Right now, in the right way: U. S. Food and Drug Administration's expanded access program and patient rights

Based on approval data, the U. S. Food and Drug Administration (FDA) program that allows for early access to drugs and medical devices before marketing approval, is a very effective means for seriously ill individuals to be treated with investigational agents. Currently the FDA receives, reviews, and approves >99% of over 1000 requests each year. This program is called “expanded access” (EA), also referred to as “compassionate use”. In addition, recent changes in the FDA EA process have streamlined the application to further decrease the timeline and surmount procedural barriers. For individual patient access, a 2-page form is submitted and reviewed within days. If the request represents an emergency, this can be done over the phone (often within hours of the request). The EA approval rate and abbreviated application process suggest that the FDA is not an impediment to patients gaining access to experimental therapeutics. Nonetheless, a United States federal Right-to-Try (RtT) law was passed and signed by President Trump. This legislation, first at the state and now at the federal level, evolved from the efforts of the libertarian Goldwater Institute to make experimental drugs available to patients who seek them by removing oversight of the FDA and local Institutional Review Boards (IRB; ethics committees) Parties that support the RtT legislation identify the FDA as the primary obstacle to accessing experimental therapies.

Authors
M E Blair Holbein, Kevin J Weatherwax, Misty Gravelin, Raymond Hutchinson, George A Mashour
Journal
Journal of clinical and translational science
Publication Date
PMCID
PMC6202008
DOI
10.1017/cts.2018.318
Linked Authors
Misty
Gravelin
Project Manager
Kevin
Weatherwax
Managing Director, Michigan Institute for Clinical and Health Research (MICHR; University of Michigan CTSA Hub); Co-PI Transforming Expanded Access to Maximize Support and Study (TEAMSS); Co-director, MICHR Center for Drug Repurposing
Blair
Holbein
Director Regulatory Support Office and Assistant Professor
George
Mashour, MD PhD
Co-Director, University of Michigan CTSA; Robert B. Sweet Professor & Chair, Department of Anesthesiology