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 HOLLEN, 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. BOUSTANY, 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. 

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled, 

3 SECTION 1. SHORT TITLE; REFERENCES. 

4 (a) SHORT TITLE.—This Act may be cited as the 

5 ‘‘Creating Hope Act of 2011’’. 


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1 (b) REFERENCES.—Wherever in this Act an amend2 ment is expressed in terms of an amendment to a section 

3 or other provision, the reference shall be considered to be 

4 made to a section or other provision of the Federal Food, 

5 Drug, and Cosmetic Act (21 U.S.C. 301 et seq.). 

6 SEC. 2. IMPROVEMENT OF THE TROPICAL DISEASE VOUCH7 ER PROGRAM. 

8 (a) HEADING.—The heading of section 524 (21 

9 U.S.C. 360n) is amended to read as follows: ‘‘PRIORITY

10 REVIEW TO ENCOURAGE TREATMENTS FOR TROP11 ICAL DISEASES AND RARE PEDIATRIC DISEASES’’. 

12 (b) DEFINITIONS.—Section 524(a) (21 U.S.C. 

13 360n(a)) is amended— 

14 (1) by redesignating paragraphs (3) and (4) as 

15 paragraphs (6) and (7), respectively; 

16 (2) by redesignating paragraphs (1) and (2) as 

17 paragraphs (2) and (3), respectively; 

18 (3) by inserting after ‘‘In this section:’’, the fol19 lowing: 

20 ‘‘(1) ELIGIBLE TREATMENT.—The term ‘eligi21 ble treatment’ means a new drug, including a bio22 logical product that is a new drug, that is the sub23 ject of an application submitted under section 

24 505(b)(1) of this Act or section 351(a) of the Public 

25 Health Service Act, if that drug contains no active 


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1 ingredient (including any ester or salt of the active 

2 ingredient) that has been previously approved in any 

3 other application under section 505(b)(1), 505(b)(2), 

4 or 505(j) of this Act or section 351(a) or 351(k) of 

5 the Public Health Service Act.’’; 

6 (4) in paragraph (3), as so redesignated, by in7 serting ‘‘or rare pediatric disease product applica8 tion’’ after ‘‘tropical disease product application’’ 

9 each place that phrase appears; 

10 (5) by inserting after paragraph (3) the fol11 lowing: 

12 ‘‘(4) RARE PEDIATRIC DISEASE.—The term 

13 ‘rare pediatric disease’ means a disease that meets 

14 each of the following criteria: 

15 ‘‘(A) The disease primarily affects individ16 uals aged from birth to 18 years, including age 

17 groups often called neonates, infants, children, 

18 and adolescents. 

19 ‘‘(B) The disease is a rare disease or con20 dition, within the meaning of section 526. 

21 ‘‘(5) RARE PEDIATRIC DISEASE PRODUCT AP22 PLICATION.—The term ‘rare pediatric disease prod23 uct application’ means a human drug application, as 

24 defined in section 735(1)— 


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1 ‘‘(A) for prevention or treatment of a rare 

2 pediatric disease; 

3 ‘‘(B) that the Secretary deems eligible for 

4 priority review; 

5 ‘‘(C) that is for an eligible treatment; 

6 ‘‘(D) that relies on clinical data derived 

7 from studies examining a pediatric population 

8 and dosages of the drug intended for that popu9 lation; and 

10 ‘‘(E) that does not seek approval for an 

11 adult indication in the original rare pediatric 

12 disease product application.’’; 

13 (6) in paragraph (6), as so redesignated— 

14 (A) by redesignating subparagraph (Q) as 

15 subparagraph (R); and 

16 (B) by inserting after subparagraph (P) 

17 the following: 

18 ‘‘(Q) Chagas Disease.’’; and 

19 (7) by amending paragraph (7), as so redesig20 nated, to read as follows: 

21 ‘‘(7) TROPICAL DISEASE PRODUCT APPLICA22 TION.—The term ‘tropical disease product applica23 tion’ means a human drug application, as defined in 

24 section 735(1)— 


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1 ‘‘(A) for prevention or treatment of a trop2 ical disease; 

3 ‘‘(B) that the Secretary deems eligible for 

4 priority review; 

5 ‘‘(C) that is for an eligible treatment; and 

6 ‘‘(D) that the sponsor affirms in the appli7 cation is for a drug that has not been approved 

8 for commercial marketing for any tropical dis9 ease indication by a government authority out10 side of the United States for more than 24 

11 months before the tropical disease product ap12 plication is submitted.’’. 

13 (c) RULES REGARDING USE AND TRANSFER OF PRI14 ORITY REVIEW VOUCHERS.—Section 524(b) (21 U.S.C. 

15 360n(b)) is amended— 

16 (1) in paragraph (1), by inserting ‘‘or rare pe17 diatric disease product application’’ after ‘‘tropical 

18 disease product application’’ each place that phrase 

19 appears; 

20 (2) by amending paragraph (2) to read as fol21 lows: 

22 ‘‘(2) TRANSFERABILITY.— 

23 ‘‘(A) IN GENERAL.—The sponsor of a trop24 ical disease product application or rare pediatric 

25 disease product application that receives a priVerDate Mar 15 2010 04:19 Sep 28, 2011 Jkt 099200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\H3059.IH H3059

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1 ority review voucher under this section may 

2 transfer (including by sale) the entitlement to 

3 such voucher. There is no limit on the number 

4 of times a priority review voucher may be trans5 ferred before such voucher is used. 

6 ‘‘(B) CONDITIONS OF TRANSFER.—If a 

7 sponsor transfers a priority review voucher 

8 after such sponsor has provided notification to 

9 the Secretary under paragraph (4)(A) of the in10 tent of such sponsor to use the voucher, the 

11 transfer shall be subject to the provisions of 

12 subparagraphs (B) and (C) of paragraph (4). 

13 ‘‘(C) NOTIFICATION OF TRANSFER.—The 

14 person to whom a voucher is transferred under 

15 paragraph (4)(B)(i) shall notify the Secretary 

16 of such change in ownership of the voucher not 

17 later than 30 days after such transfer.’’; 

18 (3) by amending paragraph (3) to read as fol19 lows: 

20 ‘‘(3) LIMITATION FOR PRIOR APPLICATIONS.— 

21 ‘‘(A) TROPICAL DISEASE PRODUCT APPLI22 CATIONS.—A sponsor of a tropical disease prod23 uct application may not receive a priority review 

24 voucher under this section if the tropical disVerDate Mar 15 2010 04:19 Sep 28, 2011 Jkt 099200 PO 00000 Frm 00006 Fmt 6652 Sfmt 6201 E:\BILLS\H3059.IH H3059

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1 ease product application was submitted to the 

2 Secretary prior to September 27, 2007. 

3 ‘‘(B) RARE PEDIATRIC DISEASE PRODUCT

4 APPLICATIONS.—A sponsor of a rare pediatric 

5 disease product application may not receive a 

6 priority review voucher under this section if the 

7 rare pediatric disease product application was 

8 submitted to the Secretary prior to the date 

9 that is 90 days after the date of enactment of 

10 the Creating Hope Act of 2011.’’; and 

11 (4) by amending paragraph (4) to read as fol12 lows: 

13 ‘‘(4) NOTIFICATION.— 

14 ‘‘(A) TIMING.—At least 90 days before the 

15 date on which a human drug application for 

16 which the sponsor intends to use a priority re17 view voucher is submitted, the sponsor of such 

18 human drug application shall notify the Sec19 retary of the intent of such sponsor to submit 

20 the human drug application. 

21 ‘‘(B) TRANSFER OF VOUCHER AFTER NO22 TIFICATION.— 

23 ‘‘(i) IN GENERAL.—The sponsor of a 

24 human drug application that provides noti25 fication of the intent of such sponsor to 


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1 use the voucher for the human drug appli2 cation may transfer the voucher after such 

3 notification is provided, if such sponsor has 

4 not yet submitted the human drug applica5 tion described in the notification. 

6 ‘‘(ii) EXCEPTION.—The person to 

7 whom a voucher is transferred under 

8 clause (i) (referred to in this paragraph as 

9 the ‘transferee’) shall give notification of 

10 the intent of such transferee to use the 

11 voucher in accordance with this subsection, 

12 unless— 

13 ‘‘(I) the transferee uses the 

14 voucher for a human drug application 

15 including the same indications as the 

16 human drug application described in 

17 the transferor’s notification; and 

18 ‘‘(II) the transferee notifies the 

19 Secretary within 30 days of the trans20 fer of the intent of such transferee to 

21 use the voucher for such purpose. 

22 ‘‘(iii) INTERNAL TRANSFER.—If the 

23 sponsor transfers a voucher internally for 

24 use with a drug application including one 

25 or more indications that were not included 


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1 in the drug application that was the sub2 ject of the notification of such sponsor, the 

3 sponsor shall notify the Secretary of the 

4 transfer in accordance with this subsection. 

5 ‘‘(C) FEE DUE UPON NOTIFICATION; CRED6 IT FOR TRANSFERRED VOUCHER.— 

7 ‘‘(i) DUE UPON NOTIFICATION.—The 

8 notification under this subsection shall be 

9 a legally binding commitment to pay for 

10 the user fee to be assessed in accordance 

11 with this section. Such fee shall be payable 

12 by the sponsor upon the submission by 

13 such sponsor of such notification. 

14 ‘‘(ii) CREDIT.—If a sponsor pays a 

15 user fee upon providing notification of the 

16 intent of such sponsor to use a priority re17 view voucher, but later transfers the vouch18 er for which such sponsor gave notifica19 tion, the Secretary shall credit the user 

20 fees paid to the next human drug applica21 tion for which a sponsor provides notifica22 tion of the intent of such sponsor to use 

23 the same transferred voucher. 

24 ‘‘(iii) DIFFERENCE IN FEE.—The Sec25 retary may require a sponsor using a 


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1 transferred voucher to pay the difference 

2 between the credit associated with the 

3 transferred voucher and the user fee pre4 vailing at the time the sponsor submits no5 tification of the intent of such sponsor to 

6 use the transferred voucher. This provision 

7 does not apply in cases where a transferee 

8 is exempted from submitting notification 

9 under this paragraph.’’. 

10 (d) PAYMENT.—Section 524(c)(4) (21 U.S.C. 

11 360n(c)(4)) is amended— 

12 (1) in subparagraph (A), by striking ‘‘submis13 sion of a human drug application under section 

14 505(b)(1) or section 351 of the Public Health Serv15 ices Act for which the priority review voucher is 

16 used.’’ and inserting ‘‘notification by a sponsor of 

17 the intent of such sponsor to use the voucher, as 

18 specified in subsection (b)(4)(A). All other user fees 

19 associated with the human drug application shall be 

20 due as required by the Secretary or under applicable 

21 law.’’; and 

22 (2) in subparagraph (C), by striking the period 

23 at the end and inserting ‘‘, except as specified in 

24 subsection (b)(4)(C).’’. 


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1 (e) DESIGNATION PROCESS; PRODUCT IMPLEMENTA2 TION REQUIREMENT.—Section 524 (21 U.S.C. 360n) is 

3 amended by adding at the end the following new sub4 sections: 

5 ‘‘(d) DESIGNATION PROCESS.— 

6 ‘‘(1) DESIGNATION OF RARE PEDIATRIC DIS7 EASES.— 

8 ‘‘(A) IN GENERAL.—Upon the request of 

9 the manufacturer or the sponsor of a new drug, 

10 the Secretary may designate that the new drug 

11 is for a rare pediatric disease. Such a request 

12 for designation, if sought, shall be made when 

13 requesting designation of orphan disease status 

14 under section 526 or fast-track designation 

15 under section 506. Requesting designation of 

16 rare pediatric disease status under this para17 graph is not a prerequisite to receiving a pri18 ority review voucher. 

19 ‘‘(B) DETERMINATION BY SECRETARY.— 

20 Not later than 60 days after a request is sub21 mitted under subparagraph (A), the Secretary 

22 shall determine whether the disease or condition 

23 that is the subject of such request is a rare pe24 diatric disease. 


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1 ‘‘(2) DESIGNATION OF ELIGIBLE TREAT2 MENTS.— 

3 ‘‘(A) IN GENERAL.—Upon the request of 

4 the manufacturer or the sponsor of a new drug, 

5 the Secretary may designate that a new drug is 

6 an eligible treatment. Such a request for des7 ignation, if sought, shall be made when request8 ing fast-track designation under section 506. 

9 Requesting designation that a new drug is an 

10 eligible treatment is not a prerequisite to receiv11 ing a priority review voucher. 

12 ‘‘(B) DETERMINATION BY SECRETARY.— 

13 Not later than 60 days after a request is sub14 mitted under subparagraph (A), the Secretary 

15 shall determine whether the new drug that is 

16 the subject of such request is an eligible treat17 ment. 

18 ‘‘(e) PRODUCT IMPLEMENTATION FOR RARE PEDI19 ATRIC DISEASE PRODUCTS.— 

20 ‘‘(1) IN GENERAL.—The Secretary shall deem a 

21 rare pediatric disease product application incomplete 

22 if such application does not contain a description of 

23 the plan of the sponsor of such application to mar24 ket the product in the United States. 

25 ‘‘(2) GOOD FAITH INTENT TO MARKET.— 


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1 ‘‘(A) GOOD FAITH INTENT.—The Sec2 retary may refuse to issue a priority review 

3 voucher upon the approval of a rare pediatric 

4 disease product application if the Secretary 

5 finds that the sponsor of such application lacks 

6 a good faith intention to market the product in 

7 the United States. The Secretary may consider 

8 any fact relevant to this determination, includ9 ing the history of such sponsor of producing 

10 rare pediatric disease products for which such 

11 sponsor received a priority review voucher, or12 phan drugs for which the sponsor received ex13 clusivity under section 527, or pediatric drugs 

14 for which the sponsor received an additional 6 

15 months of exclusivity under section 505A. 

16 ‘‘(B) PRESUMPTION.—The sponsor may 

17 establish a presumption of good faith by dem18 onstrating that such sponsor has allocated suffi19 cient resources or otherwise arranged for the 

20 production (by the sponsor or by another manu21 facturer) of the rare pediatric disease product 

22 in a manner sufficient to meet the expected de23 mand for the product during the 5-year period 

24 following approval of the application. 


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1 ‘‘(C) GUIDANCE.—If the Secretary re2 quires sponsors seeking a priority review vouch3 er to demonstrate a good faith intent to market 

4 the rare pediatric disease product in the United 

5 States, the Secretary shall first issue a guid6 ance document setting forth the required evi7 dentiary support necessary to demonstrate such 

8 a good faith intent. 

9 ‘‘(3) POSTAPPROVAL PRODUCTION REPORT.— 

10 ‘‘(A) REPORT REQUIRED.—The sponsor of 

11 an approved rare pediatric disease product shall 

12 submit a report to the Secretary not later than 

13 5 years after the approval of the applicable rare 

14 pediatric disease product application. Such re15 port shall provide the following information, 

16 with respect to each of the first 4 years after 

17 approval of such product: 

18 ‘‘(i) The estimated population in the 

19 United States suffering from the rare pedi20 atric disease. 

21 ‘‘(ii) The estimated demand in the 

22 United States for such rare pediatric dis23 ease product. 


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1 ‘‘(iii) The actual amount of such rare 

2 pediatric disease product distributed in the 

3 United States. 

4 ‘‘(B) PUBLICATION UPON FAILURE TO

5 DEMONSTRATE GOOD FAITH EFFORT TO MAR6 KET.—The Secretary may publish the results of 

7 a report submitted under subparagraph (A) in 

8 the Federal Register if the Secretary finds that 

9 the sponsor that submitted such report has not 

10 made a good faith effort to meet the demand in 

11 the United States for the product that is the 

12 subject of such report during each of the first 

13 4 years after approval of such product. 

14 ‘‘(f) PRODUCTION REPORT FOR TROPICAL DISEASE

15 PRODUCTS.— 

16 ‘‘(1) REPORT REQUIRED.—The sponsor of an 

17 approved tropical disease product shall submit a re18 port to the Secretary not later than 5 years after the 

19 approval of the applicable rare tropical disease prod20 uct application. Such report shall provide the fol21 lowing information, with respect to each of the first 

22 4 years after approval of such product: 

23 ‘‘(A) The estimated global population suf24 fering from the tropical disease. 


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1 ‘‘(B) The estimated global demand for 

2 such tropical disease product. 

3 ‘‘(C) The actual amount of such tropical 

4 disease product distributed globally. 

5 ‘‘(2) PUBLICATION UPON FAILURE TO DEM6 ONSTRATE GOOD FAITH EFFORT TO MARKET.—The 

7 Secretary may publish the results of a report sub8 mitted under paragraph (1) in the Federal Register 

9 if the Secretary finds that the sponsor that sub10 mitted such report has not made a good faith effort 

11 to meet the global demand for the product that is 

12 the subject of such report during each of the first 

13 4 years after approval of such product. 

14 ‘‘(g) NOTICE OF ISSUANCE AND USE OF VOUCHER.— 

15 The Secretary shall publish a notice in the Federal Reg16 ister and on the Web site of the Food and Drug Adminis17 tration not later than 30 days after the occurrence of each 

18 of the following: 

19 ‘‘(1) The Secretary issues a priority review 

20 voucher under this section. 

21 ‘‘(2) A sponsor submits a human drug applica22 tion for which such sponsor uses a priority review 

23 voucher. 

24 ‘‘(h) ELIGIBILITY FOR OTHER PROGRAMS.—A spon25 sor who seeks a priority review voucher under this section 


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1 may participate in any other incentive program, including 

2 the programs the Secretary has implemented under this 

3 Act, if the sponsor meets the applicable criteria of such 

4 other incentive program. 

5 ‘‘(i) RELATION TO OTHER PROVISIONS.—The provi6 sions of this section shall supplement, not supplant, any 

7 other provisions of this Act or the Public Health Service 

8 Act that encourage the development of drugs for tropical 

9 diseases and rare pediatric diseases.’’. 

10 (f) CONFORMING AMENDMENT.—Section 740(b) of 

11 the Agricultural, Rural Development, Food and Drug Ad12 ministration, and Related Agencies Appropriations Act, 

13 2010 (21 U.S.C. 360aa note) is amended by striking 

14 ‘‘(a)(3)’’ each place such term appears and inserting 

15 ‘‘(a)(6)’’. 

16 SEC. 3. EFFECTIVE DATE. 

17 This Act (and the amendments made by this Act) 

18 shall take effect on the date that is 90 days after the date 

19 of enactment of this Act. 

Æ 


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