§360bbb–0a Investigational drugs for use by eligible patients

Summary

Lets certain very sick patients get an unapproved drug when they have no other approved options and cannot join a clinical trial. To qualify, a patient must have a life‑threatening condition, have tried all approved treatments, be unable to join a trial as a doctor certifies, and give written informed consent (or have a legal representative give it). The certifying doctor must be in good standing with their licensing board and must not be paid by the drug maker for that certification. An “eligible investigational drug” must have finished a Phase 1 (first‑in‑human) trial, not be approved for any use, and either have a formal application filed or be in a clinical trial meant to prove effectiveness with an active investigational new drug application. The drug’s development must still be ongoing and not halted or abandoned. Drugs given this way are allowed to skip some usual FDA rules if the sponsor and others follow certain investigational‑drug requirements. The FDA generally cannot use patient results from these uses to delay or hurt the drug’s approval unless the agency decides the results are critical to safety or the sponsor asks to use them. If the agency decides they are critical, the decision must be written, explain the public health reason, and come from the center director or higher. Drug makers must give the FDA an annual summary with doses supplied, patients treated, uses, and any known serious adverse events by the deadline the FDA sets. The FDA will post an annual public report about these uses and how patient outcomes were handled in reviews.