 |
 |
 |
FDA
Home Page | Search FDA Site |
FDA A-Z Index | Contact FDA
Contents of this page:
Sec. 524. Priority review to encourage treatments for tropical diseases
Sec. 525. Recommendations for Investigations of Drugs for Rare Diseases or Conditions
Sec. 526. Designation of Drugs for Rare Diseases or Conditions
Sec, 527. Protection for Drugs for Rare Diseases or Conditions
Sec. 528. Open Protocols for Investigations of Drugs for Rare Diseases or Conditions
Cross-reference of FD&C Act and U.S. Code section numbers
SEC. 524. [21 USC § 360n] Priority review to encourage treatments for tropical diseases
(a) Definitions. In this section:
(1) Priority review. The term "priority review", with respect to a human drug application as defined in section 735(1) [21 USC § 379g(l)], 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(c) of the Food and Drug Administration Amendments Act of 2007 [21 USC § 379g note].
(2) Priority review voucher. The term "priority review voucher" means a voucher issued by the Secretary to the sponsor of a tropical disease product application that entitles the holder of such voucher to priority review of a single human drug application submitted under section 505(b)(1) [21 USC § 355(b)(1)] or section 351 of the Public Health Service Act [42 USC § 262] after the date of approval of the tropical disease product application.
(3) Tropical disease. The term "tropical disease" means any of the following:
(A) Tuberculosis.
(B) Malaria.
(C) Blinding trachoma.
(D) Buruli Ulcer.
(E) Cholera.
(F) Dengue/dengue haemorrhagic fever.
(G) Dracunculiasis (guinea-worm disease).
(H) Fascioliasis.
(I) Human African trypanosomiasis.
(J) Leishmaniasis.
(K) Leprosy.
(L) Lymphatic filariasis.
(M) Onchocerciasis.
(N) Schistosomiasis.
(O) Soil transmitted helmithiasis.
(P) Yaws.
(Q) Any other infectious disease for which there is no significant market in developed nations and that disproportionately affects poor and marginalized populations, designated by regulation by the Secretary.
(4) Tropical disease product application. The term "tropical disease product application" means an application that--
(A) is a human drug application as defined in section 735(1) [21 USC § 379g(1)]--
(i) for prevention or treatment of a tropical disease; and
(ii) the Secretary deems eligible for priority review;
(B) is approved after the date of the enactment of the Food and Drug Administration Amendments Act of 2007 [enacted Sept. 27, 2007], by the Secretary for use in the prevention, detection, or treatment of a tropical disease; and
(C) is for a human drug, no active ingredient (including any ester or salt of the active ingredient) of which has been approved in any other application under section 505(b)(1) [21 USC § 355(b)(1)] or section 351 of the Public Health Service Act [42 USC § 262].
(b) Priority review voucher.
(1) In general. The Secretary shall award a priority review voucher to the sponsor of a tropical disease product application upon approval by the Secretary of such tropical disease product application.
(2) Transferability. The sponsor of a tropical disease product that receives a priority review voucher under this section may transfer (including by sale) the entitlement to such voucher to a sponsor of a human drug for which an application under section 505(b)(1) [21 USC § 355(b)(1)] or section 351 of the Public Health Service Act [42 USC § 262] will be submitted after the date of the approval of the tropical disease product application.
(3) Limitation.
(A) No award for prior approved application. A sponsor of a tropical disease product may not receive a priority review voucher under this section if the tropical disease product application was submitted to the Secretary prior to the date of the enactment of this section.
(B) One-year waiting period. The Secretary shall issue a priority review voucher to the sponsor of a tropical disease product no earlier than the date that is 1 year after the date of the enactment of the Food and Drug Administration Amendments Act of 2007 [enacted Sept. 27, 2007].
(4) Notification. The sponsor of a human drug application shall notify the Secretary not later than 365 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.
(c) Priority review user fee.
(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 chapter VII [21 USC §§ 371 et seq.].
(2) Fee amount. The amount of the priority review user fee shall be determined each fiscal year by the Secretary and based on the average cost incurred by the agency in the review of a human drug application 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, 2007, for that fiscal year, the amount of the priority review user fee.
(4) Payment.
(A) In general. The priority review user fee required by this subsection shall be due upon the submission of a human drug application under section 505(b)(1) [21 USC § 355(b)(1)] or section 351 of the Public Health Services Act [42 USC § 262] for which the priority review voucher is used.
(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.
(5) Offsetting collections. 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 appropriation Acts.
SEC. 525. [21 U.S.C. 360aa] Recommendations for investigations of drugs for rare diseases or conditions 1
(a) The sponsor of a drug for a disease or condition which is rare in the States may request the Secretary to provide written recommendations for the nonclinical and clinical investigations which must be conducted with the drug before—
(1) it may be approved for such disease or condition under section 505, or
(2) if the drug is a biological product, it may be licensed for such disease or condition under section 351 of the Public Health Service Act.
If the Secretary has reason to believe that a drug for which a request is made under this section is a drug for a disease or condition which is rare in the States, the Secretary shall provide the person making the request written recommendations for the nonclinical and clinical investigations which the Secretary believes, on the basis of information available to the Secretary at the time of the request under this section, would be necessary for approval of such drug for such disease or condition under section 505 or licensing of such drug for such disease or condition under section 351 of the Public Health Service Act.
(b) The Secretary shall by regulation promulgate procedures for the implementation of subsection (a).
SEC. 526 . [21 U.S.C. 360bb] Designation of drugs for rare disesases or conditions.
(a)(1) The manufacturer or the sponsor of a drug may request the Secretary to designate the drug as a drug for a rare disease or condition. A request for designation of a drug shall be made before the submission of an application under section 505(b) for the drug, or the submission of an application for licensing of the drug under section 351 of the Public Health Service Act. If the Secretary finds that a drug for which a request is submitted under this subsection is being or will be investigated for a rare disease or condition and—
(A) if an application for such drug is approved under section 505, or
(B) if a license for such drug is issued under section 351of the Public Health Service Act, the approval, certification, or license would be for use for such disease or condition, the Secretary shall designate the drug as a drug for such disease or condition. A request for a designation of a drug under this subsection shall contain the consent of the applicant to notice being given by the Secretary under subsection (b) respecting the designation of the drug.
(2) For purposes of paragraph (1), the term ‘‘rare disease or condition’’ means any disease or condition which (A) affects less than 200,000 persons in the United States, or (B) affects more than 200,000 in the United States and for which there is no reasonable expectation that the cost of developing and making available in the United States a drug for such disease or condition will be recovered from sales in the United States of such drug. Determinations under the preceding sentence with respect to any drug shall be made on the basis of the facts and circumstances as of the date the request for designation of the drug under this subsection is made.
(b) A designation of a drug under subsection (a) shall be subject to the condition that—
(1) if an application was approved for the drug under section 505(b) or a license was issued for the drug under section 351 of the Public Health Service Act, the manufacturer of the drug will notify the Secretary of any discontinuance of the production of the drug at least one year before discontinuance, and
(2) if an application has not been approved for the drug under section 505(b) or a license has not been issued for the drug under section 351 of the Public Health Service Act and if preclinical investigations or investigations under section 505(i) are being conducted with the drug, the manufacturer or sponsor of the drug will notify the Secretary of any decision to discontinue active pursuit of approval of an application under section 505(b) or approval of a license under section 351 of the Public Health Service Act.
(c) Notice respecting the designation of a drug under subsection (a) shall be made available to the public.
(d) The Secretary shall by regulation promulgate procedures for the implementation of subsection (a).
PROTECTION FOR DRUGS FOR RARE DISEASES OR CONDITIONS
SEC. 527 . [21 U.S.C. 360cc] (a) Except as provided in subsection (b), if the Secretary—
(1) approves an application filed pursuant to section 505, or
(2) issues a license under section 351 of the Public Health Service Act for a drug designated under section 526 for a rare disease or condition, the Secretary may not approve another application under section 505 or issue another license under section 351 of the Public Health Service Act for such drug for such disease or condition for a person who is not the holder of such approved application, of or of such license until the expiration of seven years from the date of the approval of the approved application, or the issuance of the license. Section 505(c)(2) does not apply to the refusal to approve an application under the preceding sentence.
(b) If an application filed pursuant to section 505 is approved for a drug designated under section 526 for a rare disease or condition or if a license is issued under section 351 of the Public Health Service Act for such a drug, the Secretary may, during the seven year period beginning on the date of the application approval or of the issuance of the license, approve another application under section 505 or issue a license under section 351 of the Public Health Service Act, for such drug for such disease or condition for a person who is not the holder of such approved application or of such license if—
(1) the Secretary finds, after providing the holder notice and opportunity for the submission of views, that in such period the holder of the approved application or of the license cannot assure the availability of sufficient quantities of the drug to meet the needs of persons with the disease or condition for which the drug was designated; or
(2) such holder provides the Secretary in writing the consent of such holder for the approval of other applications or the issuance of other licenses before the expiration of such seven year period.
OPEN PROTOCOLS FOR INVESTIGATIONS OF DRUGS FOR RARE DISEASES OR CONDITIONS
SEC. 528 . [21 U.S.C. 360dd] If a drug is designated under section 526 as a drug for a rare disease or condition and if notice of a claimed exemption under section 505(i) or regulations issued thereunder is filed for such drug, the Secretary shall encourage the sponsor of such drug to design protocols for clinical investigations of the drug which may be conducted under the exemption to permit the addition to the investigations of persons with the disease or condition who need the drug to treat the disease or condition and who cannot be satisfactorily treated by available alternative drugs.
1. Section 5 of the Orphan Drug Act (21 U.S.C. 360ee) (Public Law 97–414), which is included in the appendix, establishes a program to make grants and enter into contracts regarding the development of drugs for rare diseases and conditions. Authorizations of appropriations for that program are currently provided through fiscal year 2006. See section 3 of Public Law 107–281 (116 Stat. 1993).