259 
whether the approved strategy should be modi-
fied to— 
‘‘(i) ensure the benefits of the drug 
outweigh the risks of the drug; or 
‘‘(ii) minimize the burden on the 
health care delivery system of complying 
with the strategy.’’; and 
(C) by striking subparagraph (D); 
(3) in paragraph (3), by striking ‘‘for a drug 
shall include—’’ and all that follows and inserting 
the following ‘‘for a drug shall include, with respect 
to each goal included in the strategy, an assessment 
of the extent to which the approved strategy, includ-
ing each element of the strategy, is meeting the goal 
or whether 1 or more such goals or such elements 
should be modified.’’; and 
(4) by amending paragraph (4) to read as fol-
lows: 
‘‘(4) MODIFICATION.— 
‘‘(A) ON
INITIATIVE
OF
RESPONSIBLE 
PERSON.—After the approval of a risk evalua-
tion and mitigation strategy by the Secretary, 
the responsible person may, at any time, submit 
to the Secretary a proposal to modify the ap-
proved strategy. Such proposal may propose the 
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addition, modification, or removal of any goal 
or element of the approved strategy and shall 
include an adequate rationale to support such 
proposed addition, modification, or removal of 
any goal or element of the strategy. 
‘‘(B) ON
INITIATIVE
OF
SECRETARY.— 
After the approval of a risk evaluation and 
mitigation strategy by the Secretary, the Sec-
retary may, at any time, require a responsible 
person to submit a proposed modification to the 
strategy within 120 days or within such reason-
able time as the Secretary specifies, if the Sec-
retary, in consultation with the offices described 
in subsection (c)(2), determines that 1 or more 
goals or elements should be added, modified, or 
removed from the approved strategy to— 
‘‘(i) ensure the benefits of the drug 
outweigh the risks of the drug; or 
‘‘(ii) minimize the burden on the 
health care delivery system of complying 
with the strategy.’’. 
(b) REVIEW OF PROPOSED STRATEGIES; REVIEW OF 
ASSESSMENTS
AND
MODIFICATIONS
OF
APPROVED 
STRATEGIES.—Section 505–1(h) (21 U.S.C. 355–1(h)) is 
amended— 
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(1) in the subsection heading by inserting ‘‘AND 
MODIFICATIONS’’ 
after 
‘‘REVIEW
OF
ASSESS-
MENTS’’; 
(2) in paragraph (1)— 
(A) by inserting ‘‘and proposed modifica-
tion to’’ after ‘‘under subsection (a) and each 
assessment of’’; and 
(B) by inserting ‘‘, and, if necessary, 
promptly initiate discussions with the respon-
sible person about such proposed strategy, as-
sessment, or modification’’ after ‘‘subsection 
(g)’’; 
(3) by striking paragraph (2); 
(4) by redesignating paragraphs (3) through 
(9) as paragraphs (2) through (8), respectively; 
(5) in paragraph (2), as redesignated by para-
graph (4)— 
(A) by amending subparagraph (A) to read 
as follows: 
‘‘(A) IN GENERAL.— 
‘‘(i) TIMEFRAME.—Unless the dispute 
resolution process described under para-
graph (3) or (4) applies, and, except as 
provided in clause (ii) or clause (iii) below, 
the Secretary, in consultation with the of-
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fices described in subsection (c)(2), shall 
review and act on the proposed risk evalua-
tion and mitigation strategy for a drug or 
any proposed modification to any required 
strategy within 180 days of receipt of the 
proposed strategy or modification. 
‘‘(ii) MINOR
MODIFICATIONS.—The 
Secretary shall review and act on a pro-
posed minor modification, as defined by 
the Secretary in guidance, within 60 days 
of receipt of such modification. 
‘‘(iii) REMS MODIFICATION DUE TO 
SAFETY LABEL CHANGES.—Not later than 
60 days after the Secretary receives a pro-
posed modification to an approved risk 
evaluation and mitigation strategy to con-
form the strategy to approved safety label 
changes, including safety labeling changes 
initiated by the sponsor in accordance with 
FDA regulatory requirements, or to a safe-
ty label change that the Secretary has di-
rected the holder of the application to 
make pursuant to section 505(o)(4), the 
Secretary shall review and act on such pro-
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posed modification to the approved strat-
egy. 
‘‘(iv) GUIDANCE.—The Secretary shall 
establish, through guidance, that respon-
sible persons may implement certain modi-
fications to an approved risk evaluation 
and mitigation strategy following notifica-
tion to the Secretary.’’; and 
(B) by amending subparagraph (C) to read 
as follows: 
‘‘(C) PUBLIC AVAILABILITY.—Upon acting 
on a proposed risk evaluation and mitigation 
strategy or proposed modification to a risk eval-
uation and mitigation strategy under subpara-
graph (A), the Secretary shall make publicly 
available an action letter describing the actions 
taken by the Secretary under such subpara-
graph (A).’’. 
(6) in paragraph (4), as redesignated by para-
graph (4)— 
(A) in subparagraph (A)(i)— 
(i) by striking ‘‘Not earlier than 15 
days, and not later than 35 days, after dis-
cussions under paragraph (2) have begun, 
the’’ and inserting ‘‘The’’; and 
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264 
(ii) by inserting ‘‘, after the sponsor is 
required to make a submission under sub-
section (a)(2) or (g),’’ before ‘‘request in 
writing’’; and 
(B) in subparagraph (I)— 
(i) by striking clauses (i) and (ii); and 
(ii) by striking ‘‘if the Secretary—’’ 
and inserting ‘‘if the Secretary has com-
plied with the timing requirements of 
scheduling review by the Drug Safety 
Oversight Board, providing a written rec-
ommendation, and issuing an action letter 
under subparagraphs (B), (F), and (G), 
respectively.’’; 
(7) in paragraph (5), as redesignated by para-
graph (4)— 
(A) in subparagraph (A), by striking ‘‘any 
of subparagraph (B) through (D)’’ and insert-
ing ‘‘subparagraph (B) or (C)’’; and 
(B) in subparagraph (C), by striking 
‘‘paragraph (4) or (5)’’ and inserting ‘‘para-
graph (3) or (4)’’; and 
(8) in paragraph (8), as redesignated by para-
graph (4), by striking ‘‘paragraphs (7) and (8)’’ and 
inserting ‘‘paragraphs (6) and (7).’’. 
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(c) GUIDANCE.—Not later than 1 year after the date 
of enactment of this Act, the Secretary of Health and 
Human Services shall issue guidance that, for purposes 
of section 505–1(h)(2)(A) of the Federal Food, Drug, and 
Cosmetic Act (21 U.S.C. 355–1(h)(2)(A)), describes the 
types of modifications to approved risk evaluation and 
mitigation strategies that shall be considered to be minor 
modifications of such strategies. 
SEC. 868. CONSULTATION WITH EXTERNAL EXPERTS ON 
RARE DISEASES, TARGETED THERAPIES, AND 
GENETIC TARGETING OF TREATMENTS. 
Subchapter E of chapter V (21 U.S.C. 360bbb et 
seq.) is amended by adding at the end the following: 
‘‘SEC. 568. CONSULTATION WITH EXTERNAL EXPERTS ON 
RARE DISEASES, TARGETED THERAPIES, AND 
GENETIC TARGETING OF TREATMENTS. 
‘‘(a) IN GENERAL.—For the purpose of promoting 
the efficiency of and informing the review by the Food 
and Drug Administration of new drugs and biological 
products for rare diseases and drugs and biological prod-
ucts that are genetically targeted, the following shall 
apply: 
‘‘(1) CONSULTATION WITH STAKEHOLDERS.— 
Consistent with sections X.C and IX.E.4 of the 
PDUFA Reauthorization Performance Goals and 
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Procedures Fiscal Years 2013 through 2017, as ref-
erenced in the letters described in section 101(b) of 
the Prescription Drug User Fee Amendments of 
2012, the Secretary shall ensure that opportunities 
exist, at a time the Secretary determines appro-
priate, for consultations with stakeholders on the 
topics described in subsection (b). 
‘‘(2) CONSULTATION
WITH
EXTERNAL
EX-
PERTS.— 
‘‘(A) IN
GENERAL.—The Secretary shall 
develop and maintain a list of external experts 
who, because of their special expertise, are 
qualified to provide advice on rare disease 
issues, including topics described in subsection 
(c). The Secretary may, when appropriate to 
address a specific regulatory question, consult 
such external experts on issues related to the 
review of new drugs and biological products for 
rare diseases and drugs and biological products 
that are genetically targeted, including the top-
ics described in subsection (b), when such con-
sultation is necessary because the Secretary 
lacks the specific scientific, medical, or tech-
nical expertise necessary for the performance of 
the Secretary’s regulatory responsibilities and 
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the necessary expertise can be provided by the 
external experts. 
‘‘(B) EXTERNAL EXPERTS.—For purposes 
of subparagraph (A), external experts are indi-
viduals who possess scientific or medical train-
ing that the Secretary lacks with respect to one 
or more rare diseases. 
‘‘(b) TOPICS FOR CONSULTATION.—Topics for con-
sultation pursuant to this section may include— 
‘‘(1) rare diseases; 
‘‘(2) the severity of rare diseases; 
‘‘(3) the unmet medical need associated with 
rare diseases; 
‘‘(4) the willingness and ability of individuals 
with a rare disease to participate in clinical trials; 
‘‘(5) an assessment of the benefits and risks of 
therapies to treat rare diseases; 
‘‘(6) the general design of clinical trials for rare 
disease populations and subpopulations; and 
‘‘(7) the demographics and the clinical descrip-
tion of patient populations. 
‘‘(c) CLASSIFICATION AS SPECIAL GOVERNMENT EM-
PLOYEES.—The external experts who are consulted under 
this section may be considered special government employ-
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ees, as defined under section 202 of title 18, United States 
Code. 
‘‘(d) PROTECTION OF CONFIDENTIAL INFORMATION 
AND TRADE SECRETS.— 
‘‘(1) RULE
OF
CONSTRUCTION.—Nothing in 
this section shall be construed to alter the protec-
tions offered by laws, regulations, and policies gov-
erning disclosure of confidential commercial or trade 
secret information, and any other information ex-
empt from disclosure pursuant to section 552(b) of 
title 5, United States Code, as such provisions would 
be applied to consultation with individuals and orga-
nizations prior to the date of enactment of this sec-
tion. 
‘‘(2) CONSENT REQUIRED FOR DISCLOSURE.— 
The Secretary shall not disclose confidential com-
mercial or trade secret information to an expert con-
sulted under this section without the written consent 
of the sponsor unless the expert is a special govern-
ment employee (as defined under section 202 of title 
18, United States Code) or the disclosure is other-
wise authorized by law. 
‘‘(e) OTHER CONSULTATION.—Nothing in this sec-
tion shall be construed to limit the ability of the Secretary 
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to consult with individuals and organizations as authorized 
prior to the date of enactment of this section. 
‘‘(f) NO RIGHT OR OBLIGATION.— 
‘‘(1) NO RIGHT TO CONSULTATION.—Nothing 
in this section shall be construed to create a legal 
right for a consultation on any matter or require the 
Secretary to meet with any particular expert or 
stakeholder. 
‘‘(2) NO ALTERING OF GOALS.—Nothing in this 
section shall be construed to alter agreed upon goals 
and procedures identified in the letters described in 
section 101(b) of the Prescription Drug User Fee 
Amendments of 2012. 
‘‘(3) NO CHANGE TO NUMBER OF REVIEW CY-
CLES.—Nothing in this section is intended to in-
crease the number of review cycles as in effect before 
the date of enactment of this section. 
‘‘(g) NO DELAY IN PRODUCT REVIEW.—Prior to a 
consultation with an external expert, as described in this 
section, relating to an investigational new drug application 
under section 505(i), a new drug application under section 
505(b), or a biologics license application under section 351 
of the Public Health Service Act, the Director of the Cen-
ter for Drug Evaluation and Research or the Director of 
the Center for Biologics Evaluation and Research (or ap-
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