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[Congressional Bills 115th Congress]
[From the U.S. Government Publishing Office]
[S. 1048 Introduced in Senate (IS)]

<DOC>

115th CONGRESS
  1st Session
                                S. 1048

To expand patient access to experimental treatments in clinical trials, 
                        and for other purposes.

_______________________________________________________________________

                   IN THE SENATE OF THE UNITED STATES

                              May 4, 2017

Mr. Hatch (for himself, Mr. Bennet, Mr. Burr, and Mr. Casey) introduced 
the following bill; which was read twice and referred to the Committee 
               on Health, Education, Labor, and Pensions

_______________________________________________________________________

                                 A BILL

 
To expand patient access to experimental treatments in clinical trials, 
                        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 ``Enhanced Clinical Trial Design Act 
of 2017''.

SEC. 2. PATIENT ACCESS TO EXPERIMENTAL TREATMENTS.

    (a) Public Meeting.--
            (1) In general.--The Secretary of Health and Human Services 
        (referred to in this Act as the ``Secretary''), acting through 
        the Commissioner of Food and Drugs, in coordination with the 
        Director of the National Institutes of Health, and in 
        consultation with patients, health care providers, drug 
        sponsors, bioethicists, and other stakeholders, shall, not 
        later than 180 days after the date of enactment of this Act, 
        convene a public meeting to discuss clinical trial inclusion 
        and exclusion criteria to inform the guidance under subsection 
        (c). The Secretary shall inform the Comptroller General of the 
        United States of the date when the public meeting will take 
        place.
            (2) Topics.--The Secretary shall provide a publicly 
        available report on the topics discussed at the meeting 
        described in paragraph (1) within 30 days of such meeting. Such 
        topics shall include discussion of--
                    (A) the rationale for, and potential barriers for 
                patients created by, clinical trial inclusion and 
                exclusion criteria;
                    (B) how patient populations most likely to be 
                affected by a drug can benefit from the results of 
                trials that employ alternative designs, as well as 
                potential risks associated with alternative clinical 
                trial designs;
                    (C) barriers to participation in clinical trials, 
                including--
                            (i) information regarding any potential 
                        risks and benefits of participation;
                            (ii) regulatory, geographical, and 
                        socioeconomic barriers; and
                            (iii) the impact of exclusion criteria on 
                        the enrollment in clinical trials of infants 
                        and children, pregnant and lactating women, 
                        seniors, individuals with advanced disease, and 
                        individuals with co-morbid conditions;
                    (D) clinical trial designs and methods that 
                increase enrollment of more diverse patient populations 
                while facilitating the collection of data to support 
                substantial evidence of safety and effectiveness; and
                    (E) how changes to clinical trial inclusion and 
                exclusion criteria may impact the complexity of the 
                clinical trial design and length of clinical trials, 
                and potential approaches to mitigating those impacts to 
                ensure that the ability to demonstrate safety and 
                effectiveness is not hindered through potential changes 
                in eligibility criteria.
    (b) Report.--Not later than 1 year after the Secretary issues a 
report on the topics discussed at the public meeting under subsection 
(a)(2), the Comptroller General of the United States shall report to 
the Committee on Health, Education, Labor, and Pensions of the Senate 
and the Committee on Energy and Commerce of the House of 
Representatives on individual access to investigational drugs through 
the expanded access program under section 561(b) of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 360bbb(b)). The report shall 
include--
            (1) a description of actions taken by manufacturers under 
        section 561A of the Federal Food, Drug, and Cosmetic Act (21 
        U.S.C. 360bbb-0);
            (2) consideration of whether Form FDA 3926 and the guidance 
        document entitled ``Expanded Access to Investigational Drugs 
        for Treatment Use--Questions and Answers'', issued by the Food 
        and Drug Administration in June 2016, has reduced application 
        burden with respect to individuals and physicians seeking 
        access to investigational new drugs pursuant to section 561(b) 
        of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb) 
        and improved clarity for patients, physicians, and drug 
        manufacturers about such process;
            (3) consideration of whether the guidance or regulations 
        released or updated under section 561 of the Federal Food, 
        Drug, and Cosmetic Act (21 U.S.C. 360bbb) have improved access 
        for individual patients who do not qualify for clinical trials 
        of such investigational drugs, and what barriers to such access 
        remain;
            (4) an assessment of how patients and health care providers 
        navigate different avenues to engage with the Food and Drug 
        Administration or drug sponsors on expanded access; and
            (5) an analysis of the Secretary's report under subsection 
        (a)(2).
    (c) Guidance.--
            (1) In general.--Not later than 180 days after the 
        publication of the report under subsection (a) the Secretary, 
        acting through the Commissioner of Food and Drugs, shall issue 
        one or more draft guidances regarding eligibility criteria for 
        clinical trials. Not later than 18 months after the public 
        comment period on each such draft guidance ends, the Secretary 
        shall issue a revised draft guidance or final guidance.
            (2) Contents.--The guidance documents described in 
        paragraph (1) shall address methodological approaches that a 
        manufacturer or sponsor of an investigation of a new drug may 
        take to--
                    (A) broaden eligibility criteria for clinical 
                trials, especially with respect to drugs for the 
                treatment of serious and life-threatening conditions or 
                diseases for which there is an unmet medical need; and
                    (B) develop eligibility criteria for, and increase 
                trial recruitment to, clinical trials so that 
                enrollment in such trials more accurately reflects the 
                patients most likely to receive the drug, as applicable 
                and as appropriate, while supporting findings of 
                substantial evidence of safety and effectiveness.

SEC. 3. IMPROVING INSTITUTIONAL REVIEW BOARD REVIEW OF SINGLE PATIENT 
              EXPANDED ACCESS PROTOCOL.

    Not later than 1 year after the date of enactment of this Act, the 
Secretary of Health and Human Services (referred to in this section as 
the ``Secretary''), acting through the Commissioner of Food and Drugs, 
shall issue guidance or regulations, or revise existing guidance or 
regulations, to streamline the institutional review board review for 
individual pediatric and adult patient expanded access protocol under 
561(b) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
360bbb(b)). Such guidance or regulation may include a description of 
the conditions under which an institutional review board chair (or 
designee) may review individual patient expanded access protocol 
submitted under section 505(i) of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 355(i)) for a drug and how centralized institutional 
review boards may facilitate the use of expanded access protocols. The 
Secretary shall update any relevant forms associated with individual 
patient expanded access protocol as necessary.

SEC. 4. EXPANDED ACCESS POLICY TRANSPARENCY.

    Section 561A(f) of the Federal Food, Drug, and Cosmetic Act (21 
U.S.C. 360bbb-0(f)) is amended--
            (1) in the matter preceding paragraph (1), by striking 
        ``later'' and inserting ``earlier'';
            (2) by striking paragraph (1);
            (3) by redesignating paragraph (2) as paragraph (1);
            (4) in paragraph (1) as so redesignated, by striking the 
        period at the end and inserting ``; or''; and
            (5) by adding at the end the following:
            ``(2) as applicable, 15 days after the drug receives a 
        designation as a breakthrough therapy, fast track product, or 
        regenerative advanced therapy under subsection (a), (b), or 
        (g), respectively, of section 506.''.
                                 <all>
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