Belgium
Kagan Atikeler, MPharm.
Sanofi, Ankara, 06, Turkey
PURPOSE: ISPOR's patient-centered special interest group defines 'patient engagement in research' as “the active, meaningful, and collaborative interaction between patients and researchers across all stages of the research process, where research decision-making is guided by patients’ contributions as partners, recognizing their specific experiences, values, and expertise.” In the past, this definition was largely aspirational. This workshop will introduce participants to real-world examples of how global pharmaceutical and research organizations operationalize meaningful patient engagement and measure its' impact across research portfolios. Topics covered include strategies for collecting high-quality patient experience data (PED), partnering with cross-functional teams to integrate PED across key decisions, and measuring the impact of patient engagement across drug and core outcome set development.
DESCRIPTION: Elisabeth Oehrlein will moderate the session, introduce key terminology, and engage the audience to consider how the processes, tools, and lessons learned from the case studies can apply to their work. Beyza Klein will describe the Novartis approach of collaborating with the patient community; building a thorough understanding of patients' life experiences to guide decision-making; and establishing standardized methods to measure the impact of patient engagement efforts throughout a product's lifecycle. She will outline how pharmaceutical companies and other stakeholders can contribute to creating a patient-centered health ecosystem by adopting systematic approaches to measure and communicate the outcomes of their patient engagement initiatives. Stacie Cavallaro will describe how BioMarin adapted the National Health Council's Patient Experience Mapping Toolkit to standardize a process for capturing and utilizing patient and caregiver insights starting early in clinical development. Jennifer Bright will describe the International Consortium for Health Outcomes Measurement’s (ICHOM) approach to engaging patient representatives to identify the outcomes that matter most to patients, develop core outcome sets, and drive adoption and reporting of these measures worldwide to drive value-based healthcare.
Elisabeth Oehrlein, PhD, MS
Applied Patient Experience, LLC, Washington, DC, USA
Jennifer Bright, MPA
ICHOM, Alexandria, VA, USA
Stacie Cavallaro, BS
BioMarin Pharmaceutical, San Rafael, CA, USA
Beyza Klein, MA
Novartis, Basel, Basel, Switzerland
Thomas Hubbard, MPP
Network for Excellence in Health Innovation, Natick, MA, USA
Alison Sexton Ward, PhD
USC Schaeffer Center, Los Angeles, CA, USA
Michael Sherman, MD, MBA, MS
RA Ventures, Boston, MA, USA
Value and health technology assessment (V/HTA) bodies have established opportunities for patients and other stakeholders to contribute to assessments. These opportunities have revealed gaps in data and evidence to support patient-centered, “whole health” V/HTA.
To overcome gaps, patient groups, individually or in partnership with V/HTA bodies, have conducted patient surveys to inform reviews, providing information about patient experiences with treatment not collected in clinical trials or claims data, such as indirect costs or caregiver burden. These surveys often represent the best available data, reduce data gaps, and advance V/HTA centered on patients' experiences, perspectives, and needs. However, there is often discordance between the information submitted to health economic modelers and the information collected in patient experience surveys. There can also be a lack of understanding of how survey data impacts model outputs.
This workshop will present a disease-agnostic survey template, highlight the merits of a universal survey template, consider relevance to specific economic modeling decisions, and explore modifications to drive meaningful adoption and use in real-world settings.
Kimberly Westrich, MA
National Pharmaceutical Council, Herndon, VA, USA
Kayleigh Majercak, MS
University of Maryland School of Pharmacy, Washington, DC, USA
Joe Vandigo, MBA, PhD
Applied Patient Experience, LLC, Greensburg, PA, USA
Steven A. Farmer, MD, PhD
Centers for Medicare & Medicaid Services, Baltimore, MD, USA
Douglas Kelly, MD
Peter Neumann, ScD
Paul Arora, PhD
University of Toronto, Toronto, ON, Canada
Mei Yang, Ph.D.
Happy Life Technology, Short Hills, NJ, USA
Anirban Basu, PhD, MS
University of Washington, Seattle, WA, USA
William Herring, PhD
RTI Health Solutions, Research Triangle Park, NC, USA
Amanda Cole, PhD
Office of Health Economics, London, LON, United Kingdom
Boshen Jiao, PhD, MPH
Harvard T.H. Chan School of Public Health, Boston, MA, USA
Phaedra Corso, PhD, MPA
Indiana University, Indianapolis, IN, USA
Julia F. Slejko, PhD
University of Maryland Baltimore, Baltimore, MD, USA
ISSUE: The impact on biopharmaceutical R&D from reduced expected reward size has been a recurring theme in the ongoing debates on the Inflation Reduction Act (IRA) in the US. There is currently no consensus from the HEOR community on the scale and magnitude of the elasticity of innovation to expected reward. Existing empirical evidence differed in their methods, data sources, and estimates. A more thorough understanding of the scale of elasticity of innovation is essential to inform policy making, ensuring the balance between affordability and innovation. The purpose of this session is to review the strengths and weaknesses of existing evidence, identify areas for further research, and discuss the implications of the findings on policy making and social welfare.
OVERVIEW: Gunnar Esiason will first describe the importance of pharmaceutical innovation for patients based on his lived experience with cystic fibrosis, and underscore the need to understand the true scale of the elasticity of innovation (10 minutes). Peter Kolchinsky will demonstrate the impacts of proposed policy changes from IRA using net present value modeling, and show how regulatory changes can impact R&D decisions from an investor perspective (10 minutes). Lotte Steuten will present a select set of existing empirical estimates from the literature, and discuss the strengths and limitations of each approach (10 minutes). Amitabh Chandra will highlight areas that should be prioritized for further research, and the implications for more informed policy making and welfare of current and future patients. The session will end with Q&A with the audience (20 minutes). Polling questions will be used throughout to identify consensus on the range of empirical estimates of innovation and priorities of areas for further research. This session will be relevant for a wide range of stakeholders including patients, employers, payors, modelers, policymakers, and value assessors.
Gunnar Esiason, MBA, MPH
Boomer Esiason Foundation, New York, NY, USA
Amitabh Chandra, PhD
Katherine Lofgren, PhD, MPH
Abett, Inc., Denver, CO, USA
Jalpa A. Doshi, PhD
University of Pennsylvania, Philadelphia, PA, USA
Luke Prettol, BA
AT&T, Spring, TX, USA
ISSUE: The World Health Organization (WHO) defines health equity as the absence of unfair, avoidable, or remediable differences in health among population groups defined socially, economically, demographically, or geographically. According to this definition, an increase in distributional fairness in health outcomes across these groups implies a positive health equity impact. Attention and commitment to improving health equity has grown, as evidenced by initiatives like the Centers of Medicare and Medicaid Services (CMS) updating the basis of 2027 Star Ratings to “Health Equity Index” (HEI) from the current “Reward Factor”. Consequently, methods like DCEA, have evolved from conventional CEA to also quantify health equity impact. This session will feature a debate among a methodologist, physician, and payer on whether and how DCEA can inform and improve health equity, with an oncology focus.
OVERVIEW: DCEA is an intuitively appealing extension of conventional CEA to quantify health inequality impact of a medical intervention. The purpose of this panel is to posit the question, how far can DCEA go in informing health equity-related decision-making in oncology from multi-dimensional perspectives including a methodologist, clinician, and payer. After a session introduction and opening statement on the oncology context of DCEA (Grossman ~5 minutes), panel members will discuss its methodological considerations (Jansen), clinical relevance (Trinh), and payer value (Lopes) (~10 minutes each). Subsequently, panel members will have a debate led by the moderator (25 minutes). In addition to audience questions, the following will be addressed: Is DCEA credible, useful, implementable, impactful, or even the right measure for health equity? How does DCEA fit in with existing frameworks that address health equity such as CMS AHEAD Model, Enhancing Oncology Model (EOM), and National Committee for Quality Assurance (NCQA) Health Equity Accreditation for health plans? How can methodologists best work with physicians and payers to operationalize and optimize DCEA in practice?
Jamie Grossman, PhD, MBA
Bayer Pharmaceuticals, Westerville, OH, USA
Maria Lopes, MD, MS
MMDLOPES, LLC, Cresskill, NJ, USA
Quoc-Dien Trinh, MD, MBA
Brigham and Women’s Faulkner Hospital, Chestnut Hill, MA, USA
Seamus Kent, PhD
Erasmus University Rotterdam, Rotterdam, ZH, Netherlands
Gregory Calip, PharmD, MPH, PhD
AbbVie Inc., North Chicago, IL, USA
Grace Hsu, MSc
Cytel Canada Health Inc., Waltham, MA, USA
Xiaoliang Wang, PhD, MPH
Flatiron Health, Inc., New York, NY, USA
PURPOSE: Bayesian borrowing methods are seeing growing uptake in regulatory settings for supplementing limited sample sizes in rare indications–particularly in pediatric populations. These methods can allow for the amount of information borrowing to depend on the degree of heterogeneity across data sources, with greater borrowing from the external data when outcomes are compatible across data sources and down-weighting of the external data when outcomes are incompatible. This dynamic borrowing approach has also been used to partially borrow information across different subpopulations in basket trials, to supplement limited sample sizes in pediatric trials by borrowing from adult trial populations, and to augment small concurrent control arms in randomized controlled trials. The goal of the workshop will be to introduce these methods to a health economics and outcome research (HEOR) audience and outline some recent applications. Lastly, potential for application of these methods to support future health technology assessments (HTA) will be discussed.
DESCRIPTION: Dr. Arora will provide a brief introduction to the topic (5 minutes). Dr. Springford will outline the challenges faced when evaluating novel therapies targeting rare indications, including challenges of recruiting enough patients to power trials, survival data immaturity, and potential barriers to conducting randomized controlled trials, and will provide an introduction to Bayesian borrowing methods, including static and dynamic borrowing approaches and use of Bayesian priors. (15 minutes) Ms. Mackay will provide an introduction and overview of several prior-based and hierarchical modelling approaches that have seen recent application in regulatory and HTA settings. (15 minutes) Dr. Dayimu will then discuss an application to economic evaluation in a basket trial setting. (15 minutes). The session will conclude with a Q&A / panel discussion on the potential for Bayesian borrowing methods in future HTA applications. An interactive smartphone-based polling feature will be used to encourage audience participation.
Alimu Dayimu, PhD
University of Cambridge, Cambridge, United Kingdom
Emma Mackay, MA, MSc
Inka Health, Toronto, ON, Canada
Aaron Springford, PhD
Cytel, Toronto, ON, Canada
PURPOSE: Medication adherence is an important determinant of patient outcomes, their quality of life, and overall healthcare utilization. Identifying prevalent adherence patterns can assist decision-makers in targeting these populations and tailoring interventions to enhance medication adherence; however, current evaluation of adherence is limited. For instance, the Pharmacy Quality Alliance (PQA) organization has endorsed the use of the proportion of days covered (PDC) as the primary method of measuring adherence. While PDC is easy to calculate, there are several drawbacks to using it as the primary measure of medication adherence. As an alternative, group-based trajectory modelling (GBTM) that can determine and cluster groups of patients based on their dynamic adherence patterns may be better suited to study the dynamic nature of medication adherence behaviours. In this workshop, we aim to introduce concept of GBTM and provide a step-by-step overview on how to utilize these models to study medication adherence. At the end of this workshop, participants will gain a better understanding of how to conduct GBTM with R.
DESCRIPTION: Workshop participants with intermediate-level subject knowledge will be familiarized with key considerations when assessing medication adherence using GBTM. Dr. Park will begin the workshop by introducing the concept of medication adherence and the limitations of PDC (10minutes). Next Dr. Thorlund will introduce the concept of GBTM and other latent trajectory analyses (10-minutes). Dr. Diop will demonstrate how GBTM can be used to study medication adherence using a case study and pre-population R codes (20 minutes). Dr. Metcalfe will conclude by discussing the Reporting Guidelines for GBTM and other latent trajectory analyses and ways to avoid common pitfalls of GBTM (10-minutes). In addition to conducting real-time polling to assess the audience's experience with medication adherence studies, we will allocate time for questions (10 minutes).
Jay JH Park, PhD
McMaster University, Hamilton, ON, Canada
Awa Diop, PhD
Core Clinical Sciences, Montreal, QC, Canada
Rebecca Metcalfe, PhD
Core Clinical Sciences, Calgary, BC, Canada
Reed Johnson, PhD
Department of Population Health Sciences, Durham, NC, USA
Lisa C Rosenblatt, MD, MPH
Bristol Myers Squibb, Lawrenceville, NJ, USA
Michael Taylor, PhD
Genenentech, Redwood City, CA, USA
Rifky Tkatch, PhD
Optum, Oak Park, MI, USA
Bryan Bennett, PhD, Cpsychol
Jazz Pharmaceuticals, Uxbridge, Uxbridge, United Kingdom
David Alderson, MBA, MEng
Cogentia Healthcare Consulting, Cambridge, United Kingdom
Diana Brixner, RPh, PhD, FAMCP
University of Utah College of Pharmacy, Salt Lake City, UT, USA
Christopher Teale, BSc(Hons) MORS MSIB
TealeHealth, Chesterfield, Derbyshire, United Kingdom
Ulrich Neumann, MBA, MSC, MA
Johnson & Johnson, Titusville, NJ, USA
A. Mark Fendrick, MD
University of Michigan, Ann Arbor, MI, USA
Annette James, MAAA, FSA, FCA
American Academy of Actuaries, Washington, DC, USA
Shannon Cope, MSc
PRECISIONheor, VANCOUVER, BC, Canada
Harlan Campbell, PhD
University of British Columbia, Rossland, BC, Canada
Neil Hawkins, MSc, PhD, MBA, CStat
University of Glasgow, Oxford, United Kingdom
PURPOSE: Target Trial Emulation (TTE) represents an advancement in HEOR by offering a rigorous approach to establish treatment effectiveness from observational data, thus overcoming the limitations of traditional real-world evidence (RWE) studies. This workshop will delve into how TTE addresses the critical challenge of bridging the gap between randomized controlled trials and RWE, enhancing the accuracy and applicability of healthcare interventions in diverse patient populations, including case studies involving target trial emulation applications in practice. Topics include (1) how to benchmark studies, (2) per-protocol estimation and what adherence means, (3) external comparators from RWE, and (4) data limitations, including unmeasured confounders and missing data problems
DESCRIPTION: Participants will gain insights into crucial TTE aspects, including benchmarking studies, per-protocol estimation and understanding adherence, leveraging real-world external comparators, and addressing missing data. Eran Bendavid will introduce TTE, emphasizing its significance in bridging the gap between observational studies and randomized controlled trials. The discussion leaders will guide participants through real-world case studies illuminating the practical application of TTE. Manuel Gomes will describe the nuances of benchmarking in TTE and its role in ensuring robust study design and interpretation. He will also speak about the intricacies of per-protocol estimation and challenges in determining treatment adherence, with practical insights from relevant case studies. Alind Gupta will describe the use of external comparators in the context of target trial emulation, focusing on their calibration, application and interpretation. Dr Gupta will also address challenges related to data limitations, including unmeasured confounders and missing data. Audience participation will be encouraged through interactive discussions, where attendees can share their perspectives and experiences. We will tailor this workshop for researchers, statisticians, and practitioners interested in gaining practical insights into applying TTE in real-world scenarios.
Alind Gupta, PhD
Eran Bendavid, MD, MS, AB
Stanford Univeristy, Stanford, CA, USA
Manuel Gomes, PhD
University College London, London, United Kingdom
Adrian Towse, MA, MPhil
Danny Yeh, PhD
AESARA, Burlingame, CA, USA
Selena Daniels, PharmD, PhD
US Food and Drug Administration, Silver Spring, MD, USA
Ebony Dashiell-Aje, PhD
Natalie Engmann, PhD
Denali Therapeutics, South San Francisco, CA, USA
Nicole Lyn, MPH
Daniel de Vicente, BS
ASMD España, Madrid, Spain
Michael Drummond, PhD
University of York, Lichfield, Staffordshire, United Kingdom
ISSUE: The cost-effectiveness of diagnostic tests hinges on their ability to correctly diagnose a condition and guide appropriate treatment, which is expected to improve clinical outcomes. How might diagnostic tests be valued for conditions that lack optimal treatment? Many infectious diseases provide good examples – e.g. norovirus, rhinovirus, bacterial vaginosis. There are currently no treatment options for norovirus and rhinovirus. Despite antibiotics being available for bacterial vaginosis, symptom persistence/recurrence rates are high. For these conditions where improvements in clinical outcomes may not be achieved, how is the value of a correct diagnosis perceived? How does the valuation criteria differ for diagnostics intended for conditions with good treatment options? Does disease severity affect the perceived value? The aims of this issue panel are: 1) to use infectious disease examples to explore more complex relationships between treatment effectiveness and diagnostic value across multiple perspectives; 2) explore different value parameters for diagnostics and how these may be captured in cost-effectiveness analysis.
OVERVIEW: The moderator will present a 5-minute overview of the issue. Each panel member will then have 10 minutes to present their perspective. This will include theoretical consideration of how test value is quantified in cost-effectiveness analysis; manufacturer perspective on the unmet need for improved testing modalities for infectious diseases; and payer perspective on current priorities and considerations for diagnostic reimbursement. After the presentations, the moderator will summarise and pull out the key themes and consider wider applicability to additional disease areas before a 20-minute panel discussion with audience participation. It’s expected that researchers and manufacturers in the diagnostic tests field and payers would benefit from this discussion.
Karissa Johnston, PhD
Broadstreet Health Economics & Outcomes Research, Vancouver, BC, Canada
Lon Castle, MD
eviCore healthcare, Bluffton, SC, USA
Mindy Cheng, MS, PhD
Cepheid, Sunnyvale, CA, USA
Beth Devine, PhD, PharmD, MBA
Yilin Chen, MPH, PhD
Curta, Inc., Seattle, WA, USA
PURPOSE: The ISPOR value flower has provided the field with a list of ‘what’ novel value elements should be incorporated into cost-effectiveness analysis (CEA), among them the value of health equity. Health equity, while important to consider, does not have an established system of measurement and weighting on economic evaluation. Recent measurement and methodological advancements that consider the importance of health equity on decision-making can play into approaches for generalized cost-effectiveness analysis (GCEA).
DESCRIPTION: Padula will provide an overview GCEA and the relative importance of adjusting economic models for health equity. Lakdawalla will introduce the generalized risk-adjusted cost-effectiveness (GRACE) framework as a way of overcoming discriminatory decision-making by adjusting for baseline disability and concerns about health equity. Basu will introduce a new measurement system to health utility, Healthy Years in Total (HYT) as an alternative to QALYs that addressed health equity concerns. Vasco Pontinha will introduce a novel method to encapsulate health equity based on budgetary constraints for mainstream health technologies. At the end of the workshop, the discussants will poll the audience about which method(s) may be able to gain traction to make U.S. health technology assessment processes more equitable.
Vasco Pontinha, PhD, MPharm, MBA
Virginia Commonwealth University School of Pharmacy, Richmond, VA, USA
PURPOSE: Since 2018, China has adopted a centralized drug review and price negotiation process to inform national coverage decision making. Quality-adjusted life year (QALY) is a recommended outcome measure for this process. However, all existing patient-reported outcome measures (PROMs) were developed in western countries. There was a need for PROMs that measure important aspects of health perceived by Chinese populations. The China Health-Related Health Outcome Measures (CHROME) were developed for this purpose. CHROME is a system of preference-based PROMs that consists of a core module for generic use and a series of disease-specific modules for measuring disease or condition-specific impact. This system has a novel design in which both generic and disease specific measurement needs are integrated throughout the conceptualization, validation, and valuation. Therefore, they can be used in clinical and economic evaluations with consistency and transitivity, while maintaining independence. The primary goal of this workshop is to introduce the development and validation of the CHROME system and its application to support drug price negotiation in China.
Kun Zhao, PhD
Tsinghua University, Beijing, Beijing, China
Qiang Fu, PhD
China National Health Development Research Center, Bei Jing, China
Xue Li, PhD
China National Health Development Research Center, Beijing, China
Feng Xie, PhD
PURPOSE: Anchored covariate-adjusted indirect treatment comparisons (ITCs) inform reimbursement decisions when (1) there are no head-to-head trials between the treatments of interest, (2) there is a common comparator arm shared by two studies, and (3) there are patient-level data limitations. Matching-adjusted indirect comparison (MAIC) is currently the most widely used method in HTA but is not always possible. We begin with an overview of newly proposed MAIC weighting schemes and approaches for assessing the numerical feasibility of conducting an MAIC. We then consider the "two-stage MAIC", an extension to MAIC which has been recently proposed and shown to yield improved precision and efficiency, while maintaining similarly low levels of bias when assumptions are met. We review the two-stage MAIC and how it has recently been applied to achieve greater precision. Finally, certain approaches in causal inference are known to be “doubly-robust” meaning that they will provide unbiased estimates so long as either the trial allocation model or the outcome model is correctly specified. However, doubly-robust methods for ITCs have not yet been proposed. Here we consider the potential of “augmented” MAIC for doubly-robust estimation in anchored ITCs.
DESCRIPTION: Workshop attendees will review the main advantages and limitations of MAIC and be introduced to newly proposed methods. The audience will be asked (real-time polling) to consider if these “advanced” methods should be more widely considered in HTA . Ms. Cope will chair the session and introduce the main issues involved with existing MAIC methods and summarize recent work on alternative weighting schemes and feasibility assessment (20 min). Dr. Remiro-Azócar will summarize his recent work on the two-stage MAIC method (15 min) and Dr. Campbell will consider the potential of “augmented” methods for doubly-robust estimation (15 min). To conclude, an audience discussion will consider the potential and priorities for MAIC methods research (10 min).
Antonio Remiro-Azócar, PhD
Bayer, London, LON, United Kingdom
PURPOSE: Real world evidence (RWE) is only trusted by regulatory bodies, health technology assessment (HTA) organizations, payers, clinicians and patients if the underlying real-world data (RWD) is demonstrated to be high quality and fit-for-purpose. Electronic health records (EHRs) are an important source of real-world data: they are the primary record of the patient symptoms, diagnoses, treatments, outcomes, and in many cases, preferences and experiences. However, EHRs were not designed for research, and clinician engagement and interaction with EHRs varies widely from scope of practice to health care organization type. Clinician documentation in EHRs can often be limited by time pressure and administrative burden. Moreover, much information is captured in unstructured formats within the EHR, and coding systems and other factors often vary between health system EHRs. Patient records may also be split between multiple health systems and private practice EHRs. In this workshop, we will explore how all of these factors impact RWE generated from EHR data, and how these challenges can be overcome to improve trust and transparency around uses and the reliability of EHR data for research and regulatory purposes.
DESCRIPTION: In this workshop, Rachele Hendricks-Sturrup will describe health system practices in generating RWE to support learning health system goals, and Duke-Margolis’ current focus on operationalizing EHR data relevance, reliability, and quality to build trust and transparency among health system stakeholders. Mac Bonafede, Elise Berliner and Louis Ehwerhemuepha will take some of those examples and describe the strengths and weaknesses of EHR data for the specific questions and how they are working to improve the validity and quality of EHR data to answer these questions. The panel will take questions on specific challenges audience members are facing using EHR data and other RWD, and brainstorm solutions with panel and audience members.
Elise Berliner, PhD
Oracle Life Sciences, Austin, TX, USA
Mac Bonafede, PhD
Veradigm, Brentwood, NH, USA
Louis Ehwerhemuepha, PhD
Children's Hospital of Orange County, Orange, CA, USA
Rachele Hendricks-Sturrup, DHSc, MSc, MA
Duke-Margolis Center for Health Policy, Washington, DC, USA
Padula will provide an overview GCEA and the relative importance of adjusting economic models for health equity. Lakdawalla will introduce the generalized risk-adjusted cost-effectiveness (GRACE) framework as a way of overcoming discriminatory decision-making by adjusting for baseline disability and concerns about health equity. Basu will introduce a new measurement system to health utility, Healthy Years in Total (HYT) as an alternative to QALYs that addressed health equity concerns. Vasco Pontinha will introduce a novel method to encapsulate health equity based on budgetary constraints for mainstream health technologies. At the end of the workshop, the discussants will poll the audience about which method(s) may be able to gain traction to make U.S. health technology assessment processes more equitable.