114TH CONGRESS 

2D SESSION S. 1878 

AN ACT 

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, 
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1 SECTION 1. SHORT TITLE. 

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

3 of 2016’’. 

4 SEC. 2. REAUTHORIZATION OF PROGRAM FOR PRIORITY 

5 REVIEW TO ENCOURAGE TREATMENTS FOR 

6 RARE PEDIATRIC DISEASES. 

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

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

9 (1) in subsection (a)— 

10 (A) in paragraph (3), by amending sub11 paragraph (A) to read as follows: 

12 ‘‘(A) The disease is a serious or life-threat13 ening disease in which the serious or life-threat14 ening manifestations primarily affect individ15 uals aged from birth to 18 years, including age 

16 groups often called neonates, infants, children, 

17 and adolescents.’’; and 

18 (B) in paragraph (4)(F), by striking ‘‘Pre19 scription Drug User Fee Amendments of 2012’’ 

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

21 (2) in subsection (b)— 

22 (A) by striking paragraph (4) and insert23 ing the following: 

24 ‘‘(4) NOTIFICATION.— 

25 ‘‘(A) SPONSOR OF A RARE PEDIATRIC DIS26 EASE PRODUCT.— 
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1 ‘‘(i) IN GENERAL.—Beginning on the 

2 date that is 90 days after the date of en3 actment of the Advancing Hope Act of 

4 2016, the sponsor of a rare pediatric dis5 ease product application that intends to re6 quest a priority review voucher under this 

7 section shall notify the Secretary of such 

8 intent upon submission of the rare pedi9 atric disease product application that is the 

10 basis of the request for a priority review 

11 voucher. 

12 ‘‘(ii) APPLICATIONS SUBMITTED BUT

13 NOT YET APPROVED.—The sponsor of a 

14 rare pediatric disease product application 

15 that was submitted and that has not been 

16 approved as of the date of enactment of 

17 the Advancing Hope Act of 2016 shall be 

18 considered eligible for a priority review 

19 voucher, if— 

20 ‘‘(I) such sponsor has submitted 

21 such rare pediatric disease product 

22 application— 

23 ‘‘(aa) on or after the date 

24 that is 90 days after the date of 

25 enactment of the Prescription 
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1 Drug User Fee Amendments of 

2 2012; and 

3 ‘‘(bb) on or before the date 

4 of enactment of the Advancing 

5 Hope Act of 2016; and 

6 ‘‘(II) such application otherwise 

7 meets the criteria for a priority review 

8 voucher under this section. 

9 ‘‘(B) SPONSOR OF A DRUG APPLICATION

10 USING A PRIORITY REVIEW VOUCHER.— 

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

12 human drug application shall notify the 

13 Secretary not later than 90 days prior to 

14 submission of the human drug application 

15 that is the subject of a priority review 

16 voucher of an intent to submit the human 

17 drug application, including the date on 

18 which the sponsor intends to submit the 

19 application. Such notification shall be a le20 gally binding commitment to pay the user 

21 fee to be assessed in accordance with this 

22 section. 

23 ‘‘(ii) TRANSFER AFTER NOTICE.—The 

24 sponsor of a human drug application that 

25 provides notification of the intent of such 
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1 sponsor to use the voucher for the human 

2 drug application under clause (i) may 

3 transfer the voucher after such notification 

4 is provided, if such sponsor has not yet 

5 submitted the human drug application de6 scribed in the notification.’’; and 

7 (B) by striking paragraph (5) and insert8 ing the following: 

9 ‘‘(5) TERMINATION OF AUTHORITY.—The Sec10 retary may not award any priority review vouchers 

11 under paragraph (1) after December 31, 2016.’’; 

12 and 

13 (3) in subsection (g), by inserting before the pe14 riod ‘‘, except that no sponsor of a rare pediatric 

15 disease product application may receive more than 

16 one priority review voucher issued under any section 

17 of this Act with respect to the drug for which the 

18 application is made.’’ 

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

20 or the amendments made by this Act, shall be construed 

21 to affect the validity of a priority review voucher that was 

22 issued under section 529 of the Federal Food, Drug, and 

23 Cosmetic Act (21 U.S.C. 360ff) before the date of enact24 ment of this Act. 
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1 SEC. 3. GAO REPORT. 

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

3 States shall conduct a study on the effectiveness of award4 ing priority review vouchers under section 529 of the Fed5 eral Food, Drug, and Cosmetic Act (21 U.S.C. 360ff) in 

6 providing incentives for the development of drugs that 

7 treat or prevent rare pediatric diseases (as defined in sub8 section (a)(3) of such section) that would not otherwise 

9 have been developed. In conducting such study, the Comp10 troller General shall examine the following: 

11 (1) The indications for which each drug for 

12 which a priority review voucher was awarded under 

13 such section 529 was approved under section 

14 505(b)(1) of the Federal Food, Drug, and Cosmetic 

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

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

17 (2) Whether the priority review voucher im18 pacted sponsors’ decisions to invest in developing a 

19 drug to treat or prevent a rare pediatric disease. 

20 (3) An analysis of the drugs for which such pri21 ority review vouchers were used, which shall in22 clude— 

23 (A) the indications for which such drugs 

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

25 Federal Food, Drug, and Cosmetic Act (21 
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1 U.S.C. 355(b)(1)) or section 351(a) of the Pub2 lic Health Service Act (42 U.S.C. 262(a)); 

3 (B) whether unmet medical needs were ad4 dressed through the approval of such drugs, in5 cluding, for each such drug— 

6 (i) if an alternative therapy was pre7 viously available to treat the indication; 

8 and 

9 (ii) if the drug provided a benefit or 

10 advantage over another available therapy; 

11 (C) the number of patients potentially 

12 treated by such drugs; 

13 (D) the value of the priority review vouch14 er if transferred; and 

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

16 which a priority review voucher was awarded 

17 and the date on which it was used. 

18 (4) With respect to the priority review voucher 

19 program under section 529 of the Federal Food, 

20 Drug, and Cosmetic Act (21 U.S.C. 360ff)— 

21 (A) the resources used by the Food and 

22 Drug Administration in implementing such pro23 gram, including the effect of such program on 

24 the Food and Drug Administration’s review of 
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1 drugs for which a priority review voucher was 

2 not awarded or used; 

3 (B) the impact of the program on the pub4 lic health as a result of the review and approval 

5 of drugs that received a priority review voucher 

6 and products that were the subject of a re7 deemed priority review voucher; and 

8 (C) alternative approaches to improving 

9 such program so that the program is appro10 priately targeted toward providing incentives for 

11 the development of clinically important drugs 

12 that— 

13 (i) prevent or treat rare pediatric dis14 eases; and 

15 (ii) would likely not otherwise have 

16 been developed to prevent or treat such 

17 diseases. 

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

19 Comptroller General of the United States shall submit to 

20 the Committee on Health, Education, Labor, and Pen21 sions of the Senate and the Committee on Energy and 

22 Commerce of the House of Representatives a report con-
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1 taining the results of the study of conducted under sub2 section (a). 

Passed the Senate September 22, 2016. 

Attest: 

Secretary. 
114TH CONGRESS 

2D SESSION S. 1878 

AN ACT 

To extend the pediatric priority review voucher 

program.