For patients facing serious or life-threatening conditions, waiting for full regulatory approval of a promising therapy is often not an option. Expanded Access Programs (EAPs), sometimes referred to as compassionate use programs, have long provided a regulatory pathway for patients to receive investigational treatments outside of clinical trials.

In a recent FDA warning letter involving investigational distribution practices, the agency cited failures tied to regulatory authorization and documentation requirements. The specific facts are unique to that company, but the broader lesson applies to every emerging biotech developing an investigational product.