II 

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. 

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled, 

3 SECTION 1. SHORT TITLE. 

4 This Act may be cited as the ‘‘Enhanced Clinical 

5 Trial Design Act of 2017’’. 

6 SEC. 2. PATIENT ACCESS TO EXPERIMENTAL TREATMENTS. 

7 (a) PUBLIC MEETING.— 

8 (1) IN GENERAL.—The Secretary of Health and 

9 Human Services (referred to in this Act as the ‘‘Sec10 retary’’), acting through the Commissioner of Food 


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1 and Drugs, in coordination with the Director of the 

2 National Institutes of Health, and in consultation 

3 with patients, health care providers, drug sponsors, 

4 bioethicists, and other stakeholders, shall, not later 

5 than 180 days after the date of enactment of this 

6 Act, convene a public meeting to discuss clinical trial 

7 inclusion and exclusion criteria to inform the guid8 ance under subsection (c). The Secretary shall in9 form the Comptroller General of the United States 

10 of the date when the public meeting will take place. 

11 (2) TOPICS.—The Secretary shall provide a 

12 publicly available report on the topics discussed at 

13 the meeting described in paragraph (1) within 30 

14 days of such meeting. Such topics shall include dis15 cussion of— 

16 (A) the rationale for, and potential barriers 

17 for patients created by, clinical trial inclusion 

18 and exclusion criteria; 

19 (B) how patient populations most likely to 

20 be affected by a drug can benefit from the re21 sults of trials that employ alternative designs, 

22 as well as potential risks associated with alter23 native clinical trial designs; 

24 (C) barriers to participation in clinical 

25 trials, including— 


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1 (i) information regarding any poten2 tial risks and benefits of participation; 

3 (ii) regulatory, geographical, and so4 cioeconomic barriers; and 

5 (iii) the impact of exclusion criteria on 

6 the enrollment in clinical trials of infants 

7 and children, pregnant and lactating 

8 women, seniors, individuals with advanced 

9 disease, and individuals with co-morbid 

10 conditions; 

11 (D) clinical trial designs and methods that 

12 increase enrollment of more diverse patient pop13 ulations while facilitating the collection of data 

14 to support substantial evidence of safety and ef15 fectiveness; and 

16 (E) how changes to clinical trial inclusion 

17 and exclusion criteria may impact the com18 plexity of the clinical trial design and length of 

19 clinical trials, and potential approaches to miti20 gating those impacts to ensure that the ability 

21 to demonstrate safety and effectiveness is not 

22 hindered through potential changes in eligibility 

23 criteria. 

24 (b) REPORT.—Not later than 1 year after the Sec25 retary issues a report on the topics discussed at the public 


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1 meeting under subsection (a)(2), the Comptroller General 

2 of the United States shall report to the Committee on 

3 Health, Education, Labor, and Pensions of the Senate and 

4 the Committee on Energy and Commerce of the House 

5 of Representatives on individual access to investigational 

6 drugs through the expanded access program under section 

7 561(b) of the Federal Food, Drug, and Cosmetic Act (21 

8 U.S.C. 360bbb(b)). The report shall include— 

9 (1) a description of actions taken by manufac10 turers under section 561A of the Federal Food, 

11 Drug, and Cosmetic Act (21 U.S.C. 360bbb–0); 

12 (2) consideration of whether Form FDA 3926 

13 and the guidance document entitled ‘‘Expanded Ac14 cess to Investigational Drugs for Treatment Use— 

15 Questions and Answers’’, issued by the Food and 

16 Drug Administration in June 2016, has reduced ap17 plication burden with respect to individuals and phy18 sicians seeking access to investigational new drugs 

19 pursuant to section 561(b) of the Federal Food, 

20 Drug, and Cosmetic Act (21 U.S.C. 360bbb) and 

21 improved clarity for patients, physicians, and drug 

22 manufacturers about such process; 

23 (3) consideration of whether the guidance or 

24 regulations released or updated under section 561 of 

25 the Federal Food, Drug, and Cosmetic Act (21 


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1 U.S.C. 360bbb) have improved access for individual 

2 patients who do not qualify for clinical trials of such 

3 investigational drugs, and what barriers to such ac4 cess remain; 

5 (4) an assessment of how patients and health 

6 care providers navigate different avenues to engage 

7 with the Food and Drug Administration or drug 

8 sponsors on expanded access; and 

9 (5) an analysis of the Secretary’s report under 

10 subsection (a)(2). 

11 (c) GUIDANCE.— 

12 (1) IN GENERAL.—Not later than 180 days 

13 after the publication of the report under subsection 

14 (a) the Secretary, acting through the Commissioner 

15 of Food and Drugs, shall issue one or more draft 

16 guidances regarding eligibility criteria for clinical 

17 trials. Not later than 18 months after the public 

18 comment period on each such draft guidance ends, 

19 the Secretary shall issue a revised draft guidance or 

20 final guidance. 

21 (2) CONTENTS.—The guidance documents de22 scribed in paragraph (1) shall address methodo23 logical approaches that a manufacturer or sponsor of 

24 an investigation of a new drug may take to— 


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1 (A) broaden eligibility criteria for clinical 

2 trials, especially with respect to drugs for the 

3 treatment of serious and life-threatening condi4 tions or diseases for which there is an unmet 

5 medical need; and 

6 (B) develop eligibility criteria for, and in7 crease trial recruitment to, clinical trials so that 

8 enrollment in such trials more accurately re9 flects the patients most likely to receive the 

10 drug, as applicable and as appropriate, while 

11 supporting findings of substantial evidence of 

12 safety and effectiveness. 

13 SEC. 3. IMPROVING INSTITUTIONAL REVIEW BOARD RE14 VIEW OF SINGLE PATIENT EXPANDED AC15 CESS PROTOCOL. 

16 Not later than 1 year after the date of enactment 

17 of this Act, the Secretary of Health and Human Services 

18 (referred to in this section as the ‘‘Secretary’’), acting 

19 through the Commissioner of Food and Drugs, shall issue 

20 guidance or regulations, or revise existing guidance or reg21 ulations, to streamline the institutional review board re22 view for individual pediatric and adult patient expanded 

23 access protocol under 561(b) of the Federal Food, Drug, 

24 and Cosmetic Act (21 U.S.C. 360bbb(b)). Such guidance 

25 or regulation may include a description of the conditions 


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1 under which an institutional review board chair (or des2 ignee) may review individual patient expanded access pro3 tocol submitted under section 505(i) of the Federal Food, 

4 Drug, and Cosmetic Act (21 U.S.C. 355(i)) for a drug 

5 and how centralized institutional review boards may facili6 tate the use of expanded access protocols. The Secretary 

7 shall update any relevant forms associated with individual 

8 patient expanded access protocol as necessary. 

9 SEC. 4. EXPANDED ACCESS POLICY TRANSPARENCY. 

10 Section 561A(f) of the Federal Food, Drug, and Cos11 metic Act (21 U.S.C. 360bbb–0(f)) is amended— 

12 (1) in the matter preceding paragraph (1), by 

13 striking ‘‘later’’ and inserting ‘‘earlier’’; 

14 (2) by striking paragraph (1); 

15 (3) by redesignating paragraph (2) as para16 graph (1); 

17 (4) in paragraph (1) as so redesignated, by 

18 striking the period at the end and inserting ‘‘; or’’; 

19 and 

20 (5) by adding at the end the following: 

21 ‘‘(2) as applicable, 15 days after the drug re22 ceives a designation as a breakthrough therapy, fast 

23 track product, or regenerative advanced therapy 


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1 under subsection (a), (b), or (g), respectively, of sec2 tion 506.’’. 

Æ 


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