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.

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.

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.

Anne Donguy is a writer and patient advocate.

Sukhun Kang is a PhD candidate in Strategy & Entrepreneurship at the London Business School. His academic advisor is Gary Dushnitsky, and he expects to finish the program in year 2023. His research interests are in innovation and entrepreneurship with a particular focus on biopharmaceutical and high-tech industry. Prior to joining the doctoral program, he worked as a semiconductor engineer at Samsung Electronics and led an Internet startup in 2010. These roles afforded him a diverse set of experiences, from designing products to managing a venture, which informs his research. He holds a BS in Computer Engineering from the University of Illinois and Master's degrees from the University of Southern California in Computer Engineering and Entrepreneurship & Innovation.

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).

Hayley Belli is an Assistant Professor of Biostatistics within the Department of Population Health at NYU Grossman School of Medicine, where she conducts research in the design, conduct, and analysis of pragmatic and adaptive clinical trials. Her work develops and tests interventions that utilize behavioral economic theory and digital health tools to improve clinical outcomes for chronic conditions, including diabetes, dementia, and migraines. She is also interested in pragmatic studies embedded within electronic health records, and methods for extracting evidence from real world data in the context of Expanded Access programs. She is a member of the Working Group on Compassionate Use and Pre-Approval Access within the Division of Medical Ethics, and co-chairs the Ethics and Real-World Evidence Research Project.

Rakesh Davda's background is in working across Pharmaceutical Sales, Medical Affairs and Market Access, including Observational Studies and Global Value Dossiers. Extensive knowledge in International Health Technology Assessment, Pricing and Reimbursement (MSc), with practical experience gained through working on National Institute for Health and Care Excellence (NICE) technology appraisals. Acknowledging the importance of explanatory trials in clinical development, interest in opportunities to understand the real-world benefit of an intervention. Focused on exploring innovative solutions and methodology to generate data to bring therapies to patients with unmet clinical faster. Experience working with biotech and pharmaceutical companies on EAP RWD collection to supplement broader strategic objectives, with related experience in Oncology, Hematology and Rare Disease.

Dr. Concato is Associate Director for Real-World Evidence Analytics in the Office of Medical Policy (OMP), Center for Drug Evaluation and Research (CDER), Food and Drug Administration (FDA). After conducting clinical research studies for more than 25 years as an independent investigator and research center director at Yale University & the Department of Veterans Affairs, he now develops, coordinates, and implements medical policy programs and strategic initiatives, with a focus on real-world evidence (RWE). These efforts include overseeing RWE guidance development and demonstration projects, as well as engaging external stakeholders and contributing to the review of RWE submissions, in support of the agency’s evaluation of RWE as mandated by the 21st Century Cures Act.

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.

Peter Marks received an MD, PhD from New York University and completed Internal Medicine Residency and Hematology/Oncology Fellowship at Brigham and Women's Hospital. He has worked in academic medicine and in industry prior to joining FDA in 2012 at Deputy Center Director of the Center for Biologics Evaluation and Research. He became Center Director in 2016 and has been heavily involved in pandemic response since 2020.

Christopher Robertson is N. Neal Pike Scholar and Professor of Law at Boston University. Robertson previously taught at University of Arizona, and has visited at Harvard, NYU, and the London School of Economics. Robertson's more than 50 scholarly articles in leading journals, such as JAMA, NEJM, and Science, and has been featured in Wall Street Journal, NBC News, NPR, and the Washington Post. Robertson has co-edited three books, and in 2019 Harvard University Press published, Exposed: Why Our Health Insurance is Incomplete and What Can Be Done About It. Robertson graduated magna cum laude from Harvard Law School and earned a doctorate in Philosophy at Washington University in St. Louis.

Steve Usdin is a senior editor at BioCentury Publications. Based in Washington, D.C., he is responsible for covering issues at the intersection of public policy, science and politics, ranging from stem cells and cloning to drug safety and the impact of public policy on innovation.

Peter Marks received an MD, PhD from New York University and completed Internal Medicine Residency and Hematology/Oncology Fellowship at Brigham and Women's Hospital. He has worked in academic medicine and in industry prior to joining FDA in 2012 at Deputy Center Director of the Center for Biologics Evaluation and Research. He became Center Director in 2016 and has been heavily involved in pandemic response since 2020.

Paul is the Head of Global Governance in the cross-divisional Chief Medical Office at Novartis, with responsibility for the oversight, standards, training and systems for compassionate use/expanded access, grants and post-registration medical programs (e.g. Investigator initiated trials, research collaborations, non-interventional studies, registries, and patient support programs). He has 20 years’ experience in the Pharma industry across multiple therapeutic areas and roles. Prior to joining the pharmaceutical industry, he practiced as a clinical pharmacist, and trained in law, business and general management. He previously served as an industry representative on the WHO/Roll-Back Malaria (RBM) global case management working group, managed many partnerships (Public-Private/Private-Private) and is a regular speaker/faculty at various conferences and stakeholder discussions on Global Health, Patient Access, Bioethics and Medical Governance.

Anne Donguy is a writer and patient advocate.

Luciana Borio, M.D., is a Venture Partner at ARCH Venture Partners and a senior fellow for global health at the Council on Foreign Relations. Dr. Borio specializes in biodefense, emerging infectious diseases, medical product development, and complex public health emergencies. Prior to joining ARCH, she was a senior vice president at In-Q-Tel, a non-profit, strategic investment firm that works to support the U.S. Intelligence Community. Past positions include serving as a member of President Biden’s transition COVID-19 Advisory Board and Director for Medical and Biodefense Preparedness at the National Security Council (2017-2019). Prior to that, she was the Acting Chief Scientist of the U.S. Food and Drug Administration (2015-2017) and the Assistant Commissioner for Counterterrorism Policy of the FDA (2010-2017).

Gregg Gonsalves is an expert in policy modeling on infectious disease and substance use, as well as the intersection of public policy and health equity. His research focuses on the use of quantitative models for improving the response to epidemic diseases. For more than 30 years, he worked on HIV/AIDS and other global health issues with several organizations, including the AIDS Coalition to Unleash Power, the Treatment Action Group, Gay Men’s Health Crisis, and the AIDS and Rights Alliance for Southern Africa. He was also a fellow at the Open Society Foundations and in the Department of Global Health and Social Medicine at Harvard Medical School from 2011-2012. He is a 2011 graduate of Yale College and received his PhD from Yale Graduate School of Arts and Sciences/School of Public Health in 2017. He is a 2018 MacArthur Fellow.

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.

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.

Phil is a graduate of the University of Washington where he was on the 1991 National Championship UW Football team. He has spent the past 25 years building a career in developing technology solutions for brands such as Sony, Hewlett Packard, and Home Depot. A loving husband to his wife Jennifer, Phil is also a dedicated father to four children (Arianne-19, Hunter-18, Parker-14, and Whitney-11). Phil was diagnosed with ALS in August of 2018 and immediately dedicated himself to making a difference in the fight against this horrific disease. Phil is active in the ALS Community and works with multiple ALS organizations with promoting ALS legislation and policy issues, increasing awareness and raising funds to find effective treatments and cures, and providing much needed support services for ALS patients and families.

Holly Fernandez Lynch, JD, MBE, is an Assistant Professor of Medical Ethics and Law at the University of Pennsylvania. Her scholarship focuses on clinical research ethics, access to investigational medicines, Food and Drug Administration policy, and the ethics of gatekeeping in health care. She is founder of the Consortium to Advance Effective Research Ethics Oversight (AEREO), which aims to understand, evaluate, and improve IRB quality and effectiveness through empirical research, and currently serves on the boards of Public Responsibility in Medicine & Research (PRIM&R) and the American Society of Law, Medicine, and Ethics.

Ms. Suzanne McGurn joined CADTH in July 2020 as its President and Chief Executive Officer. She brings to the role a deep understanding of the complex issues surrounding the management of pharmaceuticals, medical devices, and clinical interventions in Canadian health systems. Prior to joining CADTH, Ms. McGurn’s distinguished career spanned clinical practice, patient support, and senior roles in government. Within the Ontario Ministry of Health, she served as the Assistant Deputy Minister of the Drugs and Devices Division and the Executive Officer of the Ontario Public Drug Programs. She also led the implementation of the pan-Canadian Pharmaceutical Alliance and served as its first chair. Ms. McGurn holds both a Bachelor of Nursing Sciences and a Master of Public Administration from Queen’s University.

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 is also the elected co-chair of the Northeast ALS Consortium, an international, independent, non-profit group of researchers who collaboratively conduct clinical research in ALS and other motor neuron diseases. 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.

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.

Dr. Knoepfler is a Professor of Cell Biology & Human Anatomy at UC Davis School of Medicine. His research is focused on the epigenomic mechanisms of stem cells and brain cancers as well as the role of stem cells in tumors. In addition, he conducts educational outreach, bioethics and policy research on regenerative medicine issues. He is the author of three books on stem cells and CRISPR.

Dr. Carlos H. Villa is currently the Associate Director for Special Programs in the Office of Blood Research and Review at CBER/FDA. He joined the FDA in 2018, whereupon he served as a Medical Officer and clinical reviewer prior to his current role. Before joining the FDA, he was a clinical instructor in the Division of Transfusion Medicine and Therapeutic Pathology at the Hospital of the University of Pennsylvania, where he also completed his clinical residency and fellowship training. Dr. Villa has a PhD in Pharmacology with post-doctoral research experience in pharmacology and drug delivery. Dr. Villa’s clinical and scientific interests include transfusion medicine, immunohematology, and specialty coagulation testing.

Jordan Sloshower, MD, MSc is a psychiatrist and researcher whose work focuses on therapeutic applications of psychedelic substances, and in particular, how these novel treatments can be delivered in a manner that promotes holistic healing and social justice. He is currently a Lecturer in the Yale Department of Psychiatry, where he serves as an investigator and therapist in several clinical trials of psilocybin-assisted therapy. Jordan is also a clinical investigator in MAPS’ Expanded Access Program for MDMA-assisted therapy of PTSD and serves as a lead trainer with Usona Institute’s facilitator training program. Most recently, he was elected to the Board of Directors of the American Psychedelic Practitioners Association, reflecting his commitment to ethical stewardship of psychedelic medicines.

Edith Eby is Vice President, Global Medical Operations, Governance and Community in Pfizer Worldwide Medical & Safety. Edith is responsible for Global Medical Operations including grants, transparency reporting, publications and external funding; Medical Quality Oversight and Governance, Enterprise Medical Community Initiatives, and Expanded Access/Compassionate Use. Since joining Pfizer in 2002, Edith’s roles have included Oncology Medical Affairs, Global Medical Governance Lead and VP External Medical Communications. Areas of expertise are global field based medical, medical affairs, compliance, education and research grants, pediatric oncology and pharmacy. Edith obtained her BS in Pharmacy and PharmD from the University of Texas and completed an Oncology Specialty Residency at UTMD Anderson Cancer Center. Prior to joining Pfizer, Edith practiced as a pediatric oncology pharmacist at Texas Children’s Cancer Center.

Lesha D. Shah MD is dually board certified in Psychiatry and Child & Adolescent Psychiatry, and is Assistant Professor of Psychiatry at the Icahn School of Medicine at Mount Sinai. As medical education and supervisor for clinical training, she is committed to mentorship of medical students, residents and fellows. Dr. Shah’s primary academic focus is on pediatric research ethics and physician perspectives around medical decision-making for children. She studies issues of consent and capacity as they interface with family complexity and innovative medicine including clinical trials. She Co-Chairs the Pediatric Gene Therapy & Medical Ethics working group, is assistant editor for the Journal of the American Academy of Child and Adolescent Psychiatry, and serves on the Institutional Review Board at the Icahn School of Medicine at Mount Sinai.

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.

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.

Meghan Blair is currently the Associate Director of Regulatory Affairs at the Clinical Research Unit of the Abramson Cancer Center within the University of Pennsylvania. Meghan earned her graduate (MS) degree from the University of Rochester, School of Medicine and Dentistry, in Toxicology after completing her undergraduate (BS) work in Pharmacology and Toxicology at the University of the Sciences in Philadelphia (now USciences). She brings more than 10 years’ experience in an academic medical setting with principle responsibilities related to regulatory affairs and research compliance. Meghan has held previous positions within the University of Pennsylvania IRB, as enrollment site clinical staff at the Hospital of the University of Pennsylvania, and as a laboratory scientist at the University of Rochester School of Medicine. In her current role, she is instrumental in creating quality systems approaches for the conduct of clinical research, developing scalable quality and regulatory programs to support the use of investigational FDA regulated products (in both clinical research and clinical care), and strategic development for the regulatory affairs aspects of large scale research programs.

Rafael is currently a consultant to the biotechnology industry and most recently was the senior vice president of medical affairs, policy, and patient engagement at BridgeBio Gene Therapy. He has worked in global clinical research and development and patient advocacy in the biotechnology industry for nearly 30 years. His approach is to be highly considerate of the ethical implications of conducting and participating in clinical research, especially in rare diseases and in vulnerable and developing-world populations. He has served as adjunct faculty at the University of California Berkeley's Clinical Research Management Program and as an educator in human rights and justice.

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.

Andrew McFadyen is the Executive Director of The Isaac Foundation. He founded The Isaac Foundation, a non-profit organization, to fund viable and innovative research projects that aim to find a cure for MPS VI, a rare and progressive disease of which his eldest son, Isaac, was diagnosed. The Isaac Foundation has funded numerous international research grants since 2006, totalling well over $1 million. Isaac, the McFadyens, and The Isaac Foundation have been featured in articles in newspapers throughout the United States and Canada, including The Globe and Mail, The Independent, Kingston Life Magazine, and Sun Media.