For 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 Consistent with sections X.C and IX.E.4 of the PDUFA Reauthorization Performance Goals and Procedures Fiscal Years 2013 through 2017, as referenced in the letters described in section 101(b) of the Prescription Drug User Fee Amendments of 2012, the Secretary shall ensure that opportunities exist, at a time the Secretary determines appropriate, for consultations with stakeholders on the topics described in subsection (b).

The Secretary shall develop and maintain a list of external experts who, because of their special expertise, are qualified to provide advice on rare disease issues, including topics described in subsection (b). The Secretary may, when appropriate to address a specific regulatory question, consult such external experts on issues related to the review of new drugs and biological products for rare diseases and drugs and biological products that are genetically targeted, including the topics described in subsection (b), when such consultation is necessary because the Secretary lacks the specific scientific, medical, or technical expertise necessary for the performance of the Secretary’s regulatory responsibilities and the necessary expertise can be provided by the external experts.

(A) In general The Secretary shall develop and maintain a list of external experts who, because of their special expertise, are qualified to provide advice on rare disease issues, including topics described in subsection (b). The Secretary may, when appropriate to address a specific regulatory question, consult such external experts on issues related to the review of new drugs and biological products for rare diseases and drugs and biological products that are genetically targeted, including the topics described in subsection (b), when such consultation is necessary because the Secretary lacks the specific scientific, medical, or technical expertise necessary for the performance of the Secretary’s regulatory responsibilities and the necessary expertise can be provided by the external experts.

(B) External experts For purposes of subparagraph (A), external experts are individuals who possess scientific or medical training that the Secretary lacks with respect to one or more rare diseases.

Topics 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; and

(7) the demographics and the clinical description of patient populations.

The external experts who are consulted under this section may be considered special government employees, as defined under section 202 of title 18.

Nothing in this section shall be construed to alter the protections offered by laws, regulations, and policies governing disclosure of confidential commercial or trade secret information, and any other information exempt from disclosure pursuant to section 552(b) of title 5 as such provisions would be applied to consultation with individuals and organizations prior to July 9, 2012.

(1) Rule of construction Nothing in this section shall be construed to alter the protections offered by laws, regulations, and policies governing disclosure of confidential commercial or trade secret information, and any other information exempt from disclosure pursuant to section 552(b) of title 5 as such provisions would be applied to consultation with individuals and organizations prior to July 9, 2012.

(2) Consent required for disclosure The Secretary shall not disclose confidential commercial or trade secret information to an expert consulted under this section without the written consent of the sponsor unless the expert is a special government employee (as defined under section 202 of title 18) or the disclosure is otherwise authorized by law.

Nothing in this section shall be construed to limit the ability of the Secretary to consult with individuals and organizations as authorized prior to July 9, 2012.

Nothing in this section shall be construed to create a legal right for a consultation on any matter or require the Secretary to meet with any particular expert or stakeholder.

(1) No right to consultation Nothing in this section shall be construed to create a legal right for a consultation on any matter or require the Secretary to meet with any particular expert or stakeholder.

(2) No altering of goals Nothing in this section shall be construed to alter agreed upon goals and procedures identified 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 Nothing in this section is intended to increase the number of review cycles as in effect before July 9, 2012.

Prior 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—

Prior 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 The requirements of this subsection shall apply only in instances where the consultation is undertaken solely under the authority of this section. The requirements of this subsection shall not apply to any consultation initiated under any other authority.

(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.)