In this section:

(1) Priority review 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.

(2) Priority review voucher 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:

(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.

The 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— (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 [42 U.S.C. 262(a), 262(k)];

(B) is 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)];

(C) the Secretary deems eligible for priority review;

(D) that [1] 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.

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—

(1) In general 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.

(A) In general 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.

(B) Notification of transfer 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.

(3) Limitation 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.

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—

(A) Sponsor of a rare pediatric disease product (i) In general 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. (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 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.

The Secretary may not award any priority review vouchers under paragraph (1) after September 30, 2020, unless the rare pediatric disease product application—

(A) is for a drug that, not later than September 30, 2020, is designated under subsection (d) as a drug for a rare pediatric disease; and

(B) is, not later than September 30, 2022, 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)].

The amount of the priority review user fee shall be determined each fiscal year by the Secretary, based on the difference between—

(1) In general 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—

(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 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). All other user fees associated with the human drug application shall be due as required by the Secretary or under applicable law.

(A) In general 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). All other user fees associated with the human drug application shall be due as required by the Secretary or under applicable law.

(B) Complete application 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.

(C) No waivers, exemptions, reductions, or refunds 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—

(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.

Upon the request of the manufacturer or the sponsor of a new drug, the Secretary may designate—

Upon 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 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—

(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.

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:

(1) Revocation 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:

(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.

(C) The actual amount of such rare pediatric disease product distributed in the United States.

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:

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:

(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.

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—

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

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

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..[2]

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.

In conducting the study under subparagraph (A), the Comptroller General shall examine the following:

In conducting the study under subparagraph (A), the Comptroller General shall examine the following:

(A) In general 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.

(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 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).

(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.)