21 U.S.C. § 360bbb–8 — Consultation with external experts on rare diseases, targeted therapies, and genetic targeting of treatments

(a) In general To help the FDA review new drugs and biological products for rare diseases, and drugs and biological products that are genetically targeted, more efficiently: (1) Consultation with stakeholders. Following goals set out in the PDUFA Reauthorization Performance Goals letters described in section 101(b) of the Prescription Drug User Fee Amendments of 2012, the Secretary must make sure there are chances, at appropriate times, to consult with stakeholders on the topics listed in subsection (b). (2) Consultation with external experts. (A) The Secretary must build and keep a list of outside experts qualified — because of their special knowledge — to advise on rare disease issues, including the topics in subsection (b). The Secretary may consult them on a specific regulatory question about reviewing rare-disease or gene-targeted drugs and biologics, but only when the FDA itself lacks the needed scientific, medical, or technical expertise and the outside expert can supply it. (B) "External experts" means people with scientific or medical training in one or more rare diseases that the Secretary doesn't have in-house. (b) Topics for consultation Consultations under this section can cover: (1) rare diseases generally; (2) how severe a rare disease is; (3) the unmet medical need tied to it; (4) whether people with the disease are willing and able to join clinical trials; (5) weighing the benefits and risks of therapies for it; (6) how to design clinical trials for rare-disease populations and subgroups; (7) the makeup and clinical description of the patient population; and (8) the science of studying small populations. (c) Classification as special government employees Experts consulted under this section can be treated as "special government employees," as section 202 of title 18 defines that term. (d) Protection of confidential information and trade secrets (1) Rule of construction. This section does not weaken the existing laws, regulations, and policies protecting confidential commercial or trade secret information, or other information exempt from disclosure under section 552(b) of title 5, as those protections applied to consultations with outside individuals and organizations before July 9, 2012. (2) Consent required for disclosure. The Secretary cannot share confidential commercial or trade secret information with an expert consulted under this section without the sponsor's written consent — unless that expert is a special government employee, as defined in section 202 of title 18, or unless some other law authorizes the disclosure. (e) Other consultation Nothing here limits the Secretary's ability to consult with individuals and organizations in ways that were already allowed before July 9, 2012. (f) No right or obligation (1) No right to consultation. This section doesn't give anyone a legal right to a consultation, and doesn't force the Secretary to meet with any particular expert or stakeholder. (2) No altering of goals. It doesn't change the goals and procedures already agreed to in the letters described in section 101(b) of the Prescription Drug User Fee Amendments of 2012. (3) No change to number of review cycles. It's not meant to increase the number of review cycles beyond what applied before July 9, 2012. (g) No delay in product review (1) In general. Before consulting an external expert about an investigational new drug application under section 355(i), a new drug application under section 355(b), or a biologics license application under section 262 of title 42, the Director of the Center for Drug Evaluation and Research or the Center for Biologics Evaluation and Research, or the relevant Division Director, must first decide either (A) that the consultation will help the FDA finish its review and will address specific problems with the application, or (B) that the sponsor itself agreed to the consultation. (2) Limitation. This requirement only applies when the consultation happens solely under this section's authority — not when it's done under some other legal authority.