SEC. 1002. NIH INNOVATION FUND. 
(a) USE OF INNOVATION FUND.—Section 402(b) of 
the Public Health Service Act is amended— 
(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 insti-
tutes and national centers for conducting and sup-
porting innovation fund initiatives identified under 
paragraph (3) of such section.’’. 
(b) ESTABLISHMENT OF INNOVATION FUND.—Sec-
tion 402A of the Public Health Service Act is amended— 
(1) by redesignating subsection (e) as sub-
section (f); and 
(2) by inserting after subsection (d) the fol-
lowing new subsection: 
‘‘(e) NIH INNOVATION FUND.— 
‘‘(1) ESTABLISHMENT.—For the purpose of al-
locations under section 402(b)(25), there is estab-
lished a fund to be known as the NIH Innovation 
Fund. The Director of NIH shall, with respect to 
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funds appropriated to the NIH Innovation Fund, al-
locate such funds to support biomedical research 
through the funding of basic, translational, and clin-
ical 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 pre-
ceding sentence shall be in addition to any 
amounts otherwise made available to the Na-
tional 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— 
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‘‘(i) not less than $500,000,000 shall 
be for the Accelerating Advancement Pro-
gram under paragraph (5); 
‘‘(ii) not less than 35 percent of such 
amounts remaining after subtracting the 
allocation for the Accelerating Advance-
ment Program shall be for early stage in-
vestigators as defined in subsection (7); 
‘‘(iii) not less than 20 percent of such 
amounts remaining after subtracting the 
allocation for the Accelerating Advance-
ment 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 al-
location 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 sec-
tion 241, subsection (c), subsection (d), or 
any other provision of law (other than sec-
tion 402(b)(25) and this subsection); or 
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‘‘(ii) the Nonrecurring expenses fund 
under section 223 of division G of the Con-
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 para-
graph (7)). 
‘‘(D) Research to be carried out by a small 
business concern (as defined in section 3 of the 
Small Business Act). 
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‘‘(E) The Accelerating Advancement Pro-
gram under paragraph (5). 
‘‘(F) Development and implementation of 
the strategic plan under paragraph (6). 
‘‘(4) COORDINATION.—In funding programs 
and activities through the NIH Innovation Fund, 
the Secretary, 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
PRO-
GRAM.—The Director of NIH shall establish a pro-
gram, to be known as the Accelerating Advancement 
Program, under which— 
‘‘(A) the Director of NIH partners with 
national research institutes and national centers 
to accomplish important biomedical research ob-
jectives; and 
‘‘(B) for every $1 made available by the 
Director of NIH to a national research institute 
or national center for a research project, the in-
stitute or center makes $1 available for such 
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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 
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 effi-
cient and effective focus of biomedical re-
search in a manner that leverages the best 
scientific opportunities through a delibera-
tive planning process; 
‘‘(ii) identifies areas, to be known as 
strategic focus areas, in which the re-
sources of the NIH Innovation Fund can 
contribute to the goals of expanding knowl-
edge to address, 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 
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pathogen under section 505E(f) of the 
Federal Food, Drug, and Cosmetic 
Act or listed or designated as a trop-
ical disease under section 524 of such 
Act; and 
‘‘(IV) antibiotics; 
‘‘(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— 
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‘‘(i) the investigator’s terminal degree; 
or 
‘‘(ii) a medical residency (or the 
equivalent).’’. 
(c) SUPPLEMENT, NOT SUPPLANT; PROHIBITION 
AGAINST TRANSFER.—Funds appropriated pursuant to 
section 402A(e) of the Public Health Service Act, as in-
serted by subsection (b)— 
(1) shall be used to supplement, not supplant, 
the funds otherwise allocated by the National Insti-
tutes of Health for biomedical research; and 
(2) notwithstanding any transfer authority in 
any appropriation Act, shall not be used for any 
purpose other than allocating funds for conducting 
and supporting innovation fund initiatives as de-
scribed 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: 
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‘‘(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 devel-
opment, use, and updating of the research strategic 
plan under subsection (m);’’; and 
(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 effi-
cient and effective focus of biomedical re-
search in a manner that leverages the best 
scientific opportunities through a delibera-
tive planning process; 
‘‘(ii) identifies areas, to be known 
strategic focus areas, in which the re-
sources of the National Institutes of 
Health can best contribute to the goal of 
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expanding knowledge on human health in 
the United States through biomedical re-
search; and 
‘‘(iii) includes objectives for each such 
strategic focus area. 
‘‘(B) ENTITIES DESCRIBED.—The entities 
described in this subparagraph are the directors 
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 na-
tional research institutes and national centers 
that— 
‘‘(i) have a common template; and 
‘‘(ii) identify strategic focus areas in 
which the resources of the national re-
search institutes and national centers can 
best contribute to the goal of expanding 
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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 Na-
tional Institutes of Health in bio-
medical research; 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 Pri-
ority Focus Areas, which best serve the 
goals of preventing or eliminating the bur-
den of a disease or condition and scientif-
ically merit enhanced and focused research 
over the next 5 years. 
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‘‘(B) RARE AND PEDIATRIC DISEASES AND 
CONDITIONS.—In developing and maintaining a 
strategic plan under this subsection, the Direc-
tor of NIH shall ensure that rare and pediatric 
diseases and conditions remain a priority. 
‘‘(4) INITIAL PLAN.—Not later than 270 days 
after the date of enactment of this subsection, the 
Director 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 
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with the directors of the national research insti-
tutes and national centers, stakeholders in the 
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 
NATIONAL 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 insti-
tutes, as well as national centers, shall report di-
rectly 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 na-
tional research institute or national center may 
be removed 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 direc-
tor of a national research institute or national 
center becomes vacant before the end of such 
director’s term, the director appointed to fill the 
vacancy shall be appointed for a 5-year term 
starting 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 
appointed for a 5-year term under this sub-
section starting on such date of enactment.’’. 
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(b) COMPENSATION
TO CONSULTANTS
OR INDI-
VIDUAL 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 fol-
lowing: 
‘‘(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 
research institute or national center— 
‘‘(A) shall review and approve the award; and 
‘‘(B) shall take into consideration— 
‘‘(i) the mission of the national research 
institute or national center and the scientific 
priorities identified in the strategic plan under 
section 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 re-
sults of such study, including recommendations on 
how to prevent such duplication. 
SEC. 1023. REDUCING ADMINISTRATIVE BURDENS OF RE-
SEARCHERS. 
(a) IMPLEMENTATION OF MEASURES TO REDUCE 
ADMINISTRATIVE BURDENS.—The Director of the Na-
tional Institutes of Health shall implement measures to 
reduce the administrative burdens of researchers funded 
by the National Institutes of Health, taking into account 
the recommendations, evaluations, and plans researched 
by the following entities: 
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(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 Re-
search Business Models Working Group. 
(b) REPORTS.—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 
pursuant 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: 
‘‘(E) during the conduct of research by the 
National Institutes of Health.’’. 
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SEC. 1025. NIH TRAVEL. 
It is the sense of Congress that participation in or 
sponsorship of scientific conferences and meetings is es-
sential 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 appro-
priation of funds as described in subsection (g)’’ and 
inserting ‘‘the availability of funds as described in 
subsection (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 
transactions authority in entering into trans-
actions 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 sub-
section (f). 
SEC. 1027. NCATS PHASE IIB RESTRICTION. 
Section 479 of the Public Health Service Act (42 
U.S.C. 287) is amended— 
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(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 
research projects that pursue innovative approaches 
to major contemporary challenges in biomedical re-
search 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 
national research institute, for such projects.’’. 
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Subtitle C—Supporting Young 
Emerging Scientists 
SEC. 1041. IMPROVEMENT OF LOAN REPAYMENT PRO-
GRAMS 
OF 
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; pediatric research loan repayment 
program) as section 487G; and 
(2) by inserting after section 487G, as so redes-
ignated, the following: 
‘‘SEC. 487H. LOAN REPAYMENT PROGRAM. 
‘‘(a) IN GENERAL.—The Secretary shall establish a 
program, based on workforce and scientific needs, of en-
tering 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 re-
search, not more than $50,000 of the principal and inter-
est of the educational 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 
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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 sub-
section (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 estab-
lished under this section to the same extent and in the 
same manner as such provisions apply to the National 
Health Service Corps Loan Repayment Program estab-
lished under section 338B. 
‘‘(e) AVAILABILITY OF APPROPRIATIONS.—Amounts 
appropriated for a fiscal year for contracts under sub-
section (a) are authorized to remain available until the ex-
piration of the second fiscal year beginning after the fiscal 
year for which the amounts were appropriated.’’. 
(b) UPDATE OF OTHER LOAN REPAYMENT PRO-
GRAMS.— 
(1) Section 464z–5(a) of the Public Health 
Service Act (42 U.S.C.285t–2(a)) is amended— 
(A) 
in 
subsection 
(a), 
by 
striking 
‘‘$35,000’’ and inserting ‘‘$50,000’’; and 
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(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— 
(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 
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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 
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— 
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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.’’. 
(7) Section 487F of such Act (42 U.S.C. 288– 
6, as added by section 1002(b) of Public Law 106– 
310, is amended— 
(A) in subsection (a)(1), by striking 
‘‘$35,000’’ and inserting ‘‘$50,000’’; 
(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-
imum amount specified in such subsection (a) 
of such section.’’; and 
(C) by redesignating such section as sec-
tion 487G. 
SEC. 1042. REPORT. 
Not later than 18 months after the date of the enact-
ment of this Act, the Director of the National Institutes 
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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 
facilitate the successful transition or conclusion of re-
search programs, or for other purposes, as determined by 
the Director of NIH, in consultation with the directors 
of the national research institutes and national centers. 
‘‘(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-
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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 
Development and in collaboration with other 
appropriate 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’’; 
(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 
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(D) by striking ‘‘; or’’ at the end and in-
serting 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 in-
serting ‘‘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 coop-
erative agreements to supplement the salaries of new 
and early investigators who participate in the global 
pediatric 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 new and early investigators, to entities that par-
ticipate in the global pediatric clinical study net-
work; 
(3) the Food and Drug Administration should 
engage the European Medicines Agency and other 
foreign regulatory entities during the formation of 
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the global pediatric clinical study network to encour-
age their participation; and 
(4) once a global pediatric clinical study net-
work is established and becomes operational, the 
Food and Drug Administration should continue to 
engage the European Medicines Agency and other 
foreign regulatory entities to encourage and facili-
tate their participation 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 work-
shop 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 
(2) acceptable scientific justifications for ex-
cluding 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 Di-
rector of the National Institutes of Health shall publish 
guidelines— 
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(1) addressing the consideration of age as an 
inclusion variable in research involving human sub-
jects; and 
(2) identifying criteria for justifications for any 
age-related exclusions in such research. 
(c) PUBLIC AVAILABILITY OF FINDINGS AND CON-
CLUSIONS.—The Director of the National Institutes of 
Health shall— 
(1) make the findings and conclusion 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 pub-
lic on such website the number of children included 
in research that is conducted or supported by the 
National Institutes of Health, disaggregated by de-
velopmentally appropriate age group, race, and gen-
der. 
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Subtitle F—Advancement of 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) is amended by adding at the end the fol-
lowing: 
‘‘(m) 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 research 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 
following: 
‘‘(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 
paragraph (2)(A)(ii)(II), is submitted by per-
sons and posted by the Director of NIH in a 
standardized format and shall include at 
least— 
‘‘(i) the disease or indication being 
studied; 
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‘‘(ii) inclusion criteria such as age, 
gender, diagnosis or diagnoses, lab values, 
or imaging results; and 
‘‘(iii) exclusion criteria such as spe-
cific diagnosis or diagnoses, lab 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 
Classification of Diseases or the Current Proce-
dural Terminology, that facilitate electronic 
matching to data in electronic health records or 
other 
relevant 
health 
information 
tech-
nologies.’’. 
(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 (in-
cluding patients, researchers, physicians, industry rep-
resentatives, health information technology providers, the 
Food and Drug Administration, and standard setting or-
ganizations such as CDISC that have experience working 
with Federal agencies to standardize health data submis-
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sions) to receive advice on enhancements to the clinical 
trial registry data bank under section 402(j) of the Public 
Health Service Act (42 U.S.C. 282(j)) (including enhance-
ments 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 Di-
rector of the National Institutes of Health, shall enter into 
a cooperative 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 program with respect to all clinical trial data ob-
tained from qualified clinical trials for purposes of reg-
istered users conducting further research on such data. 
(b) APPLICATION.—Eligible entities seeking to enter 
into a cooperative agreement, contract, or grant with the 
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Secretary under this section shall submit to the Secretary 
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 with an eligi-
ble entity unless such entity submits an application includ-
ing 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 pur-
poses of sponsoring, operating, or participating in a 
clinical 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 
organizations with input from diverse stakeholder 
groups, and information demonstrating that the eli-
gible entity can de-identify clinical trial data con-
sistent with the requirements of section 164.514 of 
title 45, Code of Federal Regulations (or successor 
regulations). 
(3) A description of the system the eligible enti-
ty will use to store and maintain such data, and in-
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formation demonstrating that this system will com-
ply with applicable standards and requirements for 
ensuring the security of the clinical trial data. 
(4) A certification that the eligible entity will 
allow only registered users to access and use de- 
identified clinical trial data, gathered from qualified 
clinical 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 described in (e)(4)(B), and such other 
carefully controlled contractual terms as may be de-
fined by the Secretary. 
(5) Evidence demonstrating the ability of the 
eligible entity to ensure that registered users dis-
seminate the results of the research conducted in ac-
cordance with this section to interested parties to 
serve as a guide to future medical product develop-
ment or scientific 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 in sub-
section (d). 
(c) EXTENSION, EXPANSION, TERMINATION.—The 
Secretary, 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 terminate the pilot program established under 
such subsection, in whole, or in part. 
(d) STUDY AND REPORT.— 
(1) IN GENERAL.—The Secretary shall conduct 
a study and issue a report to the Congress, with re-
spect to the pilot program established under sub-
section (a), not later than 6 years after the date on 
which the pilot program is established under sub-
section (a). 
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(2) STUDY.—The study under paragraph (1) 
shall— 
(A) review the effectiveness of the pilot 
program 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 inquir-
ies, or clinical trials that have resulted from ac-
cessing clinical trial data under the pilot pro-
gram 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 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 emphasis of privacy and data in-
tegrity practices used in the program. 
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(e) DEFINITIONS.—In this section: 
(1) The term ‘‘eligible entity’’ means an entity 
that has experienced personnel with clinical and 
other technical expertise in the biomedical sciences 
and biomedical ethics and that is— 
(A) an institution of higher education (as 
such term is defined in section 1001 of the 
Higher 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 sec-
tion 501(a) of such title. 
(2) The term ‘‘medical product’’ means a drug 
(as defined in subsection (g) of section 201 of the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
331)), a device (as defined in subsection (h) of such 
section), 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 
Department of Health and Human Services with re-
spect to a medical product— 
(A) that was— 
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(i) approved or cleared under section 
505, 510(k), or 515, or has an exemption 
for investigational use in effect under sec-
tion 505 or 520(m), of the Federal Food, 
Drug, and Cosmetic Act (42 U.S.C. 301 et 
seq.); or 
(ii) licensed under section 351 of the 
Public Health Service Act (42 U.S.C. 262) 
or has an exemption for investigational use 
in effect under such section 351; or 
(B) that is an investigational product for 
which the original development was discon-
tinued 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 
undertaken by the sponsor of the original 
development program, its successors, as-
signs, 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 sci-
entific 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-identified clinical trial data from qualified 
clinical trials accessed through an eligible enti-
ty, use such data for reasons not specified in 
the research 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 Pre-
vention and in coordination with other agencies as deter-
mined appropriate 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 
designed in a manner that facilitates further research on 
neurological 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 as-
sociated with neurological diseases, including 
genetic 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 
treatment approaches for the diseases; and 
‘‘(E) the development of outcomes meas-
ures; 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 appro-
priate 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, 
public 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 
Secretary shall make information and analysis in the Na-
tional Neurological Diseases Surveillance System avail-
able, as appropriate— 
‘‘(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 avail-
able, 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 sub-
mit a report to the Congress concerning the implementa-
tion 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 appro-
priated $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 facilitate and expedite the development of medical 
products 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 de-
scribed in this subsection are to establish or facilitate the 
collection, maintenance, analysis, and interpretation of 
data regarding the natural history of diseases, with a par-
ticular 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 to 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 re-
spect to— 
‘‘(A) the design and conduct of disease 
studies; 
‘‘(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 par-
ticipates under subsection (a) shall be made available, con-
sistent with otherwise applicable Federal and State pri-
vacy laws, to the public (including patient advocacy 
groups, researchers, and drug developers) to help facilitate 
and expedite medical product development programs. 
‘‘(e) 
CONFIDENTIALITY.—Notwithstanding 
sub-
section (d), nothing in this section authorizes the disclo-
sure of any information that is a trade secret or commer-
cial or financial information that is privileged or confiden-
tial and subject 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.—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: 
‘‘(a) 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 regulation). 
‘‘(b) 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 
Secretary shall revise or clarify the rule to allow the use 
and disclosure of protected health information by a cov-
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ered entity 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 operations described in subparagraph (1) of the defi-
nition 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 
information described in subsection (a)— 
‘‘(1) the Secretary shall revise or clarify the 
Rule so that the disclosure may be made by the cov-
ered entity to only— 
‘‘(A) another covered entity for health care 
operations (as defined in such 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 such 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 dis-
closure of protected health information for research pur-
poses 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 in-
formation for research purposes are not subject to the lim-
itation 
on 
remuneration 
described 
in 
section 
164.502(a)(5)(ii)(B)(2)(ii) of part 164. 
‘‘(b) PERMITTED USES
AND DISCLOSURES.—The 
Secretary shall revise or clarify the Rule so that research 
activities, including comparative research activities, re-
lated to the quality, safety, or effectiveness of a product 
or activity that is regulated by the Food and Drug Admin-
istration are included as public health activities for pur-
poses of which a covered entity may disclose protected 
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health information 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 
information 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 
copied 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 
clarify the Rule to specify that an authorization for the 
use or disclosure of protected health information, with re-
spect to an individual, for future research purposes shall 
be deemed to contain a sufficient description of the pur-
pose of the use or disclosure if the authorization— 
‘‘(1) sufficiently describes the purposes such 
that it would be reasonable for the individual to ex-
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pect 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 
purposes such as is described by subsection (a), the cov-
ered entity may not make any further uses or disclosures 
based on that authorization, except, as provided in para-
graph (b)(5) of section 164.508 of part 164, to the extent 
that the covered entity has taken action in reliance on the 
authorization.’’. 
(b) REVISION OF REGULATIONS.—Not later than 12 
months after the date of the enactment of this Act, the 
Secretary of Health and Human Services shall revise and 
clarify the provisions of title 45, Code of Federal Regula-
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tions, 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 fol-
lowing: 
‘‘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 Govern-
ment. 
‘‘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. 
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‘‘SEC. 281B. DUTIES. 
‘‘For the purpose described in section 281A, the 
Council shall— 
‘‘(1) foster collaboration and coordination 
among the entities that comprise the Council, includ-
ing academia, government agencies, industry, health 
care payors and providers, patient advocates, and 
others engaged in the cycle of discovery, develop-
ment, and delivery of life-saving and health-enhanc-
ing innovative interventions; 
‘‘(2) undertake communication and dissemina-
tion activities; 
‘‘(3) publish information on the activities fund-
ed under section 281D; 
‘‘(4) establish a strategic agenda for accel-
erating 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; 
‘‘(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; 
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‘‘(8) propose recommendations that will facili-
tate 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 
composed of the ex officio members under sub-
paragraph (B) and the appointed members 
under subparagraph (C). All members of the 
Board shall be voting members. 
‘‘(B) EX OFFICIO MEMBERS.—The ex offi-
cio members of the Board shall be the following 
individuals or their designees: 
‘‘(i) The Director of the National In-
stitutes of Health. 
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‘‘(ii) The Commissioner of Food and 
Drugs. 
‘‘(iii) The Administrator of the Cen-
ters 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 by the Comptroller 
General of the United States from a list of 
nominations submitted by leading trade as-
sociations— 
‘‘(I) 4 of whom shall be rep-
resentatives of the biopharmaceutical 
industry; 
‘‘(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 
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‘‘(ii) 9 shall be appointed by the 
Comptroller General of the United States, 
after soliciting nominations— 
‘‘(I) 2 of whom shall be rep-
resentatives of academic researchers; 
‘‘(II) 3 of whom shall be rep-
resentative 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 majority vote from among the members of 
the Board. 
‘‘(2) TERMS AND VACANCIES.— 
‘‘(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— 
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‘‘(i) shall not affect the power of the 
remaining 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 
individual. 
‘‘(3) RESPONSIBILITIES.—Not later than 90 
days after the date on which the Council is incor-
porated and its Board of Directors is fully con-
stituted, the Board of Directors shall establish by-
laws and policies for the Council that— 
‘‘(A) are published in the Federal Register 
and available for public comment; 
‘‘(B) establish policies for the selection 
and, as applicable, appointment of— 
‘‘(i) the officers, employees, agents, 
and contractors of the Council; and 
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‘‘(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 Direc-
tors shall— 
‘‘(i) meet on a quarterly basis; and 
‘‘(ii) submit to Congress, and make 
publicly available, the minutes of such 
meetings. 
‘‘(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 de-
scribed in section 281A; and 
‘‘(ii) annually thereafter, in consulta-
tion with the Executive Director, review 
and update such agenda. 
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‘‘(b) APPOINTMENT
AND
INCORPORATION.—Not 
later than 6 months after the date of enactment of the 
21st Century 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 
necessary to incorporate the Council. 
‘‘(c) NONPROFIT STATUS.—In carrying out this part, 
the Board of Directors shall establish such policies and 
bylaws, and the Executive Director shall carry out such 
activities, 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 Direc-
tor shall— 
‘‘(1) be the chief executive officer of the Coun-
cil; and 
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‘‘(2) subject to the oversight of the Board of 
Directors, be responsible for the day-to-day manage-
ment of the Council. 
‘‘SEC. 281D. OPERATIONAL ACTIVITIES AND ASSISTANCE. 
‘‘(a) IN GENERAL.—The Council shall establish a 
sufficient operational infrastructure to fulfill the duties 
specified in section 281B. 
‘‘(b) PRIVATE SECTOR MATCHING FUNDS.—The 
Council may accept financial or in-kind support from par-
ticipating 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 ap-
propriate congressional committees a report on the per-
formance of the Council. In preparing such report, the 
Council shall consult with a nongovernmental consultant 
with appropriate expertise. 
‘‘SEC. 281F. FUNDING. 
‘‘For the each of fiscal years 2016 through 2023, 
there is authorized to be appropriated $10,000,000 to the 
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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 Sec-
retary shall implement’’ and all that follows through 
‘‘premarket 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 frame-
work in the new drug approval process— 
‘‘(A) to facilitate the balanced consider-
ation of benefits and risks; and 
‘‘(B) to develop and implement a con-
sistent and systematic approach to the discus-
sion of, regulatory decisionmaking with respect 
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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 
ASSESSMENT FRAMEWORK.— 
‘‘(1) IN GENERAL.—Not later than two years 
after the date of the enactment of this subsection, 
the Secretary shall establish and implement proc-
esses under which— 
‘‘(A) an entity seeking to develop patient 
experience 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 sum-
maries and analyses of such data; 
‘‘(B) the Secretary may request such an 
entity to submit such documents, data, and 
summaries and analyses; and 
‘‘(C) patient experience data may be devel-
oped and used to enhance the structured risk- 
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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, pa-
tient advocacy organizations, disease research foun-
dations, medical researchers, research sponsors, or 
other parties determined appropriate by the Sec-
retary that is intended to facilitate or enhance the 
Secretary’s risk-benefit assessments, including infor-
mation 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 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
355), as added by subsection (a). Such guidance 
shall include— 
(A) with respect to draft guidance docu-
ments, data, or summaries and analyses sub-
mitted to the Secretary under paragraph (1)(A) 
of such subsection, guidance— 
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(i) specifying the timelines for the re-
view of such documents, data, or sum-
maries 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 mainte-
nance of registries designed to increase un-
derstanding of the natural history of a dis-
ease; 
(C) methodological approaches that may be 
used to assess patients’ beliefs with respect to 
the benefits and risks in the management of the 
patient’s disease; and 
(D) methodologies, standards, and poten-
tial experimental designs for patient-reported 
outcomes. 
(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 Secretary shall issue final guidance on the im-
plementation 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 fol-
lowing 12-month period, the Secretary of 
Health and Human Services shall convene a 
workshop to obtain input regarding methodolo-
gies for developing the guidance under para-
graph (1), including the collection of patient ex-
perience 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 Adminis-
tration; 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 
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and Human Services shall convene a public meeting 
to solicit input on the guidance. 
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 section 507 of the Federal Food, 
Drug, and Cosmetic Act, as added by subsection (c)) 
can play a valuable role in helping develop and qual-
ify drug development tools. 
(b) SENSE OF CONGRESS.—It is the sense of Con-
gress that— 
(1) Congress should promote and facilitate a 
collaborative effort among the biomedical research 
consortia described in subsection (a)(3)— 
(A) to develop, through a transparent pub-
lic process, data standards and scientific ap-
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proaches to data collection accepted by the 
medical and clinical research community for 
purposes of qualifying drug development tools; 
(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 de-
velopment 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 Cos-
metic 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 estab-
lish a process for the qualification of drug develop-
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ment tools for a proposed context of use under 
which— 
‘‘(A)(i) a requestor initiates such process 
by submitting a letter of intent to the Sec-
retary; and 
‘‘(ii) the Secretary shall accept or decline 
to accept such letter of intent; 
‘‘(B)(i) if the Secretary accepts the letter 
of intent, a requestor shall submit a qualifica-
tion plan to the Secretary; and 
‘‘(ii) the Secretary shall accept or decline 
to accept the qualification plan; and 
‘‘(C)(i) if the Secretary accepts the quali-
fication plan, the requestor submits to the Sec-
retary a full qualification package; 
‘‘(ii) the Secretary shall determine whether 
to accept such qualification package for review; 
and 
‘‘(iii) if the Secretary accepts such quali-
fication package for review, conduct such review 
in accordance with this section. 
‘‘(2) ACCEPTANCE
AND
REVIEW
OF
SUBMIS-
SIONS.— 
‘‘(A) IN GENERAL.—The succeeding provi-
sions of this paragraph shall apply with respect 
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to the treatment of a letter of intent, a quali-
fication plan, or a full qualification package 
submitted under paragraph (1) (referred to in 
this paragraph as ‘qualification submissions’). 
‘‘(B) ACCEPTANCE FACTORS; NON-ACCEPT-
ANCE.—The Secretary shall determine whether 
to accept a qualification submission based on 
factors 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 qualification of a drug development tool for 
its proposed 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 
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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 
and other expert stakeholders, of such a 
drug development tool and its proposed 
context 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 re-
spect 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 
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‘‘(B) determine whether the drug develop-
ment tool at issue is qualified for its proposed 
context of use. 
‘‘(4) QUALIFICATION.—The Secretary shall de-
termine whether a drug development tool is qualified 
for a proposed context of use based on the scientific 
merit of a full qualification package reviewed under 
paragraph (3). 
‘‘(b) EFFECT OF QUALIFICATION.— 
‘‘(1) IN GENERAL.—A drug development tool 
determined to be qualified under subsection (a)(4) 
for a proposed context of use specified by the re-
questor 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 
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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 
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 grant-
ed a request for a meeting with the Secretary 
to discuss the basis of the Secretary’s decision 
to rescind or modify the determination before 
the effective date of the rescission or modifica-
tion. 
‘‘(c) TRANSPARENCY.— 
‘‘(1) IN GENERAL.—Subject to paragraph (3), 
the Secretary shall make publicly available, and up-
date on at least a biannual basis, on the Internet 
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website of the Food and Drug Administration the 
following: 
‘‘(A) Information with respect to each 
qualification submission under the qualification 
process under subsection (a), including— 
‘‘(i) the stage of the review process 
applicable to the submission; 
‘‘(ii) the date of the most recent 
change in stage status; 
‘‘(iii) whether the external scientific 
experts 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. 
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‘‘(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 
(including in accordance with section 
506(c)) under section 505 of this Act or 
section 351 of the Public Health Service 
Act. 
‘‘(2) RELATION TO TRADE SECRETS ACT.—In-
formation 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 
disclose any information contained in an application 
submitted under section 505 of this Act or section 
351 of the Public Health Service Act that is con-
fidential commercial or trade secret information sub-
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ject to section 552(b)(4) of title 5, United States 
Code, or section 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 
subsection (c) or (d) of section 505, including the 
substantial 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 to this Act or the 
Public Health Service Act, as applicable (as in effect 
before 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 
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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 
(20 U.S.C. 1001)), patient advocacy groups, indus-
try representatives, clinical and scientific experts, 
and other relevant entities and individuals. 
‘‘(3) CLINICAL
OUTCOME
ASSESSMENT.—(A) 
The term ‘clinical outcome assessment’ means a 
measurement of a patient’s symptoms, overall men-
tal 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. 
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‘‘(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 sta-
tus of the patient’s health condition without amend-
ment or interpretation of the patient’s report by a 
clinician or any other person. 
‘‘(7) QUALIFICATION.—The terms ‘qualifica-
tion’ and ‘qualified’ mean a determination by the 
Secretary that a drug development tool and its pro-
posed context of use can be relied upon to have a 
specific interpretation and application in drug devel-
opment 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. 
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‘‘(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 measure-
ment 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 accel-
erated approval of a drug or biological product 
in accordance with section 506(c).’’. 
(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 
interested 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-
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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 proc-
ess under such section 507— 
(i) describes the requirements that en-
tities seeking to qualify a drug develop-
ment tool under such section shall observe 
when engaging in such process; 
(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 
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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 
shall issue draft guidance under paragraph (1) on 
the implementation of section 507 of the Federal 
Food, Drug, and Cosmetic Act (as added by sub-
section (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 pe-
riod for the draft guidance closes. 
(3) TAXONOMY.— 
(A) IN
GENERAL.—For purposes of in-
forming guidance under this subsection, the 
Secretary shall, in consultation with biomedical 
research consortia and other interested parties 
through a collaborative public process, establish 
a taxonomy for the classification 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 shall make such tax-
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onomy publicly available in draft form for pub-
lic comment. The Secretary shall finalize the 
taxonomy not later than 12 months after the 
close of the public comment period. 
(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 pub-
lic meeting to describe and solicit public input re-
garding the qualification process under section 507 
of the Federal Food, Drug, and Cosmetic Act, as 
added by subsection (c). 
(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 de-
velopment tool (as defined in subsection (f) of 
such section); 
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(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 
development 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 
(3) the drug development tools qualified 
through such qualification process, specified by type 
of tool, such as a biomarker or clinical outcome as-
sessment (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-
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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 sec-
tion 351(a)(3) of the Public Health Service Act, that 
the Secretary agree to an accelerated approval devel-
opment 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— 
‘‘(A) the surrogate endpoint to be assessed 
under such plan; 
‘‘(B) the design of the study that will uti-
lize the surrogate endpoint; and 
‘‘(C) the magnitude of the effect of the 
drug on the surrogate endpoint that is the sub-
ject of the agreement that would be sufficient 
to form the primary basis of a claim that the 
drug is effective. 
‘‘(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— 
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‘‘(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 ac-
celerated approval under subsection (c). 
‘‘(4) SPONSOR
CONSULTATION.—If the Sec-
retary requires the modification or termination of an 
accelerated approval development plan under para-
graph (3), the sponsor shall be granted a request for 
a meeting to discuss the basis of the Secretary’s de-
cision before the effective date of the modification or 
termination. 
‘‘(5) DEFINITION.—In this section, the term 
‘accelerated approval development plan’ means a de-
velopment plan agreed upon by the Secretary and 
the sponsor submitting the plan that contains study 
parameters 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 ac-
celerated approval of a drug in accordance with 
subsection (c).’’. 
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(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 in-
serting ‘‘(f) CONSTRUCTION’’. 
Subtitle C—FDA Advancement of 
Precision Medicine 
SEC. 2041. PRECISION MEDICINE GUIDANCE AND OTHER 
PROGRAMS OF FOOD AND DRUG ADMINIS-
TRATION. 
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 
periodically update guidance to assist sponsors in the de-
velopment of a precision drug or biological product. Such 
guidance shall— 
‘‘(1) define the term ‘precision drug or biologi-
cal product’; and 
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‘‘(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 iden-
tify subsets of patients as likely responders to thera-
pies in order to streamline the conduct of clinical 
trials; 
‘‘(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. 
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‘‘(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 Public Health Service Act, for the treatment of a seri-
ous or life-threatening disease or condition and has been 
designated under section 526 as a drug for a rare disease 
or condition, the Secretary may— 
‘‘(1) consistent with applicable standards for 
approval, rely upon data or information previously 
submitted by the sponsor of the precision drug or bi-
ological product, or another sponsor, provided that 
the sponsor of the precision drug or biological prod-
uct has obtained a contractual right of reference to 
such other sponsor’s data and information, in an ap-
plication approved under section 505(c) or licensed 
under section 351(a) of the Public Health Service 
Act, as applicable— 
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‘‘(A) for a different drug or biological 
product; or 
‘‘(B) for a different indication for such 
precision 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 appropriate; and 
‘‘(2) as appropriate, consider the application for 
approval of such precision drug or biological product 
to be eligible for expedited review and approval pro-
grams described in section 506, including acceler-
ated 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 au-
thorized under this Act prior to enactment of this 
section, with respect to a sponsor’s ability to ref-
erence information contained in another application 
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submitted under section 505(b)(1) of this Act or sec-
tion 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 STATIS-
TICAL METHODS IN CLINICAL PROTOCOLS FOR DRUGS 
AND BIOLOGICAL PRODUCTS.—For purposes of assisting 
sponsors in incorporating adaptive trial design and 
Bayesian methods into proposed clinical protocols and ap-
plications for new drugs under section 505 of the Federal 
Food, Drug, and Cosmetic Act (21 U.S.C. 355) and bio-
logical 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— 
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(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 para-
graph (1) shall address— 
(A) the use of adaptive trial designs and 
Bayesian methods in clinical trials, including 
clinical trials proposed or submitted to help sat-
isfy the substantial evidence standard under 
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 modeling and simulations prior to— 
(i) completion of such modeling or 
simulations; or 
(ii) the submission of resulting infor-
mation to the Secretary; 
(C) the types of quantitative and quali-
tative information that should be submitted for 
review; and 
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(D) recommended analysis methodologies. 
(3) PUBLIC
MEETING.—Prior to updating or 
developing the guidances required by paragraph (1), 
the Secretary shall consult with stakeholders, includ-
ing representatives of regulated industry, academia, 
patient 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 
date of the public meeting required by para-
graph (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, as amended by section 2021, is further amended by 
inserting after section 505E of such Act (21 U.S.C. 355f) 
the following: 
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‘‘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 
clinical experience— 
‘‘(1) to help support the approval of a new indi-
cation for a drug approved under section 505(b); 
and 
‘‘(2) to help support or satisfy postapproval 
study requirements. 
‘‘(b) EVIDENCE FROM CLINICAL EXPERIENCE DE-
FINED.—In this section, the term ‘evidence from clinical 
experience’ means data regarding the usage, or the poten-
tial benefits or risks, of a drug derived from sources other 
than randomized clinical trials, including from observa-
tional 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 
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100 
safety surveillance, registry, claims, and pa-
tient-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 gen-
erated through clinical experience; and 
‘‘(D) the priority areas, remaining chal-
lenges, and potential pilot opportunities that 
the program established under this section will 
address. 
‘‘(3) CONSULTATION.— 
‘‘(A) IN GENERAL.—In developing the pro-
gram framework under this subsection, the Sec-
retary shall consult with regulated industry, 
academia, medical professional organizations, 
representatives of patient advocacy organiza-
tions, 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 
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101 
‘‘(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 enact-
ment of this section and in accordance with the framework 
established under subsection (c), implement the program 
to evaluate the potential use of evidence from clinical expe-
rience. 
‘‘(e) GUIDANCE
FOR
INDUSTRY.—The Secretary 
shall— 
‘‘(1) utilize the program established in sub-
section (d), its activities, and any subsequent pilots 
or written reports, to inform a guidance for industry 
on— 
‘‘(A) the circumstances under which spon-
sors of drugs and the Secretary may rely on 
evidence from clinical experience for the pur-
poses 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; 
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102 
‘‘(2) not later than 36 months after the date of 
enactment of this section, issue draft guidance for 
industry 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 non-specified use. 
‘‘(2) This section shall not be construed to 
alter— 
‘‘(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. 
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103 
‘‘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 in 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 author-
ized 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-
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 
approved 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 
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104 
and infrastructure the Secretary determines ap-
propriate 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 inter-
ested 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— 
‘‘(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 
conduct 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 
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105 
‘‘(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 pub-
lic health activities, permitting the use and dis-
closure of protected health information as de-
scribed in section 164.512(b)(1)(iii) of title 45, 
Code of Federal Regulations (or any successor 
regulation) and exempted as a public health ac-
tivity as described in section 46.101(b)(5) of 
title 46, Code of Federal Regulations (or any 
successor regulation); and 
‘‘(B) deem safety surveillance performed at 
the request of the Food and Drug Administra-
tion or under such jurisdiction by a sponsor 
with responsibility for a drug approved under 
this section or section 351 of the Public Health 
Services Act using the Sentinel System surveil-
lance infrastructure authorized under section 
505(k), including use of analytic tools and 
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106 
querying capabilities developed to implement 
the active postmarket surveillance system de-
scribed in this section, public health activities 
as described in section 164.512(b)(1)(iii) of title 
45, Code of Federal Regulations (or any suc-
cessor regulation) and exempted as a public 
health 
activity 
as 
described 
in 
section 
46.101(b)(5) of title 46, Code of Federal Regu-
lations (or any successor regulation). 
‘‘(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.’’. 
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 
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107 
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 
include 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 in-
dications, and such approval or licensure remains in 
effect; 
‘‘(2) the supplemental application is for ap-
proval or licensure (as applicable) under such section 
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 ap-
proved under such section 505(c) or licensed under 
such section 351(a); 
‘‘(4) the supplemental application incorporates 
or supplements the data submitted in the application 
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108 
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 
PROGRAM.—The Secretary shall post on the public website 
of the Food and Drug Administration and update annu-
ally— 
‘‘(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 
‘‘(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 
cancer, as determined appropriate by the Sec-
retary; or 
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109 
‘‘(B) such other types of indications as the 
Secretary determines to be subject to the 
streamlined 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 
indication for use of a drug.’’. 
(b) SENSE OF CONGRESS.—It is the sense of Con-
gress that the streamlined data review program under sec-
tion 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 cer-
tain supplemental applications based on qualified data 
summaries (as defined in such section 505H). 
(c) GUIDANCE; REGULATIONS.—The Commissioner 
of Food and Drugs— 
(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 sub-
section (a), not later than 24 months after the 
date of enactment of this Act; and 
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110 
(B) include in such guidance the process 
for expanding the types of indications to be 
subject to the streamlined data review program, 
as authorized 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 
necessary 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 
Administration should continue to expedite the approval 
of drugs designated as breakthrough therapies pursuant 
to section 506(a) of the Federal Food, Drug, and Cos-
metic Act (21 U.S.C. 356(a)) by approving drugs so des-
ignated as early as possible in the clinical development 
process, regardless of the phase of development, provided 
that the Secretary of Health and Human Services deter-
mines that an application for such a drug meets the stand-
ards of evidence of safety 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)). 
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111 
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, 
monitoring, or treatment of one or more serious diseases 
or conditions shall make publicly available the policy of 
the manufacturer or distributor on evaluating and re-
sponding 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 of distributor’s investigational drugs. 
‘‘(b) CONTENT OF POLICY.—A policy described in 
subsection (a) shall include making publicly available— 
‘‘(1) contact information for the manufacturer 
or distributor to facilitate communication about re-
quests described in subsection (a); 
‘‘(2) procedures for making such requests; 
‘‘(3) the general criteria the manufacturer or 
distributor will consider or use to approve such re-
quests; and 
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‘‘(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 
policies by manufacturers and distributors under sub-
section (a) shall not serve as a guarantee of access to any 
specific investigational 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 
enactment 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 Regu-
lations (or any successor regulations)) with respect 
to such investigational 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 enti-
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tled ‘‘Expanded Access to Investigational Drugs for Treat-
ment Use—Qs & As’’ and dated May 2013. 
(b) CONTENTS.—The final guidance referred to in 
subsection (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’’; 
(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 
selection of drugs for managed care or other similar 
organizations’’ and inserting ‘‘a payor, formulary 
committee, or other similar entity with knowledge 
and expertise in the area of health care economic 
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114 
analysis, carrying out its responsibilities for the se-
lection 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 economic information provided to such a com-
mittee or entity in accordance with this paragraph’’ 
and inserting ‘‘, is based on competent and reliable 
scientific evidence, and includes, where applicable, a 
conspicuous and prominent statement describing any 
material differences between the health care eco-
nomic 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 section 505(a) or in subsections (a) and (k) 
of section 351 of the Public Health Service Act shall 
not apply to health care economic information pro-
vided to such a payor, committee, or entity in ac-
cordance with this paragraph’’; and 
(5) by striking ‘‘In this paragraph, the term’’ 
and all that follows and inserting the following: 
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‘‘(2)(A) For purposes of this paragraph, the term 
‘health care economic information’ means any analysis (in-
cluding the clinical data, inputs, clinical or other assump-
tions, methods, results, and other components underlying 
or comprising the analysis) that identifies, measures, or 
describes the economic consequences, which may be based 
on the separate or aggregated clinical consequences of the 
represented health outcomes, of the use of a drug. Such 
analyses may be comparative to the use of another drug, 
to another health care intervention, or to no intervention. 
‘‘(B) Such term does not include any analysis that 
relates only to an indication that is not approved under 
section 505 or under section 351 of the Public Health 
Service Act for such drug.’’. 
SEC. 2102. FACILITATING RESPONSIBLE COMMUNICATION 
OF 
SCIENTIFIC 
AND 
MEDICAL 
DEVELOP-
MENTS. 
(a) GUIDANCE.—Not later than 18 months after the 
date of enactment of this Act, the Secretary of Health and 
Human Services shall issue draft guidance on facilitating 
the responsible dissemination of truthful and non-mis-
leading scientific and medical information not included in 
the approved labeling of drugs and devices. 
(b) DEFINITION.—In this section, the terms ‘‘drug’’ 
and ‘‘device’’ have the meaning given to such terms in sec-
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116 
tion 201 of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C. 321). 
Subtitle G—Antibiotic Drug 
Development 
SEC. 2121. APPROVAL OF CERTAIN DRUGS FOR USE IN A 
LIMITED POPULATION OF PATIENTS. 
(a) PURPOSE.—The purpose of this section is to help 
expedite the development and availability of treatments for 
serious or life-threatening bacterial or fungal infections in 
patients with unmet needs, while maintaining safety and 
effectiveness standards for such treatments, taking into 
account the severity of the infection and the availability 
or lack of alternative treatments. 
(b) APPROVAL
OF CERTAIN ANTIBACTERIAL
AND 
ANTIFUNGAL DRUGS.—Section 505 of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 355), as amended by 
section 2001, is further amended by adding at the end 
the following new subsection: 
‘‘(z) APPROVAL OF CERTAIN ANTIBACTERIAL AND 
ANTIFUNGAL DRUGS
FOR USE
IN
A LIMITED POPU-
LATION OF PATIENTS.— 
‘‘(1) PROCESS.—At the request of the sponsor 
of an antibacterial or antifungal drug that is in-
tended to treat a serious or life-threatening infec-
tion, the Secretary— 
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‘‘(A) may execute a written agreement 
with the sponsor on the process for developing 
data to support an application for approval of 
such drug, for use in a limited population of pa-
tients in accordance with this subsection; 
‘‘(B) shall proceed with the development 
and approval of such a drug in accordance with 
this subsection only if a written agreement is 
reached under subparagraph (A); 
‘‘(C) shall provide the sponsor with an op-
portunity to request meetings under paragraph 
(2); 
‘‘(D) if a written agreement is reached 
under subparagraph (A), may approve the drug 
under this subsection for such use — 
‘‘(i) in a limited population of patients 
for which there is an unmet medical need; 
‘‘(ii) based on a streamlined develop-
ment program; and 
‘‘(iii) only if the standards for ap-
proval under subsections (c) and (d) of this 
section or licensure under section 351 of 
the Public Health Service Act, as applica-
ble, are met; and 
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‘‘(E) in approving a drug in accordance 
with this subsection, subject to subparagraph 
(D)(iii), may rely upon— 
‘‘(i) traditional endpoints, alternate 
endpoints, or a combination of traditional 
and alternate endpoints, and, as appro-
priate, data sets of a limited size; and 
‘‘(ii)(I) additional data, including pre-
clinical, pharmacologic, or pathophysiologic 
evidence; 
‘‘(II) nonclinical susceptibility and 
pharmacokinetic data; 
‘‘(III) data from phase 2 clinical 
trials; and 
‘‘(IV) such other confirmatory evi-
dence as the Secretary determines appro-
priate to approve the drug. 
‘‘(2) FORMAL MEETINGS.— 
‘‘(A) IN GENERAL.—To help expedite and 
facilitate the development and review of a drug 
for which a sponsor intends to request approval 
in accordance with this subsection, the Sec-
retary may, at the request of the sponsor, con-
duct meetings that provide early consultation, 
timely advice, and sufficient opportunities to 
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119 
develop an agreement described in paragraph 
(1)(A) and help the sponsor design and conduct 
a drug development program as efficiently as 
possible, including the following types of meet-
ings: 
‘‘(i) An early consultation meeting. 
‘‘(ii) An assessment meeting. 
‘‘(iii) A postapproval meeting. 
‘‘(B) NO ALTERING OF GOALS.—Nothing 
in this paragraph shall be construed to alter 
agreed upon goals and procedures identified in 
the letters described in section 101(b) of the 
Prescription Drug User Fee Amendments of 
2012. 
‘‘(C) BREAKTHROUGH THERAPIES.—In the 
case of a drug designated as a breakthrough 
therapy under section 506(a), the sponsor of 
such drug may elect to utilize meetings pro-
vided under such section with respect to such 
drug in lieu of meetings described in subpara-
graph (A). 
‘‘(3) LABELING
REQUIREMENT.—The labeling 
of an antibacterial or antifungal drug approved in 
accordance with this subsection shall contain the 
statement ‘Limited Population’ in a prominent man-
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120 
ner and adjacent to, and not more prominent than, 
the brand name of the product. The prescribing in-
formation for such antibacterial or antifungal drug 
required by section 201.57 of title 21, Code of Fed-
eral Regulations (or any successor regulation) shall 
also include the following statement: ‘This drug is 
indicated for use in a limited and specific population 
of patients.’. 
‘‘(4) PROMOTIONAL
MATERIALS.—The provi-
sions of section 506(c)(2)(B) shall apply with re-
spect to approval in accordance with this subsection 
to the same extent and in the same manner as such 
provisions apply with respect to accelerated approval 
in accordance with section 506(c)(1). 
‘‘(5) TERMINATION OF REQUIREMENTS OR CON-
DITIONS.—If a drug is approved in accordance with 
this subsection for an indication in a limited popu-
lation of patients and is subsequently approved or li-
censed under this section or section 351 of the Pub-
lic Health Service Act, other than in accordance with 
this subsection, for— 
‘‘(A) the same indication and the same 
conditions of use, the Secretary shall remove 
any labeling requirements or postmarketing 
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121 
conditions that were made applicable to the 
drug under this subsection; or 
‘‘(B) a different indication or condition of 
use, the Secretary shall not apply the labeling 
requirements and postmarketing conditions that 
were made applicable to the drug under this 
subsection to the subsequent approval of the 
drug for such different indication or condition 
of use. 
‘‘(6) RELATION TO OTHER PROVISIONS.—Noth-
ing in this subsection shall be construed to prohibit 
the approval of a drug for use in a limited popu-
lation of patients in accordance with this subsection, 
in combination with— 
‘‘(A) an agreement on the design and size 
of a clinical trial pursuant to subparagraphs 
(B) and (C) of subsection (b)(5); 
‘‘(B) designation and treatment of the 
drug as a breakthrough therapy under section 
506(a); 
‘‘(C) designation and treatment of the 
drug as a fast track product under section 
506(b); or 
‘‘(D) accelerated approval of the drug in 
accordance with section 506(c). 
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122 
‘‘(7) RULE
OF
CONSTRUCTION.—Nothing in 
this subsection shall be construed— 
‘‘(A) to alter the standards of evidence 
under subsection (c) or (d) (including the sub-
stantial evidence standard in subsection (d)); 
‘‘(B) to waive or otherwise preclude the ap-
plication of requirements under subsection (o); 
‘‘(C) to otherwise, in any way, limit the au-
thority of the Secretary to approve products 
pursuant to this Act and the Public Health 
Service Act as authorized prior to the date of 
enactment of this subsection; or 
‘‘(D) to restrict in any manner, the pre-
scribing of antibiotics or other products by 
health care providers, or to otherwise limit or 
restrict the practice of health care. 
‘‘(8) EFFECTIVE
IMMEDIATELY.—The Sec-
retary shall have the authorities vested in the Sec-
retary by this subsection beginning on the date of 
enactment of this subsection, irrespective of when 
and whether the Secretary promulgates final regula-
tions or guidance. 
‘‘(9) DEFINITIONS.—In this subsection: 
‘‘(A) EARLY
CONSULTATION
MEETING.— 
The term ‘early consultation meeting’ means a 
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123 
pre-investigational new drug meeting or an end- 
of-phase 1 meeting that— 
‘‘(i) is conducted to review and reach 
a written agreement— 
‘‘(I) on the scope of the stream-
lined development plan for a drug for 
which a sponsor intends to request ap-
proval in accordance with this sub-
section; and 
‘‘(II) which, as appropriate, may 
include agreement on the design and 
size of necessary preclinical and clin-
ical studies early in the development 
process, including clinical trials whose 
data are intended to form the primary 
basis for an effectiveness claim; and 
‘‘(ii) provides an opportunity to dis-
cuss expectations of the Secretary regard-
ing studies or other information that the 
Secretary deems appropriate for purposes 
of applying paragraph (5), relating to the 
termination of labeling requirements or 
postmarketing conditions. 
‘‘(B) ASSESSMENT
MEETING.—The term 
‘assessment meeting’ means an end-of-phase 2 
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124 
meeting, pre-new drug application meeting, or 
pre-biologics license application meeting con-
ducted to resolve questions and issues raised 
during the course of clinical investigations, and 
details addressed in the written agreement re-
garding postapproval commitments or expan-
sion of approved uses. 
‘‘(C) POSTAPPROVAL MEETING.—The term 
‘postapproval meeting’ means a meeting fol-
lowing initial approval or licensure of the drug 
for use in a limited population, to discuss any 
issues identified by the Secretary or the sponsor 
regarding postapproval commitments or expan-
sion of approved uses.’’. 
(c) GUIDANCE.—Not later than 18 months after the 
date of enactment of this Act, the Secretary of Health and 
Human Services, acting through the Commissioner of 
Food and Drugs, shall issue draft guidance describing cri-
teria, process, and other general considerations for dem-
onstrating the safety and effectiveness of antibacterial and 
antifungal drugs to be approved for use in a limited popu-
lation in accordance with section 505(z) of the Federal 
Food, Drug, and Cosmetic Act, as added by subsection 
(b). 
(d) CONFORMING AMENDMENTS.— 
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125 
(1) LICENSURE OF CERTAIN BIOLOGICAL PROD-
UCTS.—Section 351(j) of the Public Health Service 
Act (42 U.S.C. 262(j)) is amended— 
(A) 
by 
striking 
‘‘(j)’’ 
and 
inserting 
‘‘(j)(1)’’; 
(B) by inserting ‘‘505(z),’’ after ‘‘505(p),’’; 
and 
(C) by adding at the end the following new 
paragraph: 
‘‘(2) In applying section 505(z) of the Federal Food, 
Drug, and Cosmetic Act to the licensure of biological prod-
ucts under this section— 
‘‘(A) references to an antibacterial or antifungal 
drug that is intended to treat a serious or life- 
threatening infection shall be construed to refer to 
a biological product intended to treat a serious or 
life-threatening bacterial or fungal infection; and 
‘‘(B) references to approval of a drug under 
section 505(c) of such Act shall be construed to 
refer to a licensure of a biological product under 
subsection (a) of this section.’’. 
(2) MISBRANDING.—Section 502 of the Federal 
Food, Drug, and Cosmetic Act (21 U.S.C. 352) is 
amended by adding at the end the following new 
subsection: 
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126 
‘‘(dd) If it is a drug approved in accordance with sec-
tion 505(z) and its labeling does not meet the require-
ments under paragraph (3) of such subsection, subject to 
paragraph (5) of such subsection.’’. 
(e) EVALUATION.— 
(1) ASSESSMENT.—Not later than 48 months 
after the date of enactment of this Act, the Sec-
retary of Health and Human Services shall publish 
for public comment an assessment of the program 
established under section 505(z) of the Federal 
Food, Drug, and Cosmetic Act, as added by sub-
section (b). Such assessment shall determine if the 
limited-use pathway established under such section 
505(z) has improved or is likely to improve patient 
access to novel antibacterial or antifungal treat-
ments and assess how the pathway could be ex-
panded to cover products for serious or life-threat-
ening diseases or conditions beyond bacterial and 
fungal infections. 
(2) MEETING.—Not later than 90 days after 
the date of the publication of such assessment, the 
Secretary, acting through the Commissioner of Food 
and Drugs shall hold a public meeting to discuss the 
findings of the assessment, during which public 
stakeholders may present their views on the success 
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127 
of the program established under section 505(z) of 
the Federal Food, Drug, and Cosmetic Act, as 
added by subsection (b), and the appropriateness of 
expanding such program. 
(f) EXPANSION OF PROGRAM.—If the Secretary of 
Health and Human Services determines, based on the as-
sessment under subsection (e)(1), evaluation of the assess-
ment, and any other relevant information, that the public 
health would benefit from expansion of the limited-use 
pathway established under section 505(z) of the Federal 
Food, Drug, and Cosmetic Act (as added by subsection 
(b)) beyond the drugs approved in accordance with such 
section, the Secretary may expand such limited-use path-
way in accordance with such a determination. The ap-
proval of any drugs under any such expansion shall be 
subject to the considerations and requirements described 
in such section 505(z) for purposes of expansion to other 
serious or life-threatening diseases or conditions. 
(g) MONITORING.—The Public Health Service Act is 
amended by inserting after section 317T (42 U.S.C. 
247b–22) the following: 
‘‘SEC. 
317U. 
MONITORING 
ANTIBACTERIAL 
AND 
ANTIFUNGAL DRUG USE AND RESISTANCE. 
‘‘(a) MONITORING.—The Secretary shall use an ap-
propriate monitoring system to monitor— 
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‘‘(1) the use of antibacterial and antifungal 
drugs, including those receiving approval or licensure 
for a limited population pursuant to section 505(z) 
of the Federal Food, Drug, and Cosmetic Act; and 
‘‘(2) changes in bacterial and fungal resistance 
to drugs. 
‘‘(b) PUBLIC AVAILABILITY
OF DATA.—The Sec-
retary shall make summaries of the data derived from 
monitoring under this section publicly available for the 
purposes of— 
‘‘(1) improving the monitoring of important 
trends in antibacterial and antifungal resistance; 
and 
‘‘(2) ensuring appropriate stewardship of anti-
bacterial and antifungal drugs, including those re-
ceiving approval or licensure for a limited population 
pursuant to section 505(z) of the Federal Food, 
Drug, and Cosmetic Act.’’. 
SEC. 2122. SUSCEPTIBILITY TEST INTERPRETIVE CRITERIA 
FOR MICROORGANISMS. 
(a) IN GENERAL.—Section 511 of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 360a) is amended to 
read as follows: 
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‘‘SEC. 511. IDENTIFYING AND UPDATING SUSCEPTIBILITY 
TEST INTERPRETIVE CRITERIA FOR MICRO-
ORGANISMS. 
‘‘(a) PURPOSE; IDENTIFICATION OF CRITERIA.— 
‘‘(1) PURPOSE.—The purpose of this section is 
to provide the Secretary with an expedited, flexible 
method for— 
‘‘(A) clearance or premarket approval of 
antimicrobial susceptibility testing devices uti-
lizing updated, recognized susceptibility test in-
terpretive criteria to characterize the in vitro 
susceptibility of particular bacteria, fungi, or 
other microorganisms to antimicrobial drugs; 
and 
‘‘(B) providing public notice of the avail-
ability of recognized interpretive criteria to 
meet premarket submission requirements or 
other requirements under this Act for anti-
microbial susceptibility testing devices. 
‘‘(2) IN GENERAL.—The Secretary shall iden-
tify appropriate susceptibility test interpretive cri-
teria with respect to antimicrobial drugs— 
‘‘(A) if such criteria are available on the 
date of approval of the drug under section 505 
of this Act or licensure of the drug under sec-
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130 
tion 351 of the Public Health Service Act (as 
applicable), upon such approval or licensure; or 
‘‘(B) if such criteria are unavailable on 
such date, on the date on which such criteria 
are available for such drug. 
‘‘(3) BASES
FOR
INITIAL
IDENTIFICATION.— 
The Secretary shall identify appropriate suscepti-
bility test interpretive criteria under paragraph (2), 
based on the Secretary’s review of, to the extent 
available and relevant— 
‘‘(A) preclinical and clinical data, including 
pharmacokinetic, pharmacodynamic, and epide-
miological data; 
‘‘(B) Bayesian and pharmacometric statis-
tical methodologies; and 
‘‘(C) such other evidence and information 
as the Secretary considers appropriate. 
‘‘(b) SUSCEPTIBILITY TEST INTERPRETIVE CRITERIA 
WEBSITE.— 
‘‘(1) IN GENERAL.—Not later than 1 year after 
the date of the enactment of the 21st Century Cures 
Act, the Secretary shall establish, and maintain 
thereafter, on the website of the Food and Drug Ad-
ministration, a dedicated website that contains a list 
of any appropriate new or updated susceptibility test 
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