21 U.S.C. § 360aa — Recommendations for investigations of drugs for rare diseases or conditions

(a) Request by sponsor; response by Secretary The sponsor of a drug meant for a disease or condition that is rare in the United States can ask the Secretary for written recommendations. The sponsor wants to know what non-clinical investigations, like lab and animal studies, and what clinical investigations, meaning studies in people, the drug needs before it can be approved under section 355, or, if it is a biological product, licensed under section 262 of title 42. If the Secretary has reason to believe the drug really is for a disease or condition that is rare in the United States, the Secretary must give the sponsor written recommendations. Those recommendations cover the non-clinical and clinical investigations the Secretary believes, based on the information the Secretary has at the time of the request, would be necessary for approval of the drug for that disease or condition under section 355, or for licensing it under section 262 of title 42. (b) Regulations The Secretary must issue regulations laying out the procedures for carrying out subsection (a).