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# GAO Report on Patient Access to Investigational Drugs
- URL: https://www.fdaweb.com/gao-report-on-patient-access-to-investigational-drugs/
- Published: 2019-09-09T12:00:00.000Z
- Updated: 2026-09-15T01:48:28.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5144949

A new Government Accountability Office (GAO) [report](https://www.gao.gov/assets/710/701243.pdf?ref=fdaweb.com) finds that FDA has ongoing efforts to help drug manufacturers identify the circumstances under which they could broaden clinical trial eligibility criteria to include patients who are commonly excluded, such as pediatric patients and patients with impaired liver and kidney function, without compromising study results. The report was mandated by Congress in the FDA Reauthorization Act of 2017, which called for a review of actions taken to facilitate access to investigational drugs. The report describes (**1**) actions FDA and drug manufacturers have taken to broaden eligibility criteria for clinical trials, (**2**) actions FDA has taken to facilitate access to investigational drugs outside of clinical trials, and (**3**) information drug manufacturers have communicated to patients and physicians about access to investigational drugs outside of clinical trials.

The GAO report notes that FDA issued guidance in March with recommendations on ways manufacturers could broaden eligibility criteria for cancer clinical trials, when clinically appropriate. In June, it issued related guidance that applies to a wider range of clinical trials beyond cancer trials.

Outside of clinical trials, patients with certain medical conditions may request to obtain access to investigational drugs, GAO’s report says. “This can occur under FDA’s expanded access program, or through the 2018 ‘Right to Try’ law. Under either pathway, a patient can only access the investigational drug if its manufacturer agrees to the request. FDA has taken steps to facilitate access to investigational drugs outside of clinical trials, and most manufacturers in GAO’s review communicated information to patients and physicians through their Web sites about how to access their investigational drugs outside of clinical trials.”

Additionally, the report notes that since 2017, FDA took steps to simplify its expanded access program to make it easier for patients. To address concerns raised by manufacturers, FDA clarified guidance on how it would review data resulting from the program. Seven of the 10 manufacturers GAO interviewed viewed the guidance as an improvement.