II

103D CONGRESS

1ST SESSION S. 1203

To establish a Center for Rare Disease Research in the National Institutes

of Health, and for other purposes.

IN THE SENATE OF THE UNITED STATES

JULY 1 (legislative day, JUNE 30), 1993

Mr. HATFIELD introduced the following bill; which was read twice and

referred to the Committee on Labor and Human Resources

A BILL

To establish a Center for Rare Disease Research in 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 ‘‘Center for Rare

5 Disease Research Act of 1993’’.

6 SEC. 2. ESTABLISHMENT OF CENTER FOR RARE DISEASE

7 RESEARCH.

8 Part E of title IV of the Public Health Service Act

9 (42 U.S.C. 287 et seq.) is amended by adding at the end

10 thereof the following new subpart:
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1 ‘‘Subpart 4—Center for Rare Disease Research

2 ‘‘SEC. 486A. ESTABLISHMENT.

3 ‘‘(a) IN GENERAL.—There shall be established in the

4 Office of the Director of the National Institutes of Health

5 a Center for Rare Disease Research (hereafter referred

6 to in this section as the ‘Center’). The Director of the Na7 tional Institutes of Health shall appoint an individual with

8 expertise in rare diseases to serve as the Director of

9 the Center (hereafter referred to in this section as the

10 ‘Director’).

11 ‘‘(b) PURPOSE.—The general purpose of the Center

12 is to promote and coordinate the conduct of research on

13 rare diseases and to establish and manage a rare disease

14 research clinical database. In carrying out the purpose of

15 the Center, the Director shall—

16 ‘‘(1) conduct and support research and research

17 training;

18 ‘‘(2) award grants and contracts;

19 ‘‘(3) identify projects of research on rare dis20 eases that should be conducted or supported by the

21 National Institutes of Health;

22 ‘‘(4) disseminate information among the insti23 tutes and the public on rare diseases;

24 ‘‘(5) develop and maintain a central database

25 on current clinical research projects for rare dis26 eases;
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1 ‘‘(6) encourage the participation of a diversity

2 of individuals in the conduct of rare disease re3 search; and

4 ‘‘(7) coordinate the conduct of rare disease re5 search among all institutes and other Federal agen6 cies.

7 ‘‘(c) STRATEGIC RESEARCH PLAN.—The Director

8 shall develop a comprehensive plan for the conduct and

9 support of research on rare diseases. The plan shall—

10 ‘‘(1) identify current research activities con11 ducted or supported by the Federal Government and

12 private entities, opportunities and needs for addi13 tional research and priorities for such research;

14 ‘‘(2) make recommendations for the improved

15 coordination of research conducted by the Federal

16 Government among its agencies and private entities;

17 ‘‘(3) give emphasis to areas with respect to

18 which little research has been conducted;

19 ‘‘(4) examine the extent of research on gene

20 therapy and genetic transfers and develop a plan to

21 enhance the extent of research on gene therapy, par22 ticularly for rare diseases;

23 ‘‘(5) determine the need for registries of re24 search subjects and epidemiological studies of rare

25 disease populations;
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1 ‘‘(6) identify the obstacles to the development

2 of treatments for rare diseases; and

3 ‘‘(7) examine training and education require4 ments for physicians treating rare diseases.

5 ‘‘(d) COORDINATING COMMITTEE.—

6 ‘‘(1) ESTABLISHMENT.—The Secretary shall es7 tablish a Coordinating Committee on Rare Disease

8 Research (hereafter referred to as the ‘Coordinating

9 Committee’) to be composed of—

10 ‘‘(A) the Directors of the agencies and in11 stitutes of the National Institutes of Health;

12 ‘‘(B) the Director of the Center who shall

13 serve as the chairperson of the Coordinating

14 Committee;

15 ‘‘(C) the Administrator of the Veterans

16 Administration;

17 ‘‘(D) the Secretary of Defense;

18 ‘‘(E) the Administrator of the Food and

19 Drug Administration;

20 ‘‘(F) the Administrator of the Health Care

21 Financing Administration; and

22 ‘‘(G) the Director of the Center for Dis23 ease Control and Prevention.

24 ‘‘(2) DUTIES.—The Coordinating Committee

25 shall—
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1 ‘‘(A) identify needs for research on rare

2 diseases;

3 ‘‘(B) estimate the funds needed during

4 each fiscal year to adequately fund research on

5 rare diseases;

6 ‘‘(C) encourage the agencies and Institutes

7 of the National Institutes of Health to support

8 and conduct research on rare diseases;

9 ‘‘(D) coordinate research on rare diseases

10 in the Federal Government in an effort to avoid

11 duplication and enhance research in areas tradi12 tionally not funded;

13 ‘‘(E) ensure that a diversity of individuals

14 are able to participate as research subjects in

15 projects conducting research on rare diseases;

16 and

17 ‘‘(F) biennially prepare and submit to the

18 Secretary and the Congress a report concerning

19 the activities of the Coordinating Committee.

20 ‘‘(e) NATIONAL ADVISORY BOARD ON RARE DISEASE

21 RESEARCH.—

22 ‘‘(1) ESTABLISHMENT.—The Director shall es23 tablish an advisory board to be known as the Advi24 sory Board on Rare Disease Research (hereafter re25 ferred to in this section as the ‘Board’).
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1 ‘‘(2) DUTIES.—The Board shall review and as2 sess Federal research needs, priorities, activities,

3 funding and findings regarding rare diseases and

4 shall advise the Director on the development and im5 plementation of the research plan required under

6 subsection (c).

7 ‘‘(3) COMPOSITION.—The Board shall be com8 posed of 15 individuals to be appointed by the Direc9 tor of the National Institutes of Health, of which

10 eight individuals shall be representatives of health

11 and scientific disciplines with respect to rare dis12 eases and seven individuals shall be representing the

13 interest of individuals with rare diseases. Such indi14 viduals shall not be officers or employees of the

15 Federal Government.

16 ‘‘(4) EX OFFICIO MEMBERS.—The members of

17 the Coordinating Committee shall serve as ex officio

18 members of the Board.

19 ‘‘(5) CHAIRPERSON.—The members of the

20 Board shall annually select an individual to serve as

21 the chairperson of the Board.

22 ‘‘(f) NATIONAL RARE DISEASE CLINICAL AND IN23 FORMATIONAL DATABASE.—

24 ‘‘(1) ESTABLISHMENT.—The Director shall es25 tablish a database that—
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1 ‘‘(A) identifies the extent, location, and

2 sponsor of current research conducted on rare

3 diseases;

4 ‘‘(B) connects researchers of rare diseases

5 with patients needed as subjects for clinical

6 trials;

7 ‘‘(C) provides physicians and individuals

8 with information concerning the location and

9 sponsors of clinical trials on rare diseases; and

10 ‘‘(D) connects patients with support

11 groups in rare diseases.

12 ‘‘(2) AVAILABILITY.—The Director shall ensure

13 that information in the database is available to the

14 general public.

15 ‘‘(g) AUTHORIZATION OF APPROPRIATIONS.—There

16 are authorized to be appropriated to carry out this section,

17 $10,000,000 for each of the fiscal years 1994 through

18 1996.

19 ‘‘(h) DEFINITION.—As used in this section, the term

20 ‘rare disease’ means any disease or condition that affects

21 fewer than 200,000 individuals in the United States.’’.

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