21 USC 360 - Priority review to encourage treatments for rare pediatric diseases

View all text of Part B [§ 360aa - § 360ff-1]

§ 360ff. Priority review to encourage treatments for rare pediatric diseases

(a) DefinitionsIn this section:

(1) Priority review

(2) Priority review voucher

(3) Rare pediatric diseaseThe term “rare pediatric disease” means a disease that meets each of the following criteria:

(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.

(B) The disease is a rare disease or condition, within the meaning of section 360bb of this title.

(4) Rare pediatric disease product applicationThe term “rare pediatric disease product application” means a human drug application, as defined in section 379g(1) of this title, that—

(A) is for a drug or biological product that is for the prevention or treatment of a rare pediatric disease;

(B)

(i) is for such a drug—(I) that contains no active moiety (as defined by the Secretary in section 314.3 of title 21, Code of Federal Regulations (or any successor regulations)) that has been previously approved in any other application under subsection (b)(1), (b)(2), or (j) of section 355 of this title; and(II) that is the subject of an application submitted under section 355(b)(1) of this title; or

(ii) is for such a biological product—(I) that contains no active ingredient that has been previously approved in any other application under section 351(a) or 351(k) of the Public Health Service Act [42 U.S.C. 262(a), 262(k)]; and(II) that is the subject of an application submitted under section 351(a) of the Public Health Service Act [42 U.S.C. 262(a)];

(D) that 1

1 So in original. The word “that” probably should not appear.

relies on clinical data derived from studies examining a pediatric population and dosages of the drug intended for that population;

(E) that 1 does not seek approval for an adult indication in the original rare pediatric disease product application; and

(F) is approved after September 30, 2016.

(b) Priority review voucher

(1) In general

(2) Transferability

(A) In general

(B) Notification of transfer

(3) Limitation

(4) Notification

(A) Sponsor of a rare pediatric disease product

(i) In general

(ii) Applications submitted but not yet approvedThe 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—(I) such sponsor has submitted such rare pediatric disease product application—(aa) on or after the date that is 90 days after July 9, 2012; and(bb) on or before September 30, 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

(ii) Transfer after notice

(5) Termination of authorityThe Secretary may not award any priority review vouchers under paragraph (1) after December 20, 2024, unless the rare pediatric disease product application—

(A) is for a drug that, not later than December 20, 2024, is designated under subsection (d) as a drug for a rare pediatric disease; and

(B) is, not later than September 30, 2026, approved under section 355(b)(1) of this title or section 351(a) of the Public Health Service Act [42 U.S.C. 262(a)].

(1) In general

(2) Fee amountThe amount of the priority review user fee shall be determined each fiscal year by the Secretary, based on the difference between—

(A) the average cost incurred by the Food and Drug Administration in the review of a human drug application subject to priority review in the previous fiscal year; and

(B) the average cost incurred by the Food and Drug Administration in the review of a human drug application that is not subject to priority review in the previous fiscal year.

(3) Annual fee setting

(4) Payment

(A) In general

(B) Complete application

(5) Offsetting collectionsFees collected pursuant to this subsection for any fiscal year—

(A) shall be deposited and credited as offsetting collections to the account providing appropriations to the Food and Drug Administration; and

(B) shall not be collected for any fiscal year except to the extent provided in advance in appropriations Acts.

(d) Designation process

(1) In generalUpon the request of the manufacturer or the sponsor of a new drug, the Secretary may designate—

(A) the new drug as a drug for a rare pediatric disease; and

(B) the application for the new drug as a rare pediatric disease product application.

(2) Request for designation

(3) Determination by SecretaryNot later than 60 days after a request is submitted under paragraph (1), the Secretary shall determine whether—

(A) the disease or condition that is the subject of such request is a rare pediatric disease; and

(B) the application for the new drug is a rare pediatric disease product application.

(e) Marketing of rare pediatric disease products

(1) Revocation

(2) Postapproval production reportThe 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:

(A) The estimated population in the United States suffering from the rare pediatric disease.

(B) The estimated demand in the United States for such rare pediatric disease product.

(f) Notice and report

(1) Notice of issuance of voucher and approval of products under voucherThe 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:

(A) The Secretary issues a priority review voucher under this section.

(B) The Secretary approves a drug pursuant to 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 which the sponsor of the application used a priority review voucher under this section.

(2) NotificationIf, 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—

(A) notifying such Committees of the use of such voucher; and

(B) identifying the drug for which such priority review voucher is used.

(g) Eligibility for other programs

(h) Relation to other provisions

(i) GAO study and report

(1) Study

(A) In general

(B) Contents of studyIn conducting the study under subparagraph (A), the Comptroller General shall examine the following:

(i) The indications for which each rare disease product for which a priority review voucher was awarded was approved under section 355 of this title or section 351 of the Public Health Service Act [42 U.S.C. 262].

(ii) Whether, and to what extent, an unmet need related to the treatment or prevention of a rare pediatric disease was met through the approval of such a rare disease product.

(iii) The value of the priority review voucher if transferred.

(iv) Identification of each drug for which a priority review voucher was used.

(v) The length of the period of time between the date on which a priority review voucher was awarded and the date on which it was used.

(2) Report

(June 25, 1938, ch. 675, § 529, as added Pub. L. 112–144, title IX, § 908, July 9, 2012, 126 Stat. 1094; amended Pub. L. 114–113, div. A, title VII, § 765, Dec. 18, 2015, 129 Stat. 2286; Pub. L. 114–229, § 2(a), Sept. 30, 2016, 130 Stat. 943; Pub. L. 114–255, div. A, title III, § 3013(a), Dec. 13, 2016, 130 Stat. 1093; Pub. L. 116–159, div. C, title I, § 2105, Oct. 1, 2020, 134 Stat. 729; Pub. L. 116–215, div. B, title II, § 1211, Dec. 11, 2020, 134 Stat. 1045; Pub. L. 116–260, div. BB, title III, § 321, Dec. 27, 2020, 134 Stat. 2932; Pub. L. 117–9, § 1(a)(4), Apr. 23, 2021, 135 Stat. 257; Pub. L. 118–83, div. B, title II, § 202, Sept. 26, 2024, 138 Stat. 1538.)