COMMITTEE MEMBERS

Jeff (Jeffrey) Kahn, Ph.D., M.P.H. (Committee Chair), is the Andreas C. Dracopoulos Director of the Johns Hopkins Berman Institute of Bioethics, a position he assumed in July 2016. Since 2011, he has been the inaugural Robert Henry Levi and Ryda Hecht Levi Professor of Bioethics and Public Policy at the Berman Institute. He is also Professor in the Department of Health Policy and Management of the Johns Hopkins Bloomberg School of Public Health. He is an internationally recognized expert in bioethics, exploring the intersection of ethics and health/science policy, including human and animal research ethics, public health, and ethical issues in emerging biomedical technologies. Dr. Kahn has served on numerous governmental and international advisory panels, including most recently on the International Commission on the Clinical Use of Heritable Human Genome Editing. He is currently chair of the National Academies of Sciences, Engineering, and Medicine (National Academies) Committee on Aerospace Medicine and Medicine of Extreme Environments and has previously chaired the National Academies Committee on the Use of Chimpanzees in Biomedical and Behavioral Research (2011); the Committee on Ethics Principles and Guidelines for Health Standards for Long Duration and Exploration Spaceflights (2014); and the Committee on the Ethical, Social, and Policy Considerations of Mitochondrial Replacement Techniques (2016). Dr. Kahn has served as chair of the National Academy of Medicine Board on Health Sciences Policy and as a member of the National Academy of Medicine Council. He received his B.A. (microbiology) from the University of California, Los Angeles, his M.P.H. from Johns Hopkins University School of Hygiene and Public Health, and his Ph.D. (philosophy/bioethics) from Georgetown University.

Ron (Ronald) Bartek, M.Sc., is co-founder and president of Friedreich’s Ataxia Research Alliance (FARA). Formerly, he was director and chair of the National Organization for Rare Disorders. He also served as past president and current member of the board of directors of the Alliance for a Stronger FDA. Mr. Bartek co-founded the NCATS Alliance, now serving as chair of its board of directors. He was formerly on the board of directors of the Alliance for Regenerative Medicine. He was a member of the NIH/NCATS National Advisory Council and the NIH Neurological Institute National Advisory Council, having previously served as chair of the NCATS Cures Acceleration Network Review Board. Mr. Bartek has been recognized by FDA Office of Orphan Drug Development as one of “30 Heroes changing lives of rare disease patients.” He is a former member of the FDA/CTTI Patient Engagement Collaborative. Mr. Bartek has 20 years of federal executive and legislative experience in defense, foreign policy, and intelligence. Mr. Bartek received his B.S. from U.S. Military Academy, West Point, and his M.A. from Georgetown University.

Terry Jo Bichell, Ph.D., M.P.H., is the CEO and founder of COMBINEDBrain as well as a lecturer at Vanderbilt University. She worked primarily as a public health nurse-midwife until her youngest child, Lou, was diagnosed with Angelman syndrome in 2000. She quickly switched focus to move bench research into the first clinical trials for Angelman syndrome and to help design natural history studies. Dr. Bichell was the founding director of the A-BOM Alliance from 2016–2018. In 2019, she launched COMBINEDBrain, a pre-competitive consortium of patient advocacy organizations that works with clinicians, researchers, and pharmaceutical firms to identify outcome measures and biomarkers for rare genetic neurodevelopmental disorders. She is the vice chair of the Tennessee Rare Disease Advisory Council and teaches a course in translational neuroscience at Vanderbilt University. She previously served on the Angelman Patient Advisory Council to Hoffman La Roche, which has now been disbanded. As a parent of a child living with a rare disease, Dr. Bichell gained first-hand experience in the search for a treatment for Angelman syndrome as she accompanies her son as he participates in clinical trials. Dr. Bichell received her B.S.N. from St. Louis University, her M.P.H. from Boston University, and her Ph.D. in neuroscience from Vanderbilt University where she studied gene-environment interactions in Huntington’s disease rodent models.

Edward Botchwey, Ph.D., M.E., is a professor in the Wallace H. Coulter Department of Biomedical Engineering at Georgia Tech and Emory University. His research focuses on elucidating lipid signaling mechanisms in sickle cell disease and developing novel immunomodulatory therapies to resolve inflammation and treat organ dysfunction. Dr. Botchwey has garnered over $14 million in research funding and has published extensively on bioactive lipids and resolution pharmacology for sickle cell therapies. He conducts research in affiliation with the Marcus Center for Therapeutic Cell Characterization and Manufacturing. He has also spearheaded efforts to increase diversity and inclusion in biomedical engineering, including leading the Diversity, Equity, and Inclusion Committee for the Society for Biomaterials. Dr. Botchwey received his B.S. from the University of Maryland and his MEng and Ph.D. from the University of Pennsylvania. He completed a postdoctoral fellowship in vascular biology at The Wistar Institute.

Shein-Chung Chow, Ph.D., is a professor at the Department of Biostatistics and Bioinformatics at Duke University School of Medicine. Dr. Chow is also an adjunct professor at Peking University and Beijing Capital Medical University. Previously, he was a special government employee, appointed by FDA as a voting member of the Oncologic Drug Advisory Committee and a statistical advisor to FDA. Dr. Chow previously served as associate director of the Center for Drug Evaluation and Research’s Office of Biostatistics at FDA. Dr. Chow is currently serving on several Data Safety Monitoring Boards for clinical studies sponsored by Genetech and Merck via third party clinical research organizations, Syneos, Parexel, and Statistics Collaborative. Dr. Chow was the editor-in-chief of the Journal of Biopharmaceutical Statistics (1992–2020) and is the editor-in-chief of the Biostatistics Book Series at Chapman and Hall/CRC Press of Taylor & Francis Group. He was elected Fellow of the American Statistical Association in 1995 and elected member of the ISI (International Statistical Institute) in 1999. Dr. Chow is the author or co-author of over 200 papers and 20 books on trial design considerations and statistical methods for rare disease research. He more recently authored the book Innovative Methods for Rare Disease Drug Development. Dr. Chow received his B.S. from National Taiwan University and his Ph.D. from University of Wisconsin–Madison.

Hans-Georg Eichler, M.D., M.Sc., is the consulting physician at the Association of Austrian Social Insurances. Prior to this role, Dr. Eichler was the senior medical officer of the European Medicines Agency (EMA) from 2007–2021. Earlier in his career, Dr. Eichler was a professor of clinical pharmacology and head of the Department of Clinical Pharmacology, as well as vice-rector for research and international relations at the Medical University of Vienna. During his time in academia, he gained ample experience in clinical research, including being the primary investigator of numerous academic and industry-sponsored drug trials. In his subsequent positions, he was closely involved in the development of pharmaceutical policies from the governmental, regulatory, and public payer perspectives. Dr. Eichler serve as the vice-chair of the Scientific Advisory Committee at the Centre for Innovation in Regulatory Sciences and has done pro bono work for EURORDIS-Rare Diseases Europe, a nonprofit alliance of over 1,000 rare disease patient organizations across Europe. Dr. Eichler has received several honors and awards from different European universities and learned societies. He received his M.Sc. from the University of Surrey and his M.D. from the University of Vienna.

Pat (Patricia) Furlong, R.N., is president and CEO of Parent Project Muscular Dystrophy, which focuses on Duchenne and Becker muscular dystrophy. She led the development of Draft Guidance on Duchenne, which was submitted to FDA in 2016 and updated in 2022. She serves as a member for the Duchenne Community Advisory Board in Europe. Ms. Furlong served on the National Academies of Sciences, Engineering, and Medicine Committee on Safe and Effective Medicines for Children. She is a nurse practitioner by training, spending her early career in whole organ transplantation and renal dialysis. Since 1994, Ms. Furlong has focused on rare disease research—specifically on genetic testing, standards of care, therapy development, and regulatory processes. She has led preference studies to understand the patient’s perspective of benefit and risk. Ms. Furlong has worked with rare disease groups as they prepare for interactions with regulatory agencies in the United States and Europe. Ms. Furlong is a member of the World Duchenne Organization’s board, data safety monitoring boards for the Rare Disease Research Network and Cooperative International Neuromuscular Research Group, and New York University’s Pediatric Gene Therapy Medical Ethics Group. Ms. Furlong serves on the Clinical Trials Transformation Initiative executive committee and is a board member with the National Health Council. She received her R.N./B.S.N. degree from Mount St. Joseph University.

Steven Galson M.D., M.P.H., is a senior advisor to Boston Consulting Group and serves on the board of directors of Biocryst Pharmaceuticals and Elephas Biosciences. He is a consultant for Skyline Therapeutics. Until June 2020, Dr. Galson was a senior vice president of research and development at Amgen Inc. He spent more than 20 years in government service, including 2 years as acting surgeon general of the United States. He served as director of FDA’s Center for Drug Evaluation and Research, where he provided leadership for the center’s national and international programs in pharmaceutical regulation. Dr. Galson began his public health service career as an epidemiological investigator at the U.S. Centers For Disease Control and Prevention. He was also the chief medical officer at both the Environmental Protection Agency and the U.S. Department of Energy. Dr. Galson is a trustee of the Keck Graduate Institute and a member of the Executive Committee of the Clinical Trial Transformation Initiative. In 2008, he received an honorary Doctor of Public Service degree from Drexel University School of Public Health, and in 2015, he received the Jacobi Medallion Award from Icahn Mount Sinai School of Medicine. In 2018 he was named health leader of the year from the Commissioned Officers Association of the U.S. Public Health Service. Dr. Galson has been a member of the Forum on Drug Discovery Development and Translation of the National Academies of Sciences, Engineering, and Medicine for 20 years. He has also served two terms as the forum’s co-chair. Dr. Galson received his B.S. from Stony Brook University, his M.P.H. from Harvard University, and his M.D. from Icahn Mount Sinai School of Medicine.

Gavin Huntley-Fenner, Ph.D., is the co-founder and principal human factors consultant at Huntley-Fenner Advisors, which provides scientific advisory services. Specializing in creative and scientifically based approaches to assessing risks and benefits, Dr. Huntley-Fenner brings more than 25 years of both academic and business experience to bear on the crafting of innovative and effective communication to consumers. Dr. Huntley-Fenner also serves as an expert legal consultant, public speaker, and facilitator of risk analysis teams, where he is noted for his ability to effectively articulate solutions to complex problems. He has served on the U.S. FDA Risk Communication Advisory Committee (2004–2009) and on the National Academies of Sciences Engineering and Medicine (National Academies) Mutual Recognition Agreements in the Regulation of Medicines Study Committee (2019–2020). He is currently a member of the National Academies Environmental Health Matters Initiative Steering Committee and is also a member of Mattel’s Medical and Scientific Advisory Council. Dr. Huntley-Fenner received his Ph.D. in brain and cognitive sciences from Massachusetts Institute of Technology in 1995 and his B.A. in cognitive sciences from Vassar College in 1990.

Anaeze Offodile II, M.D., M.P.H., is an executive vice president and the chief strategy officer of Memorial Sloan Kettering Cancer Center (MSK). Dr. Offodile is a member of the Forum on Drug Discovery Development and Translation of the National Academies of Sciences, Engineering, and Medicine (National Academies). He is a double board-certified physician with clinical expertise in oncologic reconstruction, a health services researcher with a focus on alternative payment models and care redesign, and a healthcare administrator with management experience in academia. He leads strategy efforts and care transformation initiatives at MSK by continuing to develop the core infrastructure, management systems, and processes for enterprise strategy and business development. Dr. Offodile pilots new initiatives, facilitates alignment on strategic institutional priorities, leverages data sources to cultivate innovative digital analytics and products, develops collaborations with outside groups, and partners with key internal leaders to competitively position MSK for the future. He received his B.S. from Kent State University, his M.P.H. from Johns Hopkins Bloomberg School of Public Health, and his M.D. from Columbia University.

Anne Pariser, M.D., is a physician, currently working with the Indian Health Service at the Crow/Northern Cheyenne Hospital in Crow Agency, Montana. Prior to this, she was the VP, medical and regulatory affairs at Alltrna, a biotech company developing tRNAs as therapeutic agents for rare genetic diseases (through March 2024), where she continues to provide part-time consulting services. Previously, she was the director of the Office of Rare Diseases Research at the NIH National Center for Advancing Translational Sciences from 2017–2022. From 2000–2017, she worked at the FDA CDER Office of New Drugs (OND), where she led the first specialized rare diseases review team for Inborn Errors of Metabolism. In 2010, Dr. Pariser founded the Rare Diseases Program (RDP) within OND/CDER, a congressional mandate intended to accelerate and improve rare diseases drug review within FDA that focused on the development of policy, guidance, training, and coordination of regulatory science to benefit rare disease programs. Dr. Pariser’s research has focused on rare diseases regulatory and translational sciences, and she is the author of approximately 50 papers on these topics. Dr. Pariser has received numerous awards from FDA, NIH, HHS and other stakeholders for her service to the rare disease community. This includes the National Organization for Rare Disorders’ National Public Health Leadership Award and being named, along with the OND/CDER’s Rare Diseases Program, as one of the 30 Rare Disease Heroes on the 30th anniversary of the Orphan Drug Act. She is the chair of the Regulatory Scientific Committee at the International Rare Disease Research Consortium, a collaborative initiative uniting national and international nonprofits, industry, patient advocacy organizations, and scientists to promote collaboration for rare disease research. She is also a volunteer member of the board of directors for the Undiagnosed Diseases Network Foundation—a nonprofit that fosters collaboration among patients, clinicians, and scientists to bridge diagnosis, research, and clinical care for undiagnosed patients with rare and ultra-rare diseases. Dr. Pariser received her B.S. from Bates College and her M.D. from Georgetown University School of Medicine. She is board certified in internal medicine.

Jonathan Watanabe, PharmD., Ph.D., is associate dean of assessment and quality at the UC Irvine School of Pharmacy & Pharmaceutical Sciences and director of the UCI Center for Data-Driven Drugs Research and Policy. Dr. Watanabe is a member of the Forum on Drug Discovery Development and Translation of the National Academies of Sciences, Engineering, and Medicine (National Academies). Dr. Watanabe is an appointed member of the non-partisan California Health Benefits Review Program Faculty Task Force of the University of California funded by the State of California Legislature. He has been involved in research and policy efforts salient to rare diseases, including service on the National Academies’ Committee on Making Medicines Affordable and participation in the National Academies’ workshop series on Examining the Impact of Real-World Evidence on Medical Product Development. The latter also entailed publication of guidance manuscripts on conducting real-world trials in special populations. Additionally, he has received grant approval for a pending industry-funded project that will use publicly available data to examine global definitions of what deems drug as a ‘biosimilar.’ He has published original research examining the increase in high-spend medications covered by Medicare Part D that serve small populations in the United States. He was the National Academy of Medicine (NAM) Anniversary Fellow in Pharmacy from 2016 to 2018. From 2018 to 2021, he served as a Scholar in the NAM Emerging Leaders in Health and Medicine Program. Dr. Watanabe was the inaugural recipient of the University of Washington/Allergan Global Health Economics and Outcomes Research Fellowship (2007 to 2009). He received his B.S. from the University of Washington, his Pharm.D. from the University of Southern California, and his M.S. and Ph.D. from the University of Washington Comparative Health Outcomes, Policy, and Economics Institute. He is a board-certified geriatrics pharmacist.

NATIONAL ACADEMY OF MEDICINE FELLOW

Sanket Dhruva, M.D., M.H.S., is an assistant professor of medicine at the University of California, San Francisco (UCSF) and a cardiologist at the San Francisco Veterans Affairs Medical Center. His research, clinical, and education interests focus on understanding and strengthening the evidence base for the safe and effective use of drugs and medical devices in diverse populations, with the goal of improving the quality of care and clinical outcomes for patients. He identifies solutions to improve equity in the development and dissemination of these therapies. Dr. Dhruva currently serves on the Medicare Evidence Development & Coverage Advisory Committee and Institute for Clinical and Economic Review California Technology Assessment Forum. He has authored more than 185 peer-reviewed publications and has been funded by the Greenwall Foundation, Department of Veterans Affairs, NIH, Food and Drug Administration, National Evaluation System for Health Technology, National Institute for Health Care Management, and Arnold Ventures. Dr. Dhruva received his BA in political science and molecular and cell biology from the University of California, Berkeley. He graduated with an MD from UCSF. He completed residency in internal medicine at UCSF and fellowship in cardiovascular medicine at the University of California, Davis. He subsequently completed an MHS at Yale University

STUDY STAFF AND CONSULTANT

Carolyn K. Shore, Ph.D., is a senior program officer with the Board on Health Sciences Policy of the National Academies of Sciences, Engineering, and Medicine. She is co-director of the National Academies study on Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union and staff director of the Forum on Drug Discovery, Development, and Translation. Before joining the National Academies, Dr. Shore was an officer on Pew’s antibiotic resistance project, leading work on research and policies to spur the discovery and development of urgently needed antibacterial therapies. She previously served as a foreign affairs officer at the U.S. Department of State, where she led an initiative on open data and innovation-based solutions to global challenges. She also served as the State Department’s representative to intergovernmental organizations focusing on food safety, plant and animal health, biosecurity, and agricultural trade policy. Previously, Dr. Shore was an American Society for Microbiology congressional fellow, working on science-based policy related to antibiotic stewardship and other public health issues. She holds a doctoral degree in microbiology and molecular genetics from Harvard University. As a graduate student, she studied antimalarial drug resistance in Senegal and worked jointly between the Medicines for Malaria Venture, Genzyme Corporation, and the Broad Institute of Harvard and MIT to discover new anti-malarial compounds. Dr. Shore was awarded a Fulbright Fellowship for work at the University of Queensland in Brisbane, Australia, and a National Institutes of Health Training Grant for postdoctoral work at the University of Iowa.

Tequam Worku, M.P.H. (Study Co-Director), is a program officer for the Board on Health Sciences Policy at the National Academies of Sciences, Engineering, and Medicine. Most recently, she worked on a study Examining the Working Definition for Long COVID and serves as the staff lead for an Action Collaborative on Engaging Community Practices in Clinical Trials. Her previous work with the National Academies includes directing a study on Improving the CDC Quarantine Station Network’s Response to Emerging Threats with the Board on Global Health. Prior to that, she worked at the Association of State and Territorial Health Officials as a senior analyst for Clinical to Community Connections, managing federally funded projects on community health workers and ending the HIV epidemic. Her past experience also includes working on projects related to chronic diseases and the development of healthy communities, including the promotion of healthy aging and hypertension prevention and control (the Million Hearts Initiative). Ms. Worku has worked on various research projects on topics including breast cancer disparities and cultural competency in health care. Additionally, she has worked internationally supporting knowledge management and data analysis efforts at the national level. She is committed to efforts aimed at bridging disparities in health and has been actively involved in health-equity initiatives. She earned her B.A. in biology from University of Maryland Baltimore County, an M.P.H. from The George Washington University, and is currently a Dr.P.H. candidate at Morgan State University.

Carson Smith, M.S., is a research associate with the Board on Health Sciences Policy of the National Academies of Sciences, Engineering, and Medicine. Prior to joining the National Academies in August 2023, he was a research assistant for the Human Factors and Aging Laboratory at the University of Illinois at Urbana-Champaign. In this role, he supported research into usefulness, ease of use, and adoption of assistive technology among older adults. He also previously worked in research at the National Center for Human Factors in Healthcare within the MedStar Institute for Innovation. Mr. Smith received his B.S. in interdisciplinary health sciences and his M.Sc. in health technology from the University of Illinois at Urbana-Champaign.

Noah Ontjes, M.A., is an associate program officer with the Board on Health Sciences Policy of the National Academies of Sciences, Engineering, and Medicine. He currently staffs the Forum on Drug Discovery, Development, and Translation, co-leading projects on engaging community practices in clinical trials and preparing the future workforce in drug research and development. He attended Wake Forest University where he graduated with a B.S. in biology and a triple minor in bioethics, chemistry, and psychology. His interest in the multiple factors that influence one’s health paired with his love of different perspectives led him to pursue an M.A. in Bioethics at Wake Forest University. During graduate school, he successfully defended his thesis on the reasonable person standard of disclosure in genetic research as well as collaborated on a published paper concerning the ethical considerations of electroconvulsive therapy on incapacitated patients. Overall, he likes to categorize himself as someone who is intellectually curious.

Kyle Cavagnini, Ph.D., is an associate program officer with the Board on Health Sciences Policy. They currently staff the Forum on Drug Discovery, Development, and Translation, where their portfolio includes pre-clinical research and clinical trial diversity. Dr. Cavagnini previously worked with the National Academies Institute for Laboratory Animal Research, where they supported the Standing Committee for the Care and Use of Animals in Research, and workshop committees engaged in the One Health field. Prior to joining the National Academies, Dr. Cavagnini completed a science policy fellowship with the Federation of American Societies for Experimental Biology and was a Fulbright Fellow in the Department of Biomedicine at the University of Bergen, Norway. They earned their Ph.D. in biological chemistry from the Johns Hopkins University School of Medicine, where their doctoral research focused on genomic contributions to metabolic sensing in the liver and other tissues. They received undergraduate degrees in biochemistry and philosophy from the University of North Carolina at Asheville.

Melvin Joppy is a senior program assistant on the Board on Health Sciences Policy of the National Academies of Sciences, Engineering, and Medicine. He previously served as a program assistant at the Department of Energy (DOE) in the Office of Basic Energy Sciences. Prior to DOE, Mr. Joppy served as the committee manager for the Presidential Advisory Council on HIV/AIDS within the U.S. Department of Health and Human Services. Mr. Joppy received his B.S. in communications from Bowie State University.

Clare Stroud, Ph.D., is senior board director for the Board on Health Sciences Policy at the National Academies of Sciences, Engineering, and Medicine. In this capacity, she oversees a program of activities aimed at fostering the basic biomedical and clinical research enterprises; addressing the ethical, legal, and social contexts of scientific and technologic advances related to health; and strengthening the preparedness, resilience, and sustainability of communities. Previously, she served as director of the National Academies’ Forum on Neuroscience and Nervous System Disorders, which brings together leaders from government, academia, industry, and nonprofit organizations to discuss key challenges and emerging issues in neuroscience research, development of therapies for nervous system disorders, and related ethical and societal issues. She also led consensus studies and contributed to projects on topics such as pain management, medications for opioid use disorder, traumatic brain injury, preventing cognitive decline and dementia, supporting persons living with dementia and their caregivers, the health and well-being of young adults, and disaster preparedness and response. Dr. Stroud first joined the National Academies as a Mirzayan Science and Technology Policy Graduate Fellow. She has also been an associate at AmericaSpeaks, a nonprofit organization that engaged citizens in decision making on important public policy issues. Dr. Stroud received her PhD from the University of Maryland, College Park, with research focused on the cognitive neuroscience of language, and her bachelor’s degree from Queen’s University in Canada.

Erin Hammers Forstag, J.D., M.P.H., supported the study as the science writer. She has been writing for the National Academies of Sciences, Engineering, and Medicine and other organizations for over 10 years, covering topics including COVID-19, mitochondrial replacement therapy, DNA forensics, and health professionals education. She obtained her JD from Georgetown University Law Center in 2012 and her Master’s in Public Health from Columbia University in 2006.