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

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.