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

Abstract

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 [1]. This program is called “expanded access” (EA), also referred to as “compassionate use” [2, 3]. In addition, recent changes in the FDA EA process have streamlined the application to further decrease the timeline and surmount procedural barriers [3]. 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 [4, 5]. 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) [6]. Parties that support the RtT legislation identify the FDA as the primary obstacle to accessing experimental therapies. What seems to have been lost in the public discussions is a clear presentation of the critical benefits and protections afforded the patient by the current EA system that are missing in RtT (Table 1). Lawmakers and RtT advocates do not appear to understand or acknowledge the inherent risk in experimental therapies, which may have been tested in as few as a dozen patients in a phase 1 trial. Rather than serving as an impediment, the FDA serves as a safeguard for patients facing desperate, complex situations. While much of the current literature focuses on the redundancy and ethical arguments about the legislation [7–10], there are pragmatic and unacknowledged issues surrounding patient rights that are maintained by EA. Importantly, there is a real value to patients that the FDA, as well as other partners in the current EA system, bring to the process. The RtT law, as written, does not preclude the use of the existing EA process. We believe that is very important to make providers aware of EA and that it offers the best option for patients. The FDA, IRBs, and research institutions play critical roles in the EA process by providing patients with the safest possible access to investigational agents. Table 1 Comparison of patient rights under US Food and Drug Administration (FDA) expanded access program and Right-to-Try law