As methods of real-world evidence (RWE) research have rapidly developed and RWE is increasingly considered by regulators and health technology assessment (HTA) bodies, a wealth of recommendations on how to assess RWE studies have been published. Yet there remains some uncertainty on how decision-makers review the scientific validity of RWE submissions and inconsistencies in their review remain. This event will bring together regulators, HTA bodies and other stakeholders, to review the state of RWE submissions for labeling and coverage decision-making and how they are reviewed for their scientific merit.
For in-person attendees: The roundtable will be held in the Pechet Family Conference Room, Joseph B. Martin Conference Center, Harvard Medical School, 77 Avenue Louis Pasteur, Boston, MA 02115. This room is located on the first floor (see map). Public parking is available nearby. Breakfast will be served from 8:00-9:00AM ET, with lunch served after the event beginning at 1:30PM ET.
For online attendees: The roundtable will be hosted via Zoom. Webinar details will be shared with attendees one week before the event.
In-person and online attendees are encouraged to submit questions during the event using Slido. To submit your questions during each session, please join the Q&A at slido.com, meeting code #6966426.
Please contact Shruti Belitkar or Matt Martin with registration questions or if you require any accommodations to participate fully in the event.
All times listed below are in Eastern Time (ET).
Sebastian Schneeweiss, Professor of Medicine and Epidemiology, Harvard Medical School; Chief, Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women's Hospital
Aaron Kesselheim, Professor of Medicine, Harvard Medical School; Director, Program On Regulation, Therapeutics, And Law (PORTAL), Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women's Hospital
Presenters:
Discussants:
Moderator:
Presenter:
Discussants:
Moderator:
Presenter:
Discussants:
Moderator:
Presenter:
Discussants:
Moderator:
Dr. Folusa Agboola serves as the Senior Vice President of Research at ICER, where she provides methodological guidance on reviews, manages the broader health technology assessment process, and oversees the evidence synthesis team. Her expertise is in the application of principles of evidence synthesis in healthcare and public health decision-making. Before joining ICER in 2016, Dr. Agboola worked at the Mass General Brigham Center for Drug Policy. There, she developed economic evaluations to accompany rigorous reviews of clinical evidence on new therapies, assessed the potential cost and budgetary impact of a wide range of clinical interventions, performed clinical forecasting, and developed pharmacoeconomic models. Prior to that role, she was an Academic Fellow at the Harvard T.H. Chan School of Public Health. She earned her medical degree from the University of Ilorin, Nigeria, and her Master of Public Health in Quantitative Methods from Harvard University.
Anne d'Andon is an Independent Consultant, having most recently served as the Medical Director at CEMKA, providing medical and methodological support. She contributes to the development of the consulting and study activity (market access, pharmacoepidemiology, public health evaluation, medical data summaries) at the consulting company, and her mission is to explore the needs in terms of studies on emerging health fields.
Trained in cardiovascular pharmacology, human genetics and molecular biology, innovation has always attracted d'Andon. After working on the genetics of arterial hypertension at INSERM, she contributed to the organization of clinical research at AP-HP and then led the scientific directions of patient associations. Since 2008, she was responsible for the evaluation of medicines at the High Authority for Health (HAS) adding to her assets, national and international expertise in technological evaluation, in the city or in the hospital, as well as in the whole life cycle of the drug.
Dr. Rimma Berenstein is the deputy head of the pharmaceuticals department at G-BA. She holds a doctorate in medical biotechnology and has comprehensive experience in the HTA assessment of medicinal products. Since 2017 she has been working as a scientific advisor and team coordinator in the G-BA's Pharmaceuticals Department focusing on medicinal products for oncological and haematological indications. Since 2021, she has been coordinating the area of routine care data collection at G-BA. She is a member of the JCA subgroup and the HTA coordination group.
Dr. Marie Bradley is a Senior Advisor on the Real-World Evidence Analytics team in the Office of Medical Policy, Center for Drug Evaluation and Research (CDER), FDA. Her responsibilities related to real-world evidence (RWE) include serving as program lead for the FDA Advancing Real World Evidence Program and lead for a portfolio of externally conducted RWE demonstration projects, evaluating real-world evidence protocols, participating in internal Agency processes, interacting with external stakeholders, and contributing to guidance development.
She is a pharmacoepidemiologist and a pharmacist with over 15 years of experience working in regulatory, government, and academic sectors in US and the UK, including 11 years at the FDA. Dr. Bradley has a PhD in Pharmacoepidemiology and a Masters in Pharmacy degree from Queen’s University Belfast as well as a Masters in Public Health degree from London School of Hygiene and Tropical Medicine.
Prof. Karl Broich is Head and President of the BfArM (Federal Institute for Drugs and Medical Devices, Germany) and Honorary Professor for Psychiatry and Clinical Neuropsycopharmacology at the University of Bonn. From 1985 to 2000, Dr. Broich performed clinical and research work at hospitals of the universities of Bonn, Halle/Saale and Philadelphia (PennU) (Board certifications in Neurology, Psychiatry, Behavioral Psychotherapy).
He served as head of the Section Neurology/Psychiatry from 2000 to 2005 and became the department head from 2005to 2009. Since 2009, Dr. Broich has been the deputy head (Vice-President) at the BfArM. He also served as an alternate member of the Committee for Medicinal Products for Human Use (CHMP) from 2005 to 2009 and serves as chair of the CNS-Working Party at the European Medicines Agency (EMA) since 2013. Current research activities include clinical trials methodology CNS, biomarkers in drug development, Alzheimer’s disease and other neurodegenerative disorders. He holds memberships in several learned societies of the CNS field. Dr. Broich has authored and served as a co-author for more than 140 publications (peer reviewed articles, reviews, and book sections).
Dr. Kaysiaryna Bykov is a pharmacoepidemiologist and an Assistant Professor of Medicine at the Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women’s Hospital and Harvard Medical School. She has been working with electronic healthcare data to evaluate drug utilization, impact of health care interventions, and medication safety and effectiveness for more than a decade. Her current research focuses on the development and application of epidemiologic methods for evaluating the safety and effectiveness of complex drug exposures, including extended treatments, treatment combinations, and drug-drug interactions. Dr. Bykov also serves as an Associate Editor of Pharmacoepidemiology and Drug Safety and is a Fellow of International Society for Pharmacoepidemiology (ISPE).
Gianmario Candore has over 20 years of experience in data analytics and observational research across both regulatory authorities and industry. At the European Medicines Agency, he contributed to key Real-World Evidence initiatives, including the HMA-EMA Steering Group recommendations, the creation of DARWIN EU®, and served as co-chair of ENCePP®.
Since 2022, he has been with Bayer, leading evidence generation from multiple data sources and advancing innovative research methods with international experts. He is actively involved in EFPIA and IHI projects, represents industry at ENCePP®, and regularly contributes to professional and educational initiatives.
Dr. Steven Farmer is a board-certified cardiologist and is a Senior Partner of ABIG Health. He previously served as Chief Strategy Officer for Coverage at the Centers for Medicare & Medicaid Services (CMS). In that role, he led an effort to streamline and accelerate the development of evidence-based coverage policies. He was the principal architect of the Transitional Coverage for Emerging Technologies (TCET) pathway and led the initiative to incorporate real-world evidence into coverage decision-making. Before his role as CSO, he served as a Senior Advisor in the Center for Medicare and Medicaid Innovation, where he assisted in developing and refining value-based payment programs, with a particular emphasis on the Bundled Payments for Care Improvement Advanced model.
Prof. Wim Goettsch is a Special Advisor in Health Technology Assessment (HTA) at the Dutch National Health Care Institute (ZIN). In his position at ZIN, he is a Member of the EU HTAR Coordination Group and its subgroup on Joint Scientific Consultation (JSC).
Between 2010 and 2013, he was the Deputy Secretary of the Medicinal Products Reimbursement Committee at the Dutch National Health Care Institute. He was the Director of the EUnetHTA JA3 (2016-2020) Directorate and Chair of the Executive Board of EUnetHTA between June 2016 and March 2018. Since December 2022 he also has a position as a Professor and Chair on HTA of pharmaceuticals at Utrecht University (NL), where he has been leading an H2020 consortium with fifteen partners around Europe called HTx, new methods for Health Technology Assessment (2019-2024). Since 2024, he is also the coordinator of a new Horizon Europe Project called SUSTAIN-HTA, an EU-wide initiative to build a supporting infrastructure to ensure ongoing implementation of the latest and fit-for-purpose HTA methodologies and tools in practice. He has more than 150 publications in peer-reviewed international journals.
Niklas Hedberg is the Chief Pharmacist at the Dental and Pharmaceutical Benefits Agency (TLV) in Sweden. Niklas is the HTAR Coordination Group co-chair for medicinal products. He was the Chair of the Executive Board for EUnetHTA21 (2021-2023) and for EUnetHTA JA3 (2018- 2021). Niklas has been working with HTA and pricing and reimbursement since 2001.
Ashley Jaksa is the Principal, Research Partnerships at Target RWE, where she leads the strategic growth of research collaborations. Her role centers on developing and executing high-impact research partnerships with pharmaceutical, biotechnology, and other stakeholders to redefine how retrospective and prospective data are used to advance disease research and treatments.
Prior to joining Target RWE, Ashley held roles at Aetion, CVS Health, and Context Matters, whereshe contributed to the evolution of evidence generation strategies across the health care ecosystem. Her research focuses on improving the use of real-world evidence(RWE) in regulatory, payer, and health technology assessment decision-making. She has presented her work at leading conferences including ISPOR, HTAi, and AMCP, and her research has been published in a range of peer-reviewed journals. Ashley holds a Bachelor’s degree from the University of Michigan and a Masterof Public Health from Yale University.
Ashley is an active contributor to the RWE community, serving on ISPOR’s Real-World Evidence and RWE4Decisions Steering Committees, SustainHTA’s Advisory Council, AMCP’s RWE Research Initiative, and the Editorial Board of the Journal of Comparative Effectiveness Research. She is also a former Chair of ISPOR’s Real-World Evidence Subject Matter Interest Group.
Páll Jónsson is Programme Director for Data and Evidence at UK’s National Institute for Health and Care Excellence (NICE). He has a strategic role in ensuring NICE is at the forefront of harnessing new and emerging opportunities to inform NICE’s guidance for the health and care sectors. His remit includes data access and analysis, real-world evidence methods, implementation of AI in HTA and guidelines, and evidence services. Páll holds a doctorate in biochemistry and bioinformatics from the University College London. Prior to his current role, he served as Associate Director for Science Policy and Research, overseeing a portfolio of international research projects in areas such as big data and real-world evidence.
Dr. Kesselheim is Professor of Medicine at Harvard Medical School and a faculty member in the Division of Pharmacoepidemiology and Pharmacoeconomics in the Department of Medicine at Brigham and Women’s Hospital. His research focuses on the effects of intellectual property laws and regulatory policies on pharmaceutical development, the drug approval process, and the costs, availability, and use of prescription drugs both domestically and in resource-poor settings. He has also investigated how other issues at the intersection of law and public health can affect the healthcare system, including health care fraud, expert testimony in malpractice cases, and insurance reimbursement practices. He is a member of the New York State Bar and is a Patent Attorney.
Dr. Li is a Principal Investigator and Associate Professor at Tsinghua University. Dr. Li’s team utilizes pharmacoepidemiology methodologies, digital health technologies and multi-omics techniques to critically assess emerging issues and tools on the development, evaluation, regulation and use of therapeutics during clinical development and in real-world settings.
Claire Martin is Global Policy Lead, Real-World Evidence at Bayer AG. She is responsible for policy topics related to real-world data and evidence and its role in healthcare decision making, as well as stakeholder engagement, and establishing demonstration projects and collaborative initiatives aimed at advancing the field of real-world evidence. Prior to her current role, Claire was Head of Global Market Access Stakeholder Engagement, Cardio-Renal, at Bayer AG and was responsible for leading the development of strategic stakeholder engagement initiatives to enable patient access.
Throughout her career, Claire has worked in healthcare policy, market access, advocacy and communications for the pharmaceutical industry, consultancies and non-governmental organisations in Europe, the UK and Japan. She has led and contributed to various multi-stakeholder initiatives on topics ranging from assessing and using patient preferences in the development and regulation of medicines to advancing public policy in areas such as global health, cardiovascular disease, and women’s health.
Dr. Mackenzie Mills is an Associate Director of the Medical Technology Research Group and CEO of HTA-Hive. He has worked in the field of health economics for over 10 years, with a focus on market access, health technology assessment, real world evidence, sustainable financing, and drug pricing and has numerous publications on these topics. Mackenzie’s previous work involves global consulting and research activities for a range of public and private organizations including the European Commission, the World Health Organization, the OECD, the European Federation of Pharmaceutical Industries and Associations, and a number of global biopharmaceutical companies.
He has participated in speaking engagements at several conferences across Europe, the Middle East and LATAM and delivering executive training courses including the LSE Market Access Academy and the WHO workshop on strategic procurement with a focus on negotiations. Mackenzie holds a PhD in Pharmaceutical Policy (LSE,UK), MSc in International Health Policy (LSE, UK), and BSc in Biochemistry (University of British Columbia, Canada).
Dr. Rebecca Nebel is a Senior Director of Science and Regulatory Advocacy at PhRMA and leads advocacy efforts to advance FDA regulatory policy on key issues including digital health, regulatory information and technology, and combination products. Prior to joining PhRMA, Rebecca was the Director of Scientific Programs at the Society for Women’s Health Research and a project manager at the National Institutes of Health. She was also a Christine Mirzayan Science & Technology Policy Graduate Fellow at the National Academies of Sciences, Engineering, and Medicine. Rebecca received her PhD in biomedical sciences from Albert Einstein College of Medicine and her BS in biological sciences from Binghamton University.
Dr. Susana Perez-Gutthann has over 3 decades of experience in the research-driven international pharmaceutical environment developing and leading teams of epidemiologists and biostatisticians and driving strategy and research programs for safety, pharmacovigilance, risk management, development, real-world evidence, and regulatory activities applying public health and epidemiologic methods. She is an active leader and past president of the International Society of Pharmacoepidemiology (ISPE) and past vice chair of the Steering Committee of the European Medicines Agency European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP).
Dr. Perez-Gutthann’s roles as an advisor and in international working groups have included ENCePP research guidance, International Scientific Advisory Board to the Medicines and Healthcare Products Regulatory Agency General Practice Research Database, ISPE Working Groups on Risk Management Guidances (Food and Drug Administration, European Medicines Agency), Good Pharmacoepidemiology Practice, and confidentiality of patient databases in Europe. Currently, Dr. Perez-Gutthann serves as a member and chair of several scientific advisory boards for studies, research programs, strategic and regulatory risk management, and real-world evidence topics. Her prior positions include global head of epidemiology at Pharmacia, Novartis, and Ciba-Geigy; European Head of Epidemiology at Pfizer; and postdoctoral fellow in the Division of Clinical Epidemiology at Johns Hopkins.
Dr. Laura Pizzi is the Chief Science Officer for ISPOR—The Professional Society for Health Economics and Outcomes Research. 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.
Dr. Pizzi brings more than 25years 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.
Donna Rivera, PharmD, MSc, FISPE, is as the Vice President of Life Sciences at Datavant. In this role, she guides life sciences organizations in advancing evidence generation strategies that span the entire medical product development continuum, including supporting innovative trial approaches and expanding data enrichment strategies through the use of real-world data (RWD), pragmatic designs, and external control arms with Datavant's partners.
Dr. Rivera brings more than a decade of experience working at the intersection of pharmacoepidemiology, oncology RWD, innovative trial design, and regulatory science. Most recently, she served as Associate Director for Pharmacoepidemiology at the Food and Drug Administration Oncology Center of Excellence, where she founded and led the Oncology Real-World Evidence Program.
Earlier in her career at the National Cancer Institute (NCI), she led an initiative to link large-scale, longitudinal treatment data with the Surveillance, Epidemiology, and End Results (SEER)program through collaborative public-private partnerships. She has published more than 90peer-reviewed papers, presented at national and international conferences, and is a fellow of the International Society for Pharmacoepidemiology. She earned her Doctor of Pharmacy and Master of Science in Pharmaceutical Sciences with a concentration in Pharmaceutical Outcomes and Policy from the University of Florida College of Pharmacy, and completed a postdoctoral fellowship in pharmacoepidemiology and pharmacogenomics at the NCI.
Dr. Sebastian Schneeweiss is Professor of Medicine and Epidemiology at Harvard Medical School and Vice Chief of the Division of Pharmacoepidemiology and Pharmacoeconomics of the Department of Medicine, Brigham and Women’s Hospital, a world-leading research and training center.
His research is funded by multiple NIH, PCORI, and FDA grants and focuses on the comparative effectiveness and safety of biopharmaceuticals. He has developed analytic methods to improve the scientific validity of epidemiologic analyses using complex longitudinal healthcare databases particularly for newly marketed medical products. Applying such methods transparently and in rapid cycles for sequential medication effectiveness monitoring is the overarching theme of his research. His work is published in >300 articles, many of them in high-ranking journals. He is Aetion Inc.’s Science Lead, where he develops a worldwide network of rapid-cycle analytics platforms that produces evidence fit for decision making in healthcare.
Dr. Schneeweiss is Director of the Harvard-Brigham Drug Safety Research Center funded by FDA/CDER and Co-Chair of the Methods Core of the FDA Sentinel program. He is voting consultant to the FDA Drug Safety and Risk Management Advisory Committee and was inaugural member of the Methods Committee of the Patient Centered Outcomes Research Institute. He was President of the International Society for Pharmacoepidemiology, inaugural member of the PCORI Methods Committee and is Fellow of the American College of Epidemiology, the American College of Clinical Pharmacology, and the International Society for Pharmacoepidemiology. At Harvard he teaches courses on Database Analytics for Pharmacoepidemiology and on Effectiveness Research in Longitudinal Healthcare Databases among others. He received his medical training at the Ludwig-Maximilians University of Munich and his doctoral degree in pharmacoepidemiology from Harvard.
Dr. Mark Sculpher is Professor of Health Economics and Head of Department for the Centre for Health Economics at the University of York. He is also Co-Director of the Policy Research Unit in Economic Evaluation of Health and Care Interventions (EEPRU). He has been based at the University of York since 1997. Between 1988 and 1997, he worked at the Health Economics Research Group at Brunel University; during 1998 he was a visitor in the Department of Clinical Epidemiology and Biostatistics at McMaster University in Canada.
Dr. Sculpher has worked extensively in the field of economic evaluation in health, with experience in areas including heart disease and various cancer. He has also contributed to methods in the field, in particular relating to decision analytic modelling and handling uncertainty.
Dr. Álmath Spooner is a qualified pharmacist and barrister-at-law with a PhD from Trinity College Dublin. Over the course of two decades, Álmath acquired a mix of public and private sector experience. Prior to joining industry, Dr. Spooner held leadership roles at the Irish Health Authority (HPRA), and at European Medicines Agency (EMA) as the first Vice Chair of the EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) where she served for the maximum of two terms (2012-2018).
Álmath was called to the Bar of Ireland in 2018 and practiced as a Barrister prior to joining AbbVie's Global Regulatory Policy and Intelligence team in 2020. Álmath leads the European region for Regulatory Policy at AbbVie and is the global policy topic lead for Real World Evidence and Patient Focused Drug Development. Álmath chairs EFPIA’s expert working on Integrated Evidence Generation and Use and co-chairs a subgroup on Patient Engagement. She is also a member of EFPIA’s Innovation Board Sponsored Committee and Regulatory Strategy Committee. Álmath has participated in various international initiatives including ICH, ICMRA, ISPE and CIOMS and has represented the EU in ICH EWGs.
Dr. Yoshiaki Uyama is currently Associate Executive Director, Pharmaceuticals & Medical Devices Agency (PMDA) of Japan. He is responsible for regulatory science research and data science in the agency. He has many experiences in new drug evaluation and real world data utilization, including the ICH E15, E16 and E17 Expert Working Groups, and Pharmacoepidemiology Discussion Group of ICH. He is also served as a member of the DIA Council of Regulators and a board member of the International Society for Pharmacoepidemiology as well as a Visiting Professor in Chiba University (Graduate School of Medicine), Nagoya University (Graduate School of Medicine) and Nagoya City University (Graduate School of Pharmaceutical Science).
This roundtable is organized by the Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women’s Hospital and Harvard Medical School. The event is supported by Bayer AG, who also contributed its organization. For questions about the event, please contact Shruti Belitkar or Matt Martin.
