II

107TH CONGRESS

1ST SESSION S. 1379

To amend the Public Health Service Act to establish an Office of Rare

Diseases at the National Institutes of Health, and for other purposes.

IN THE SENATE OF THE UNITED STATES

AUGUST 3, 2001

Mr. KENNEDY (for himself and Mr. HATCH) introduced the following bill;

which was read twice and referred to the Committee on Health, Education, Labor, and Pensions

A BILL

To amend the Public Health Service Act to establish an

Office of Rare Diseases at the National Institutes of

Health, and for other purposes.

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled,

3 SECTION 1. SHORT TITLE.

4 This Act may be cited as the ‘‘Rare Diseases Act of

5 2001’’.

6 SEC. 2. FINDINGS AND PURPOSES.

7 (a) FINDINGS.—Congress makes the following find8 ings:
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1 (1) Rare diseases and disorders are those which

2 affect small patient populations, typically popu3 lations smaller than 200,000 individuals in the

4 United States. Such diseases and conditions include

5 Huntington’s disease, amyotrophic lateral sclerosis

6 (Lou Gehrig’s disease), Tourette syndrome, Crohn’s

7 disease, cystic fibrosis, cystinosis, and Duchenne

8 muscular dystrophy.

9 (2) For many years, the 25,000,000 Americans

10 suffering from the over 6,000 rare diseases and dis11 orders were denied access to effective medicines be12 cause prescription drug manufacturers could rarely

13 make a profit from marketing drugs for such small

14 groups of patients. The prescription drug industry

15 did not adequately fund research into such treat16 ments. Despite the urgent health need for these

17 medicines, they came to be known as ‘‘orphan

18 drugs’’ because no companies would commercialize

19 them.

20 (3) During the 1970s, an organization called

21 the National Organization for Rare Disorders

22 (NORD) was founded to provide services and to

23 lobby on behalf of patients with rare diseases and

24 disorders. NORD was instrumental in pressing Con-
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1 gress for legislation to encourage the development of

2 orphan drugs.

3 (4) The Orphan Drug Act created financial in4 centives for the research and production of such or5 phan drugs. New federal programs at the National

6 Institutes of Health and the Food and Drug Admin7 istration encouraged clinical research and commer8 cial product development for products that target

9 rare diseases. An Orphan Products Board was estab10 lished to promote the development of drugs and de11 vices for rare diseases or disorders.

12 (5) Before 1983, some 38 orphan drugs had

13 been developed. Since the enactment of the Orphan

14 Drug Act, more than 220 new orphan drugs have

15 been approved and marketed in the United States

16 and more than 800 additional drugs are in the re17 search pipeline.

18 (6) Despite the tremendous success of the Or19 phan Drug Act, rare diseases and disorders deserve

20 greater emphasis in the national biomedical research

21 enterprise. The Office of Rare Diseases at the Na22 tional Institutes of Health was created in 1993, but

23 lacks a statutory authorization.

24 (7) The National Institutes of Health has re25 ceived a substantial increase in research funding
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1 from Congress for the purpose of expanding the na2 tional investment of the United States in behavioral

3 and biomedical research.

4 (8) Notwithstanding such increases, funding for

5 rare diseases and disorders at the National Insti6 tutes of Health has not increased appreciably.

7 (9) To redress this oversight, the Department

8 of Health and Human Services has proposed the es9 tablishment of a network of regional centers of excel10 lence for research on rare diseases.

11 (10) The Food and Drug Administration sup12 ports small clinical trials through Orphan Products

13 Research Grants. Such grants embody successful

14 partnerships of government and industry, and have

15 led to the development of at least 23 drugs and four

16 medical devices for rare diseases and disorders. Yet

17 the appropriations in Fiscal Year 2001 for such

18 grants were less than in Fiscal Year 1995.

19 (b) PURPOSES.—The purposes of this Act are to—

20 (1) amend the Public Health Service Act to es21 tablish an Office of Rare Diseases at the National

22 Institutes of Health; and

23 (2) increase the national investment in the de24 velopment of diagnostics and treatments for patients

25 with rare diseases and disorders.
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1 TITLE I—NATIONAL INSTITUTES

2 OF HEALTH

3 SEC. 101. NIH OFFICE OF RARE DISEASES.

4 Title IV of the Public Health Service Act (42 U.S.C.

5 281 et seq.) is amended by inserting after section 404D

6 the following:

7 ‘‘OFFICE OF RARE DISEASES

8 ‘‘SEC. 404E. (a) ESTABLISHMENT.—There is estab9 lished within the Office of the Director of NIH an office

10 to be known as the Office of Rare Diseases (in this section

11 referred to as the ‘Office’), which shall be headed by a

12 Director (in this section referred to as the ‘Director’), ap13 pointed by the Director of NIH.

14 ‘‘(b) DUTIES.—

15 ‘‘(1) IN GENERAL.—The Director of the Office

16 shall carry out the following:

17 ‘‘(A) The Director shall recommend an

18 agenda for conducting and supporting research

19 on rare diseases through the national research

20 institutes and centers. The agenda shall provide

21 for a broad range of research and education ac22 tivities, including scientific workshops and

23 symposia to identify research opportunities for

24 rare diseases.
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1 ‘‘(B) The Director shall, with respect to

2 rare diseases, promote coordination and co3 operation among the national research insti4 tutes and centers and entities whose research is

5 supported by such institutes.

6 ‘‘(C) The Director shall enter into coopera7 tive agreements with and make grants for re8 gional centers of excellence on rare diseases in

9 accordance with section 404F.

10 ‘‘(D) The Director shall promote the suffi11 cient allocation of the resources of the National

12 Institutes of Health for conducting and sup13 porting research on rare diseases.

14 ‘‘(E) The Director shall promote and en15 courage the establishment of a centralized

16 clearinghouse for rare and genetic disease infor17 mation that will provide understandable infor18 mation about these diseases to the public, med19 ical professionals, patients and families.

20 ‘‘(F) The Director shall biennially prepare

21 a report that describes the research and edu22 cation activities on rare diseases being con23 ducted or supported through the national re24 search institutes and centers, and that identi25 fies particular projects or types of projects that
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1 should in the future be conducted or supported

2 by the national research institutes and centers

3 or other entities in the field of research on rare

4 diseases.

5 ‘‘(G) The Director shall prepare the NIH

6 Director’s annual report to Congress on rare

7 disease research conducted by or supported

8 through the national research institutes and

9 centers.

10 ‘‘(2) PRINCIPAL ADVISOR REGARDING ORPHAN

11 DISEASES.—With respect to rare diseases, the Direc12 tor shall serve as the principal advisor to the Direc13 tor of NIH and shall provide advice to other relevant

14 agencies. The Director shall provide liaison with na15 tional and international patient, health and scientific

16 organizations concerned with rare diseases.

17 ‘‘(c) DEFINITION.—For purposes of this section, the

18 term ‘rare disease’ means any disease or condition that

19 affects less than 200,000 persons in the United States.

20 ‘‘(d) AUTHORIZATION OF APPROPRIATIONS.—For the

21 purpose of carrying out this section, there are authorized

22 to be appropriated $4,000,000 for fiscal year 2002, and

23 such sums as may be necessary for each subsequent fiscal

24 year.’’.
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1 SEC. 102. RARE DISEASE REGIONAL CENTERS OF EXCEL2 LENCE.

3 Title IV of the Public Health Service Act (42 U.S.C.

4 281 et seq.), as amended by section 101, is further amend5 ed by inserting after section 404E the following:

6 ‘‘RARE DISEASE REGIONAL CENTERS OF EXCELLENCE

7 ‘‘SEC. 404F. (a) COOPERATIVE AGREEMENTS AND

8 GRANTS.—

9 ‘‘(1) IN GENERAL.—The Director of the Office

10 of Rare Diseases (in this section referred to as the

11 ‘Director’) shall enter into cooperative agreements

12 with and make grants to public or private nonprofit

13 entities to pay all or part of the cost of planning, es14 tablishing, or strengthening, and providing basic op15 erating support for regional centers of excellence for

16 clinical research into, training in, and demonstration

17 of diagnostic, prevention, control, and treatment

18 methods for rare diseases.

19 ‘‘(2) POLICIES.—A cooperative agreement or

20 grant under paragraph (1) shall be entered into in

21 accordance with policies established by the Director

22 of NIH.

23 ‘‘(b) COORDINATION WITH OTHER INSTITUTES.—

24 The Director shall coordinate the activities under this sec25 tion with similar activities conducted by other national re26 search institutes, centers and agencies of the National In-
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1 stitutes of Health and by the Food and Drug Administra2 tion to the extent that such institutes, centers and agen3 cies have responsibilities that are related to rare diseases.

4 ‘‘(c) USES FOR FEDERAL PAYMENTS UNDER COOP5 ERATIVE AGREEMENTS OR GRANTS.—Federal payments

6 made under a cooperative agreement or grant under sub7 section (a) may be used for—

8 ‘‘(1) staffing, administrative, and other basic

9 operating costs, including such patient care costs as

10 are required for research;

11 ‘‘(2) clinical training, including training for al12 lied health professionals, continuing education for

13 health professionals and allied health professions

14 personnel, and information programs for the public

15 with respect to rare diseases; and

16 ‘‘(3) clinical research and demonstration pro17 grams.

18 ‘‘(d) PERIOD OF SUPPORT; ADDITIONAL PERIODS.—

19 Support of a center under subsection (a) may be for a

20 period of not to exceed 5 years. Such period may be ex21 tended by the Director for additional periods of not more

22 than 5 years if the operations of such center have been

23 reviewed by an appropriate technical and scientific peer

24 review group established by the Director and if such group
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1 has recommended to the Director that such period should

2 be extended.

3 ‘‘(e) AUTHORIZATION OF APPROPRIATIONS.—For the

4 purpose of carrying out this section, there are authorized

5 to be appropriated $20,000,000 for fiscal year 2002, and

6 such sums as may be necessary for each subsequent fiscal

7 year.’’.

8 TITLE II—FOOD AND DRUG

9 ADMINISTRATION

10 SEC. 201. GRANTS AND CONTRACTS FOR THE DEVELOP11 MENT OF ORPHAN DRUGS.

12 Subsection (c) of section 5 of the Orphan Drug Act

13 (21 U.S.C. 360ee(c)) is amended to read as follows:

14 ‘‘(c) For grants and contracts under subsection (a)

15 there are authorized to be appropriated $25,000,000 for

16 fiscal year 2002, and such sums as may be necessary for

17 each subsequent fiscal year.’’.

18 SEC. 202. TECHNICAL AMENDMENT.

19 Section 527(a) of the Federal Food, Drug, and Cos20 metic Act (21 U.S.C.360cc(a)) is amended in the matter

21 following paragraph (2)—

22 (1) by striking ‘‘, of such certification,’’; and

23 (2) by striking ‘‘, the issuance of the certifi24 cation,’’.

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