270 
propriate Division Director), as appropriate, shall deter-
mine that— 
‘‘(1) such consultation will— 
‘‘(A) facilitate the Secretary’s ability to 
complete the Secretary’s review; 
‘‘(B) address outstanding deficiencies in 
the application; and 
‘‘(C) increase the likelihood of an approval 
decision in the current review cycle; or 
‘‘(2) the sponsor authorized such consultation.’’. 
SEC. 869. BREAKTHROUGH THERAPIES. 
(a) IN GENERAL.—Section 506 (21 U.S.C. 356), as 
amended by section 841, is further amended— 
(1) by redesignating subsection (d) as sub-
section (f); 
(2) by redesignating subsections (a) through (c) 
as subsections (b) through (d), respectively; 
(3) by inserting before subsection (b), as so re-
designated, the following: 
‘‘(a) DESIGNATION OF A DRUG AS A BREAKTHROUGH 
THERAPY.— 
‘‘(1) IN GENERAL.—The Secretary shall, at the 
request of the sponsor of a drug, expedite the devel-
opment and review of such drug if the drug is in-
tended, alone or in combination with 1 or more other 
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drugs, to treat a serious or life-threatening disease 
or condition and preliminary clinical evidence indi-
cates that the drug may demonstrate substantial im-
provement over existing therapies on 1 or more clini-
cally significant endpoints, such as substantial treat-
ment effects observed early in clinical development. 
(In this section, such a drug is referred to as a 
‘breakthrough therapy’.) 
‘‘(2) REQUEST FOR DESIGNATION.—The spon-
sor of a drug may request the Secretary to designate 
the drug as a breakthrough therapy. A request for 
the designation may be made concurrently with, or 
at any time after, the submission of an application 
for the investigation of the drug under section 505(i) 
or section 351(a)(3) of the Public Health Service 
Act. 
‘‘(3) DESIGNATION.— 
‘‘(A) IN GENERAL.—Not later than 60 cal-
endar days after the receipt of a request under 
paragraph (2), the Secretary shall determine 
whether the drug that is the subject of the re-
quest meets the criteria described in paragraph 
(1). If the Secretary finds that the drug meets 
the criteria, the Secretary shall designate the 
drug as a breakthrough therapy and shall take 
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such actions as are appropriate to expedite the 
development and review of the application for 
approval of such drug. 
‘‘(B) ACTIONS.—The actions to expedite 
the development and review of an application 
under subparagraph (A) may include, as appro-
priate— 
‘‘(i) holding meetings with the sponsor 
and the review team throughout the devel-
opment of the drug; 
‘‘(ii) providing timely advice to, and 
interactive communication with, the spon-
sor regarding the development of the drug 
to ensure that the development program to 
gather the non-clinical and clinical data 
necessary for approval is as efficient as 
practicable; 
‘‘(iii) involving senior managers and 
experienced review staff, as appropriate, in 
a collaborative, cross-disciplinary review; 
‘‘(iv) assigning a cross-disciplinary 
project lead for the Food and Drug Ad-
ministration review team to facilitate an 
efficient review of the development pro-
gram and to serve as a scientific liaison be-
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tween the review team and the sponsor; 
and 
‘‘(v) taking steps to ensure that the 
design of the clinical trials is as efficient as 
practicable, when scientifically appropriate, 
such as by minimizing the number of pa-
tients exposed to a potentially less effica-
cious treatment.’’; 
(4) in subsection (f)(1), as so redesignated, by 
striking ‘‘applicable to accelerated approval’’ and in-
serting ‘‘applicable to breakthrough therapies, accel-
erated approval, and’’; and 
(5) by adding at the end the following: 
‘‘(g) REPORT.—Beginning in fiscal year 2013, the 
Secretary shall annually prepare and submit to the Com-
mittee on Health, Education, Labor, and Pensions of the 
Senate and the Committee on Energy and Commerce of 
the House of Representatives, and make publicly available, 
with respect to this section for the previous fiscal year— 
‘‘(1) the number of drugs for which a sponsor 
requested designation as a breakthrough therapy; 
‘‘(2) the number of products designated as a 
breakthrough therapy; and 
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‘‘(3) for each product designated as a break-
through therapy, a summary of the actions taken 
under subsection (a)(3).’’. 
(b) GUIDANCE; AMENDED REGULATIONS.— 
(1) IN GENERAL.— 
(A) 
GUIDANCE.—Not 
later 
than 
months after the date of enactment of this Act, 
the Secretary of Health and Human Services 
(referred to in this section as the ‘‘Secretary’’) 
shall issue draft guidance on implementing the 
requirements with respect to breakthrough 
therapies, as set forth in section 506(a) of the 
Federal Food, Drug, and Cosmetic Act (21 
U.S.C. 356(a)), as amended by this section. 
The Secretary shall issue final guidance not 
later than 1 year after the close of the comment 
period for the draft guidance. 
(B) AMENDED REGULATIONS.— 
(i) IN GENERAL.—If the Secretary de-
termines that it is necessary to amend the 
regulations under title 21, Code of Federal 
Regulations in order to implement the 
amendments made by this section to sec-
tion 506(a) of the Federal Food, Drug, 
and Cosmetic Act, the Secretary shall 
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amend such regulations not later than 2 
years after the date of enactment of this 
Act. 
(ii) PROCEDURE.—In amending regu-
lations under clause (i), the Secretary 
shall— 
(I) issue a notice of proposed 
rulemaking that includes the proposed 
regulation; 
(II) provide a period of not less 
than 60 days for comments on the 
proposed regulation; and 
(III) publish the final regulation 
not less than 30 days before the effec-
tive date of the regulation. 
(iii) RESTRICTIONS.—Notwithstanding 
any other provision of law, the Secretary 
shall promulgate regulations implementing 
the amendments made by section only as 
described in clause (ii). 
(2) REQUIREMENTS.—Guidance issued under 
this section shall— 
(A) specify the process and criteria by 
which the Secretary makes a designation under 
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section 506(a)(3) of the Federal Food, Drug, 
and Cosmetic Act; and 
(B) specify the actions the Secretary shall 
take to expedite the development and review of 
a breakthrough therapy pursuant to such des-
ignation under such section 506(a)(3), includ-
ing updating good review management practices 
to reflect breakthrough therapies. 
(c) INDEPENDENT REVIEW.—Not later than 3 years 
after the date of enactment of this Act, the Comptroller 
General of the United States, in consultation with appro-
priate experts, shall assess the manner by which the Food 
and Drug Administration has applied the processes de-
scribed in section 506(a) of the Federal Food, Drug, and 
Cosmetic Act, as amended by this section, and the impact 
of such processes on the development and timely avail-
ability of innovative treatments for patients affected by se-
rious or life-threatening conditions. Such assessment shall 
be made publicly available upon completion. 
(d) CONFORMING AMENDMENTS.—Section 506B(e) 
(21 U.S.C. 356b) is amended by striking ‘‘section 
506(b)(2)(A)’’ each place such term appears and inserting 
‘‘section 506(c)(2)(A)’’. 
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SEC. 870. GRANTS AND CONTRACTS FOR THE DEVELOP-
MENT OF ORPHAN DRUGS. 
(a) QUALIFIED
TESTING
DEFINITION.—Section 
5(b)(1)(A)(ii) of the Orphan Drug Act (21 U.S.C. 
360ee(b)(1)(A)(ii)) is amended by striking ‘‘after the date 
such drug is designated under section 526 of such Act 
and’’. 
(b) AUTHORIZATION OF APPROPRIATIONS.—Section 
5(c) of the Orphan Drug Act (21 U.S.C. 360ee(c)) is 
amended to read as follows: 
‘‘(c) AUTHORIZATION
OF
APPROPRIATIONS.—For 
grants and contracts under subsection (a), there is author-
ized to be appropriated $30,000,000 for each of fiscal 
years 2013 through 2017.’’. 
TITLE IX—DRUG SHORTAGES 
SEC. 901. DISCONTINUANCE AND INTERRUPTIONS OF MAN-
UFACTURING OF CERTAIN DRUGS. 
(a) IN GENERAL.—Section 506C (21 U.S.C. 356c) 
is amended to read as follows: 
‘‘SEC. 506C. DISCONTINUANCE AND INTERRUPTIONS OF 
MANUFACTURING OF CERTAIN DRUGS. 
‘‘(a) IN GENERAL.—A manufacturer of a drug sub-
ject to section 503(b)(1)— 
‘‘(1) that is— 
‘‘(A) life-supporting; 
‘‘(B) life-sustaining; or 
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‘‘(C) intended for use in the prevention or 
treatment of a debilitating disease or condition; 
and 
‘‘(2) that is not a radio pharmaceutical drug 
product, a product derived from human plasma pro-
tein and their recombinant analogs, or any other 
product as designated by the Secretary, 
shall notify the Secretary of a discontinuance of the manu-
facture of the drug, or an interruption of the manufacture 
of the drug that is likely to lead to a meaningful disruption 
in the manufacturer’s supply of the drug, and the reason 
for such discontinuance or interruption, in accordance 
with subsection (b). 
‘‘(b) TIMING.—A notice required by subsection (a) 
shall be submitted to the Secretary— 
‘‘(1) at least 6 months prior to the date of the 
discontinuance or interruption; or 
‘‘(2) if compliance with paragraph (1) is not 
possible, as soon as practicable. 
‘‘(c) DISTRIBUTION.—To the maximum extent prac-
ticable, the Secretary shall distribute information on the 
discontinuation or interruption of the manufacture of the 
drugs described in subsection (a) to appropriate organiza-
tions, including physician, health provider, and patient or-
ganizations, as described in section 506D. 
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‘‘(d) CONFIDENTIALITY.—Nothing in this section 
shall be construed as authorizing the Secretary to disclose 
any information that is a trade secret or confidential infor-
mation subject to section 552(b)(4) of title 5, United 
States Code, or section 1905 of title 18, United States 
Code. 
‘‘(e) COORDINATION WITH ATTORNEY GENERAL.— 
Not later than 30 days after the receipt of a notification 
described in subsection (a), the Secretary shall— 
‘‘(1) determine whether the notification pertains 
to a controlled substance subject to a production 
quota under section 306 of the Controlled Sub-
stances Act; and 
‘‘(2) if necessary, as determined by the Sec-
retary— 
‘‘(A) notify the Attorney General that the 
Secretary has received such a notification; 
‘‘(B) request that the Attorney General in-
crease the aggregate and individual production 
quotas under section 306 of the Controlled Sub-
stances Act applicable to such controlled sub-
stance and any ingredient therein to a level the 
Secretary deems necessary to address a short-
age of a controlled substance based on the best 
available market data; and 
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‘‘(C) if the Attorney General determines 
that the level requested is not necessary to ad-
dress a shortage of a controlled substance, the 
Attorney General shall provide to the Secretary 
a written response detailing the basis for the 
Attorney General’s determination. 
The Secretary shall make the written response pro-
vided under subparagraph (C) available to the public 
on the Web site of the Food and Drug Administra-
tion. 
‘‘(f) FAILURE TO MEET REQUIREMENTS.—If a per-
son fails to submit information required under subsection 
(a) in accordance with subsection (b)— 
‘‘(1) the Secretary shall issue a letter to such 
person informing such person of such failure; 
‘‘(2) not later than 30 calendar days after the 
issuance of a letter under paragraph (1), the person 
who receives such letter shall submit to the Sec-
retary a written response to such letter setting forth 
the basis for noncompliance and providing informa-
tion required under subsection (a); and 
‘‘(3) not later than 45 calendar days after the 
issuance of a letter under paragraph (1), the Sec-
retary shall make such letter and any response to 
such letter under paragraph (2) available to the pub-
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lic on the Web site of the Food and Drug Adminis-
tration, with appropriate redactions made to protect 
information described in subsection (d), except that, 
if the Secretary determines that the letter under 
paragraph (1) was issued in error or, after review of 
such response, the person had a reasonable basis for 
not notifying as required under subsection (a), the 
requirements of this paragraph shall not apply.’’. 
(b) REGULATIONS.— 
(1) IN GENERAL.—Not later than 18 months 
after the date of the enactment of this Act, the Sec-
retary of Health and Human Services, after issuing 
a notice of proposed rule and holding a public hear-
ing, shall promulgate final regulations that imple-
ment the amendment made by subsection (a). 
(2) CONTENTS.—Such regulations shall, for 
purposes of section 506C of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 356c)— 
(A) define the terms ‘‘life-supporting’’, 
‘‘life-sustaining’’, and ‘‘intended for use in the 
prevention or treatment of a debilitating disease 
or condition’’; and 
(B) define the term ‘‘interruption of the 
manufacture of the drug that is likely to lead 
to a meaningful disruption in the manufactur-
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er’s supply of the drug’’ to mean a change in 
production that is highly likely to lead to more 
than a negligible reduction in the supply of the 
drug and affects the ability of the manufacturer 
to meet demand for such drug, but not to in-
clude a change in production due to matters 
such as routine maintenance or insignificant 
changes in manufacturing so long as the manu-
facturer expects to resume operations in a short 
period of time. 
SEC. 902. DRUG SHORTAGE LIST. 
Title V (21 U.S.C. 351 et seq.) is amended by insert-
ing after section 506C the following new section: 
‘‘SEC. 506D. DRUG SHORTAGE LIST. 
‘‘(a) ESTABLISHMENT.—The Secretary shall main-
tain an up-to-date list of drugs that are determined by 
the Secretary to be in shortage in the United States. 
‘‘(b) CONTENTS.—For each drug on such list, the 
Secretary shall include the following information: 
‘‘(1) The name of the drug in shortage. 
‘‘(2) The name of each manufacturer of such 
drug. 
‘‘(3) The reason for the shortage, as determined 
by the Secretary, selecting from the following cat-
egories: 
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‘‘(A) Requirements related to complying 
with good manufacturing practices. 
‘‘(B) Regulatory delay. 
‘‘(C) Shortage of an active ingredient. 
‘‘(D) Shortage of an inactive ingredient 
component. 
‘‘(E) Discontinuation of the manufacture 
of the drug. 
‘‘(F) Delay in shipping of the drug. 
‘‘(G) Demand increase for the drug. 
‘‘(4) The estimated duration of the shortage as 
determined by the Secretary. 
‘‘(c) PUBLIC AVAILABILITY.— 
‘‘(1) IN GENERAL.—Subject to paragraphs (2) 
and (3), the Secretary shall make the information in 
such list publicly available. 
‘‘(2) TRADE SECRETS AND CONFIDENTIAL IN-
FORMATION.—Nothing in this section alters or 
amends section 1905 of title 18, United States Code, 
or section 552(b)(4) of title 5 of such Code. 
‘‘(3) PUBLIC HEALTH EXCEPTION.—The Sec-
retary may choose not to make information collected 
under this section publicly available under paragraph 
(1) if the Secretary determines that disclosure of 
such information would adversely affect the public 
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health (such as by increasing the possibility of 
hoarding or other disruption of the availability of 
drug products to patients).’’. 
SEC. 903. QUOTAS APPLICABLE TO DRUGS IN SHORTAGE. 
Section 306 of the Controlled Substances Act (21 
U.S.C. 826) is amended by adding at the end the fol-
lowing: 
‘‘(h)(1) Not later than 30 days after the receipt of 
a request described in paragraph (2), the Attorney Gen-
eral shall— 
‘‘(A) complete review of such request; and 
‘‘(B)(i) as necessary to address a shortage of a 
controlled substance, increase the aggregate and in-
dividual production quotas under this section appli-
cable to such controlled substance and any ingre-
dient therein to the level requested; or 
‘‘(ii) if the Attorney General determines that 
the level requested is not necessary to address a 
shortage of a controlled substance, the Attorney 
General shall provide a written response detailing 
the basis for the Attorney General’s determination. 
The Secretary shall make the written response pro-
vided under subparagraph (B)(ii) available to the 
public on the Web site of the Food and Drug Ad-
ministration. 
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‘‘(2) A request is described in this paragraph if— 
‘‘(A) the request pertains to a controlled sub-
stance on the list of drugs in shortage maintained 
under section 506D of the Federal Food, Drug, and 
Cosmetic Act; 
‘‘(B) the request is submitted by the manufac-
turer of the controlled substance; and 
‘‘(C) the controlled substance is in schedule 
II.’’. 
SEC. 904. EXPEDITED REVIEW OF MAJOR MANUFACTURING 
CHANGES FOR POTENTIAL AND VERIFIED 
SHORTAGES OF DRUGS THAT ARE LIFE-SUP-
PORTING, LIFE-SUSTAINING, OR INTENDED 
FOR USE IN THE PREVENTION OF A DEBILI-
TATING DISEASE OR CONDITION. 
Subsection (c) of section 506A (21 U.S.C. 356a) is 
amended by adding at the end the following new para-
graph: 
‘‘(3) CHANGES
ADDRESSING
A
DRUG
SHORT-
AGE.— 
‘‘(A) CERTIFICATION.— 
‘‘(i) DESCRIPTION.—A certification is 
described in this subparagraph if the man-
ufacturer, having notified the Secretary of 
an interruption or discontinuance of a drug 
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in accordance with Section 506C, certifies 
(in such certification) that the major man-
ufacturing change for which approval is 
being sought may prevent or alleviate a 
discontinuance or interruption of such 
drug. 
‘‘(ii) BAD
FAITH
EXCEPTION.—Sub-
paragraphs (B) and (C) do not apply in 
the case of a certification which the Sec-
retary determines to be made in bad faith. 
‘‘(B) EXPEDITED
REVIEW.—If a certifi-
cation described in subparagraph (A) is sub-
mitted in connection with a supplemental appli-
cation for a major manufacturing change, the 
Secretary shall— 
‘‘(i) expedite any technical review or 
inspection necessary for consideration of 
the supplemental application; 
‘‘(ii) provide any technical assistance 
necessary to facilitate approval of the sup-
plemental application; and 
‘‘(iii) not later than 60 days after re-
ceipt of the certification, complete review 
of the supplemental application.’’. 
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