I 

114TH CONGRESS 

1ST SESSION H. R. 2338 

To amend the Federal Food, Drug, and Cosmetic Act to provide for the 

development and use of patient experience data to enhance the structured 

risk-benefit assessment framework, and for other purposes. 

IN THE HOUSE OF REPRESENTATIVES 

MAY 14, 2015 

Mr. PITTS 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 to 

provide for the development and use of patient experience 

data to enhance the structured risk-benefit assessment 

framework, 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. DEVELOPMENT AND USE OF PATIENT EXPERI4 ENCE DATA TO ENHANCE STRUCTURED RISK5 BENEFIT ASSESSMENT FRAMEWORK. 

6 (a) IN GENERAL.—Section 505 of the Federal Food, 

7 Drug, and Cosmetic Act (21 U.S.C. 355) is amended— 


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1 (1) in subsection (d), by striking ‘‘The Sec2 retary shall implement’’ and all that follows through 

3 ‘‘premarket approval of a drug.’’; and 

4 (2) by adding at the end the following new sub5 sections: 

6 ‘‘(x) STRUCTURED RISK-BENEFIT ASSESSMENT

7 FRAMEWORK.— 

8 ‘‘(1) IN GENERAL.—The Secretary shall imple9 ment a structured risk-benefit assessment frame10 work in the new drug approval process— 

11 ‘‘(A) to facilitate the balanced consider12 ation of benefits and risks; and 

13 ‘‘(B) to develop and implement a con14 sistent and systematic approach to the discus15 sion of, regulatory decisionmaking with respect 

16 to, and the communication of, the benefits and 

17 risks of new drugs. 

18 ‘‘(2) RULE OF CONSTRUCTION.—Nothing in 

19 paragraph (1) shall alter the criteria for evaluating 

20 an application for premarket approval of a drug. 

21 ‘‘(y) DEVELOPMENT AND USE OF PATIENT EXPERI22 ENCE DATA TO ENHANCE STRUCTURED RISK-BENEFIT

23 ASSESSMENT FRAMEWORK.— 

24 ‘‘(1) IN GENERAL.—Not later than two years 

25 after the date of the enactment of this subsection, 


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1 the Secretary shall establish and implement proc2 esses under which— 

3 ‘‘(A) an entity seeking to develop patient 

4 experience data may submit to the Secretary— 

5 ‘‘(i) initial research concepts for feed6 back from the Secretary; and 

7 ‘‘(ii) with respect to patient experience 

8 data collected by the entity, draft guidance 

9 documents, completed data, and sum10 maries and analyses of such data; 

11 ‘‘(B) the Secretary may request such an 

12 entity to submit such documents, data, and 

13 summaries and analyses; and 

14 ‘‘(C) patient experience data may be devel15 oped and used to enhance the structured risk16 benefit assessment framework under subsection 

17 (x). 

18 ‘‘(2) PATIENT EXPERIENCE DATA.—In this sub19 section, the term ‘patient experience data’ means 

20 data collected by patients, parents, caregivers, pa21 tient advocacy organizations, disease research foun22 dations, medical researchers, research sponsors or 

23 other parties determined appropriate by the Sec24 retary that is intended to facilitate or enhance the 

25 Secretary’s risk-benefit assessments, including inforVerDate Sep 11 2014 01:18 May 20, 2015 Jkt 049200 PO 00000 Frm 00003 Fmt 6652 Sfmt 6201 E:\BILLS\H2338.IH H2338

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1 mation about the impact of a disease or a therapy 

2 on patients’ lives.’’. 

3 (b) GUIDANCE.— 

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

5 Human Services shall publish guidance on the imple6 mentation of subsection (y) of section 505 of the 

7 Federal Food, Drug, and Cosmetic Act (21 U.S.C. 

8 355), as added by subsection (a). Such guidance 

9 shall include— 

10 (A) with respect to draft guidance docu11 ments, data, or summaries and analyses sub12 mitted to the Secretary under paragraph (1)(A) 

13 of such subsection, guidance— 

14 (i) specifying the timelines for the re15 view of such documents, data, or sum16 maries and analyses by the Secretary; and 

17 (ii) on how the Secretary will use such 

18 documents, data, or summaries and anal19 yses to update any guidance documents 

20 published under this subsection or publish 

21 new guidance; 

22 (B) with respect to the collection and anal23 ysis of patient experience data (as defined in 

24 paragraph (2) of such subsection (y)), guidance 

25 on— 


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1 (i) methodological considerations for 

2 the collection of patient experience data, 

3 which may include structured approaches 

4 to gathering information on— 

5 (I) the experience of a patient liv6 ing with a particular disease; 

7 (II) the burden of living with or 

8 managing the disease; 

9 (III) the impact of the disease on 

10 daily life and long-term functioning; 

11 and 

12 (IV) the effect of current thera13 peutic options on different aspects of 

14 the disease; and 

15 (ii) the establishment and mainte16 nance of registries designed to increase un17 derstanding of the natural history of a dis18 ease; 

19 (C) methodological approaches that may be 

20 used to assess patients’ beliefs with respect to 

21 the benefits and risks in the management of the 

22 patient’s disease; and 

23 (D) methodologies, standards, and poten24 tial experimental designs for patient-reported 

25 outcomes. 


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1 (2) TIMING.—Not later than three years after 

2 the date of the enactment of this Act, the Secretary 

3 of Health and Human Services shall issue draft 

4 guidance on the implementation of subsection (y) of 

5 section 505 of the Federal Food, Drug, and Cos6 metic Act (21 U.S.C. 355), as added by subsection 

7 (a). The Secretary shall issue final guidance on the 

8 implementation of such subsection not later than one 

9 year after the date on which the comment period for 

10 the draft guidance closes. 

11 (3) WORKSHOPS.— 

12 (A) IN GENERAL.—Not later than 6 

13 months after the date of the enactment of this 

14 Act and once every 6 months during the fol15 lowing 12-month period, the Secretary of 

16 Health and Human Services shall convene a 

17 workshop to obtain input regarding methodolo18 gies for developing the guidance under para19 graph (1), including the collection of patient ex20 perience data. 

21 (B) ATTENDEES.—A workshop convened 

22 under this paragraph shall include— 

23 (i) patients; 


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1 (ii) representatives from patient advo2 cacy organizations, biopharmaceutical com3 panies, and disease research foundations; 

4 (iii) representatives of the reviewing 

5 divisions of the Food and Drug Adminis6 tration; and 

7 (iv) methodological experts with sig8 nificant expertise in patient experience 

9 data. 

10 (4) PUBLIC MEETING.—Not later than 90 days 

11 after the date on which the draft guidance is pub12 lished under this subsection, the Secretary of Health 

13 and Human Services shall convene a public meeting 

14 to solicit input on the guidance. 

Æ 


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