IB 
Union Calendar No. 142 
114TH CONGRESS 
1ST SESSION 
H. R. 6 
[Report No. 114–190, Part I] 
To accelerate the discovery, development, and delivery of 21st century cures, 
and for other purposes. 
IN THE HOUSE OF REPRESENTATIVES 
MAY 19, 2015 
Mr. UPTON (for himself, Ms. DEGETTE, Mr. PITTS, Mr. PALLONE, and Mr. 
GENE GREEN of Texas) introduced the following bill; which was referred 
to the Committee on Energy and Commerce, and in addition to the Com-
mittee on Ways and Means, for a period to be subsequently determined 
by the Speaker, in each case for consideration of such provisions as fall 
within the jurisdiction of the committee concerned 
JULY 7, 2015 
Additional sponsors: Mr. BARTON, Mr. CRAMER, Mr. BUCSHON, Mr. BILI-
RAKIS, Mrs. BLACKBURN, Mrs. BROOKS of Indiana, Mr. BURGESS, Mrs. 
ELLMERS of North Carolina, Mr. GRIFFITH, Mr. GUTHRIE, Mr. LANCE, 
Mr. MCKINLEY, Mrs. MCMORRIS RODGERS, Mr. MULLIN, Mr. MURPHY 
of Pennsylvania, Mr. SHIMKUS, Mr. WALDEN, Mr. WHITFIELD, Mr. ROS-
KAM, Mr. HANNA, Mr. MCCAUL, Mrs. COMSTOCK, Mr. HARRIS, Mr. 
MARCHANT, Mr. YARMUTH, Ms. CASTOR of Florida, Mr. LOEBSACK, Ms. 
SCHAKOWSKY, Mr. TONKO, Ms. MOORE, Mr. VEASEY, Mrs. DINGELL, 
Mr. FATTAH, Mr. SCHRADER, Mr. NOLAN, Ms. ESHOO, Mr. WELCH, Mr. 
DAVID SCOTT of Georgia, Mr. PERLMUTTER, Mr. COURTNEY, Mr. 
COHEN, Mr. DESAULNIER, Mr. LONG, Mr. BUTTERFIELD, Ms. CLARKE 
of New York, Mr. MICHAEL F. DOYLE of Pennsylvania, Mr. RUSH, Mr. 
ENGEL, Mr. MCNERNEY, Ms. MATSUI, Mr. FLORES, Mr. JOHNSON of 
Ohio, Mr. GIBSON, Mr. KENNEDY, Mr. BEN RAY LUJA´N of New Mexico, 
Mr. WALZ, Mr. CASTRO of Texas, Mrs. BUSTOS, Ms. FRANKEL of Flor-
ida, Ms. BROWNLEY of California, Mr. COSTA, Mrs. WAGNER, Mr. ROD-
NEY DAVIS of Illinois, Mr. SCALISE, Mr. LATTA, Mr. HARPER, Mr. 
OLSON, Mr. KINZINGER of Illinois, Mr. POMPEO, Mr. COLLINS of New 
York, Mrs. MIMI WALTERS of California, Mr. ALLEN, Mr. SARBANES, 
Ms. LEE, Mrs. MILLER of Michigan, Mr. HIGGINS, Mr. HUFFMAN, Mr. 
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KILDEE, Mr. QUIGLEY, Mr. TAKAI, Mr. HECK of Nevada, Mr. PIERLUISI, 
Mr. BENISHEK, Mrs. WALORSKI, Mr. ISRAEL, Mr. HULTGREN, Ms. 
KUSTER, Mr. YODER, Mr. DENT, Mr. CURBELO of Florida, Mrs. KIRK-
PATRICK, Mr. POLIS, Mr. O’ROURKE, Mr. HUDSON, Mr. CA´RDENAS, Mrs. 
CAPPS, Mr. ROE of Tennessee, Mr. BISHOP of Michigan, Mr. ROSS, Mr. 
PAYNE, Ms. NORTON, Mr. DOLD, Mr. TURNER, Mr. COFFMAN, Mr. 
SIRES, Mr. MEADOWS, Mr. COSTELLO of Pennsylvania, Mr. GUTIE´RREZ, 
Mr. MESSER, Mr. WALBERG, Mr. SALMON, Mr. JENKINS of West Vir-
ginia, Mr. POCAN, Mr. PETERS, Mrs. TORRES, Ms. LOFGREN, Ms. MENG, 
Mr. TAKANO, Mr. PITTENGER, Mr. DENHAM, Mr. BARLETTA, Mr. 
HARDY, Mr. RUPPERSBERGER, Mr. VARGAS, Mr. GARAMENDI, Mr. HECK 
of Washington, Mr. BERA, Mr. LUETKEMEYER, Mr. ZELDIN, Ms. CLARK 
of Massachusetts, Mr. BOST, Mrs. LAWRENCE, Ms. VELA´ZQUEZ, Mr. 
ROGERS of Alabama, Ms. EDWARDS, Mr. YOUNG of Iowa, Mr. KATKO, 
Mr. MOULTON, Mr. FORTENBERRY, Mr. MCHENRY, Mr. HINOJOSA, Mr. 
CARTWRIGHT, 
Mr. 
JOYCE, 
Mr. 
VALADAO, 
Mr. 
ASHFORD, 
Mr. 
MOOLENAAR, Mr. DONOVAN, Mr. WENSTRUP, Mr. ELLISON, Mr. HAS-
TINGS, Mrs. CAROLYN B. MALONEY of New York, Mr. NADLER, Ms. 
WASSERMAN SCHULTZ, Mr. TROTT, Ms. KAPTUR, Ms. KELLY of Illinois, 
Mr. RANGEL, Ms. FUDGE, Mr. MCGOVERN, Mr. REED, Mr. HUIZENGA 
of Michigan, Mr. LAMALFA, Mr. CRENSHAW, Ms. SINEMA, Ms. MCCOL-
LUM, Mr. WEBSTER of Florida, Ms. BORDALLO, Mr. GIBBS, Mr. 
BRENDAN F. BOYLE of Pennsylvania, Mr. CONAWAY, Mr. KNIGHT, Mr. 
WOMACK, Mr. DESJARLAIS, Mr. ROYCE, Mr. DIAZ-BALART, Ms. 
STEFANIK, Mr. ZINKE, Mr. CAPUANO, Ms. JACKSON LEE, Mr. TIBERI, 
Ms. HERRERA BEUTLER, Mr. VAN HOLLEN, Mr. GUINTA, Ms. ROS- 
LEHTINEN, Mr. SIMPSON, Ms. BONAMICI, Mr. CICILLINE, Mr. LIPINSKI, 
Miss RICE of New York, Mr. PAULSEN, Ms. SPEIER, Mr. COOK, Mr. 
LANGEVIN, Mr. STIVERS, Mr. CLEAVER, Mr. COLLINS of Georgia, Mr. 
HUNTER, Ms. HAHN, Mr. RIGELL, Mr. CHABOT, Ms. DELBENE, Mr. 
LOBIONDO, Ms. ROYBAL-ALLARD, Mr. PERRY, Mr. DELANEY, Mr. 
LEWIS, Mr. TED LIEU of California, Mr. JOHNSON of Georgia, Ms. WIL-
SON of Florida, Mr. KEATING, Ms. BROWN of Florida, Mr. KIND, Mr. 
MARINO, Mr. BRADY of Pennsylvania, Mr. FARR, Mr. CALVERT, Mr. 
LARSON of Connecticut, Ms. JUDY CHU of California, Mr. HOYER, Ms. 
EDDIE BERNICE JOHNSON of Texas, Ms. MAXINE WATERS of California, 
Mr. LOWENTHAL, Mr. RYAN of Ohio, Mrs. DAVIS of California, Mr. KIL-
MER, and Ms. DUCKWORTH 
JULY 7, 2015 
Reported from the Committee on Energy and Commerce with an amendment 
[Strike out all after the enacting clause and insert the part printed in italic] 
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JULY 7, 2015 
The Committee on Ways and Means discharged; committed to the Committee 
of the Whole House on the State of the Union and ordered to be printed 
[For text of introduced bill, see copy of bill as introduced on May 19, 2015] 
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A BILL 
To accelerate the discovery, development, and delivery of 
21st century cures, and for other purposes. 
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Be it enacted by the Senate and House of Representa-
tives of the United States of America in Congress assembled, 
SECTION 1. SHORT TITLE; TABLE OF CONTENTS. 
(a) SHORT TITLE.—This Act may be cited as the ‘‘21st 
Century Cures Act’’. 
(b) TABLE OF CONTENTS.—The table of contents for 
this Act is as follows: 
Sec. 1. Short title; table of contents. 
TITLE I—DISCOVERY 
Subtitle A—National Institutes of Health Funding 
Sec. 1001. National Institutes of Health reauthorization. 
Sec. 1002. NIH Innovation Fund. 
Subtitle B—National Institutes of Health Planning and Administration 
Sec. 1021. NIH research strategic plan. 
Sec. 1022. Increasing accountability at the National Institutes of Health. 
Sec. 1023. Reducing administrative burdens of researchers. 
Sec. 1024. Exemption for the National Institutes of Health from the Paperwork 
Reduction Act requirements. 
Sec. 1025. NIH travel. 
Sec. 1026. Other transactions authority. 
Sec. 1027. NCATS phase IIB restriction. 
Sec. 1028. High-risk, high-reward research. 
Sec. 1029. Sense of Congress on increased inclusion of underrepresented commu-
nities in clinical trials. 
Subtitle C—Supporting Young Emerging Scientists 
Sec. 1041. Improvement of loan repayment programs of the National Institutes 
of Health. 
Sec. 1042. Report. 
Subtitle D—Capstone Grant Program 
Sec. 1061. Capstone award. 
Subtitle E—Promoting Pediatric Research Through the National Institutes of 
Health 
Sec. 1081. National pediatric research network. 
Sec. 1082. Global pediatric clinical study network sense of Congress. 
Sec. 1083. Appropriate age groupings in clinical research. 
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Subtitle F—Advancement of the National Institutes of Health Research and 
Data Access 
Sec. 1101. Sharing of data generated through NIH-funded research. 
Sec. 1102. Standardization of data in Clinical Trial Registry Data Bank on eli-
gibility for clinical trials. 
Subtitle G—Facilitating Collaborative Research 
Sec. 1121. Clinical trial data system. 
Sec. 1122. National neurological diseases surveillance system. 
Sec. 1123. Data on natural history of diseases. 
Sec. 1124. Accessing, sharing, and using health data for research purposes. 
Subtitle H—Council for 21st Century Cures 
Sec. 1141. Council for 21st Century Cures. 
TITLE II—DEVELOPMENT 
Subtitle A—Patient-Focused Drug Development 
Sec. 2001. Development and use of patient experience data to enhance structured 
risk-benefit assessment framework. 
Subtitle B—Qualification and Use of Drug Development Tools 
Sec. 2021. Qualification of drug development tools. 
Sec. 2022. Accelerated approval development plan. 
Subtitle C—FDA Advancement of Precision Medicine 
Sec. 2041. Precision medicine guidance and other programs of Food and Drug 
Administration. 
Subtitle D—Modern Trial Design and Evidence Development 
Sec. 2061. Broader application of Bayesian statistics and adaptive trial designs. 
Sec. 2062. Utilizing evidence from clinical experience. 
Sec. 2063. Streamlined data review program. 
Subtitle E—Expediting Patient Access 
Sec. 2081. Sense of Congress. 
Sec. 2082. Expanded access policy. 
Sec. 2083. Finalizing draft guidance on expanded access. 
Subtitle F—Facilitating Responsible Manufacturer Communications 
Sec. 2101. Facilitating dissemination of health care economic information. 
Sec. 2102. Facilitating responsible communication of scientific and medical devel-
opments. 
Subtitle G—Antibiotic Drug Development 
Sec. 2121. Approval of certain drugs for use in a limited population of patients. 
Sec. 2122. Susceptibility test interpretive criteria for microorganisms. 
Sec. 2123. Encouraging the development and use of new antimicrobial drugs. 
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Subtitle H—Vaccine Access, Certainty, and Innovation 
Sec. 2141. Timely review of vaccines by the Advisory Committee on Immuniza-
tion Practices. 
Sec. 2142. Review of processes and consistency of ACIP recommendations. 
Sec. 2143. Meetings between CDC and vaccine developers. 
Subtitle I—Orphan Product Extensions Now; Incentives for Certain Products 
for Limited Populations 
Sec. 2151. Extension of exclusivity periods for a drug approved for a new indica-
tion for a rare disease or condition. 
Sec. 2152. Reauthorization of rare pediatric disease priority review voucher in-
centive program. 
Subtitle J—Domestic Manufacturing and Export Efficiencies 
Sec. 2161. Grants for studying the process of continuous drug manufacturing. 
Sec. 2162. Re-exportation among members of the European Economic Area. 
Subtitle K—Enhancing Combination Products Review 
Sec. 2181. Enhancing combination products review. 
Subtitle L—Priority Review for Breakthrough Devices 
Sec. 2201. Priority review for breakthrough devices. 
Subtitle M—Medical Device Regulatory Process Improvements 
Sec. 2221. Third-party quality system assessment. 
Sec. 2222. Valid scientific evidence. 
Sec. 2223. Training and oversight in least burdensome appropriate means con-
cept. 
Sec. 2224. Recognition of standards. 
Sec. 2225. Easing regulatory burden with respect to certain class I and class II 
devices. 
Sec. 2226. Advisory committee process. 
Sec. 2227. Humanitarian device exemption application. 
Sec. 2228. CLIA waiver study design guidance for in vitro diagnostics. 
Subtitle N—Sensible Oversight for Technology Which Advances Regulatory 
Efficiency 
Sec. 2241. Health software. 
Sec. 2242. Applicability and inapplicability of regulation. 
Sec. 2243. Exclusion from definition of device. 
Subtitle O—Streamlining Clinical Trials 
Sec. 2261. Protection of human subjects in research; applicability of rules. 
Sec. 2262. Use of non-local institutional review boards for review of investiga-
tional device exemptions and human device exemptions. 
Sec. 2263. Alteration or waiver of informed consent for clinical investigations. 
Subtitle P—Improving Scientific Expertise and Outreach at FDA 
Sec. 2281. Silvio O. Conte Senior Biomedical Research Service. 
Sec. 2282. Enabling FDA scientific engagement. 
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Sec. 2283. Reagan-Udall Foundation for the Food and Drug Administration. 
Sec. 2284. Collection of certain voluntary information exempted from Paperwork 
Reduction Act. 
Sec. 2285. Hiring authority for scientific, technical, and professional personnel. 
Subtitle Q—Exempting From Sequestration Certain User Fees 
Sec. 2301. Exempting from sequestration certain user fees of Food and Drug Ad-
ministration. 
TITLE III—DELIVERY 
Subtitle A—Interoperability 
Sec. 3001. Ensuring interoperability of health information technology. 
Subtitle B—Telehealth 
Sec. 3021. Telehealth services under the Medicare program. 
Subtitle C—Encouraging Continuing Medical Education for Physicians 
Sec. 3041. Exempting from manufacturer transparency reporting certain trans-
fers used for educational purposes. 
Subtitle D—Disposable Medical Technologies 
Sec. 3061. Treatment of certain items and devices. 
Subtitle E—Local Coverage Decision Reforms 
Sec. 3081. Improvements in the Medicare local coverage determination (LCD) 
process. 
Subtitle F—Medicare Pharmaceutical and Technology Ombudsman 
Sec. 3101. Medicare pharmaceutical and technology ombudsman. 
Subtitle G—Medicare Site-of-Service Price Transparency 
Sec. 3121. Medicare site-of-Service price transparency. 
Subtitle H—Medicare Part D Patient Safety and Drug Abuse Prevention 
Sec. 3141. Programs to prevent prescription drug abuse under Medicare parts C 
and D. 
TITLE IV—MEDICAID, MEDICARE, AND OTHER REFORMS 
Subtitle A—Medicaid and Medicare Reforms 
Sec. 4001. Limiting Federal Medicaid reimbursement to States for durable med-
ical equipment (DME) to Medicare payment rates. 
Sec. 4002. Medicare payment incentive for the transition from traditional x-ray 
imaging to digital radiography and other Medicare imaging 
payment provision. 
Sec. 4003. Implementation of Office of Inspector General recommendation to 
delay certain Medicare prescription drug plan prepayments. 
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Subtitle B—Cures Innovation Fund 
Sec. 4041. Cures Innovation Fund. 
Subtitle C—Other Reforms 
Sec. 4061. SPR drawdown. 
Subtitle D—Miscellaneous 
Sec. 4081. Lyme disease and other tick-borne diseases. 
TITLE I—DISCOVERY 
Subtitle A—National Institutes of 
Health Funding 
SEC. 1001. NATIONAL INSTITUTES OF HEALTH REAUTHOR-
IZATION. 
Section 402A(a)(1) of the Public Health Service Act 
(42 U.S.C. 282a(a)(1)) is amended— 
(1) in subparagraph (B), by striking at the end 
‘‘and’’; 
(2) in subparagraph (C), by striking at the end 
the period and inserting a semicolon; and 
(3) by adding at the end the following new sub-
paragraphs: 
‘‘(D) $31,811,000,000 for fiscal year 2016; 
‘‘(E) $33,331,000,000 for fiscal year 2017; 
and 
‘‘(F) $34,851,000,000 for fiscal year 2018.’’. 
SEC. 1002. NIH INNOVATION FUND. 
(a) USE OF INNOVATION FUND.—Section 402(b) of the 
Public Health Service Act (42 U.S.C. 282(b)) is amended— 
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(1) in paragraph (23), by striking at the end 
‘‘and’’; 
(2) in paragraph (24), by striking at the end the 
period and inserting ‘‘; and’’; and 
(3) by inserting after paragraph (24), the fol-
lowing new paragraph: 
‘‘(25) shall, with respect to funds appropriated 
under section 402A(e) to the NIH Innovation Fund, 
allocate such funds to the national research institutes 
and national centers for conducting and supporting 
innovation fund initiatives identified under para-
graph (3) of such section.’’. 
(b) ESTABLISHMENT OF INNOVATION FUND.—Section 
402A of the Public Health Service Act (42 U.S.C. 282a)is 
amended— 
(1) by redesignating subsection (e) as subsection 
(f); and 
(2) by inserting after subsection (d) the following 
new subsection: 
‘‘(e) NIH INNOVATION FUND.— 
‘‘(1) ESTABLISHMENT.—For the purpose of allo-
cations under section 402(b)(25), there is established 
a fund to be known as the NIH Innovation Fund. The 
Director of NIH shall, with respect to funds appro-
priated to the NIH Innovation Fund, allocate such 
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funds to support biomedical research through the 
funding of basic, translational, and clinical research. 
‘‘(2) AMOUNTS MADE AVAILABLE TO FUND.— 
‘‘(A) IN
GENERAL.—Subject to subpara-
graph (B), there is authorized to be appro-
priated, and appropriated, to the NIH Innova-
tion Fund out of any funds in the Treasury not 
otherwise appropriated, $2,000,000,000 for each 
of fiscal years 2016 through 2020. The amounts 
appropriated to the Fund by the preceding sen-
tence shall be in addition to any amounts other-
wise made available to the National Institutes of 
Health. 
‘‘(B) AVAILABILITY
SUBJECT
TO
APPRO-
PRIATIONS.—Amounts in the Fund shall not be 
available except to the extent and in such 
amounts as are provided in advance in appro-
priation Acts. 
‘‘(C) ALLOCATION
OF
AMOUNTS.—Of the 
amounts made available from the NIH Innova-
tion 
Fund 
for 
allocations 
under 
section 
402(b)(25) for a fiscal year— 
‘‘(i) not less than $500,000,000 shall be 
for the Accelerating Advancement Program 
under paragraph (5); 
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‘‘(ii) not less than 35 percent of such 
amounts remaining after subtracting the al-
location for the Accelerating Advancement 
Program shall be for early stage investiga-
tors (as defined in paragraph (7)); 
‘‘(iii) not less than 20 percent of such 
amounts remaining after subtracting the al-
location for the Accelerating Advancement 
Program shall be for high-risk, high-reward 
research under section 409K; and 
‘‘(iv) not more than 10 percent of such 
amounts (without subtracting the allocation 
for the Accelerating Advancement Program) 
shall be for intramural research. 
‘‘(D) INAPPLICABILITY OF CERTAIN PROVI-
SIONS.—Amounts in the NIH Innovation Fund 
shall not be subject to— 
‘‘(i) any transfer authority of the Sec-
retary or the Director of NIH under section 
241, subsection (c), subsection (d), or any 
other provision of law (other than section 
402(b)(25) and this subsection); or 
‘‘(ii) the Nonrecurring expenses fund 
under section 223 of division G of the Con-
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solidated Appropriations Act, 2008 (42 
U.S.C. 3514a). 
‘‘(3) AUTHORIZED USES.—Amounts in the NIH 
Innovation Fund established under paragraph (1) 
may be used only to conduct or support innovative 
biomedical research through the following: 
‘‘(A) Research in which— 
‘‘(i) a principal investigator has a spe-
cific project or specific objectives; and 
‘‘(ii) funding is tied to pursuit of such 
project or objectives. 
‘‘(B) Research in which— 
‘‘(i) a principal investigator has shown 
promise in biomedical research; and 
‘‘(ii) funding is not tied to a specific 
project or specific objectives. 
‘‘(C) Research to be carried out by an early 
stage investigator (as defined in paragraph (7)). 
‘‘(D) Research to be carried out by a small 
business concern (as defined in section 3 of the 
Small Business Act). 
‘‘(E) The Accelerating Advancement Pro-
gram under paragraph (5). 
‘‘(F) Development and implementation of 
the strategic plan under paragraph (6). 
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‘‘(4) COORDINATION.—In funding programs and 
activities through the NIH Innovation Fund, the Sec-
retary, acting through the Director of NIH, shall— 
‘‘(A) ensure coordination among the na-
tional research institutes, the national centers, 
and other departments, agencies, and offices of 
the Federal Government; and 
‘‘(B) minimize unnecessary duplication. 
‘‘(5) ACCELERATING ADVANCEMENT PROGRAM.— 
The Director of NIH shall establish a program, to be 
known as the Accelerating Advancement Program, 
under which— 
‘‘(A) the Director of NIH partners with na-
tional research institutes and national centers to 
accomplish important biomedical research objec-
tives; and 
‘‘(B) for every $1 made available by the Di-
rector of NIH to a national research institute or 
national center for a research project, the insti-
tute or center makes $1 available for such project 
from funds that are not derived from the NIH 
Innovation Fund. 
‘‘(6) STRATEGIC PLAN.— 
‘‘(A) IN GENERAL.—The Director of NIH 
shall ensure that scientifically based strategic 
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planning is implemented in support of research 
priorities, including through development, use, 
and updating of a research strategic plan that— 
‘‘(i) is designed to increase the efficient 
and effective focus of biomedical research in 
a manner that leverages the best scientific 
opportunities through a deliberative plan-
ning process; 
‘‘(ii) identifies areas, to be known as 
strategic focus areas, in which the resources 
of the NIH Innovation Fund can contribute 
to the goals of expanding knowledge to ad-
dress, and find more effective treatments for, 
unmet medical needs in the United States, 
including the areas of— 
‘‘(I) biomarkers; 
‘‘(II) precision medicine; 
‘‘(III) infectious diseases, includ-
ing pathogens listed as a qualifying 
pathogen under section 505E(f) of the 
Federal Food, Drug, and Cosmetic Act 
or listed or designated as a tropical 
disease under section 524 of such Act; 
and 
‘‘(IV) antibiotics; 
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‘‘(iii) includes objectives for each such 
strategic focus area; and 
‘‘(iv) ensures that basic research re-
mains a priority. 
‘‘(B) UPDATES AND REVIEWS.—The Direc-
tor shall review and, as appropriate, update the 
research strategic plan under subparagraph (A) 
not less than every 18 months. 
‘‘(7) DEFINITION.—In this subsection, the term 
‘early stage investigator’ means an investigator 
who— 
‘‘(A) will be the principal investigator or 
the program director of the proposed research; 
‘‘(B) has never been awarded, or has been 
awarded only once, a substantial, competing 
grant by the National Institutes of Health for 
independent research; and 
‘‘(C) is within 10 years of having com-
pleted— 
‘‘(i) the investigator’s terminal degree; 
or 
‘‘(ii) a medical residency (or the equiv-
alent).’’. 
(c) SUPPLEMENT, NOT
SUPPLANT; PROHIBITION 
AGAINST TRANSFER.—Funds appropriated pursuant to sec-
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tion 402A(e) of the Public Health Service Act, as inserted 
by subsection (b)— 
(1) shall be used to supplement, not supplant, the 
funds otherwise allocated by the National Institutes of 
Health for biomedical research; and 
(2) notwithstanding any transfer authority in 
any appropriation Act, shall not be used for any pur-
pose other than allocating funds for conducting and 
supporting innovation fund initiatives as described in 
section 402(b)(25) of the Public Health Service Act, 
as added by subsection (a). 
Subtitle B—National Institutes of 
Health Planning and Adminis-
tration 
SEC. 1021. NIH RESEARCH STRATEGIC PLAN. 
Section 402 of the Public Health Service Act (42 
U.S.C. 282) is amended— 
(1) in subsection (b), by amending paragraph 
(5) to read as follows: 
‘‘(5) shall ensure that scientifically based stra-
tegic planning is implemented in support of research 
priorities as determined by the agencies of the Na-
tional Institutes of Health, including through develop-
ment, use, and updating of the research strategic plan 
under subsection (m);’’; and 
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(2) by adding at the end the following: 
‘‘(m) RESEARCH STRATEGIC PLAN.— 
‘‘(1) FIVE-YEAR
PLANS
FOR
BIOMEDICAL
RE-
SEARCH STRATEGY.— 
‘‘(A) IN
GENERAL.—For each successive 
five-year period beginning with the period of fis-
cal years 2016 through 2020, the Director of 
NIH, in consultation with the entities described 
in subparagraph (B), shall develop and main-
tain a biomedical research strategic plan that— 
‘‘(i) is designed to increase the efficient 
and effective focus of biomedical research in 
a manner that leverages the best scientific 
opportunities through a deliberative plan-
ning process; 
‘‘(ii) identifies areas, to be known as 
strategic focus areas, in which the resources 
of the National Institutes of Health can best 
contribute to the goal of expanding knowl-
edge on human health in the United States 
through biomedical research; and 
‘‘(iii) includes objectives for each such 
strategic focus area. 
‘‘(B) ENTITIES
DESCRIBED.—The entities 
described in this subparagraph are the directors 
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of the national research institutes and national 
centers, researchers, patient advocacy groups, 
and industry leaders. 
‘‘(2) USE OF PLAN.—The Director of NIH and 
the directors of the national research institutes and 
national centers shall use the strategic plan— 
‘‘(A) to identify research opportunities; and 
‘‘(B) to develop individual strategic plans 
for the research activities of each of the national 
research institutes and national centers that— 
‘‘(i) have a common template; and 
‘‘(ii) identify strategic focus areas in 
which the resources of the national research 
institutes and national centers can best con-
tribute to the goal of expanding knowledge 
on human health in the United States 
through biomedical research. 
‘‘(3) CONTENTS OF PLANS.— 
‘‘(A) STRATEGIC FOCUS AREAS.—The stra-
tegic focus areas identified pursuant to para-
graph (1)(A)(ii) shall— 
‘‘(i) be identified in a manner that— 
‘‘(I) considers the return on in-
vestment to the United States public 
through the investments of the National 
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Institutes of Health in biomedical re-
search; and 
‘‘(II) contributes to expanding 
knowledge to improve the United 
States public’s health through bio-
medical research; and 
‘‘(ii) include overarching and trans- 
National Institutes of Health strategic focus 
areas, to be known as Mission Priority 
Focus Areas, which best serve the goals of 
preventing or eliminating the burden of a 
disease or condition and scientifically merit 
enhanced and focused research over the next 
5 years. 
‘‘(B) RARE AND PEDIATRIC DISEASES AND 
CONDITIONS.—In developing and maintaining a 
strategic plan under this subsection, the Director 
of NIH shall ensure that rare and pediatric dis-
eases and conditions remain a priority. 
‘‘(C) 
WORKFORCE.—In 
developing 
and 
maintaining a strategic plan under this sub-
section, the Director of NIH shall ensure that 
maintaining the biomedical workforce of the fu-
ture, including the participation by scientists 
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from groups traditionally underrepresented in 
the scientific workforce, remains a priority. 
‘‘(4) INITIAL PLAN.—Not later than 270 days 
after the date of enactment of this subsection, the Di-
rector of NIH and the directors of the national re-
search institutes and national centers shall— 
‘‘(A) complete the initial strategic plan re-
quired by paragraphs (1) and (2); and 
‘‘(B) make such initial strategic plan pub-
licly available on the website of the National In-
stitutes of Health. 
‘‘(5) REVIEW; UPDATES.— 
‘‘(A) PROGRESS
REVIEWS.—Not less than 
annually, the Director of NIH, in consultation 
with the directors of the national research insti-
tutes and national centers, shall conduct progress 
reviews for each strategic focus area identified 
under paragraph (1)(A)(ii). 
‘‘(B) UPDATES.—Not later than the end of 
the 5-year period covered by the initial strategic 
plan under this subsection, and every 5 years 
thereafter, the Director of NIH, in consultation 
with the directors of the national research insti-
tutes and national centers, stakeholders in the 
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scientific field, advocates, and the public at 
large, shall— 
‘‘(i) conduct a review of the plan, in-
cluding each strategic focus area identified 
under paragraph (2)(B); and 
‘‘(ii) update such plan in accordance 
with this section.’’. 
SEC. 1022. INCREASING ACCOUNTABILITY AT THE NA-
TIONAL INSTITUTES OF HEALTH. 
(a) APPOINTMENT AND TERMS OF DIRECTORS OF NA-
TIONAL
RESEARCH
INSTITUTES
AND
NATIONAL
CEN-
TERS.—Subsection (a) of section 405 of the Public Health 
Service Act (42 U.S.C. 284) is amended to read as follows: 
‘‘(a) APPOINTMENT; TERMS.— 
‘‘(1) APPOINTMENT.—The Director of the Na-
tional Cancer Institute shall be appointed by the 
President and the directors of the other national re-
search institutes, as well as the directors of the na-
tional centers, shall be appointed by the Director of 
NIH. The directors of the national research institutes, 
as well as national centers, shall report directly to the 
Director of NIH. 
‘‘(2) TERMS.— 
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‘‘(A) IN GENERAL.—The term of office of a 
director of a national research institute or na-
tional center shall be 5 years. 
‘‘(B) REMOVAL.—The director of a national 
research institute or national center may be re-
moved from office by the Director of NIH prior 
to the expiration of such director’s 5-year term. 
‘‘(C) REAPPOINTMENT.—At the end of the 
term of a director of a national research insti-
tute or national center, the director may be re-
appointed. There is no limit on the number of 
terms a director may serve. 
‘‘(D) VACANCIES.—If the office of a director 
of a national research institute or national cen-
ter becomes vacant before the end of such direc-
tor’s term, the director appointed to fill the va-
cancy shall be appointed for a 5-year term start-
ing on the date of such appointment. 
‘‘(E) TRANSITIONAL PROVISION.—Each di-
rector of a national research institute or na-
tional center serving on the date of enactment of 
the 21st Century Cures Act is deemed to be ap-
pointed for a 5-year term under this subsection 
starting on such date of enactment.’’. 
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(b) COMPENSATION TO CONSULTANTS OR INDIVIDUAL 
SCIENTISTS.—Section 202 of the Departments of Labor, 
Health and Human Services, and Education, and Related 
Agencies Appropriations Act, 1993 (Public Law 102–394; 
42 U.S.C. 238f note) is amended by striking ‘‘portable 
structures;’’ and all that follows and inserting ‘‘portable 
structures.’’. 
(c) REVIEW OF CERTAIN AWARDS BY DIRECTORS.— 
Section 405(b) of the Public Health Service Act (42 U.S.C. 
284(b)) is amended by adding at the end the following: 
‘‘(3) Before an award is made by a national research 
institute or by a national center for a grant for a research 
program or project (commonly referred to as an ‘R-series 
grant’), other than an award constituting a noncompeting 
renewal of such grant, or a noncompeting administrative 
supplement to such grant, the director of such national re-
search institute or national center— 
‘‘(A) shall review and approve the award; and 
‘‘(B) shall take into consideration— 
‘‘(i) the mission of the national research in-
stitute or national center and the scientific pri-
orities identified in the strategic plan under sec-
tion 402(m); and 
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‘‘(ii) whether other agencies are funding 
programs or projects to accomplish the same 
goal.’’. 
(d) IOM STUDY ON DUPLICATION IN FEDERAL BIO-
MEDICAL
RESEARCH.—The Secretary of Health and 
Human Services shall enter into an arrangement with the 
Institute of Medicine of the National Academies (or, if the 
Institute declines, another appropriate entity) under which 
the Institute (or other appropriate entity) not later than 
2 years after the date of enactment of this Act will— 
(1) complete a study on the extent to which bio-
medical research conducted or supported by Federal 
agencies is duplicative; and 
(2) submit a report to the Congress on the results 
of such study, including recommendations on how to 
prevent such duplication. 
SEC. 1023. REDUCING ADMINISTRATIVE BURDENS OF RE-
SEARCHERS. 
(a) PLAN PREPARATION
AND IMPLEMENTATION
OF 
MEASURES TO REDUCE ADMINISTRATIVE BURDENS.—The 
Director of the National Institutes of Health shall prepare 
a plan, including time frames, and implement measures to 
reduce the administrative burdens of researchers funded by 
the National Institutes of Health, taking into account the 
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recommendations, evaluations, and plans researched by the 
following entities: 
(1) The Scientific Management Review Board. 
(2) The National Academy of Sciences. 
(3) The 2007 and 2012 Faculty Burden Survey 
conducted by The Federal Demonstration Partner-
ship. 
(4) Relevant recommendations from the Research 
Business Models Working Group. 
(b) REPORT.—Not later than two years after the date 
of enactment of this Act, the Director of the National Insti-
tutes of Health shall submit to Congress a report on the 
extent to which the Director has implemented measures pur-
suant to subsection (a). 
SEC. 1024. EXEMPTION FOR THE NATIONAL INSTITUTES OF 
HEALTH FROM THE PAPERWORK REDUCTION 
ACT REQUIREMENTS. 
Section 3518(c)(1) of title 44, United States Code, is 
amended— 
(1) in subparagraph (C), by striking ‘‘; or’’ and 
inserting a semicolon; 
(2) in subparagraph (D), by striking the period 
at the end and inserting ‘‘; or’’; and 
(3) by inserting at the end the following new 
subparagraph: 
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‘‘(E) during the conduct of research by the Na-
tional Institutes of Health.’’. 
SEC. 1025. NIH TRAVEL. 
It is the sense of Congress that participation in or 
sponsorship of scientific conferences and meetings is essen-
tial to the mission of the National Institutes of Health. 
SEC. 1026. OTHER TRANSACTIONS AUTHORITY. 
Section 480 of the Public Health Service Act (42 
U.S.C. 287a) is amended— 
(1) in subsection (b), by striking ‘‘the appropria-
tion of funds as described in subsection (g)’’ and in-
serting ‘‘the availability of funds as described in sub-
section (f)’’; 
(2) in subsection (e)(3), by amending subpara-
graph (C) to read as follows: 
‘‘(C) OTHER TRANSACTIONS AUTHORITY.— 
The Director of the Center shall have other trans-
actions authority in entering into transactions 
to fund projects in accordance with the terms 
and conditions of this section.’’; 
(3) by striking subsection (f); and 
(4) by redesignating subsection (g) as subsection 
(f). 
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SEC. 1027. NCATS PHASE IIB RESTRICTION. 
Section 479 of the Public Health Service Act (42 
U.S.C. 287) is amended— 
(1) prior to making the amendments under 
paragraph (2), by striking ‘‘IIB’’ each place it ap-
pears and inserting ‘‘III’’; and 
(2) by striking ‘‘IIA’’ each place it appears and 
inserting ‘‘IIB’’. 
SEC. 1028. HIGH-RISK, HIGH-REWARD RESEARCH. 
Part B of title IV of the Public Health Service Act 
(42 U.S.C. 284 et seq.) is amended by adding at the end 
the following: 
‘‘SEC. 409K. HIGH-RISK, HIGH-REWARD RESEARCH PRO-
GRAM. 
‘‘The director of each national research institute shall, 
as appropriate— 
‘‘(1) establish programs to conduct or support re-
search projects that pursue innovative approaches to 
major contemporary challenges in biomedical research 
that involve inherent high risk, but have the potential 
to lead to breakthroughs; and 
‘‘(2) set aside a specific percentage of funding, to 
be determined by the Director of NIH for each na-
tional research institute, for such projects.’’. 
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SEC. 1029. SENSE OF CONGRESS ON INCREASED INCLUSION 
OF UNDERREPRESENTED COMMUNITIES IN 
CLINICAL TRIALS. 
It is the sense of Congress that the National Institute 
on Minority Health and Health Disparities (NIMHD) 
should include within its strategic plan ways to increase 
representation of underrepresented communities in clinical 
trials. 
Subtitle C—Supporting Young 
Emerging Scientists 
SEC. 1041. IMPROVEMENT OF LOAN REPAYMENT PROGRAMS 
OF THE NATIONAL INSTITUTES OF HEALTH. 
(a) IN GENERAL.—Part G of title IV of the Public 
Health Service (42 U.S.C. 288 et seq.) is amended— 
(1) by redesignating the second section 487F (42 
U.S.C. 288–6; relating to pediatric research loan re-
payment program) as section 487G; and 
(2) by inserting after section 487G, as so redesig-
nated, the following: 
‘‘SEC. 487H. LOAN REPAYMENT PROGRAM. 
‘‘(a) IN GENERAL.—The Secretary shall establish a 
program, based on workforce and scientific needs, of enter-
ing into contracts with qualified health professionals under 
which such health professionals agree to engage in research 
in consideration of the Federal Government agreeing to 
pay, for each year of engaging in such research, not more 
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than $50,000 of the principal and interest of the edu-
cational loans of such health professionals. 
‘‘(b) ADJUSTMENT FOR INFLATION.—Beginning with 
respect to fiscal year 2017, the Secretary may increase the 
maximum amount specified in subsection (a) by an amount 
that is determined by the Secretary, on an annual basis, 
to reflect inflation. 
‘‘(c) LIMITATION.—The Secretary may not enter into 
a contract with a health professional pursuant to subsection 
(a) unless such professional has a substantial amount of 
educational loans relative to income. 
‘‘(d) APPLICABILITY
OF CERTAIN PROVISIONS RE-
GARDING OBLIGATED SERVICE.—Except to the extent in-
consistent with this section, the provisions of sections 338B, 
338C, and 338E shall apply to the program established 
under this section to the same extent and in the same man-
ner as such provisions apply to the National Health Service 
Corps Loan Repayment Program established under section 
338B. 
‘‘(e) AVAILABILITY
OF APPROPRIATIONS.—Amounts 
appropriated for a fiscal year for contracts under subsection 
(a) are authorized to remain available until the expiration 
of the second fiscal year beginning after the fiscal year for 
which the amounts were appropriated.’’. 
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(b) UPDATE
OF OTHER LOAN REPAYMENT PRO-
GRAMS.— 
(1) Section 464z–5(a) of the Public Health Serv-
ice Act (42 U.S.C.285t–2(a)) is amended— 
(A) by striking ‘‘$35,000’’ and inserting 
‘‘$50,000’’; and 
(B) by adding at the end the following new 
sentence: ‘‘Subsection (b) of section 487H shall 
apply with respect to the maximum amount 
specified in this subsection in the same manner 
as it applies to the maximum amount specified 
in subsection (a) of such section.’’. 
(2) Section 487A(a) of such Act (42 U.S.C. 288– 
1(a)) is amended— 
(A) by striking ‘‘$35,000’’ and inserting 
‘‘$50,000’’; and 
(B) by adding at the end the following new 
sentence: ‘‘Subsection (b) of section 487H shall 
apply with respect to the maximum amount 
specified in this subsection in the same manner 
as it applies to the maximum amount specified 
in subsection (a) of such section.’’. 
(3) Section 487B(a) of such Act (42 U.S.C. 288– 
2(a)) is amended— 
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(A) by striking ‘‘$35,000’’ and inserting 
‘‘$50,000’’; and 
(B) by adding at the end the following new 
sentence: ‘‘Subsection (b) of section 487H shall 
apply with respect to the maximum amount 
specified in this subsection in the same manner 
as it applies to the maximum amount specified 
in such subsection (a) of such section.’’. 
(4) Section 487C(a)(1) of such Act (42 U.S.C. 
288–3(a)(1)) is amended— 
(A) by striking ‘‘$35,000’’ and inserting 
‘‘$50,000’’; and 
(B) by adding at the end the following new 
sentence: ‘‘Subsection (b) of section 487H shall 
apply with respect to the maximum amount 
specified in this paragraph in the same manner 
as it applies to the maximum amount specified 
in such subsection (a) of such section.’’. 
(5) Section 487E(a)(1) of such Act (42 U.S.C. 
288–5(a)(1)) is amended— 
(A) by striking ‘‘$35,000’’ and inserting 
‘‘$50,000’’; and 
(B) by adding at the end the following new 
sentence: ‘‘Subsection (b) of section 487H shall 
apply with respect to the maximum amount 
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specified in this paragraph in the same manner 
as it applies to the maximum amount specified 
in such subsection (a) of such section.’’. 
(6) Section 487F(a) of such Act (42 U.S.C. 288– 
5a(a)), as added by section 205 of Public Law 106– 
505, is amended— 
(A) by striking ‘‘$35,000’’ and inserting 
‘‘$50,000’’; and 
(B) by adding at the end the following new 
sentence: ‘‘Subsection (b) of section 487H shall 
apply with respect to the maximum amount 
specified in this subsection in the same manner 
as it applies to the maximum amount specified 
in such subsection (a) of such section.’’. 
(7) Section 487G of such Act (42 U.S.C. 288–6, 
as redesignated by section 1041(a)(1)), is further 
amended— 
(A) in subsection (a)(1), by striking 
‘‘$35,000’’ and inserting ‘‘$50,000’’; and 
(B) in subsection (b), by adding at the end 
the following new sentence: ‘‘Subsection (b) of 
section 487H shall apply with respect to the 
maximum amount specified in subsection (a)(1) 
in the same manner as it applies to the max-
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imum amount specified in such subsection (a) of 
such section.’’. 
SEC. 1042. REPORT. 
Not later than 18 months after the date of the enact-
ment of this Act, the Director of the National Institutes of 
Health shall submit to Congress a report on efforts of the 
National Institutes of Health to attract, retain, and develop 
emerging scientists. 
Subtitle D—Capstone Grant 
Program 
SEC. 1061. CAPSTONE AWARD. 
Part G of title IV of the Public Health Service Act 
(42 U.S.C. 288 et seq.) is amended by adding at the end 
the following: 
‘‘SEC. 490. CAPSTONE AWARD. 
‘‘(a) IN GENERAL.—The Secretary may make awards 
(each of which, hereafter in this section, referred to as a 
‘Capstone Award’) to support outstanding scientists who 
have been funded by the National Institutes of Health. 
‘‘(b) PURPOSE.—Capstone Awards shall be made to fa-
cilitate the successful transition or conclusion of research 
programs, or for other purposes, as determined by the Direc-
tor of NIH, in consultation with the directors of the na-
tional research institutes and national centers. 
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‘‘(c) DURATION
AND AMOUNT.—The duration and 
amount of each Capstone Award shall be determined by the 
Director of NIH in consultation with the directors of the 
national research institutes and national centers. 
‘‘(d) LIMITATION.—Individuals who have received a 
Capstone Award shall not be eligible to have principle in-
vestigator status on subsequent awards from the National 
Institutes of Health.’’. 
Subtitle 
E—Promoting 
Pediatric 
Research Through the National 
Institutes of Health 
SEC. 1081. NATIONAL PEDIATRIC RESEARCH NETWORK. 
Section 409D(d) of the Public Health Service Act (42 
U.S.C. 284h(d)) is amended— 
(1) in paragraph (1)— 
(A) by striking ‘‘in consultation with the 
Director of the Eunice Kennedy Shriver Na-
tional Institute of Child Health and Human De-
velopment and in collaboration with other ap-
propriate national research institutes and na-
tional centers that carry out activities involving 
pediatric research’’ and inserting ‘‘in collabora-
tion with the national research institutes and 
national centers that carry out activities involv-
ing pediatric research’’; 
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(B) by striking subparagraph (B); 
(C) by striking ‘‘may be comprised of, as 
appropriate’’ and all that follows through ‘‘the 
pediatric research consortia’’ and inserting 
‘‘may be comprised of, as appropriate, the pedi-
atric research consortia’’; and 
(D) by striking ‘‘; or’’ at the end and insert-
ing a period; and 
(2) in paragraph (1), paragraph (2)(A), the first 
sentence of paragraph (2)(E), and paragraph (4), by 
striking ‘‘may’’ each place it appears and inserting 
‘‘shall’’. 
SEC. 1082. GLOBAL PEDIATRIC CLINICAL STUDY NETWORK 
SENSE OF CONGRESS. 
It is the sense of Congress that— 
(1) the National Institutes of Health should en-
courage a global pediatric clinical study network 
through the allocation of grants, contracts, or coopera-
tive agreements to supplement the salaries of new and 
early investigators who participate in the global pedi-
atric clinical study network; 
(2) National Institutes of Health grants, con-
tracts, or cooperative agreements should be awarded, 
solely for the purpose of supplementing the salaries of 
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new and early investigators, to entities that partici-
pate in the global pediatric clinical study network; 
(3) the Food and Drug Administration should 
engage the European Medicines Agency and other for-
eign regulatory entities during the formation of the 
global pediatric clinical study network to encourage 
their participation; and 
(4) once a global pediatric clinical study network 
is established and becomes operational, the Food and 
Drug Administration should continue to engage the 
European Medicines Agency and other foreign regu-
latory entities to encourage and facilitate their par-
ticipation in the network with the goal of enhancing 
the global reach of the network. 
SEC. 1083. APPROPRIATE AGE GROUPINGS IN CLINICAL RE-
SEARCH. 
(a) INPUT FROM EXPERTS.—Not later than 180 days 
after the date of enactment of this Act, the Director of the 
National Institutes of Health shall convene a workshop of 
experts on pediatrics and experts on geriatrics to provide 
input on— 
(1) appropriate age groupings to be included in 
research studies involving human subjects; and 
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(2) acceptable scientific justifications for exclud-
ing participants from a range of age groups from 
human subjects research studies. 
(b) GUIDELINES.—Not later than 180 days after the 
conclusion of the workshop under subsection (a), the Direc-
tor of the National Institutes of Health shall publish guide-
lines— 
(1) addressing the consideration of age as an in-
clusion variable in research involving human subjects; 
and 
(2) identifying criteria for justifications for any 
age-related exclusions in such research. 
(c) PUBLIC AVAILABILITY OF FINDINGS AND CONCLU-
SIONS.—The Director of the National Institutes of Health 
shall— 
(1) make the findings and conclusions resulting 
from the workshop under subsection (a) available to 
the public on the website of the National Institutes of 
Health; and 
(2) not less than biennially, disclose to the public 
on such website the number of children included in re-
search that is conducted or supported by the National 
Institutes of Health, disaggregated by developmentally 
appropriate age group, race, and gender. 
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Subtitle F—Advancement of the Na-
tional Institutes of Health Re-
search and Data Access 
SEC. 1101. SHARING OF DATA GENERATED THROUGH NIH- 
FUNDED RESEARCH. 
Section 402 of the Public Health Service Act (42 
U.S.C. 282) (as amended by section 1021(2)) is further 
amended by adding at the end the following: 
‘‘(n) SHARING OF DATA GENERATED THROUGH NIH- 
FUNDED RESEARCH.— 
‘‘(1) AUTHORITY.—Subject to paragraph (2), the 
Director of NIH may require recipients of the award 
of an NIH grant or other financial support, provided 
that the research is fully funded through such grant 
or other support, to share scientific data generated 
from research conducted through such support for re-
search purposes. 
‘‘(2) LIMITATION.—The Director of NIH shall 
not require the sharing of data that is inconsistent 
with applicable law and policy protecting— 
‘‘(A) privacy and confidentiality; 
‘‘(B) proprietary interests; 
‘‘(C) business confidential information; 
‘‘(D) intellectual property rights; and 
‘‘(E) other relevant rights.’’. 
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SEC. 1102. STANDARDIZATION OF DATA IN CLINICAL TRIAL 
REGISTRY DATA BANK ON ELIGIBILITY FOR 
CLINICAL TRIALS. 
(a) STANDARDIZATION.— 
(1) IN GENERAL.—Section 402(j) of the Public 
Health Service Act (42 U.S.C. 282(j)) is amended— 
(A) by redesignating paragraph (7) as 
paragraph (8); and 
(B) by inserting after paragraph (6) the fol-
lowing: 
‘‘(7) STANDARDIZATION.—The Director of NIH 
shall— 
‘‘(A) ensure that the registry and results 
data bank is easily used by the public; 
‘‘(B) ensure that entries in the registry and 
results data bank are easily compared; 
‘‘(C) ensure that information required to be 
submitted to the registry and results data bank, 
including recruitment information under para-
graph (2)(A)(ii)(II), is submitted by persons and 
posted by the Director of NIH in a standardized 
format and includes at least— 
‘‘(i) the disease or indication being 
studied; 
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‘‘(ii) inclusion criteria such as age, 
gender, diagnosis or diagnoses, laboratory 
values, or imaging results; and 
‘‘(iii) exclusion criteria such as specific 
diagnosis or diagnoses, laboratory values, or 
prohibited medications; and 
‘‘(D) to the extent possible, in carrying out 
this paragraph, make use of standard health care 
terminologies, such as the International Classi-
fication of Diseases or the Current Procedural 
Terminology, that facilitate electronic matching 
to data in electronic health records or other rel-
evant health information technologies.’’. 
(2) CONFORMING
AMENDMENT.—Clause (iv) of 
section 402(j)(2)(B) of the Public Health Service Act 
(42 U.S.C. 282(j)(2)(B)) is hereby stricken. 
(b) CONSULTATION.—Not later than 90 days after the 
date of enactment of this Act, the Secretary of Health and 
Human Services shall consult with stakeholders (including 
patients, researchers, physicians, industry representatives, 
health information technology providers, the Food and Drug 
Administration, and standard setting organizations such as 
CDISC that have experience working with Federal agencies 
to standardize health data submissions) to receive advice 
on enhancements to the clinical trial registry data bank 
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under section 402(j) of the Public Health Service Act (42 
U.S.C. 282(j)) (including enhancements to usability, 
functionality, and search capability) that are necessary to 
implement paragraph (7) of section 402(j) of such Act, as 
added by subsection (a). 
(c) APPLICABILITY.—Not later than 18 months after 
the date of enactment of this Act, the Secretary of Health 
and Human Services shall begin implementation of para-
graph (7) of section 402(j) of the Public Health Service Act, 
as added by subsection (a). 
Subtitle G—Facilitating 
Collaborative Research 
SEC. 1121. CLINICAL TRIAL DATA SYSTEM. 
(a) ESTABLISHMENT.—The Secretary, acting through 
the Commissioner of Food and Drugs and the Director of 
the National Institutes of Health, shall enter into a coopera-
tive agreement, contract, or grant for a period of 7 years, 
to be known as the Clinical Trial Data System Agreement, 
with one or more eligible entities to implement a pilot pro-
gram with respect to all clinical trial data obtained from 
qualified clinical trials for purposes of registered users con-
ducting further research on such data. 
(b) APPLICATION.—Eligible entities seeking to enter 
into a cooperative agreement, contract, or grant with the 
Secretary under this section shall submit to the Secretary 
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an application in such time and manner, and containing 
such information, as the Secretary may require in accord-
ance with this section. The Secretary shall not enter into 
a cooperative agreement, contract, or grant under this sec-
tion with an eligible entity unless such entity submits an 
application including the following: 
(1) A certification that the eligible entity is not 
currently and does not plan to be involved in spon-
soring, operating, or participating in a clinical trial 
nor collaborating with another entity for the purposes 
of sponsoring, operating, or participating in a clin-
ical trial. 
(2) Information demonstrating that the eligible 
entity can compile clinical trial data in standardized 
formats using terminologies and standards that have 
been developed by recognized standards developing or-
ganizations with input from diverse stakeholder 
groups, and information demonstrating that the eligi-
ble entity can de-identify clinical trial data consistent 
with the requirements of section 164.514 of title 45, 
Code of Federal Regulations (or successor regula-
tions). 
(3) A description of the system the eligible entity 
will use to store and maintain such data, and infor-
mation demonstrating that this system will comply 
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with applicable standards and requirements for en-
suring the security of the clinical trial data. 
(4) A certification that the eligible entity will 
allow only registered users to access and use de-iden-
tified clinical trial data, gathered from qualified clin-
ical trials, and that the eligible entity will allow each 
registered user to access and use such data only after 
such registered user agrees in writing to the terms de-
scribed in (e)(4)(B), and such other carefully con-
trolled contractual terms as may be defined by the 
Secretary. 
(5) Evidence demonstrating the ability of the eli-
gible entity to ensure that registered users disseminate 
the results of the research conducted in accordance 
with this section to interested parties to serve as a 
guide to future medical product development or sci-
entific research. 
(6) The plan of the eligible entity for securing 
funding for the activities it would conduct under the 
clinical trial data system agreement from govern-
mental sources and private foundations, entities, and 
individuals. 
(7) Evidence demonstrating a proven track 
record of— 
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(A) being a neutral third party in working 
with medical product manufacturers, academic 
institutions, and the Food and Drug Adminis-
tration; and 
(B) having the ability to protect confiden-
tial data. 
(8) An agreement that the eligible entity will 
work with the Comptroller General of the United 
States for purposes of the study and report under sub-
section (d). 
(c) EXTENSION, EXPANSION, TERMINATION.—The Sec-
retary, acting through the Commissioner of Food and Drugs 
and the Director of the National Institutes of Health, upon 
the expiration of the 7-year period referred to in subsection 
(a), may extend (including permanently), expand, or termi-
nate the pilot program established under such subsection, 
in whole or in part. 
(d) STUDY AND REPORT.— 
(1) IN GENERAL.—The Comptroller General of 
the United States shall conduct a study and issue a 
report to the Congress and the Secretary with respect 
to the pilot program established under subsection (a), 
not later than 6 years after the date on which the 
pilot program is established under subsection (a). 
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(2) STUDY.—The study under paragraph (1) 
shall— 
(A) review the effectiveness of the pilot pro-
gram established under subsection (a); and 
(B) be designed to formulate recommenda-
tions on improvements to the program. 
(3) REPORT.—The report under paragraph (1) 
shall contain at least the following information: 
(A) The new discoveries, research inquiries, 
or clinical trials that have resulted from access-
ing clinical trial data under the pilot program 
established under subsection (a). 
(B) The number of times scientists have 
accessed such data, disaggregated by research 
area and clinical trial phase. 
(C) An analysis of whether the program has 
helped to reduce adverse events in clinical trials. 
(D) An analysis of whether scientists have 
raised any concerns about the burden of having 
to share data with the system established under 
the program and a description, if any, of such 
burden. 
(E) An analysis of privacy and data integ-
rity practices used in the program. 
(e) DEFINITIONS.—In this section: 
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(1) The term ‘‘eligible entity’’ means an entity 
that has experienced personnel with clinical and other 
technical expertise in the biomedical sciences and bio-
medical ethics and that is— 
(A) an institution of higher education (as 
such term is defined in section 1001 of the High-
er Education Act of 1965 (20 U.S.C. 1001)) or 
a consortium of such institutions; or 
(B) an organization described in section 
501(c)(3) of title 26 of the Internal Revenue Code 
of 1986 and exempt from tax under section 
501(a) of such title. 
(2) The term ‘‘medical product’’ means a drug 
(as defined in section 201(g) of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 331(g))), a device 
(as defined in section 201(h) of such Act (21 U.S.C. 
331(h)), a biological product (as defined in section 
351 of the Public Health Service Act (42 U.S.C. 
262)), or any combination thereof. 
(3) The term ‘‘qualified clinical trial’’ means a 
clinical trial sponsored solely by an agency of the De-
partment of Health and Human Services with respect 
to a medical product— 
(A) that— 
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(i) was approved or cleared under sec-
tion 505, 510(k), or 515, or has an exemp-
tion for investigational use in effect under 
section 505 or 520(m), of the Federal Food, 
Drug, and Cosmetic Act (42 U.S.C. 301 et 
seq.); or 
(ii) was licensed under section 351 of 
the Public Health Service Act (42 U.S.C. 
262) or has an exemption for investiga-
tional use in effect under such section 351; 
or 
(B) that is an investigational product for 
which the original development was discontinued 
and with respect to which— 
(i) no additional work to support ap-
proval, licensure, or clearance of such med-
ical product is being or is planned to be un-
dertaken by the sponsor of the original de-
velopment program, its successors, assigns, 
or collaborators; and 
(ii) the sponsor of the original inves-
tigational development program has pro-
vided its consent to the Secretary for inclu-
sion of data regarding such product in the 
system established under this section. 
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(4) The term ‘‘registered user’’ means a scientific 
or medical researcher who has— 
(A) a legitimate biomedical research pur-
pose for accessing information from the clinical 
trials data system and has appropriate quali-
fications to conduct such research; and 
(B) agreed in writing not to transfer to any 
other person that is not a registered user de-iden-
tified clinical trial data from qualified clinical 
trials accessed through an eligible entity, use 
such data for reasons not specified in the re-
search proposal, or seek to re-identify qualified 
clinical trial participants. 
(5) The term ‘‘Secretary’’ means the Secretary of 
Health and Human Services. 
SEC. 1122. NATIONAL NEUROLOGICAL DISEASES SURVEIL-
LANCE SYSTEM. 
Part P of title III of the Public Health Service Act 
(42 U.S.C. 280g et seq.) is amended by adding at the end 
the following: 
‘‘SEC. 399V–6 SURVEILLANCE OF NEUROLOGICAL DISEASES. 
‘‘(a) IN GENERAL.—The Secretary, acting through the 
Director of the Centers for Disease Control and Prevention 
and in coordination with other agencies as determined ap-
propriate by the Secretary, shall— 
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‘‘(1) enhance and expand infrastructure and ac-
tivities to track the epidemiology of neurological dis-
eases, including multiple sclerosis and Parkinson’s 
disease; and 
‘‘(2) incorporate information obtained through 
such activities into a statistically sound, scientifically 
credible, integrated surveillance system, to be known 
as the National Neurological Diseases Surveillance 
System. 
‘‘(b) RESEARCH.—The Secretary shall ensure that the 
National Neurological Diseases Surveillance System is de-
signed in a manner that facilitates further research on neu-
rological diseases. 
‘‘(c) CONTENT.—In carrying out subsection (a), the 
Secretary— 
‘‘(1) shall provide for the collection and storage 
of information on the incidence and prevalence of 
neurological diseases in the United States; 
‘‘(2) to the extent practicable, shall provide for 
the collection and storage of other available informa-
tion on neurological diseases, such as information 
concerning— 
‘‘(A) demographics and other information 
associated or possibly associated with neuro-
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logical diseases, such as age, race, ethnicity, sex, 
geographic location, and family history; 
‘‘(B) risk factors associated or possibly asso-
ciated with neurological diseases, including ge-
netic and environmental risk factors; and 
‘‘(C) diagnosis and progression markers; 
‘‘(3) may provide for the collection and storage 
of information relevant to analysis on neurological 
diseases, such as information concerning— 
‘‘(A) the epidemiology of the diseases; 
‘‘(B) the natural history of the diseases; 
‘‘(C) the prevention of the diseases; 
‘‘(D) the detection, management, and treat-
ment approaches for the diseases; and 
‘‘(E) the development of outcomes measures; 
and 
‘‘(4) may address issues identified during the 
consultation process under subsection (d). 
‘‘(d) CONSULTATION.—In carrying out this section, the 
Secretary shall consult with individuals with appropriate 
expertise, including— 
‘‘(1) epidemiologists with experience in disease 
surveillance or registries; 
‘‘(2) representatives of national voluntary health 
associations that— 
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‘‘(A) focus on neurological diseases, includ-
ing multiple sclerosis and Parkinson’s disease; 
and 
‘‘(B) have demonstrated experience in re-
search, care, or patient services; 
‘‘(3) health information technology experts or 
other information management specialists; 
‘‘(4) clinicians with expertise in neurological 
diseases; and 
‘‘(5) research scientists with experience con-
ducting translational research or utilizing surveil-
lance systems for scientific research purposes. 
‘‘(e) GRANTS.—The Secretary may award grants to, 
or enter into contracts or cooperative agreements with, pub-
lic or private nonprofit entities to carry out activities under 
this section. 
‘‘(f) COORDINATION WITH OTHER FEDERAL, STATE, 
AND LOCAL AGENCIES.—Subject to subsection (h), the Sec-
retary shall make information and analysis in the National 
Neurological Diseases Surveillance System available, as ap-
propriate— 
‘‘(1) to Federal departments and agencies, such 
as the National Institutes of Health, the Food and 
Drug Administration, the Centers for Medicare & 
Medicaid Services, the Agency for Healthcare Re-
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search and Quality, the Department of Veterans Af-
fairs, and the Department of Defense; and 
‘‘(2) to State and local agencies. 
‘‘(g) PUBLIC ACCESS.—Subject to subsection (h), the 
Secretary shall make information and analysis in the Na-
tional Neurological Diseases Surveillance System available, 
as appropriate, to the public, including researchers. 
‘‘(h) PRIVACY.—The Secretary shall ensure that pri-
vacy and security protections applicable to the National 
Neurological Diseases Surveillance System are at least as 
stringent as the privacy and security protections under 
HIPAA privacy and security law (as defined in section 
3009(a)(2)). 
‘‘(i) REPORT.—Not later than 4 years after the date 
of the enactment of this section, the Secretary shall submit 
a report to the Congress concerning the implementation of 
this section. Such report shall include information on— 
‘‘(1) the development and maintenance of the 
National Neurological Diseases Surveillance System; 
‘‘(2) the type of information collected and stored 
in the System; 
‘‘(3) the use and availability of such informa-
tion, including guidelines for such use; and 
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‘‘(4) the use and coordination of databases that 
collect or maintain information on neurological dis-
eases. 
‘‘(j) DEFINITION.—In this section, the term ‘national 
voluntary health association’ means a national nonprofit 
organization with chapters, other affiliated organizations, 
or networks in States throughout the United States. 
‘‘(k) AUTHORIZATION OF APPROPRIATIONS.—To carry 
out this section, there is authorized to be appropriated 
$5,000,000 for each of fiscal years 2016 through 2020.’’. 
SEC. 1123. DATA ON NATURAL HISTORY OF DISEASES. 
(a) SENSE OF CONGRESS.—It is the sense of the Con-
gress that studies on the natural history of diseases can help 
to facilitate and expedite the development of medical prod-
ucts for such diseases. 
(b) AUTHORITY.—Part A of title II of the Public 
Health Service Act (42 U.S.C. 202 et seq.) is amended by 
adding at the end the following: 
‘‘SEC. 229A. DATA ON NATURAL HISTORY OF DISEASES. 
‘‘(a) IN GENERAL.—The Secretary may, for the pur-
poses described in subsection (b)— 
‘‘(1) participate in public-private partnerships 
engaged in one or more activities specified in sub-
section (c); and 
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‘‘(2) award grants to patient advocacy groups or 
other organizations determined appropriate by the 
Secretary. 
‘‘(b) PURPOSES DESCRIBED.—The purposes described 
in this subsection are to establish or facilitate the collection, 
maintenance, analysis, and interpretation of data regard-
ing the natural history of diseases, with a particular focus 
on rare diseases. 
‘‘(c) ACTIVITIES
OF
PUBLIC-PRIVATE
PARTNER-
SHIPS.—The activities of public-private partnerships in 
which the Secretary may participate for purposes of this 
section include— 
‘‘(1) cooperating with other entities that sponsor 
or maintain disease registries, including disease reg-
istries and disease registry platforms for rare dis-
eases; 
‘‘(2) developing or enhancing a secure informa-
tion technology system that— 
‘‘(A) has the capacity to support data needs 
across a wide range of disease studies; 
‘‘(B) is easily modified as knowledge is 
gained during such studies; and 
‘‘(C) is capable of handling increasing 
amounts of data as more studies are carried out; 
and 
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‘‘(3) providing advice to clinical researchers, pa-
tient advocacy groups, and other entities with respect 
to— 
‘‘(A) the design and conduct of disease stud-
ies; 
‘‘(B) the modification of any such ongoing 
studies; and 
‘‘(C) addressing associated patient privacy 
issues. 
‘‘(d) AVAILABILITY OF DATA ON NATURAL HISTORY OF 
DISEASES.—Data relating to the natural history of diseases 
obtained, aggregated, or otherwise maintained by a public- 
private partnership in which the Secretary participates 
under subsection (a) shall be made available, consistent 
with otherwise applicable Federal and State privacy laws, 
to the public (including patient advocacy groups, research-
ers, and drug developers) to help to facilitate and expedite 
medical product development programs. 
‘‘(e) CONFIDENTIALITY.—Notwithstanding subsection 
(d), nothing in this section authorizes the disclosure of any 
information that is a trade secret or commercial or finan-
cial information that is privileged or confidential and sub-
ject to section 552(b)(4) of title 5, United States Code, or 
section 1905 of title 18, United States Code. 
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‘‘(f) AUTHORIZATION OF APPROPRIATIONS.—There is 
authorized to be appropriated to carry out this section 
$5,000,000 for each of fiscal years 2016 through 2020.’’. 
SEC. 1124. ACCESSING, SHARING, AND USING HEALTH DATA 
FOR RESEARCH PURPOSES. 
(a) IN GENERAL.—(1) The HITECH Act (title XIII 
of division A of Public Law 111–5) is amended by adding 
at the end of subtitle D of such Act (42 U.S.C. 17921 et 
seq.) the following: 
‘‘PART 4—ACCESSING, SHARING, AND USING 
HEALTH DATA FOR RESEARCH PURPOSES 
‘‘SEC. 13441. REFERENCES. 
‘‘In this part: 
‘‘(1) THE RULE.—References to ‘the Rule’ refer 
to part 160 or part 164, as appropriate, of title 45, 
Code of Federal Regulations (or any successor regula-
tion). 
‘‘(2) PART 164.—References to a specified section 
of ‘part 164’, refer to such specified section of part 
164 of title 45, Code of Federal Regulations (or any 
successor section). 
‘‘SEC. 13442. DEFINING HEALTH DATA RESEARCH AS PART 
OF HEALTH CARE OPERATIONS. 
‘‘(a) IN GENERAL.—Subject to subsection (b), the Sec-
retary shall revise or clarify the Rule to allow the use and 
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disclosure of protected health information by a covered enti-
ty for research purposes, including studies whose purpose 
is to obtain generalizable knowledge, to be treated as the 
use and disclosure of such information for health care oper-
ations described in subparagraph (1) of the definition of 
health care operations in section 164.501 of part 164. 
‘‘(b) MODIFICATIONS TO RULES FOR DISCLOSURES 
FOR HEALTH CARE OPERATIONS.—In applying section 
164.506 of part 164 to the disclosure of protected health in-
formation described in subsection (a)— 
‘‘(1) the Secretary shall revise or clarify the Rule 
so that the disclosure may be made by the covered en-
tity to only— 
‘‘(A) another covered entity for health care 
operations (as defined in section 164.501 of part 
164); 
‘‘(B) a business associate that has entered 
into a contract under section 164.504(e) of part 
164 with a disclosing covered entity to perform 
health care operations; or 
‘‘(C) a business associate that has entered 
into a contract under section 164.504(e) of part 
164 for the purpose of data aggregation (as de-
fined in section 164.501 of part 164); and 
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‘‘(2) the Secretary shall further revise or clarify 
the Rule so that the limitation specified by section 
164.506(c)(4) of part 164 does not apply to disclo-
sures that are described by subsection (a). 
‘‘(c) RULE OF CONSTRUCTION.—This section shall not 
be construed as prohibiting or restricting a use or disclosure 
of protected health information for research purposes that 
is otherwise permitted under part 164. 
‘‘SEC. 13443. TREATING DISCLOSURES OF PROTECTED 
HEALTH INFORMATION FOR RESEARCH SIMI-
LARLY TO DISCLOSURES OF SUCH INFORMA-
TION FOR PUBLIC HEALTH PURPOSES. 
‘‘(a) REMUNERATION.—The Secretary shall revise or 
clarify the Rule so that disclosures of protected health infor-
mation for research purposes are not subject to the limita-
tion 
on 
remuneration 
described 
in 
section 
164.502(a)(5)(ii)(B)(2)(ii) of part 164. 
‘‘(b) PERMITTED USES AND DISCLOSURES.—The Sec-
retary shall revise or clarify the Rule so that research ac-
tivities, including comparative research activities, related 
to the quality, safety, or effectiveness of a product or activ-
ity that is regulated by the Food and Drug Administration 
are included as public health activities for purposes of 
which a covered entity may disclose protected health infor-
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mation to a person described in section 164.512(b)(1)(iii) 
of part 164. 
‘‘SEC. 13444. PERMITTING REMOTE ACCESS TO PROTECTED 
HEALTH INFORMATION BY RESEARCHERS. 
‘‘The Secretary shall revise or clarify the Rule so that 
subparagraph (B) of section 164.512(i)(1)(ii) of part 164 
(prohibiting the removal of protected health information by 
a researcher) shall not prohibit remote access to health in-
formation by a researcher so long as— 
‘‘(1) appropriate security and privacy safe-
guards are maintained by the covered entity and the 
researcher; and 
‘‘(2) the protected health information is not cop-
ied or otherwise retained by the researcher. 
‘‘SEC. 13445. ALLOWING ONE-TIME AUTHORIZATION OF USE 
AND DISCLOSURE OF PROTECTED HEALTH 
INFORMATION FOR RESEARCH PURPOSES. 
‘‘(a) IN GENERAL.—The Secretary shall revise or clar-
ify the Rule to specify that an authorization for the use 
or disclosure of protected health information, with respect 
to an individual, for future research purposes shall be 
deemed to contain a sufficient description of the purpose 
of the use or disclosure if the authorization— 
‘‘(1) sufficiently describes the purposes such that 
it would be reasonable for the individual to expect 
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that the protected health information could be used or 
disclosed for such future research; 
‘‘(2) either— 
‘‘(A) states that the authorization will ex-
pire on a particular date or on the occurrence of 
a particular event; or 
‘‘(B) states that the authorization will re-
main valid unless and until it is revoked by the 
individual; and 
‘‘(3) provides instruction to the individual on 
how to revoke such authorization at any time. 
‘‘(b) REVOCATION
OF
AUTHORIZATION.—The Sec-
retary shall revise or clarify the Rule to specify that, if an 
individual revokes an authorization for future research pur-
poses such as is described by subsection (a), the covered enti-
ty may not make any further uses or disclosures based on 
that authorization, except, as provided in paragraph (b)(5) 
of section 164.508 of part 164, to the extent that the covered 
entity has taken action in reliance on the authorization.’’. 
(2) The table of sections in section 13001(b) of such 
Act is amended by adding at the end of the items relating 
to subtitle D the following new items: 
‘‘PART 4—ACCESSING, SHARING, AND USING HEALTH DATA FOR RESEARCH 
PURPOSES 
‘‘Sec. 13441. References. 
‘‘Sec. 13442. Defining health data research as part of health care operations. 
‘‘Sec. 13443. Treating disclosures of protected health information for research 
similarly to disclosures of such information for public health 
purposes. 
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‘‘Sec. 13444. Permitting remote access to protected health information by re-
searchers. 
‘‘Sec. 13445. Allowing one-time authorization of use and disclosure of protected 
health information for research purposes.’’. 
(b) REVISION OF REGULATIONS.—Not later than 12 
months after the date of the enactment of this Act, the Sec-
retary of Health and Human Services shall revise and clar-
ify the provisions of title 45, Code of Federal Regulations, 
for consistency with part 4 of subtitle D of the HITECH 
Act, as added by subsection (a). 
Subtitle H—Council for 21st 
Century Cures 
SEC. 1141. COUNCIL FOR 21ST CENTURY CURES. 
Title II of the Public Health Service Act (42 U.S.C. 
202 et seq.) is amended by adding at the end the following: 
‘‘PART E—COUNCIL FOR 21ST CENTURY CURES 
‘‘SEC. 281. ESTABLISHMENT. 
‘‘A nonprofit corporation to be known as the Council 
for 21st Century Cures (referred to in this part as the 
‘Council’) shall be established in accordance with this sec-
tion. The Council shall be a public-private partnership 
headed by an Executive Director (referred to in this part 
as the ‘Executive Director’), appointed by the members of 
the Board of Directors. The Council shall not be an agency 
or instrumentality of the United States Government. 
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‘‘SEC. 281A. PURPOSE. 
‘‘The purpose of the Council is to accelerate the dis-
covery, development, and delivery in the United States of 
innovative cures, treatments, and preventive measures for 
patients. 
‘‘SEC. 281B. DUTIES. 
‘‘For the purpose described in section 281A, the Coun-
cil shall— 
‘‘(1) foster collaboration and coordination among 
the entities that comprise the Council, including aca-
demia, government agencies, industry, health care 
payors and providers, patient advocates, and others 
engaged in the cycle of discovery, development, and 
delivery of life-saving and health-enhancing innova-
tive interventions; 
‘‘(2) undertake communication and dissemina-
tion activities; 
‘‘(3) publish information on the activities funded 
under section 281D; 
‘‘(4) establish a strategic agenda for accelerating 
the discovery, development, and delivery in the 
United States of innovative cures, treatments, and 
preventive measures for patients; 
‘‘(5) identify gaps and opportunities within and 
across the discovery, development, and delivery cycle; 
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‘‘(6) develop and propose recommendations based 
on the gaps and opportunities so identified; 
‘‘(7) facilitate the interoperability of the compo-
nents of the discovery, development, and delivery 
cycle; 
‘‘(8) propose recommendations that will facilitate 
precompetitive collaboration; 
‘‘(9) identify opportunities to work with, but not 
duplicate the efforts of, nonprofit organizations and 
other public-private partnerships; and 
‘‘(10) identify opportunities for collaboration 
with organizations operating outside of the United 
States, such as the Innovative Medicines Initiative of 
the European Union. 
‘‘SEC. 281C. ORGANIZATION; ADMINISTRATION. 
‘‘(a) BOARD OF DIRECTORS.— 
‘‘(1) ESTABLISHMENT.— 
‘‘(A) IN GENERAL.—The Council shall have 
a Board of Directors (in this part referred to as 
the ‘Board of Directors’), which shall be com-
posed of the ex officio members under subpara-
graph (B) and the appointed members under 
subparagraph (C). All members of the Board 
shall be voting members. 
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‘‘(B) EX OFFICIO MEMBERS.—The ex officio 
members of the Board shall be the following indi-
viduals or their designees: 
‘‘(i) The Director of the National Insti-
tutes of Health. 
‘‘(ii) The Commissioner of Food and 
Drugs. 
‘‘(iii) The Administrator of the Centers 
for Medicare & Medicaid Services. 
‘‘(iv) The heads of five other Federal 
agencies deemed by the Secretary to be en-
gaged in biomedical research and develop-
ment. 
‘‘(C) 
APPOINTED
MEMBERS.—The 
ap-
pointed members of the Board shall consist of 17 
individuals, of whom— 
‘‘(i) 8 shall be appointed by the Comp-
troller General of the United States from a 
list of nominations submitted by leading 
trade associations— 
‘‘(I) 4 of whom shall be represent-
atives of the biopharmaceutical indus-
try; 
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‘‘(II) 2 of whom shall be rep-
resentatives of the medical device in-
dustry; and 
‘‘(III) 2 of whom shall be rep-
resentatives of the information and 
digital technology industry; and 
‘‘(ii) 9 shall be appointed by the 
Comptroller General of the United States, 
after soliciting nominations— 
‘‘(I) 2 of whom shall be represent-
atives of academic researchers; 
‘‘(II) 3 of whom shall be rep-
resentatives of patients; 
‘‘(III) 2 of whom shall be rep-
resentatives of health care providers; 
and 
‘‘(IV) 2 of whom shall be rep-
resentatives of health care plans and 
insurers. 
‘‘(D) CHAIR.—The Chair of the Board shall 
be selected by the members of the Board by ma-
jority vote from among the members of the 
Board. 
‘‘(2) TERMS AND VACANCIES.— 
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‘‘(A) IN GENERAL.—The term of office of 
each member of the Board appointed under 
paragraph (1)(C) shall be 5 years. 
‘‘(B) VACANCY.—Any vacancy in the mem-
bership of the Board— 
‘‘(i) shall not affect the power of the re-
maining members to execute the duties of 
the Board; and 
‘‘(ii) shall be filled by appointment by 
the appointed members described in para-
graph (1)(C) by majority vote. 
‘‘(C) PARTIAL TERM.—If a member of the 
Board does not serve the full term applicable 
under subparagraph (A), the individual ap-
pointed under subparagraph (B) to fill the re-
sulting vacancy shall be appointed for the re-
mainder of the term of the predecessor of the in-
dividual. 
‘‘(3) RESPONSIBILITIES.—Not later than 90 days 
after the date on which the Council is incorporated 
and its Board of Directors is fully constituted, the 
Board of Directors shall establish bylaws and policies 
for the Council that— 
‘‘(A) are published in the Federal Register 
and available for public comment; 
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‘‘(B) establish policies for the selection and, 
as applicable, appointment of— 
‘‘(i) the officers, employees, agents, and 
contractors of the Council; and 
‘‘(ii) the members of any committees of 
the Council; 
‘‘(C) establish policies, including ethical 
standards, for the conduct of programs and other 
activities under section 281D; and 
‘‘(D) establish specific duties of the Execu-
tive Director. 
‘‘(4) MEETINGS.— 
‘‘(A) IN GENERAL.—The Board of Directors 
shall— 
‘‘(i) meet on a quarterly basis; and 
‘‘(ii) submit to Congress, and make 
publicly available, the minutes of such meet-
ings. 
‘‘(B) AGENDA.—The Board of Directors 
shall, not later than 3 months after the incorpo-
ration of the Council— 
‘‘(i) issue an agenda (in this part re-
ferred to as the ‘agenda’) outlining how the 
Council will achieve the purpose described 
in section 281A; and 
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‘‘(ii) annually thereafter, in consulta-
tion with the Executive Director, review 
and update such agenda. 
‘‘(b) APPOINTMENT AND INCORPORATION.—Not later 
than 6 months after the date of enactment of the 21st Cen-
tury Cures Act— 
‘‘(1) the Comptroller General of the United 
States shall appoint the appointed members of the 
Board of Directors under subsection (a)(1)(C); and 
‘‘(2) the ex officio members of the Board of Di-
rectors under subsection (a)(1)(B) shall serve as 
incorporators and shall take whatever actions are nec-
essary to incorporate the Council. 
‘‘(c) NONPROFIT STATUS.—In carrying out this part, 
the Board of Directors shall establish such policies and by-
laws, and the Executive Director shall carry out such ac-
tivities, as may be necessary to ensure that the Council 
maintains status as an organization that— 
‘‘(1) is described in subsection (c)(3) of section 
501 of the Internal Revenue Code of 1986; and 
‘‘(2) is, under subsection (a) of such section, ex-
empt from taxation. 
‘‘(d) EXECUTIVE DIRECTOR.—The Executive Director 
shall— 
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‘‘(1) be the chief executive officer of the Council; 
and 
‘‘(2) subject to the oversight of the Board of Di-
rectors, be responsible for the day-to-day management 
of the Council. 
‘‘SEC. 281D. OPERATIONAL ACTIVITIES AND ASSISTANCE. 
‘‘(a) IN GENERAL.—The Council shall establish a suffi-
cient operational infrastructure to fulfill the duties specified 
in section 281B. 
‘‘(b) PRIVATE SECTOR MATCHING FUNDS.—The Coun-
cil may accept financial or in-kind support from partici-
pating entities or private foundations or organizations 
when such support is deemed appropriate. 
‘‘SEC. 281E. TERMINATION; REPORT. 
‘‘(a) IN GENERAL.—The Council shall terminate on 
September 30, 2023. 
‘‘(b) REPORT.—Not later than one year after the date 
on which the Council is established and each year thereafter, 
the Executive Director shall submit to the appropriate con-
gressional committees a report on the performance of the 
Council. In preparing such report, the Council shall consult 
with a nongovernmental consultant with appropriate exper-
tise. 
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‘‘SEC. 281F. FUNDING. 
‘‘For the each of fiscal years 2016 through 2023, there 
is authorized to be appropriated $10,000,000 to the Council 
for purposes of carrying out the duties of the Council under 
this part.’’. 
TITLE II—DEVELOPMENT 
Subtitle A—Patient-Focused Drug 
Development 
SEC. 2001. DEVELOPMENT AND USE OF PATIENT EXPERI-
ENCE DATA TO ENHANCE STRUCTURED RISK- 
BENEFIT ASSESSMENT FRAMEWORK. 
(a) IN GENERAL.—Section 505 of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 355) is amended— 
(1) in subsection (d), by striking ‘‘The Secretary 
shall implement’’ and all that follows through ‘‘pre-
market approval of a drug.’’; and 
(2) by adding at the end the following new sub-
sections: 
‘‘(x) 
STRUCTURED
RISK-BENEFIT
ASSESSMENT 
FRAMEWORK.— 
‘‘(1) IN GENERAL.—The Secretary shall imple-
ment a structured risk-benefit assessment framework 
in the new drug approval process— 
‘‘(A) to facilitate the balanced consideration 
of benefits and risks; and 
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‘‘(B) to develop and implement a consistent 
and systematic approach to the discussion of, 
regulatory decisionmaking with respect to, and 
the communication of, the benefits and risks of 
new drugs. 
‘‘(2) RULE
OF
CONSTRUCTION.—Nothing in 
paragraph (1) shall alter the criteria for evaluating 
an application for premarket approval of a drug. 
‘‘(y) DEVELOPMENT AND USE OF PATIENT EXPERI-
ENCE DATA TO ENHANCE STRUCTURED RISK-BENEFIT AS-
SESSMENT FRAMEWORK.— 
‘‘(1) IN
GENERAL.—Not later than two years 
after the date of the enactment of this subsection, the 
Secretary shall establish and implement processes 
under which— 
‘‘(A) an entity seeking to develop patient ex-
perience data may submit to the Secretary— 
‘‘(i) initial research concepts for feed-
back from the Secretary; and 
‘‘(ii) with respect to patient experience 
data collected by the entity, draft guidance 
documents, completed data, and summaries 
and analyses of such data; 
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‘‘(B) the Secretary may request such an en-
tity to submit such documents, data, and sum-
maries and analyses; and 
‘‘(C) patient experience data may be devel-
oped and used to enhance the structured risk- 
benefit assessment framework under subsection 
(x). 
‘‘(2) PATIENT EXPERIENCE DATA.—In this sub-
section, the term ‘patient experience data’ means data 
collected by patients, parents, caregivers, patient ad-
vocacy organizations, disease research foundations, 
medical researchers, research sponsors, or other par-
ties determined appropriate by the Secretary that is 
intended to facilitate or enhance the Secretary’s risk- 
benefit assessments, including information about the 
impact of a disease or a therapy on patients’ lives.’’. 
(b) GUIDANCE.— 
(1) IN GENERAL.—The Secretary of Health and 
Human Services shall publish guidance on the imple-
mentation of subsection (y) of section 505 of the Fed-
eral Food, Drug, and Cosmetic Act (21 U.S.C. 355), 
as added by subsection (a). Such guidance shall in-
clude— 
(A) with respect to draft guidance docu-
ments, data, or summaries and analyses sub-
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mitted to the Secretary under paragraph (1)(A) 
of such subsection, guidance— 
(i) specifying the timelines for the re-
view of such documents, data, or summaries 
and analyses by the Secretary; and 
(ii) on how the Secretary will use such 
documents, data, or summaries and anal-
yses to update any guidance documents 
published under this subsection or publish 
new guidance; 
(B) with respect to the collection and anal-
ysis of patient experience data (as defined in 
paragraph (2) of such subsection (y)), guidance 
on— 
(i) methodological considerations for 
the collection of patient experience data, 
which may include structured approaches to 
gathering information on— 
(I) the experience of a patient liv-
ing with a particular disease; 
(II) the burden of living with or 
managing the disease; 
(III) the impact of the disease on 
daily life and long-term functioning; 
and 
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(IV) the effect of current thera-
peutic options on different aspects of 
the disease; and 
(ii) the establishment and maintenance 
of registries designed to increase under-
standing of the natural history of a disease; 
(C) methodological approaches that may be 
used to assess patients’ beliefs with respect to the 
benefits and risks in the management of the pa-
tient’s disease; and 
(D) methodologies, standards, and potential 
experimental designs for patient-reported out-
comes. 
(2) TIMING.—Not later than 3 years after the 
date of the enactment of this Act, the Secretary of 
Health and Human Services shall issue draft guid-
ance on the implementation of subsection (y) of sec-
tion 505 of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C. 355), as added by subsection (a). The Sec-
retary shall issue final guidance on the implementa-
tion of such subsection not later than one year after 
the date on which the comment period for the draft 
guidance closes. 
(3) WORKSHOPS.— 
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(A) IN GENERAL.—Not later than 6 months 
after the date of the enactment of this Act and 
once every 6 months during the following 12- 
month period, the Secretary of Health and 
Human Services shall convene a workshop to ob-
tain input regarding methodologies for devel-
oping the guidance under paragraph (1), includ-
ing the collection of patient experience data. 
(B) ATTENDEES.—A workshop convened 
under this paragraph shall include— 
(i) patients; 
(ii) representatives from patient advo-
cacy organizations, biopharmaceutical com-
panies, and disease research foundations; 
(iii) representatives of the reviewing 
divisions of the Food and Drug Administra-
tion; and 
(iv) methodological experts with sig-
nificant expertise in patient experience 
data. 
(4) PUBLIC MEETING.—Not later than 90 days 
after the date on which the draft guidance is pub-
lished under this subsection, the Secretary of Health 
and Human Services shall convene a public meeting 
to solicit input on the guidance. 
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Subtitle B—Qualification and Use 
of Drug Development Tools 
SEC. 2021. QUALIFICATION OF DRUG DEVELOPMENT TOOLS. 
(a) FINDINGS.—Congress finds the following: 
(1) Development of new drugs has become in-
creasingly challenging and resource intensive. 
(2) Development of drug development tools can 
benefit the availability of new medical therapies by 
helping to translate scientific discoveries into clinical 
applications. 
(3) Biomedical research consortia (as defined in 
section 507(f) of the Federal Food, Drug, and Cos-
metic Act, as added by subsection (c)) can play a val-
uable role in helping to develop and qualify drug de-
velopment tools. 
(b) SENSE OF CONGRESS.—It is the sense of Congress 
that— 
(1) Congress should promote and facilitate a col-
laborative effort among the biomedical research con-
sortia described in subsection (a)(3)— 
(A) to develop, through a transparent public 
process, data standards and scientific approaches 
to data collection accepted by the medical and 
clinical research community for purposes of 
qualifying drug development tools; 
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(B) to coordinate efforts toward developing 
and qualifying drug development tools in key 
therapeutic areas; and 
(C) to encourage the development of acces-
sible databases for collecting relevant drug devel-
opment tool data for such purposes; and 
(2) an entity seeking to qualify a drug develop-
ment tool should be encouraged, in addition to con-
sultation with the Secretary, to consult with bio-
medical research consortia and other individuals and 
entities with expert knowledge and insights that may 
assist the requestor and benefit the process for such 
qualification. 
(c) QUALIFICATION OF DRUG DEVELOPMENT TOOLS.— 
Chapter V of the Federal Food, Drug, and Cosmetic Act 
is amended by inserting after section 506F the following 
new section: 
‘‘SEC. 507. QUALIFICATION OF DRUG DEVELOPMENT TOOLS. 
‘‘(a) PROCESS FOR QUALIFICATION.— 
‘‘(1) IN GENERAL.—The Secretary shall establish 
a process for the qualification of drug development 
tools for a proposed context of use under which— 
‘‘(A)(i) a requestor initiates such process by 
submitting a letter of intent to the Secretary; 
and 
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‘‘(B) ACCEPTANCE
FACTORS; NONACCEPT-
ANCE.—The Secretary shall determine whether to 
accept a qualification submission based on fac-
tors which may include the scientific merit of the 
submission and the available resources of the 
Food and Drug Administration to review the 
qualification submission. A determination not to 
accept a submission under paragraph (1) shall 
not be construed as a final determination by the 
Secretary under this section regarding the quali-
fication of a drug development tool for its pro-
posed context of use. 
‘‘(C) PRIORITIZATION
OF
QUALIFICATION 
REVIEW.—The Secretary may prioritize the re-
view of a full qualification package submitted 
under paragraph (1) with respect to a drug de-
velopment tool, based on factors determined ap-
propriate by the Secretary, including— 
‘‘(i) as applicable, the severity, rarity, 
or prevalence of the disease or condition 
targeted by the drug development tool and 
the availability or lack of alternative treat-
ments for such disease or condition; and 
‘‘(ii) the identification, by the Sec-
retary or by biomedical research consortia 
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and other expert stakeholders, of such a 
drug development tool and its proposed con-
text of use as a public health priority. 
‘‘(D) ENGAGEMENT
OF
EXTERNAL
EX-
PERTS.—The Secretary may, for purposes of the 
review of qualification submissions, through the 
use of cooperative agreements, grants, or other 
appropriate mechanisms, consult with bio-
medical research consortia and may consider the 
recommendations of such consortia with respect 
to the review of any qualification plan submitted 
under paragraph (1) or the review of any full 
qualification package under paragraph (3). 
‘‘(3) REVIEW
OF
FULL
QUALIFICATION
PACK-
AGE.—The Secretary shall— 
‘‘(A) conduct a comprehensive review of a 
full qualification package accepted under para-
graph (1)(C); and 
‘‘(B) determine whether the drug develop-
ment tool at issue is qualified for its proposed 
context of use. 
‘‘(4) QUALIFICATION.—The Secretary shall deter-
mine whether a drug development tool is qualified for 
a proposed context of use based on the scientific merit 
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of a full qualification package reviewed under para-
graph (3). 
‘‘(b) EFFECT OF QUALIFICATION.— 
‘‘(1) IN GENERAL.—A drug development tool de-
termined to be qualified under subsection (a)(4) for a 
proposed context of use specified by the requestor may 
be used by any person in such context of use for the 
purposes described in paragraph (2). 
‘‘(2) USE OF A DRUG DEVELOPMENT TOOL.— 
Subject to paragraph (3), a drug development tool 
qualified under this section may be used for— 
‘‘(A) supporting or obtaining approval or 
licensure (as applicable) of a drug or biological 
product (including in accordance with section 
506(c)) under section 505 of this Act or section 
351 of the Public Health Service Act; or 
‘‘(B) supporting the investigational use of a 
drug or biological product under section 505(i) 
of this Act or section 351(a)(3) of the Public 
Health Service Act. 
‘‘(3) RESCISSION OR MODIFICATION.— 
‘‘(A) IN GENERAL.—The Secretary may re-
scind or modify a determination under this sec-
tion to qualify a drug development tool if the 
Secretary determines that the drug development 
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tool is not appropriate for the proposed context 
of use specified by the requestor. Such a deter-
mination may be based on new information that 
calls into question the basis for such qualifica-
tion. 
‘‘(B) MEETING FOR REVIEW.—If the Sec-
retary rescinds or modifies under subparagraph 
(A) a determination to qualify a drug develop-
ment tool, the requestor involved shall be granted 
a request for a meeting with the Secretary to dis-
cuss the basis of the Secretary’s decision to re-
scind or modify the determination before the ef-
fective date of the rescission or modification. 
‘‘(c) TRANSPARENCY.— 
‘‘(1) IN GENERAL.—Subject to paragraph (3), the 
Secretary shall make publicly available, and update 
on at least a biannual basis, on the Internet website 
of the Food and Drug Administration the following: 
‘‘(A) Information with respect to each qual-
ification submission under the qualification 
process under subsection (a), including— 
‘‘(i) the stage of the review process ap-
plicable to the submission; 
‘‘(ii) the date of the most recent change 
in stage status; 
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‘‘(iii) whether the external scientific ex-
perts were utilized in the development of a 
qualification plan or the review of a full 
qualification package; and 
‘‘(iv) 
submissions 
from 
requestors 
under the qualification process under sub-
section (a), including any data and evi-
dence contained in such submissions, and 
any updates to such submissions. 
‘‘(B) The Secretary’s formal written deter-
minations in response to such qualification sub-
missions. 
‘‘(C) Any rescissions or modifications under 
subsection (b)(3) of a determination to qualify a 
drug development tool. 
‘‘(D) Summary reviews that document con-
clusions and recommendations for determina-
tions to qualify drug development tools under 
subsection (a). 
‘‘(E) A comprehensive list of— 
‘‘(i) all drug development tools quali-
fied under subsection (a); and 
‘‘(ii) all surrogate endpoints which 
were the basis of approval or licensure (as 
applicable) of a drug or biological product 
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(including in accordance with section 
506(c)) under section 505 of this Act or sec-
tion 351 of the Public Health Service Act. 
‘‘(2) RELATION TO TRADE SECRETS ACT.—Infor-
mation made publicly available by the Secretary 
under paragraph (1) shall be considered a disclosure 
authorized by law for purposes of section 1905 of title 
18, United States Code. 
‘‘(3) APPLICABILITY.—Nothing in this section 
shall be construed as authorizing the Secretary to dis-
close any information contained in an application 
submitted under section 505 of this Act or section 351 
of the Public Health Service Act that is confidential 
commercial or trade secret information subject to sec-
tion 552(b)(4) of title 5, United States Code, or sec-
tion 1905 of title 18, United States Code. 
‘‘(d) RULE OF CONSTRUCTION.—Nothing in this sec-
tion shall be construed— 
‘‘(1) to alter the standards of evidence under sub-
section (c) or (d) of section 505, including the sub-
stantial evidence standard in such subsection (d), or 
under section 351 of the Public Health Service Act (as 
applicable); or 
‘‘(2) to limit the authority of the Secretary to 
approve or license products under this Act or the Pub-
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lic Health Service Act, as applicable (as in effect be-
fore the date of the enactment of the 21st Century 
Cures Act). 
‘‘(e) AUTHORIZATION OF APPROPRIATIONS.—There are 
authorized to be appropriated to carry out this section, 
$10,000,000 for each of fiscal years 2016 through 2020. 
‘‘(f) DEFINITIONS.—In this section: 
‘‘(1) BIOMARKER.—(A) The term ‘biomarker’ 
means a characteristic (such as a physiologic, 
pathologic, or anatomic characteristic or measure-
ment) that is objectively measured and evaluated as 
an indicator of normal biologic processes, pathologic 
processes, or biological responses to a therapeutic 
intervention; and 
‘‘(B) such term includes a surrogate endpoint. 
‘‘(2) BIOMEDICAL RESEARCH CONSORTIA.—The 
term ‘biomedical research consortia’ means collabo-
rative groups that may take the form of public-pri-
vate partnerships and may include government agen-
cies, institutions of higher education (as defined in 
section 101(a) of the Higher Education Act of 1965, 
patient advocacy groups, industry representatives, 
clinical and scientific experts, and other relevant enti-
ties and individuals. 
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‘‘(3) CLINICAL OUTCOME ASSESSMENT.—(A) The 
term ‘clinical outcome assessment’ means a measure-
ment of a patient’s symptoms, overall mental state, or 
the effects of a disease or condition on how the patient 
functions; and 
‘‘(B) such term includes a patient-reported out-
come. 
‘‘(4) CONTEXT OF USE.—The term ‘context of 
use’ means, with respect to a drug development tool, 
a statement that describes the circumstances under 
which the drug development tool is to be used in drug 
development and regulatory review. 
‘‘(5) DRUG
DEVELOPMENT
TOOL.—The term 
‘drug development tool’ includes— 
‘‘(A) a biomarker; 
‘‘(B) a clinical outcome assessment; and 
‘‘(C) any other method, material, or meas-
ure that the Secretary determines aids drug de-
velopment and regulatory review for purposes of 
this section. 
‘‘(6) PATIENT-REPORTED OUTCOME.—The term 
‘patient-reported outcome’ means a measurement 
based on a report from a patient regarding the status 
of the patient’s health condition without amendment 
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or interpretation of the patient’s report by a clinician 
or any other person. 
‘‘(7) QUALIFICATION.—The terms ‘qualification’ 
and ‘qualified’ mean a determination by the Sec-
retary that a drug development tool and its proposed 
context of use can be relied upon to have a specific 
interpretation and application in drug development 
and regulatory review under this Act. 
‘‘(8) REQUESTOR.—The term ‘requestor’ means 
an entity or entities, including a drug sponsor or a 
biomedical research consortia, seeking to qualify a 
drug development tool for a proposed context of use 
under this section. 
‘‘(9) SURROGATE ENDPOINT.—The term ‘surro-
gate endpoint’ means a marker, such as a laboratory 
measurement, radiographic image, physical sign, or 
other measure, that is not itself a direct measurement 
of clinical benefit, and— 
‘‘(A) is known to predict clinical benefit 
and could be used to support traditional ap-
proval of a drug or biological product; or 
‘‘(B) is reasonably likely to predict clinical 
benefit and could be used to support the acceler-
ated approval of a drug or biological product in 
accordance with section 506(c).’’. 
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(d) GUIDANCE.— 
(1) IN GENERAL.—The Secretary of Health and 
Human Services shall, in consultation with bio-
medical research consortia (as defined in subsection 
(f) of section 507 the Federal Food, Drug, and Cos-
metic Act (as added by subsection (c))) and other in-
terested parties through a collaborative public process, 
issue guidance to implement such section 507 that— 
(A) provides a conceptual framework de-
scribing appropriate standards and scientific 
approaches to support the development of bio-
markers delineated under the taxonomy estab-
lished under paragraph (3); 
(B) 
makes 
recommendations 
for 
dem-
onstrating that a surrogate endpoint is reason-
ably likely to predict clinical benefit for the pur-
pose of supporting the accelerated approval of a 
drug under section 506(c) of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 356(c)); 
(C) with respect to the qualification process 
under such section 507— 
(i) describes the requirements that enti-
ties seeking to qualify a drug development 
tool under such section shall observe when 
engaging in such process; 
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(ii) outlines reasonable timeframes for 
the Secretary’s review of letters, qualifica-
tion plans, or full qualification packages 
submitted under such process; and 
(iii) establishes a process by which 
such entities or the Secretary may consult 
with biomedical research consortia and 
other individuals and entities with expert 
knowledge and insights that may assist the 
Secretary in the review of qualification 
plans and full qualification submissions 
under such section; and 
(D) includes such other information as the 
Secretary determines appropriate. 
(2) TIMING.—Not later than 24 months after the 
date of the enactment of this Act, the Secretary of 
Health and Human Services shall issue draft guid-
ance under paragraph (1) on the implementation of 
section 507 of the Federal Food, Drug, and Cosmetic 
Act (as added by subsection (c)). The Secretary shall 
issue final guidance on the implementation of such 
section not later than 6 months after the date on 
which the comment period for the draft guidance 
closes. 
(3) TAXONOMY.— 
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(A) IN GENERAL.—For purposes of inform-
ing guidance under this subsection, the Secretary 
of Health and Human Services shall, in con-
sultation with biomedical research consortia and 
other interested parties through a collaborative 
public process, establish a taxonomy for the clas-
sification of biomarkers (and related scientific 
concepts) for use in drug development. 
(B) PUBLIC AVAILABILITY.—Not later than 
12 months after the date of the enactment of this 
Act, the Secretary of Health and Human Serv-
ices shall make such taxonomy publicly available 
in draft form for public comment. The Secretary 
shall finalize the taxonomy not later than 12 
months after the close of the public comment pe-
riod. 
(e) MEETING AND REPORT.— 
(1) MEETING.—Not later than 12 months after 
the date of the enactment of this Act, the Secretary of 
Health and Human Services shall convene a public 
meeting to describe and solicit public input regarding 
the qualification process under section 507 of the Fed-
eral Food, Drug, and Cosmetic Act, as added by sub-
section (c). 
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(2) REPORT.—Not later than 5 years after the 
date of the enactment of this Act, the Secretary shall 
make publicly available on the Internet website of the 
Food and Drug Administration a report. Such report 
shall include, with respect to the qualification process 
under section 507 of the Federal Food, Drug, and 
Cosmetic Act, as added by subsection (c), information 
on— 
(A) the number of requests submitted, as a 
letter of intent, for qualification of a drug devel-
opment tool (as defined in subsection (f) of such 
section); 
(B) the number of such requests accepted 
and determined to be eligible for submission of a 
qualification plan or full qualification package 
(as such terms are defined in such subsection), 
respectively; 
(C) the number of such requests for which 
external scientific experts were utilized in the de-
velopment of a qualification plan or review of a 
full qualification package; and 
(D) the number of qualification plans and 
full qualification packages, respectively, sub-
mitted to the Secretary; and 
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(3) the drug development tools qualified through 
such qualification process, specified by type of tool, 
such as a biomarker or clinical outcome assessment 
(as such terms are defined in subsection (f) of such 
section 507). 
SEC. 2022. ACCELERATED APPROVAL DEVELOPMENT PLAN. 
(a) IN GENERAL.—Section 506 of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 356) is amended by 
adding the following subsection: 
‘‘(g) 
ACCELERATED
APPROVAL
DEVELOPMENT 
PLAN.— 
‘‘(1) IN GENERAL.—In the case of a drug that 
the Secretary determines may be eligible for acceler-
ated approval in accordance with subsection (c), the 
sponsor of such drug may request, at any time after 
the submission of an application for the investigation 
of the drug under section 505(i) of this Act or section 
351(a)(3) of the Public Health Service Act, that the 
Secretary agree to an accelerated approval develop-
ment plan described in paragraph (2). 
‘‘(2) PLAN
DESCRIBED.—A plan described in 
this paragraph, with respect to a drug described in 
paragraph (1), is an accelerated approval develop-
ment plan, which shall include agreement on— 
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‘‘(A) the surrogate endpoint to be assessed 
under such plan; 
‘‘(B) the design of the study that will utilize 
the surrogate endpoint; and 
‘‘(C) the magnitude of the effect of the drug 
on the surrogate endpoint that is the subject of 
the agreement that would be sufficient to form 
the primary basis of a claim that the drug is ef-
fective. 
‘‘(3) MODIFICATION; TERMINATION.—The Sec-
retary may require the sponsor of a drug that is the 
subject of an accelerated approval development plan 
to modify or terminate the plan if additional data or 
information indicates that— 
‘‘(A) the plan as originally agreed upon is 
no longer sufficient to demonstrate the safety and 
effectiveness of the drug involved; or 
‘‘(B) the drug is no longer eligible for accel-
erated approval under subsection (c). 
‘‘(4) SPONSOR CONSULTATION.—If the Secretary 
requires the modification or termination of an accel-
erated approval development plan under paragraph 
(3), the sponsor shall be granted a request for a meet-
ing to discuss the basis of the Secretary’s decision be-
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fore the effective date of the modification or termi-
nation. 
‘‘(5) DEFINITION.—In this section, the term ‘ac-
celerated approval development plan’ means a devel-
opment plan agreed upon by the Secretary and the 
sponsor submitting the plan that contains study pa-
rameters for the use of a surrogate endpoint that— 
‘‘(A) is reasonably likely to predict clinical 
benefit; and 
‘‘(B) is intended to be the basis of the accel-
erated approval of a drug in accordance with 
subsection (c).’’. 
(b) TECHNICAL AMENDMENTS.—Section 506 of the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 356) is 
amended— 
(1) by striking ‘‘(f) AWARENESS EFFORTS’’ and 
inserting ‘‘(e) AWARENESS EFFORTS’’; and 
(2) by striking ‘‘(e) CONSTRUCTION’’ and insert-
ing ‘‘(f) CONSTRUCTION’’. 
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Subtitle C—FDA Advancement of 
Precision Medicine 
SEC. 2041. PRECISION MEDICINE GUIDANCE AND OTHER 
PROGRAMS OF FOOD AND DRUG ADMINISTRA-
TION. 
Chapter V of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 351 et seq.) is amended by adding at the 
end the following: 
‘‘Subchapter J—Precision Medicine 
‘‘SEC. 591. GENERAL AGENCY GUIDANCE ON PRECISION 
MEDICINE. 
‘‘(a) IN GENERAL.—The Secretary shall issue and pe-
riodically update guidance to assist sponsors in the develop-
ment of a precision drug or biological product. Such guid-
ance shall— 
‘‘(1) define the term ‘precision drug or biological 
product’; and 
‘‘(2) address the topics described in subsection 
(b). 
‘‘(b) CERTAIN ISSUES.—The topics to be addressed by 
guidance under subsection (a) are— 
‘‘(1) the evidence needed to support the use of 
biomarkers (as defined in section 507(e)) that identify 
subsets of patients as likely responders to therapies in 
order to streamline the conduct of clinical trials; 
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‘‘(2) recommendations for the design of studies to 
demonstrate the validity of a biomarker as a pre-
dictor of drug or biological product response; 
‘‘(3) the manner and extent to which a benefit- 
risk assessment may be affected when clinical trials 
are limited to patient population subsets that are 
identified using biomarkers; 
‘‘(4) the development of companion diagnostics 
in the context of a drug development program; and 
‘‘(5) considerations for developing biomarkers 
that inform prescribing decisions for a drug or bio-
logical product, and when information regarding a 
biomarker may be included in the approved prescrip-
tion labeling for a precision drug or biological prod-
uct. 
‘‘(c) DATE CERTAIN FOR INITIAL GUIDANCE.—The 
Secretary shall issue guidance under subsection (a) not 
later than 18 months after the date of the enactment of the 
21st Century Cures Act. 
‘‘SEC. 592. PRECISION MEDICINE REGARDING ORPHAN- 
DRUG 
AND 
EXPEDITED-APPROVAL 
PRO-
GRAMS. 
‘‘(a) IN GENERAL.—In the case of a precision drug 
or biological product that is the subject of an application 
submitted under section 505(b)(1), or section 351(a) of the 
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Public Health Service Act, for the treatment of a serious 
or life-threatening disease or condition and has been des-
ignated under section 526 as a drug for a rare disease or 
condition, the Secretary may— 
‘‘(1) consistent with applicable standards for ap-
proval, rely upon data or information previously sub-
mitted by the sponsor of the precision drug or biologi-
cal product, or another sponsor, provided that the 
sponsor of the precision drug or biological product 
has obtained a contractual right of reference to such 
other sponsor’s data and information, in an applica-
tion approved under section 505(c) or licensed under 
section 351(a) of the Public Health Service Act, as 
applicable— 
‘‘(A) for a different drug or biological prod-
uct; or 
‘‘(B) for a different indication for such pre-
cision drug or biological product, 
in order to expedite clinical development for a preci-
sion drug or biological product that is using the same 
or similar approach as that used to support approval 
of the prior approved application or license, as ap-
propriate; and 
‘‘(2) as appropriate, consider the application for 
approval of such precision drug or biological product 
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to be eligible for expedited review and approval pro-
grams described in section 506, including accelerated 
approval in accordance with subsection (c) of such 
section. 
‘‘(b) RULE OF CONSTRUCTION.—Nothing in this sec-
tion shall be construed to— 
‘‘(1) limit the authority of the Secretary to ap-
prove products pursuant to this Act and the Public 
Health Service Act as authorized prior to the date of 
enactment of this section; or 
‘‘(2) confer any new rights, beyond those author-
ized under this Act prior to enactment of this section, 
with respect to a sponsor’s ability to reference infor-
mation contained in another application submitted 
under section 505(b)(1) of this Act or section 351(a) 
of the Public Health Service Act.’’. 
Subtitle D—Modern Trial Design 
and Evidence Development 
SEC. 2061. BROADER APPLICATION OF BAYESIAN STATIS-
TICS AND ADAPTIVE TRIAL DESIGNS. 
(a) PROPOSALS FOR USE OF INNOVATIVE STATISTICAL 
METHODS IN CLINICAL PROTOCOLS FOR DRUGS AND BIO-
LOGICAL PRODUCTS.—For purposes of assisting sponsors in 
incorporating adaptive trial design and Bayesian methods 
into proposed clinical protocols and applications for new 
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100 
drugs under section 505 of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 355) and biological products under 
section 351 of the Public Health Service Act (42 U.S.C. 
262), the Secretary shall conduct a public meeting and issue 
guidance in accordance with subsection (b). 
(b) GUIDANCE ADDRESSING USE OF ADAPTIVE TRIAL 
DESIGNS AND BAYESIAN METHODS.— 
(1) IN GENERAL.—The Secretary of Health and 
Human Services, acting through the Commissioner of 
Food and Drugs (in this subsection referred to as the 
‘‘Secretary’’), shall— 
(A) update and finalize the draft guidance 
addressing the use of adaptive trial design for 
drugs and biological products; and 
(B) issue draft guidance on the use of 
Bayesian methods in the development and regu-
latory review and approval or licensure of drugs 
and biological products. 
(2) CONTENTS.—The guidances under paragraph 
(1) shall address— 
(A) the use of adaptive trial designs and 
Bayesian methods in clinical trials, including 
clinical trials proposed or submitted to help to 
satisfy the substantial evidence standard under 
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101 
section 505(d) of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 355(d)); 
(B) how sponsors may obtain feedback from 
the Secretary on technical issues related to mod-
eling and simulations prior to— 
(i) completion of such modeling or sim-
ulations; or 
(ii) the submission of resulting infor-
mation to the Secretary; 
(C) the types of quantitative and qualitative 
information that should be submitted for review; 
and 
(D) recommended analysis methodologies. 
(3) PUBLIC MEETING.—Prior to updating or de-
veloping the guidances required by paragraph (1), the 
Secretary shall consult with stakeholders, including 
representatives of regulated industry, academia, pa-
tient advocacy organizations, and disease research 
foundations, through a public meeting to be held not 
later than 1 year after the date of enactment of this 
Act. 
(4) SCHEDULE.—The Secretary shall publish— 
(A) the final guidance required by para-
graph (1)(A) not later than 18 months after the 
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102 
date of the public meeting required by paragraph 
(3); and 
(B) the guidance required by paragraph 
(1)(B) not later than 48 months after the date of 
the public meeting required by paragraph (3). 
SEC. 2062. UTILIZING EVIDENCE FROM CLINICAL EXPERI-
ENCE. 
Chapter V of the Federal Food, Drug, and Cosmetic 
Act is amended by inserting after section 505E of such Act 
(21 U.S.C. 355f) the following: 
‘‘SEC. 505F. UTILIZING EVIDENCE FROM CLINICAL EXPERI-
ENCE. 
‘‘(a) IN GENERAL.—The Secretary shall establish a 
program to evaluate the potential use of evidence from clin-
ical experience— 
‘‘(1) to help to support the approval of a new in-
dication for a drug approved under section 505(b); 
and 
‘‘(2) to help to support or satisfy postapproval 
study requirements. 
‘‘(b) EVIDENCE FROM CLINICAL EXPERIENCE DE-
FINED.—In this section, the term ‘evidence from clinical ex-
perience’ means data regarding the usage, or the potential 
benefits or risks, of a drug derived from sources other than 
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103 
randomized clinical trials, including from observational 
studies, registries, and therapeutic use. 
‘‘(c) PROGRAM FRAMEWORK.— 
‘‘(1) IN GENERAL.—Not later than 18 months 
after the date of enactment of this section, the Sec-
retary shall establish a draft framework for imple-
mentation of the program under this section. 
‘‘(2) CONTENTS
OF
FRAMEWORK.—The frame-
work shall include information describing— 
‘‘(A) the current sources of data developed 
through clinical experience, including ongoing 
safety surveillance, registry, claims, and patient- 
centered outcomes research activities; 
‘‘(B) the gaps in current data collection ac-
tivities; 
‘‘(C) the current standards and methodolo-
gies for collection and analysis of data generated 
through clinical experience; and 
‘‘(D) the priority areas, remaining chal-
lenges, and potential pilot opportunities that the 
program established under this section will ad-
dress. 
‘‘(3) CONSULTATION.— 
‘‘(A) IN GENERAL.—In developing the pro-
gram framework under this subsection, the Sec-
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104 
retary shall consult with regulated industry, aca-
demia, medical professional organizations, rep-
resentatives of patient advocacy organizations, 
disease research foundations, and other interested 
parties. 
‘‘(B) PROCESS.—The consultation under 
subparagraph (A) may be carried out through 
approaches such as— 
‘‘(i) a public-private partnership with 
the entities described in such subparagraph 
in which the Secretary may participate; or 
‘‘(ii) a contract, grant, or other ar-
rangement, as determined appropriate by 
the Secretary with such a partnership or an 
independent research organization. 
‘‘(d) PROGRAM
IMPLEMENTATION.—The Secretary 
shall, not later than 24 months after the date of enactment 
of this section and in accordance with the framework estab-
lished under subsection (c), implement the program to 
evaluate the potential use of evidence from clinical experi-
ence. 
‘‘(e) GUIDANCE
FOR
INDUSTRY.—The Secretary 
shall— 
‘‘(1) utilize the program established under sub-
section (a), its activities, and any subsequent pilots or 
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written reports, to inform a guidance for industry 
on— 
‘‘(A) the circumstances under which spon-
sors of drugs and the Secretary may rely on evi-
dence from clinical experience for the purposes 
described in subsection (a)(1) or (a)(2); and 
‘‘(B) the appropriate standards and meth-
odologies for collection and analysis of evidence 
from clinical experience submitted for such pur-
poses; 
‘‘(2) not later than 36 months after the date of 
enactment of this section, issue draft guidance for in-
dustry as described in paragraph (1); and 
‘‘(3) not later than 48 months after the date of 
enactment of this section, after providing an oppor-
tunity for public comment on the draft guidance, 
issue final guidance. 
‘‘(f) RULE OF CONSTRUCTION.— 
‘‘(1) Subject to paragraph (2), nothing in this 
section prohibits the Secretary from using evidence 
from clinical experience for purposes not specified in 
this section, provided the Secretary determines that 
sufficient basis exists for any such nonspecified use. 
‘‘(2) This section shall not be construed to 
alter— 
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‘‘(A) the standards of evidence under— 
‘‘(i) subsection (c) or (d) of section 505, 
including the substantial evidence standard 
in such subsection (d); or 
‘‘(ii) section 351(a) of the Public 
Health Service Act; or 
‘‘(B) the Secretary’s authority to require 
postapproval studies or clinical trials, or the 
standards of evidence under which studies or 
trials are evaluated. 
‘‘SEC. 505G. COLLECTING EVIDENCE FROM CLINICAL EXPE-
RIENCE THROUGH TARGETED EXTENSIONS 
OF THE SENTINEL SYSTEM. 
‘‘(a) IN GENERAL.—The Secretary shall, in parallel to 
implementing the program established under section 505F 
and in order to build capacity for utilizing the evidence 
from clinical experience described in that section, identify 
and execute pilot demonstrations to extend existing use of 
the Sentinel System surveillance infrastructure authorized 
under section 505(k). 
‘‘(b) PILOT DEMONSTRATIONS.— 
‘‘(1) IN GENERAL.—The Secretary— 
‘‘(A) shall design and implement pilot dem-
onstrations to utilize data captured through the 
Sentinel System surveillance infrastructure au-
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thorized under section 505(k) for purposes of, as 
appropriate— 
‘‘(i) generating evidence from clinical 
experience to improve characterization or 
assessment of risks or benefits of a drug ap-
proved under section 505(c); 
‘‘(ii) protecting the public health; or 
‘‘(iii) advancing patient-centered care; 
and 
‘‘(B) may make strategic linkages with 
sources of complementary public health data and 
infrastructure the Secretary determines appro-
priate and necessary. 
‘‘(2) CONSULTATION.—In developing the pilot 
demonstrations under this subsection, the Secretary 
shall— 
‘‘(A) consult with regulated industry, aca-
demia, medical professional organizations, rep-
resentatives of patient advocacy organizations, 
disease research foundations, and other interested 
parties through a public process; and 
‘‘(B) develop a framework to promote ap-
propriate transparency and dialogue about re-
search conducted under these pilot demonstra-
tions, including by— 
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‘‘(i) providing adequate notice to a 
sponsor of a drug approved under section 
505 or section 351 of the Public Health 
Service Act of the Secretary’s intent to con-
duct analyses of such sponsor’s drug or 
drugs under these pilot demonstrations; 
‘‘(ii) providing adequate notice of the 
findings related to analyses described in 
clause (i) and an opportunity for the spon-
sor of such drug or drugs to comment on 
such findings; and 
‘‘(iii) ensuring the protection from 
public disclosure of any information that is 
a trade secret or confidential information 
subject to section 552(b)(4) of title 5, United 
States Code, or section 1905 of title 18, 
United States Code. 
‘‘(3) PUBLIC
HEALTH
EXEMPTION.—The Sec-
retary may— 
‘‘(A) deem such pilot demonstrations public 
health activities, permitting the use and disclo-
sure of protected health information as described 
in section 164.512(b)(1)(iii) of title 45, Code of 
Federal Regulations (or any successor regula-
tion) and exempted as a public health activity as 
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109 
described in section 46.101(b)(5) of title 46, Code 
of Federal Regulations (or any successor regula-
tion); and 
‘‘(B) deem safety surveillance performed at 
the request of the Food and Drug Administration 
or under such jurisdiction by a sponsor with re-
sponsibility for a drug approved under this sec-
tion or section 351 of the Public Health Services 
Act using the Sentinel System surveillance infra-
structure authorized under section 505(k), in-
cluding use of analytic tools and querying capa-
bilities developed to implement the active 
postmarket surveillance system described in this 
section, public health activities as described in 
section 164.512(b)(1)(iii) of title 45, Code of 
Federal Regulations (or any successor regula-
tion) and exempted as a public health activity as 
described in section 46.101(b)(5) of title 46, Code 
of Federal Regulations (or any successor regula-
tion). 
‘‘(c) AUTHORIZATION OF APPROPRIATIONS.—There are 
authorized to be appropriated to carry out this section 
$3,000,000 for each of fiscal years 2016 through 2020.’’. 
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110 
SEC. 2063. STREAMLINED DATA REVIEW PROGRAM. 
(a) IN GENERAL.—Chapter V of the Federal Food, 
Drug, and Cosmetic Act, as amended by section 2062, is 
further amended by inserting after section 505G of such Act 
the following: 
‘‘SEC. 505H. STREAMLINED DATA REVIEW PROGRAM. 
‘‘(a) IN GENERAL.—The Secretary shall establish a 
streamlined data review program under which a holder of 
an approved application submitted under section 505(b)(1) 
or under section 351(a) of the Public Health Service Act 
may, to support the approval or licensure (as applicable) 
of the use of the drug that is the subject of such approved 
application for a new qualified indication, submit qualified 
data summaries. 
‘‘(b) ELIGIBILITY.—In carrying out the streamlined 
data review program under subsection (a), the Secretary 
may authorize the holder of the approved application to in-
clude one or more qualified data summaries described in 
subsection (a) in a supplemental application if— 
‘‘(1) the drug has been approved under section 
505(c) of this Act or licensed under section 351(a) of 
the Public Health Service Act for one or more indica-
tions, and such approval or licensure remains in ef-
fect; 
‘‘(2) the supplemental application is for ap-
proval or licensure (as applicable) under such section 
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505(c) or 351(a) of the use of the drug for a new 
qualified indication under such section 505(c) or 
351(a); 
‘‘(3) there is an existing database acceptable to 
the Secretary regarding the safety of the drug devel-
oped for one or more indications of the drug approved 
under such section 505(c) or licensed under such sec-
tion 351(a); 
‘‘(4) the supplemental application incorporates 
or supplements the data submitted in the application 
for approval or licensure referred to in paragraph (1); 
and 
‘‘(5) the full data sets used to develop the quali-
fied data summaries are submitted, unless the Sec-
retary determines that the full data sets are not re-
quired. 
‘‘(c) PUBLIC AVAILABILITY OF INFORMATION ON PRO-
GRAM.—The Secretary shall post on the public website of 
the Food and Drug Administration and update annually— 
‘‘(1) the number of applications reviewed under 
the streamlined data review program; 
‘‘(2) the average time for completion of review 
under the streamlined data review program versus 
other review of applications for new indications; and 
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112 
‘‘(3) the number of applications reviewed under 
the streamlined data review program for which the 
Food and Drug Administration made use of full data 
sets in addition to the qualified data summary. 
‘‘(d) DEFINITIONS.—In this section: 
‘‘(1) The term ‘qualified indication’ means— 
‘‘(A) an indication for the treatment of can-
cer, as determined appropriate by the Secretary; 
or 
‘‘(B) such other types of indications as the 
Secretary determines to be subject to the stream-
lined data review program under this section. 
‘‘(2) The term ‘qualified data summary’ means 
a summary of clinical data intended to demonstrate 
safety and effectiveness with respect to a qualified in-
dication for use of a drug.’’. 
(b) SENSE OF CONGRESS.—It is the sense of Congress 
that the streamlined data review program under section 
505H of the Federal Food, Drug, and Cosmetic Act, as 
added by subsection (a), should enable the Food and Drug 
Administration to make approval decisions for certain sup-
plemental applications based on qualified data summaries 
(as defined in such section 505H). 
(c) GUIDANCE; REGULATIONS.—The Commissioner of 
Food and Drugs— 
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113 
(1) shall— 
(A) issue final guidance for implementation 
of the streamlined data review program estab-
lished under section 505H of the Federal Food, 
Drug, and Cosmetic Act, as added by subsection 
(a), not later than 24 months after the date of 
enactment of this Act; and 
(B) include in such guidance the process for 
expanding the types of indications to be subject 
to the streamlined data review program, as au-
thorized by section 505H(c)(1)(B) of such Act; 
and 
(2) in addition to issuing guidance under para-
graph (1), may issue such regulations as may be nec-
essary for implementation of the program. 
Subtitle E—Expediting Patient 
Access 
SEC. 2081. SENSE OF CONGRESS. 
It is the sense of Congress that the Food and Drug Ad-
ministration should continue to expedite the approval of 
drugs designated as breakthrough therapies pursuant to sec-
tion 506(a) of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C. 356(a)) by approving drugs so designated as 
early as possible in the clinical development process, regard-
less of the phase of development, provided that the Secretary 
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114 
of Health and Human Services determines that an applica-
tion for such a drug meets the standards of evidence of safe-
ty and effectiveness under section 505 of such Act (21 U.S.C. 
355), including the substantial evidence standard under 
subsection (d) of such section or under section 351(a) of the 
Public Health Service Act (42 U.S.C. 262(a)). 
SEC. 2082. EXPANDED ACCESS POLICY. 
Chapter V of the Federal Food, Drug, and Cosmetic 
Act is amended by inserting after section 561 (21 U.S.C. 
360bbb) the following: 
‘‘SEC. 561A. EXPANDED ACCESS POLICY REQUIRED FOR IN-
VESTIGATIONAL DRUGS. 
‘‘(a) IN GENERAL.—The manufacturer or distributor 
of one or more investigational drugs for the diagnosis, mon-
itoring, or treatment of one or more serious diseases or con-
ditions shall make publicly available the policy of the man-
ufacturer or distributor on evaluating and responding to 
requests submitted under section 561(b) for provision of 
such a drug. A manufacturer or distributor may satisfy the 
requirement of the preceding sentence by posting such policy 
as generally applicable to all of such manufacturer’s or dis-
tributor’s investigational drugs. 
‘‘(b) CONTENT OF POLICY.—A policy described in sub-
section (a) shall include making publicly available— 
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115 
‘‘(1) contact information for the manufacturer or 
distributor to facilitate communication about requests 
described in subsection (a); 
‘‘(2) procedures for making such requests; 
‘‘(3) the general criteria the manufacturer or dis-
tributor will consider or use to approve such requests; 
and 
‘‘(4) the length of time the manufacturer or dis-
tributor anticipates will be necessary to acknowledge 
receipt of such requests. 
‘‘(c) NO GUARANTEE OF ACCESS.—The posting of poli-
cies by manufacturers and distributors under subsection (a) 
shall not serve as a guarantee of access to any specific inves-
tigational drug by any individual patient. 
‘‘(d) REVISED
POLICY.—A manufacturer or dis-
tributor that has made a policy publicly available as re-
quired by this section may revise the policy at any time. 
‘‘(e) APPLICATION.—This section shall apply to a 
manufacturer or distributor with respect to an investiga-
tional drug beginning on the later of— 
‘‘(1) the date that is 60 days after the date of en-
actment of the 21st Century Cures Act; or 
‘‘(2) the first initiation of a phase 2 or phase 3 
study (as such terms are defined in section 312.21(b) 
and (c) of title 21, Code of Federal Regulations (or 
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116 
any successor regulations)) with respect to such inves-
tigational new drug.’’. 
SEC. 2083. FINALIZING DRAFT GUIDANCE ON EXPANDED 
ACCESS. 
(a) IN GENERAL.—Not later than 12 months after the 
date of enactment of this Act, 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 sub-
section (a) shall clearly define how the Secretary of Health 
and Human Services 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)). 
Subtitle 
F—Facilitating 
Respon-
sible Manufacturer Communica-
tions 
SEC. 2101. FACILITATING DISSEMINATION OF HEALTH CARE 
ECONOMIC INFORMATION. 
Section 502(a) of the Federal Food, Drug, and Cos-
metic Act (21 U.S.C. 352(a)) is amended— 
(1) by striking ‘‘(a) If its’’ and inserting ‘‘(a)(1) 
If its’’; 
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(2) by striking ‘‘a formulary committee, or other 
similar entity, in the course of the committee or the 
entity carrying out its responsibilities for the selec-
tion of drugs for managed care or other similar orga-
nizations’’ and inserting ‘‘a payor, formulary com-
mittee, or other similar entity with knowledge and ex-
pertise in the area of health care economic analysis, 
carrying out its responsibilities for the selection of 
drugs for coverage or reimbursement’’; 
(3) by striking ‘‘directly relates’’ and inserting 
‘‘relates’’; 
(4) by striking ‘‘and is based on competent and 
reliable scientific evidence. The requirements set forth 
in section 505(a) or in section 351(a) of the Public 
Health Service Act shall not apply to health care eco-
nomic information provided to such a committee or 
entity in accordance with this paragraph’’ and in-
serting ‘‘, is based on competent and reliable scientific 
evidence, and includes, where applicable, a con-
spicuous and prominent statement describing any 
material differences between the health care economic 
information and the labeling approved for the drug 
under section 505 or under section 351 of the Public 
Health Service Act. The requirements set forth in sec-
tion 505(a) or in subsections (a) and (k) of section 
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135 
other similar source acceptable to the 
Secretary; and 
‘‘(ii) recognized in whole, or in part, 
by the Secretary under subsection (c). 
‘‘(B) OTHER LIST.—The Interpretive Cri-
teria Website shall, in addition to the list de-
scribed in subparagraph (A), include a list of in-
terpretive criteria, if any, that the Secretary has 
determined to be appropriate with respect to le-
gally marketed antimicrobial drugs, where— 
‘‘(i) the Secretary does not recognize, 
in whole or in part, an interpretive criteria 
standard described under subparagraph (A) 
otherwise applicable to such a drug; 
‘‘(ii) the Secretary withdraws under 
subsection (c)(1)(B) recognition of a stand-
ard, in whole or in part, otherwise applica-
ble to such a drug; 
‘‘(iii) the Secretary approves an appli-
cation under section 505 of this Act or sec-
tion 351 of the Public Health Service Act, 
as applicable, with respect to marketing of 
such a drug for which there are no relevant 
interpretive criteria included in a standard 
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174 
controlled substance from the United States delivers to 
the Attorney General— 
‘‘(i) documentation certifying that such re- 
exportation has occurred; and 
‘‘(ii) information concerning the consignee, 
country, and product.’’; and 
(2) by adding at the end the following: 
‘‘(g) LIMITATION.—The Attorney General shall not 
promulgate nor enforce any regulation, subregulatory guid-
ance, or enforcement policy which impedes re-exportation 
among European Economic Area countries (as provided in 
subsection (f)(5)), including by promulgating or enforcing 
any requirement that— 
‘‘(1) re-exportation from the first country to the 
second country or re-exportation from the second 
country to another country (as such terms are used 
in subsection (f)) occur within a specified period of 
time; or 
‘‘(2) information concerning the consignee, coun-
try, and product be provided prior to exportation of 
the controlled substance from the United States or 
prior to each re-exportation among members of the 
European Economic Area.’’. 
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