[Congressional Bills 112th Congress]
[From the U.S. Government Publishing Office]
[H.R. 4132 Introduced in House (IH)]

112th CONGRESS
  2d Session
                                H. R. 4132

  To amend section 506 of the Federal Food, Drug, and Cosmetic Act to 
expedited approval of drugs for serious or life-threatening diseases or 
                              conditions.


_______________________________________________________________________


                    IN THE HOUSE OF REPRESENTATIVES

                             March 5, 2012

Mr. Stearns (for himself and Mr. Towns) introduced the following bill; 
       which was referred to the Committee on Energy and Commerce

_______________________________________________________________________

                                 A BILL


 
  To amend section 506 of the Federal Food, Drug, and Cosmetic Act to 
expedited approval of drugs for serious or life-threatening diseases or 
                              conditions.

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

SECTION 1. SHORT TITLE.

    This Act may be cited as the ``Faster Access to Specialized 
Treatments Act'' or ``FAST Act''.

SEC. 2. FINDINGS; SENSE OF CONGRESS.

    (a) Findings.--Congress finds the following:
            (1) The Food and Drug Administration (FDA) serves a 
        critical role in helping to assure that new medicines are safe 
        and effective. Regulatory innovation is one element of the 
        Nation's strategy to address serious and life-threatening 
        diseases or conditions by promoting investment in and 
        development of innovative treatments for unmet medical needs.
            (2) Over the previous two decades, since the accelerated 
        approval mechanism was established, advances in medical 
        sciences, including genomics, molecular biology, and 
        bioinformatics, have provided an unprecedented understanding of 
        the underlying biological mechanism and pathogenesis of 
        disease. A new generation of modern, targeted medicines is 
        currently under development to treat serious and life-
        threatening diseases, some applying drug development strategies 
        based on biomarkers or pharmacogenomics, predictive toxicology, 
        clinical trial enrichment techniques, and novel clinical trial 
        designs, such as adaptive clinical trials.
            (3) As a result of these remarkable scientific and medical 
        advances, FDA should be encouraged to implement more broadly 
        effective processes for the expedited development and review of 
        innovative new medicines intended to address unmet medical 
        needs for serious or life-threatening diseases or conditions, 
        including those for rare diseases or conditions, using a broad 
        range of surrogate or clinical endpoints and modern scientific 
        tools earlier in the drug development cycle when appropriate. 
        This may result in fewer, smaller, or shorter clinical trials 
        for the intended patient population or targeted subpopulation 
        without compromising or altering FDA's existing high standards 
        for the approval of drugs.
            (4) Patients benefit from expedited access to safe and 
        effective innovative therapies to treat unmet medical needs for 
        serious or life-threatening diseases or conditions.
            (5) For these reasons, the existing statutory authority 
        governing expedited approval of drugs or serious or life-
        threatening conditions should be amended in order to enhance 
        FDA's authority to consider appropriate scientific data, 
        methods, and tools, and to expedite development and access to 
        novel treatments for patients with a broad range oofserious or 
        life-threatening diseases or conditions.
    (b) Sense of Congress.--It is the sense of Congress that the Food 
and Drug Administration should apply the accelerated approval and the 
fast track provisions set forth in section 506 of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 356), as amended by section 3, to the 
greatest extent possible to help expedite the development and 
availability to patients of treatments for serious or life-threatening 
diseases or conditions while maintaining appropriate safety and 
effectiveness standards for such treatments.

SEC. 3. EXPEDITED APPROVAL OF DRUGS FOR SERIOUS OR LIFE-THREATENING 
              DISEASES OR CONDITIONS.

    Section 506 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
356) is amended to read as follows:

``SEC. 506. EXPEDITED APPROVAL OF DRUGS FOR SERIOUS OR LIFE-THREATENING 
              DISEASES OR CONDITIONS.

    ``(a) Designation of Drug as a Fast Track Product.--
            ``(1) In general.--The Secretary shall, at the request of 
        the sponsor of a new drug, facilitate the development and 
        expedite the review of such drug if it is intended, whether 
        alone or in combination with one or more other drugs, for the 
        treatment of a serious or life-threatening disease or 
        condition, and it demonstrates the potential to address unmet 
        medical needs for such a disease or condition. (In this 
        section, such a drug is referred to as a `fast track product'.)
            ``(2) Request for designation.--The sponsor of a new drug 
        may request the Secretary to designate the drug as a fast track 
        product. A request for the designation may be made concurrently 
        with, or at any time after, 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.--Within 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 fast track product and shall take such 
        actions as are appropriate to expedite the development and 
        review of the application for approval of such product.
    ``(b) Accelerated Approval of a Drug for a Serious or Life-
Threatening Disease or Condition, Including a Fast Track Product.--
            ``(1) In general.--The Secretary may approve an application 
        for approval of a product for a serious or life-threatening 
        disease or condition, including a fast track product, under 
        section 505(c) or section 351(a) of the Public Health Service 
        Act upon making a determination (taking into account the 
        severity or rarity of the disease or condition and the 
        availability of alternative treatments) that the product has an 
        effect on--
                    ``(A) a surrogate endpoint that is reasonably 
                likely to predict clinical benefit; or
                    ``(B) a clinical endpoint, including an endpoint 
                that can be measured earlier than irreversible 
                morbidity or mortality, that is reasonably likely to 
                predict an effect on irreversible morbidity or 
                mortality or other clinical benefit.
        The evidence to support that an endpoint is reasonably likely 
        to predict clinical benefit may include epidemiological, 
        pathophysiologic, pharmacologic, therapeutic or other evidence 
        developed using, for example, biomarkers, or other scientific 
        methods or tools.
            ``(2) Limitation.--Approval of a product under this 
        subsection may, as determined by the Secretary, be subject to 
        the following requirements--
                    ``(A) that the sponsor conduct appropriate post-
                approval studies to verify and describe the predicted 
                effect of the product on irreversible morbidity or 
                mortality or other clinical benefit; and
                    ``(B) that the sponsor submit copies of all 
                promotional materials related to the product, at least 
                30 days prior to dissemination of the materials 
                during--
                            ``(i) the preapproval review period; and
                            ``(ii) following approval, for a period 
                        that the Secretary determines to be 
                        appropriate.
            ``(3) Expedited withdrawal of approval.--The Secretary may 
        withdraw approval of a product approved pursuant to this 
        subsection using expedited procedures (as prescribed by the 
        Secretary in regulations, which shall include an opportunity 
        for an informal hearing) if--
                    ``(A) the sponsor fails to conduct any required 
                post-approval study of the product with due diligence;
                    ``(B) a study required to verify and describe the 
                predicted effect on irreversible morbidity or mortality 
                or other clinical benefit of the product fails to 
                verify and describe such effect or benefit;
                    ``(C) other evidence demonstrates that the product 
                is not safe or effective under the conditions of use; 
                or
                    ``(D) the sponsor disseminates false or misleading 
                promotional materials with respect to the product.
    ``(c) Review of Incomplete Applications for Approval of a Fast 
Track Product.--
            ``(1) In general.--If the Secretary determines, after 
        preliminary evaluation of clinical data submitted by the 
        sponsor, that a fast track product may be effective, the 
        Secretary shall evaluate for filing, and may commence review of 
        portions of, an application for the approval of the product 
        before the sponsor submits a complete application. The 
        Secretary shall commence such review only if the applicant--
                    ``(A) provides a schedule for submission of 
                information necessary to make the application complete; 
                and
                    ``(B) pays any fee that may be required under 
                section 736.
            ``(2) Exception.--Any time period for review of human drug 
        applications that has been agreed to by the Secretary and that 
        has been set forth in goals identified in letters of the 
        Secretary (relating to the use of fees collected under section 
        736 to expedite the drug development process and the review of 
        human drug applications) shall not apply to an application 
        submitted under paragraph (1) until the date on which the 
        application is complete.
    ``(d) Awareness Efforts.--The Secretary shall--
            ``(1) develop and disseminate to physicians, patient 
        organizations, pharmaceutical and biotechnology companies, and 
        other appropriate persons a description of the provisions of 
        this section applicable to accelerated approval and fast track 
        products; and
            ``(2) establish a program to encourage the development of 
        surrogate and clinical endpoints, including biomarkers, and 
        other scientific methods and tools that can assist the 
        Secretary in determining whether the evidence submitted in an 
        application is reasonably likely to predict clinical benefit 
        for serious or life-threatening conditions for which there 
        exist significant unmet medical needs.''.

SEC. 4. GUIDANCE; AMENDED REGULATIONS.

    (a) Initial Guidance.--Not later than one year after the date of 
enactment of this Act, the Secretary of Health and Human Services 
(hereinafter ``the Secretary'') shall issue draft guidance to implement 
the amendments made by section 3.
    (b) Final Guidance.--Not later than one year after the issuance of 
draft guidance under subsection (a), after an opportunity for public 
comment, the Secretary shall issue--
            (1) final guidance to implement the amendments made by 
        section 3; and
            (2) amend the regulations governing accelerated approval in 
        parts 314 and 601 of title 21, Code of Federal Regulations, as 
        necessary to conform such regulations with the amendments made 
        by section 3.
    (c) Considerations.--In developing the guidance under subsections 
(a) and (b)(1) and the amendments under subsection (b)(2), the 
Secretary shall consider--
            (1) issues arising under the accelerated approval and fast 
        track processes under section 506 of the Federal Food, Drug, 
        and Cosmetic Act (as amended by section 3) for drugs designated 
        for a rare disease or condition under section 526 of the 
        Federal, Food, Drug, and Cosmetic Act; and
            (2) how to incorporate novel approaches to the review of 
        surrogate endpoints based on pathophysiologic and pharmacologic 
        evidence in such guidance, especially in instances where the 
        low prevalence of a disease renders the existence or collection 
        of other types of data unlikely or impractical.
    (d) No Delay in Review or Approval.--The issuance (or non-issuance) 
of guidance or conforming regulations implementing the amendments made 
by section 3 shall not preclude the review of, or action on, a request 
for designation or an application for approval submitted pursuant to 
section 506 of the Federal Food, Drug, and Cosmetic Act, as amended by 
section 3.

SEC. 5. INDEPENDENT REVIEW.

    (a) In General.--The Secretary shall, in conjunction with other 
planned reviews of the new drug review process, contract with an 
independent entity with expertise in assessing the quality and 
efficiency of biopharmaceutical development and regulatory review 
programs, to evaluate the Food and Drug Administration's application of 
the processes described in section 506 of the Federal Food, Drug, and 
Cosmetic Act, as amended by section 3, and the impact of such processes 
on the development and timely availability of innovative treatments for 
patients suffering from serious or life-threatening conditions.
    (b) Consultation.--Any evaluation under subsection (a) shall 
include consultation with regulated industries, patient advocacy and 
disease research foundations, and relevant academic medical centers.

SEC. 6. RULE OF CONSTRUCTION.

    The amendments made to section 506(b) of the Federal Food, Drug and 
Cosmetic Act by this Act shall be construed in a manner that encourages 
the Secretary to utilize innovative approaches for the assessment of 
products under accelerated approval while maintaining appropriate 
safety and effectiveness standards for such products.
                                 <all>