I 
112TH CONGRESS 
1ST SESSION H. R. 3059 
To amend the Federal Food, Drug, and Cosmetic Act to improve the priority 
review voucher incentive program relating to tropical and rare pediatric 
diseases. 
IN THE HOUSE OF REPRESENTATIVES 
SEPTEMBER 23, 2011 
Mr. MCCAUL (for himself, Mr. BUTTERFIELD, Mrs. MYRICK, Mr. VAN HOL-
LEN, Mr. BURGESS, Ms. SPEIER, Mr. KELLY, Mr. JOHNSON of Georgia, 
Mr. DAVIS of Illinois, Mr. TOWNS, Mrs. CHRISTENSEN, Mr. RUSH, Mr. 
CUELLAR, Mr. BILBRAY, Mr. WOLF, Mrs. MCMORRIS RODGERS, Mr. 
KEATING, Mr. OLSON, Mr. CANSECO, Mr. ROGERS of Alabama, Mr. BOU-
STANY, Mr. DAVIS of Kentucky, Ms. ROS-LEHTINEN, Ms. PELOSI, and 
Mr. ROTHMAN of New Jersey) introduced the following bill; which was 
referred to the Committee on Energy and Commerce 
A BILL 
To amend the Federal Food, Drug, and Cosmetic Act to 
improve the priority review voucher incentive program 
relating to tropical and rare pediatric diseases. 
Be it enacted by the Senate and House of Representa-
tives of the United States of America in Congress assembled, 
SECTION 1. SHORT TITLE; REFERENCES. 
(a) SHORT TITLE.—This Act may be cited as the 
‘‘Creating Hope Act of 2011’’. 
VerDate Mar 15 2010 
04:19 Sep 28, 2011
Jkt 099200
PO 00000
Frm 00001
Fmt 6652
Sfmt 6201
E:\BILLS\H3059.IH
H3059
jbell on DSK7SPTVN1PROD with BILLS
(b) REFERENCES.—Wherever in this Act an amend-
ment is expressed in terms of an amendment to a section 
or other provision, the reference shall be considered to be 
made to a section or other provision of the Federal Food, 
Drug, and Cosmetic Act (21 U.S.C. 301 et seq.). 
SEC. 2. IMPROVEMENT OF THE TROPICAL DISEASE VOUCH-
ER PROGRAM. 
(a) HEADING.—The heading of section 524 (21 
U.S.C. 360n) is amended to read as follows: ‘‘PRIORITY 
REVIEW TO ENCOURAGE TREATMENTS FOR TROP-
ICAL DISEASES AND RARE PEDIATRIC DISEASES’’. 
(b) DEFINITIONS.—Section 524(a) (21 U.S.C. 
360n(a)) is amended— 
(1) by redesignating paragraphs (3) and (4) as 
paragraphs (6) and (7), respectively; 
(2) by redesignating paragraphs (1) and (2) as 
paragraphs (2) and (3), respectively; 
(3) by inserting after ‘‘In this section:’’, the fol-
lowing: 
‘‘(1) ELIGIBLE TREATMENT.—The term ‘eligi-
ble treatment’ means a new drug, including a bio-
logical product that is a new drug, that is the sub-
ject of an application submitted under section 
505(b)(1) of this Act or section 351(a) of the Public 
Health Service Act, if that drug contains no active 
VerDate Mar 15 2010 
04:19 Sep 28, 2011
Jkt 099200
PO 00000
Frm 00002
Fmt 6652
Sfmt 6201
E:\BILLS\H3059.IH
H3059
jbell on DSK7SPTVN1PROD with BILLS
ingredient (including any ester or salt of the active 
ingredient) that has been previously approved in any 
other application under section 505(b)(1), 505(b)(2), 
or 505(j) of this Act or section 351(a) or 351(k) of 
the Public Health Service Act.’’; 
(4) in paragraph (3), as so redesignated, by in-
serting ‘‘or rare pediatric disease product applica-
tion’’ after ‘‘tropical disease product application’’ 
each place that phrase appears; 
(5) by inserting after paragraph (3) the fol-
lowing: 
‘‘(4) RARE
PEDIATRIC
DISEASE.—The term 
‘rare pediatric disease’ means a disease that meets 
each of the following criteria: 
‘‘(A) The disease primarily affects individ-
uals aged from birth to 18 years, including age 
groups often called neonates, infants, children, 
and adolescents. 
‘‘(B) The disease is a rare disease or con-
dition, within the meaning of section 526. 
‘‘(5) RARE PEDIATRIC DISEASE PRODUCT AP-
PLICATION.—The term ‘rare pediatric disease prod-
uct application’ means a human drug application, as 
defined in section 735(1)— 
VerDate Mar 15 2010 
04:19 Sep 28, 2011
Jkt 099200
PO 00000
Frm 00003
Fmt 6652
Sfmt 6201
E:\BILLS\H3059.IH
H3059
jbell on DSK7SPTVN1PROD with BILLS
‘‘(A) for prevention or treatment of a rare 
pediatric disease; 
‘‘(B) that the Secretary deems eligible for 
priority review; 
‘‘(C) that is for an eligible treatment; 
‘‘(D) that relies on clinical data derived 
from studies examining a pediatric population 
and dosages of the drug intended for that popu-
lation; and 
‘‘(E) that does not seek approval for an 
adult indication in the original rare pediatric 
disease product application.’’; 
(6) in paragraph (6), as so redesignated— 
(A) by redesignating subparagraph (Q) as 
subparagraph (R); and 
(B) by inserting after subparagraph (P) 
the following: 
‘‘(Q) Chagas Disease.’’; and 
(7) by amending paragraph (7), as so redesig-
nated, to read as follows: 
‘‘(7) TROPICAL
DISEASE
PRODUCT
APPLICA-
TION.—The term ‘tropical disease product applica-
tion’ means a human drug application, as defined in 
section 735(1)— 
VerDate Mar 15 2010 
04:19 Sep 28, 2011
Jkt 099200
PO 00000
Frm 00004
Fmt 6652
Sfmt 6201
E:\BILLS\H3059.IH
H3059
jbell on DSK7SPTVN1PROD with BILLS
‘‘(A) for prevention or treatment of a trop-
ical disease; 
‘‘(B) that the Secretary deems eligible for 
priority review; 
‘‘(C) that is for an eligible treatment; and 
‘‘(D) that the sponsor affirms in the appli-
cation is for a drug that has not been approved 
for commercial marketing for any tropical dis-
ease indication by a government authority out-
side of the United States for more than 24 
months before the tropical disease product ap-
plication is submitted.’’. 
(c) RULES REGARDING USE AND TRANSFER OF PRI-
ORITY REVIEW VOUCHERS.—Section 524(b) (21 U.S.C. 
360n(b)) is amended— 
(1) in paragraph (1), by inserting ‘‘or rare pe-
diatric disease product application’’ after ‘‘tropical 
disease product application’’ each place that phrase 
appears; 
(2) by amending paragraph (2) to read as fol-
lows: 
‘‘(2) TRANSFERABILITY.— 
‘‘(A) IN GENERAL.—The sponsor of a trop-
ical disease product application or rare pediatric 
disease product application that receives a pri-
VerDate Mar 15 2010 
04:19 Sep 28, 2011
Jkt 099200
PO 00000
Frm 00005
Fmt 6652
Sfmt 6201
E:\BILLS\H3059.IH
H3059
jbell on DSK7SPTVN1PROD with BILLS
ority review voucher under this section may 
transfer (including by sale) the entitlement to 
such voucher. There is no limit on the number 
of times a priority review voucher may be trans-
ferred before such voucher is used. 
‘‘(B) CONDITIONS
OF
TRANSFER.—If a 
sponsor transfers a priority review voucher 
after such sponsor has provided notification to 
the Secretary under paragraph (4)(A) of the in-
tent of such sponsor to use the voucher, the 
transfer shall be subject to the provisions of 
subparagraphs (B) and (C) of paragraph (4). 
‘‘(C) NOTIFICATION OF TRANSFER.—The 
person to whom a voucher is transferred under 
paragraph (4)(B)(i) shall notify the Secretary 
of such change in ownership of the voucher not 
later than 30 days after such transfer.’’; 
(3) by amending paragraph (3) to read as fol-
lows: 
‘‘(3) LIMITATION FOR PRIOR APPLICATIONS.— 
‘‘(A) TROPICAL DISEASE PRODUCT APPLI-
CATIONS.—A sponsor of a tropical disease prod-
uct application may not receive a priority review 
voucher under this section if the tropical dis-
VerDate Mar 15 2010 
04:19 Sep 28, 2011
Jkt 099200
PO 00000
Frm 00006
Fmt 6652
Sfmt 6201
E:\BILLS\H3059.IH
H3059
jbell on DSK7SPTVN1PROD with BILLS
in the drug application that was the sub-
ject of the notification of such sponsor, the 
sponsor shall notify the Secretary of the 
transfer in accordance with this subsection. 
‘‘(C) FEE DUE UPON NOTIFICATION; CRED-
IT FOR TRANSFERRED VOUCHER.— 
‘‘(i) DUE UPON NOTIFICATION.—The 
notification under this subsection shall be 
a legally binding commitment to pay for 
the user fee to be assessed in accordance 
with this section. Such fee shall be payable 
by the sponsor upon the submission by 
such sponsor of such notification. 
‘‘(ii) CREDIT.—If a sponsor pays a 
user fee upon providing notification of the 
intent of such sponsor to use a priority re-
view voucher, but later transfers the vouch-
er for which such sponsor gave notifica-
tion, the Secretary shall credit the user 
fees paid to the next human drug applica-
tion for which a sponsor provides notifica-
tion of the intent of such sponsor to use 
the same transferred voucher. 
‘‘(iii) DIFFERENCE IN FEE.—The Sec-
retary may require a sponsor using a 
VerDate Mar 15 2010 
04:19 Sep 28, 2011
Jkt 099200
PO 00000
Frm 00009
Fmt 6652
Sfmt 6201
E:\BILLS\H3059.IH
H3059
jbell on DSK7SPTVN1PROD with BILLS
transferred voucher to pay the difference 
between the credit associated with the 
transferred voucher and the user fee pre-
vailing at the time the sponsor submits no-
tification of the intent of such sponsor to 
use the transferred voucher. This provision 
does not apply in cases where a transferee 
is exempted from submitting notification 
under this paragraph.’’. 
(d) 
PAYMENT.—Section 
524(c)(4) 
(21 
U.S.C. 
360n(c)(4)) is amended— 
(1) in subparagraph (A), by striking ‘‘submis-
sion of a human drug application under section 
505(b)(1) or section 351 of the Public Health Serv-
ices Act for which the priority review voucher is 
used.’’ and inserting ‘‘notification by a sponsor of 
the intent of such sponsor to use the voucher, as 
specified in subsection (b)(4)(A). All other user fees 
associated with the human drug application shall be 
due as required by the Secretary or under applicable 
law.’’; and 
(2) in subparagraph (C), by striking the period 
at the end and inserting ‘‘, except as specified in 
subsection (b)(4)(C).’’. 
VerDate Mar 15 2010 
04:19 Sep 28, 2011
Jkt 099200
PO 00000
Frm 00010
Fmt 6652
Sfmt 6201
E:\BILLS\H3059.IH
H3059
jbell on DSK7SPTVN1PROD with BILLS
(e) DESIGNATION PROCESS; PRODUCT IMPLEMENTA-
TION REQUIREMENT.—Section 524 (21 U.S.C. 360n) is 
amended by adding at the end the following new sub-
sections: 
‘‘(d) DESIGNATION PROCESS.— 
‘‘(1) DESIGNATION
OF
RARE
PEDIATRIC
DIS-
EASES.— 
‘‘(A) IN GENERAL.—Upon the request of 
the manufacturer or the sponsor of a new drug, 
the Secretary may designate that the new drug 
is for a rare pediatric disease. Such a request 
for designation, if sought, shall be made when 
requesting designation of orphan disease status 
under section 526 or fast-track designation 
under section 506. Requesting designation of 
rare pediatric disease status under this para-
graph is not a prerequisite to receiving a pri-
ority review voucher. 
‘‘(B) DETERMINATION
BY
SECRETARY.— 
Not later than 60 days after a request is sub-
mitted under subparagraph (A), the Secretary 
shall determine whether the disease or condition 
that is the subject of such request is a rare pe-
diatric disease. 
VerDate Mar 15 2010 
04:19 Sep 28, 2011
Jkt 099200
PO 00000
Frm 00011
Fmt 6652
Sfmt 6201
E:\BILLS\H3059.IH
H3059
jbell on DSK7SPTVN1PROD with BILLS
‘‘(2) 
DESIGNATION
OF
ELIGIBLE
TREAT-
MENTS.— 
‘‘(A) IN GENERAL.—Upon the request of 
the manufacturer or the sponsor of a new drug, 
the Secretary may designate that a new drug is 
an eligible treatment. Such a request for des-
ignation, if sought, shall be made when request-
ing fast-track designation under section 506. 
Requesting designation that a new drug is an 
eligible treatment is not a prerequisite to receiv-
ing a priority review voucher. 
‘‘(B) DETERMINATION
BY
SECRETARY.— 
Not later than 60 days after a request is sub-
mitted under subparagraph (A), the Secretary 
shall determine whether the new drug that is 
the subject of such request is an eligible treat-
ment. 
‘‘(e) PRODUCT IMPLEMENTATION FOR RARE PEDI-
ATRIC DISEASE PRODUCTS.— 
‘‘(1) IN GENERAL.—The Secretary shall deem a 
rare pediatric disease product application incomplete 
if such application does not contain a description of 
the plan of the sponsor of such application to mar-
ket the product in the United States. 
‘‘(2) GOOD FAITH INTENT TO MARKET.— 
VerDate Mar 15 2010 
04:19 Sep 28, 2011
Jkt 099200
PO 00000
Frm 00012
Fmt 6652
Sfmt 6201
E:\BILLS\H3059.IH
H3059
jbell on DSK7SPTVN1PROD with BILLS
‘‘(A) GOOD
FAITH
INTENT.—The Sec-
retary may refuse to issue a priority review 
voucher upon the approval of a rare pediatric 
disease product application if the Secretary 
finds that the sponsor of such application lacks 
a good faith intention to market the product in 
the United States. The Secretary may consider 
any fact relevant to this determination, includ-
ing the history of such sponsor of producing 
rare pediatric disease products for which such 
sponsor received a priority review voucher, or-
phan drugs for which the sponsor received ex-
clusivity under section 527, or pediatric drugs 
for which the sponsor received an additional 6 
months of exclusivity under section 505A. 
‘‘(B) PRESUMPTION.—The sponsor may 
establish a presumption of good faith by dem-
onstrating that such sponsor has allocated suffi-
cient resources or otherwise arranged for the 
production (by the sponsor or by another manu-
facturer) of the rare pediatric disease product 
in a manner sufficient to meet the expected de-
mand for the product during the 5-year period 
following approval of the application. 
VerDate Mar 15 2010 
04:19 Sep 28, 2011
Jkt 099200
PO 00000
Frm 00013
Fmt 6652
Sfmt 6201
E:\BILLS\H3059.IH
H3059
jbell on DSK7SPTVN1PROD with BILLS
‘‘(C) GUIDANCE.—If the Secretary re-
quires sponsors seeking a priority review vouch-
er to demonstrate a good faith intent to market 
the rare pediatric disease product in the United 
States, the Secretary shall first issue a guid-
ance document setting forth the required evi-
dentiary support necessary to demonstrate such 
a good faith intent. 
‘‘(3) POSTAPPROVAL PRODUCTION REPORT.— 
‘‘(A) REPORT REQUIRED.—The sponsor of 
an approved rare pediatric disease product shall 
submit a report to the Secretary not later than 
5 years after the approval of the applicable rare 
pediatric disease product application. Such re-
port shall provide the following information, 
with respect to each of the first 4 years after 
approval of such product: 
‘‘(i) The estimated population in the 
United States suffering from the rare pedi-
atric disease. 
‘‘(ii) The estimated demand in the 
United States for such rare pediatric dis-
ease product. 
VerDate Mar 15 2010 
04:19 Sep 28, 2011
Jkt 099200
PO 00000
Frm 00014
Fmt 6652
Sfmt 6201
E:\BILLS\H3059.IH
H3059
jbell on DSK7SPTVN1PROD with BILLS