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, regulation, and patient advocacy. This enables us to draw on the perspectives of all stakeholders involved in this preapproval access.

Arthur L. Caplan, PhD, Co-Chair

Dr. Caplan is the Drs. William F. and Virginia Connolly Mitty Professor of Bioethics. He founded the Division of Medical Ethics in 2012 and was its director through December 2025. He co-chairs the Johnson & Johnson Compassionate Use Advisory Committees (CompAC). He is the author and editor of 32 books and more than 880 papers in peer-reviewed journals. During the COVID-19 pandemic, he served 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, and developed an ethical framework for distributing novel drugs and vaccines for Johnson & Johnson and Moderna. In 2016, the National Organization for Rare Disorders (NORD) honored him with its Rare Impact Award; he received the Food and Drug Law Institute’s Distinguished Service Leadership Award the same year. In 2019, he received the Reagan-Udall Foundation for the FDA’s Innovation Award. Learn more about Dr. Caplan, and contact him at Arthur.Caplan@NYULangone.org.

Alison Bateman-House, PhD, MPH, MA, Co-Chair

Dr. Bateman-House is an associate professor in the Section of Medical Ethics at NYU Grossman School of Medicine. She has published and spoken extensively on access to investigational medical products, clinical trial conduct and design, individualized therapeutics, and research and public health ethics. She co-chairs the Working Group on Pediatric Gene Therapy & Medical Ethics, which studies ethical issues surrounding the development and use of gene therapies for children, especially those with rare conditions. PGTME creates forums for the difficult but essential policy discussions necessary to the advancement of these technological capabilities. She chairs the Compassionate Use Advisory Committee (CompAC), the first independent body of its kind created to advise a pharmaceutical company on the ethical, transparent allocation of its scarce products in development. This research was awarded the 2019 Reagan-Udall Foundation for the FDA’s Innovation Award. Learn more about Dr. Bateman-House, and contact her at Alison.Bateman-House@NYULangone.org.

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

Dr. Andrews is a neurologist at NYU Langone Health with a specialized focus on neuromuscular diseases, particularly amyotrophic lateral sclerosis (ALS). She is the director of the ALS clinic and the director of clinical trials. She is the elected co-chair at the Network of Excellence for ALS (NEALS)—an international consortium focused on developing novel treatments. She is a member of the steering committee for the Cures Collective, a nonprofit organization devoted to unifying efforts against neurodegenerative diseases to expedite treatments and improve care accessibility. Dr. Andrews hold board certifications in neurology, neuromuscular disease, and electrodiagnostic medicine. She received the Diamond Award for ALS Research and the Healey International Prize for Innovation in ALS and has held fellowships in the American Academy of Neurology (FAAN) and the American Neurological Association (FANA). 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 JA2289@CUMC.Columbia.edu.

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 Hayley.Belli@NYULangone.org.

Taryn L. Blanchard, MS

Ms. Blanchard is a clinical trial manager at Kumquat Biosciences, with extensive experience leading global oncology clinical trials across Phases I–III. She specializes in driving cross-functional execution from clinical trial start-up through closeout, including delivery of key milestones, such database lock and clinical study reports to support biologics license applications (BLAs). Ms. Blanchard has managed multiple concurrent oncology studies, overseeing vendor partnerships, CROs, and internal clinical operations teams to ensure quality, compliance, and timely progress. Earlier in her career, she worked as a medicinal chemist at Gilead Sciences, where she synthesized novel small molecules and co-authored multiple patents related to integrin inhibitors. She holds an MS in organic chemistry from the California Institute of Technology, a BA in chemistry from Connecticut College (magna cum laude, Phi Beta Kappa), and a certificate in Clinical Research Conduct and Management from UC Berkeley Extension. Contact her at tarynlblanchard@gmail.com.

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 Pat@Parentprojectmd.org.

Cláudia Hirawat

Ms. Hirawat is the owner and executive chair of VOZ Advisors, a global consulting firm focused exclusively on patient advocacy and engagement. She was formerly the president of PTC Therapeutics, where her key accomplishments include leading multiple business functions, such as corporate and business development, as well as multiple rounds of private and public financing. Ms. Hirawat was the lead negotiator of multiple collaborations (Genzyme, Pfizer, Schering-Plough, Hoffman-La Roche, Celgene) including the collaboration between PTC, Hoffman-LaRoche, and the SMA Foundation that led to the development of Evrysdi (risdiplam) for the treatment of spinal muscular atrophy (SMA). She serves as adjunct faculty in NYU Grossman School of Medicine’s Section of Medical Ethics. Contact her at CHirawat@Vozadvisors.com.

Kay Holcombe, MS

Ms. Holcombe, now retired, most recently served as senior adviser to the Milken Institute Center for Public Health and senior vice president for science policy at BIO (Biotechnology Innovation Organization). She has extensive experience in government health policy in the federal government executive branch and Congress, and in the profit and nonprofit sectors. She received an MS in chemistry from the University of Virginia and a BS in chemistry from the University of Illinois, where she graduated with honors and was elected to Phi Beta Kappa, Phi Kappa Phi, and Iota Sigma Pi. She currently serves on the boards of the Milken Institute for Public Health and the Critical Path Institute. She is chair of the board of the National Organization for Rare Disorders (NORD). Contact her at KHolcombe20@Gmail.com.

Sukhun Kang, PhD, MS

Dr. Kang is an assistant professor of technology management at the University of California, Santa Barbara. His research interests are in strategy and innovation, with a particular focus on the biopharmaceutical and high-tech industries. He received his PhD in Strategy and Entrepreneurship from London Business School. 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 the University of Southern California. Learn more about Dr. Kang and contact him at SukhunKang@ucsb.edu.

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 Karell@Chop.edu.

Lisa Kearns, MS, MA

Ms. Kearns is the senior research associate in the Section 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 the section’s Working Group on Pediatric Gene Therapy and Medical Ethics and the deputy chair of the Compassionate Use Advisory Committees. She is also the associate director of the section’s High School Bioethics Project. Contact her at Lisa.Kearns@NYULangone.org.

Richard Klein

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 RKlein123@AOL.com.

Connie Law, PharmD

Dr. Law is a clinical pharmacist specializing in drug information and policy. She is currently practicing at the Children's Hospital of Philadelphia (CHOP) and an adjunct faculty at Wilkes University. She has experience in taking care of pediatric patients with thrombotic disorder and pediatric patients diagnosed with rare diseases. She serves as the secretary of the Drug Use Evaluation committee at CHOP, which is involve with the provision of drug therapy use with limited and sometime no pediatric information. She received a PharmD from the Philadelphia College of Pharmacy and completed her pharmacy practice residency at the Philadelphia VA Medical Center and drug information residency at Thomas Jefferson University and Jefferson Health. Contact her at LawC1@Chop.edu.

Holly Fernandez Lynch, JD, MBE

Professor Fernandez Lynch is associate professor of medical ethics and law 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 quality and effectiveness, and FDA policy and practice. 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. She is a Fellow of the Hastings Center and a National Academy Emerging Leader in Health and Medicine. Contact her at LynchHF@Pennmedicine.UPenn.edu.

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 ethics lead for Precision Child Health at the Hospital for Sick Children in Toronto, Ontario. He is also the executive director for the Isaac Foundation, a nonprofit organization dedicated to helping families battling rare diseases, and he has worked extensively in the pre-approval access to medicines space. Mr. McFadyen has worked closely with and been a long-time advisor to governments and pharmaceutical companies to help ensure access to novel therapeutics for patients in need, create equitable expanded access programs, and fairly ration pre-approval access to scarce resources. He has testified as an expert witness in both the U.S. Senate and Canadian parliamentary steering committees on access to treatments for patients with rare diseases. He is a longtime member of the Section of Medical Ethics Working Group on Pediatric Gene Therapy and Medical Ethics. Contact him at andrew.mcfadyen@sickkids.ca.

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 (preapproval 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 LindsayMcnair333@Gmail.com.

Ken Menkhaus, PhD

Dr. Menkhaus is the C. Louise Nelson Professor of Political Science at Davidson College, NC, where he has taught since 1991. He is author of more than 50 articles and monographs, has testified to congressional subcommittees on five occasions, and has been interviewed on CNN, the BBC, MSNBC, NPR’s “All Things Considered,” and the PBS “NewsHour.” In 2018, he was diagnosed with ALS and has since shifted some of his research energies to the politics, policies, and ethics of expanded access to experimental therapies for ALS. From 2020–23 he served on the ALS Association Board of Trustees and continues to serve on its Policy Advocacy and Care Services committees.

Jennifer Miller, PhD

Dr. Miller is director of the Yale Bioethics Lab, co-director of the Yale Program for Biomedical Ethics, and an associate professor in Yale School of Medicine. She is also the director of the Good Pharma Scorecard (an index that ranks and rates pharmaceutical companies on their bioethical performance) and founder of the nonprofit Bioethics International. Her current research focuses on ethics, access, and governance in biomedical innovation, healthcare data sharing, and AI-enabled medical care. She also specializes in developing and using metrics to enhance accountability, patient centricity, and social responsibility across the pharmaceutical industry. Prior to joining Yale’s faculty, she was an assistant professor (tenure track) at NYU School of Medicine and completed training in physics, regulatory governance, bioethics, and business ethics at Fordham University, Duke University, Regina Apostolorum Pontifical University, and Harvard University. Contact her at Jennifer.E.Miller@Yale.edu.

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 Kenneth.Moch@Gmail.com.

Regine Nshimiyimana Maniraho, DNP, PharmB, FNP

Dr. Nshimiyimana Maniraho is a trained pharmacist and nurse practitioner with specialties in family (FNP), acute care (AGACNP), and oncology (AOCNP). Her academic journey spanned the National University of Rwanda, Thomas Jefferson University, the University of Pennsylvania, and George Washington University. She practices as a nurse practitioner at the Sidney Kimmel Cancer Center-Jefferson Health and is an adjunct faculty member at Thomas Jefferson University. She also leads the liaison between Thomas Jefferson University and the University of Rwanda as part of the Jefferson Consortium for African Partnerships. Contact her at Regine.Nshimiyimana-Maniraho@Jefferson.edu.

Barbara Redman, PhD, MBE, RN

Dr. Redman is an associate of the Section 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 Reconstructing Research Integrity (Springer/Nature, 2023). Contact her at BKRedman@Comcast.net.

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 CTR00@BU.edu.

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 DScheer@Scheerandco.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 Lesha.Shah@MountSinai.org.

David Wallach, MPH, CIP

Mr. Wallach joined NYU Langone in May 2016 and now serves as director of Regulatory Affairs & Business Operations. He leads teams that support FDA IND/IDE submissions, < < clinicaltrials.gov compliance, DSMB management, and strategic research initiatives. He also serves as an instructor in the MS in Clinical Research program at NYU, teaching a course on federal regulations and agencies. Previously, he spent more than 14 years at the Albert Einstein College of Medicine, ultimately serving as IRB director for Einstein, Montefiore, two NYC H+H hospitals, and Yeshiva University. He holds an MPH from Albert Einstein and has been a Certified IRB Professional since 2005. Contact him at David.Wallach@NYULangone.org.

Tom Watson, BSc

Mr. Watson is the chief executive officer at Bionical Emas. He has more than 25 years of experience within the pharmaceutical industry across senior roles in the U.K. and U.S. He founded Bionical EAP in 2018. He is a leading figure within the EAP space and has partnered with pharma and biotech companies to develop hundreds of global programs, allowing patients to gain access to potentially lifesaving treatments. He is leading Bionical toward its vision of advancing access to treatments that will have the biggest impact on human health, creating a meaningful impact in the world. Contact him at Tom.Watson@BionicalEmas.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 Alvarez Award-winning science memoir, A Series of Catastrophes & Miracles, and now speaks to and consults for numerous health organizations across the country. She holds certifications from the narrative medicine program at Columbia University and the conflict resolution program at Drew University. She is currently a doctoral candidate in medical humanities at Drew, focusing on negotiation in informed consent. Contact her at Embeedub@Gmail.com.