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

IMPORTANCE: The Right to Try Act of 2017 allows patients with life-threatening conditions to access investigational medicines outside clinical trials without oversight from the US Food and Drug Administration (FDA). A better understanding of existing expanded access programs can inform the consideration and implementation of both the federal Right to Try Act and state right-to-try laws. OBJECTIVE: To determine the timing and duration of expanded access programs for investigational medicines initiated prior to FDA approval. DESIGN AND SETTING: This cross-sectional study examined expanded access and compassionate use programs registered through August 1, 2017, identified from ClinicalTrials.gov and publicly available FDA documents. MAIN OUTCOMES AND MEASURES: Start date of each program and 3 key regulatory dates (investigational new drug application activation, initial new drug application submission, and FDA approval), and timing and duration of expanded access availability in relation to new drug application submission and FDA approval. RESULTS: Through ClinicalTrials.gov, 92 FDA-approved drugs and biologics with associated expanded access programs initiated prior to FDA approval were identified. These programs were initiated between September 1996 and June 2017 for medicines that were most commonly used to treat cancer (n = 46 [50.0%]); metabolic, endocrine, and genetic diseases (n = 16 [17.4%]); and infectious diseases (n = 14 [15.2%]). The median (interquartile range) premarket expanded access availability was 10.0 (6.0-19.5) months, constituting a median (interquartile range) of 14% (7%-25%) of the premarket clinical development period (investigational new drug application activation to FDA approval). Of 92 expanded access programs, 64 (69.6%) were initiated just before or after new drug application submission: 24 (26.1%) were initiated during the 6-month period before, and 40 (43.5%) in the 6 months after. CONCLUSIONS AND RELEVANCE: Over the past 2 decades, expanded access programs have provided access to investigational medicines for which evidence of safety and effectiveness was established. For medicines that ultimately receive FDA approval, these findings suggest that the FDA and pharmaceutical industry have established a balance between investigational new drug access and protection of patients from therapies without established safety. This balance may be compromised by policy makers seeking to speed access to investigational medicines through the Right to Try Act.