Our drug development process has produced many life-saving medications, but patients experiencing rare diseases and similar conditions often are left with limited options for treatment. For an approved treatment to be developed, research on a new candidate or existing drug must validate safety and efficacy based on contemporary research expectations. Randomized clinical trials are conducted for this purpose, but they are also costly, laborious, and time-consuming. For this reason, The 21st Century Cures Act mandates that the US Food and Drug Administration look for alternative methods for approving drugs, in particular exploring the uses of real-world data and evidence. Expanded access (“compassionate use”) is a pathway for the clinical treatment of patients using drugs that are not yet approved for prescribing in the United States. Using real-world evidence generated from expanded-access patients presents an opportunity to provide critical data on patient outcomes that can serve regulatory approval in conjunction with other observational datasets or clinical trials, and in limited circumstances may be the best data available for regulatory review. In doing so, we may also support and encourage patient-centered care and a personalized medicine approach to drug development.

Expanded access (EA) provides a pathway for the clinical use of investigational products (drugs, biologics, and medical devices) for patients who are without satisfactory therapeutic options and for whom a clinical trial is not available. Academic medical centers (AMCs) are likely to encounter EA requests, but it is unknown what support is available at these institutions for physicians seeking EA for patients. Methods: A landscape assessment was conducted at AMCs, focused on those within the Clinical and Translational Science Awards (CTSA) consortium. Results: Forty-seven responses were evaluated including 42 CTSA hubs. The large majority (43 of 47 respondents) reported using single-patient EA, while 37 reported multi-patient industry sponsored EA and 37 reported multi-patient investigator-initiated EA. Only half reported central tracking of EA requests. Support was available at 89% of sites for single-patient EA but less often for multi-patient EA. Types of support varied and were focused largely on the initial submission to the FDA. Conclusion: Use of and support for EA is widespread at AMCs, with support focused on single-patient requests. Gaps in support are common for activities after initial submission, such as FDA reporting and data collection.