Ethical Issues in Preapproval Access to Investigational Drugs in the U.S.

The U.S. FDA Expanded Access Program allows certain patients, under certain circumstances, to access investigational (experimental) drugs in development outside clinical trials, before they’re approved by the agency. Also known as preapproval access or compassionate use, the program has been largely successful since its inception in the 1980s, although not without controversy. This presentation will explain the history of this preapproval access pathway and the many ethical concerns it raises for all stakeholders: patients, physicians, regulators, legislators, and drug manufacturers. It will also discuss how the novel coronavirus pandemic has brought the program to the forefront, as patients and physicians have looked to unproven therapies to treat a potentially deadly disease, and examine the tension between the importance of clinical trials in bringing promising drugs to market (public good) and patients’ desire for “last chance” therapies (individual rights).