§ 360aa. Recommendations for investigations of drugs for rare diseases or conditions.

• (a) Request by sponsor; response by Secretary

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 non-clinical and clinical investigations which must be conducted with the drug before -

• (1) it may be approved for such disease or condition under section 355 of this title,
• (2) if the drug is an antibiotic, it may be certified for such disease or condition under section 357 of this title, or
• (3) if the drug is a biological product, it may be licensed for such disease or condition under section 262 of title 42.

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 non-clinical 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 355 of this title, certification of such drug for such disease or condition under section 357 of this title, or licensing of such drug for such disease or condition under section 262 of title 42.

• (b) Regulations

The Secretary shall by regulation promulgate procedures for the implementation of subsection (a) of this section.

§ 360bb. Designation of drugs for rare diseases or conditions.

• (a) Request by sponsor; preconditions; ''rare disease or condition'' defined
  • (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 355(b) of this title for the drug, the submission of an application for certification of the drug under section 357 of this title, or the submission of an application for licensing of the drug under section 262 of title 42. 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 355 of this title,
    • (B) if a certification for such drug is issued under section 357 of this title, or
    • (C) if a license for such drug is issued under section 262 of title 42, 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) of this section 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) Notification of discontinuance of drug or application as condition

A designation of a drug under subsection (a) of this section shall be subject to the condition that -

• (1) if an application was approved for the drug under section 355(b) of this title, a certificate was issued for the drug under section 357 of this title, or a license was issued for the drug under section 262 of title 42, 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 355(b) of this title, a certificate has not been issued for the drug under section 357 of this title, or a license has not been issued for the drug under section 262 of title 42 and if preclinical investigations or investigations under section 355(i) of this title 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 355(b) of this title, approval of an application for certification under section 357 of this title, or approval of a license under section 262 of title 42.

• (c) Notice to public

Notice respecting the designation of a drug under subsection (a) of this section shall be made available to the public.

• (d) Regulations

The Secretary shall by regulation promulgate procedures for the implementation of subsection (a) of this section.

§ 360cc. Protection for drugs for rare diseases or conditions.

• (a) Exclusive approval, certification, or license

Except as provided in subsection (b) of this section, if the Secretary -

• (1) approves an application filed pursuant to section 355 of this title,
• (2) issues a certification under section 357 of this title, or
• (3) issues a license under section 262 of title 42 for a drug designated under section 360bb of this title for a rare disease or condition, the Secretary may not approve another application under section 355 of this title, issue another certification under section 357 of this title, or issue another license under section 262 of title 42 for such drug for such disease or condition for a person who is not the holder of such approved application, of such certification, or of such license until the expiration of seven years from the date of the approval of the approved application, the issuance of the certification, or the issuance of the license. Section 355(c)(2) of this title does not apply to the refusal to approve an application under the preceding sentence.

• (b) Exceptions

If an application filed pursuant to section 355 of this title is approved for a drug designated under section 360bb of this title for a rare disease or condition, if a certification is issued under section 357 of this title for such a drug, or if a license is issued under section 262 of title 42 for such a drug, the Secretary may, during the seven-year period beginning on the date of the application approval, of the issuance of the certification under section 357 of this title, or of the issuance of the license, approve another application under section 355 of this title, issue another certification under section 357 of this title, or issue a license under section 262 of title 42, for such drug for such disease or condition for a person who is not the holder of such approved application, of such certification, 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, of such certification, 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, issuance of other certifications, or the issuance of other licenses before the expiration of such seven-year period.

§ 360dd. Open protocols for investigations of drugs for rare diseases or conditions.

If a drug is designated under section 360bb of this title as a drug for a rare disease or condition and if notice of a claimed exemption under section 355(i) of this title 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.