21 USC § 360bbb–8 - Consultation with external experts on rare diseases, targeted therapies, and genetic targeting of treatments

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This preliminary release may be subject to further revision before it is released again as a final version. As with other online versions of the Code, the U.S. Code Classification Tables should be consulted for the latest laws affecting the Code. Those using the USCPrelim should verify the text against the printed slip laws available from GPO (Government Printing Office), the laws as shown on THOMAS (a legislative service of the Library of Congress), and the final version of the Code when it becomes available.

Current through Pub. L. 112-173. (See Public Laws for the current Congress.)

(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 (c). 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; and

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

(c) Classification as special government employees

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

(d) Protection of confidential information and trade secrets

(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.

(e) Other consultation

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.

(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

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

(g) No delay in product review

(1) In general

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.

This preliminary release may be subject to further revision before it is released again as a final version. As with other online versions of the Code, the U.S. Code Classification Tables should be consulted for the latest laws affecting the Code. Those using the USCPrelim should verify the text against the printed slip laws available from GPO (Government Printing Office), the laws as shown on THOMAS (a legislative service of the Library of Congress), and the final version of the Code when it becomes available.

Current through Pub. L. 112-173. (See Public Laws for the current Congress.)

Source

(June 25, 1938, ch. 675, § 569, as added Pub. L. 112–144, title IX, § 903,July 9, 2012, 126 Stat. 1088.)

References in Text

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

The table below lists the classification updates, since Jan. 3, 2012, for this section. Updates to a broader range of sections may be found at the update page for containing chapter, title, etc.

The most recent Classification Table update that we have noticed was Wednesday, December 26, 2012

An empty table indicates that we see no relevant changes listed in the classification tables. If you suspect that our system may be missing something, please double-check with the Office of the Law Revision Counsel.

How To Use

Multiple entries for a section are listed most recent first, within the section.

The Session Year indicates which session of Congress was responsible for the changes classified. The Congress number forms the first part of the Public Law number; each Congress has two sessions.

Abbreviations used in the Description of Change column:

An empty field implies a standard amendment.
"new" means a new section or new note, or all new text of an existing section or note.
"nt" means note.
"nt [tbl]" means note [table].
"prec" means preceding.
"fr" means a transfer from another section.
"to" means a transfer to another section.
"omitted" means the section is omitted.
"repealed" means the section is repealed.
"nt ed change" and "ed change" - See the Editorial Classification Change Table [pdf].

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Sections with change type "new" are a special case, still under development. All are now listed, at the title level only.

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General Reference

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21 USC	Description of Change	Session Year	Public Law	Statutes at Large
§ 360bbb-8	new	2012	112-144 [Sec.] 903 "569"	126 Stat. 1088

21 USC § 360bbb–8 - Consultation with external experts on rare diseases, targeted therapies, and genetic targeting of treatments

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