S. 1878 

One Hundred Fourteenth Congress 

of the 

United States of America 

AT THE SECOND SESSION 

Begun and held at the City of Washington on Monday, 

the fourth day of January, two thousand and sixteen 

An Act 

To extend the pediatric priority review voucher program. 

Be it enacted by the Senate and House of Representatives of 

the United States of America in Congress assembled, 

SECTION 1. SHORT TITLE. 

This Act may be cited as the ‘‘Advancing Hope Act of 2016’’. 

SEC. 2. REAUTHORIZATION OF PROGRAM FOR PRIORITY REVIEW TO 

ENCOURAGE TREATMENTS FOR RARE PEDIATRIC DISEASES. 

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

Cosmetic Act (21 U.S.C. 360ff) is amended— 

(1) in subsection (a)— 

(A) in paragraph (3), by amending subparagraph (A) 

to read as follows: 

‘‘(A) The disease is a serious or life-threatening disease 

in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years, 

including age groups often called neonates, infants, children, and adolescents.’’; and 

(B) in paragraph (4)(F), by striking ‘‘Prescription Drug 

User Fee Amendments of 2012’’ and inserting ‘‘Advancing 

Hope Act of 2016’’; 

(2) in subsection (b)— 

(A) by striking paragraph (4) and inserting the following: 

‘‘(4) NOTIFICATION.— 

‘‘(A) SPONSOR OF A RARE PEDIATRIC DISEASE PRODUCT.— 

‘‘(i) IN GENERAL.—Beginning on the date that is 

90 days after the date of enactment of the Advancing 

Hope Act of 2016, the sponsor of a rare pediatric disease product application that intends to request a priority review voucher under this section shall notify 

the Secretary of such intent upon submission of the 

rare pediatric disease product application that is the 

basis of the request for a priority review voucher. 

‘‘(ii) APPLICATIONS SUBMITTED BUT NOT YET

APPROVED.—The sponsor of a rare pediatric disease 

product application that was submitted and that has 

not been approved as of the date of enactment of the 

Advancing Hope Act of 2016 shall be considered eligible 

for a priority review voucher, if— 

‘‘(I) such sponsor has submitted such rare pediatric disease product application— 
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‘‘(aa) on or after the date that is 90 days 

after the date of enactment of the Prescription 

Drug User Fee Amendments of 2012; and 

‘‘(bb) on or before the date of enactment 

of the Advancing Hope Act of 2016; and 

‘‘(II) such application otherwise meets the criteria for a priority review voucher under this section. 

‘‘(B) SPONSOR OF A DRUG APPLICATION USING A PRIORITY

REVIEW VOUCHER.— 

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

application shall notify the Secretary not later than 

90 days prior to submission of the human drug application that is the subject of a priority review voucher 

of an intent to submit the human drug application, 

including the date on which the sponsor intends to 

submit the application. Such notification shall be a 

legally binding commitment to pay the user fee to 

be assessed in accordance with this section. 

‘‘(ii) TRANSFER AFTER NOTICE.—The sponsor of a 

human drug application that provides notification of 

the intent of such sponsor to use the voucher for the 

human drug application under clause (i) may transfer 

the voucher after such notification is provided, if such 

sponsor has not yet submitted the human drug application described in the notification.’’; and 

(B) by striking paragraph (5) and inserting the following: 

‘‘(5) TERMINATION OF AUTHORITY.—The Secretary may not 

award any priority review vouchers under paragraph (1) after 

December 31, 2016.’’; and 

(3) in subsection (g), by inserting before the period ‘‘, except 

that no sponsor of a rare pediatric disease product application 

may receive more than one priority review voucher issued under 

any section of this Act with respect to the drug for which 

the application is made.’’ 

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

amendments made by this Act, shall be construed to affect the 

validity of a priority review voucher that was issued under section 

529 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360ff) 

before the date of enactment of this Act. 

SEC. 3. GAO REPORT. 

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

conduct a study on the effectiveness of awarding priority review 

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

Act (21 U.S.C. 360ff) in providing incentives for the development 

of drugs that treat or prevent rare pediatric diseases (as defined 

in subsection (a)(3) of such section) that would not otherwise have 

been developed. In conducting such study, the Comptroller General 

shall examine the following: 

(1) The indications for which each drug for which a priority 

review voucher was awarded under such section 529 was 

approved under section 505(b)(1) of the Federal Food, Drug, 

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

the Public Health Service Act (42 U.S.C. 262(a)). 
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(2) Whether the priority review voucher impacted sponsors’ 

decisions to invest in developing a drug to treat or prevent 

a rare pediatric disease. 

(3) An analysis of the drugs for which such priority review 

vouchers were used, which shall include— 

(A) the indications for which such drugs were approved 

under section 505(b)(1) of the Federal Food, Drug, and 

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

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

(B) whether unmet medical needs were addressed 

through the approval of such drugs, including, for each 

such drug— 

(i) if an alternative therapy was previously available to treat the indication; and 

(ii) if the drug provided a benefit or advantage 

over another available therapy; 

(C) the number of patients potentially treated by such 

drugs; 

(D) the value of the priority review voucher if transferred; and 

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

a priority review voucher was awarded and the date on 

which it was used. 

(4) With respect to the priority review voucher program 

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

Act (21 U.S.C. 360ff)— 

(A) the resources used by the Food and Drug Administration in implementing such program, including the effect 

of such program on the Food and Drug Administration’s 

review of drugs for which a priority review voucher was 

not awarded or used; 

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

as a result of the review and approval of drugs that received 

a priority review voucher and products that were the subject of a redeemed priority review voucher; and 

(C) alternative approaches to improving such program 

so that the program is appropriately targeted toward providing incentives for the development of clinically important drugs that— 

(i) prevent or treat rare pediatric diseases; and 

(ii) would likely not otherwise have been developed 

to prevent or treat such diseases. 

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

General of the United States shall submit to the Committee on 

Health, Education, Labor, and Pensions of the Senate and the 
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Committee on Energy and Commerce of the House of Representatives a report containing the results of the study of conducted 

under subsection (a). 

Speaker of the House of Representatives. 

Vice President of the United States and

President of the Senate.