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

(a)

In general

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).

(2)

Consultation with 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.

(b)

Topics for consultation

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;

(7)

the demographics and the clinical description of patient populations; and

(8)

the science of small population studies.

(c)

Classification as special government employees

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

(d)

Protection of confidential information and trade secrets

(1)

Rule of construction

section 552(b) of title 5July 9, 2012Nothing 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 as such provisions would be applied to consultation with individuals and organizations prior to .

(2)

Consent required for disclosure

section 202 of title 18The 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 ) or the disclosure is otherwise authorized by law.

(e)

Other consultation

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

(f)

No right or obligation

(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

July 9, 2012Nothing in this section is intended to increase the number of review cycles as in effect before .

(g)

No delay in product review

(1)

In general

section 355(i) of this title section 355(b) of this title section 262 of title 42Prior to a consultation with an external expert, as described in this section, relating to an investigational new drug application under , a new drug application under , or a biologics license application under , 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 Pub. L. 112–144, title IX, § 903July 9, 2012126 Stat. 1088 Pub. L. 114–255, div. A, title III, § 3101(a)(2)(O)Dec. 13, 2016130 Stat. 1154 Pub. L. 117–328, div. FF, title III, § 3202(e)Dec. 29, 2022136 Stat. 5812(, as added , , ; amended , , ; , , .)

Editorial Notes

References in Text

section 101(b) of Pub. L. 112–144 section 379g of this titleSection 101(b) of the Prescription Drug User Fee Amendments of 2012, referred to in subsecs. (a)(1) and (f)(2), is , which is set out as a note under .

Amendments

Pub. L. 117–3282022—Subsec. (b)(8). added par. (8).

Pub. L. 114–2552016—Subsec. (a)(2)(A). substituted “subsection (b)” for “subsection (c)” before period in first sentence.