Working Group on Compassionate Use & Preapproval Access Members | NYU Langone Health

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Division of Medical Ethics Working Group on Compassionate Use & Preapproval Access Working Group on Compassionate Use & Preapproval Access Members

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 arthur.caplan@nyulangone.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 alison.bateman-house@nyulangone.org.

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 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.

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 nancyadubler@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. 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 has served on the business advisory board of the National Hemophilia Foundation and the board of directors of Parent Project Muscular Dystrophy. Contact her at chirawat@vozadvisors.com.

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 kholcombe20@gmail.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.

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, and she is a member of CUPA’s Bioethics of Individualized Therapeutics Subgroup. In addition to being a member of 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 lisa.kearns@nyulangone.org.

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 laura.kimberly@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.

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 aisha.langford@nyulangone.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 lynchhf@pennmedicine.upenn.edu.

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. Contact him at mcfadyena@me.com.

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. Contact her at lindsaymcnair333@gmail.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 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 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 regine.nshimiyimana-maniraho@jefferson.edu.

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 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.

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 russell.teagarden@gmail.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 david.wallach@nyulangone.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 tom.watson@bionical-emas.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 embeedub@gmail.com.