I 
112TH CONGRESS 
2D SESSION 
H. R. 5334 
To amend chapter V of the Federal Food, Drug, and Cosmetic Act to 
expedite the development and review of breakthrough therapies. 
IN THE HOUSE OF REPRESENTATIVES 
MAY 7, 2012 
Mr. BILBRAY (for himself and Ms. DEGETTE) introduced the following bill; 
which was referred to the Committee on Energy and Commerce 
A BILL 
To amend chapter V of the Federal Food, Drug, and Cos-
metic Act to expedite the development and review of 
breakthrough therapies. 
Be it enacted by the Senate and House of Representa-
tives of the United States of America in Congress assembled, 
SECTION 1. BREAKTHROUGH THERAPIES. 
(a) IN GENERAL.—Section 506 (21 U.S.C. 356) is 
amended— 
(1) by redesignating subsection (d) as sub-
section (f); 
(2) by redesignating subsections (a) through (c) 
as subsections (b) through (d), respectively; 
VerDate Mar 15 2010 
06:46 May 09, 2012
Jkt 019200
PO 00000
Frm 00001
Fmt 6652
Sfmt 6201
E:\BILLS\H5334.IH
H5334
rfrederick on DSK6VPTVN1PROD with BILLS
(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 
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.— 
VerDate Mar 15 2010 
06:46 May 09, 2012
Jkt 019200
PO 00000
Frm 00002
Fmt 6652
Sfmt 6201
E:\BILLS\H5334.IH
H5334
rfrederick on DSK6VPTVN1PROD with BILLS
‘‘(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 
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; 
VerDate Mar 15 2010 
06:46 May 09, 2012
Jkt 019200
PO 00000
Frm 00003
Fmt 6652
Sfmt 6201
E:\BILLS\H5334.IH
H5334
rfrederick on DSK6VPTVN1PROD with BILLS
‘‘(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-
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 
VerDate Mar 15 2010 
06:46 May 09, 2012
Jkt 019200
PO 00000
Frm 00004
Fmt 6652
Sfmt 6201
E:\BILLS\H5334.IH
H5334
rfrederick on DSK6VPTVN1PROD with BILLS
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; 
and 
‘‘(2) the number of products designated as a 
breakthrough therapy.’’. 
(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 
VerDate Mar 15 2010 
06:46 May 09, 2012
Jkt 019200
PO 00000
Frm 00005
Fmt 6652
Sfmt 6201
E:\BILLS\H5334.IH
H5334
rfrederick on DSK6VPTVN1PROD with BILLS
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 
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 this section only 
as described in clause (ii). 
VerDate Mar 15 2010 
06:46 May 09, 2012
Jkt 019200
PO 00000
Frm 00006
Fmt 6652
Sfmt 6201
E:\BILLS\H5334.IH
H5334
rfrederick on DSK6VPTVN1PROD with BILLS
(2) REQUIREMENTS.—Guidance issued under 
this section shall— 
(A) specify the process and criteria by 
which the Secretary makes a designation under 
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 
VerDate Mar 15 2010 
06:46 May 09, 2012
Jkt 019200
PO 00000
Frm 00007
Fmt 6652
Sfmt 6201
E:\BILLS\H5334.IH
H5334
rfrederick on DSK6VPTVN1PROD with BILLS
506(b)(2)(A)’’ each place such term appears and inserting 
‘‘section 506(c)(2)(A)’’. 
Æ 
VerDate Mar 15 2010 
06:46 May 09, 2012
Jkt 019200
PO 00000
Frm 00008
Fmt 6652
Sfmt 6301
E:\BILLS\H5334.IH
H5334
rfrederick on DSK6VPTVN1PROD with BILLS