HR 5334 IHCommentsClose CommentsPermalink

112th CONGRESSCommentsClose CommentsPermalink

2d SessionCommentsClose CommentsPermalink

H. R. 5334CommentsClose CommentsPermalink

To amend chapter V of the Federal Food, Drug, and Cosmetic Act to expedite the development and review of breakthrough therapies.CommentsClose CommentsPermalink

IN THE HOUSE OF REPRESENTATIVESCommentsClose CommentsPermalink

May 7, 2012CommentsClose CommentsPermalink

Mr. BILBRAY (for himself and Ms. DEGETTE) introduced the following bill; which was referred to the Committee on Energy and CommerceCommentsClose CommentsPermalink

A BILLCommentsClose CommentsPermalink

To amend chapter V of the Federal Food, Drug, and Cosmetic Act to expedite the development and review of breakthrough therapies.CommentsClose CommentsPermalink

Be it enacted by the Senate and House of Representatives of the United States of America in Congress assembled,CommentsClose CommentsPermalink

SECTION 1. BREAKTHROUGH THERAPIES.

(a) In General- Section 506 (21 U.S.C. 356) is amended--CommentsClose CommentsPermalink





(1) by redesignating subsection (d) as subsection (f);CommentsClose CommentsPermalink





(2) by redesignating subsections (a) through (c) as subsections (b) through (d), respectively;CommentsClose CommentsPermalink





(3) by inserting before subsection (b), as so redesignated, the following:CommentsClose CommentsPermalink





‘(a) Designation of a Drug as a Breakthrough Therapy-CommentsClose CommentsPermalink





‘(1) IN GENERAL- The Secretary shall, at the request of the sponsor of a drug, expedite the development and review of such drug if the drug is intended, alone or in combination with 1 or more other drugs, to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on 1 or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. (In this section, such a drug is referred to as a ‘breakthrough therapy’.)CommentsClose CommentsPermalink





‘(2) REQUEST FOR DESIGNATION- The sponsor of a drug may request the Secretary to designate the drug as a breakthrough therapy. A request for the designation may be made concurrently with, or at any time after, the submission of an application for the investigation of the drug under section 505(i) or section 351(a)(3) of the Public Health Service Act.CommentsClose CommentsPermalink





‘(3) DESIGNATION-CommentsClose CommentsPermalink





‘(A) IN GENERAL- Not later than 60 calendar days after the receipt of a request under paragraph (2), the Secretary shall determine whether the drug that is the subject of the request meets the criteria described in paragraph (1). If the Secretary finds that the drug meets the criteria, the Secretary shall designate the drug as a breakthrough therapy and shall take such actions as are appropriate to expedite the development and review of the application for approval of such drug.CommentsClose CommentsPermalink





‘(B) ACTIONS- The actions to expedite the development and review of an application under subparagraph (A) may include, as appropriate--CommentsClose CommentsPermalink





‘(i) holding meetings with the sponsor and the review team throughout the development of the drug;CommentsClose CommentsPermalink





‘(ii) providing timely advice to, and interactive communication with, the sponsor regarding the development of the drug to ensure that the development program to gather the non-clinical and clinical data necessary for approval is as efficient as practicable;CommentsClose CommentsPermalink





‘(iii) involving senior managers and experienced review staff, as appropriate, in a collaborative, cross-disciplinary review;CommentsClose CommentsPermalink





‘(iv) assigning a cross-disciplinary project lead for the Food and Drug Administration review team to facilitate an efficient review of the development program and to serve as a scientific liaison between the review team and the sponsor; andCommentsClose CommentsPermalink





‘(v) taking steps to ensure that the design of the clinical trials is as efficient as practicable, when scientifically appropriate, such as by minimizing the number of patients exposed to a potentially less efficacious treatment.’;CommentsClose CommentsPermalink





(4) in subsection (f)(1), as so redesignated, by striking ‘applicable to accelerated approval’ and inserting ‘applicable to breakthrough therapies, accelerated approval, and’; andCommentsClose CommentsPermalink





(5) by adding at the end the following:CommentsClose CommentsPermalink





‘(g) Report- Beginning in fiscal year 2013, the Secretary shall annually prepare and submit to the Committee on Health, Education, Labor, and Pensions of the Senate and the Committee on Energy and Commerce of the House of Representatives, and make publicly available, with respect to this section for the previous fiscal year--CommentsClose CommentsPermalink





‘(1) the number of drugs for which a sponsor requested designation as a breakthrough therapy; andCommentsClose CommentsPermalink





‘(2) the number of products designated as a breakthrough therapy.’.CommentsClose CommentsPermalink





(b) Guidance; Amended Regulations-CommentsClose CommentsPermalink





(1) IN GENERAL-CommentsClose CommentsPermalink





(A) GUIDANCE- Not later than 18 months after the date of enactment of this Act, the Secretary of Health and Human Services (referred to in this section as the ‘Secretary’) shall issue draft guidance on implementing the requirements with respect to breakthrough therapies, as set forth in section 506(a) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 356(a)), as amended by this section. The Secretary shall issue final guidance not later than 1 year after the close of the comment period for the draft guidance.CommentsClose CommentsPermalink





(B) AMENDED REGULATIONS-CommentsClose CommentsPermalink





(i) IN GENERAL- If the Secretary determines that it is necessary to amend the regulations under title 21, Code of Federal Regulations in order to implement the amendments made by this section to section 506(a) of the Federal Food, Drug, and Cosmetic Act, the Secretary shall amend such regulations not later than 2 years after the date of enactment of this Act.CommentsClose CommentsPermalink





(ii) PROCEDURE- In amending regulations under clause (i), the Secretary shall--CommentsClose CommentsPermalink





(I) issue a notice of proposed rulemaking that includes the proposed regulation;CommentsClose CommentsPermalink





(II) provide a period of not less than 60 days for comments on the proposed regulation; andCommentsClose CommentsPermalink





(III) publish the final regulation not less than 30 days before the effective date of the regulation.CommentsClose CommentsPermalink





(iii) RESTRICTIONS- Notwithstanding any other provision of law, the Secretary shall promulgate regulations implementing the amendments made by this section only as described in clause (ii).CommentsClose CommentsPermalink





(2) REQUIREMENTS- Guidance issued under this section shall--CommentsClose CommentsPermalink





(A) specify the process and criteria by which the Secretary makes a designation under section 506(a)(3) of the Federal Food, Drug, and Cosmetic Act; andCommentsClose CommentsPermalink





(B) specify the actions the Secretary shall take to expedite the development and review of a breakthrough therapy pursuant to such designation under such section 506(a)(3), including updating good review management practices to reflect breakthrough therapies.CommentsClose CommentsPermalink





(c) Independent Review- Not later than 3 years after the date of enactment of this Act, the Comptroller General of the United States, in consultation with appropriate experts, shall assess the manner by which the Food and Drug Administration has applied the processes described in section 506(a) of the Federal Food, Drug, and Cosmetic Act, as amended by this section, and the impact of such processes on the development and timely availability of innovative treatments for patients affected by serious or life-threatening conditions. Such assessment shall be made publicly available upon completion.CommentsClose CommentsPermalink





(d) Conforming Amendments- Section 506B(e) (21 U.S.C. 356b) is amended by striking ‘section 506(b)(2)(A)’ each place such term appears and inserting ‘section 506(c)(2)(A)’.CommentsClose CommentsPermalink