Division of Medical Ethics Working Group on Compassionate Use & Preapproval Access Members
Members of the Working Group on Compassionate Use and Preapproval Access (CUPA), who come from within NYU Langone and from outside entities, represent a wide variety of fields, including medicine, law, industry, and patient advocacy. This enables us to draw on the perspectives of all stakeholders involved in this preapproval access.
Arthur L. Caplan, PhD
Dr. Caplan is the Drs. William F. and Virginia Connolly Mitty Professor and founding head of the Division of Medical Ethics at NYU Langone. He is the chair of the Division of Medical Ethics Compassionate Use Advisory Committees and the author or editor of 32 books and more than 700 papers in peer-reviewed journals. His most recent books are Getting to Good: Research Integrity in the Biomedical Sciences, with Barbara Redman (Springer, 2018), and Vaccination Ethics and Policy, with Jason Schwartz (MIT Press, 2017). Learn more about Dr. Caplan.
Alison Bateman-House, PhD, MPH
Dr. Bateman-House is an assistant professor in the Division of Medical Ethics. She serves as the chair of the NYU/Janssen Pharmaceuticals Compassionate Use Advisory Committees for Infectious Diseases and Neurology/Psychology. She has published and spoken extensively about requests for non-trial access to investigational drugs. She has also written on the history and ethics of using humans as research subjects and on clinical trial accessibility. Learn more about Dr. Bateman-House.
Jinsy A. Andrews, MD, MSc
Dr. Andrews is an assistant professor of neurology in Columbia University’s Division of Neuromuscular Medicine and the director of neuromuscular clinical trials in the Department of Neurology. She has extensive experience in conducting human clinical trials in neuromuscular disorders in academic and industry settings, as well as participating in expanded access programs. She received a BS from Union College, an MSc in biostatistics from Columbia University Mailman School of Public Health, and an MD from Albany Medical College.
Hayley M. Belli, PhD, MS
Dr. Belli has been a postdoctoral fellow in in NYU Langone’s Division of Biostatistics since December 2017. She conducts research in the design, implementation, and statistical analysis of clinical trials with a focus on adaptive, pragmatic studies. Her work is supported through NYU Langone’s Clinical and Translational Science Institute TL1 Training Program. She earned a PhD in biomedical engineering and an MS in applied mathematics at Northwestern University.
David R. Curry, MS
Mr. Curry is a co-founder and president of the GE2P2 Global Foundation, a U.S.-based nongovernmental organization with the mission to advance ethical and scientific rigor in research and evidence generation. The foundation’s Center for Access to Medicines focuses on bioethical issues across the clinical development life cycle including compassionate use/prelicensure/expanded access, and access to essential medicines in low-resource settings and humanitarian contexts globally. He is an associate faculty member of the Division of Medical Ethics.
Nancy N. Dubler, LLB
Ms. Dubler is a consultant for ethics for the New York City Health and Hospitals Corporation, an adjunct professor of the Division of Medical Ethics, a professor emerita of bioethics at the Albert Einstein College of Medicine, and the founding director of the Montefiore Medical Center Division of Bioethics and the Montefiore/Einstein Certificate Program in Bioethics and Medical Humanities. Learn more about Ms. Dubler.
Kelly McBride Folkers, MA
Ms. Folkers is a senior research associate in the Division of Medical Ethics and an adjunct lecturer at the City University of New York. Her work focuses on a variety of topics related to preapproval access to investigational medical products, including the role of institutional review boards (IRBs) in expanded access, clinical trial equity, medical crowdfunding, innovative treatment for transgender youth, use of real-world evidence in regulatory decision-making, and federal expanded access policy.
Pat Furlong, MS, RN, BSN
Ms. Furlong is the founding president and CEO of Parent Project Muscular Dystrophy (PPMD) and one of the foremost authorities on Duchenne muscular dystrophy in the world. In 1994, she founded PPMD with other parents of young men with Duchenne muscular dystrophy to change the course of the disease and ultimately find a cure. Together they accelerate research, raise their voices in Washington, demand optimal care for all young men, and educate the global community.
Ms. Hirawat is the owner and executive chair of VOZ Advisors and a venture partner at Nest Bio. Until 2015, she was president of PTC Therapeutics, Inc. She served as chair of the International Circle of Ambassadors for EURORDIS (the European Organisation for Rare Diseases) until 2018 and continues to serve as an active volunteer at Rare Diseases International and the NGO Committee for Rare Diseases. She has served on the advisory board of the National Hemophilia Foundation and the board of directors of Parent Project Muscular Dystrophy.
Kay Holcombe, MS
Ms. Holcombe serves as senior adviser to the Milken Institute, focusing on the Lynda and Stewart Resnick Center for Public Health. She was senior vice president for science policy at BIO (Biotechnology Innovation Organization) and has extensive experience in government health policy and the nonprofit sector. She received an MS in chemistry, graduating with honors, from the University of Virginia and was elected to Phi Beta Kappa, Phi Kappa Phi, and Iota Sigma Pi.
Lisa Kearns, MS, MA
Ms. Kearns, senior research associate in the Division of Medical Ethics, has spoken and published extensively on state and federal right to try legislation. She is the deputy chair of the Compassionate Use Advisory Committees Oncology and Neurology/Psychology panels. She has an MA in philosophy from the University of North Carolina at Chapel Hill and an MS in bioethics from Columbia University.
Laura Kimberly, MSW, MBE
Ms. Kimberly is an assistant research scientist in the Hansjörg Wyss Department of Plastic Surgery and an associate of the Division of Medical Ethics. She is also a doctoral candidate at the Columbia School of Social Work. Laura studies the ethical and psychosocial implications of innovative medical interventions including novel forms of organ and tissue transplantation. She received master’s degrees in social work and bioethics from the University of Pennsylvania.
Mr. Klein, an internationally recognized expert in preapproval access to therapeutic agents, worked at the FDA for more than 40 years before leaving the agency in 2017. While there he worked closely with patient communities in a variety of areas including treatment access to unapproved drugs, product safety, and clinical trial design. Prior to working in patient engagement, he helped develop policies and regulations for the protection of human research subjects and provided guidance for IRBs.
Andrew McFadyen, BA, BEd
Mr. McFadyen is the executive director for the Isaac Foundation, a nonprofit organization he founded to fund research on MPS (mucopolysaccharidosis), a rare and progressive disease affecting his eldest son. He has led numerous advocacy efforts to shape public policy throughout Canada with respect to availability of treatments for children dying from rare diseases, and he fights for fair and equitable access to treatments for children affected by rare diseases throughout Canada and the United States.
Lindsay McNair, MD, MPH, MS
Dr. McNair is the chief medical officer of the WIRB-Copernicus Group (WCG). Before joining WCG, she worked with multiple biopharma companies, providing medical guidance on clinical development strategies, study designs for new drug studies, and medical oversight of all phases of clinical trials. She currently oversees the medical functions of WCG and provides advice to biopharma companies and institutions on IRB oversight and the ethical conduct of research. She also received an MS in bioethics.
Jennifer Miller, PhD
Dr. Miller, assistant professor at Yale School of Medicine, is the founder of Bioethics International and the Good Pharma Scorecard. She taught at NYU Grossman School of Medicine and Duke University and is a member of the World Economic Forum. She has advised pharmacy and therapeutics committees, the Centers for Disease Control and Prevention, the American Medical Association, United Nations, the National Institutes of Health (NIH), and the Patient-Centered Outcomes Research Institute (PCORI)–NIH Collaboratory. Her work explores the ethics of drug development, access to medicines, and big data in healthcare. She trained at Fordham University, Pontifical University System, Duke University, and Harvard University.
Kenneth I. Moch, MBA
Mr. Moch has been CEO or co-founder of five companies that pioneered novel therapies for life-threatening diseases: Cognition Therapeutics (Alzheimer's disease), where he currently serves as president and CEO; Chimerix (antiviral); Alteon (diabetes and cardiovascular); Biocyte (cord blood stem cell transplantation); and the Liposome Company (anti-cancer and anti-fungal). He chairs the Biotechnology Innovation Organization’s bioethics committee and co-chairs its ECS Policy Subcommittee focused on the development of new medicines for highly prevalent chronic diseases.
Barbara Redman, PhD, MBE, RN
Dr. Redman is an associate of the Division of Medical Ethics. Her work focuses on research ethics and, to a lesser extent, chronic disease ethics. In CUPA, she has focused on the role of IRBs in approving preapproval access, publishing (with Dr. Bateman-House) “Institutional Review Boards as Arbiters of Expanded Access to Unapproved Drugs: Time for a Change?” in Therapeutic Innovation & Regulatory Science. Her most recent book is Getting to Good: Research Integrity in the Biomedical Sciences, with Dr. Caplan (Springer, 2018).
Christopher T. Robertson, JD, PhD
Dr. Robertson is associate dean for research and innovation and founder of the Regulatory Science Program at the University of Arizona. He is the author of Exposed: Why Our Health Insurance Is Incomplete and What Can Be Done About It (HUP 2019), co-editor of two other books, and author of more than 50 journal articles. A philosopher and legal scholar, he has taught bioethics, health law, and torts at NYU School of Law, Harvard Law School, and Washington University in St. Louis.
David I. Scheer, MS
Mr. Scheer is president of Scheer & Company, Inc., a venture capital, corporate strategy, and transactional advisory services firm focused on the life sciences. He has been involved in the founding and/or been a board member of many life sciences and biotechnology companies. He received an AB, cum laude, in biochemical sciences from Harvard College and an MS in cell, molecular, and developmental biology from Yale University.
Lesha D. Shah, MD
Dr. Shah is assistant professor of psychiatry at the Icahn School of Medicine at Mount Sinai and medical director of Child, Adolescent and Family Services. She studies issues of consent and capacity as they interface with family complexity, mental illness, and innovative medicine including gene therapies and research studies. With clinical expertise in pediatric psychiatric care in the hospital setting, she is interested in physician perspectives on medical decision-making for children and medical education.
J. Russell Teagarden, DMH, MA
Dr. Teagarden has served in clinical and executive roles in clinical pharmacy practice, clinical research, university teaching, pharmacy benefit management, and nonprofit patient advocacy. He prepared for these roles through formal education and training in pharmacy, research methods, bioethics, and medical humanities.
David Wallach, MPH, CIP
Mr. Wallach joined NYU Langone in May 2016 as director of Research Regulatory Services. He oversees the human research quality assurance/improvement program, clinicaltrials.gov registration and reporting, and regulatory services, providing support for faculty-held investigational new drug applications and investigational device exemptions. For more than 14 years, he worked at the Albert Einstein College of Medicine IRB, culminating in his role as the IRB director, where he oversaw IRB operations for Einstein, Montefiore Medical Center, NYC Health + Hospitals/Jacobi, NYC Health + Hospitals/North Central Bronx, and Yeshiva University.
Tom Watson, BSc
Mr. Watson is the executive vice president for Early Access Programs at Bionical. He has partnered with pharma and biotech companies to design strategies for preapproval access and implement global programs, allowing patients access to treatments that would otherwise be unavailable. Previous roles include executive director for TW Consulting Group and head of U.S. business development for Clinigen Group. He has developed thinking and strategy for many of the top 20 pharma companies in this area.
Mary Elizabeth Williams
Ms. Williams is a journalist and an author. In 2011, facing metastatic melanoma, she became one of the first people in the world in a groundbreaking immunotherapy clinical trial. She chronicled her experiences in her 2016 science memoir, A Series of Catastrophes & Miracles, and now speaks to and consults for numerous health organizations across the country. She is currently enrolled in the narrative medicine program at Columbia University. Learn more about Ms. Williams.