Title 21 — Food and Drugs
Part B · Chapter 9 — Federal Food, Drug, and Cosmetic Act
§ 360aa. Recommendations for investigations of drugs for rare diseases or conditions
Statutory text
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—
it may be approved for such disease or condition under section 355 of this title, or
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.
Regulations
The Secretary shall by regulation promulgate procedures for the implementation of subsection (a).
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.
Pub. L. 97–414 § 2(a) · 96 Stat. 2049
Enacted · 21 U.S.C. § 360aa
June 25, 1938, ch. 675, § 525, as added Pub. L. 97–414, § 2(a), Jan. 4, 1983, 96 Stat. 2049
Source evidence
Code source credit
June 25, 1938, ch. 675, § 525, as added Pub. L. 97–414, § 2(a), Jan. 4, 1983, 96 Stat. 2049Enacted 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.”.Orphan Drug Amendments of 1985
Pub. L. 99–91 § 3(a)(1) · 99 Stat. 387
Struck · 21 U.S.C. § 360aa(a)
Pub. L. 99–91 struck out “or” at end of par. (1)
Inserted · 21 U.S.C. § 360aa(a)
Pub. L. 99–91 inserted par. (2)
Redesignated · 21 U.S.C. § 360aa(a)
Pub. L. 99–91 redesignated former par. (2) as (3)
Struck and inserted · 21 U.S.C. § 360aa(a)
Pub. L. 99–91 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
Substituted · 21 U.S.C. § 360aa(a)
Pub. L. 99–91 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”
Source evidence
Code amendment note
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”.Enacted provision · exact public law section
(1) Section 525(a) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360aa(a)) is amended— (A) by striking out “or” at the end of paragraph (1), by redesignating paragraph (2) as paragraph (3), and by inserting after paragraph (1) the following: “(2) if the drug is an antibiotic, it may be certified for such disease or condition under section 507, or”; (B) by striking out “before” in paragraph (3) (as so redesignated); (C) by inserting after “505” in the last sentence a comma and the following: “certification of such drug for such disease or condition under section 507,”; and (D) by striking out “licensing under section 351 of the Public Health Service Act for such disease or condition” and inserting in lieu thereof “licensing of such drug for such disease or condition under section 351 of the Public Health Service Act”.Effective date note
Pub. L. 99–91, § 8, Aug. 15, 1985, 99 Stat. 392, provided that:Food and Drug Administration Modernization Act of 1997
Pub. L. 105–115 § 125(b)(2)(G) · 111 Stat. 2296
Struck · 21 U.S.C. § 360aa(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
Source evidence
Code amendment note
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.Enacted provision · exact public law section
(G) Section 525(a) (21 U.S.C. 360aa(a)) is amended by striking “, certification of such drug for such disease or condition under section 507,”.Food and Drug Administration Modernization Act of 1997
Pub. L. 105–115 § 125(b)(2)(F) · 111 Stat. 2296
Inserted · 21 U.S.C. § 360aa(a)(1)(3)
Pub. L. 105–115, § 125(b)(2)(F), inserted “or” at end of par. (1)
Redesignated · 21 U.S.C. § 360aa(a)(1)(3)
Pub. L. 105–115, § 125(b)(2)(F), redesignated par. (3) as (2)
Struck · 21 U.S.C. § 360aa(a)(1)(3)
Pub. L. 105–115, § 125(b)(2)(F), 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”
Source evidence
Code amendment note
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”.Enacted provision · exact public law section
(F) Section 525(a) (21 U.S.C. 360aa(a)) is amended by inserting “or” at the end of paragraph (1), by striking paragraph (2), and by redesignating paragraph (3) as paragraph (2).
10 of 10 references resolved to an enrolled provision
Editorial Notes
Amendments1985–1997 · 2 rounds
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 Subsidiaries
Effective Date of 1985 Amendment
Pub. L. 99–91, § 8, , 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 .
“(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 []. The amendment made by section 6(b) [amending section 6022 of Title 42] shall take effect . The amendments made by section 7 [amending section 295g–1 of Title 42] shall take effect and shall cease to be in effect after .”
Rare Disease Endpoint Advancement Pilot Program
Pub. L. 117–328, div. FF, title III, § 3208, , 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 , 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 , 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 , 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 , 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 .”
ALS and Other Rare Neurodegenerative Disease Action Plan
Pub. L. 117–79, § 4, , 135 Stat. 1536, provided that:
“(a) In General.—
Not later than 6 months after the date of enactment of this Act [], 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, , 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), , 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 .
Congressional Findings
Pub. L. 97–414, § 1(b), , 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.”
Credits
1938–1997 · 3 acts
- 1938June 25, 1938, ch. 675, § 525, as added Pub. L. 97–414, § 2(a), , 96 Stat. 2049
- 1985amended Pub. L. 99–91, § 3(a)(1), , 99 Stat. 387
- 1997Pub. L. 105–115, title I, § 125(b)(2)(F), (G), , 111 Stat. 2325, 2326.
Show as published
(June 25, 1938, ch. 675, § 525, as added Pub. L. 97–414, § 2(a), , 96 Stat. 2049; amended Pub. L. 99–91, § 3(a)(1), , 99 Stat. 387; Pub. L. 105–115, title I, § 125(b)(2)(F), (G), , 111 Stat. 2325, 2326.)