[Congressional Bills 113th Congress]
[From the U.S. Government Publishing Office]
[H.R. 5805 Introduced in House (IH)]

113th CONGRESS
  2d Session
                                H. R. 5805

   To amend the Federal Food, Drug, and Cosmetic Act with respect to 
    expanding access for breakthrough drugs, and for other purposes.


_______________________________________________________________________


                    IN THE HOUSE OF REPRESENTATIVES

                            December 8, 2014

  Mr. McCaul introduced the following bill; which was referred to the 
                    Committee on Energy and Commerce

_______________________________________________________________________

                                 A BILL


 
   To amend the Federal Food, Drug, and Cosmetic Act with respect to 
    expanding access for breakthrough drugs, and for other purposes.

    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 ``Andrea Sloan Compassionate Use 
Reform and Enhancement Act'' or the ``Andrea Sloan CURE Act''.

SEC. 2. EXPANDED ACCESS POLICY AS CONDITION OF EXPEDITED APPROVAL.

    Section 561 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
360bbb) is amended--
            (1) by redesignating subsections (d) and (e) as subsections 
        (e) and (f), respectively; and
            (2) by inserting after subsection (c) the following new 
        subsection:
    ``(d) Expanded Access Policy Required for Covered Breakthrough 
Drugs.--
            ``(1) In general.--With respect to a qualified breakthrough 
        drug, not later than 30 days after the date on which the drug 
        meets the definition of a covered breakthrough drug (as 
        specified in paragraph (2)), the sponsor of the covered 
        breakthrough drug shall submit to the Secretary and make 
        publicly available the policy of the sponsor with respect to 
        requests submitted under subsection (b). In the case of such a 
        policy under which the sponsor accepts such requests, such 
        policy shall include--
                    ``(A) a single point of contact who receives and 
                processes such requests;
                    ``(B) procedures for making such requests;
                    ``(C) the minimum criteria for the sponsor's 
                consideration or approval of such requests; and
                    ``(D) the amount of time the sponsor anticipates 
                will be necessary to make a decision on such requests.
            ``(2) Covered breakthrough drug.--In this subsection, the 
        term `covered breakthrough drug' means a drug--
                    ``(A) that is designated as a breakthrough therapy 
                or as a fast track product or is approved under 
                accelerated approval under section 506;
                    ``(B) that is designated under section 505E(d) as a 
                qualified infectious disease product; or
                    ``(C) the sponsor of which is awarded a priority 
                review voucher under section 524 or 529.''.

SEC. 3. NOTIFICATION OF SUBMITTERS OF COMPASSIONATE USE REQUESTS.

    Section 561 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
360bbb), as amended by section 2, is further amended--
            (1) by redesignating subsections (e) and (f) (as 
        redesignated by section 2(1)) as subsections (f) and (g), 
        respectively; and
            (2) by inserting after subsection (d) (as inserted by 
        section 2(2)) the following new subsection:
    ``(e) Notification of Submitters of Requests.--In the case of the 
denial by a manufacturer or distributor of a request under subsection 
(b), not later than 5 days after the date of such denial, the 
manufacturer or distributor, as applicable, shall submit to the person 
(or physician) who made the request written notice of the denial, 
including an explanation for the denial.''.

SEC. 4. GAO QUALITATIVE ANALYSIS ON INDIVIDUAL PATIENT ACCESS TO 
              UNAPPROVED THERAPIES AND DIAGNOSTICS.

    Not later than 180 days after the date of the enactment of this Act 
and each year thereafter, the Comptroller General of the United States 
shall submit to the Committee on Energy and Commerce of the House of 
Representatives and the Committee on Health, Education, Labor and 
Pensions of the Senate a report containing a qualitative analysis of 
the extent to which individual patients have access to investigational 
drugs pursuant to subsection (b) of section 561 of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 360bbb) and recommendations for 
improving such access. In preparing such report, the Comptroller 
General shall conduct a qualitative analysis of the following:
            (1) Whether there are any identifiable patterns in requests 
        submitted under subsection (b) of such section, such as the 
        types of indications for which requests for individual patient 
        access are sought or the reasons for the denial of such 
        requests.
            (2) What the primary barriers are to drug sponsors granting 
        requests for individual patient access.
            (3) How the Secretary evaluates safety and efficacy data 
        submitted in connection with such requests.
            (4) The amount of time that--
                    (A) a physician typically takes to complete the 
                paperwork necessary to make such a request;
                    (B) a drug sponsor takes to process such a request 
                and to issue a decision with respect to the request; 
                and
                    (C) the Secretary takes to process such a request 
                and to issue a decision with respect to the request.
            (5) How regulations, guidance, policies, or practices may 
        be modified, streamlined, expanded, or discontinued to reduce 
        or prevent delays in approving such requests.
            (6) The number of such requests that, for the period 
        covered by the report--
                    (A) were approved by drug sponsors and the Food and 
                Drug Administration;
                    (B) were approved by drug sponsors but denied by 
                the Food and Drug Administration; and
                    (C) were denied by drug sponsors.
            (7) How to encourage drug sponsors to grant requests for 
        expanded access under such section 561, including requests for 
        emergency use, intermediate-size patient populations, and large 
        patient populations under a specified indication.
            (8) Whether and to what extent adverse events reported to 
        the Secretary as a result of individual use of an 
        investigational drug or investigational device under such 
        section 561 affected the development or approval of any drug or 
        device.

SEC. 5. EXPANDED ACCESS TASK FORCE.

    (a) Establishment.--The Secretary of Health and Human Services 
shall establish a task force within the Department of Health and Human 
Services to explore mechanisms for improving the access individual 
patients have to investigational drugs pursuant to subsection (b) of 
section 561 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
360bbb), to be known as the ``Expanded Access Task Force'' (in this 
section referred to as the ``Task Force''). Not later than 90 days 
after the date on which the Comptroller General of the United States 
submits the first report required under section 4, the Task Force shall 
be convened.
    (b) Membership.--
            (1) Composition.--The Task Force shall be composed of not 
        more than 9 voting members appointed as follows:
                    (A) One member to serve as Chairman of the Task 
                Force, appointed by the Speaker of the House of 
                Representatives.
                    (B) One representative from the Department of 
                Health and Human Services, appointed by the Secretary 
                of Health and Human Services.
                    (C) Four representatives appointed by the Majority 
                Leader of the House of Representatives, in consultation 
                with the Minority Leader of the House of 
                Representatives, and the Chairman and the Ranking 
                Member of the Committee on Energy and Commerce of the 
                House of Representatives, including--
                            (i) one representative of a 
                        biopharmaceutical company of less than 250 
                        full-time employees;
                            (ii) one representative of the rare disease 
                        patient community;
                            (iii) one representative of the health care 
                        provider community; and
                            (iv) one bioethicist.
                    (D) Three representatives appointed by Majority 
                Leader of the Senate, in consultation with the Minority 
                Leader of the Senate, and the Chairman and the Ranking 
                Member of the Health, Education, Labor and Pensions 
                Committee of the Senate, including--
                            (i) one representative of the 
                        biopharmaceutical industry;
                            (ii) one representative of the patient 
                        community; and
                            (iii) one representative of the health care 
                        payor community.
            (2) Compensation.--Members of the Task Force shall serve 
        without compensation.
    (c) Duties.--The Task Force shall comprehensively evaluate the 
access individual patients have to investigational drugs pursuant to 
subsection (b) of section 561 of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 360bbb), taking into account--
            (1) the unique challenges faced by children with likely 
        fatal diseases for which there is not a comparable or 
        satisfactory alternative therapy available;
            (2) possible incentives for biopharmaceutical companies and 
        providers to approve requests submitted under such subsection;
            (3) how the Secretary of Health and Human Services 
        interprets and takes into consideration adverse event data 
        reported in the case of data from use under a request submitted 
        under such subsection;
            (4) ways to streamline and standardize the process for 
        submitting requests under such subsection; and
            (5) the costs incurred by biopharmaceutical companies for 
        the time, effort, and delivery of investigational drugs to 
        patients for the diagnosis, monitoring, or treatment of a 
        serious disease or condition under such subsection.
    (d) Report.--Not later than 180 days after the date on which the 
Task Force is convened, the Task Force shall submit to the Committee on 
Energy and Commerce of the House of Representatives and the Committee 
on Health, Education, Labor and Pensions of the Senate a report in an 
electronic format describing the specific recommendations of the Task 
Force for improving the access individual patients have to 
investigational drugs pursuant to subsection (b) of section 561 of the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb).
    (e) Termination.--The task force shall terminate upon submission of 
the report required under subsection (d).

SEC. 6. FINALIZING DRAFT GUIDANCE ON EXPANDED ACCESS.

    (a) In General.--Not later than 180 days after the date on which 
the Expanded Access Task Force established under section 5 submits the 
report under subsection (d) of such section, the Secretary of Health 
and Human Services shall finalize the draft guidance entitled 
``Expanded Access to Investigational Drugs for Treatment Use--Qs & As'' 
and dated May 2013.
    (b) Contents.--The final guidance referred to in subsection (a) 
shall--
            (1) clearly define how the Secretary interprets and uses 
        adverse drug event data reported by investigators in the case 
        of data reported from use under a request submitted under 
        section 561(b) of the Federal Food, Drug, and Cosmetic Act (21 
        U.S.C. 360bbb(b)); and
            (2) take into account the report of the Expanded Access 
        Task Force submitted under section 5(d) and the first report of 
        the Comptroller General of the United States submitted under 
        section 4.
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