21 U.S.C. § 360ee (FOOD AND DRUGS)
§ 360ee. Grants and contracts for development of drugs for rare diseases and conditions
(a) Authority of Secretary
The Secretary may make grants to and enter into contracts with public and private entities and individuals to assist in (1) defraying the costs of developing drugs for rare diseases or conditions, including qualified testing expenses, (2) defraying the costs of developing medical devices for rare diseases or conditions, (3) defraying the costs of developing medical foods for rare diseases or conditions, and (4) developing regulatory science pertaining to the chemistry, manufacturing, and controls of individualized medical products to treat individuals with rare diseases or conditions.
(b) Definitions
For purposes of subsection (a):
(1) The term “qualified testing” means—
(A) human clinical testing—
(i) which is carried out under an exemption for a drug for a rare disease or condition under section 355(i) of this title (or regulations issued under such section); and
(ii) which occurs before the date on which an application with respect to such drug is submitted under section 355(b) of this title or under section 262 of title 42;
(B) preclinical testing involving a drug for a rare disease or condition which occurs after the date such drug is designated under section 360bb of this title and before the date on which an application with respect to such drug is submitted under section 355(b) of this title or under section 262 of title 42; and
(C) prospectively planned and designed observational studies and other analyses conducted to assist in the understanding of the natural history of a rare disease or condition and in the development of a therapy, including studies and analyses to—
(i) develop or validate a drug development tool related to a rare disease or condition; or
(ii) understand the full spectrum of the disease manifestations, including describing genotypic and phenotypic variability and identifying and defining distinct subpopulations affected by a rare disease or condition.
(2) The term “rare disease or condition” means (1) in the case of a drug, 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, (2) in the case of a medical device, any disease or condition that occurs so infrequently in the United States that there is no reasonable expectation that a medical device for such disease or condition will be developed without assistance under subsection (a), and (3) in the case of a medical food, any disease or condition that occurs so infrequently in the United States that there is no reasonable expectation that a medical food for such disease or condition will be developed without assistance under subsection (a). 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 section 360bb of this title is made.
(3) The term “medical food” means a food which is formulated to be consumed or administered enterally under the supervision of a physician and which is intended for the specific dietary management of a disease or condition for which distinctive nutritional requirements, based on recognized scientific principles, are established by medical evaluation.
(c) Authorization of appropriations
For grants and contracts under subsection (a), there is authorized to be appropriated $6,904,110 for the period beginning on October 1, 2022 and ending on December 23, 2022.1
(Pub. L. 97–414, § 5, Jan. 4, 1983, 96 Stat. 2056; Pub. L. 98–551, § 4(b), Oct. 30, 1984, 98 Stat. 2817; Pub. L. 99–91, § 5, Aug. 15, 1985, 99 Stat. 391; Pub. L. 100–290, § 3(a)–(c), Apr. 18, 1988, 102 Stat. 90, 91; Pub. L. 105–115, title I, § 125(b)(2)(N), Nov. 21, 1997, 111 Stat. 2326; Pub. L. 107–281, § 3, Nov. 6, 2002, 116 Stat. 1993; Pub. L. 110–85, title XI, § 1112(b), Sept. 27, 2007, 121 Stat. 976; Pub. L. 112–144, title IX, § 906, July 9, 2012, 126 Stat. 1092; Pub. L. 114–255, div. A, title III, § 3015, Dec. 13, 2016, 130 Stat. 1094; Pub. L. 115–52, title VI, § 603, Aug. 18, 2017, 131 Stat. 1048; Pub. L. 117–180, div. F, title V, § 5006, Sept. 30, 2022, 136 Stat. 2167; Pub. L. 117–229, div. C, title III, § 307, Dec. 16, 2022, 136 Stat. 2312; Pub. L. 117–328, div. FF, title III, § 3107, Dec. 29, 2022, 136 Stat. 5808.)
heading: Editorial Notes
heading: Codification
text: Section was enacted as part of the Orphan Drug Act, and not as part of the Federal Food, Drug, and Cosmetic Act which comprises this chapter.
heading: Amendments
text: 2022—Subsec. (a)(4). Pub. L. 117–328, § 3107(1), added par. (4).
Subsec. (c). Pub. L. 117–328, § 3107(2), which directed the substitution of “$30,000,000 for each of fiscal years 2023 through 2027” for “$6,328,767 for the period beginning on October 1, 2022, and ending on December 23, 2022”, could not be executed because “$6,328,767” did not appear after the intervening amendment by Pub. L. 117–229. See below.
Pub. L. 117–229 substituted “$6,904,110 for the period beginning on October 1, 2022 and ending on December 23, 2022” for “$6,328,767 for the period beginning on October 1, 2022, and ending on December 16, 2022”.
Pub. L. 117–180 substituted “$6,328,767 for the period beginning on October 1, 2022, and ending on December 16, 2022” for “$30,000,000 for each of fiscal years 2018 through 2022”.
2017—Subsec. (c). Pub. L. 115–52 substituted “2018 through 2022” for “2013 through 2017”.
2016—Subsec. (a)(1). Pub. L. 114–255, § 3015(1), added par. (1) and struck out former par. (1) which read as follows: “defraying the costs of qualified testing expenses incurred in connection with the development of drugs for rare diseases and conditions,”.
Subsec. (b)(1)(C). Pub. L. 114–255, § 3015(2), added subpar. (C).
2012—Subsec. (b)(1)(A)(ii). Pub. L. 112–144, § 906(a), struck out “after the date such drug is designated under section 360bb of this title and” after “which occurs”.
Subsec. (c). Pub. L. 112–144, § 906(b), amended subsec. (c) generally. Prior to amendment, text read as follows: “For grants and contracts under subsection (a), there is authorized to be appropriated $30,000,000 for each of fiscal years 2008 through 2012.”
2007—Subsec. (c). Pub. L. 110–85 amended subsec. (c) generally. Prior to amendment, subsec. (c) read as follows: “For grants and contracts under subsection (a) of this section, there are authorized to be appropriated such sums as already have been appropriated for fiscal year 2002, and $25,000,000 for each of the fiscal years 2003 through 2006.”
2002—Subsec. (c). Pub. L. 107–281 amended subsec. (c) generally. Prior to amendment, subsec. (c) read as follows: “For grants and contracts under subsection (a) of this section there are authorized to be appropriated $10,000,000 for fiscal year 1988, $12,000,000 for fiscal year 1989, $14,000,000 for fiscal year 1990.”
1997—Subsec. (b)(1)(A)(ii), (B). Pub. L. 105–115 struck out “or 357” after “355(b)”.
1988—Subsec. (a). Pub. L. 100–290, § 3(a)(1), (b)(1), inserted “(1)” after “assist in” and added pars. (2) and (3).
Subsec. (b)(2). Pub. L. 100–290, § 3(a)(2), (b)(2), inserted “(1) in the case of a drug,” after “means”, added cls. (2) and (3), and substituted “under section 360bb of this title” for “under this subsection” in last sentence.
Subsec. (b)(3). Pub. L. 100–290, § 3(b)(3), added par. (3).
Subsec. (c). Pub. L. 100–290, § 3(c), amended subsec. (c) generally. Prior to amendment, subsec. (c) read as follows: “For grants and contracts under subsection (a) of this section there are authorized to be appropriated $4,000,000 for fiscal year 1986, $4,000,000 for fiscal year 1987, and $4,000,000 for fiscal year 1988.”
1985—Subsec. (a). Pub. L. 99–91, § 5(a)(1), struck out “clinical” before “testing”.
Subsec. (b)(1). Pub. L. 99–91, § 5(a)(2), substituted provisions defining “qualified testing” for provisions defining “qualified clinical testing”.
Subsec. (c). Pub. L. 99–91, § 5(b), substituted provisions authorizing appropriations for fiscal years 1986 to 1988, for provisions authorizing appropriations for fiscal years 1983 and the two succeeding fiscal years.
1984—Subsec. (b)(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”.
heading: Statutory Notes and Related Subsidiaries
heading: Effective Date of 1985 Amendment
text: Amendment by Pub. L. 99–91 effective Oct. 1, 1985, see section 8(a) of Pub. L. 99–91, set out as a note under section 360aa of this title.
heading: Grants for Research on Therapies for ALS
text: Pub. L. 117–79, § 2, Dec. 23, 2021, 135 Stat. 1533, provided that:
“(a) In General.—
The Secretary of Health and Human Services (referred to in this section as the ‘Secretary’) shall award grants to participating entities for purposes of scientific research utilizing data from expanded access to investigational drugs for individuals who are not otherwise eligible for clinical trials for the prevention, diagnosis, mitigation, treatment, or cure of amyotrophic lateral sclerosis. In the case of a participating entity seeking such a grant, an expanded access request must be submitted, and allowed to proceed by the Secretary, under section 561 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb) and part 312 of title 21, Code of Federal Regulations (or any successor regulations), before the application for such grant is submitted.
“(b) Application.—
“(1) In general.—
A participating entity seeking a grant under this section shall submit to the Secretary an application at such time, in such manner, and containing such information as the Secretary shall specify.
“(2) Use of data.—
An application submitted under paragraph (1) shall include a description of how data generated through an expanded access request under section 561 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb) with respect to the investigational drug involved will be used to support research or development related to the prevention, diagnosis, mitigation, treatment, or cure of amyotrophic lateral sclerosis.
“(3) Noninterference with clinical trials.—
An application submitted under paragraph (1) shall include a description of how the proposed expanded access program will be designed so as not to interfere with patient enrollment in ongoing clinical trials for investigational therapies for the prevention, diagnosis, mitigation, treatment, or cure of amyotrophic lateral sclerosis.
“(c) Selection.—
Consistent with sections 406 and 492 of the Public Health Service Act (42 U.S.C. 284a, 289a), the Secretary shall, in determining whether to award a grant under this section, confirm that—
“(1) such grant will be used to support a scientific research objective relating to the prevention, diagnosis, mitigation, treatment, or cure of amyotrophic lateral sclerosis (as described in subsection (a));
“(2) such grant shall not have the effect of diminishing eligibility for, or impeding enrollment of, ongoing clinical trials for the prevention, diagnosis, mitigation, treatment, or cure of amyotrophic lateral sclerosis by determining that individuals who receive expanded access to investigational drugs through such a grant are not eligible for enrollment in—
“(A) ongoing clinical trials that are registered on ClinicalTrials.gov (or successor website), with respect to a drug for the prevention, diagnosis, mitigation, treatment, or cure of amyotrophic lateral sclerosis; or
“(B) clinical trials for the prevention, diagnosis, mitigation, treatment, or cure of amyotrophic lateral sclerosis for which an exemption under section 505(i) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(i)) has been granted by the Food and Drug Administration and which are expected to begin enrollment within one year; and
“(3) the resulting project funded by such grant will allow for equitable access to investigational drugs by minority and underserved populations.
“(d) Use of Funds.—
A participating entity shall use funds received through the grant—
“(1) to pay the manufacturer or sponsor for the direct costs of the investigational drug, as authorized under section 312.8(d) of title 21, Code of Federal Regulations (or successor regulations), to prevent, diagnose, mitigate, treat, or cure amyotrophic lateral sclerosis that is the subject of an expanded access request described in subsection (a), if such costs are justified as part of peer review of the grant;
“(2) for the entity’s direct costs incurred in providing such drug consistent with the research mission of the grant; or
“(3) for the direct and indirect costs of the entity in conducting research with respect to such drug.
“(e) Definitions.—
In this section:
“(1) The term ‘participating entity’ means a participating clinical trial site or sites sponsored by a small business concern (as defined in section 3(a) of the Small Business Act (15 U.S.C. 632(a))) that is the sponsor of a drug that is the subject of an investigational new drug application under section 505(i) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(i)) to prevent, diagnose, mitigate, treat, or cure amyotrophic lateral sclerosis.
“(2) The term ‘participating clinical trial’ means a phase 3 clinical trial conducted pursuant to an exemption under section 505(i) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(i)) or section 351(a) of the Public Health Service Act (42 U.S.C. 262(a)) to investigate a drug intended to prevent, diagnose, mitigate, treat, or cure amyotrophic lateral sclerosis.
“(3) The term ‘participating clinical trial site’ means a health care facility, or network of facilities, at which patients participating in a participating clinical trial receive an investigational drug through such trial.
“(f) Sunset.—
The Secretary may not award grants under this section on or after September 30, 2026.”
heading: Findings and Purposes
text: Pub. L. 107–281, § 2, Nov. 6, 2002, 116 Stat. 1992, provided that:
“(a) Findings.—
Congress makes the following findings:
“(1) Rare diseases and disorders are those which affect small patient populations, typically populations smaller than 200,000 individuals in the United States. Such diseases and conditions include Huntington’s disease, amyotrophic lateral sclerosis (Lou Gehrig’s disease), Tourette syndrome, Crohn’s disease, cystic fibrosis, cystinosis, and Duchenne muscular dystrophy.
“(2) For many years, the 25,000,000 Americans suffering from the over 6,000 rare diseases and disorders were denied access to effective medicines because prescription drug manufacturers could rarely make a profit from marketing drugs for such small groups of patients. The prescription drug industry did not adequately fund research into such treatments. Despite the urgent health need for these medicines, they came to be known as ‘orphan drugs’ because no companies would commercialize them.
“(3) During the 1970s, an organization called the National Organization for Rare Disorders (NORD) was founded to provide services and to lobby on behalf of patients with rare diseases and disorders. NORD was instrumental in pressing Congress for legislation to encourage the development of orphan drugs.
“(4) The Orphan Drug Act [see Short Title of 1983 Amendments note set out under section 301 of this title] created financial incentives for the research and production of such orphan drugs. New Federal programs at the National Institutes of Health and the Food and Drug Administration encouraged clinical research and commercial product development for products that target rare diseases. An Orphan Products Board was established to promote the development of drugs and devices for rare diseases or disorders.
“(5) Before 1983, some 38 orphan drugs had been developed. Since the enactment of the Orphan Drug Act [Jan. 4, 1983], more than 220 new orphan drugs have been approved and marketed in the United States and more than 800 additional drugs are in the research pipeline.
“(6) Despite the tremendous success of the Orphan Drug Act, rare diseases and disorders deserve greater emphasis in the national biomedical research enterprise.
“(7) The Food and Drug Administration supports small clinical trials through Orphan Products Research Grants. Such grants embody successful partnerships of government and industry, and have led to the development of at least 23 drugs and four medical devices for rare diseases and disorders. Yet the appropriations in fiscal year 2001 for such grants were less than in fiscal year 1995.
“(b) Purposes.—
The purpose of this Act [see Short Title of 2002 Amendments note set out under section 301 of this title] is to increase the national investment in the development of diagnostics and treatments for patients with rare diseases and disorders.”