article: SECTION 1. BREAKTHROUGH THERAPIES. (a) In General.--Section 506 (21 U.S.C. 356) is amended-- (1) by redesignating subsection (d) as subsection (f); (2) by redesignating subsections (a) through (c) as subsections (b) through (d), respectively; (3) by inserting before subsection (b), as so redesignated, the following: ``(a) Designation of a Drug as a Breakthrough Therapy.-- ``(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'.) ``(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. ``(3) Designation.-- ``(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. ``(B) Actions.--The actions to expedite the development and review of an application under subparagraph (A) may include, as appropriate-- ``(i) holding meetings with the sponsor and the review team throughout the development of the drug; ``(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; ``(iii) involving senior managers and experienced review staff, as appropriate, in a collaborative, cross-disciplinary review; ``(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; and ``(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.''; (4) in subsection (f)(1), as so redesignated, by striking ``applicable to accelerated approval'' and inserting ``applicable to breakthrough therapies, accelerated approval, and''; and (5) by adding at the end the following: ``(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-- ``(1) the number of drugs for which a sponsor requested designation as a breakthrough therapy; and ``(2) the number of products designated as a breakthrough therapy.''. (b) Guidance; Amended Regulations.-- (1) In general.-- (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. (B) Amended regulations.-- (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. (ii) Procedure.--In amending regulations under clause (i), the Secretary shall-- (I) issue a notice of proposed rulemaking that includes the proposed regulation; (II) provide a period of not less than 60 days for comments on the proposed regulation; and (III) publish the final regulation not less than 30 days before the effective date of the regulation. (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). (2) Requirements.--Guidance issued under this section shall-- (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; and (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. (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. (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)''.
summary: Amends the Federal Food, Drug, and Cosmetic Act to direct the Secretary of Health and Human Services (HHS), at the request of the sponsor of a drug, to expedite the drug's development and review if: (1) it is intended, either alone or in combination, to treat a serious life-threatening disease or condition; and (2) preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. Authorizes the drug's sponsor to request the Secretary to designate the drug as a breakthrough therapy. Requires the Secretary, within 60 days of such request, to determine whether the drug meets such criteria and, if so, make such designation, followed by appropriate actions to expedite its development and review for approval. Requires an annual report from the Secretary to Congress on drugs for which breakthrough designations were requested and approved. Directs the Secretary to issue guidance on implementing requirements with respect to breakthrough therapies and to amend promulgated regulations. Requires the Comptroller General to assess the manner by which the Food and Drug Administration (FDA) has applied the processes established by this Act and their impact on the development and timely availability of innovative treatments for patients affected by serious or life-threatening conditions.