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, Chair
Dr. Caplan is the Drs. William F. and Virginia Connolly Mitty Professor and founding head of the Division of Medical Ethics. He co-chairs the Compassionate Use Advisory Committees (CompAC) and is the author or editor of 32 books and more than 750 papers in peer-reviewed journals. His most recent books are Getting to Good: Research Integrity in the Biomedical Sciences, with Barbara Redman, and Vaccination Ethics and Policy, with Jason Schwartz. During the coronavirus (COVID-19) pandemic, he is co-directing a new advisory group on sports and recreation for the U.S. Conference of Mayors, creating a working group on vaccine challenge studies, developing an ethical framework for distributing drugs and vaccines for Johnson & Johnson, and setting policy with the Western Institutional Review Board for research studies. Learn more about Dr. Caplan.
Alison Bateman-House, PhD, MPH, MA
Dr. Bateman-House is an assistant professor in the Division of Medical Ethics. She co-chairs the interdisciplinary Working Group on Pediatric Gene Therapy and Medical Ethics and serves as the chair of the Compassionate Use Advisory Committees for infectious diseases and neurology/psychology. She has published and spoken extensively on access to investigational drugs and the history and ethics of using humans as research subjects. Learn more about Dr. Bateman-House.
M. Sage Gustafson, MA
Ms. Gustafson is the CUPA project manager and a research associate in the Division of Medical Ethics. She graduated from Duke University with a master of arts in bioethics and science policy, where she focused her studies on the ethical responsibilities of science communicators and the intersection of gender and racial discrimination in health policy. Before her graduate work, she was a flute performer and music educator in Oregon.
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 is an assistant professor in the Division of Biostatistics at NYU Langone. She conducts research in the design, implementation, and statistical analysis of clinical trials with a focus on adaptive, pragmatic studies. Prior to this appointment, she was a postdoctoral fellow in the Division of Biostatistics. She earned a PhD in biomedical engineering and an MS in applied mathematics at Northwestern University. Learn more about Dr. Belli.
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.
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 a member of the Working Group on Pediatric Gene Therapy and Medical Ethics and 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, PhD, MSW, MBE
Dr. Kimberly is an assistant research scientist in the Hansjörg Wyss Department of Plastic Surgery and an associate of the Division of Medical Ethics. Her research examines ethical and psychosocial implications of innovative medical interventions with a focus on social justice and health equity. She worked previously in health policy administration, research, and education. Dr. Kimberly received master’s degrees in social work and bioethics from the University of Pennsylvania, and holds a PhD in social work with a concentration in social policy and administration from Columbia University.
Mr. Klein, an internationally recognized expert in preapproval access to therapeutic agents, is the director of Expanded Access Programs and Policy for the GE2P2 Global Foundation. He 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.
Aisha Langford, PhD, MPH
Dr. Langford is an assistant professor in the Department of Population Health at NYU Langone and co-directs the Recruitment and Retention Core within NYU Langone Health’s Clinical and Translational Science Institute. She explores how health communication can improve individual decision-making and reduce population health disparities for conditions or behaviors that lead to preventable mortality and morbidity. Dr. Langford’s current research focuses on cardiovascular disease and cancer prevention and control. Learn more about Dr. Langford.
Holly Fernandez Lynch, JD, MBE
Ms. Lynch is the John Russell Dickson, MD, Presidential Assistant Professor of Medical Ethics and Health Policy at the University of Pennsylvania, where she also did her training. Her scholarship focuses on the law and ethics of healthcare gatekeeping, with an emphasis on research with human subjects, preapproval access to investigational drugs, and conflicts of conscience in medicine. She was previously a regulatory attorney in private practice and a bioethicist serving at the National Institutes of Health (NIH).
Andrew McFadyen, BA, BEd
Mr. McFadyen is the executive director for the Isaac Foundation, a nonprofit organization dedicated to helping families battling rare diseases. He is a member of the Division of Medical Ethics Working Group on Pediatric Gene Therapy and Medical Ethics. He is also an associate fellow of the GE2P2 Global Foundation and a member of its Independent Bioethics Advisory Committee, which provides consultative services to biopharma organizations on clinical trials, expanded access programs, and in other areas.
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.
Jennifer Miller, PhD
Dr. Miller is an assistant professor at Yale School of Medicine, founder of Bioethics International and the Good Pharma Scorecard, and a member of the World Economic Forum. She has advised pharmacy and therapeutics committees; the Centers for Disease Control and Prevention, American Medical Association, United Nations, and National Institutes of Health; 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. Her training is in physics, bioethics, regulatory governance, and institutional corruption from Fordham University, Regina Apostolorum Pontifical University, 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); Chimerix (antivirals and biodefense); Alteon (diabetes and cardiovascular); Biocyte (cord blood stem cell transplantation); and The Liposome Company (anti-cancer and anti-fungal). He has served as chair of the Biotechnology Innovation Organization’s bioethics committee and co-chair of its Emerging Companies Section Strategy and Policy Committee, which is focused on issues surrounding the development of new medicines. He received an AB in biochemistry with a minor in health policy from Princeton University and an MBA from the Stanford Graduate School of Business.
Regine Nshimiyimana Maniraho, DNP, PharmB, FNP
Dr. Nshimiyimana Maniraho is a former pharmacist currently practicing as an oncology nurse practitioner and an adjunct faculty at Thomas Jefferson University and Jefferson Health. She has experience in taking care of patients diagnosed with cancer who require inpatient treatment. She received a PharmB from the National University of Rwanda, an MSN from Thomas Jefferson University, a post-MSN from the University of Pennsylvania, and a DNP from the George Washington University.
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 Robertson, JD, PhD
Dr. Robertson is the N. Neal Pike Scholar and Professor of Law at Boston University. He is the author of Exposed: Why Our Health Insurance Is Incomplete and What Can Be Done About It (HUP 2019) and co-editor of three books on medical devices, healthcare decisions, and bias. He has taught bioethics, health law, and torts at University of Arizona, NYU Law, Harvard Law, and Washington University in St. Louis and served as a reporter for the Health Law Monitoring Committee of the Uniform Law Commission.
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.