Providing Patients with Critical or Life-Threatening Illnesses Access to Experimental Drug Therapy: A Guide to Clinical Trials and the US FDA Expanded Access Program

Abstract

This review article considers two alternative options to standard treatment for desperately ill patients when standard treatments are no longer working: clinical trials and the Food and Drug Administration’s (FDA’s) expanded access program. The article describes the history of drug regulation in the United States, pointing out that the clinical research process to demonstrate safety and efficacy has lengthened the time to market approval. The author describes the advantages and disadvantages of clinical trials and of expanded access primarily for patients, with a discussion of the FDA’s expanded access program, including descriptions of single patient, intermediate-size patient populations and widespread treatment uses. The advantages of the FDA’s expanded access program over the new federal right-to-try law are also discussed. Alternative clinical designs are introduced into consideration as a way to improve the clinical trial process, reduce the time to market approval and perhaps interest more patients in enrolling in trials.