21 U.S.C. § 360bb — Designation of drugs for rare diseases or

Page 394 TITLE 21—FOOD AND DRUGS § 360bb 1 See References in Text note below. ‘‘(4) because so few individuals are affected by any one rare disease or condition, a pharmaceutical com- pany which develops an orphan drug may reasonably expect the drug to generate relatively small sales in comparison to the cost of developing the drug and consequently to incur a financial loss; ‘‘(5) there is reason to believe that some promising orphan drugs will not be developed unless changes are made in the applicable Federal laws to reduce the costs of developing such drugs and to provide finan- cial incentives to develop such drugs; and ‘‘(6) it is in the public interest to provide such changes and incentives for the development of orphan drugs.’’ § 360bb. Designation of drugs for rare diseases or conditions (a) Request by sponsor; preconditions; ‘‘rare dis- ease 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 re- quest for designation of a drug shall be made be- fore the submission of an application under sec- tion 355(b) of this title for the drug, or the sub- mission of an application for licensing of the drug under section 262 of title 42. If the Sec- retary 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 ap- proved under section 355 of this title, or (B) 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 Sec- retary shall designate the drug as a drug for such disease or condition. A request for a des- ignation of a drug under this subsection shall contain the consent of the applicant to notice being given by the Secretary under subsection (b) 1 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 condi- tion 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 cir- cumstances as of the date the request for des- ignation of the drug under this subsection is made. (b) Notification of discontinuance of drug or ap- plication as condition 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 355(b) of this title or a li- cense 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 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 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) shall be made available to the public. (d) Regulations The Secretary shall by regulation promulgate procedures for the implementation of subsection (a). (June 25, 1938, ch. 675, § 526, as added Pub. L. 97–414, § 2(a), Jan. 4, 1983, 96 Stat. 2050; amended Pub. L. 98–551, § 4(a), Oct. 30, 1984, 98 Stat. 2817; Pub. L. 99–91, § 3(a)(2), Aug. 15, 1985, 99 Stat. 387; Pub. L. 100–290, § 2, Apr. 18, 1988, 102 Stat. 90; Pub. L. 105–115, title I, § 125(b)(2)(H), (I), Nov. 21, 1997, 111 Stat. 2326.) Editorial Notes REFERENCES IN TEXT Subsection (b), referred to in subsec. (a)(1), was redes- ignated as subsec. (c) of this section by Pub. L. 100–290, § 2(b), Apr. 18, 1988, 102 Stat. 90. AMENDMENTS 1997—Subsec. (a)(1). Pub. L. 105–115, § 125(b)(2)(H), struck out ‘‘the submission of an application for cer- tification of the drug under section 357 of this title,’’ before ‘‘or the submission of an application for licens- ing 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’’. Subsec. (b)(1). Pub. L. 105–115, § 125(b)(2)(I)(i), struck out ‘‘, a certificate was issued for the drug under sec- tion 357 of this title,’’ before ‘‘or a license was issued’’. 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’’. 1988—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 sub- mission 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.’’ Subsecs. (b) to (d). Pub. L. 100–290, § 2(b), added sub- sec. (b) and redesignated former subsecs. (b) and (c) as (c) and (d), respectively. 1985—Subsec. (a)(1). Pub. L. 99–91 struck out ‘‘or’’ at end of subpar. (A), struck out subpar. (B) and sub- stituted subpars. (B) and (C), and inserted ‘‘, certification,’’ after ‘‘approval’’. 1984—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 infre- quently in the United States that’’. Page 395 TITLE 21—FOOD AND DRUGS § 360cc 1 See References in Text note below. Statutory Notes and Related Subsidiaries EFFECTIVE DATE OF 1985 AMENDMENT 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. § 360cc. Protection for drugs for rare diseases or conditions (a) Exclusive approval, certification, or license Except as provided in subsection (b), if the Secretary— (1) approves an application filed pursuant to section 355 of this title, or (2) 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 Sec- retary may not approve another application under section 355 of this title or issue another li- cense under section 262 of title 42 for the same drug for the same disease or condition for a per- son who is not the holder of such approved appli- cation 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 li- cense. Section 355(c)(2) 1 of this title does not apply to the refusal to approve an application under the preceding sentence. (b) Exceptions During the 7-year period described in sub- section (a) for an approved application under section 355 of this title or license under section 262 of title 42, the Secretary may approve an ap- plication or issue a license for a drug that is otherwise the same, as determined by the Sec- retary, as the already approved drug for the same rare disease or condition if— (1) the Secretary finds, after providing the holder of exclusive approval or licensure no- tice and opportunity for the submission of views, that during such period the holder of the exclusive approval or licensure cannot en- sure 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) the holder provides the Secretary in writ- ing 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. (c) Condition of clinical superiority (1) In general If a sponsor of a drug that is designated under section 360bb of this title and is other- wise the same, as determined by the Sec- retary, as an already approved or licensed drug is seeking exclusive approval or exclusive licensure described in subsection (a) for the same rare disease or condition as the already approved drug, the Secretary shall require such sponsor, as a condition of such exclusive approval or licensure, to demonstrate that such drug is clinically superior to any already approved or licensed drug that is the same drug. (2) Definition For purposes of paragraph (1), the term ‘‘clinically superior’’ with respect to a drug means that the drug provides a significant therapeutic advantage over and above an al- ready approved or licensed drug in terms of greater efficacy, greater safety, or by pro- viding a major contribution to patient care. (3) Applicability This subsection applies to any drug des- ignated under section 360bb of this title for which an application was approved under sec- tion 355 of this title or licensed under section 262 of title 42 after August 18, 2017, regardless of the date on which such drug was designated under section 360bb of this title. (d) Regulations The Secretary may promulgate regulations for the implementation of subsection (c). Beginning on August 18, 2017, until such time as the Sec- retary promulgates regulations in accordance with this subsection, the Secretary may apply any definitions set forth in regulations that were promulgated prior to such date, to the ex- tent such definitions are not inconsistent with the terms of this section, as amended by such Act. (e) Demonstration of clinical superiority stand- ard To assist sponsors in demonstrating clinical superiority as described in subsection (c), the Secretary— (1) upon the designation of any drug under section 360bb of this title, shall notify the sponsor of such drug in writing of the basis for the designation, including, as applicable, any plausible hypothesis offered by the sponsor and relied upon by the Secretary that the drug is clinically superior to a previously approved drug; and (2) upon granting exclusive approval or li- censure under subsection (a) on the basis of a demonstration of clinical superiority as de- scribed in subsection (c), shall publish a sum- mary of the clinical superiority findings. (June 25, 1938, ch. 675, § 527, as added Pub. L. 97–414, § 2(a), Jan. 4, 1983, 96 Stat. 2050; amended Pub. L. 98–417, title I, § 102(b)(6), Sept. 24, 1984, 98 Stat. 1593; Pub. L. 99–91, §§ 2, 3(a)(3), Aug. 15, 1985, 99 Stat. 387, 388; Pub. L. 103–80, § 3(v), Aug. 13, 1993, 107 Stat. 778; Pub. L. 105–115, title I, § 125(b)(2)(J), (K), Nov. 21, 1997, 111 Stat. 2326; Pub. L. 107–281, § 4, Nov. 6, 2002, 116 Stat. 1993; Pub. L. 115–52, title VI, § 607(a), Aug. 18, 2017, 131 Stat. 1049; Pub. L. 116–260, div. BB, title III, § 323, Dec. 27, 2020, 134 Stat. 2933.) Editorial Notes REFERENCES IN TEXT Section 355(c)(2) of this title, referred to in subsec. (a), was redesignated as section 355(c)(1)(B) of this title by Pub. L. 98–417, title I, § 102(a)(2), Sept. 24, 1984, 98 Stat. 1592. This section, as amended by such Act, referred to in subsec. (d), means this section as amended by the FDA Reauthorization Act of 2017, Pub. L. 115–52. AMENDMENTS 2020—Subsec. (c)(3). Pub. L. 116–260 added par. (3).