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Questions:

00:00: Introduction

01:23: Health economists now sit closer to power than ever. Is their role still primarily to inform policy, or has the field crossed into shaping it – and where should that line be drawn?

03:46: How can economists best help policymakers understand alternative options and opportunity costs without drifting into advocacy?

06:19: Have we reached a genuine stand-off in the healthcare value debate, or have underlying philosophical differences simply become impossible to smooth over?

15:58: Why do health systems and even health economists struggle to confront the reality of scarcity, and what real-world examples illustrate this?

21:22: Is HEOR being asked to stretch beyond its traditional focus on efficiency and equity into broader industrial strategy, and are we equipped to do that well?

Michael Drummond

Professor Emeritus and former Director, Centre for Health Economics, University of York

Michael Drummond is Professor Emeritus and former Director of the Centre for Health Economics at the University of York in the UK. His main field of interest is in the methodology and practise of economic evaluation in health care. He is the author of two major textbooks and more than 750 scientific papers. He has advised several governments on the assessment of health technologies and acted as a consultant to many life sciences companies. In October 2010 he was made a member of the National Academy of Medicine in the USA. He served for 14 years as Co-Editor-in-Chief of Value in Health and was made Editor Emeritus in May 2024.

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Questions:

00:00: Introduction

01:16: What’s changing in pricing reform, and why is this moment important?

03:11: How do MFN and reference pricing work, and how do they differ in purpose and impact?

06:18: Why are these tools so appealing to policymakers?

07:00: MFN pricing is often framed as a fairness issue. From an access and economics perspective, where does that argument fall short?

09:35: One concern is that MFN doesn’t just affect the US, it reshapes global pricing behavior. What ripple effects do you see internationally?

12:40: External reference pricing has been around for decades. Why does it feel more consequential, and more dangerous, now than it did 10 or 15 years ago?

15:18: People still talk about ‘Europe’ as if it’s one access environment. Why is that framing fundamentally flawed?

18:30: From your experience in consulting and industry, what does true organizational readiness for European access actually look like?

21:01: How early is ‘early enough’ when it comes to European access planning, and what needs to be decided before Phase III?

22:58: With more coordination across Europe, there was hope for simplification. Are we actually seeing simplification, or just a new layer of complexity?

25:39: As a CrossFit coach, you use “explain, demonstrate, practice, correct.” How does that approach apply to communicating value in access today?

27:15: What does it take to tell an access story that resonates with payers, policymakers, and patients beyond the science and policy?

28:38: Rapid Fire Questions

Ramiro Gilardino

Healthcare Executive specializing in Global Access & Health Policy

Ramiro Gilardino, MD, MSc, is a health policy and innovation specialist with a background spanning global institutions and the private sector. He brings over a decade of experience in HTA, market access policy, and evidence strategy. Ramiro has led and contributed to international collaborations focused on aligning HTA methodologies, advancing affordability frameworks, and enabling lifecycle approaches to decision-making. He currently serves on the Board of Directors at ISPOR, where he champions stakeholder engagement, equity, and policy reform to enhance patient access and making healthcare accessible and affordable.

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Questions:

00:00: Introduction

01:24: What do you see as the real purpose of health economics today?

05:17: Australia’s National Health and Medical Research Strategy is being revisited for the first time in a decade. What is the significance of reassessing R&D spending now, and how could it reshape priorities across hospitals, universities, and health-service research?

09:06: Australia’s PBAC is known globally for rigor and transparency. What does PBAC consistently get right – and where could it improve?

12:48: It can take nearly three years from FDA approval to PBAC listing. Where could the system meaningfully shorten that pathway without weakening the evidence?

19:41: More than 300 oncology drugs were approved globally in five years, yet few reached Australia. Are sponsors more deterred by pricing expectations or by process complexity?

22:48: What does smart, ethical engagement between academia and industry look like in 2025?

25:16: Many universities push for impact yet primarily reward publications. How can institutions better recognize and value policy-relevant or commercial translation work?

29:37: ISPOR’s 2030 Vision calls for bridging evidence and equitable access. What is the single biggest shift needed to achieve that?

32:21: Amid geopolitical and economic pressures, how do you see global pricing and value frameworks evolving – and what might this mean for patient access?

34:01: Healthcare spending is outpacing GDP worldwide. Is sustainability fundamentally an economic problem or a political one?

36:30: If Australia could share HTA workload with one other agency, who would it be, and why?

37:52: You’ve mentored a generation of health economists. What advice would you give your younger self, or emerging economists hoping to influence policy today?

40:56: Rapid Fire Questions

Paul Scuffham

Founder, Centre for Applied Health Economics

Chair of Health Economics, School of Medicine & Dentistry, Griffith University

Professor Scuffham’s research is focused on identifying the value for money of healthcare interventions, elicitation of preferences from patients and the public, and methods of priority setting. He applies his research interests across the breadth of cardiac conditions, genomics, digital health, vaccines and more. At Griffith University, Queensland Australia, he founded the Centre for Applied Health Economics and was Director of the former Menzies Health Institute Queensland, a large research institute with 13 centers and over 500 staff. In his professional society, he was the inaugural President of the Australian Chapter of ISPOR, and past Chair of the Asia Consortium. He was awarded ISPOR’s Marilyn Dix-Smith Leadership Award in 2025. He is a Fellow, Board member, and Chair of the Policy Advisory Committee of the Australian Academy of Health & Medical Sciences. He is a member of the Research Committee of the National Health and Medical Research Committee and a member of the Public Health and Health Systems Committee of the Medical Research Future Fund & NHMRC.

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01:08: How have your personal experiences shaped your understanding of evidence, access, and impact in health policy?

03:24: What core principles have remained constant throughout your work across advocacy, government, and consulting in health policy?

05:20: Can you share an example of a well-designed policy that failed at the implementation stage, and what you learned from it?

06:26: What lessons from implementing the ACA have shaped your approach to structuring practical, workable health policy frameworks today?

08:45: If you were designing a US HTA framework today, what would you build differently to make it work in practice?

11:28: What are your reflections on ICER’s current assessment process, and how could it be improved to make its findings more useful and actionable across the healthcare system?

14:25: Why does the US continue to struggle to fully integrate evidence generation into its pricing and access frameworks?

16:35: If you could design an incentive structure to encourage pharmaceutical companies to share their data, what would it look like and how would you make it work in practice?

20:03: What do you see as the most effective way to align FDA and CMS around patient-centered access?

25:11: If you could design a structured communication process between CMS and industry, what would it look like?

27:28: Can you share an example of how stakeholder feedback directly improved CMS implementation efforts?

35:06: What emerging policy and regulatory trends will most influence pharma market access and RWE over the next decade, and which are still being underestimated?

39:57: Amid rising costs and solvency challenges, what gives you confidence that the US can bend the healthcare cost curve without stifling innovation?

Kristi Martin

Director, Camber Collective

Kristi Martin brings over 20 years of experience shaping health policy through practical, impactful solutions. Currently a Director at Camber Collective, she pairs analytic rigor with a human-centered approach to advance strategies that improve health and well-being. She guides mission-driven organizations through complex health policy landscapes through her own firm, Highway 136 Consulting, which was founded in 2020.

Kristi’s career spans government, philanthropy, and consulting – always focused on making healthcare better and fairer. Most recently, she served as Chief of Staff for Medicare at the Centers for Medicare & Medicaid Services (CMS), where she led strategy and execution for major initiatives under the Inflation Reduction Act, including Medicare drug price negotiations.

Previously, Kristi was Vice President for Health Care at Arnold Ventures, managing their portfolio to lower prescription drug costs. As Managing Director at Waxman Strategies, she provided strategic counsel to foundations and advocacy organizations on issues ranging from prescription drug pricing to women’s health. Earlier, she played a key role in implementing provisions of the Affordable Care Act, advising senior officials, and coordinating policy across federal agencies at the Department of Health and Human Services, CMS, and the Office of Personnel Management. Her federal service across these agencies as well as Government Accountability Office and National Institutes of Health gave her a strong foundation in policy development and implementation. Her roots in DC are in patient advocacy as she started her career with Easter Seals and the American Cancer Society.

Known for turning bold ideas into action, Kristi has advised the White House, represented US interests internationally, and built consensus among diverse stakeholders. She holds a Master of Public Administration from George Washington University and earned both a master’s and bachelor’s degree from the University of Kentucky.

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Questions:

00:00: Introduction

01:15: You’ve worked across government, pharma, consulting, NGOs, and academia. What ties it all together, and what are you trying to solve now?

03:35: Was there a moment that drew you to health economics, or was it a gradual pull?

05:47: You’ve written on value frameworks and outcomes for reimbursement. If you could redesign HTA from scratch, what would you toss and what would you keep?

09:53: You’ve said incentives drive behavior, and competing incentives are a constant challenge. What’s the most misaligned incentive in healthcare today – and who does it hurt most?

15:19: With growing scrutiny of RWE and digital health, is oversight enabling or stifling innovation?

17:54: What’s one policy or regulation you believe is well-intentioned, but has led to poor health or economic outcomes?

22:31: On July 31, President Trump asked 17 pharma companies to match US drug prices to other developed nations. As a health economist, do you think this will work?

27:14: Rapid Fire Questions

Jason Shafrin

Senior Managing Director, Center for Health Economics and Policy, FTI Consulting

Jason Shafrin, PhD, is a Senior Managing Director at FTI Consulting's Center for Healthcare Economics and Policy. Dr Shafrin has over 20 years of health economics research experience serving as trusted advisor and expert to a wide variety of healthcare and life sciences companies, governments and non-governmental organizations (NGOs). Dr Shafrin is an Adjunct Professor at the University of Southern California, the former Director of Research at the Innovation and Value Initiative and the Founder and Editor of the Healthcare Economist blog. Dr Shafrin is an expert in applied economics with a specialty in health economics research. Over his career, he has developed novel methodological approaches, advanced quantitative analyses and innovative modeling to address some of the most difficult health economics problems and provide evidence-based solutions for clients across the healthcare industry. His work has assisted clients in addressing policy changes, navigating regulatory processes, evaluating pricing, value and outcomes and supporting innovation and transformative change. Dr Shafrin’s extensive experience includes projects on value assessment, alternative payment model design, discrete choice experiment survey, real-world data analysis, and digital medicine.

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Questions:

00:00: Introduction

01:02: You entered biopharma through econometrics and discrimination litigation – what connects that work to what you’re solving now?

04:50: You built analytical tools to prioritize policy battles. What’s one battle you lost that still keeps you up at night?

09:03: You’ve said predicting this administration’s moves is impossible. What tools help you turn that chaos into strategy?

14:13: You’ve warned that China is overtaking US clinical development. How real is this shift, what’s driving it, and what are we still in denial about?

18:39: You’ve advocated for more carrots than sticks in policy. What’s the most promising incentive we’re overlooking to drive real health outcomes?

20:32: What, if anything, should health economists and outcomes researchers be saying about issues like biosimilars potentially disappearing and China’s rise?

23:04: If rebates disappeared tomorrow, what reform would you pursue to make formularies more clinically honest and economically sustainable?

25:48: You publish in both journals and op-eds—how do you balance academic neutrality with policy persuasion?

28:38: At recent ISPORs, there’s been debate that HEOR, RWE and market access departments struggle to clearly demonstrate their impact within pharma and policy. What’s your take?

30:22: Can you share an example where health economics truly shaped a policy decision?

32:12: Rapid Fire Questions

Kirsten Axelsen

Senior Policy Advisor, DLA Piper

Kirsten works with leaders in life sciences to navigate policy, competition, reimbursement and public perception for successful product launch, growth and profitability. She has developed and executed strategies at all stages of the biopharmaceutical lifecycle in all major markets. Kirsten brings more than 25 years of experience working in life sciences. She is a biopharmaceutical consultant and an Aspen Institute Health Fellow.

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Questions:

00:00: Introduction

01:03: You work at the intersection of health economics, policy, and investment. How do these domains influence one another in your work, and what gaps are you aiming to bridge?

04:34: Who are you informing differently than has been typical?

07:21: You’ve worked across leading institutions in healthcare. What connects these roles, and what core question or challenge has guided your career??

09:36: How does traditional cost-effectiveness modeling differ from risk-adjusted NPV models, and do you see a hybrid approach emerging? Who might lead that shift?

16:11: If you could redesign the value framework, what elements do you think are missing – and why?

19:27: As a CBO health adviser, what perspective do you bring, and what impact do you hope to make?

22:12: With policies like the IRA taking shape, are we striking the right balance between cost control and long-term innovation, or are new incentives needed?

24:55: In your view, what makes healthcare evidence resonate, and why is some data overlooked?

27:35: As health equity gains attention in economic evaluation, how is HEOR adapting – and where is it still falling short?

31:02: What advice would you offer to women starting out in health economics today?

33:45: Rapid fire questions

Melanie Whittington

Managing Director and Head of the Leerink Center for Pharmacoeconomics, MEDACorp

Melanie (Mel) Whittington is a Managing Director and Head of the Leerink Center for Pharmacoeconomics, which is a division of MEDACorp and an affiliate of Leerink Partners. In this role, she leads pharmacoeconomic evaluations of the societal impact of biopharmaceutical innovations. She is also a Senior Fellow within the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center where she is interested in testing novel health economic methods.

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Questions:

00:00: Introduction

01:26: What’s something outside your professional life that has shaped how you show up day to day?

07:14: Can you share a time when data conflicted with lived experience and shifted your perspective or approach to research?

11:06: Has a single patient story ever changed your perspective more than large datasets? How should research methods account for that?

16:03: As ISPOR President, what conversations are we still not having loudly enough in HEOR or HTA circles?

20:08: As AI scales, how can we ensure underrepresented regions aren’t left behind – and what’s your approach to bridging that gap?

24:49: What can behavioral science teach us about improving health outcomes that traditional models miss?

27:55: How should we address gaps in data that overlook patients’ economic realities, like out-of-pocket costs or caregiving burdens?

30:56: How do we train the next generation of HEOR professionals not just to code, but to care? Where are we missing the mark?

39:38: Rapid Fire Questions

Eberechukwu Onukwugha

Professor and Executive Director of Pharmaceutical Research Computing, University of Maryland, Baltimore

Eberechukwu Onukwugha, PhD, is a Professor in the Department of Practice, Sciences, and Health Outcomes Research and is Executive Director of Pharmaceutical Research Computing at University of Maryland School of Pharmacy. She received a doctorate degree in economics from Virginia Tech and has 20 years of experience conducting health economics and outcomes research. She has authored 140 peer-reviewed articles. She is an editorial board member for PharmacoEconomics and an Associate Editor for Ethnicity & Disease.

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Jeff Trotter doesn’t just study evidence—he shapes how we think about it. A longtime advocate for the thoughtful use of real-world evidence (RWE) in healthcare decision-making, Jeff has helped pharma companies, service providers, and policymakers navigate the evolving landscape of value, validity, and what it means to “know” something in medicine. As the industry embraces new technologies, new stakeholders, and new threats to scientific integrity, Jeff remains a clear, incisive voice—grounded in rigor, yet open to rethinking the gameplan. In this conversation, we talk ROI, regulatory shifts, and what happens when facts become optional.

Questions:

00:00: Introduction

00:53: Since our last conversation, what shifts in the RWE landscape have caught your attention?

10:31: The ROI of RWE is getting clearer, but are we asking the right questions about value?

18:25: What happens when society starts to reject facts?

26:00: What’s one RWE project that exceeded expectations?

32:30: Is observational research declining due to the rise of secondary real-world data and AI, or is it poised for a resurgence thanks to its flexibility?

35:12: Can you share an example where you reached a demographic not captured in secondary data?

37:57: How are CROs being challenged to evolve in this new RWE era?

41:58: You’ve been a thought leader in this space for decades. What’s changed most about how you think about your own role in it?

44:29: Rapid Fire Questions

Jeff Trotter

RWE Pioneer

As an entrepreneur, researcher, consultant, and innovator, Jeff Trotter has been a pioneer in the evolving health economics and outcomes research community and is an industry leader in the design and implementation of patient registries, observational studies, and other RWE initiatives. Having founded, led, and sold two companies in the pharma services space and served as an executive with several global CROs, Jeff currently supports the scientific and strategic RWE initiatives of life-sciences and related organizations through his independent consultancy, J Trotter Research & Consulting.

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Note, this interview took place prior to the Datavant acquisition of Aetion.

Questions:

00:00: Introduction

00:32: Tell us about your career trajectory, and what your focus is now.

05:00: How do you define ‘regulatory-grade’ RWE at Aetion, and how does it differ from evidence used for publications or internal decisions?

09:25: Do you think we've overreached in using RWD, and, if so, in what areas?

12:10: What is Aetion doing to improve RWD quality?

17:44: How are you applying your AI background to data analytics in your current work?

23:41: With rising scrutiny around commercial influence in RWE, what role do public-interest audits, independent replication, or open protocols play at Aetion?

30:29: How is Aetion addressing missing public health data, and can commercial groups help fill these gaps for public benefit?

32:04: Which thinkers have influenced your views on uncertainty and evidence, and how do those ideas shape Aetion’s platform?

34:31: Rapid Fire Questions

Jeremy Rassen

Co-founder & CEO, Aetion

Dr Rassen is an epidemiologist and computer scientist with more than 25 years of experience in how causal inference and AI can drive understanding of the safety, effectiveness and value of medical treatments. He co-founded Aetion to pursue a vision of technology providing the speed, scale and rigor required to answer high-stakes questions in healthcare. Dr Rassen has overseen Aetion’s work with 18 of the 20 top global pharma companies, as well as many other customers around healthcare. During the COVID-19 pandemic, he initiated Aetion’s research collaborations with the US FDA and was a leading voice on how RWE could be applied to decisions around COVID-19 and other critical decisions in healthcare. Prior to Aetion, Dr Rassen was an assistant professor of medicine at Harvard Medical School, where he developed methods to improve the quality and validity of RWD studies. He started his career in Silicon Valley, where he was the first employee at E.piphany to join the company’s founders to build a novel, data-driven customer relationship management (CRM) platform and sell it to Fortune 50 companies. Dr Rassen received a Bachelor of Arts in Computer Science from Harvard University and a Doctor of Science in Epidemiology from the Harvard TH Chan School of Public Health. He is a fellow and board member of the International Society of Pharmacoepidemiology.

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Questions:

00:00: Introduction

00:44: Tell us about your career over the years, and what your focus is now.

03:59: ICER has been a disruptor in the value assessment space. Where do you see it heading over the next five years?

10:02: What is the goal of ICER’s HEMA collaboration with NICE and CDA?

13:00: What do you see as ICER’s biggest strategic blind spot, and how are you addressing it?

16:28: Looking back, what work do you feel made the greatest impact, and what work should have made a difference but didn’t?

20:35: With CMS now negotiating drug prices under the IRA, how is ICER positioning itself?

23:55: What kinds of activities are you invited to participate in that you are not involved in right now, and why?

26:47: You often balance scientific rigor with public trust – how do you strategically prioritize transparency without paralyzing decision-making?

29:58: How is ICER using AI, and assessing its role in value assessment?

31:37: ICER’s review process is grounded in ethics. Have the ethical considerations changed at any point over the past 19 years?

33:14: Rapid Fire Questions

Sarah K Emond, MPP

President and Chief Executive Officer, ICER

With 25 years of experience in the business and policy of health care, Sarah leads the Institute for Clinical and Economic Review, a leading non-profit health policy research organization, as President and Chief Executive Officer. She joined ICER in 2009 as its first Chief Operating Officer and third employee, and has worked to grow the organization’s approach, scope, and impact over the years.

Prior to joining ICER, Sarah spent time as a communications consultant, with six years in the corporate communications and investor relations department at a commercial-stage biopharmaceutical company, and several years with a health care communications firm. Sarah began her health care career in clinical research at Beth Israel Deaconess Medical Center in Boston.

A graduate of the Heller School for Social Policy and Management at Brandeis University, Sarah holds a Master of Public Policy degree with a concentration in health policy. Sarah also received a bachelor’s degree in biological sciences from Smith College. Sarah speaks frequently at national conferences on the topics of prescription drug pricing policy, comparative effectiveness research, and value-based health care. In her free time, Sarah enjoys skiing and hiking in the White Mountains of New Hampshire.

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Questions:

00:00: Introduction

00:40: What is ISPOR’s vision for 2030?

03:39: Which stakeholders and sectors will be key to driving the Whole Health agenda?

10:19: What policy challenges is ISPOR actively addressing?

15:07: The balance between cost-effectiveness and affordability has always been challenging. Is ISPOR making this a core focus or will it remain a peripheral issue for healthcare researchers?

20:38: HEOR and Market Access have grown into distinct but connected fields. How is ISPOR working to improve their efficiency and alignment?

26:46: Is ISPOR involved in any work to demonstrate the value of HEOR?

35:09: Where does ISPOR stand on DEI policy, and what role is it playing in promoting inclusion in research?

42:10: Now that the initial hype around AI has settled, what are the 2–3 key areas ISPOR is focusing on to guide the conversation?

46:33: Rapid Fire Questions

Laura Pizzi

Chief Science Officer, ISPOR—The Professional Society for Health Economics and Outcomes Research

Dr Laura Pizzi is the chief science officer for ISPOR—The Professional Society for Health Economics and Outcomes Research. ISPOR is the leading global professional society for health economics and outcomes research (HEOR). In her role, Dr Pizzi provides strategic scientific leadership and oversees the organization’s major initiatives, including the development of ISPOR’s science strategy, content priorities, and global initiatives such as Special Interest Groups, Patient Council, regional Patient Representative Roundtables, Health Technology Assessment (HTA) Roundtables, and the organization’s AI and Digital Health member initiatives. Her team is also responsible for engaging with global healthcare authorities to support the use of HEOR in healthcare decision making. With a strong passion for education, Dr Pizzi co-led the development of ISPOR ’s first Health Economics and Outcomes Research Competency Framework, which outlines the core knowledge and skills essential to the HEOR profession.

Dr Pizzi brings more than 25 years of academic and research expertise to ISPOR. She has led multidisciplinary teams of methodologists, statisticians, and clinicians in designing and conducting cost and outcomes analyses for both pharmacologic therapies and nonpharmacologic interventions. Much of her work has focused on improving the quality of and access to treatments for older adults in underrepresented communities for diseases of aging such as dementia, diabetes, chronic vision diseases, and vaccine-preventable illness. Her achievements include leading health economic analyses for numerous multicenter clinical trials, testifying before the US House of Representatives Committee on Ways and Means on Medicare spending, authoring a textbook on economic evaluation, and publishing more than 100 peer-reviewed manuscripts and 120 research presentations.

In addition to her leadership role at ISPOR, Dr Pizzi serves as research professor at Rutgers University, Ernest Mario School of Pharmacy. Her academic career has centered on advancing the science of health economics, influencing healthcare policy, and training the next generation of HEOR professionals.

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Questions:

00:00: Introduction

01:15: Transforming pharmaceutical discovery to accelerate drug development has been discussed for decades. What sets your perspective apart, and what makes your proposals unique?

17:05: Is what you're talking about a new way of value assessment, or is it implementation science?

27:16: What new terminology should we get comfortable with?

33:24: There have been some recent publications and conversation from major associations evaluating the value of HEOR. How can the field stay relevant and closely connected to customers?

44:20: Rapid Fire Questions

Melvin 'Skip' Olson

Founder, Olson Strategies

Skip Olson is the Founder of Olson Strategies and focuses on using his 30 years of experience in the pharmaceutical industry to develop strategies and solutions on topics related to Evidence and Implementation Science. He was recently the Global Head of Integrated Evidence Strategy and Innovation at Novartis, where he was responsible for promoting the very best in research methodology and applications of Real World and other Evidence across all therapeutic areas and around the globe to drive better decision-making. He comes from a background in HE&OR, where he has led the use of RWE to transform the generation of patient insights and value for money assessments. He earned a ScD in Biostatistics from Harvard University.

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Questions:

00:00: Introduction

01:47: As the head of A Rare Cause, what is the company’s mission, and what problem are you addressing?

04:03: What are the unique issues around rare diseases in Africa that sets it apart from other continents or populations? How are you tackling these?

11:17: What ethical challenges do you face in rare diseases, and how are these issues unique to Africa yet relevant globally?

17:49: Let’s talk about rare disease drug pricing. What challenges do you face, and what unique solutions have you proposed to Pharma, NGOs, and policymakers?

21:27: Can you elaborate on your proposed subscription model for rare diseases?

27:08: How do your patients define value?

28:43: Over your multi-faceted career addressing rare diseases over several continents, what are your top learnings?

31:06: What do you wish stakeholders knew, and how would you like them to join you in solving these challenges?

36:43: Rapid Fire Questions

Christian Hendriksz

Chief Community Impact Officer, A Rare Cause

Chris Hendriksz qualified as a medical doctor in South Africa in 1985 from the University of Pretoria. He completed his Master’s degree in Sports Medicine at the same university. Following a rare disease diagnosis of his own child, he moved to the UK, where he continued his studies, becoming an expert in inborn errors of metabolism across all ages. He obtained his MRCP and FRCPCH with subspecialty registration after training at the world renowned Willink Unit in Manchester, UK.

He spent 22 years in the UK National Health Service in various roles leading both pediatric Birmingham Children’s Hospital and adult Salford Royal NHS foundation trusts rare disease centres with patient populations in excess of 1500 at each unit. Currently, he is an Extraordinary Professor of Human Metabolomics at North-West University, Potchefstroom, South Africa, and an Extraordinary Professor of Paediatrics and Child Health at the Steve Biko Academic Unit, University of Pretoria. He was employed by Nestlé Health Science as the Global Clinical Development Lead for Rare Diseases, IEM, and Innovative Pharmaceuticals until his retirement at the end of March 2024. Post-retirement, he will continue his lifelong passion for medical education and supporting rare disease service developments in low- and middle-income countries.

He is currently the Chief Community Impact officer for A Rare Cause, a non-profit organization based in the UK that educates clinicians on rare disease management in more than 50 countries, with the list growing yearly. This creates, “hope for those with the least chance of being recognized,” in his own words. He also provides expert knowledge as a consultant for FYMCA Medical Ltd, a family-owned company, to patient organizations, foundations, regulators, and payors in his field of expertise.

His publication list includes more than 250 works, with the majority focusing on rare disease. He has numerous publications related to clinical trials, quality of life, clinical guidelines, and review papers, as well as several book chapters in the field of inborn errors of metabolism.

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Questions:

00:00: Introduction

00:41: Tell me about your current position, and your areas of past and current research?

03:36: What could we do better in HTA for the US and worldwide?

08:39: How would you respond to the claim that HEOR analysis has contributed to rising drug prices, reduced affordability, and increased disparities?

12:28: What major research question would you focus on if you had the resources to tackle it?

16:28: Why don’t health economists have more impact on drug development, on healthcare access?

21:06: What did you learn during the COVID Simulator, and how would you change future simulations?

Jagpreet Chhatwal

Director, Institute for Technology Assessment, Massachusetts General Hospital; and Associate Professor, Harvard University

Jagpreet Chhatwal, PhD is Director of the Institute for Technology Assessment at Massachusetts General Hospital, and an Associate Professor at Harvard Medical School. His research has shaped national and global health policies across diverse disease areas. He has provided expert advice to the White House, the World Health Organization, and CDC on hepatitis C elimination. Dr. Chhatwal has authored over 120 research studies, and his work has been featured in major media outlets such as CNN, Forbes, The New York Times, The Wall Street Journal, and National Public Radio. In addition to his academic pursuits, he is a meditation instructor and has been practicing meditation for over two decades.

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Questions:

00:00: Could you give us a brief overview of your career?

02:34: What is your “why” – the driving force behind what you do, and your involvement in the initiatives you've led or supported?

05:14: What have you learned about the science of innovation in your own work in terms of process, mistakes, identifying goals, etc.

06:33: Put AI into context with respect to transformative potential. Identify the critical areas of opportunity and ones that you’ve moved down the list. Why?

14:50: Can you identify the more advanced HTAs with respect to AI acceptance and usage, and what do researchers need to do better or differently to align with this intention?

22:10: Biases in AI are a significant concern. Describe 1 or 2 major concerns and explain how our field is addressing this bias?

24:28: Tell us more about the EHR framework for data quality report.

26:47: Tell me about the biggest lesson you’ve learned in your career and how did it change who you are as a researcher and a thought leader?

27:52: Rapid fire questions

Rachael Fleurence

Senior Advisor, National Institute of Biomedical Imaging and Bioengineering

Dr Fleurence is a Senior Advisor to Dr Francis Collins at the National Institutes of Health where her team is spearheading a national initiative to eliminate hepatitis C in the US. Dr Fleurence is also affiliated with the National Institute of Biomedical Imaging and Bioengineering (NIBIB) where she is focused on advances in Artificial Intelligence and Machine Learning. Dr Fleurence currently co-leads the ISPOR Task Force on the suitability of EHR data for Health Technology Assessments. Previously, Dr Fleurence served as a senior health policy advisor at the White House, where she worked on several scientific priorities for the Administration, including the development of a national elimination plan for hepatitis C and the development of the President’s Council of Advisors in Science and Technology (PCAST) recommendations to improve patient safety and reduce medical harm in the US. Prior to her role at the White House, Dr Fleurence served as Senior Advisor to the NIH Director at the NIH. As part of the federal response to COVID-19, Dr Fleurence led the novel, “Say Yes! Covid Test.” This program was the first of its kind in to provide free rapid antigen tests to communities across the country.

Dr Fleurence served on the White House Pandemic Testing Board policy group, and the White House Office of Science Technology Policy (OSTP) Pandemic Innovation Task Force in 2021–2022. In 2021 and 2022, Dr Fleurence was a recipient of three NIH Director’s Award for exceptional dedication and innovation in advancing COVID-19 testing and diagnostics efforts. In 2022, Dr Fleurence was a recipient of the Health and Human Services Secretary’s award for Distinguished Service. She was the recipient of the 2023 “National Champion for Global Hepatitis Elimination” along with Dr Francis Collins.

Prior to her role at the White House and NIH, Dr Fleurence served as the Executive Director of the National Evaluation System for health Technology Coordinating Center (NESTcc), and served as the Program Director for PCORnet at the US Patient-Centered Outcomes Research Institute (PCORI). Dr Fleurence received a BA from Cambridge University (UK), an MA in business management from ESSEC-Paris (France), and an MSc in health economics and PhD in health sciences, both from the University of York (UK).

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00:31: Could you give us a brief overview of your career?

04:40: Flatiron FORUM (FLATIRON FORUM (Fostering Oncology RWE Uses and Methods): What are some of the short- and long-term goals?

09:00: The use of real-world data from various countries has sparked concerns about external validity or transportability, leading to a preference for local data. How have you tackled this?

14:47: Why are SDoH and racial/ethnic inequities not a guiding factor in HEOR research?

20:31: What can you tell us about the recent ISPOR SUITABILITY checklist for EHRs for HTA use on data delineation and data fitness for purpose, and what do you think it will accomplish?

25:37: How are you utilizing LLMs to scale real-world data curation?

36:28: How is Flatiron engaging with CMS and other stakeholders on the Inflation Reduction Act?

38:01: Rapid fire questions

Blythe Adamson, PhD, MPH

Head of Outcomes Research at Flatiron Health & Founder of Infectious Economics

Dr Blythe Adamson is the head of international outcomes research and evidence generation at Flatiron Health.

As a visionary senior leader at Flatiron Health, her team pioneered deep learning language models for extraction of clinical details from EHR documents, breaking the limits of what was possible for humans to do alone. Learning from the experience of millions of patients with cancer, they generate evidence of treatment effectiveness and value used by governments around the world. Dr Adamson co-invented a patented clinical decision-support tool, enabled by machine learning, that is used by cancer clinics to benefit patients.

She holds degrees in microbiology, epidemiology, and pharmaceutical economics with a focus on infectious disease prevention. Dr Adamson has held roles at the Bill and Melinda Gates Foundation Institute for Disease Modeling, the NIAID HIV Vaccine Trials Network, and Flatiron Health.

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00:00: Tell us about the genesis of your involvement in Duchenne’s Muscular Dystrophy and how did you move into patient advocacy?

04:34: Was there a particular event or challenge that moved you from a parent to a more holistic patient advocacy role?

07:23: How did you envision a process to set up a clinical trial website focused on patients and clinical sites in the UK?

10:00: What is Project Hercules?

14:55: You had multiple pharma companies and a university along with your patient group involved in Project Hercules; who made financial contributions?

16:58: What is the current status and future focus for Project Hercules?

20:39: How did your group engage directly with NICE and NHS around Project Hercules?

22:09: I’ve heard you say that you struggle with NICE Rare Disease standards being too high. How are you focusing on this via Duchenne’s UK?

24:35: From a policy standpoint, you took on the disease stages of Duchenne’s and made them more granular and introduced a Transitional Stage; do you believe that all progressive rare diseases should receive this kind of review?

26:03: What learnings from Project Hercules can you offer to other patient advocacy groups?

29:21: What is the geographical focus of Project Hercules?

30:03: Have you noticed a change in NICE over the 13 years you’ve been working with them from a patient advocacy standpoint?

32:50: NICE is opening their process more to patients; have you sat on decision-making committees at the HTA level or what is your organization’s role at this level?

34:22: What are we missing about patient centricity and what do you wish we did better?

42:22: What makes a stakeholder want to work with you or a patient advocacy group?

48:07: In 13 years, Duchenne’s UK has grown in its breadth and impact. What mistakes, if any, have you made and what do you think about when you reflect back on the history of your organization?

50:24: What is your short-term goal for the next 18 months?

52:18: If we’re having this conversation in 10 years, what will be discussing?

53:10: Rapid fire questions

Emily Reuben OBE

CEO & Co-founder, Duchenne UK

Emily has a degree in modern history from Oxford University, and had a successful career as a reporter and anchor for Channel 4 News and CNN International.

After her son was diagnosed with Duchenne muscular dystrophy (DMD), she established the Duchenne Children’s Trust. The charity joined forces with Joining Jack and is now Duchenne UK. Duchenne UK has raised more than £27 million to accelerate the search for treatments for DMD, improve the standards of care for it in the UK, and develop technology to help people with DMD live more independently.

She sits on the steering group for the Association of the British Pharmaceutical Industry’s Patient Organisation Forum. She is a member of the Medicines and Healthcare products Regulatory Agency’s Patient Group Consultative Forum, as well as the Steering Group for the regulator’s Innovative Licensing and Access Pathway.

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00:38: Have patient aspects always been of interest in HTAs?

04:54: What is considered patient involvement in HTA?

06:23: Are there differences in how patient participation is presented in countries with HTAs? Can they be categorized based on the degree of development of this participation?

12:18: You mentioned the new guideline for patient involvement in the Joint Clinical Assessment. How will this impact patient involvement?

13:18: You explained that patient involvement includes both research into patient aspects and patient participation in HTA. What are the expected outcomes from each, and what are the specific differences?

15:58: Regarding research, what are the barriers and enablers to patient-based evidence?

20:56: What methodologies exist for measuring what matters to patients?

24:40: It seems there is a need to adapt HTA methodologies to better incorporate patient involvement and address emerging healthcare challenges. What changes in HTA and patient involvement do you foresee in the future?

30:10: What is necessary to continue improving patient involvement in HTA?

31:22: What is your call to action?

34:29: Rapid fire questions

Walter Toro

Executive Director, Global HEOR/RWE

Novartis Gene Therapies

Dr Walter Toro, an accomplished MD and PhD in Health Economics, is a seasoned executive trained across Colombia, Spain, and the USA. His expertise lies in Pharma/Biotech's HEOR and Market Access domains. Based in the Chicago Area, he serves as Executive Director, Global HEOR/RWE in Gene Therapies at Novartis. With over 20 years of experience spanning Pfizer, Bayer, Abbott/AbbVie, and Astellas, he excels in evidence generation, launching new drugs (including blockbusters), optimizing access, and shaping policies.

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00:15: You both have led and served on Boards. When is the time to form a Board, does the make-up of the Board differ, and what are some of the challenges once you have a Board?

11:07: What should a founder do with a “NO”? How can they turn that “NO“ into a better next pitch?

13:29: Let’s put the shoe on the other foot. From an investor standpoint, what pain points are you dealing with that you wish founders would consider so that the two parties can work in the most mutually agreeable and successful way?

17:20: What does due diligence look like, and what should founders be prepared for? Does this differ by geographic location and if so, how?

36:05: Founding a company, expanding reach, scaling for growth, are necessities that many founders/CEOs have little experience in, and often the investors are negotiating sticky situations. Let’s talk about some of these challenges.

49:00: Final thoughts

Speakers

Natalie Douglas

Founder and Healthcare Specialist Advisor

Lucidity LLC

Natalie is a +30-year life sciences and healthcare executive and multi-award winning, former global CEO with extensive Board level experience (Executive/NED/Strategic Advisory) in building and transforming mid-market PE backed businesses in pharmaceutical and healthcare services.

An entrepreneur CEO, she built a global market leading pharmaceutical services company securing debt financing and private equity investment. Following its acquisition, as CEO, Natalie led a significant turnaround of one of the largest private equity-backed clinical homecare and speciality pharmacy companies.

Natalie has held several NED positions in emerging life sciences and data analytics companies and today is Chair of two VC backed med tech companies. She is Founder of New Jersey based Lucidity LLC, an international health technology investment vehicle and provider of strategic advisory, operational expertise and due diligence services to PE/VC investors. She is highly curious about AI and data analytics, broadly interested in the application of technology and how it can be applied to improve healthcare service delivery, patient and care provider outcomes, health system efficiencies and establishing greater biopharma value demonstration.

She is also a mentor and coach to commercially inexperienced CEO’s and management teams.

Natalie splits her time between USA, UK and Switzerland.

M. Ragan Hart

Senior Associate, MultiCare Capital Partners

Founding Partner, Evergreen Value Advisory

M. Ragan Hart, MS, PhD is an investor for MultiCare Capital Partners (MCP). MCP is the innovation and corporate venture arm of MultiCare, a 12-hospital community-based regional health system, headquartered in Tacoma, Washington. As the conduit between MultiCare and a larger entrepreneurial ecosystem, MCP exists to fulfill MultiCare’s mission of partnering for healing and a healthy future.

Ragan is also a Founding Partner of Evergreen Value Advisory and previously co-founded MDisrupt, a commercial enablement platform for digital health and healthtech companies, and prior to that role co-led venture creation efforts for F-Prime Capital Partners focused on health data sciences. She earned her BS from Auburn University, her MS (genetic epidemiology) and PhD (public health genetics, concentration applied health economics) from the University of Washington, and conducted her postdoctoral fellowship at Stanford University (biomedical data science).

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00:00: Introduction

03:14: What is the big picture investment arena for RWE, HEOR, and Pricing/Access companies? What is catching the eye and the wallet of the market? And what trends are looming over the next 5 years?

09:30: What are the funding avenues available to founders and when should they be considering these options?

16:36: How do founders know “it’s time” to seek outside funding, and what research should a founder do before identifying investment channels?

34:09: After a founder has identified potential investors, how do you make the first connection so that it sets you up for future successful conversation?

45:42: What makes a good pitch, and who should be involved in developing the pitch? What are common features of a failed pitch or blind spots?

Speakers:

Natalie Douglas

Founder and Healthcare Specialist Advisor

Lucidity LLC

Natalie is a +30-year life sciences and healthcare executive and multi-award winning, former global CEO with extensive Board level experience (Executive/NED/Strategic Advisory) in building and transforming mid-market PE backed businesses in pharmaceutical and healthcare services.

An entrepreneur CEO, she built a global market leading pharmaceutical services company securing debt financing and private equity investment. Following its acquisition, as CEO, Natalie led a significant turnaround of one of the largest private equity-backed clinical homecare and speciality pharmacy companies.

Natalie has held several NED positions in emerging life sciences and data analytics companies and today is Chair of two VC backed med tech companies. She is Founder of New Jersey based Lucidity LLC, an international health technology investment vehicle and provider of strategic advisory, operational expertise and due diligence services to PE/VC investors. She is highly curious about AI and data analytics, broadly interested in the application of technology and how it can be applied to improve healthcare service delivery, patient and care provider outcomes, health system efficiencies and establishing greater biopharma value demonstration. She is also a mentor and coach to commercially inexperienced CEO’s and management teams. Natalie splits her time between USA, UK and Switzerland.

M. Ragan Hart

Senior Associate, MultiCare Capital Partners

Founding Partner, Evergreen Value Advisory

M. Ragan Hart, MS, PhD is an investor for MultiCare Capital Partners (MCP). MCP is the innovation and corporate venture arm of MultiCare, a 12-hospital community-based regional health system, headquartered in Tacoma, Washington. As the conduit between MultiCare and a larger entrepreneurial ecosystem, MCP exists to fulfill MultiCare’s mission of partnering for healing and a healthy future.

Ragan is also a Founding Partner of Evergreen Value Advisory and previously co-founded MDisrupt, a commercial enablement platform for digital health and healthtech companies, and prior to that role co-led venture creation efforts for F-Prime Capital Partners focused on health data sciences. She earned her BS from Auburn University, her MS (genetic epidemiology) and PhD (public health genetics, concentration applied health economics) from the University of Washington, and conducted her postdoctoral fellowship at Stanford University (biomedical data science).

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00:00: You’ve been advocating for the EU HTA legislation. What changes do you see occurring as a result of how manufacturers are pursuing HTA and price approval?

11:01: You mentioned that many people fear that there will be several base-case scenarios, the so-called PICOs. Please explain this and the implications.

16:04: What are the cross-border implications of the EU HTA legislation with the US IRA?

23:49: Are there any unintended consequences that you predict as a result of this legislation, from an overall launch perspective, pursuit of multiple indications, and for how portfolios are managed?

27:03: Does Medicare have a ‘value framework?’ How will that be actualized in a way that can be used by manufacturers in development? What does CMS need to share/publicly state, will they, and if not, what can we impute?

30:55: You’ve written on IEPs. What are the biggest barriers to implementation, and advancing this idea from the 'theoretical’ to a standard practice?

44:09: Where does health equity fit into HTA decision-making?

49:57: You now serve as a Strategic Advisor to ISPOR after serving in numerous positions over the organization’s history. Can you tell us about some of the critical issues in which you are engaging, and what are you hoping to achieve?

54:19: Rapid Fire Questions

Jens Grueger

Partner & Senior Expert for Pricing and Market Access in Healthcare, Boston Consulting Group (BCG)

Jens Grueger, PhD, is a Partner at the Boston Consulting Group (BCG), based in Zurich, and BCG's senior expert for pricing and market access in health care. He is a fellow of the BCG Henderson Institute and Affiliate Professor of Health Economics in the Comparative Health Outcomes, Economics and Policy (CHOICE) Institute at the University of Washington in Seattle, USA. He was the 2020–2021 President of ISPOR, the largest professional society for health economics and outcomes research. Before joining BCG in 2019, Jens led the Global Pricing, Market Access, Health Economics and Health Policy functions at Novartis (1999–2009), Pfizer (2009–2011) and Roche (2011–2019).

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00:00: Tell us about your career over the years, and what your focus is now.

04:46: You’ve written an interesting blog on LinkedIn summarizing the RWE Market and its segmentation, which garnered a lot of feedback. What was your reason for writing this, and what feedback did you get? Why does this matter, and to whom?

18:05: You’ve published a commentary in Value in Health entitled, “Replication of Randomized Controlled Trials Using Real-World Data: What Could Go Wrong?” In it, you call into question the appropriateness of using RCT findings as a standard by which RWE should be judged, especially by the FDA. What is the fundamental issue here? What in fact could go wrong?

27:01: You note that, “the train has left the station with RCT replication.” What are the unintended consequences in, say, 5 years if this is the position adopted by FDA and industry?

29:16: Another one of your pet projects is a survey of researcher pain points when using RWD sources to generate RWE. Can you tell us more about this?

33:42: Back at the 2018 ISPOR EU Congress, you organized a panel discussion ominously titled, “Will Machines Soon Make Health Economists Obsolete?” How do you see AI impacting HEOR and RWE now and in the future?

41:29: You now serve as a Strategic Advisor to ISPOR after serving in numerous positions over the organization's history. Can you tell us about some of the critical issues in which you are engaging, and what are you hoping to achieve?

47:01: Rapid Fire Questions

David Thompson

Founder & Principal Consultant, Rubidoux Research LLC

David Thompson, PhD is a health economist with 35 years experience consulting in the HEOR/RWE domain. He is Founder & Principal Consultant of Rubidoux Research LLC, a company dedicated to helping individuals and businesses achieve their commercial objectives in the ever-evolving HEOR/RWE marketplace. Prior to founding Rubidoux Research, he held leadership positions with various CROs and HEOR consultancies.

Dr Thompson is a long-time contributor to ISPOR, having served as Editor-in-Chief of Value & Outcomes Spotlight 2008–2020, a member of the Board of Directors 2020–2023, and currently a Special Board Advisor. He also has conducted more than 50 workshops, issue panels, symposia, and short courses at the various ISPOR conferences. Among various other affiliations, he sits on the advisory board and work streams of selected RWE policy groups, including Duke-Margolis RWE Collaborative.

He received his BS in Economics from the University of California, Riverside and his PhD in Economics from the University of Massachusetts at Amherst.

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Chief Strategy Officer, National Pharmaceutical Council (NPC)

Kimberly Westrich, MA, is the Chief Strategy Officer of the NPC, which sponsors and conducts research on health policy issues related to the development and use of innovative biopharmaceuticals to improve the health of patients. NPC’s research contributes to the body of evidence that supports discussions and decisions about patient access to treatments, appropriate use, and the value innovative treatments provide to both patients and the healthcare system.

Ms Westrich provides strategic guidance to NPC’s policy research and communications activities. She leads several research initiatives across NPC’s portfolio, including employer-sponsored insurance. She has published extensively on issues related to value assessment frameworks, patient-centered formulary and benefit design, value-based contracts, quality performance measurement, and accountable care organizations.

Ms Westrich began her NPC career in 2000. She has also served as Director of Research at the Pharmaceutical Research and Manufacturers of America (PhRMA), worked as a healthcare consultant at The Lewin Group and Xcenda, and as a health economics and outcomes researcher at Johnson & Johnson.

Ms Westrich is a certified yoga teacher and life coach with a passion for helping others learn and thrive. She received her master’s degree in economics from Northwestern University and her undergraduate degree in economics and mathematics from the College of William and Mary.

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Chief Executive, National Institute for Health and Care Excellence (NICE), UK

Sam Roberts is the Chief Executive of the National Institute for Health and Care Excellence. She has worked in healthcare for 20 years in a combination of clinical, operational, private sector and academic roles. She originally trained as a doctor before undertaking an MBA and joining McKinsey and Company. Her focus for the last 15 years has been on improving quality of clinical care and supporting uptake of innovation - as Head of Operations at a large teaching hospital in London (UCLH), Director of Innovation, Research and Life Sciences at NHS England and Improvement and Chief Executive of the Accelerated Access Collaborative. Prior to joining NICE she invested in early stage digital health and diagnostics companies. She has an academic interest in the influence of health economic analysis on policy and clinical practice, having worked at the London School of Economics followed by doctoral studies at the University of Oxford.

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Director, Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center; and Professor, Tufts University School of Medicine

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); and 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 panel of health advisors at 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.

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Former Pharma Executive and Independent Consultant for real-world data and real-world evidence

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 to 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.

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Adjunct Professor of Epidemiology at the University of North Carolina, and Chief Scientific Officer, IQVIA Real-World Solutions, Retired

Nancy Dreyer is Adjunct Professor of Epidemiology at the University of North Carolina at Chapel Hill, a Trustee of Brandeis University and Chief Scientific Officer, IQVIA Real World Solutions. Recently retired from full-time employment, she maintains an active career, including consulting, serving on advisory boards, teaching for ISPE and the ISPOR RWE leadership team, and publishing. She earned a Master of Public Health Degree and PhD in Epidemiology from UNC. She is a Fellow of both the International Society of Pharmacoepidemiology (ISPE) and the Drug Information Association. Her work has been used to advance global use of real-world evidence by medical product regulators, with her publications cited in recent guidelines in the US, Europe, and China.

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Director of Robert J. Margolis Center and Professor of Business, Medicine, and Policy, Duke-Margolis Center for Health Policy

Mark McClellan MD, PhD, served as the Commissioner of the US Food and Drug Administration (FDA) from 2002 to 2004, and the Administrator of Centers of Medicare and Medicaid from 2004 to 2006, where he developed and implemented major reforms in health policy, including the Medicare prescription drug benefit, and Medicare and Medicaid payment reforms. He has also previously served as a member of the President’s Council of Economic Advisers and Senior Director for Healthcare Policy at the White House, and as Deputy Assistant Secretary for Economic Policy at the Department of the Treasury. He is currently the Director of Robert J. Margolis Center and a Professor of Business, Medicine, and Policy at the Duke-Margolis Center for Health Policy.

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RWE Pioneer

As an entrepreneur, researcher, consultant, and innovator, Jeff Trotter has been a pioneer in the evolving health economics and outcomes research community and is an industry leader in the design and implementation of patient registries, observational studies, and other RWE (real-world evidence) initiatives. Jeff currently supports the scientific and strategic RWE initiatives of life-sciences and related organizations through his eponymous consultancy, J Trotter Research & Consulting.

In 1990, Jeff founded Ovation Research Group, an internationally known research and consulting firm (acquired by ICON Clinical Research) and has been in executive management with other research companies (ClinTrials Research, ICON, PharmaNet/i3, inVentiv Health (now Syneos)). Most recently, Jeff served as Senior Vice President – Worldwide Evidence for Worldwide Clinical Trials, a leading mid-sized contract research organization, after Worldwide acquired the Late Stage (RWE) research business unit of Continuum Clinical, a professional services company including additional capabilities in Patient Recruitment and Medical Communications, that Jeff co-founded in 2014.

Jeff has spoken widely in front of varied industry audiences and has published over 75 original articles and research papers — including a book for the American Hospital Association, The Quest for Cost-Effectiveness in Healthcare: Achieving Clinical Excellence While Controlling Cost. Jeff is a founding member of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), and holds a Master of Management (MBA) degree from Northwestern University’s Kellogg School of Management and a Bachelor’s degree in Accounting from the University of Illinois.

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CEO

OM1, Inc.

Dr Richard Gliklich is the CEO of OM1, a data and technology company focused on real-world data and artificial intelligence (AI) to support medical research, outcomes measurement and personalized medicine. Previously, he was founder and CEO of Outcome, which he led from inception through its acquisition by Quintiles (IQVIA) and an XIR with General Catalyst Partners. Dr Gliklich is well known in the areas of registries, outcomes and analytics. He is senior editor of the landmark publication by the U.S. Agency for Healthcare Research and Quality (AHRQ) handbook ‘Registries for Evaluating Patient Outcomes: A User’s Guide’ and the PI for the Outcomes Measures Framework, which focuses on standardization of outcomes measurement. He is a frequent speaker and author on using high-quality real-world data and advanced analytics for drug development, peri-approval monitoring, value-based care and clinical decision support. Dr Gliklich also holds several patents for health outcomes systems, machine learning and predictive analytics. Dr Gliklich is a graduate of Yale University and Harvard Medical School, and a former Charles A Dana Scholar at the University of Pennsylvania. He is also a surgeon and the Leffenfeld Professor at Harvard Medical School. 

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