21 U.S.C. § 360aa

Recommendations for investigations of drugs for rare diseases or conditions

Read at: OLRCuscode.house.gov CornellLII GovInfogovinfo.gov JustiaTitle 21 CasesGoogle Scholar

(a) Request by sponsor; response by SecretaryThe 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, or(2) 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 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).

(June 25, 1938, ch. 675, § 525, as added Pub. L. 97–414, § 2(a), Jan. 4, 1983, 96 Stat. 2049; amended Pub. L. 99–91, § 3(a)(1), Aug. 15, 1985, 99 Stat. 387; Pub. L. 105–115, title I, § 125(b)(2)(F), (G), Nov. 21, 1997, 111 Stat. 2325, 2326.)Editorial NotesAmendments

1997—Subsec. (a). Pub. L. 105–115, § 125(b)(2)(G), struck out “, certification of such drug for such disease or condition under section 357 of this title,” before “or licensing of such drug” in closing provisions.

Subsec. (a)(1) to (3). Pub. L. 105–115, § 125(b)(2)(F), inserted “or” at end of par. (1), redesignated par. (3) as (2), and struck out former par. (2), which read as follows: “if the drug is an antibiotic, it may be certified for such disease or condition under section 357 of this title, or”.

1985—Subsec. (a). Pub. L. 99–91 struck out “or” at end of par. (1), inserted par. (2), redesignated former par. (2) as (3) and struck out “before” after “product,”, and in last sentence inserted provisions relating to certification of such drug for disease or condition under section 357 of this title and substituted “licensing of such drug for such disease or condition under section 262 of title 42” for “licensing under section 262 of title 42 for such disease or condition”.

Statutory Notes and Related SubsidiariesEffective Date of 1985 Amendment

Pub. L. 99–91, § 8, Aug. 15, 1985, 99 Stat. 392, provided that:“(a)General Rule.—Except as provided in subsection (b), this Act and the amendments made by this Act [amending this section, sections 360bb, 360cc, and 360ee of this title, and sections 295g–1 and 6022 of Title 42, The Public Health and Welfare, and enacting provisions set out as notes under section 301 of this title and section 236 of Title 42] shall take effect October 1, 1985.“(b)Exception.—The amendments made by sections 2, 3, and 6(a) [amending this section and sections 360bb and 360cc of this title] shall take effect on the date of the enactment of this Act [Aug. 15, 1985]. The amendment made by section 6(b) [amending section 6022 of Title 42] shall take effect October 19, 1984. The amendments made by section 7 [amending section 295g–1 of Title 42] shall take effect October 1, 1984 and shall cease to be in effect after September 30, 1985.”

Rare Disease Endpoint Advancement Pilot Program

Pub. L. 117–328, div. FF, title III, § 3208, Dec. 29, 2022, 136 Stat. 5821, provided that:“(a)In General.—The Secretary [of Health and Human Services] shall establish a pilot program under which the Secretary establishes procedures to provide increased interaction with sponsors of rare disease drug development programs for purposes of advancing the development of efficacy endpoints, including surrogate and intermediate endpoints, for drugs intended to treat rare diseases, including through—“(1) determining eligibility of participants for such program; and“(2) developing and implementing a process for applying to, and participating in, such a program.“(b)Public Workshops.—The Secretary shall conduct up to 3 public workshops, which shall be completed not later than September 30, 2026, to discuss topics relevant to the development of endpoints for rare diseases, which may include discussions about—“(1) novel endpoints developed through the pilot program established under this section; and“(2) as appropriate, the use of real world evidence and real world data to support the validation of efficacy endpoints, including surrogate and intermediate endpoints, for rare diseases.“(c)Reports.—“(1)Interim report.—Not later than September 30, 2026, the Secretary shall submit to the Committee on Health, Education, Labor, and Pensions of the Senate and the Committee on Energy and Commerce of the House of Representatives a report describing the completed and ongoing activities in the pilot program established under this section and public workshops described in subsection (b).“(2)Final report.—Not later than September 30, 2027, the Secretary shall submit to the Committee on Health, Education, Labor, and Pensions of the Senate and the Committee on Energy and Commerce of the House of Representatives a report describing the outcomes of the pilot program established under this section.“(d)Guidance.—Not later than September 30, 2027, the Secretary shall issue guidance describing best practices and strategies for development of efficacy endpoints, including surrogate and intermediate endpoints, for rare diseases.“(e)Sunset.—The Secretary may not accept any new application or request to participate in the program established by this section on or after October 1, 2027.”

ALS and Other Rare Neurodegenerative Disease Action Plan

Pub. L. 117–79, § 4, Dec. 23, 2021, 135 Stat. 1536, provided that:“(a)In General.—Not later than 6 months after the date of enactment of this Act [Dec. 23, 2021], the Commissioner of Food and Drugs shall publish on the website of the Food and Drug Administration an action plan describing actions the Food and Drug Administration intends to take during the 5-year period following publication of the plan with respect to program enhancements, policy development, regulatory science initiatives, and other appropriate initiatives to—“(1) foster the development of safe and effective drugs that improve or extend, or both, the lives of people living with amyotrophic lateral sclerosis and other rare neurodegenerative diseases; and“(2) facilitate access to investigational drugs for amyotrophic lateral sclerosis and other rare neurodegenerative diseases.“(b)Contents.—The initial action plan published under subsection (a) shall—“(1) identify appropriate representation from within the Food and Drug Administration to be responsible for implementation of such action plan;“(2) include elements to facilitate—“(A) interactions and collaboration between the Food and Drug Administration, including the review centers thereof, and stakeholders including patients, sponsors, and the external biomedical research community;“(B) consideration of cross-cutting clinical and regulatory policy issues, including consistency of regulatory advice and decisionmaking;“(C) identification of key regulatory science and policy issues critical to advancing development of safe and effective drugs; and“(D) enhancement of collaboration and engagement of the relevant centers and offices of the Food and Drug Administration with other operating divisions within the Department of Health and Human Services, the Partnership, and the broader neurodegenerative disease community; and“(3) be subject to revision, as determined appropriate by the Secretary of Health and Human Services.”

Review Groups on Rare Diseases and Neglected Diseases of the Developing World; Report; Guidance; Standards

Pub. L. 111–80, title VII, § 740, Oct. 21, 2009, 123 Stat. 2127, provided that:“(a) The Commissioner of Food and Drugs shall establish within the Food and Drug Administration a review group which shall recommend to the Commissioner of Food and Drugs appropriate preclinical, trial design, and regulatory paradigms and optimal solutions for the prevention, diagnosis, and treatment of rare diseases: Provided, That the Commissioner of Food and Drugs shall appoint individuals employed by the Food and Drug Administration to serve on the review group: Provided further, That members of the review group shall have specific expertise relating to the development of articles for use in the prevention, diagnosis, or treatment of rare diseases, including specific expertise in developing or carrying out clinical trials.“(b) The Commissioner of Food and Drugs shall establish within the Food and Drug Administration a review group which shall recommend to the Commissioner of Food and Drugs appropriate preclinical, trial design, and regulatory paradigms and optimal solutions for the prevention, diagnosis, and treatment of neglected diseases of the developing world: Provided, That the Commissioner of Food and Drugs shall appoint individuals employed by the Food and Drug Administration to serve on the review group: Provided further, That members of the review group shall have specific expertise relating to the development of articles for use in the prevention, diagnosis, or treatment of neglected diseases of the developing world, including specific expertise in developing or carrying out clinical trials: Provided further, That for the purposes of this section the term ‘neglected disease of the developing world’ means a tropical disease, as defined in section 524(a)(3) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360n(a)(3)).“(c) The Commissioner of Food and Drugs shall—“(1) submit, not later than 1 year after the date of the establishment of review groups under subsections (a) and (b), a report to Congress that describes both the findings and recommendations made by the review groups under subsections (a) and (b);“(2) issue, not later than 180 days after submission of the report to Congress under paragraph (1), guidance based on such recommendations for articles for use in the prevention, diagnosis, and treatment of rare diseases and for such uses in neglected diseases of the developing world; and“(3) develop, not later than 180 days after submission of the report to Congress under paragraph (1), internal review standards based on such recommendations for articles for use in the prevention, diagnosis, and treatment of rare diseases and for such uses in neglected diseases of the developing world.”

Study

Pub. L. 100–290, § 3(d), Apr. 18, 1988, 102 Stat. 91, directed Secretary of Health and Human Services to conduct a study to determine whether the application of subchapter B of chapter V of the Federal Food, Drug, and Cosmetic Act, 21 U.S.C. 360aa et seq. (relating to drugs for rare diseases and conditions), and 26 U.S.C. 28 (relating to tax credit) to medical devices or medical foods for rare diseases or conditions or to both was needed to encourage development of such devices and foods and report results of the study to Congress not later than one year after Apr. 18, 1988.

Congressional Findings

Pub. L. 97–414, § 1(b), Jan. 4, 1983, 96 Stat. 2049, provided that: “The Congress finds that—“(1) there are many diseases and conditions, such as Huntington’s disease, myoclonus, ALS (Lou Gehrig’s disease), Tourette syndrome, and muscular dystrophy which affect such small numbers of individuals residing in the United States that the diseases and conditions are considered rare in the United States;“(2) adequate drugs for many of such diseases and conditions have not been developed;“(3) drugs for these diseases and conditions are commonly referred to as ‘orphan drugs’;“(4) because so few individuals are affected by any one rare disease or condition, a pharmaceutical company 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 financial 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.”

Notes of Decisions

Cited in 11 cases (4 in the last 5 years), 1987–2025 · leading case: Eagle Pharm., Inc. v. Alex Azar, II, 952 F.3d 323 (D.C. Cir. 2020).

Sort: Relevance Newest Treatment

Eagle Pharm., Inc. v. Alex Azar, II, 952 F.3d 323 (D.C. Cir. 2020). · cites it 2× “(Eagle) as an “orphan drug” under the Orphan Drug Act (ODA), 21 U.S.C. §§ 360aa–360ee. In 2015, the FDA approved Eagle’s drug for marketing but denied Eagle’s request for a seven-year period of marketing exclusivity under 21 U.”

CLS Bank Int'l v. Alice Corp. Pty. Ltd., 717 F.3d 1269 (Fed. Cir. 2013). “5 The court 5 Illustration is seen in the Orphan Drug Act, 21 U.S.C. §360aa–360ee (1997), which provides patent-like exclusivity and is reported to have provided treatment for CLS BANK INTERNATIONAL v.”

Philadelphia Beverage Ass'n v. City of Philadelphia & Frank Breslin, in His Off. Capacity as Comm'r of the Philadelphia Dep't of Revenue, 164 A.3d 576 (Pa. Commw. Ct. 2017). “2 21 U.S.C. §§360aa—360ff-1. 2 The PBT defines “dealer” as “[a]ny person engaged in the business of selling sugar-sweetened beverages for retail sale within the City” and defines “distributor” as “[a]ny person who supplies sugar-sweetened beverages to a dealer.”

Catalyst Pharm., Inc. v. Xavier Becerra, 14 F.4th 1299 (11th Cir. 2021). “2049 (codified as amended at 21 U.S.C. §§ 360aa–360ee). The Orphan Drug Act 1 Catalyst named Alex Azar, Secretary of Health and Human Services; Norman Sharpless, Acting Commissioner of the FDA; the U.”

All. Sec. Prods., Inc. v. Fleming Co., 471 F. Supp. 2d 452 (S.D.N.Y. 2007). “21 U.S.C. § 360aa et seq. 34 . Id. § 360bb.”

Jazz Pharm., Inc. v. Robert F. Kennedy Jr., 141 F.4th 254 (D.C. Cir. 2025). · cites it 3× “2049 , 2049–51, 2056–57 (1983) (codified as amended at 21 U.S.C. §§ 360aa–360ee). After having found that pharmaceutical companies needed financial incentives to make drug development for rare diseases economically feasible, the Congress determined it was in the public interest…”

Genentech, Inc. v. Bowen, 676 F. Supp. 301 (D.D.C. 1987). “See 21 U.S.C. § 360aa(a) (orphan drug manufacturer may request from the FDA written recommendations for clinical and non-clinical tests necessary for approval).”

In Re Neopharm, Inc. Sec. Litig., 705 F. Supp. 2d 946 (N.D. Ill. 2010). “The Orphan Drug Act, codified at 21 U.S.C. § 360aa et seq., provides incentives to companies to develop and market drugs for rare diseases — ones affecting fewer than 200,000 people in the United States, or "for which there is no reasonable expectation that the cost of…”

Catalyst Pharm., Inc. v. Azar (S.D. Fla. 2020). · cites it 2× “2049 (1983); 21 U.S.C. §§ 360aa–360ee. A. Orphan Drug Act Lambert-Eaton Myasthenic Syndrome (“LEMS”) is an “orphan disease” — a disease that affects so few people compared to the general population that drug companies do not have the financial incentive to develop drugs to treat…”

United Therapeutics Corp. (Tax Ct. 2023). “The Orphan Drug Credit In 1983, approximately two years after first establishing the research credit, Congress enacted the orphan drug credit as part of the Orphan Drug Act, Pub.”

Zamfirova v. Amag Pharm., Inc. (D.N.J. 2021). “Plaintiffs indicate that the FDA’s review found that the 2003 trial (1) “failed to identify the optimal time to start taking Makena”; (2) “one study center accounted for nearly half of the subjects, calling into question the effectiveness of the study’s randomizations”; and (3)…”

— 21 U.S.C. § 360aa(a) — 2 cases

Eagle Pharm., Inc. v. Alex Azar, II, 952 F.3d 323 (D.C. Cir. 2020). “(Eagle) as an “orphan drug” under the Orphan Drug Act (ODA), 21 U.S.C. §§ 360aa–360ee. In 2015, the FDA approved Eagle’s drug for marketing but denied Eagle’s request for a seven-year period of marketing exclusivity under 21 U.”

Annotations are extracted automatically from the opinions in the Syfert caselaw corpus and ranked by authority, recency, and treatment. Dots show Syfertize treatment of the citing case itself.