21 U.S.C. § 360bbb–0a — Investigational drugs for use by eligible patients

(a) Definitions This section defines three terms. An "eligible patient" is a patient who meets three things. (A) A doctor has diagnosed them with a life-threatening disease or condition, using the FDA's definition in 21 CFR 312.81. (B) The patient has tried all approved treatments and can't join a clinical trial for the drug. A physician must certify this. That physician must be in good standing with their licensing board, and the manufacturer cannot pay that physician directly for the certification. (C) The patient — or their legal representative — has given written informed consent to the treating physician about the investigational drug. An "eligible investigational drug" is an investigational drug, as defined in section 360bbb, that meets four conditions. (A) It has finished a Phase 1 clinical trial. (B) It has not been approved or licensed under section 355 of this title or section 351 of the Public Health Service Act. (C) Either an approval application has been filed for it, or it is being tested in a trial meant to be the main basis for an approval application, with an active investigational new drug application behind that trial. (D) The manufacturer is still actively developing or producing it — it hasn't been discontinued or put on clinical hold. A "Phase 1 trial" is a Phase 1 clinical investigation as described in 21 CFR 312.21. (b) Exemptions If an eligible investigational drug is given to an eligible patient following this section's rules, it is exempt from several requirements: sections 352(f), 353(b)(4), 355(a), and 355(i) of this title; section 351(a) of the Public Health Service Act; and parts 50, 56, and 312 of title 21 of the Code of Federal Regulations. This exemption only applies if the sponsor, manufacturer, distributor, prescriber, dispenser, or anyone else providing the drug still follows three specific safety rules for investigational drugs: 21 CFR 312.6, 312.7, and 312.8(d)(1). (c) Use of clinical outcomes (1) In general. The Secretary generally cannot use what happens to a patient under this section — a "clinical outcome" — to slow down or hurt the drug's official review or approval. There are two exceptions: (A) the Secretary decides, following the process in paragraph (2), that the outcome is critical to judging the drug's safety; or (B) the sponsor itself asks the Secretary to use the outcome. (2) Limitation. If the Secretary makes that determination under (1)(A), the Secretary must give the sponsor written notice explaining the public health reason for it, and that notice becomes part of the official record. Only the director of the FDA center reviewing the drug can make this decision — it can't be handed down to someone lower. (d) Reporting (1) In general. The drug's manufacturer or sponsor must send the Secretary an annual summary of how the drug was used under this section — how many doses were given, how many patients were treated, what the drug was used for, and any serious adverse events. The Secretary sets the deadline for this summary by regulation and may fold it into the drug's regular annual report. (2) Posting of information. The Secretary must post an annual public report on the FDA website. It must show how many drugs had clinical outcomes used under (c)(1)(A) (Secretary-required use), how many under (c)(1)(B) (sponsor-requested use), and how many were not used at all in reviewing an approval application.