Enhanced Clinical Trial Design Act of 2017

To expand patient access to experimental treatments in clinical trials, and for other purposes. 1. Short title This Act may be cited as the Enhanced Clinical Trial Design Act of 2017 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 (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) (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 21 U.S.C. 360bbb (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 (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. 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 21 U.S.C. 360bbb(b) 21 U.S.C. 355(i) 4. Expanded access policy transparency Section 561A(f) of the Federal Food, Drug, and Cosmetic Act ( 21 U.S.C. 360bbb–0(f) (1) in the matter preceding paragraph (1), by striking later 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 (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. .

Summary: Enhanced Clinical Trial Design Act of 2017 This bill requires the Food and Drug Administration (FDA), in coordination with the National Institutes of Health, to convene a meeting to discuss clinical trial inclusion and exclusion criteria. The FDA must report on the meeting and issue guidance regarding eligibility criteria for clinical trials. The Government Accountability Office must report on individual access to investigational drugs for serious conditions through the FDA's expanded access program (i.e., compassionate use). The FDA must streamline review by institutional review boards of expanded access protocols for individual patients. The bill amends the Federal Food, Drug, and Cosmetic Act to require the manufacturer or distributor of an investigational drug for a serious condition that is designated a breakthrough therapy, fast track product, or regenerative advanced therapy to publish its expanded access policy not later than 15 days after the designation.