‘‘(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
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—
‘‘(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;
‘‘(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—
(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;
(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
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.
(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—
(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:
‘‘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-
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
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).
‘‘(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
‘‘(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
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
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.
‘‘(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.
‘‘(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—
(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
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
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,
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).
100 
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
101 
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—
102 
‘‘(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-
103 
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
104 
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
105 
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
106 
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—
107 
(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
108 
(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:
109 
‘‘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
110 
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
111 
‘‘(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;
112 
‘‘(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.
113 
‘‘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
114 
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
115 
‘‘(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
116 
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
117 
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
118 
‘‘(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).
119 
(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-
120 
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.
121 
‘‘(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
122 
‘‘(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)).
123 
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-
124 
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.
125 
‘‘(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
126 
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);
127 
‘‘(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;
128 
‘‘(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
129 
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-