HR 6 RFS 
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— 
(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.’’. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00039
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
(7) Section 487G of such Act (42 U.S.C. 288– 
6, as redesignated by subsection (a)(1)), is further 
amended— 
(A) in subsection (a)(1), by striking 
‘‘$35,000’’ and inserting ‘‘$50,000’’; and 
(B) in subsection (b), by adding at the end 
the following new sentence: ‘‘Subsection (b) of 
section 487H shall apply with respect to the 
maximum amount specified in subsection (a)(1) 
in the same manner as it applies to the max-
imum amount specified in such subsection (a) 
of such section.’’. 
SEC. 1042. REPORT. 
Not later than 18 months after the date of the enact-
ment of this Act, the Director of the National Institutes 
of Health shall submit to Congress a report on efforts of 
the National Institutes of Health to attract, retain, and 
develop emerging scientists, including underrepresented 
individuals in the sciences, such as women and other mi-
norities. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00040
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
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-
vestigator status on subsequent awards from the National 
Institutes of Health.’’. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00041
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
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 
(D) by striking ‘‘; or’’ at the end and in-
serting a period; and 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00042
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
(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 
the global pediatric clinical study network to encour-
age their participation; and 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00043
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
(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— 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00044
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
(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 conclusions resulting 
from the workshop under subsection (a) available to 
the public on the website of the National Institutes 
of Health; and 
(2) not less than biennially, disclose to the 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. 
Subtitle F—Advancement of the 
National Institutes of Health Re-
search and Data Access 
SEC. 1101. STANDARDIZATION OF DATA IN CLINICAL TRIAL 
REGISTRY DATA BANK ON ELIGIBILITY FOR 
CLINICAL TRIALS. 
(a) STANDARDIZATION.— 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00045
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
(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 includes at least— 
‘‘(i) the disease or indication being 
studied; 
‘‘(ii) inclusion criteria such as age, 
gender, diagnosis or diagnoses, laboratory 
values, or imaging results; and 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00046
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
‘‘(iii) exclusion criteria such as spe-
cific diagnosis or diagnoses, laboratory val-
ues, 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-
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-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00047
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
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 
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-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00048
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
ance with this section. The Secretary shall not enter into 
a cooperative agreement, contract, or grant under this sec-
tion with an eligible entity unless such entity submits an 
application including the following: 
(1) A certification that the eligible entity is not 
currently and does not plan to be involved in spon-
soring, operating, or participating in a clinical trial 
nor collaborating with another entity for the 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-
formation demonstrating that this system will com-
ply with applicable standards and requirements for 
ensuring the security of the clinical trial data. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00049
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
(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— 
(A) being a neutral third party in working 
with medical product manufacturers, academic 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00050
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
institutions, and the Food and Drug Adminis-
tration; and 
(B) having the ability to protect confiden-
tial data. 
(8) An agreement that the eligible entity will 
work with the Comptroller General of the United 
States for purposes of the study and report under 
subsection (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 Comptroller General of 
the United States shall conduct a study and issue a 
report to the Congress and the Secretary 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). 
(2) STUDY.—The study under paragraph (1) 
shall— 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00051
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
(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 to reduce adverse events in clinical 
trials. 
(D) An analysis of whether scientists have 
raised any concerns about the burden of having 
to share data with the system established under 
the program and, if so, a description of such 
concerns. 
(E) An analysis of privacy and data integ-
rity practices used in the program. 
(e) DEFINITIONS.—In this section: 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00052
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
(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 section 201(g) of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 331(g))), a de-
vice (as defined in section 201(h) of such Act (21 
U.S.C. 331(h)), a biological product (as defined in 
section 351 of the Public Health Service Act (42 
U.S.C. 262)), or any combination thereof. 
(3) The term ‘‘qualified clinical trial’’ means a 
clinical trial sponsored solely by an agency of the 
Department of Health and Human Services with re-
spect to a medical product— 
(A) that— 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00053
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
(i) was approved or cleared under sec-
tion 505, 510(k), or 515, or has an exemp-
tion for investigational use in effect under 
section 505 or 520(m), of the Federal 
Food, Drug, and Cosmetic Act (42 U.S.C. 
301 et seq.); or 
(ii) was licensed under section 351 of 
the Public Health Service Act (42 U.S.C. 
262) or has an exemption for investiga-
tional use in effect under such section 351; 
or 
(B) that is an investigational product for 
which the original development was 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. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00054
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
(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— 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00055
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
‘‘(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-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00056
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
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— 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00057
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
‘‘(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-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00058
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00059
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
‘‘(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 to 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, acting through 
the Commissioner of Food and Drugs, may, for the pur-
poses described in subsection (b)— 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00060
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
‘‘(1) participate in public-private partnerships 
engaged in one or more activities specified in sub-
section (c); and 
‘‘(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 that spon-
sor or maintain disease registries, including disease 
registries 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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00061
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
‘‘(C) is capable of handling increasing 
amounts of data as more studies are carried 
out; and 
‘‘(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 to facili-
tate 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-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00062
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
tial and subject to section 552(b)(4) of title 5, United 
States Code, or section 1905 of title 18, United States 
Code. 
‘‘(f) AUTHORIZATION OF APPROPRIATIONS.—There 
is authorized to be appropriated to carry out this section 
$5,000,000 for each of fiscal years 2016 through 2020.’’. 
SEC. 1124. ACCESSING, SHARING, AND USING HEALTH DATA 
FOR RESEARCH PURPOSES. 
(a) IN GENERAL.—(1) The HITECH Act (title XIII 
of division A of Public Law 111–5) is amended by adding 
at the end of subtitle D of such Act (42 U.S.C. 17921 
et seq.) the following: 
‘‘PART 4—ACCESSING, SHARING, AND USING 
HEALTH DATA FOR RESEARCH PURPOSES 
‘‘SEC. 13441. REFERENCES. 
‘‘In this part: 
‘‘(1) THE RULE.—References to ‘the Rule’ refer 
to part 160 or part 164, as appropriate, of title 45, 
Code of Federal Regulations (or any successor regu-
lation). 
‘‘(2) PART 164.—References to a specified sec-
tion of ‘part 164’, refer to such specified section of 
part 164 of title 45, Code of Federal Regulations (or 
any successor section). 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00063
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
‘‘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-
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 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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00064
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
‘‘(C) a business associate that has entered 
into a contract under section 164.504(e) of part 
164 for the purpose of data aggregation (as de-
fined in section 164.501 of part 164); and 
‘‘(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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00065
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
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 
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) does 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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00066
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
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-
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.’’. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00067
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
(2) The table of sections in section 13001(b) of such 
Act is amended by adding at the end of the items relating 
to subtitle D the following new items: 
‘‘PART 4—ACCESSING, SHARING, AND USING HEALTH DATA FOR RESEARCH 
PURPOSES 
‘‘Sec. 13441. References. 
‘‘Sec. 13442. Defining health data research as part of health care operations. 
‘‘Sec. 13443. Treating disclosures of protected health information for research 
similarly to disclosures of such information for public health 
purposes. 
‘‘Sec. 13444. Permitting remote access to protected health information by re-
searchers. 
‘‘Sec. 13445. Allowing one-time authorization of use and disclosure of protected 
health information for research purposes.’’. 
(b) REVISION OF REGULATIONS.—Not later than 12 
months after the date of the enactment of this Act, the 
Secretary of Health and Human Services shall revise and 
clarify the provisions of title 45, Code of Federal Regula-
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-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00068
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
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. 
‘‘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; 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00069
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
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. 
‘‘(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) eight shall be appointed by the 
Comptroller General of the United States 
from a list of nominations submitted by 
leading trade associations— 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00071
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
‘‘(I) four of whom shall be rep-
resentatives of the biopharmaceutical 
industry; 
‘‘(II) two of whom shall be rep-
resentatives of the medical device in-
dustry; and 
‘‘(III) two of whom shall be rep-
resentatives of the information and 
digital technology industry; and 
‘‘(ii) nine shall be appointed by the 
Comptroller General of the United States, 
after soliciting nominations— 
‘‘(I) two of whom shall be rep-
resentatives of academic researchers; 
‘‘(II) three of whom shall be rep-
resentatives of patients; 
‘‘(III) two of whom shall be rep-
resentatives of health care providers; 
and 
‘‘(IV) two 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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00072
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
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— 
‘‘(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-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00073
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
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 
‘‘(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— 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00074
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
‘‘(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. 
‘‘(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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00075
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
‘‘(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 
‘‘(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 1 year after the date 
on which the Council is established and each year there-
after, the Executive Director shall submit to the appro-
priate congressional committees a report on the perform-
ance of the Council. In preparing such report, the Council 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00076
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
shall consult with a nongovernmental consultant with ap-
propriate expertise. 
‘‘SEC. 281F. FUNDING. 
‘‘For the each of fiscal years 2016 through 2023, 
there is authorized to be appropriated $10,000,000 to the 
Council for purposes of carrying out the duties of the 
Council under this part.’’. 
TITLE II—DEVELOPMENT 
Subtitle A—Patient-Focused Drug 
Development 
SEC. 2001. DEVELOPMENT AND USE OF PATIENT EXPERI-
ENCE DATA TO ENHANCE STRUCTURED RISK- 
BENEFIT ASSESSMENT FRAMEWORK. 
(a) IN GENERAL.—Section 505 of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 355) is amended— 
(1) in subsection (d), by striking ‘‘The 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— 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00077
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
‘‘(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 
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 2 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; 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00078
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
‘‘(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- 
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— 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00079
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
(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— 
(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; 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00080
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
(III) the impact of the disease on 
daily life and long-term functioning; 
and 
(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 1 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00081
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
year after the date on which the comment period for 
the draft guidance closes. 
(3) WORKSHOPS.— 
(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. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00082
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
(4) PUBLIC MEETING.—Not later than 90 days 
after the date on which the draft guidance is pub-
lished under this subsection, the Secretary of Health 
and Human Services shall convene a public meeting 
to solicit input on the guidance. 
Subtitle B—Qualification and Use 
of Drug Development Tools 
SEC. 
2021. 
QUALIFICATION 
OF 
DRUG 
DEVELOPMENT 
TOOLS. 
(a) FINDINGS.—Congress finds the following: 
(1) Development of new drugs has become in-
creasingly challenging and resource intensive. 
(2) Development of drug development tools can 
benefit the availability of new medical therapies by 
helping to translate scientific discoveries into clinical 
applications. 
(3) Biomedical research consortia (as defined in 
section 507(f) of the Federal Food, Drug, and Cos-
metic Act, as added by subsection (c)) can play a 
valuable role in helping to develop and qualify drug 
development tools. 
(b) SENSE OF CONGRESS.—It is the sense of Con-
gress that— 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00083
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
(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-
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: 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00084
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
‘‘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-
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 accepts or declines to 
accept such letter of intent; 
‘‘(B)(i) if the Secretary accepts the letter 
of intent, a requestor submits a qualification 
plan to the Secretary; and 
‘‘(ii) the Secretary accepts or declines 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 determines whether to 
accept such qualification package for review; 
and 
‘‘(iii) if the Secretary accepts such quali-
fication package for review, the Secretary con-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00085
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
ducts such review in accordance with this sec-
tion. 
‘‘(2) ACCEPTANCE
AND
REVIEW
OF
SUBMIS-
SIONS.— 
‘‘(A) IN GENERAL.—The succeeding provi-
sions of this paragraph shall apply with respect 
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; NONACCEPT-
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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00086
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
under paragraph (1) with respect to a drug de-
velopment tool, based on factors determined ap-
propriate by the Secretary, including— 
‘‘(i) as applicable, the severity, rarity, 
or prevalence of the disease or condition 
targeted by the drug development tool and 
the availability or lack of alternative treat-
ments for such disease or condition; and 
‘‘(ii) the identification, by the Sec-
retary or by biomedical research consortia 
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). 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00087
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
‘‘(3) REVIEW OF FULL QUALIFICATION PACK-
AGE.—The Secretary shall— 
‘‘(A) conduct a comprehensive review of a 
full qualification package accepted under para-
graph (1)(C); and 
‘‘(B) determine whether the drug develop-
ment tool at issue is qualified for its proposed 
context of use. 
‘‘(4) QUALIFICATION.—The Secretary shall 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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00088
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
506(c)) under section 505 of this Act or section 
351 of the Public Health Service Act; or 
‘‘(B) supporting the investigational use of 
a drug or biological product under section 
505(i) of this Act or section 351(a)(3) of the 
Public Health Service Act. 
‘‘(3) RESCISSION OR MODIFICATION.— 
‘‘(A) IN GENERAL.—The Secretary may re-
scind or modify a determination under this sec-
tion to qualify a drug development tool if the 
Secretary determines that the drug development 
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, on re-
quest, be granted 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. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00089
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
‘‘(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 
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. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00090
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
‘‘(C) Any rescissions or modifications 
under subsection (b)(3) of a determination to 
qualify a drug development tool. 
‘‘(D) Summary reviews that document con-
clusions and recommendations for determina-
tions to qualify drug development tools under 
subsection (a). 
‘‘(E) A comprehensive list of— 
‘‘(i) all drug development tools quali-
fied under subsection (a); and 
‘‘(ii) all surrogate endpoints which 
were the basis of approval or licensure (as 
applicable) of a drug or biological product 
(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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00091
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
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-
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 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) 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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00092
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
processes, or biological responses to a therapeutic 
intervention; and 
‘‘(B) such term includes a surrogate endpoint. 
‘‘(2) BIOMEDICAL RESEARCH CONSORTIA.—The 
term ‘biomedical research consortia’ means collabo-
rative groups that may take the form of public-pri-
vate partnerships and may include government agen-
cies, institutions of higher education (as defined in 
section 101(a) of the Higher Education Act of 
1965), patient advocacy groups, industry representa-
tives, clinical and scientific experts, and other rel-
evant 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, 
the circumstances under which the drug development 
tool is to be used in drug development and regu-
latory review. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00093
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
‘‘(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. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00094
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
‘‘(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). 
‘‘(f) 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.’’. 
(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— 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00095
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
(A) provides a conceptual framework de-
scribing appropriate standards and scientific 
approaches to support the development of bio-
markers delineated under the taxonomy estab-
lished under paragraph (3); 
(B) makes recommendations for dem-
onstrating that a surrogate endpoint is reason-
ably likely to predict clinical benefit for the pur-
pose of supporting the accelerated approval of 
a drug under section 506(c) of the Federal 
Food, Drug, and Cosmetic Act (21 U.S.C. 
356(c)); 
(C) with respect to the qualification 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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00096
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
other individuals and entities with expert 
knowledge and insights that may assist the 
Secretary in the review of qualification 
plans and full qualification submissions 
under such section; and 
(D) includes such other information as the 
Secretary determines appropriate. 
(2) TIMING.—Not later than 24 months after 
the date of the enactment of this Act, the Secretary 
of Health and Human Services shall issue draft 
guidance under paragraph (1) on the implementa-
tion of section 507 of the Federal Food, Drug, and 
Cosmetic Act (as added by subsection (c)). The Sec-
retary shall issue final guidance on the implementa-
tion of such section not later than 6 months after 
the date on which the comment period for the draft 
guidance closes. 
(3) TAXONOMY.— 
(A) IN
GENERAL.—For purposes of in-
forming guidance under this subsection, the 
Secretary of Health and Human Services shall, 
in consultation with biomedical research con-
sortia and other interested parties through a 
collaborative public process, establish a tax-
onomy for the classification of biomarkers (and 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00097
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
related scientific concepts) for use in drug de-
velopment. 
(B) PUBLIC
AVAILABILITY.—Not later 
than 12 months after the date of the enactment 
of this Act, the Secretary of Health and Human 
Services shall make such taxonomy publicly 
available in draft form for public comment. The 
Secretary shall finalize the taxonomy not later 
than 12 months after the close of the public 
comment 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, 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00098
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
HR 6 RFS 
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); 
(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; 
(D) the number of qualification plans and 
full qualification packages, respectively, sub-
mitted to the Secretary; and 
(E) the drug development tools qualified 
through such qualification process, specified by 
type of tool, such as a biomarker or clinical out-
come assessment (as such terms are defined in 
subsection (f) of such section 507). 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00099
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
100 
HR 6 RFS 
SEC. 2022. ACCELERATED APPROVAL DEVELOPMENT PLAN. 
(a) IN GENERAL.—Section 506 of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 356) is amended by 
adding the following subsection: 
‘‘(g) 
ACCELERATED
APPROVAL
DEVELOPMENT 
PLAN.— 
‘‘(1) IN GENERAL.—In the case of a drug that 
the Secretary determines may be eligible for acceler-
ated approval in accordance with subsection (c), the 
sponsor of such drug may request, at any time after 
the submission of an application for the investigation 
of the drug under section 505(i) of this Act or 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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00100
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
101 
HR 6 RFS 
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— 
‘‘(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— 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00101
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
102 
HR 6 RFS 
‘‘(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).’’. 
(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-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00102
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
103 
HR 6 RFS 
velopment of a precision drug or biological product. Such 
guidance shall— 
‘‘(1) define the term ‘precision drug or biologi-
cal product’; and 
‘‘(2) address the topics described in subsection 
(b). 
‘‘(b) CERTAIN ISSUES.—The topics to be addressed 
by guidance under subsection (a) are— 
‘‘(1) the evidence needed to support the use of 
biomarkers (as defined in section 507(e)) that 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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00103
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
104 
HR 6 RFS 
biomarker may be included in the approved prescrip-
tion labeling for a precision drug or biological prod-
uct. 
‘‘(c) DATE CERTAIN FOR INITIAL GUIDANCE.—The 
Secretary shall issue guidance under subsection (a) not 
later than 18 months after the date of the enactment of 
the 21st Century Cures Act. 
‘‘SEC. 592. PRECISION MEDICINE REGARDING ORPHAN- 
DRUG 
AND 
EXPEDITED-APPROVAL 
PRO-
GRAMS. 
‘‘(a) IN GENERAL.—In the case of a precision drug 
or biological product that is the subject of an application 
submitted under section 505(b)(1), or section 351(a) of 
the 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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00104
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
105 
HR 6 RFS 
under section 351(a) of the Public Health Service 
Act, as applicable— 
‘‘(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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00105
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
106 
HR 6 RFS 
section, with respect to a sponsor’s ability to ref-
erence information contained in another application 
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— 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00106
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
107 
HR 6 RFS 
(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 to 
satisfy 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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00107
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
108 
HR 6 RFS 
(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 is amended by inserting after section 505E of such 
Act (21 U.S.C. 355f) the following: 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00108
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
109 
HR 6 RFS 
‘‘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 to support the approval of a new 
indication for a drug approved under section 505(b); 
and 
‘‘(2) to help to support or satisfy postapproval 
study requirements. 
‘‘(b) EVIDENCE FROM CLINICAL EXPERIENCE DE-
FINED.—In this section, the term ‘evidence from clinical 
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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00109
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
110 
HR 6 RFS 
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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00110
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
111 
HR 6 RFS 
‘‘(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 under sub-
section (a), 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; 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00111
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
112 
HR 6 RFS 
‘‘(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 nonspecified 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. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00112
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
113 
HR 6 RFS 
‘‘SEC. 505G. COLLECTING EVIDENCE FROM CLINICAL EXPE-
RIENCE THROUGH TARGETED EXTENSIONS 
OF THE SENTINEL SYSTEM. 
‘‘(a) IN GENERAL.—The Secretary shall, in parallel 
to implementing the program established under section 
505F and in order to build capacity for utilizing the evi-
dence from clinical experience described in that section, 
identify and execute pilot demonstrations to extend exist-
ing use of the Sentinel System surveillance infrastructure 
authorized under section 505(k). 
‘‘(b) PILOT DEMONSTRATIONS.— 
‘‘(1) IN GENERAL.—The Secretary— 
‘‘(A) shall design and implement pilot dem-
onstrations to utilize data captured through the 
Sentinel System surveillance infrastructure au-
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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00113
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
114 
HR 6 RFS 
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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00114
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
115 
HR 6 RFS 
‘‘(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) HIPAA PRIVACY RULE; HUMAN SUBJECT 
RESEARCH REGULATION.—The Secretary may deem 
such pilot demonstrations— 
‘‘(A) public health activities, for purposes 
of which a use or disclosure of protected health 
information would be permitted as described in 
section 164.512(b)(1) of title 45, Code of Fed-
eral Regulations (or any successor regulation); 
and 
‘‘(B) outside the scope of ‘research’ as de-
fined in section 46.102(d) of title 45, Code of 
Federal Regulations (or any successor regula-
tion). 
‘‘(c) AUTHORIZATION OF APPROPRIATIONS.—There 
are authorized to be appropriated to carry out this section 
$3,000,000 for each of fiscal years 2016 through 2020.’’. 
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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00115
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
116 
HR 6 RFS 
further amended by inserting after section 505G of such 
Act the following: 
‘‘SEC. 505H. STREAMLINED DATA REVIEW PROGRAM. 
‘‘(a) IN GENERAL.—The Secretary shall establish a 
streamlined data review program under which a holder of 
an 
approved 
application 
submitted 
under 
section 
505(b)(1) or under section 351(a) of the Public Health 
Service Act may, to support the approval or licensure (as 
applicable) of the use of the drug that is the subject of 
such approved application for a new qualified indication, 
submit qualified data summaries. 
‘‘(b) ELIGIBILITY.—In carrying out the streamlined 
data review program under subsection (a), the Secretary 
may authorize the holder of the approved application to 
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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00116
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
117 
HR 6 RFS 
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 
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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00117
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
118 
HR 6 RFS 
‘‘(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 
‘‘(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). 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00118
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
119 
HR 6 RFS 
(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 
(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-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00119
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
120 
HR 6 RFS 
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)). 
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 or distributor’s investigational drugs. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00120
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
121 
HR 6 RFS 
‘‘(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 
‘‘(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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00121
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
122 
HR 6 RFS 
‘‘(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-
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)). 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00122
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
123 
HR 6 RFS 
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 
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-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00123
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
124 
HR 6 RFS 
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: 
‘‘(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 
analysis may be comparative to the use of another drug, 
to another health care intervention, or to no intervention. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00124
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
125 
HR 6 RFS 
‘‘(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 nonmis-
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-
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 
to expedite the development and availability of treatments 
for serious or life-threatening bacterial or fungal infections 
in patients with unmet needs, while maintaining safety 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00125
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
126 
HR 6 RFS 
and effectiveness standards for such treatments, taking 
into account the severity of the infection and the avail-
ability 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— 
‘‘(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 in accordance with this 
subsection only if a written agreement is 
reached under subparagraph (A); 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00126
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
127 
HR 6 RFS 
‘‘(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 
‘‘(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; 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00127
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
128 
HR 6 RFS 
‘‘(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 to expedite 
and facilitate the development and review of a 
drug for which a sponsor intends to request ap-
proval in accordance with this subsection, the 
Secretary may, at the request of the sponsor, 
conduct meetings that provide early consulta-
tion, timely advice, and sufficient opportunities 
to 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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00128
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
129 
HR 6 RFS 
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-
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-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00129
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
130 
HR 6 RFS 
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 
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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00130
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
131 
HR 6 RFS 
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). 
‘‘(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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00131
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
132 
HR 6 RFS 
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 
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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00132
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
133 
HR 6 RFS 
‘‘(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 
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-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00133
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
134 
HR 6 RFS 
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.— 
(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: 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00134
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
135 
HR 6 RFS 
‘‘(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: 
‘‘(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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00135
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
136 
HR 6 RFS 
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 
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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00136
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
137 
HR 6 RFS 
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— 
‘‘(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 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00137
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
138 
HR 6 RFS 
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: 
‘‘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-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00138
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
139 
HR 6 RFS 
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-
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— 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00139
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
140 
HR 6 RFS 
‘‘(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 
interpretive criteria standards in accordance with 
paragraph (2) (referred to in this section as the ‘In-
terpretive Criteria Website’). 
‘‘(2) LISTING OF SUSCEPTIBILITY TEST INTER-
PRETIVE CRITERIA STANDARDS.— 
‘‘(A) IN GENERAL.—The list described in 
paragraph (1) shall consist of any new or up-
dated susceptibility test interpretive criteria 
standards that are— 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00140
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
141 
HR 6 RFS 
‘‘(i) established by a nationally or 
internationally recognized standard devel-
opment organization that— 
‘‘(I) establishes and maintains 
procedures to address potential con-
flicts of interest and ensure trans-
parent decisionmaking; 
‘‘(II) holds open meetings to en-
sure that there is an opportunity for 
public input by interested parties, and 
establishes and maintains processes to 
ensure that such input is considered 
in decisionmaking; and 
‘‘(III) permits its standards to be 
made publicly available, through the 
National Library of Medicine or an-
other similar source acceptable to the 
Secretary; and 
‘‘(ii) recognized in whole, or in part, 
by the Secretary under subsection (c). 
‘‘(B) OTHER LIST.—The Interpretive Cri-
teria Website shall, in addition to the list de-
scribed in subparagraph (A), include a list of 
interpretive criteria, if any, that the Secretary 
has determined to be appropriate with respect 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00141
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
142 
HR 6 RFS 
to 
legally 
marketed 
antimicrobial 
drugs, 
where— 
‘‘(i) the Secretary does not recognize, 
in whole or in part, an interpretive criteria 
standard described under subparagraph 
(A) otherwise applicable to such a drug; 
‘‘(ii) the Secretary withdraws under 
subsection (c)(1)(B) recognition of a 
standard, in whole or in part, otherwise 
applicable to such a drug; 
‘‘(iii) the Secretary approves an appli-
cation under section 505 of this Act or sec-
tion 351 of the Public Health Service Act, 
as applicable, with respect to marketing of 
such a drug for which there are no rel-
evant interpretive criteria included in a 
standard recognized by the Secretary 
under subsection (c); or 
‘‘(iv) because the characteristics of 
such a drug differ from other drugs with 
the same active ingredient, the interpretive 
criteria with respect to such drug— 
‘‘(I) differ from otherwise appli-
cable interpretive criteria included in 
a standard listed under subparagraph 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00142
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
143 
HR 6 RFS 
(A) or interpretive criteria otherwise 
listed under this subparagraph; and 
‘‘(II) are determined by the Sec-
retary to be appropriate for the drug. 
‘‘(C) REQUIRED STATEMENTS OF LIMITA-
TIONS OF INFORMATION.—The Interpretive Cri-
teria Website shall include the following: 
‘‘(i) A statement that— 
‘‘(I) the website provides infor-
mation about the susceptibility of bac-
teria, fungi, or other microorganisms 
to a certain drug (or drugs); and 
‘‘(II) the safety and efficacy of 
the drug in treating clinical infections 
due to such bacteria, fungi, or other 
microorganisms may not have been es-
tablished in adequate and well-con-
trolled clinical trials and the clinical 
significance of such susceptibility in-
formation in such trials is unknown. 
‘‘(ii) A statement that directs health 
care practitioners to consult the approved 
product labeling for specific drugs to deter-
mine the uses for which the Food and 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00143
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
144 
HR 6 RFS 
Drug Administration has approved the 
product. 
‘‘(iii) Any other statement that the 
Secretary determines appropriate to ade-
quately convey the limitations of the data 
supporting susceptibility test interpretive 
criteria standard listed on the website. 
‘‘(3) NOTICE.—Not later than the date on 
which the Interpretive Criteria Website is estab-
lished, the Secretary shall publish a notice of that 
establishment in the Federal Register. 
‘‘(4) INAPPLICABILITY OF MISBRANDING PROVI-
SION.—The inclusion in the approved labeling of an 
antimicrobial drug of a reference or hyperlink to the 
Interpretive Criteria Website, in and of itself, shall 
not cause the drug to be misbranded in violation of 
section 502, or the regulations promulgated there-
under. 
‘‘(5) TRADE SECRETS AND CONFIDENTIAL IN-
FORMATION.—Nothing in this section shall be con-
strued as authorizing the Secretary to disclose any 
information that is a trade secret or confidential in-
formation subject to section 552(b)(4) of title 5, 
United States Code. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00144
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
145 
HR 6 RFS 
‘‘(c) RECOGNITION OF SUSCEPTIBILITY TEST INTER-
PRETIVE CRITERIA FROM STANDARD DEVELOPMENT OR-
GANIZATIONS.— 
‘‘(1) IN GENERAL.—Beginning on the date of 
the establishment of the Interpretive Criteria 
Website, and at least every 6 months thereafter, the 
Secretary shall— 
‘‘(A) evaluate any appropriate new or up-
dated susceptibility test interpretive criteria 
standards established by a nationally or inter-
nationally recognized standard development or-
ganization described in subsection (b)(2)(A)(i); 
and 
‘‘(B) publish on the public website of the 
Food and Drug Administration a notice— 
‘‘(i) withdrawing recognition of any 
different susceptibility test interpretive cri-
teria standard, in whole or in part; 
‘‘(ii) recognizing the new or updated 
standards; 
‘‘(iii) recognizing one or more parts of 
the new or updated interpretive criteria 
specified in such a standard and declining 
to recognize the remainder of such stand-
ard; and 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00145
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
146 
HR 6 RFS 
‘‘(iv) making any necessary updates to 
the lists under subsection (b)(2). 
‘‘(2) BASES FOR UPDATING INTERPRETIVE CRI-
TERIA STANDARDS.—In evaluating new or updated 
susceptibility test interpretive criteria standards 
under paragraph (1)(A), the Secretary may con-
sider— 
‘‘(A) the Secretary’s determination that 
such a standard is not applicable to a particular 
drug because the characteristics of the drug dif-
fer from other drugs with the same active in-
gredient; 
‘‘(B) information provided by interested 
third parties, including public comment on the 
annual compilation of notices published under 
paragraph (3); 
‘‘(C) any bases used to identify suscepti-
bility test interpretive criteria under subsection 
(a)(2); and 
‘‘(D) such other information or factors as 
the Secretary determines appropriate. 
‘‘(3) ANNUAL
COMPILATION
OF
NOTICES.— 
Each year, the Secretary shall compile the notices 
published under paragraph (1)(B) and publish such 
compilation in the Federal Register and provide for 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00146
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
147 
HR 6 RFS 
public comment. If the Secretary receives comments, 
the Secretary shall review such comments and, if the 
Secretary determines appropriate, update pursuant 
to this subsection susceptibility test interpretive cri-
teria standards— 
‘‘(A) recognized by the Secretary under 
this subsection; or 
‘‘(B) otherwise listed on the Interpretive 
Criteria Website under subsection (b)(2). 
‘‘(4) RELATION TO SECTION 514(c).—Any sus-
ceptibility test interpretive standard recognized 
under this subsection or any criteria otherwise listed 
under subsection (b)(2)(B) shall be deemed to be 
recognized as a standard by the Secretary under sec-
tion 514(c)(1). 
‘‘(5) VOLUNTARY USE OF INTERPRETIVE CRI-
TERIA.—Nothing in this section prohibits a person 
from seeking approval or clearance of a drug or de-
vice, or changes to the drug or the device, on the 
basis of susceptibility test interpretive criteria stand-
ards which differ from those recognized pursuant to 
paragraph (1). 
‘‘(d) ANTIMICROBIAL DRUG LABELING.— 
‘‘(1) DRUGS MARKETED PRIOR TO ESTABLISH-
MENT OF INTERPRETIVE CRITERIA WEBSITE.—With 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00147
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
148 
HR 6 RFS 
respect to an antimicrobial drug lawfully introduced 
or delivered for introduction into interstate com-
merce for commercial distribution before the estab-
lishment of the Interpretive Criteria Website, a hold-
er of an approved application under section 505 of 
this Act or section 351 of the Public Health Service 
Act, as applicable, for each such drug— 
‘‘(A) not later than 1 year after establish-
ment of the Interpretive Criteria Website, shall 
submit to the Secretary a supplemental applica-
tion for purposes of changing the drug’s label-
ing to substitute a reference or hyperlink to 
such Website for any susceptibility test inter-
pretive criteria and related information; and 
‘‘(B) may begin distribution of the drug in-
volved upon receipt by the Secretary of the sup-
plemental application for such change. 
‘‘(2) DRUGS MARKETED SUBSEQUENT TO ES-
TABLISHMENT
OF
INTERPRETIVE
CRITERIA 
WEBSITE.—With respect to antimicrobial drugs law-
fully introduced or delivered for introduction into 
interstate commerce for commercial distribution on 
or after the date of the establishment of the Inter-
pretive Criteria Website, the labeling for such a drug 
shall include, in lieu of susceptibility test interpretive 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00148
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
149 
HR 6 RFS 
criteria and related information, a reference to such 
Website. 
‘‘(e) SPECIAL CONDITION FOR MARKETING OF ANTI-
MICROBIAL SUSCEPTIBILITY TESTING DEVICES.— 
‘‘(1) IN
GENERAL.—Notwithstanding sections 
501, 502, 510, 513, and 515, if the conditions speci-
fied in paragraph (2) are met (in addition to other 
applicable provisions under this chapter) with re-
spect to an antimicrobial susceptibility testing device 
described in subsection (f)(1), the Secretary may au-
thorize the marketing of such device for a use de-
scribed in such subsection. 
‘‘(2) 
CONDITIONS
APPLICABLE
TO
ANTI-
MICROBIAL
SUSCEPTIBILITY
TESTING
DEVICES.— 
The conditions specified in this paragraph are the 
following: 
‘‘(A) The device is used to make a deter-
mination of susceptibility using susceptibility 
test interpretive criteria that are— 
‘‘(i) included in a standard recognized 
by the Secretary under subsection (c); or 
‘‘(ii) otherwise listed on the Interpre-
tive Criteria Website under subsection 
(b)(2). 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00149
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
150 
HR 6 RFS 
‘‘(B) The labeling of such device promi-
nently and conspicuously— 
‘‘(i) includes a statement that— 
‘‘(I) the device provides informa-
tion about the susceptibility of bac-
teria and fungi to certain drugs; and 
‘‘(II) the safety and efficacy of 
such drugs in treating clinical infec-
tions due to such bacteria or fungi 
may not have been established in ade-
quate and well-controlled clinical trials 
and the clinical significance of such 
susceptibility information in those in-
stances is unknown; 
‘‘(ii) includes a statement directing 
health care practitioners to consult the ap-
proved labeling for drugs tested using such 
a device, to determine the uses for which 
the Food and Drug Administration has ap-
proved such drugs; and 
‘‘(iii) includes any other statement the 
Secretary determines appropriate to ade-
quately convey the limitations of the data 
supporting the interpretive criteria de-
scribed in subparagraph (A). 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00150
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
151 
HR 6 RFS 
‘‘(f) DEFINITIONS.—In this section: 
‘‘(1) The term ‘antimicrobial susceptibility test-
ing device’ means a device that utilizes susceptibility 
test interpretive criteria to determine and report the 
in vitro susceptibility of certain microorganisms to a 
drug (or drugs). 
‘‘(2) The term ‘qualified infectious disease 
product’ means a qualified infectious disease product 
designated under section 505E(d). 
‘‘(3) The term ‘susceptibility test interpretive 
criteria’ means— 
‘‘(A) one or more specific numerical values 
which characterize the susceptibility of bacteria 
or other microorganisms to the drug tested; and 
‘‘(B) related categorizations of such sus-
ceptibility, including categorization of the drug 
as susceptible, intermediate, resistant, or such 
other term as the Secretary determines appro-
priate. 
‘‘(4)(A) The term ‘antimicrobial drug’ means, 
subject to subparagraph (B), a systemic anti-
bacterial or antifungal drug that— 
‘‘(i) is intended for human use in the treat-
ment of a disease or condition caused by a bac-
terium or fungus; 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00151
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
152 
HR 6 RFS 
‘‘(ii) may include a qualified infectious dis-
ease product designated under section 505E(d); 
and 
‘‘(iii) is subject to section 503(b)(1). 
‘‘(B) If provided by the Secretary through regu-
lations, such term may include— 
‘‘(i) drugs other than systemic anti-
bacterial and antifungal drugs; and 
‘‘(ii) biological products (as such term is 
defined in section 351 of the Public Health 
Service Act) to the extent such products exhibit 
antimicrobial activity. 
‘‘(g) RULE OF CONSTRUCTION.—Nothing in this sec-
tion shall be construed— 
‘‘(1) to alter the standards of evidence— 
‘‘(A) under subsection (c) or (d) of section 
505, including the substantial evidence stand-
ard in section 505(d), or under section 351 of 
the Public Health Service Act (as applicable); 
or 
‘‘(B) with respect to marketing authoriza-
tion for devices, under section 510, 513, or 515; 
‘‘(2) to apply with respect to any drug, device, 
or biological product, in any context other than— 
‘‘(A) an antimicrobial drug; or 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00152
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
153 
HR 6 RFS 
‘‘(B) an antimicrobial susceptibility testing 
device that uses susceptibility test interpretive 
criteria to characterize and report the in vitro 
susceptibility of certain bacteria, fungi, or other 
microorganisms to antimicrobial drugs in ac-
cordance with this section; or 
‘‘(3) unless specifically stated, to have any ef-
fect on authorities provided under other sections of 
this Act, including any regulations issued under such 
sections.’’. 
(b) CONFORMING AMENDMENTS.— 
(1) REPEAL OF RELATED AUTHORITY.—Section 
1111 of the Food and Drug Administration Amend-
ments Act of 2007 (42 U.S.C. 247d–5a; relating to 
identification of clinically susceptible concentrations 
of antimicrobials) is repealed. 
(2) CLERICAL AMENDMENT.—The table of con-
tents in section 2 of the Food and Drug Administra-
tion Amendments Act of 2007 is amended by strik-
ing the item relating to section 1111. 
(3) MISBRANDING.—Section 502 of the Federal 
Food, Drug, and Cosmetic Act (21 U.S.C. 352), as 
amended by section 2121, is further amended by 
adding at the end the following: 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00153
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
154 
HR 6 RFS 
‘‘(ee) If it is an antimicrobial drug and its labeling 
fails to conform with the requirements under section 
511(d).’’. 
(4) RECOGNITION OF INTERPRETIVE CRITERIA 
AS DEVICE STANDARD.—Section 514(c)(1)(A) of the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
360d(c)(1)(A)) is amended by inserting after ‘‘the 
Secretary shall, by publication in the Federal Reg-
ister’’ the following: ‘‘(or, with respect to suscepti-
bility test interpretive criteria or standards recog-
nized or otherwise listed under section 511, by post-
ing on the Interpretive Criteria Website in accord-
ance with such section)’’. 
(c) REPORT TO CONGRESS.—Not later than 2 years 
after the date of enactment of this Act, the Secretary of 
Health and Human Services shall submit to the Com-
mittee on Energy and Commerce of the House of Rep-
resentatives and the Committee on Health, Education, 
Labor, and Pensions of the Senate a report on the 
progress made in implementing section 511 of the Federal 
Food, Drug, and Cosmetic Act (21 U.S.C. 360a), as 
amended by this section. 
(d) REQUESTS FOR UPDATES TO INTERPRETIVE CRI-
TERIA WEBSITE.—Chapter 35 of title 44, United States 
Code, shall not apply to the collection of information from 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00154
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
155 
HR 6 RFS 
interested parties regarding the updating of lists under 
paragraph (2) of subsection (b) section 511 of the Federal 
Food, Drug, and Cosmetic Act (as amended by subsection 
(a)) and posted on the Interpretive Criteria Website estab-
lished under paragraph (1) of such subsection (b). 
(e) NO EFFECT
ON HEALTH CARE PRACTICE.— 
Nothing in this subtitle (including the amendments made 
by this subtitle) shall be construed to restrict, in any man-
ner, the prescribing or administering of antibiotics or 
other products by health care practitioners, or to limit the 
practice of health care. 
SEC. 2123. ENCOURAGING THE DEVELOPMENT AND USE OF 
DISARM DRUGS. 
(a) ADDITIONAL PAYMENT
FOR DISARM DRUGS 
UNDER MEDICARE.— 
(1) IN GENERAL.—Section 1886(d)(5) of the 
Social Security Act (42 U.S.C. 1395ww(d)(5)) is 
amended by adding at the end the following new 
subparagraph: 
‘‘(M)(i) As part of the annual rulemaking conducted 
with respect to payment for subsection (d) hospitals for 
each fiscal year beginning with fiscal year 2018, the Sec-
retary shall— 
‘‘(I) include a list of the DISARM drugs for 
such fiscal year; and 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00155
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
156 
HR 6 RFS 
‘‘(II) with respect to discharges by eligible hos-
pitals that involve a drug so listed, provide for an 
additional payment to be made under this subsection 
in accordance with the provisions of this subpara-
graph. 
‘‘(ii) Additional payments may not be made for a 
drug under this subparagraph— 
‘‘(I) other than during the 5-fiscal-year period 
beginning with the fiscal year for which the drug is 
first included in the list described in clause (i)(I); 
and 
‘‘(II) with respect to which payment has ever 
been made pursuant to subparagraph (K). 
‘‘(iii) For purposes of this subparagraph, the term 
‘DISARM drug’ means a product that is approved for use, 
or a product for which an indication is first approved for 
use, by the Food and Drug Administration on or after 
December 1, 2014, and that the Food and Drug Adminis-
tration determines is an antimicrobial product (as defined 
in clause (iv)) and is intended to treat an infection— 
‘‘(I) for which there is an unmet medical need; 
and 
‘‘(II) which is associated with high rates of 
mortality or significant patient morbidity, as deter-
mined in consultation with the Director of the Cen-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00156
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
157 
HR 6 RFS 
ters for Disease Control and Prevention and the in-
fectious disease professional community. 
‘‘(iv) For purposes of clause (iii), the term ‘anti-
microbial product’ means a product that either— 
‘‘(I) is intended to treat an infection caused by, 
or likely to be caused by, a qualifying pathogen (as 
defined under section 505E(f) of the Federal Food, 
Drug, and Cosmetic Act); or 
‘‘(II) meets the definition of a qualified infec-
tious disease product under section 505E(g) of the 
Federal Food, Drug, and Cosmetic Act. 
Such determination may be revoked only upon 
a finding that the request for such determina-
tion contained an untrue statement of material 
fact. 
‘‘(v) For purposes of this subparagraph, the term ‘eli-
gible hospital’ means a subsection (d) hospital that partici-
pates in the National Healthcare Safety Network of the 
Centers for Disease Control and Prevention (or, to the ex-
tent a similar surveillance system that includes reporting 
about antimicrobial drugs is determined by the Secretary 
to be available to such hospitals, such similar surveillance 
system as the Secretary may specify). 
‘‘(vi) Subject to the succeeding provisions of this sub-
paragraph, the additional payment under this subpara-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00157
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
158 
HR 6 RFS 
graph, with respect to a drug, shall be in the amount pro-
vided for such drug under section 1847A. 
‘‘(vii) As part of the rulemaking referred to in clause 
(i) for each fiscal year, the Secretary shall estimate— 
‘‘(I) total add-on payments (as defined in sub-
clause (I) of clause (ix)); and 
‘‘(II) total hospital payments (as defined in 
subclause (II) of such clause). 
‘‘(viii) If the total add-on payments estimated pursu-
ant to clause (vii)(I) for a fiscal year exceed 0.02 percent 
of the total hospital payments estimated pursuant to 
clause (vii)(II) for such fiscal year, the Secretary shall re-
duce in a pro rata manner the amount of each additional 
payment under this subsection pursuant to this subpara-
graph for such fiscal year in order to ensure that the total 
add-on payments estimated for such fiscal year do not ex-
ceed 0.02 percent of the total hospital payments estimated 
for such fiscal year. 
‘‘(ix) In this subparagraph: 
‘‘(I) The term ‘total add-on payments’ means, 
with respect to a fiscal year, the total amount of the 
additional payments under this subsection pursuant 
to this subparagraph for discharges in such fiscal 
year without regard to the application of clause 
(viii). 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00158
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
159 
HR 6 RFS 
‘‘(II) The term ‘total hospital payments’ means, 
with respect to a fiscal year, the total amount of 
payments made under this subsection for all dis-
charges in such fiscal year.’’. 
(2) CONFORMING AMENDMENTS.— 
(A) NO DUPLICATIVE NTAP PAYMENTS.— 
Section 1886(d)(5)(K)(vi) of the Social Security 
Act (42 U.S.C. 1395ww(d)(5)(K)(vi)) is amend-
ed by inserting ‘‘and if additional payment has 
never been made under this subsection pursu-
ant to subparagraph (M) with respect to the 
service or technology’’ before the period at the 
end. 
(B) ACCESS
TO
PRICE
INFORMATION.— 
Section 1927(b)(3)(A) of the Social Security 
Act (42 U.S.C. 1396r–8(b)(3)(A)) is amend-
ed— 
(i) in clause (ii)— 
(I) by striking ‘‘for each’’ and in-
serting ‘‘, for each’’; and 
(II) by striking ‘‘and’’ at the end; 
(ii) in clause (iii)— 
(I) in subclause (II), by inserting 
‘‘or under section 1886(d) pursuant to 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00159
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
160 
HR 6 RFS 
paragraph (5)(M) of such section,’’ 
after ‘‘1847A,’’; 
(II) in the matter following sub-
clause 
(III), 
by 
striking 
‘‘or 
1881(b)(13)(A)(ii)’’ and inserting ‘‘, 
section 1881(b)(13)(A)(ii), or section 
1886(d)(5)(M)’’; and 
(III) by striking the period at the 
end and inserting ‘‘; and’’; and 
(iii) in clause (iv), by striking the 
semicolon at the end and inserting a pe-
riod. 
(b) STUDY AND REPORT ON REMOVING BARRIERS TO 
DEVELOPMENT OF DISARM DRUGS.— 
(1) STUDY.—The Comptroller General of the 
United States shall, in consultation with the Direc-
tor of the National Institutes of Health, the Com-
missioner of Food and Drugs, and the Director of 
the Centers for Disease Control and Prevention, con-
duct a study to— 
(A) identify and examine the barriers that 
prevent the development of DISARM drugs, as 
defined in section 1886(d)(5)(M)(iii) of the So-
cial 
Security 
Act 
(42 
U.S.C. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00160
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
161 
HR 6 RFS 
1395ww(d)(5)(M)(iii)), as added by subsection 
(a)(1); and 
(B) develop recommendations for actions 
to be taken in order to overcome any barriers 
identified under subparagraph (A). 
(2) REPORT.—Not later than 1 year after the 
date of the enactment of this Act, the Comptroller 
General shall submit to Congress a report on the 
study conducted under paragraph (1). 
(c) STUDY AND REPORT ON THE IMPACT OF ADDI-
TIONAL MEDICARE PAYMENT FOR DISARM DRUGS ON 
USAGE
PRACTICES
AND
DEVELOPMENT
OF
RESIST-
ANCE.— 
(1) STUDY.—The Director of the Centers for 
Disease Control and Prevention shall conduct a 
study to examine the effects of the additional pay-
ment for DISARM drugs under the Medicare Pro-
gram provided under subparagraph (M) of section 
1886(d)(5) of the Social Security Act (42 U.S.C. 
1395ww(d)(5)), as added by subsection (a), on— 
(A) the usage of DISARM drugs (as de-
fined by clause (iii) of such subparagraph) by 
subsection (d) hospitals (as defined in section 
1886(d)(1)(B) of such Act); and 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00161
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
162 
HR 6 RFS 
(B) the development of resistance by indi-
viduals to such DISARM drugs. 
(2) REPORT.—Not later than 3 years after the 
date of the enactment of this Act, such Director 
shall submit to Congress a report on the study con-
ducted under paragraph (1). 
Subtitle H—Vaccine Access, 
Certainty, and Innovation 
SEC. 2141. TIMELY REVIEW OF VACCINES BY THE ADVISORY 
COMMITTEE ON IMMUNIZATION PRACTICES. 
Section 2102(a) of the Public Health Service Act (42 
U.S.C. 300aa–2(a)) is amended by adding at the end the 
following: 
‘‘(10) ADVISORY COMMITTEE ON IMMUNIZATION 
PRACTICES.— 
‘‘(A) STANDARD
PERIODS
OF
TIME
FOR 
MAKING RECOMMENDATIONS.—Upon the licen-
sure of any vaccine or any new indication for a 
vaccine, the Director of the Program shall di-
rect the Advisory Committee on Immunization 
Practices, at its next regularly scheduled meet-
ing, to consider the use of the vaccine. 
‘‘(B) EXPEDITED REVIEW PURSUANT TO 
REQUEST BY SPONSOR OR MANUFACTURER.—If 
the Advisory Committee does not make rec-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00162
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
163 
HR 6 RFS 
ommendations with respect to the use of a vac-
cine at the Advisory Committee’s first regularly 
scheduled meeting after the licensure of the 
vaccine or any new indication for the vaccine, 
the Advisory Committee, at the request of the 
sponsor of the vaccine, shall make such rec-
ommendations on an expedited basis. 
‘‘(C) EXPEDITED
REVIEW
FOR
BREAK-
THROUGH
THERAPIES
AND
FOR
USE
DURING 
PUBLIC HEALTH EMERGENCIES.—If a vaccine 
is designated as a breakthrough therapy under 
section 506 of the Federal Food, Drug, and 
Cosmetic Act, and is licensed under section 351 
of this Act, the Advisory Committee shall make 
recommendations with respect to the use of the 
vaccine on an expedited basis. 
‘‘(D) DEFINITION.—In this paragraph, the 
terms ‘Advisory Committee on Immunization 
Practices’ and ‘Advisory Committee’ mean the 
advisory committee on immunization practices 
established by the Secretary pursuant to section 
222, acting through the Director of the Centers 
for Disease Control and Prevention.’’. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00163
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
164 
HR 6 RFS 
SEC. 2142. REVIEW OF PROCESSES AND CONSISTENCY OF 
ACIP RECOMMENDATIONS. 
(a) REVIEW.—The Director of the Centers for Dis-
ease Control and Prevention shall conduct a review of the 
process used by the Advisory Committee on Immunization 
Practices to evaluate consistency in formulating and 
issuing recommendations pertaining to vaccines. 
(b) CONSIDERATIONS.—The review under subsection 
(a) shall include assessment of— 
(1) the criteria used to evaluate new and exist-
ing vaccines; 
(2) the Grading of Recommendations, Assess-
ment, Development, and Evaluation (GRADE) ap-
proach to the review and analysis of scientific and 
economic data, including the scientific basis for such 
approach; and 
(3) the extent to which the processes used by 
the working groups of the Advisory Committee on 
Immunization 
Practices 
are 
consistent 
among 
groups. 
(c) STAKEHOLDERS.—In carrying out the review 
under subsection (a), the Director of the Centers for Dis-
ease Control and Prevention shall solicit input from vac-
cine stakeholders. 
(d) REPORT.—Not later than 18 months after the 
date of enactment of this Act, the Director of the Centers 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00164
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
165 
HR 6 RFS 
for Disease Control and Prevention shall submit to the 
appropriate committees of the Congress and make publicly 
available a report on the results of the review under sub-
section (a), including recommendations on improving the 
consistency of the process described in such subsection. 
(e) DEFINITION.—In this section, the term ‘‘Advisory 
Committee on Immunization Practices’’ means the advi-
sory committee on immunization practices established by 
the Secretary of Health and Human Services pursuant to 
section 222 of the Public Health Service Act (42 U.S.C. 
217a), acting through the Director of the Centers for Dis-
ease Control and Prevention. 
SEC. 2143. MEETINGS BETWEEN CDC AND VACCINE DEVEL-
OPERS. 
Section 310 of the Public Health Service Act (42 
U.S.C. 242o) is amended by adding at the end the fol-
lowing: 
‘‘(c)(1) In this subsection, the term ‘vaccine devel-
oper’ means a nongovernmental entity engaged in— 
‘‘(A)(i) the development of a vaccine with the 
intent to pursue licensing of the vaccine by the Food 
and Drug Administration; or 
‘‘(ii) the production of a vaccine licensed by the 
Food and Drug Administration; and 
‘‘(B) vaccine research. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00165
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
166 
HR 6 RFS 
‘‘(2)(A) Upon the submission of a written request for 
a meeting by a vaccine developer, that includes a valid jus-
tification for the meeting, the Secretary, acting through 
the Director of the Centers for Disease Control and Pre-
vention, shall convene a meeting of representatives of the 
vaccine developer and experts from the Centers for Dis-
ease Control and Prevention in immunization programs, 
epidemiology, and other relevant areas at which the Direc-
tor (or the Director’s designee), for the purpose of inform-
ing the vaccine developer’s understanding of public health 
needs and priorities, shall provide the perspectives of the 
Centers for Disease Control and Prevention and other rel-
evant Federal agencies regarding— 
‘‘(i) public health needs, epidemiology, and im-
plementation considerations with regard to a vaccine 
developer’s potential vaccine profile; and 
‘‘(ii) potential implications of such perspectives 
for the vaccine developer’s vaccine research and de-
velopment planning. 
‘‘(B) In addition to the representatives specified in 
subparagraph (A), the Secretary may, with the agreement 
of the vaccine developer requesting a meeting under such 
subparagraph, include in such meeting representatives 
of— 
‘‘(i) the Food and Drug Administration; and 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00166
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
167 
HR 6 RFS 
‘‘(ii) the National Vaccine Program. 
‘‘(C) The Secretary shall convene a meeting re-
quested with a valid justification under subparagraph (A) 
not later than 120 days after receipt of the request for 
the meeting. 
‘‘(3)(A) Upon the submission of a written request by 
a vaccine developer, the Secretary, acting through the Di-
rector of the Centers for Disease Control and Prevention, 
shall provide to the vaccine developer any age-based or 
other demographically assessed disease epidemiological 
analyses or data that— 
‘‘(i) are specified in the request; 
‘‘(ii) have been published; 
‘‘(iii) have been performed by or are in the pos-
session of the Centers; 
‘‘(iv) are not a trade secret or commercial or fi-
nancial information that is privileged or confidential 
and subject to section 552(b)(4) of title 5, United 
States Code, or section 1905 of title 18, United 
States Code; and 
‘‘(v) do not contain individually identifiable in-
formation. 
‘‘(B) The Secretary shall provide analyses requested 
by a vaccine manufacturer under subparagraph (A) not 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00167
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
168 
HR 6 RFS 
later than 120 calendar days after receipt of the request 
for the analyses. 
‘‘(4) The Secretary shall promptly notify a vaccine 
developer if— 
‘‘(A) the Secretary becomes aware of any sig-
nificant change to information that was— 
‘‘(i) shared by the Secretary with the vac-
cine developer during a meeting under para-
graph (2); or 
‘‘(ii) provided by the Secretary to the vac-
cine developer in one or more analyses under 
paragraph (3); and 
‘‘(B) the change to such information may have 
implications for the vaccine developer’s vaccine re-
search and development.’’. 
Subtitle I—Orphan Product Exten-
sions Now; Incentives for Cer-
tain Products for Limited Popu-
lations 
SEC. 2151. EXTENSION OF EXCLUSIVITY PERIODS FOR A 
DRUG APPROVED FOR A NEW INDICATION 
FOR A RARE DISEASE OR CONDITION. 
(a) IN GENERAL.—Chapter V of the Federal Food, 
Drug, and Cosmetic Act, as amended by sections 2062 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00168
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
169 
HR 6 RFS 
and 2063, is further amended by inserting after section 
505H of such Act the following: 
‘‘SEC. 505I. EXTENSION OF EXCLUSIVITY PERIODS FOR A 
DRUG APPROVED FOR A NEW INDICATION 
FOR A RARE DISEASE OR CONDITION. 
‘‘(a) DESIGNATION.— 
‘‘(1) IN GENERAL.—The Secretary shall des-
ignate a drug as a drug approved for a new indica-
tion to prevent, diagnose, or treat a rare disease or 
condition for purposes of granting the extensions 
under subsection (b) if— 
‘‘(A) prior to approval of an application or 
supplemental application for the new indication, 
the drug was approved or licensed for mar-
keting under section 505(c) of this Act or sec-
tion 351(a) of the Public Health Service Act 
but was not so approved or licensed for the new 
indication; 
‘‘(B)(i) the sponsor of the approved or li-
censed drug files an application or a supple-
mental application for approval of the new indi-
cation for use of the drug to prevent, diagnose, 
or treat the rare disease or condition; and 
‘‘(ii) the Secretary approves the application 
or supplemental application; and 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00169
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
170 
HR 6 RFS 
‘‘(C) the application or supplemental appli-
cation for the new indication contains the con-
sent of the applicant to notice being given by 
the Secretary under paragraph (4) respecting 
the designation of the drug. 
‘‘(2) REVOCATION OF DESIGNATION.— 
‘‘(A) IN GENERAL.—Except as provided in 
subparagraph (B), a designation under para-
graph (1) shall not be revoked for any reason. 
‘‘(B) EXCEPTION.—The Secretary may re-
voke a designation of a drug under paragraph 
(1) if the Secretary finds that the application or 
supplemental application resulting in such des-
ignation contained an untrue statement of ma-
terial fact. 
‘‘(3) NOTIFICATION PRIOR TO DISCONTINUANCE 
OF
PRODUCTION
FOR
SOLELY
COMMERCIAL
REA-
SONS.—A designation of a drug under paragraph (1) 
shall be subject to the condition that the sponsor of 
the drug will notify the Secretary of any discontinu-
ance of the production of the drug for solely com-
mercial reasons at least 1 year before such dis-
continuance. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00170
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
171 
HR 6 RFS 
‘‘(4) NOTICE
TO
PUBLIC.—Notice respecting 
the designation of a drug under paragraph (1) shall 
be made available to the public. 
‘‘(b) EXTENSION.—If the Secretary designates a 
drug as a drug approved for a new indication for a rare 
disease or condition, as described in subsection (a)(1)— 
‘‘(1)(A) the 4-, 5-, and 71⁄2-year periods de-
scribed in subsections (c)(3)(E)(ii) and (j)(5)(F)(ii) 
of section 505, the 3-year periods described in 
clauses (iii) and (iv) of subsection (c)(3)(E) and 
clauses (iii) and (iv) of subsection (j)(5)(F) of sec-
tion 505, and the 7-year period described in section 
527, as applicable, shall be extended by 6 months; 
or 
‘‘(B) the 4- and 12-year periods described in 
subparagraphs (A) and (B) of section 351(k)(7) of 
the Public Health Service Act and the 7-year period 
described in section 527, as applicable, shall be ex-
tended by 6 months; and 
‘‘(2)(A) if the drug is the subject of a listed 
patent for which a certification has been submitted 
under subsection (b)(2)(A)(ii) or (j)(2)(A)(vii)(II) of 
section 505 or a listed patent for which a certifi-
cation has been submitted under subsections 
(b)(2)(A)(iii) or (j)(2)(A)(vii)(III) of section 505, 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00171
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
172 
HR 6 RFS 
the period during which an application may not be 
approved 
under 
section 
505(c)(3) 
or 
section 
505(j)(5)(B) shall be extended by a period of 6 
months after the date the patent expires (including 
any patent extensions); or 
‘‘(B) if the drug is the subject of a listed patent 
for which a certification has been submitted under 
subsection (b)(2)(A)(iv) or (j)(2)(A)(vii)(IV) of sec-
tion 505, and in the patent infringement litigation 
resulting from the certification the court determines 
that the patent is valid and would be infringed, the 
period during which an application may not be ap-
proved 
under 
section 
505(c)(3) 
or 
section 
505(j)(5)(B) shall be extended by a period of 6 
months after the date the patent expires (including 
any patent extensions). 
‘‘(c) RELATION TO PEDIATRIC AND QUALIFIED IN-
FECTIOUS DISEASE PRODUCT EXCLUSIVITY.—Any exten-
sion under subsection (b) of a period shall be in addition 
to any extension of the periods under sections 505A and 
505E of this Act and section 351(m) of the Public Health 
Service Act, as applicable, with respect to the drug. 
‘‘(d) LIMITATIONS.—The extension described in sub-
section (b) shall not apply if the drug designated under 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00172
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
173 
HR 6 RFS 
subsection (a)(1) has previously received an extension by 
operation of subsection (b). 
‘‘(e) DEFINITION.—In this section, the term ‘rare 
disease or condition’ has the meaning given to such term 
in section 526(a)(2).’’. 
(b) APPLICATION.—Section 505G of the Federal 
Food, Drug, and Cosmetic Act, as added by subsection 
(a), applies only with respect to a drug for which an appli-
cation or supplemental application described in subsection 
(a)(1)(B)(i) of such section 505G is first approved under 
section 505(c) of such Act (21 U.S.C. 355(c)) or section 
351(a) of the Public Health Service Act (42 U.S.C. 
262(a)) on or after the date of the enactment of this Act. 
(c) CONFORMING AMENDMENTS.— 
(1) RELATION TO PEDIATRIC EXCLUSIVITY FOR 
DRUGS.—Section 505A of the Federal Food, Drug, 
and Cosmetic Act (21 U.S.C. 355a) is amended— 
(A) in subsection (b), by adding at the end 
the following: 
‘‘(3) RELATION TO EXCLUSIVITY FOR A DRUG 
APPROVED FOR A NEW INDICATION FOR A RARE DIS-
EASE
OR
CONDITION.—Notwithstanding the ref-
erences in paragraph (1) to the lengths of the exclu-
sivity periods after application of pediatric exclu-
sivity, the 6-month extensions described in para-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00173
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
174 
HR 6 RFS 
graph (1) shall be in addition to any extensions 
under section 505G.’’; and 
(B) in subsection (c), by adding at the end 
the following: 
‘‘(3) RELATION TO EXCLUSIVITY FOR A DRUG 
APPROVED FOR A NEW INDICATION FOR A RARE DIS-
EASE
OR
CONDITION.—Notwithstanding the ref-
erences in paragraph (1) to the lengths of the exclu-
sivity periods after application of pediatric exclu-
sivity, the 6-month extensions described in para-
graph (1) shall be in addition to any extensions 
under section 505G.’’. 
(2) RELATION
TO
EXCLUSIVITY
FOR
NEW 
QUALIFIED
INFECTIOUS
DISEASE
PRODUCTS
THAT 
ARE DRUGS.—Subsection (b) of section 505E of the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 
355f) is amended— 
(A) by amending the subsection heading to 
read as follows: ‘‘RELATION TO PEDIATRIC EX-
CLUSIVITY AND EXCLUSIVITY FOR A DRUG AP-
PROVED FOR A NEW INDICATION FOR A RARE 
DISEASE OR CONDITION.—’’; and 
(B) by striking ‘‘any extension of the pe-
riod under section 505A’’ and inserting ‘‘any 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00174
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
175 
HR 6 RFS 
extension of the periods under sections 505A 
and 505G, as applicable,’’. 
(3) RELATION TO PEDIATRIC EXCLUSIVITY FOR 
BIOLOGICAL
PRODUCTS.—Section 351(m) of the 
Public Health Service Act (42 U.S.C. 262(m)) is 
amended by adding at the end the following: 
‘‘(5) RELATION TO EXCLUSIVITY FOR A BIO-
LOGICAL PRODUCT APPROVED FOR A NEW INDICA-
TION FOR A RARE DISEASE OR CONDITION.—Not-
withstanding the references in paragraphs (2)(A), 
(2)(B), (3)(A), and (3)(B) to the lengths of the ex-
clusivity periods after application of pediatric exclu-
sivity, the 6-month extensions described in such 
paragraphs shall be in addition to any extensions 
under section 505G.’’. 
SEC. 2152. REAUTHORIZATION OF RARE PEDIATRIC DIS-
EASE PRIORITY REVIEW VOUCHER INCEN-
TIVE PROGRAM. 
(a) IN GENERAL.—Section 529 of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 360ff) is amended— 
(1) in subsection (a)— 
(A) in paragraph (3), by amending sub-
paragraph (A) to read as follows: 
‘‘(A) The disease is a serious or life-threat-
ening disease in which the serious or life-threat-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00175
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
176 
HR 6 RFS 
ening manifestations primarily affect individ-
uals aged from birth to 18 years, including age 
groups often called neonates, infants, children, 
and adolescents.’’; and 
(B) in paragraph (4)— 
(i) in subparagraph (E), by striking 
‘‘and’’ at the end; 
(ii) in subparagraph (F), by striking 
the period at the end and inserting ‘‘; 
and’’; and 
(iii) by adding at the end the fol-
lowing: 
‘‘(G) is for a drug or biological product for 
which a priority review voucher has not been 
issued under section 524 (relating to tropical 
disease products).’’; and 
(2) in subsection (b), by striking paragraph (5) 
and inserting the following: 
‘‘(5) TERMINATION OF AUTHORITY.— 
‘‘(A) IN
GENERAL.—The Secretary may 
not award any priority review vouchers under 
paragraph (1) after December 31, 2018. 
‘‘(B) EXCEPTION.—Notwithstanding sub-
paragraph (A), the sponsor of a drug that is 
designated under subsection (d) as a drug for 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00176
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
177 
HR 6 RFS 
a rare pediatric disease and that is the subject 
of a rare pediatric disease product application 
that is submitted during the period beginning 
on the date of enactment of the 21st Century 
Cures Act and ending the date specified in sub-
paragraph (A) shall remain eligible to receive a 
priority review voucher under paragraph (1) ir-
respective of whether the rare pediatric disease 
product application with respect to such drug is 
approved after the end of such period.’’. 
(b) GAO STUDY AND REPORT.— 
(1) STUDY.—The Comptroller General of the 
United States shall conduct a study on the effective-
ness of awarding priority review vouchers under sec-
tion 529 of the Federal Food, Drug, and Cosmetic 
Act (21 U.S.C. 360ff) in providing incentives for the 
development of drugs that treat or prevent rare pe-
diatric diseases (as defined in subsection (a)(3) of 
such section) that would not otherwise have been de-
veloped. In conducting such study, the Comptroller 
General shall examine the following: 
(A) The indications for which each drug 
for which a priority review voucher was award-
ed under such section 529 was approved under 
section 505 of such Act (21 U.S.C. 355) or sec-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00177
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
178 
HR 6 RFS 
tion 351 of the Public Health Service Act (42 
U.S.C. 262). 
(B) Whether the priority review voucher 
impacted a sponsor’s decision to invest in devel-
oping a drug to treat or prevent a rare pedi-
atric disease. 
(C) An analysis of the drugs that utilized 
such priority review vouchers, which shall in-
clude— 
(i) the indications for which such 
drugs were approved under section 505 of 
the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C. 355) or section 351 of the Pub-
lic Health Service Act (42 U.S.C. 262); 
(ii) whether unmet medical needs were 
addressed through the approval of such 
drugs, including, for each such drug— 
(I) if an alternative therapy was 
previously available to treat the indi-
cation; and 
(II) the benefit or advantage the 
drug provided over another available 
therapy; 
(iii) the number of patients potentially 
treated by such drugs; 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00178
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
179 
HR 6 RFS 
(iv) the value of the priority review 
voucher if transferred; and 
(v) the length of time between the 
date on which a priority review voucher 
was awarded and the date on which it was 
used. 
(D) With respect to the priority review 
voucher program under section 529 of the Fed-
eral Food, Drug, and Cosmetic Act (21 U.S.C. 
360ff)— 
(i) the resources used by, and burden 
placed on, the Food and Drug Administra-
tion in implementing such program, includ-
ing the effect of such program on the Food 
and Drug Administration’s review of drugs 
for which a priority review voucher was not 
awarded or used; 
(ii) the impact of the program on the 
public health as a result of the expedited 
review of applications for drugs that treat 
or prevent non-serious indications that are 
generally used by the broader public; and 
(iii) alternative approaches to improv-
ing such program so that the program is 
appropriately targeted toward providing in-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00179
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
180 
HR 6 RFS 
centives for the development of clinically 
important drugs that— 
(I) prevent or treat rare pediatric 
diseases; and 
(II) would likely not otherwise 
have been developed to prevent or 
treat such diseases. 
(2) REPORT.—Not later than December 31, 
2017, the Comptroller General of the United States 
shall submit to the Committee on Energy and Com-
merce of the House of Representatives and the Com-
mittee on Health, Education, Labor, and Pensions 
of the Senate a report containing the results of the 
study of conducted under paragraph (1). 
Subtitle 
J—Domestic 
Manufac-
turing and Export Efficiencies 
SEC. 2161. GRANTS FOR STUDYING THE PROCESS OF CON-
TINUOUS DRUG MANUFACTURING. 
(a) IN GENERAL.—The Commissioner of Food and 
Drugs may award grants to institutions of higher edu-
cation and nonprofit organizations for the purpose of 
studying and recommending improvements to the process 
of continuous manufacturing of drugs and biological prod-
ucts and similar innovative monitoring and control tech-
niques. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00180
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
181 
HR 6 RFS 
(b) DEFINITIONS.—In this section: 
(1) The term ‘‘drug’’ has the meaning given to 
such term in section 201 of the Federal Food, Drug, 
and Cosmetic Act (21 U.S.C. 321). 
(2) The term ‘‘biological product’’ has the 
meaning given to such term in section 351(i) of the 
Public Health Service Act (42 U.S.C. 262(i)). 
(3) The term ‘‘institution of higher education’’ 
has the meaning given to such term in section 101 
of the Higher Education Act of 1965 (20 U.S.C. 
1001). 
(c) 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. 2162. RE-EXPORTATION AMONG MEMBERS OF THE EU-
ROPEAN ECONOMIC AREA. 
Section 1003 of the Controlled Substances Import 
and Export Act (21 U.S.C. 953) is amended— 
(1) in subsection (f)— 
(A) in paragraph (5)— 
(i) by striking ‘‘(5)’’ and inserting 
‘‘(5)(A)’’; 
(ii) by inserting ‘‘, except that the 
controlled substance may be exported from 
the second country to another country that 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00181
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
182 
HR 6 RFS 
is a member of the European Economic 
Area’’ before the period at the end; and 
(iii) by adding at the end the fol-
lowing: 
‘‘(B) Subsequent to any re-exportation de-
scribed in subparagraph (A), a controlled substance 
may continue to be exported from any country that 
is a member of the European Economic Area to any 
other such country, provided that— 
‘‘(i) the conditions applicable with respect 
to the first country under paragraphs (1), (2), 
(3), (4), (6), and (7) are met by each subse-
quent country from which the controlled sub-
stance is exported pursuant to this paragraph; 
and 
‘‘(ii) the conditions applicable with respect 
to the second country under such paragraphs 
are met by each subsequent country to which 
the controlled substance is exported pursuant to 
this paragraph.’’; and 
(B) in paragraph (6)— 
(i) by striking ‘‘(6)’’ and inserting 
‘‘(6)(A)’’; and 
(ii) by adding at the end the fol-
lowing: 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00182
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
183 
HR 6 RFS 
‘‘(B) In the case of re-exportation among mem-
bers of the European Economic Area, within 30 
days after each re-exportation, the person who ex-
ported the controlled substance from the United 
States delivers to the Attorney General— 
‘‘(i) documentation certifying that such re- 
exportation has occurred; and 
‘‘(ii) information concerning the consignee, 
country, and product.’’; and 
(2) by adding at the end the following: 
‘‘(g) LIMITATION.—Subject to paragraphs (5) and 
(6) of subsection (f) in the case of any controlled sub-
stance in schedule I or II or any narcotic drug in schedule 
III or IV, the Attorney General shall not promulgate nor 
enforce any regulation, subregulatory guidance, or en-
forcement policy which impedes re-exportation of any con-
trolled substance among European Economic Area coun-
tries, including by promulgating or enforcing any require-
ment that— 
‘‘(1) re-exportation from the first country to the 
second country or re-exportation from the second 
country to another country occur within a specified 
period of time; or 
‘‘(2) information concerning the consignee, 
country, and product be provided prior to expor-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00183
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
184 
HR 6 RFS 
tation of the controlled substance from the United 
States or prior to each re-exportation among mem-
bers of the European Economic Area.’’. 
Subtitle K—Enhancing 
Combination Products Review 
SEC. 2181. ENHANCING COMBINATION PRODUCTS REVIEW. 
Section 503(g)(4)(C) of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 353(g)(4)(C)) is amended by 
adding at the end the following new clause: 
‘‘(iii) Not later than 18 months after the date of the 
enactment of the 21st Century Cures Act, the Secretary 
shall issue final guidance that describes the responsibilities 
of each agency center regarding its review of combination 
products. The Secretary shall, after soliciting public com-
ment, review and update the guidance periodically.’’. 
Subtitle L—Priority Review for 
Breakthrough Devices 
SEC. 2201. PRIORITY REVIEW FOR BREAKTHROUGH DE-
VICES. 
(a) IN GENERAL.—Chapter V of the Federal Food, 
Drug, and Cosmetic Act is amended— 
(1) in section 515(d)— 
(A) by striking paragraph (5); and 
(B) by redesignating paragraph (6) as 
paragraph (5); and 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00184
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
185 
HR 6 RFS 
(2) by inserting after section 515A (21 U.S.C. 
360e–1) the following: 
‘‘SEC. 515B. PRIORITY REVIEW FOR BREAKTHROUGH DE-
VICES. 
‘‘(a) IN GENERAL.—In order to provide for more ef-
fective treatment or diagnosis of life-threatening or irre-
versibly debilitating human diseases or conditions, the 
Secretary shall establish a program to provide priority re-
view for devices— 
‘‘(1) representing breakthrough technologies; 
‘‘(2) for which no approved alternatives exist; 
‘‘(3) offering significant advantages over exist-
ing approved or cleared alternatives, including the 
potential to, compared to existing approved or 
cleared alternatives, reduce or eliminate the need for 
hospitalization, improve patient quality of life, facili-
tate patients’ ability to manage their own care (such 
as through self-directed personal assistance), or es-
tablish long-term clinical efficiencies; or 
‘‘(4) the availability of which is in the best in-
terest of patients. 
‘‘(b) REQUEST FOR DESIGNATION.—A sponsor of a 
device may request that the Secretary designate the device 
for priority review under this section. Any such request 
for designation may be made at any time prior to the sub-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00185
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
186 
HR 6 RFS 
mission of an application under section 515(c), a petition 
for classification under section 513(f)(2), or a notification 
under section 510(k). 
‘‘(c) DESIGNATION PROCESS.— 
‘‘(1) IN GENERAL.—Not later than 60 calendar 
days after the receipt of a request under subsection 
(b), the Secretary shall determine whether the device 
that is the subject of the request meets the criteria 
described in subsection (a). If the Secretary deter-
mines that the device meets the criteria, the Sec-
retary shall designate the device for priority review. 
‘‘(2) REVIEW.—Review of a request under sub-
section (b) shall be undertaken by a team that is 
composed of experienced staff and managers of the 
Food and Drug Administration and is chaired by a 
senior manager. 
‘‘(3) DESIGNATION DETERMINATION.—A deter-
mination approving or denying a request under sub-
section (b) shall be considered a significant decision 
under section 517A and the Secretary shall provide 
a written, substantive summary of the basis for the 
determination in accordance with section 517A(a). 
‘‘(4) RECONSIDERATION.— 
‘‘(A) REQUEST FOR RECONSIDERATION.— 
Any person whose request under subsection (b) 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00186
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
187 
HR 6 RFS 
is denied may, within 30 days of the denial, re-
quest reconsideration of the denial in accord-
ance with section 517A(b)— 
‘‘(i) based upon the submission of 
documents by such person; or 
‘‘(ii) based upon such documents and 
a meeting or teleconference. 
‘‘(B) RESPONSE.—Reconsideration of a 
designation determination under this paragraph 
shall be conducted in accordance with section 
517A(b). 
‘‘(5) WITHDRAWAL.—If the Secretary approves 
a priority review designation for a device under this 
section, the Secretary may not withdraw the des-
ignation based on the fact that the criteria specified 
in subsection (a) are no longer met because of the 
subsequent clearance or approval of another device 
that was designated under— 
‘‘(A) this section; or 
‘‘(B) section 515(d)(5) (as in effect imme-
diately prior to the enactment of the 21st Cen-
tury Cures Act). 
‘‘(d) PRIORITY REVIEW.— 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00187
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
188 
HR 6 RFS 
‘‘(1) ACTIONS.—For purposes of expediting the 
development and review of devices designated under 
subsection (c), the Secretary shall— 
‘‘(A) assign a team of staff, including a 
team leader with appropriate subject matter ex-
pertise and experience, for each device for 
which a request is submitted under subsection 
(b); 
‘‘(B) provide for oversight of the team by 
senior agency personnel to facilitate the effi-
cient development of the device and the efficient 
review of any submission described in sub-
section (b) for the device; 
‘‘(C) adopt an efficient process for timely 
dispute resolution; 
‘‘(D) provide for interactive communication 
with the sponsor of the device during the review 
process; 
‘‘(E) expedite the Secretary’s review of 
manufacturing and quality systems compliance, 
as applicable; 
‘‘(F) disclose to the sponsor in advance the 
topics of any consultation concerning the spon-
sor’s device that the Secretary intends to under-
take with external experts or an advisory com-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00188
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
189 
HR 6 RFS 
mittee and provide the sponsor an opportunity 
to recommend such external experts; 
‘‘(G) for applications submitted under sec-
tion 515(c), provide for advisory committee 
input, as the Secretary determines appropriate 
(including in response to the request of the 
sponsor); and 
‘‘(H) assign staff to be available within a 
reasonable time to address questions posed by 
institutional review committees concerning the 
conditions and clinical testing requirements ap-
plicable to the investigational use of the device 
pursuant to an exemption under section 520(g). 
‘‘(2) ADDITIONAL ACTIONS.—In addition to the 
actions described in paragraph (1), for purposes of 
expediting the development and review of devices 
designated under subsection (c), the Secretary, in 
collaboration with the device sponsor, may, as appro-
priate— 
‘‘(A) coordinate with the sponsor regarding 
early agreement on a data development plan; 
‘‘(B) take steps to ensure that the design 
of clinical trials is as efficient as practicable, 
such as through adoption of shorter or smaller 
clinical 
trials, 
application 
of 
surrogate 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00189
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
190 
HR 6 RFS 
endpoints, and use of adaptive trial designs and 
Bayesian statistics, to the extent scientifically 
appropriate; 
‘‘(C) facilitate, to the extent scientifically 
appropriate, expedited and efficient develop-
ment and review of the device through utiliza-
tion of timely postmarket data collection, with 
regard to applications for approval under sec-
tion 515(c); and 
‘‘(D) agree to clinical protocols that the 
Secretary will consider binding on the Secretary 
and the sponsor, subject to— 
‘‘(i) changes agreed to by the sponsor 
and the Secretary; 
‘‘(ii) changes that the Secretary deter-
mines are required to prevent an unreason-
able risk to the public health; or 
‘‘(iii) the identification of a substan-
tial scientific issue determined by the Sec-
retary to be essential to the safety or effec-
tiveness of the device involved. 
‘‘(e) PRIORITY REVIEW GUIDANCE.— 
‘‘(1) CONTENT.—The Secretary shall issue 
guidance on the implementation of this section. Such 
guidance shall include the following: 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00190
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
191 
HR 6 RFS 
‘‘(A) The process for a person to seek a 
priority review designation. 
‘‘(B) A template for requests under sub-
section (b). 
‘‘(C) The criteria the Secretary will use in 
evaluating a request for priority review. 
‘‘(D) The standards the Secretary will use 
in assigning a team of staff, including team 
leaders, to review devices designated for priority 
review, including any training required for such 
personnel on effective and efficient review. 
‘‘(2) PROCESS.—Prior to finalizing the guid-
ance under paragraph (1), the Secretary shall pro-
pose such guidance for public comment. 
‘‘(f) CONSTRUCTION.— 
‘‘(1) PURPOSE.—This section is intended to en-
courage the Secretary and provide the Secretary suf-
ficient authorities to apply efficient and flexible ap-
proaches to expedite the development of, and 
prioritize the agency’s review of, devices that rep-
resent breakthrough technologies. 
‘‘(2) CONSTRUCTION.—Nothing in this section 
shall be construed to alter the criteria and standards 
for evaluating an application pursuant to section 
515(c), a report and request for classification under 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00191
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
192 
HR 6 RFS 
section 513(f)(2), or a report under section 510(k), 
including the recognition of valid scientific evidence 
as described in section 513(a)(3)(B), and consider-
ation of the least burdensome means of evaluating 
device effectiveness or demonstrating substantial 
equivalence between devices with differing techno-
logical characteristics, as applicable. Nothing in this 
section alters the authority of the Secretary to act 
on an application pursuant to section 515(d) before 
completion of an establishment inspection, as the 
Secretary deems appropriate.’’. 
(b) CONFORMING AMENDMENT RELATED TO DES-
IGNATION DETERMINATIONS.—Section 517A(a)(1) of the 
Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360g– 
1(a)(1)) is amended by inserting ‘‘a request for designa-
tion under section 515B,’’ after ‘‘an application under sec-
tion 515,’’. 
Subtitle M—Medical Device 
Regulatory Process Improvements 
SEC. 2221. THIRD-PARTY QUALITY SYSTEM ASSESSMENT. 
(a) ESTABLISHMENT
OF THIRD-PARTY QUALITY 
SYSTEM ASSESSMENT PROGRAM.—Chapter V of the Fed-
eral Food, Drug, and Cosmetic Act is amended by insert-
ing after section 524A (21 U.S.C. 360n–1) the following 
new section: 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00192
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
193 
HR 6 RFS 
‘‘SEC. 524B. THIRD-PARTY QUALITY SYSTEM ASSESSMENT. 
‘‘(a) ACCREDITATION AND ASSESSMENT.— 
‘‘(1) IN GENERAL; CERTIFICATION OF DEVICE 
QUALITY SYSTEM.—The Secretary shall, in accord-
ance with this section, establish a third-party quality 
system assessment program— 
‘‘(A) to accredit persons to assess whether 
a requestor’s quality system, including its de-
sign controls, can reasonably assure the safety 
and effectiveness of in-scope devices subject to 
device-related changes; 
‘‘(B) under which accredited persons shall 
(as applicable) certify that a requestor’s quality 
system meets the criteria included in the guid-
ance issued under paragraph (5) with respect to 
the in-scope devices at issue; and 
‘‘(C) under which the Secretary shall rely 
on such certifications for purposes of deter-
mining the safety and effectiveness (or as appli-
cable, substantial equivalence) of in-scope de-
vices subject to the device-related changes in-
volved, in lieu of compliance with the following 
submission requirements: 
‘‘(i) A premarket notification. 
‘‘(ii) A 30-day notice. 
‘‘(iii) A Special PMA supplement. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00193
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
194 
HR 6 RFS 
‘‘(2) DEFINITIONS.—For purposes of this sec-
tionƒ 
‘‘(A) the term ‘device-related changes’ 
means changes made by a requestor with re-
spect to in-scope devices, which are— 
‘‘(i) changes to a device found to be 
substantially equivalent under sections 
513(i) and 510(k) to a predicate device, 
that— 
‘‘(I) would otherwise be subject 
to a premarket notification; and 
‘‘(II) do not alter— 
‘‘(aa) the intended use of 
the changed device; or 
‘‘(bb) the fundamental sci-
entific technology of such device; 
‘‘(ii) manufacturing changes subject 
to a 30-day notice; 
‘‘(iii) changes that qualify for a Spe-
cial PMA Supplement; and 
‘‘(iv) such other changes relating to 
the devices or the device manufacturing 
process as the Secretary determines appro-
priate; 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00194
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
195 
HR 6 RFS 
‘‘(B) the term ‘in-scope device’ means a 
device within the scope of devices agreed to by 
the requestor and the accredited person for pur-
poses of a request for certification under this 
section; 
‘‘(C) the term ‘premarket notification’ 
means a premarket notification under section 
510(k); 
‘‘(D) the term ‘quality system’ means the 
methods used in, and the facilities and controls 
used for, the design, manufacture, packaging, 
labeling, storage, installation, and servicing of 
devices, as described in section 520(f); 
‘‘(E) the term ‘requestor’ means a device 
manufacturer that is seeking certification under 
this section of a quality system used by such 
manufacturer; 
‘‘(F) the term ‘Special PMA’ means a Spe-
cial PMA supplement under section 814.39(d) 
of title 21, Code of Federal Regulations (or any 
successor regulations); and 
‘‘(G) the term ‘30-day notice’ means a no-
tice described in section 515(d)(6). 
‘‘(3) ACCREDITATION PROCESS; ACCREDITATION 
RENEWAL.—Except as inconsistent with this section, 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00195
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
196 
HR 6 RFS 
the process and qualifications for accreditation of 
persons and renewal of such accreditation under sec-
tion 704(g) shall apply with respect to accreditation 
of persons and renewal of such accreditation under 
this section. 
‘‘(4) USE OF ACCREDITED PARTIES TO CON-
DUCT ASSESSMENTS.— 
‘‘(A) INITIATION
OF
ASSESSMENT
SERV-
ICES.— 
‘‘(i) DATE
ASSESSMENTS
AUTHOR-
IZED.—Beginning after the date on which 
the final guidance is issued under para-
graph (5), an accredited person may con-
duct an assessment under this section. 
‘‘(ii) INITIATION OF ASSESSMENTS.— 
Use of one or more accredited persons to 
assess a requestor’s quality system under 
this section with respect to in-scope devices 
shall be at the initiation of the person who 
registers and lists the devices at issue 
under section 510. 
‘‘(B) COMPENSATION.—Compensation for 
such accredited persons shall— 
‘‘(i) be determined by agreement be-
tween the accredited person and the person 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00196
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
197 
HR 6 RFS 
who engages the services of the accredited 
person; and 
‘‘(ii) be paid by the person who en-
gages such services. 
‘‘(C) ACCREDITED PERSON SELECTION.— 
Each person who chooses to use an accredited 
person to assess a requestor’s quality system, 
as described in this section, shall select the ac-
credited person from a list of such persons pub-
lished by the Secretary in accordance with sec-
tion 704(g)(4). 
‘‘(5) 
GUIDANCE; 
CRITERIA
FOR
CERTIFI-
CATION.— 
‘‘(A) IN GENERAL.—The criteria for cer-
tification of a quality system under this section 
shall be as specified by the Secretary in guid-
ance issued under this paragraph. 
‘‘(B) CONTENTS; CRITERIA.—The guidance 
under this paragraph shall include specification 
of— 
‘‘(i) evaluative criteria to be used by 
an accredited person to assess and, as ap-
plicable, certify a requestor’s quality sys-
tem under this section with respect to in- 
scope devices; and 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00197
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
198 
HR 6 RFS 
‘‘(ii) criteria for accredited persons to 
apply for a waiver of, and exemptions 
from, the criteria under clause (i). 
‘‘(C) TIMEFRAME
FOR
ISSUING
GUID-
ANCE.—The Secretary shall issue under this 
paragraph— 
‘‘(i) draft guidance not later than 12 
months after the enactment of the 21st 
Century Cures Act; and 
‘‘(ii) final guidance not later than 12 
months after issuance of the draft guid-
ance under clause (i). 
‘‘(b) USE OF THIRD-PARTY ASSESSMENT.— 
‘‘(1) 
ASSESSMENT
SUMMARY; 
CERTIFI-
CATION.— 
‘‘(A) SUBMISSION OF ASSESSMENT TO SEC-
RETARY.—An accredited person who assesses a 
requestor’s quality system under subsection (a) 
shall submit to the Secretary a summary of the 
assessment— 
‘‘(i) within 30 days of the assessment; 
and 
‘‘(ii) which shall include (as applica-
ble)— 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00198
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
199 
HR 6 RFS 
‘‘(I) the accredited person’s cer-
tification that the requestor has satis-
fied the criteria specified in the guid-
ance issued under subsection (a)(5) 
for quality system certification with 
respect to the in-scope devices at 
issue; and 
‘‘(II) any waivers or exemptions 
from such criteria applied by the ac-
credited person. 
‘‘(B) TREATMENT
OF
ASSESSMENTS.— 
Subject to action by the Secretary under sub-
paragraph (C), with respect to assessments 
which include a certification under this sec-
tion— 
‘‘(i) the Secretary’s review of the as-
sessment summary shall be deemed com-
plete on the day that is 30 days after the 
date on which the Secretary receives the 
summary under subparagraph (A); and 
‘‘(ii) the assessment summary and 
certification of the quality system of a re-
questor shall be deemed accepted by the 
Secretary on such 30th day. 
‘‘(C) ACTIONS BY SECRETARY.— 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00199
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
200 
HR 6 RFS 
‘‘(i) IN GENERAL.—Within 30 days of 
receiving an assessment summary and cer-
tification under subparagraph (A), the Sec-
retary may, by written notice to the ac-
credited person submitting such assess-
ment certification, deem any such certifi-
cation to be provisional beyond such 30- 
day period, suspended pending further re-
view by the Secretary, or otherwise quali-
fied or cancelled, based on the Secretary’s 
determination that (as applicable)— 
‘‘(I) additional information is 
needed to support such certification; 
‘‘(II) such assessment or certifi-
cation is unwarranted; or 
‘‘(III) such action with regard to 
the certification is otherwise justified 
according to such factors and criteria 
as the Secretary finds appropriate. 
‘‘(ii) 
ACCEPTANCE
OF
CERTIFI-
CATION.—If following action by the Sec-
retary under clause (i) with respect to a 
certification, the Secretary determines that 
such certification is acceptable, the Sec-
retary shall issue written notice to the ap-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00200
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
201 
HR 6 RFS 
plicable accredited person indicating such 
acceptance. 
‘‘(2) NOTIFICATIONS TO SECRETARY BY CER-
TIFIED REQUESTORS OR ACCREDITED PERSONS FOR 
PROGRAM EVALUATION PURPOSES.— 
‘‘(A) ANNUAL SUMMARY REPORT FOR DE-
VICE-RELATED CHANGES OTHERWISE SUBJECT 
TO
PREMARKET
NOTIFICATION.—A requestor 
whose quality system is certified under this sec-
tion that effectuates device-related changes with 
respect to in-scope devices, without prior sub-
mission of a premarket notification, shall en-
sure that an annual summary report is sub-
mitted to the Secretary by the accredited per-
son which— 
‘‘(i) describes the changes made to the 
in-scope device; and 
‘‘(ii) indicates the effective dates of 
such changes. 
‘‘(B) PERIODIC NOTIFICATION FOR MANU-
FACTURING CHANGES OTHERWISE SUBJECT TO 
30-DAY
NOTICE.—A requestor whose quality 
system is certified under this section that effec-
tuates device-related changes with respect to in- 
scope devices, without prior submission of a 30- 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00201
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
202 
HR 6 RFS 
day notice, shall provide notification to the Sec-
retary of such changes in the requestor’s next 
periodic report under section 814.84(b) of title 
21, Code of Federal Regulations (or any suc-
cessor regulation). Such notification shall— 
‘‘(i) describe the changes made; and 
‘‘(ii) indicate the effective dates of 
such changes. 
‘‘(C) PERIODIC
NOTIFICATION
FOR
DE-
VICE-RELATED CHANGES OTHERWISE SUBJECT 
TO
SPECIAL
PMA
SUPPLEMENT.—A requestor 
whose quality system is certified under this sec-
tion that effectuates device-related changes with 
respect to in-scope devices, without prior sub-
mission of a Special PMA Supplement, shall 
provide notification to the Secretary of such 
changes in the requestor’s next periodic report 
under section 814.84(b) of title 21, Code of 
Federal Regulations (or any successor regula-
tion). Such notification shall— 
‘‘(i) describe the changes made, in-
cluding a full explanation of the basis for 
the changes; and 
‘‘(ii) indicate the effective dates of 
such changes. 
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00202
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS
203 
HR 6 RFS 
‘‘(D) USE
OF
NOTIFICATIONS
FOR
PRO-
GRAM
EVALUATION
PURPOSES.—Information 
submitted to the Secretary under subpara-
graphs (A) through (C) shall be used by the 
Secretary for purposes of the program evalua-
tion under subsection (d). 
‘‘(c) DURATION AND EFFECT OF CERTIFICATION.— 
A certification under this section— 
‘‘(1) shall remain in effect for a period of 2 
years from the date such certification is accepted by 
the Secretary, subject to paragraph (6); 
‘‘(2) may be renewed through the process de-
scribed in subsection (a)(3); 
‘‘(3) shall continue to apply with respect to de-
vice-related changes made during such 2-year period, 
provided the certification remains in effect, irrespec-
tive of whether such certification is renewed after 
such 2-year period; 
‘‘(4) shall have no effect on the need to comply 
with applicable submission requirements specified in 
subsection (a)(1)(C) with respect to any change per-
taining to in-scope devices which is not a device-re-
lated change under subsection (a)(2); 
‘‘(5) shall have no effect on the authority of the 
Secretary to conduct an inspection or otherwise de-
VerDate Sep 11 2014 
22:29 Jul 13, 2015
Jkt 049200
PO 00000
Frm 00203
Fmt 6652
Sfmt 6201
E:\BILLS\H6.RFS
H6
asabaliauskas on DSK5VPTVN1PROD with BILLS