FDA Updates Guide on Expanded Access to Investigational Drugs

FDA has issued updated guidance clarifying how patients, physicians, and drug developers can access investigational therapies outside of clinical trials, incorporating policy changes enacted under recent federal laws. The document, Expanded Access to Investigational Drugs for Treatment Use: Questions and Answers, reflects the agency’s latest thinking on so-called “expanded access” pathways, which allow patients with serious or life-threatening conditions to receive experimental drugs when no satisfactory treatment options are available.

FDA says the updated guidance addresses ongoing questions from industry and clinicians about how to implement expanded access regulations first formalized in 2009. It is intended to improve awareness and consistency in how expanded access requests are handled, particularly for patients with limited or no therapeutic alternatives.

The agency outlines three primary pathways: individual patient access (including emergency use), access for intermediate-size patient populations, and broader treatment use under a treatment investigational new drug (IND) application.

While the framework is intended to facilitate access, FDA emphasized that several criteria must still be met, including ensuring that use of an investigational drug does not interfere with ongoing clinical trials or compromise the drug’s development. Importantly, sponsors must agree to provide the drug — FDA cannot require companies to grant access.

The guidance also integrates statutory changes from the 21st Century Cures Act and the FDA Reauthorization Act of 2017, which introduced new transparency requirements for drug manufacturers.

Under those laws, companies developing investigational drugs must publicly post their policies for evaluating expanded access requests, including contact information, eligibility criteria, and expected response timelines. These policies must be made available early in development — either when mid- to late-stage trials begin or shortly after a drug receives certain expedited designations.

However, FDA reiterated that public disclosure of these policies does not guarantee patient access, as participation remains voluntary for drug sponsors.