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

Footnotes

1. 1 See References in Text note below.

Legislative history

Reconstructed from this section’s Code source credit and amendment notes, resolved against the local Statutes at Large. Editorial notes are evidence, not the law that made the change.

1. 1938

   Pub. L. 97–414 § 2(a) · 96 Stat. 2049

   Enacted · 21 U.S.C. § 360bb

   June 25, 1938, ch. 675, § 526, as added Pub. L. 97–414, § 2(a), Jan. 4, 1983, 96 Stat. 2050

   Source evidence

   Code source credit
   June 25, 1938, ch. 675, § 526, as added Pub. L. 97–414, § 2(a), Jan. 4, 1983, 96 Stat. 2050

   Enacted provision · exact public law section
   (a) Chapter V of the Federal Food, Drug, and Cosmetic Act is amended by adding at the end the following: “Subchapter B— Drugs for Rare Diseases or Conditions “Sec. 525. “recommendations for investigations of drugs for rare diseases or conditions (a) The sponsor of a drug for a disease or condition 21 USC 360aa. 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 505, or 21 USC 355. “(2) if the drug is a biological product, before it may be licensed for such disease or condition under section 351 of the 42 USC 262. 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 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 21 USC 355. drug for such disease or condition under section 505 or licensing under section 351 of the Public Health Service Act for such disease or condition. “(b) The Secretary shall by regulation promulgate procedures for the implementation of subsection (a). “Sec. 526. “designation of drugs for rare diseases 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. 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 the drug is a biological product, a license is issued under section 351 of the Public Health Service Act, the approval 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) “Rare disease or condition.” For purposes of paragraph (1), the term ‘rare disease or condition’ means any disease or condition which occurs so infrequently in the United States that 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) Notice. Notice respecting the designation of a drug under subsection (a) shall be made available to the public. “(c) The Secretary shall by regulation promulgate procedures for the implementation of subsection (a). “Sec. 527. “protection for unpatented drugs for rare diseases or conditions (a) Except as provided in subsection (b), if the Secretary— “(1) approves an application filed pursuant to section 505(b), 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 Ante, p. 2050. condition and for which a United States Letter of Patent may not be issued, the Secretary may not approve another application under section 505(b) or issue another license under section 351 of the 21 USC 355. 42 USC 262. 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 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(b) is approved for a drug designated under section 526 for a rare disease or condition or a license is issued under section 351 of the Public Health Service Act for such a drug and if a United States Letter of Patent may not be issued for the 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(b), or, if the drug is a biological product, 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. “Sec. 528. “open protocols for investigations of drugs for rare diseases or conditions If a drug is designated under section 526 as a 21 USC 360dd. 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.”.

2. 1984

   Health Promotion and Disease Prevention Amendments of 1984

   Pub. L. 98–551 § 4(a) · 98 Stat. 2815

   Substituted · 21 U.S.C. § 360bb(a)(2)

   Pub. L. 98–551 substituted “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” for “which occurs so infrequently in the United States that”

   Source evidence

   Code amendment note
   Subsec. (a)(2). Pub. L. 98–551 substituted “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” for “which occurs so infrequently in the United States that”.

   Enacted provision · exact public law section
   (a) Section 526(a)(2) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bb(a)(2)) is amended by striking out “which occurs so infrequently in the United States that” and inserting in lieu thereof “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”.

3. 1985

   Orphan Drug Amendments of 1985

   Pub. L. 99–91 § 3(a)(2) · 99 Stat. 387

   1. Struck · 21 U.S.C. § 360bb(a)(1)

      Pub. L. 99–91 struck out “or” at end of subpar. (A)

   2. Struck · 21 U.S.C. § 360bb(a)(1)

      Pub. L. 99–91 struck out subpar. (B)

   3. Substituted · 21 U.S.C. § 360bb(a)(1)

      Pub. L. 99–91 substituted subpars. (B) and (C)

   4. Inserted · 21 U.S.C. § 360bb(a)(1)

      Pub. L. 99–91 inserted “, certification,” after “approval”

   Source evidence

   Code amendment note
   Subsec. (a)(1). Pub. L. 99–91 struck out “or” at end of subpar. (A), struck out subpar. (B) and substituted subpars. (B) and (C), and inserted “, certification,” after “approval”.

   Enacted provision · exact public law section
   (2) Section 526(a)(1) of such Act (21 U.S.C. 360bb(a)(1)) is amended— (A) by striking out “or” at the end of subparagraph (A) and by striking out subparagraph (B) and inserting in lieu thereof the following: “(B) if a certification for such drug is issued under section 507, or “(C) if a license for such drug is issued under section 351 of the Public Health Service Act,”; and (B) by striking out “the approval or license” and inserting in lieu thereof “the approval, certification, or license”.

   Effective date note
   Amendment by Pub. L. 99–91 effective Aug. 15, 1985, see section 8(b) of Pub. L. 99–91, set out as a note under section 360aa of this title.

4. 1988

   Orphan Drug Amendments of 1988

   Pub. L. 100–290 § 2(a) · 102 Stat. 90

   Inserted · 21 U.S.C. § 360bb(a)(1)

   Pub. L. 100–290, § 2(a), inserted after first sentence “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.”

   Source evidence

   Code amendment note
   Subsec. (a)(1). Pub. L. 100–290, § 2(a), inserted after first sentence “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.”

   Enacted provision · exact public law section
   (a) Request.— Section 526(a)(1) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bb(a)(1) is amended by adding after the first sentence the following: “A request for designation of a drug shall be made before the submission of an application under section 505(b) for the drug, the submission of an application for certification of the drug under section 507, or the submission of an application for licensing of the drug under section 351 of the Public Health Service Act”.

5. 1988

   Orphan Drug Amendments of 1988

   Pub. L. 100–290 § 2(b) · 102 Stat. 90

   1. Added · 21 U.S.C. § 360bb(b)(d)

      Pub. L. 100–290, § 2(b), added subsec. (b)

   2. Redesignated · 21 U.S.C. § 360bb(b)(d)

      Pub. L. 100–290, § 2(b), redesignated former subsecs. (b) and (c) as (c) and (d), respectively

   Source evidence

   Code amendment note
   Subsecs. (b) to (d). Pub. L. 100–290, § 2(b), added subsec. (b) and redesignated former subsecs. (b) and (c) as (c) and (d), respectively.

   Enacted provision · exact public law section
   (b) Discontinuance.— Section 526 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bb) is amended by redesignating subsections (b) and (c) as subsections (c) and (d), respectively, and by adding after subsection (a) the following: “(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), a certificate was issued for the drug under section 507, or a license was issued forthedrugunder 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), a certificate has not been issued for the drug under section 507, 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), approval of an application for certification under section 507, or approval of a license under section 351 of the Public Health Service Act.”.

6. 1997

   Food and Drug Administration Modernization Act of 1997

   Pub. L. 105–115 § 125(b)(2)(H) · 111 Stat. 2296

   1. Struck · 21 U.S.C. § 360bb(a)(1)

      Pub. L. 105–115, § 125(b)(2)(H), struck out “the submission of an application for certification of the drug under section 357 of this title,” before “or the submission of an application for licensing of the drug” in introductory provisions

   2. Inserted · 21 U.S.C. § 360bb(a)(1)

      Pub. L. 105–115, § 125(b)(2)(H), inserted “or” at end of subpar. (A)

   3. Redesignated · 21 U.S.C. § 360bb(a)(1)

      Pub. L. 105–115, § 125(b)(2)(H), redesignated subpar. (C) as (B)

   4. Struck · 21 U.S.C. § 360bb(a)(1)

      Pub. L. 105–115, § 125(b)(2)(H), struck out former subpar. (B) which read as follows: “if a certification for such drug is issued under section 357 of this title, or”

   Source evidence

   Code amendment note
   Subsec. (a)(1). Pub. L. 105–115, § 125(b)(2)(H), struck out “the submission of an application for certification of the drug under section 357 of this title,” before “or the submission of an application for licensing of the drug” in introductory provisions, inserted “or” at end of subpar. (A), redesignated subpar. (C) as (B), and struck out former subpar. (B) which read as follows: “if a certification for such drug is issued under section 357 of this title, or”.

   Enacted provision · exact public law section
   (H) Section 526(a)(1) (21 U.S.C. 360bb) is amended by striking “the submission of an application for certification of the drug under section 507,”, by inserting “or” at the end of subparagraph (A), by striking subparagraph (B), and by redesignating subparagraph (C) as subparagraph (B).

7. 1997

   Food and Drug Administration Modernization Act of 1997

   Pub. L. 105–115 § 125(b)(2)(I)(i) · 111 Stat. 2296

   Struck · 21 U.S.C. § 360bb(b)(1)

   Pub. L. 105–115, § 125(b)(2)(I)(i), struck out “, a certificate was issued for the drug under section 357 of this title,” before “or a license was issued”

   Source evidence

   Code amendment note
   Subsec. (b)(1). Pub. L. 105–115, § 125(b)(2)(I)(i), struck out “, a certificate was issued for the drug under section 357 of this title,” before “or a license was issued”.

   Enacted provision · exact public law section
   (i) in paragraph (1), by striking “, a certificate was issued for the drug under section 507,”; and

8. 1997

   Food and Drug Administration Modernization Act of 1997

   Pub. L. 105–115 § 125(b)(2)(I)(ii) · 111 Stat. 2296

   Struck · 21 U.S.C. § 360bb(b)(2)

   Pub. L. 105–115, § 125(b)(2)(I)(ii), struck out “, a certificate has not been issued for the drug under section 357 of this title,” before “or a license has not been issued” and “, approval of an application for certification under section 357 of this title,” before “or approval of a license”

   Source evidence

   Code amendment note
   Subsec. (b)(2). Pub. L. 105–115, § 125(b)(2)(I)(ii), struck out “, a certificate has not been issued for the drug under section 357 of this title,” before “or a license has not been issued” and “, approval of an application for certification under section 357 of this title,” before “or approval of a license”.

   Enacted provision · exact public law section
   (ii) in paragraph (2) by striking “, a certificate has not been issued for the drug under section 507,” and by striking “, approval of an application for certification under section 507,”.

15 of 15 references resolved to an enrolled provision