II 

112TH CONGRESS 

1ST SESSION S. 606 

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 SENATE OF THE UNITED STATES 

MARCH 17, 2011 

Mr. CASEY (for himself, Mr. BROWN of Massachusetts, Mr. BROWN of Ohio, 

Mr. ISAKSON, and Mr. FRANKEN) introduced the following bill; which was 

read twice and referred to the Committee on Health, Education, Labor, 

and Pensions 

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’’. 

6 (b) REFERENCES.—Wherever in this Act an amend7 ment is expressed in terms of an amendment to a section 

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


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

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

3 SEC. 2. IMPROVEMENT OF THE TROPICAL DISEASE VOUCH4 ER PROGRAM. 

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

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

7 REVIEW TO ENCOURAGE INNOVATIVE TREATMENTS

8 FOR TROPICAL DISEASES AND RARE PEDIATRIC

9 DISEASES’’. 

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

11 360n(a)) is amended— 

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

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

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

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

16 (3) by inserting after ‘‘In this section:’’, the fol17 lowing: 

18 ‘‘(1) INNOVATIVE TREATMENT.—The term ‘in19 novative treatment’ means— 

20 ‘‘(A) a human drug that is the subject of 

21 an application submitted under section 

22 505(b)(1), if that drug contains no active ingre23 dient (including any ester or salt of the active 

24 ingredient) that has been previously approved 

25 in any other application under section 


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1 505(b)(1), 505(b)(2), or 505(j) or section 351 

2 of the Public Health Service Act; or 

3 ‘‘(B) a biological product that is the sub4 ject of an application submitted under section 

5 351(a) of the Public Health Service Act, if that 

6 biological product— 

7 ‘‘(i) does not have the same structure 

8 as a biological product that has been pre9 viously licensed in any other application 

10 under subsection (a) or (k) of section 351 

11 of the Public Health Service Act or ap12 proved under section 505 of this Act; and 

13 ‘‘(ii) is not biosimilar, within the 

14 meaning of section 351(i) of the Public 

15 Health Service Act, to a biological product 

16 that has been previously licensed in any 

17 other application under subsection (a) or 

18 (k) of section 351 of the Public Health 

19 Service Act or approved under section 505 

20 of this Act.’’; 

21 (4) in paragraph (3), as so redesignated, by in22 serting ‘‘or rare pediatric disease product applica23 tion’’ after ‘‘tropical disease product application’’ 

24 each place that phrase appears; 


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1 (5) by inserting after paragraph (3) the fol2 lowing: 

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

4 ‘rare pediatric disease’ means a disease that meets 

5 each of the following criteria: 

6 ‘‘(A) The disease is recognized in the med7 ical community as affecting a pediatric popu8 lation. 

9 ‘‘(B) The disease is a rare disease or con10 dition, within the meaning of section 526. 

11 ‘‘(5) RARE PEDIATRIC DISEASE PRODUCT AP12 PLICATION.—The term ‘rare pediatric disease prod13 uct application’ means a human drug application, as 

14 defined in section 735(1)— 

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

16 pediatric disease; 

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

18 priority review; 

19 ‘‘(C) that is for an innovative treatment; 

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

21 from studies examining a pediatric population 

22 and dosages of the drug intended for that popu23 lation; and 


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

2 adult indication in the original rare pediatric 

3 disease product application.’’; 

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

5 (A) by redesignating subparagraph (Q) as 

6 subparagraph (R); and 

7 (B) by inserting after subparagraph (P) 

8 the following: 

9 ‘‘(Q) Chagas Disease.’’; and 

10 (7) by amending paragraph (7), as so redesig11 nated, to read as follows: 

12 ‘‘(7) TROPICAL DISEASE PRODUCT APPLICA13 TION.—The term ‘tropical disease product applica14 tion’ means a human drug application, as defined in 

15 section 735(1)— 

16 ‘‘(A) for prevention or treatment of a trop17 ical disease; 

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

19 priority review; 

20 ‘‘(C) that is for an innovative treatment; 

21 and 

22 ‘‘(D) that the sponsor affirms in the appli23 cation is for a drug that has not been approved 

24 for commercial marketing for any tropical dis25 ease indication by a government authority outVerDate Mar 15 2010 04:33 Mar 22, 2011 Jkt 099200 PO 00000 Frm 00005 Fmt 6652 Sfmt 6201 E:\BILLS\S606.IS S606

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1 side of the United States for more than 24 

2 months before the tropical disease product ap3 plication is submitted.’’. 

4 (c) RULES REGARDING USE AND TRANSFER OF PRI5 ORITY REVIEW VOUCHERS.—Section 524(b) (21 U.S.C. 

6 360n(b)) is amended— 

7 (1) in paragraph (1), by inserting ‘‘or rare pe8 diatric disease product application’’ after ‘‘tropical 

9 disease product application’’ each place that phrase 

10 appears; 

11 (2) by amending paragraph (2) to read as fol12 lows: 

13 ‘‘(2) TRANSFERABILITY.— 

14 ‘‘(A) IN GENERAL.—The sponsor of a trop15 ical disease product application or rare pediatric 

16 disease product application that receives a pri17 ority review voucher under this section may 

18 transfer (including by sale) the entitlement to 

19 such voucher. There is no limit on the number 

20 of times a priority review voucher may be trans21 ferred before such voucher is used. 

22 ‘‘(B) CONDITIONS OF TRANSFER.—If a 

23 sponsor transfers a priority review voucher 

24 after such sponsor has provided notification to 

25 the Secretary under paragraph (4)(A) of the inVerDate Mar 15 2010 04:33 Mar 22, 2011 Jkt 099200 PO 00000 Frm 00006 Fmt 6652 Sfmt 6201 E:\BILLS\S606.IS S606

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1 tent of such sponsor to use the voucher, the 

2 transfer shall be subject to the provisions of 

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

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

5 person to whom a voucher is transferred under 

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

7 of such change in ownership of the voucher not 

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

9 (3) by amending paragraph (3) to read as fol10 lows: 

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

12 ‘‘(A) TROPICAL DISEASE PRODUCT APPLI13 CATIONS.—A sponsor of a tropical disease prod14 uct application may not receive a priority review 

15 voucher under this section if the tropical dis16 ease product application was submitted to the 

17 Secretary prior to September 27, 2007. 

18 ‘‘(B) RARE PEDIATRIC DISEASE PRODUCT

19 APPLICATIONS.—A sponsor of a rare pediatric 

20 disease product application may not receive a 

21 priority review voucher under this section if the 

22 rare pediatric disease product application was 

23 submitted to the Secretary prior to the date 

24 that is 90 days after the date of enactment of 

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


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1 (4) by amending paragraph (4) to read as fol2 lows: 

3 ‘‘(4) NOTIFICATION.— 

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

5 date on which a human drug application for 

6 which the sponsor intends to use a priority re7 view voucher is submitted, the sponsor of such 

8 human drug application shall notify the Sec9 retary of the intent of such sponsor to submit 

10 the human drug application. 

11 ‘‘(B) TRANSFER OF VOUCHER AFTER NO12 TIFICATION.— 

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

14 human drug application that provides noti15 fication of the intent of such sponsor to 

16 use the voucher for the human drug appli17 cation may transfer the voucher within 1 

18 year after such notification is provided, if 

19 such sponsor has not yet submitted the 

20 human drug application described in the 

21 notification. 

22 ‘‘(ii) EXCEPTION.—The person to 

23 whom a voucher is transferred under 

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

25 the ‘transferee’) shall give notification of 


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1 the intent of such transferee to use the 

2 voucher in accordance with this subsection, 

3 unless— 

4 ‘‘(I) the transferee uses the 

5 voucher for a human drug application 

6 including the same indications as the 

7 human drug application described in 

8 the transferor’s notification; and 

9 ‘‘(II) the transferee notifies the 

10 Secretary within 30 days of the trans11 fer of the intent of such transferee to 

12 use the voucher for such purpose. 

13 ‘‘(iii) INTERNAL TRANSFER.—If the 

14 sponsor transfers a voucher internally for 

15 use with a drug application including one 

16 or more indications that were not included 

17 in the drug application that was the sub18 ject of the notification of such sponsor, the 

19 sponsor shall notify the Secretary of the 

20 transfer in accordance with this subsection. 

21 ‘‘(C) FEE DUE UPON NOTIFICATION; CRED22 IT FOR TRANSFERRED VOUCHER.— 

23 ‘‘(i) DUE UPON NOTIFICATION.—The 

24 notification under this subsection shall be 

25 a legally binding commitment to pay for 


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1 the user fee to be assessed in accordance 

2 with this section. Such fee shall be payable 

3 by the sponsor upon the submission by 

4 such sponsor of such notification. 

5 ‘‘(ii) CREDIT.—If a sponsor pays a 

6 user fee upon providing notification of the 

7 intent of such sponsor to use a priority re8 view voucher, but later transfers the vouch9 er for which such sponsor gave notifica10 tion, the Secretary shall credit the user 

11 fees paid to the next human drug applica12 tion for which a sponsor provides notifica13 tion of the intent of such sponsor to use 

14 the same transferred voucher. 

15 ‘‘(iii) DIFFERENCE IN FEE.—The Sec16 retary may require a sponsor using a 

17 transferred voucher to pay the difference 

18 between the credit associated with the 

19 transferred voucher and the user fee pre20 vailing at the time the sponsor submits no21 tification of the intent of such sponsor to 

22 use the transferred voucher. This provision 

23 does not apply in cases where a transferee 

24 is exempted from submitting notification 

25 under this paragraph.’’. 


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

2 360n(c)(4)) is amended— 

3 (1) in subparagraph (A), by striking ‘‘submis4 sion of a human drug application under section 

5 505(b)(1) or section 351 of the Public Health Serv6 ices Act for which the priority review voucher is 

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

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

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

10 associated with the human drug application shall be 

11 due as required by the Secretary or under applicable 

12 law.’’; and 

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

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

15 subsection (b)(4)(C).’’. 

16 (e) DESIGNATION PROCESS; PRODUCT IMPLEMENTA17 TION REQUIREMENT.—Section 524 (21 U.S.C. 360n) is 

18 amended by adding at the end the following new sub19 sections: 

20 ‘‘(e) DESIGNATION PROCESS.— 

21 ‘‘(1) DESIGNATION OF RARE PEDIATRIC DIS22 EASES.— 

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

24 the manufacturer or the sponsor of a new drug, 

25 the Secretary may designate that the new drug 


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1 is for a rare pediatric disease. Such a request 

2 for designation, if sought, shall be made when 

3 requesting designation of orphan disease status 

4 under section 526 or fast-track designation 

5 under section 506. Requesting designation of 

6 rare pediatric disease status under this para7 graph is not a prerequisite to receiving a pri8 ority review voucher. 

9 ‘‘(B) DETERMINATION BY SECRETARY.— 

10 Not later than 60 days after a request is sub11 mitted under subparagraph (A), the Secretary 

12 shall determine whether the disease or condition 

13 that is the subject of such request is a rare pe14 diatric disease. 

15 ‘‘(2) DESIGNATION OF INNOVATIVE TREAT16 MENTS.— 

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

18 the manufacturer or the sponsor of a new drug, 

19 the Secretary may designate that a new drug is 

20 an innovative treatment. Such a request for 

21 designation, if sought, shall be made when re22 questing fast-track designation under section 

23 506. Requesting designation that a new drug is 

24 an innovative treatment is not a prerequisite to 

25 receiving a priority review voucher. 


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1 ‘‘(B) DETERMINATION BY SECRETARY.— 

2 Not later than 60 days after a request is sub3 mitted under subparagraph (A), the Secretary 

4 shall determine whether the new drug that is 

5 the subject of such request is an innovative 

6 treatment. 

7 ‘‘(f) PRODUCT IMPLEMENTATION FOR RARE PEDI8 ATRIC DISEASE PRODUCTS.— 

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

10 rare pediatric disease product application incomplete 

11 if such application does not contain a description of 

12 the plan of the sponsor of such application to mar13 ket the product in the United States. 

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

15 ‘‘(A) GOOD FAITH INTENT.—The Sec16 retary may refuse to issue a priority review 

17 voucher upon the approval of a rare pediatric 

18 disease product application if the Secretary 

19 finds that the sponsor of such application lacks 

20 a good faith intention to market the product in 

21 the United States. The Secretary may consider 

22 any fact relevant to this determination, includ23 ing the history of such sponsor of producing 

24 rare pediatric disease products for which such 

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1 phan drugs for which the sponsor received ex2 clusivity under section 527, or pediatric drugs 

3 for which the sponsor received an additional 6 

4 months of exclusivity under section 505A. 

5 ‘‘(B) PRESUMPTION.—The sponsor may 

6 establish a presumption of good faith by dem7 onstrating that such sponsor has allocated suffi8 cient resources or otherwise arranged for the 

9 production (by the sponsor or by another manu10 facturer) of the rare pediatric disease product 

11 in a manner sufficient to meet the expected de12 mand for the product during the 5-year period 

13 following approval of the application. 

14 ‘‘(C) GUIDANCE.—If the Secretary re15 quires sponsors seeking a priority review vouch16 er to demonstrate a good faith intent to market 

17 the rare pediatric disease product in the United 

18 States, the Secretary shall first issue a guid19 ance document setting forth the required evi20 dentiary support necessary to demonstrate such 

21 a good faith intent. 

22 ‘‘(3) POSTAPPROVAL PRODUCTION REPORT.— 

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

24 an approved rare pediatric disease product shall 

25 submit a report to the Secretary not later than 


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1 5 years after the approval of the applicable rare 

2 pediatric disease product application. Such re3 port shall provide the following information, 

4 with respect to each of the first 4 years after 

5 approval of such product: 

6 ‘‘(i) The estimated population in the 

7 United States suffering from the rare pedi8 atric disease. 

9 ‘‘(ii) The estimated demand in the 

10 United States for such rare pediatric dis11 ease product. 

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

13 pediatric disease product distributed in the 

14 United States. 

15 ‘‘(B) PUBLICATION UPON FAILURE TO

16 DEMONSTRATE GOOD FAITH EFFORT TO MAR17 KET.—The Secretary may publish the results of 

18 a report submitted under subparagraph (A) in 

19 the Federal Register if the Secretary finds that 

20 the sponsor that submitted such report has not 

21 made a good faith effort to meet the demand in 

22 the United States for the product that is the 

23 subject of such report during each of the first 

24 4 years after approval of such product. 


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1 ‘‘(g) PRODUCTION REPORT FOR TROPICAL DISEASE

2 PRODUCTS.— 

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

4 approved tropical disease product shall submit a re5 port to the Secretary not later than 5 years after the 

6 approval of the applicable rare tropical disease prod7 uct application. Such report shall provide the fol8 lowing information, with respect to each of the first 

9 4 years after approval of such product: 

10 ‘‘(A) The estimated global population suf11 fering from the tropical disease. 

12 ‘‘(B) The estimated global demand for 

13 such tropical disease product. 

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

15 disease product distributed globally. 

16 ‘‘(2) PUBLICATION UPON FAILURE TO DEM17 ONSTRATE GOOD FAITH EFFORT TO MARKET.—The 

18 Secretary may publish the results of a report sub19 mitted under paragraph (1) in the Federal Register 

20 if the Secretary finds that the sponsor that sub21 mitted such report has not made a good faith effort 

22 to meet the global demand for the product that is 

23 the subject of such report during each of the first 

24 4 years after approval of such product. 


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1 ‘‘(h) NOTICE OF ISSUANCE AND USE OF VOUCHER.— 

2 The Secretary shall publish a notice in the Federal Reg3 ister and on the Web site of the Food and Drug Adminis4 tration not later than 30 days after the occurrence of each 

5 of the following: 

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

7 voucher under this section. 

8 ‘‘(2) A sponsor submits a human drug applica9 tion for which such sponsor uses a priority review 

10 voucher. 

11 ‘‘(i) ELIGIBILITY FOR OTHER PROGRAMS.—A spon12 sor who seeks a priority review voucher under this section 

13 may participate in any other incentive program, including 

14 the programs the Secretary has implemented under this 

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

16 other incentive program. 

17 ‘‘(j) RELATION TO OTHER PROVISIONS.—This provi18 sions of this section shall supplement, not supplant, any 

19 other provisions of this Act or the Public Health Service 

20 Act that encourage the development of drugs for tropical 

21 diseases and rare pediatric diseases.’’. 

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

23 the Agricultural, Rural Development, Food and Drug Ad24 ministration, and Related Agencies Appropriations Act, 

25 2010 (21 U.S.C. 360aa note) is amended by striking 


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

2 ‘‘(a)(6)’’. 

3 SEC. 3. EFFECTIVE DATE. 

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

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

6 of enactment of this Act. 

Æ 


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