Availability of Investigational Medicines Through the US Food and Drug Administration’s Expanded Access and Compassionate Use Programs

Key Points Question What is the timing and duration of investigational drug availability through the US Food and Drug Administration’s (FDA’s) expanded access program? Findings In this cross-sectional study of 92 FDA-approved drugs with associated expanded access programs, the median premarket expanded access availability was 10 months. Of 92 expanded access programs, 64 (69.6%) were initiated within the 6 months preceding or following a new drug’s application submission to the FDA. Meaning The FDA and the pharmaceutical industry have established a balance between investigational new drug access and patient safety that may be compromised by policy makers seeking to speed access to investigational medicines through the Right to Try Act.