21 U.S.C. § 360bbb — Expanded access to unapproved therapies and diagnostics

(a) Emergency situations The Secretary may, under conditions the Secretary sets, allow investigational drugs or investigational devices to be shipped for diagnosing, monitoring, or treating a serious disease or condition in an emergency. (b) Individual patient access to investigational products for serious diseases A patient, acting through a physician licensed under state law, may ask a manufacturer or distributor for an investigational drug or device to diagnose, monitor, or treat a serious disease or condition. The manufacturer or distributor may provide it once four conditions are met: (1) the physician decides the patient has no comparable or satisfactory alternative therapy, and that the investigational product's likely risk isn't greater than the risk from the disease itself; (2) the Secretary decides there's enough evidence of safety and effectiveness to support that use; (3) the Secretary decides providing it won't get in the way of the clinical trials needed for eventual marketing approval; and (4) the product's sponsor or investigator submits to the Secretary a clinical protocol — following section 355(i) or 360j(g) and their regulations — describing its use in a single patient or a small group of patients. (c) Treatment investigational new drug applications and treatment investigational device exemptions When a sponsor or physician submits a protocol meant to give many eligible patients access — called an "expanded access protocol" — the Secretary must permit the product to be supplied under a treatment investigational new drug application or treatment investigational device exemption, if the Secretary finds all of the following: (1) it's meant to diagnose, monitor, or treat a serious or immediately life-threatening disease or condition; (2) there's no comparable or satisfactory alternative therapy for that population of patients; (3) either (A) it's already being tested in a controlled clinical trial under an active exemption under section 355(i) or 360j(g), or (B) every trial needed to approve that use has already been completed; (4) the trial sponsor is actively and diligently pursuing marketing approval for that use; (5) if (3)(A) applies, providing expanded access won't interfere with enrolling patients in the ongoing trials; (6) for serious diseases, there's enough evidence of safety and effectiveness to support the use; and (7) for immediately life-threatening diseases, the scientific evidence as a whole gives a reasonable basis to think the product may work, without exposing patients to an unreasonable, significant risk of illness or injury. Such a protocol must follow section 355(i) or 360j(g) and their regulations. The Secretary may tell national, state, and local medical associations and societies, voluntary health groups, and other appropriate people about available expanded-access protocols, using the same kind of information required under 42 U.S.C. 282(i)(3). (d) Termination The Secretary may end expanded access under this section, at any time, for any sponsor, physician, manufacturer, or distributor, if this section's requirements are no longer being met. (e) Definitions The terms "investigational drug," "investigational device," "treatment investigational new drug application," and "treatment investigational device exemption" mean whatever the Secretary's regulations say they mean; this section does not define them itself.