The priority review voucher program for rare pediatric diseases is extended through 2018 and revised to apply only to serious diseases.
(a) In General.—Section 529 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360ff) is amended—
(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
(i) in subparagraph (E), by striking “and” at the end;
(ii) in subparagraph (F), by striking the period at the end and inserting “; and”; and
(iii) by adding at the end the following:
“(G) is for a drug or biological product for which a priority review voucher has not been issued under section 524 (relating to tropical disease products).”; and
(2) in subsection (b), by striking paragraph (5) and inserting the following:
“(5) TERMINATION OF AUTHORITY.—
“(A) IN GENERAL.—The Secretary may not award any priority review vouchers under paragraph (1) after December 31, 2018.
“(B) EXCEPTION.—Notwithstanding subparagraph (A), the sponsor of a drug that is designated under subsection (d) as a drug for a rare pediatric disease and that is the subject of a rare pediatric disease product application that is submitted during the period beginning on the date of enactment of the 21st Century Cures Act and ending the date specified in subparagraph (A) shall remain eligible to receive a priority review voucher under paragraph (1) irrespective of whether the rare pediatric disease product application with respect to such drug is approved after the end of such period.”.
(1) 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:
(A) The indications for which each drug for which a priority review voucher was awarded under such section 529 was approved under section 505 of such Act (21 U.S.C. 355) or section 351 of the Public Health Service Act (42 U.S.C. 262).
(B) Whether the priority review voucher impacted a sponsor’s decision to invest in developing a drug to treat or prevent a rare pediatric disease.
(C) An analysis of the drugs that utilized such priority review vouchers, which shall include—
(i) the indications for which such drugs were approved under section 505 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355) or section 351 of the Public Health Service Act (42 U.S.C. 262);
(ii) 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) the benefit or advantage the drug provided over another available therapy;
(iii) the number of patients potentially treated by such drugs;
(iv) the value of the priority review voucher if transferred; and
(v) the length of time between the date on which a priority review voucher was awarded and the date on which it was used.
(D) With respect to the priority review voucher program under section 529 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360ff)—
(i) the resources used by, and burden placed on, 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;
(ii) the impact of the program on the public health as a result of the expedited review of applications for drugs that treat or prevent non-serious indications that are generally used by the broader public; and
(iii) 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.
(2) REPORT.—Not later than December 31, 2017, the Comptroller General of the United States shall submit to the Committee on Energy and Commerce of the House of Representatives and the Committee on Health, Education, Labor, and Pensions of the Senate a report containing the results of the study of conducted under paragraph (1).