View all text of Part E [§ 360bbb - § 360bbb-8d]
§ 360bbb–8. Consultation with external experts on rare diseases, targeted therapies, and genetic targeting of treatments
(a) In generalFor the purpose of promoting the efficiency of and informing the review by the Food and Drug Administration of new drugs and biological products for rare diseases and drugs and biological products that are genetically targeted, the following shall apply:
(1) Consultation with stakeholders
(2) Consultation with external experts
(A) In general
(B) External experts
(b) Topics for consultationTopics for consultation pursuant to this section may include—
(1) rare diseases;
(2) the severity of rare diseases;
(3) the unmet medical need associated with rare diseases;
(4) the willingness and ability of individuals with a rare disease to participate in clinical trials;
(5) an assessment of the benefits and risks of therapies to treat rare diseases;
(6) the general design of clinical trials for rare disease populations and subpopulations;
(7) the demographics and the clinical description of patient populations; and
(8) the science of small population studies.
(c) Classification as special government employees
(d) Protection of confidential information and trade secrets
(1) Rule of construction
(2) Consent required for disclosure
(e) Other consultation
(f) No right or obligation
(1) No right to consultation
(2) No altering of goals
(3) No change to number of review cycles
(g) No delay in product review
(1) In generalPrior to a consultation with an external expert, as described in this section, relating to an investigational new drug application under section 355(i) of this title, a new drug application under section 355(b) of this title, or a biologics license application under section 262 of title 42, the Director of the Center for Drug Evaluation and Research or the Director of the Center for Biologics Evaluation and Research (or appropriate Division Director), as appropriate, shall determine that—
(A) such consultation will—
(i) facilitate the Secretary’s ability to complete the Secretary’s review; and
(ii) address outstanding deficiencies in the application; or
(B) the sponsor authorized such consultation.
(2) Limitation
(June 25, 1938, ch. 675, § 569, as added Pub. L. 112–144, title IX, § 903, July 9, 2012, 126 Stat. 1088; amended Pub. L. 114–255, div. A, title III, § 3101(a)(2)(O), Dec. 13, 2016, 130 Stat. 1154; Pub. L. 117–328, div. FF, title III, § 3202(e), Dec. 29, 2022, 136 Stat. 5812.)