A bill to provide for the expedited development and evaluation of drugs designated as breakthrough drugs.

SECTION 1. SHORT TITLE.

    This Act may be cited as the ``Advancing Breakthrough Therapies for 
Patients Act of 2012''.

SEC. 2. BREAKTHROUGH THERAPIES AND FAST TRACK PRODUCTS.

    (a) In General.--Section 506 of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 356) is amended--
            (1) in the heading, by inserting ``breakthrough therapies 
        and'' before ``fast'';
            (2) by redesignating subsections (a) through (d) as 
        subsections (b) through (e), 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) shall include--
                            ``(i) holding meetings with the sponsor and 
                        the review team throughout the development of 
                        the drug;
                            ``(ii) providing timely advice to 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) providing timely interactive 
                        communication with sponsors;
                            ``(v) 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
                            ``(vi) 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 enrolled in the trial and the 
                        duration of the trial and considering 
                        alternatives to the traditional multi-phase, 
                        sequential development approach, designed to 
                        abbreviate, consolidate, and condense clinical 
                        trials and studies.'';
            (4) in subsection (e)(1), as so redesignated, by inserting 
        ``breakthrough therapies and'' after ``applicable to''; and
            (5) by adding at the end the following:
    ``(f) Guidance; Amended Regulations.--
            ``(1) In general.--
                    ``(A) Guidance.--Not later than 18 months after the 
                date of enactment of the Advancing Breakthrough 
                Therapies for Patients Act of 2012, the Secretary shall 
                issue draft guidance on implementing the requirements 
                with respect to breakthrough therapies, accelerated 
                approval, and fast track products as set forth in 
                subsections (a) through (c), as amended by the 
                Advancing Breakthrough Therapies for Patients Act of 
                2012. After an opportunity for public comment and not 
                later than 2 years after the date of enactment of the 
                Advancing Breakthrough Therapies for Patients Act of 
                2012, the Secretary shall issue final guidance.
                    ``(B) Amended regulations.--Not later than 2 years 
                after the date of enactment of the Advancing 
                Breakthrough Therapies for Patients Act of 2012, the 
                Secretary shall amend the applicable regulations under 
                title 21, Code of Federal Regulations, as may be 
                necessary to implement the requirements under 
                subsections (a) through (c), as amended by the 
                Advancing Breakthrough Therapies for Patients Act of 
                2012.
            ``(2) Requirements.--Guidance and regulations promulgated 
        under this section shall--
                    ``(A) distinguish between products that may qualify 
                for--
                            ``(i) treatment as a breakthrough therapy;
                            ``(ii) treatment as a fast track product;
                            ``(iii) accelerated approval; and
                            ``(iv) a combination of all of the 
                        designations described in clauses (i) through 
                        (iii); and
                    ``(B) specify the actions the Secretary shall take 
                to expedite the development and review of a 
                breakthrough therapy pursuant to such designation under 
                506(a)(3), including updating good review management 
                practices to reflect breakthrough therapies.
    ``(g) Independent Review.--Not later than 3 years after the date of 
enactment of this Act, the Secretary shall, in conjunction with other 
planned reviews, contract with an independent entity with expertise in 
assessing the quality, efficiency, and predictability of 
biopharmaceutical development and regulatory review programs to 
evaluate the manner by which the Food and Drug Administration has 
applied the processes described in the section, as amended by the 
Advancing Breakthrough Therapies for Patients Act of 2012, 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 evaluation shall be completed not later 
than 4 years after the date of enactment of the Advancing Breakthrough 
Therapies for Patients Act of 2012 and shall be made publicly available 
upon completion.
    ``(h) 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;
            ``(2) the number of products designated as a breakthrough 
        therapy; and
            ``(3) for each breakthrough therapy approved in the fiscal 
        year--
                    ``(A) the point in the drug development and review 
                process at which such breakthrough designation 
                occurred;
                    ``(B) the total time from designation as a 
                breakthrough therapy, including the total time to 
                review and act on an application designated as a 
                breakthrough therapy, to approval of the drug; and
                    ``(C) the number of breakthrough therapies approved 
                on the first review out of the total number of such 
                therapies so approved.''.
    (b) Conforming Amendments.--Section 506B(e) of the Federal Food, 
Drug, and Cosmetic Act (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: Advancing Breakthrough Therapies for Patients Act of 2012 - 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.

Directs the Secretary to issue guidance on implementing requirements with respect to breakthrough therapies and to amend promulgated regulations.

Requires the Secretary to contract with an independent entity to evaluate the manner by which the Food and Drug Administration (FDA) has applied the processes for the breakthrough therapy determination, and the impact of such processes on the development and timely availability of innovative treatments for patients affected by serious or life-threatening conditions. Requires an annual report from the Secretary to Congress on drugs for which breakthrough designations were requested and approved.