Experimental Medicines Provided to Very Ill Patients in Expanded Access Programs Receive FDA Marketing Approval

July 31, 2017

NEW YORK, Bioethics International, a not-for-profit focused on the ethics, patient-centricity, and governance of how medicines are researched, developed, marketed and made accessible to patients around the world, today announced the publication of a new study, showing the FDA marketing approval rate of experimental drugs provided to patients through the FDA's expanded access programs, in BMC Research Notes.

The paper, titled "Characterizing Expanded Access and Compassionate Use Programs for Experimental Drugs", found most drugs (76%) provided through registered expanded access and compassionate use programs, eventually receive FDA marketing approval. The study also identified the funding source, product type, conditions treated, and patient demographics for registered expanded access programs.

"Drugs provided to patients through expanded access programs, more times than not, are eventually deemed safe by the FDA and receive FDA approval," said Jennifer E. Miller, Ph.D., founder of Bioethics International, assistant professor of medical ethics in the Department of Population Health of NYU School of Medicine and lead author on the paper. "However, drug safety or efficacy data are generally not made public from these programs. We found only 2% of programs publicly report results in ClinicalTrials.gov."

Miller continues, "The pharmaceutical industry was by far the largest sponsor of expanded access programs. Some companies and universities are already establishing programs to provide seriously ill patients access to experimental therapies outside of clinical trials."

Joseph Ross, MD, Associate Professor of Medicine and Public Health at Yale, added "Our results suggest that industry and other funders of expanded access and compassionate use programs are using ClinicalTrials.gov to make the public aware of their efforts, and of these opportunities to gain access to experimental therapies, but need to do better with respect to reporting patients' resulting experiences and outcomes, to inform other patients with these diseases and their families."

About Expanded Access and Compassionate Use Programs
Expanded access programs, also called "compassionate use" programs, provide patients with serious and life threatening conditions, who have tried all other approved treatment options, access to experimental products outside of clinical trials. At least 36 States have already passed Right to Try Legislation. Proposed federal Right to Try legislation, advocated by the White House, allows physicians to prescribe experimental therapies unapproved by the U.S. Food and Drug Administration (FDA) to terminally ill patients.

About Bioethics International
Bioethics International's (BEI) mission is to empower professionals and organizations in healthcare, life science and biotechnology to make responsible decisions - on issues that impact patients the most. Currently, we focus on the ethics and governance of how medicines and vaccines are researched, developed, marketed and made accessible to patients around the world. BEI runs the Good Pharma Scorecard, an index that ranks new drugs and pharmaceutical companies on their ethics, trustworthiness and patient-centricity. For more information, please visit www.bioethicsinternational.org.