An Introduction to Preapproval Access: Expanded Access, Compassionate Use & Right to Try

Most people access unapproved medical products through clinical trials, but what happens when patients can’t get into a clinical trial, or there is no trial focused on treating their disease? Non-trial preapproval access, through Expanded Access or Right to Try, may offer a way for patients to use an investigational product. In this webinar, a panel of experts from the working group on Compassionate Use and Preapproval Access will discuss the how, who, and why of accessing unproven medical products, both inside and outside of clinical trials. The panel will consider the perspectives of various stakeholders, including clinicians, industry members, patient advocates, regulators, and bioethicists to present the most comprehensive picture of preapproval access.

Featured Speakers

Moderator

Regine 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.

Panelists

Lisa 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.

Richard Klein is an internationally recognized expert in pre-approval, or expanded access to unapproved therapeutic agents. He serves as Director, Expanded Access Programs & Policy for the GE2P2 Global Foundation, following a more than 41-year career with the Food and Drug Administration (FDA). Having worked in various capacities at FDA provides him with a well-rounded understanding of the regulatory issues that affect patients. He helped develop the revised expanded access regulations and guidelines, led the creation of the FDA expanded access website, and played an active role in the development of the streamlined application for individual patient access, and the Reagan-Udall Foundation Expanded Access Navigator. Mr. Klein spearheaded the effort to create the waiver of full-board IRB review for individual patient access to unapproved drugs and biologics. Mr. Klein served as director of the FDA’s Patient Liaison Program in the agency’s Office of Health and Constituent Affairs, the primary agency interface with patients and patient advocate communities. He interacted extensively with outside communities and within the agency’s scientific and policy offices to advocate for patient interests, and to facilitate patient engagement in regulatory activities. He worked closely with patient communities to actively address issues and concerns of patients in a variety of areas, including treatment access to unapproved drugs, product safety, and clinical trial design. Before taking on that role, he created the FDA’s HIV/AIDS Program, working with AIDS activists and advocates to coordinate their input and participation in regulatory policy and decision-making related to HIV/AIDS. Prior to working in patient engagement, he was engaged in policy development and regulation of protections for human research subjects, and provided guidance for institutional review boards (IRBs).

Jinsy 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.

Ken Moch has been CEO or co-founder of five companies pioneering novel therapies for life-threatening diseases including cancer, pathogens and Alzheimer’s, and has been instrumental in the development of therapeutic approaches including liposomes and cord blood stem cells. He has served for over a decade on the Biotechnology Innovation Organization (BIO) Governing Board, chairing BIO’s Bioethics Committee and co-chairing the Emerging Companies Section’s Strategy and Policy Committee focused on the development of medicines for highly prevalent chronic diseases. He received an AB in biochemistry with a minor in health policy from Princeton University and an MBA from Stanford University.