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 of Bioethics and founding head of the Division of Medical Ethics. He co-chairs the Johnson & Johnson Compassionate Use Advisory Committees (CompAC) and is the author and 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 COVID-19 pandemic, he serves as a member of the World Health Organization/Monitored Emergency Use of Unregistered and Experimental Interventions (WHO MEURI) Compassionate Use Advisory Committee; created a working group on vaccine challenge studies, developing an ethical framework for distributing drugs and vaccines for Johnson & Johnson; and helped set policy with WCG IRB for research studies involving new drugs and vaccines for COVID-19. Learn more about Dr. Caplan, and contact him at firstname.lastname@example.org.
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, and contact her at email@example.com.
Sharad Adekar, MD, PhD, CIP
Dr. Adekar is a lead medical chair at WCG IRB in Puyallup, Washington. He is a physician scientist with clinical experience in family practice and pediatrics and research experience in immunology, oncology, infectious diseases, and neurology. He received his medical degree (MBBS, MD) from B.J. Medical College in Pune, India, and worked on his PhD thesis at Thomas Jefferson University. He received his PhD from the University of Pune. Dr. Adekar has extensive experience in human antibodies in terms of discovery, lead optimization, and preclinical development of monoclonal antibody therapeutics. He has published various peer-reviewed articles and participated in several conference presentations that included panel discussions, invited presentations, and peer-reviewed oral presentations.
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. Contact her at firstname.lastname@example.org.
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, and contact her at email@example.com.
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. Contact her at firstname.lastname@example.org.
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. Contact her at email@example.com.
Ms. Hirawat is the owner and executive chair of VOZ Advisors. Until 2015, she was president of PTC Therapeutics, Inc. She volunteers with EURORDIS—Rare Diseases Europe in international activities promoting the inclusion of the needs of patients with rare diseases in the United Nations Sustainable Development Goals 2030. She also serves as adjunct faculty in NYU Grossman School of Medicine’s Division of Medical Ethics. Contact her at firstname.lastname@example.org.
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. Contact her at email@example.com.
Sukhun Kang, MS
Mr. Kang is a PhD candidate in strategy and entrepreneurship at London Business School. His research focuses on strategy and innovation with a particular emphasis on healthcare, biopharmaceutical, and the high-tech industry. One of his projects examines expanded access from a strategic perspective. Prior to his doctoral program, he worked as a semiconductor engineer and led an internet startup. He holds a BS in computer engineering from the University of Illinois and master's degrees in both computer engineering and entrepreneurship and innovation from University of Southern California.
Lauren Karel, PharmD, BCPS
Dr. Karel is a drug information pharmacist at Children's Hospital of Philadelphia, where she participates in formulary management. She earned her BS in biology from University of Delaware and PharmD degree from Thomas Jefferson University. Following drug information residency training at Thomas Jefferson University Hospital, she subsequently served in roles within the investigational drug service. Contact her at firstname.lastname@example.org.
Lisa Kearns, MS, MA
Ms. Kearns is a senior research associate in the Division of Medical Ethics. She has spoken and published extensively on the ethical issues surrounding preapproval and expanded access to investigational drugs, including state and federal right to try legislation. More recently, she has focused on the ethics of individualized (n-of-1) gene therapies. She is a member of CUPA’s Bioethics of Individualized Therapeutics Subgroup and the division’s Working Group on Pediatric Gene Therapy and Medical Ethics. She is the deputy chair of the Compassionate Use Advisory Committees, a participant in the NYU–University of Ghana Research Integrity Training Program, and the associate director of the division’s High School Bioethics Project. Contact her at email@example.com.
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. Contact her at firstname.lastname@example.org.
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. Contact him at email@example.com.
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, and contact her at firstname.lastname@example.org.
Holly Fernandez Lynch, JD, MBE
Ms. Lynch is the John Russell Dickson, MD, Presidential Assistant Professor of Medical Ethics and assistant professor of law (secondary) 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, institutional review board and FDA policy and practice, and conflicts of conscience in medicine. She was previously a pharmaceuticals regulatory attorney in private practice and a bioethicist serving at the National Institutes of Health, as well as a member of the Department of Health and Human Services Secretary’s Advisory Committee on Human Research Protections. She currently serves on the boards of Public Responsibility in Medicine and Research and the American Society for Law, Medicine, and Ethics. Contact her at email@example.com.
Sandy Macrae, MBChB, PhD
Dr. Macrae has served as Sangamo's president and CEO and as a member of the board of directors since June 2016. He has 20 years of experience in the pharmaceutical industry, most recently serving as the global medical officer of Takeda Pharmaceuticals, from 2012 to 2016, where he established and led the Global Medical Office, which encompasses medical affairs, regulatory affairs, pharmacovigilance, outcomes research and epidemiology, quantitative sciences, and knowledge and informatics. Dr. Macrae received his BS in pharmacology and his MBChB with honors from Glasgow University. Dr. Macrae also earned a PhD in molecular genomics at King's College, Cambridge, and is a member of the Royal College of Physicians.
Andrew McFadyen, MHSc, 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. Contact him at firstname.lastname@example.org.
Lindsay McNair, MD, MPH, MS
Dr. McNair is the principal consultant at Equipoise Consulting, LLC. From 2013 to 2023, Dr. McNair was the chief medical officer for the WIRB-Copernicus Group (WCG). As part of the WCG IRB Executive Committee, she oversaw IRB member selection and training and IRB policy development, and helped to maintain regulatory and accreditation compliance. She also provided consultation to institutions and pharma/biotech companies on a wide range of issues related to protocol design, regulatory compliance, human subject protection, and ethical policy development (pre-approval access, subject compensation). Dr. McNair is adjunct faculty at Boston University and teaches graduate courses on the scientific design of clinical research studies. She is an associate editor for the Journal of Empirical Research on Human Research Ethics. She has previously been part of the Human Subjects Review Board of the U.S. Environmental Protection Agency, various working groups within Harvard’s Multi-Regional Clinical Trials program, and the Advancing Effective Research Ethics Oversight consortium. Contact her at email@example.com.
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’s biotechnology council. Her current work centers on issues at the intersection of bioethics, health policy, and the pharmaceutical industry, and includes a focus on developing and implementing accountability metrics for ethical clinical trial design, data sharing, and access to medicines. Contact her at firstname.lastname@example.org.
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. Contact him at email@example.com.
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. Contact her at firstname.lastname@example.org.
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). Contact her at email@example.com.
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. Contact him at firstname.lastname@example.org.
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. Contact him at email@example.com.
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. Contact her at firstname.lastname@example.org.
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. Contact him at email@example.com.
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. Contact him at firstname.lastname@example.org.
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. Contact him at email@example.com.
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. Contact her at firstname.lastname@example.org.