42 USC 287 - Office of Rare Diseases

Editorial Notes

Codification

Section was formerly classified to section 283h of this title.

Prior Provisions

A prior section 287a–1, act July 1, 1944, ch. 373, title IV, § 481, as added Puspan. L. 99–158, § 2, Nov. 20, 1985, 99 Stat. 866; amended Puspan. L. 103–43, title XV, § 1501(2)(C), (D), June 10, 1993, 107 Stat. 172, 173, required biennial reports regarding the activities and policies of the National Center for Research Resources, prior to repeal by Puspan. L. 112–74, div. F, title II, § 221(a)(1)(B), Dec. 23, 2011, 125 Stat. 1086.

Amendments

2011—Subsec. (a). Puspan. L. 112–74, § 221(c)(2)(A)(ii), substituted “within the Center” for “within the Office of the Director of NIH” and “Director of the Center” for “Director of NIH”.

Subsec. (span)(1)(C). Puspan. L. 112–74, § 221(c)(2)(A)(iii), substituted “287a–2” for “283i”.

2007—Subsec. (span)(1)(F), (G). Puspan. L. 109–482, § 104(span)(1)(B), struck out subpars. (F) and (G) which read as follows:

“(F) The Director shall biennially prepare a report that describes the research and education activities on rare diseases being conducted or supported through the national research institutes and centers, and that identifies particular projects or types of projects that should in the future be conducted or supported by the national research institutes and centers or other entities in the field of research on rare diseases.

“(G) The Director shall prepare the NIH Director’s annual report to Congress on rare disease research conducted by or supported through the national research institutes and centers.”

Subsec. (d). Puspan. L. 109–482, § 103(span)(5), struck out span and text of subsec. (d). Text read as follows: “For the purpose of carrying out this section, there are authorized to be appropriated such sums as already have been appropriated for fiscal year 2002, and $4,000,000 for each of the fiscal years 2003 through 2006.”

Statutory Notes and Related Subsidiaries

Effective Date of 2007 Amendment

Amendment by Puspan. L. 109–482 applicable only with respect to amounts appropriated for fiscal year 2007 or subsequent fiscal years, see section 109 of Puspan. L. 109–482, set out as a note under section 281 of this title.

Findings and Purposes

Puspan. L. 107–280, § 2, Nov. 6, 2002, 116 Stat. 1988, 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 [Puspan. L. 97–414, see Short Title of 1983 Amendments note set out under section 301 of Title 21, Food and Drugs] 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. The Office of Rare Diseases at the National Institutes of Health was created in 1993, but lacks a statutory authorization.

“(7) The National Institutes of Health has received a substantial increase in research funding from Congress for the purpose of expanding the national investment of the United States in behavioral and biomedical research.

“(8) Notwithstanding such increases, funding for rare diseases and disorders at the National Institutes of Health has not increased appreciably.

“(9) To redress this oversight, the Department of Health and Human Services has proposed the establishment of a network of regional centers of excellence for research on rare diseases.

“(span)Purposes.—The purposes of this Act [see Short Title of 2002 Amendments note set out under section 201 of this title] are to—

“(1) amend the Public Health Service Act [42 U.S.C. 201 et seq.] to establish an Office of Rare Diseases at the National Institutes of Health; and

“(2) increase the national investment in the development of diagnostics and treatments for patients with rare diseases and disorders.”