II 

Calendar No. 415 

114TH CONGRESS 

2D SESSION S. 1878 

To extend the pediatric priority review voucher program. 

IN THE SENATE OF THE UNITED STATES 

JULY 28, 2015 

Mr. CASEY (for himself, Mr. ISAKSON, Mr. BROWN, and Mr. KIRK) introduced 

the following bill; which was read twice and referred to the Committee 

on Health, Education, Labor, and Pensions 

APRIL 5, 2016 

Reported by Mr. ALEXANDER, with an amendment 

[Strike out all after the enacting clause and insert the part printed in italic] 

A BILL 

To extend the pediatric priority review voucher program. 

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. 

4 This Act may be cited as the ‘‘Advancing Hope Act 

5 of 2015’’. 


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1 SEC. 2. REAUTHORIZATION OF PROGRAM FOR PRIORITY 

2 REVIEW TO ENCOURAGE TREATMENTS FOR 

3 RARE PEDIATRIC DISEASES. 

4 Section 529 of the Federal Food, Drug, and Cosmetic 

5 Act (21 U.S.C. 360ff) is amended— 

6 (1) in subsection (a)— 

7 (A) by amending paragraph (3) to read as 

8 follows: 

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

10 ‘rare pediatric disease’ means any of the following: 

11 ‘‘(A) A disease that meets each of the fol12 lowing criteria: 

13 ‘‘(i) The disease primarily affects indi14 viduals aged from birth to 18 years, in15 cluding age groups often called neonates, 

16 infants, children, and adolescents. 

17 ‘‘(ii) The disease is a rare disease or 

18 condition, within the meaning of section 

19 526. 

20 ‘‘(B) Any form of sickle cell disease. 

21 ‘‘(C) Any pediatric cancers.’’; and 

22 (B) in paragraph (4)— 

23 (i) in subparagraph (E), by striking 

24 ‘‘and’’ at the end; 


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1 (ii) in subparagraph (F), by striking 

2 the period at the end and inserting ‘‘; 

3 and’’; and 

4 (iii) by adding at the end the fol5 lowing: 

6 ‘‘(G) is for a drug or biological product for 

7 which a priority review voucher has not been 

8 issued under section 524 (relating to tropical 

9 disease products).’’; 

10 (2) in subsection (b), by striking paragraph (5); 

11 and 

12 (3) in subsection (d)(2), in the second sentence, 

13 by striking the period and inserting ‘‘, but the spon14 sor of a drug that intends to request a priority re15 view voucher under this section shall notify the Sec16 retary of such intent upon submission of an applica17 tion under section 505 or section 351 of the Public 

18 Health Service Act that is the basis of such re19 quest.’’. 

20 SECTION 1. SHORT TITLE. 

21 This Act may be cited as the ‘‘Advancing Hope Act 

22 of 2016’’. 


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1 SEC. 2. REAUTHORIZATION OF PROGRAM FOR PRIORITY RE2 VIEW TO ENCOURAGE TREATMENTS FOR 

3 RARE PEDIATRIC DISEASES. 

4 (a) IN GENERAL.—Section 529 of the Federal Food, 

5 Drug, and Cosmetic Act (21 U.S.C. 360ff) is amended— 

6 (1) in subsection (a)— 

7 (A) in paragraph (3), by amending sub8 paragraph (A) to read as follows: 

9 ‘‘(A) The disease is a serious or life-threat10 ening disease in which the serious or life-threat11 ening manifestations primarily affect individ12 uals aged from birth to 18 years, including age 

13 groups often called neonates, infants, children, 

14 and adolescents.’’; and 

15 (B) in paragraph (4)(F), by striking ‘‘Pre16 scription Drug User Fee Amendments of 2012’’ 

17 and inserting ‘‘Advancing Hope Act of 2016’’; 

18 (2) in subsection (b)— 

19 (A) by striking paragraph (4) and inserting 

20 the following: 

21 ‘‘(4) NOTIFICATION.— 

22 ‘‘(A) SPONSOR OF A RARE PEDIATRIC DIS23 EASE PRODUCT.— 

24 ‘‘(i) IN GENERAL.—Beginning on the 

25 date that is 90 days after the date of enact26 ment of the Advancing Hope Act of 2016, 


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1 the sponsor of a rare pediatric disease prod2 uct application that intends to request a 

3 priority review voucher under this section 

4 shall notify the Secretary of such intent 

5 upon submission of the rare pediatric dis6 ease product application that is the basis of 

7 the request for a priority review voucher. 

8 ‘‘(ii) APPLICATIONS SUBMITTED BUT

9 NOT YET APPROVED.—The sponsor of a rare 

10 pediatric disease product application that 

11 was submitted and that has not been ap12 proved as of the date of enactment of the 

13 Advancing Hope Act of 2016 shall be con14 sidered eligible for a priority review vouch15 er, if— 

16 ‘‘(I) such sponsor has submitted 

17 such rare pediatric disease product ap18 plication— 

19 ‘‘(aa) on or after the date 

20 that is 90 days after the date of 

21 enactment of the Prescription 

22 Drug User Fee Amendments of 

23 2012; and 


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1 ‘‘(bb) on or before the date of 

2 enactment of the Advancing Hope 

3 Act of 2016; and 

4 ‘‘(II) such application otherwise 

5 meets the criteria for a priority review 

6 voucher under this section. 

7 ‘‘(B) SPONSOR OF A DRUG APPLICATION

8 USING A PRIORITY REVIEW VOUCHER.— 

9 ‘‘(i) IN GENERAL.—The sponsor of a 

10 human drug application shall notify the 

11 Secretary not later than 90 days prior to 

12 submission of the human drug application 

13 that is the subject of a priority review 

14 voucher of an intent to submit the human 

15 drug application, including the date on 

16 which the sponsor intends to submit the ap17 plication. Such notification shall be a le18 gally binding commitment to pay the user 

19 fee to be assessed in accordance with this 

20 section. 

21 ‘‘(ii) TRANSFER AFTER NOTICE.—The 

22 sponsor of a human drug application that 

23 provides notification of the intent of such 

24 sponsor to use the voucher for the human 

25 drug application under clause (i) may 


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1 transfer the voucher after such notification 

2 is provided, if such sponsor has not yet sub3 mitted the human drug application de4 scribed in the notification.’’; and 

5 (B) by striking paragraph (5) and inserting 

6 the following: 

7 ‘‘(5) TERMINATION OF AUTHORITY.—The Sec8 retary may not award any priority review vouchers 

9 under paragraph (1) after September 30, 2022, unless 

10 the rare pediatric disease product application— 

11 ‘‘(A) is for a drug that, not later than Sep12 tember 30, 2022, is designated under subsection 

13 (d) as a drug for a rare pediatric disease; and 

14 ‘‘(B) is, not later than September 30, 2027, 

15 approved under section 505(b)(1) of this Act or 

16 section 351(a) of the Public Health Service Act.’’; 

17 and 

18 (3) in subsection (g), by inserting before the pe19 riod ‘‘, except that no sponsor of a rare pediatric dis20 ease product application may receive more than one 

21 priority review voucher issued under any section of 

22 this Act with respect to the drug for which the appli23 cation is made.’’ 

24 (b) RULE OF CONSTRUCTION.—Nothing in this Act, or 

25 the amendments made by this Act, shall be construed to afVerDate Sep 11 2014 20:57 Apr 05, 2016 Jkt 059200 PO 00000 Frm 00007 Fmt 6652 Sfmt 6203 E:\BILLS\S1878.RS S1878

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1 fect the validity of a priority review voucher that was issued 

2 under section 529 of the Federal Food, Drug, and Cosmetic 

3 Act (21 U.S.C. 360ff) before the date of enactment of this 

4 Act. 

5 SEC. 3. GAO REPORT. 

6 (a) STUDY.—The Comptroller General of the United 

7 States shall conduct a study on the effectiveness of awarding 

8 priority review vouchers under section 529 of the Federal 

9 Food, Drug, and Cosmetic Act (21 U.S.C. 360ff) in pro10 viding incentives for the development of drugs that treat 

11 or prevent rare pediatric diseases (as defined in subsection 

12 (a)(3) of such section) that would not otherwise have been 

13 developed. In conducting such study, the Comptroller Gen14 eral shall examine the following: 

15 (1) The indications for which each drug for 

16 which a priority review voucher was awarded under 

17 such section 529 was approved under section 

18 505(b)(1) of the Federal Food, Drug, and Cosmetic 

19 Act (21 U.S.C. 355(b)(1)) or section 351(a) of the 

20 Public Health Service Act (42 U.S.C. 262(a)). 

21 (2) Whether the priority review voucher im22 pacted sponsors’ decisions to invest in developing a 

23 drug to treat or prevent a rare pediatric disease. 


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1 (3) An analysis of the drugs for which such pri2 ority review vouchers were used, which shall in3 clude— 

4 (A) the indications for which such drugs 

5 were approved under section 505(b)(1) of the 

6 Federal Food, Drug, and Cosmetic Act (21 

7 U.S.C. 355(b)(1)) or section 351(a) of the Public 

8 Health Service Act (42 U.S.C. 262(a)); 

9 (B) whether unmet medical needs were ad10 dressed through the approval of such drugs, in11 cluding, for each such drug— 

12 (i) if an alternative therapy was pre13 viously available to treat the indication; 

14 and 

15 (ii) if the drug provided a benefit or 

16 advantage over another available therapy; 

17 (C) the number of patients potentially treat18 ed by such drugs; 

19 (D) the value of the priority review voucher 

20 if transferred; and 

21 (E) the length of time between the date on 

22 which a priority review voucher was awarded 

23 and the date on which it was used. 


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1 (4) With respect to the priority review voucher 

2 program under section 529 of the Federal Food, Drug, 

3 and Cosmetic Act (21 U.S.C. 360ff)— 

4 (A) the resources used by the Food and 

5 Drug Administration in implementing such pro6 gram, including the effect of such program on the 

7 Food and Drug Administration’s review of drugs 

8 for which a priority review voucher was not 

9 awarded or used; 

10 (B) the impact of the program on the public 

11 health as a result of the review and approval of 

12 drugs that received a priority review voucher 

13 and products that were the subject of a redeemed 

14 priority review voucher; and 

15 (C) alternative approaches to improving 

16 such program so that the program is appro17 priately targeted toward providing incentives for 

18 the development of clinically important drugs 

19 that— 

20 (i) prevent or treat rare pediatric dis21 eases; and 

22 (ii) would likely not otherwise have 

23 been developed to prevent or treat such dis24 eases. 


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1 (b) REPORT.—Not later than January 31, 2022, the 

2 Comptroller General of the United States shall submit to 

3 the Committee on Health, Education, Labor, and Pensions 

4 of the Senate and the Committee on Energy and Commerce 

5 of the House of Representatives a report containing the re6 sults of the study of conducted under subsection (a). 


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Calendar No. 415 

114TH CONGRESS 

2D SESSION S. 1878 

A BILL 

To extend the pediatric priority review voucher 

program. 

APRIL 5, 2016 

Reported with an amendment 


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