In this section:
The term "priority review", with respect to a human drug application as defined in section 379g(1) of this title, means review and action by the Secretary on such application not later than 6 months after receipt by the Secretary of such application, as described in the Manual of Policies and Procedures of the Food and Drug Administration and goals identified in the letters described in section 101(b) of the Prescription Drug User Fee Amendments of 2012.
The term "priority review voucher" means a voucher issued by the Secretary to the sponsor of a rare pediatric disease product application that entitles the holder of such voucher to priority review of a single human drug application submitted under section 355(b)(1) of this title or section 351(a) of the Public Health Service Act [42 U.S.C. 262(a)] after the date of approval of the rare pediatric disease product application.
The term "rare pediatric disease" means a disease that meets each of the following criteria:
The term "rare pediatric disease product application" means a human drug application, as defined in section 379g(1) of this title, that-
The Secretary shall award a priority review voucher to the sponsor of a rare pediatric disease product application upon approval by the Secretary of such rare pediatric disease product application.
The sponsor of a rare pediatric disease product application that receives a priority review voucher under this section may transfer (including by sale) the entitlement to such voucher. There is no limit on the number of times a priority review voucher may be transferred before such voucher is used.
Each person to whom a voucher is transferred shall notify the Secretary of such change in ownership of the voucher not later than 30 days after such transfer.
A sponsor of a rare pediatric disease product application may not receive a priority review voucher under this section if the rare pediatric disease product application was submitted to the Secretary prior to the date that is 90 days after July 9, 2012.
Beginning on the date that is 90 days after September 30, 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.
The sponsor of a rare pediatric disease product application that was submitted and that has not been approved as of September 30, 2016, shall be considered eligible for a priority review voucher, if-
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.
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.
The Secretary may not award any priority review vouchers under paragraph (1) after December 20, 2024, unless the rare pediatric disease product application-
The Secretary shall establish a user fee program under which a sponsor of a human drug application that is the subject of a priority review voucher shall pay to the Secretary a fee determined under paragraph (2). Such fee shall be in addition to any fee required to be submitted by the sponsor under subchapter VII.
The amount of the priority review user fee shall be determined each fiscal year by the Secretary, based on the difference between-
The Secretary shall establish, before the beginning of each fiscal year beginning after September 30, 2012, the amount of the priority review user fee for that fiscal year.
The priority review user fee required by this subsection shall be due upon the notification by a sponsor of the intent of such sponsor to use the voucher, as specified in subsection (b)(4)(A).2 All other user fees associated with the human drug application shall be due as required by the Secretary or under applicable law.
An application described under subparagraph (A) for which the sponsor requests the use of a priority review voucher shall be considered incomplete if the fee required by this subsection and all other applicable user fees are not paid in accordance with the Secretary's procedures for paying such fees.
The Secretary may not grant a waiver, exemption, reduction, or refund of any fees due and payable under this section.
Fees collected pursuant to this subsection for any fiscal year-
Upon the request of the manufacturer or the sponsor of a new drug, the Secretary may designate-
The request for a designation under paragraph (1) shall be made at the same time a request for designation of orphan disease status under section 360bb of this title or fast-track designation under section 356 of this title is made. Requesting designation under this subsection is not a prerequisite to receiving a priority review voucher under this section.
Not later than 60 days after a request is submitted under paragraph (1), the Secretary shall determine whether-
The Secretary may revoke any priority review voucher awarded under subsection (b) if the rare pediatric disease product for which such voucher was awarded is not marketed in the United States within the 365-day period beginning on the date of the approval of such drug under section 355 of this title or section 351 of the Public Health Service Act [42 U.S.C. 262] .
The sponsor of an approved rare pediatric disease product shall submit a report to the Secretary not later than 5 years after the approval of the applicable rare pediatric disease product application. Such report shall provide the following information, with respect to each of the first 4 years after approval of such product:
The Secretary shall publish a notice in the Federal Register and on the Internet Web site of the Food and Drug Administration not later than 30 days after the occurrence of each of the following:
If, after the last day of the 1-year period that begins on the date that the Secretary awards the third rare pediatric disease priority voucher under this section, a sponsor of an application submitted under section 355(b) of this title or section 351(a) of the Public Health Service Act [42 U.S.C. 262(a)] for a drug uses a priority review voucher under this section for such application, the Secretary 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 document-
Nothing in this section precludes a sponsor who seeks a priority review voucher under this section from participating in any other incentive program, including under this chapter, 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 chapter with respect to the drug for which the application is made..3
The provisions of this section shall supplement, not supplant, any other provisions of this chapter or the Public Health Service Act [42 U.S.C. 201 et seq.] that encourage the development of drugs for tropical diseases and rare pediatric diseases.
Beginning on the date that the Secretary awards the third rare pediatric disease priority voucher under this section, the Comptroller General of the United States shall conduct a study of the effectiveness of awarding rare pediatric disease priority vouchers under this section in the development of human drug products that treat or prevent such diseases.
In conducting the study under subparagraph (A), the Comptroller General shall examine the following:
Not later than 1 year after the date under paragraph (1)(A), the Comptroller General 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 under paragraph (1).
1So in original. The word "that" probably should not appear.
2See References in Text note below.
3So in original.
21 U.S.C. § 360ff
EDITORIAL NOTES
REFERENCES IN TEXTSection 101(b) of the Prescription Drug User Fee Amendments of 2012, referred to in subsec. (a)(1), is section 101(b) of Pub. L. 112-144 which is set out as a note under section 379g of this title.Subsection (b)(4)(A), referred to in subsec. (c)(4)(A), was part of subsec. (b)(4) of this section that was struck out by Pub. L. 114-229, §2(a)(2)(A), Sept. 30, 2016, 130 Stat. 943, which provision also added a new subsec. (b)(4) in which former subsec. (b)(4)(A) was restated as subsec. (b)(4)(B)(i).The Public Health Service Act, referred to in subsec. (h), is act July 1, 1944, ch. 373, 58 Stat. 682, which is classified generally to chapter 6A (§201 et seq.) of Title 42, The Public Health and Welfare. For complete classification of this Act to the Code, see Short Title note set out under section 201 of Title 42 and Tables.
AMENDMENTS2024-Subsec. (b)(5). Pub. L. 118-83 substituted "December 20, 2024" for "September 30, 2024" in introductory provisions and in subpar. (A). 2021-Subsec. (a)(4)(A), (B). Pub. L. 117-9 added subpars. (A) and (B) and struck out former subpars. (A) and (B) which read as follows:"(A) is for a drug or biological product- "(i) that is for the prevention or treatment of a rare pediatric disease; and"(ii) that contains no active ingredient (including any ester or salt of the active ingredient) that has been previously approved in any other application under section 355(b)(1), 355(b)(2), or 355(j) of this title or section 351(a) or 351(k) of the Public Health Service Act;"(B) is submitted under section 355(b)(1) of this title or section 351(a) of the Public Health Service Act;". 2020-Subsec. (b)(5). Pub. L. 116-260 substituted "September 30, 2024" for "December 18, 2020" in introductory provisions and in subpar. (A) and substituted "September 30, 2026" for "December 18, 2022" in subpar. (B). Pub. L. 116-215 substituted "December 18, 2020" for "December 11, 2020" in introductory provisions and in subpar. (A) and substituted "December 18, 2022" for "December 11, 2022" in subpar. (B). Pub. L. 116-159 substituted "December 11, 2020" for "September 30, 2020" in introductory provisions and in subpar. (A) and substituted "December 11, 2022" for "September 30, 2022" in subpar. (B).2016-Subsec. (a)(3)(A). Pub. L. 114-229, §2(a)(1)(A), amended subpar. (A) generally. Prior to amendment, subpar. (A) read as follows: "The disease primarily affects individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents."Subsec. (a)(4)(F). Pub. L. 114-229, §2(a)(1)(B), substituted "September 30, 2016" for "July 9, 2012". Subsec. (b)(4). Pub. L. 114-229, §2(a)(2)(A), added par. (4) and struck out former par. (4). Prior to amendment, text read as follows: "(A) 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 for the user fee to be assessed in accordance with this section."(B) 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 subparagraph (A) may transfer the voucher after such notification is provided, if such sponsor has not yet submitted the human drug application described in the notification."Subsec. (b)(5). Pub. L. 114-255 added par. (5) and struck out former par. (5). Prior to amendment, text read as follows: "The Secretary may not award any priority review vouchers under paragraph (1) after December 31, 2016." Pub. L. 114-229, §2(a)(2)(B), added par. (5) and struck out former par. (5). Prior to amendment, text read as follows: "The Secretary may not award any priority review vouchers under paragraph (1) after September 30, 2016."Subsec. (g). Pub. L. 114-229, §2(a)(3), inserted before period at end ",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 chapter with respect to the drug for which the application is made." 2015-Subsec. (b)(5). Pub. L. 114-113 substituted "September 30, 2016." for "the last day of the 1-year period that begins on the date that the Secretary awards the third rare pediatric disease priority voucher under this section."
STATUTORY NOTES AND RELATED SUBSIDIARIES
CONSTRUCTION Pub. L. 114-229, §2(b), Sept. 30, 2016, 130 Stat. 944, provided that: "Nothing in this Act [amending this section and enacting provisions set out as a note under section 301 of this title], 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 [Sept. 30, 2016]."