Calendar No. 714

103D CONGRESS

2D SESSION S. 1203

A BILL

To establish a Center for Rare Disease Research in

O

purposes.the National Institutes of Health, and for other

CTOBER 5 (legislative day, SEPTEMBER 12), 1994

Reported with an amendment
II

Calendar No. 714

103D CONGRESS

2D SESSION S. 1203

[Report No. 103–399]

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

which was read twice and referred to the Committee on Labor and

Human Resources

OCTOBER 5 (legislative day, SEPTEMBER 12), 1994

Reported by Mr. KENNEDY, with an amendment

[Strike out all after the enacting clause and insert the part printed in italic]

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’’.
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•S 1203 RS

1 SEC. 2. ESTABLISHMENT OF CENTER FOR RARE DISEASE

2 RESEARCH.

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

4 (42 U.S.C. 287 et seq.) is amended by adding at the end

5 thereof the following new subpart:

6 ‘‘Subpart 4—Center for Rare Disease Research

7 ‘‘SEC. 486A. ESTABLISHMENT.

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

9 Office of the Director of the National Institutes of Health

10 a Center for Rare Disease Research (hereafter referred

11 to in this section as the ‘Center’). The Director of the Na12 tional Institutes of Health shall appoint an individual with

13 expertise in rare diseases to serve as the Director of

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

15 ‘Director’).

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

17 is to promote and coordinate the conduct of research on

18 rare diseases and to establish and manage a rare disease

19 research clinical database. In carrying out the purpose of

20 the Center, the Director shall—

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

22 training;

23 ‘‘(2) award grants and contracts;

24 ‘‘(3) identify projects of research on rare dis25 eases that should be conducted or supported by the

26 National Institutes of Health;
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•S 1203 RS

1 ‘‘(4) disseminate information among the insti2 tutes and the public on rare diseases;

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

4 on current clinical research projects for rare dis5 eases;

6 ‘‘(6) encourage the participation of a diversity

7 of individuals in the conduct of rare disease re8 search; and

9 ‘‘(7) coordinate the conduct of rare disease re10 search among all institutes and other Federal agen11 cies.

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

13 shall develop a comprehensive plan for the conduct and

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

15 ‘‘(1) identify current research activities con16 ducted or supported by the Federal Government and

17 private entities, opportunities and needs for addi18 tional research and priorities for such research;

19 ‘‘(2) make recommendations for the improved

20 coordination of research conducted by the Federal

21 Government among its agencies and private entities;

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

23 which little research has been conducted;

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

25 therapy and genetic transfers and develop a plan to
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1 enhance the extent of research on gene therapy, par2 ticularly for rare diseases;

3 ‘‘(5) determine the need for registries of re4 search subjects and epidemiological studies of rare

5 disease populations;

6 ‘‘(6) identify the obstacles to the development

7 of treatments for rare diseases; and

8 ‘‘(7) examine training and education require9 ments for physicians treating rare diseases.

10 ‘‘(d) COORDINATING COMMITTEE.—

11 ‘‘(1) ESTABLISHMENT.—The Secretary shall es12 tablish a Coordinating Committee on Rare Disease

13 Research (hereafter referred to as the ‘Coordinating

14 Committee’) to be composed of—

15 ‘‘(A) the Directors of the agencies and in16 stitutes of the National Institutes of Health;

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

18 serve as the chairperson of the Coordinating

19 Committee;

20 ‘‘(C) the Administrator of the Veterans

21 Administration;

22 ‘‘(D) the Secretary of Defense;

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

24 Drug Administration;
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1 ‘‘(F) the Administrator of the Health Care

2 Financing Administration; and

3 ‘‘(G) the Director of the Center for Dis4 ease Control and Prevention.

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

6 shall—

7 ‘‘(A) identify needs for research on rare

8 diseases;

9 ‘‘(B) estimate the funds needed during

10 each fiscal year to adequately fund research on

11 rare diseases;

12 ‘‘(C) encourage the agencies and Institutes

13 of the National Institutes of Health to support

14 and conduct research on rare diseases;

15 ‘‘(D) coordinate research on rare diseases

16 in the Federal Government in an effort to avoid

17 duplication and enhance research in areas tradi18 tionally not funded;

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

20 are able to participate as research subjects in

21 projects conducting research on rare diseases;

22 and

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

24 Secretary and the Congress a report concerning

25 the activities of the Coordinating Committee.
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1 ‘‘(e) NATIONAL ADVISORY BOARD ON RARE DISEASE

2 RESEARCH.—

3 ‘‘(1) ESTABLISHMENT.—The Director shall es4 tablish an advisory board to be known as the Advi5 sory Board on Rare Disease Research (hereafter re6 ferred to in this section as the ‘Board’).

7 ‘‘(2) DUTIES.—The Board shall review and as8 sess Federal research needs, priorities, activities,

9 funding and findings regarding rare diseases and

10 shall advise the Director on the development and im11 plementation of the research plan required under

12 subsection (c).

13 ‘‘(3) COMPOSITION.—The Board shall be com14 posed of 15 individuals to be appointed by the Direc15 tor of the National Institutes of Health, of which

16 eight individuals shall be representatives of health

17 and scientific disciplines with respect to rare dis18 eases and seven individuals shall be representing the

19 interest of individuals with rare diseases. Such indi20 viduals shall not be officers or employees of the

21 Federal Government.

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

23 the Coordinating Committee shall serve as ex officio

24 members of the Board.
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1 ‘‘(5) CHAIRPERSON.—The members of the

2 Board shall annually select an individual to serve as

3 the chairperson of the Board.

4 ‘‘(f) NATIONAL RARE DISEASE CLINICAL AND IN5 FORMATIONAL DATABASE.—

6 ‘‘(1) ESTABLISHMENT.—The Director shall es7 tablish a database that—

8 ‘‘(A) identifies the extent, location, and

9 sponsor of current research conducted on rare

10 diseases;

11 ‘‘(B) connects researchers of rare diseases

12 with patients needed as subjects for clinical

13 trials;

14 ‘‘(C) provides physicians and individuals

15 with information concerning the location and

16 sponsors of clinical trials on rare diseases; and

17 ‘‘(D) connects patients with support

18 groups in rare diseases.

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

20 that information in the database is available to the

21 general public.

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

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

24 $10,000,000 for each of the fiscal years 1994 through

25 1996.
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1 ‘‘(h) DEFINITION.—As used in this section, the term

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

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

4 SECTION 1. SHORT TITLE.

5 This Act may be cited as the ‘‘Office for Rare Disease

6 Research Act of 1994’’.

7 SEC. 2. ESTABLISHMENT OF OFFICE FOR RARE DISEASE

8 RESEARCH.

9 Part A of title IV of the Public Health Service Act

10 (42 U.S.C. 281 et seq.) is amended by adding at the end

11 thereof the following new section:

12 ‘‘SEC. 404F. OFFICE FOR RARE DISEASE RESEARCH.

13 ‘‘(a) ESTABLISHMENT.—There is established within

14 the Office of the Director of the National Institutes of

15 Health an office to be known as the Office for Rare Disease

16 Research (in this section referred to as the ‘Office’). The

17 Office shall be headed by a director, who shall be appointed

18 by the Director of the National Institutes of Health.

19 ‘‘(b) PURPOSE.—The purpose of the Office is to pro20 mote and coordinate the conduct of research on rare diseases

21 through a strategic research plan and to establish and man22 age a rare disease research clinical database.

23 ‘‘(c) ADVISORY COUNCIL.—The Secretary shall estab24 lish an advisory council for the purpose of providing advice

25 to the director of the Office concerning carrying out the
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1 strategic research plan and other duties under this section.

2 Section 222 shall apply to such council to the same extent

3 and in the same manner as such section applies to commit4 tees or councils established under such section.

5 ‘‘(d) DUTIES.—In carrying out subsection (b), the di6 rector of the Office shall—

7 ‘‘(1) develop a comprehensive plan for the con8 duct and support of research on rare diseases;

9 ‘‘(2) coordinate and disseminate information

10 among the institutes and the public on rare diseases;

11 ‘‘(3) support research training and encourage the

12 participation of a diversity of individuals in the con13 duct of rare disease research;

14 ‘‘(4) identify projects or research on rare diseases

15 that should be conducted or supported by the National

16 Institutes of Health;

17 ‘‘(5) develop and maintain a central database on

18 current government sponsored clinical research

19 projects for rare diseases;

20 ‘‘(6) determine the need for registries of research

21 subjects and epidemiological studies of rare disease

22 populations; and

23 ‘‘(7) prepare biennial reports on the activities

24 carried out or to be carried out by the Office and sub25 mit such reports to the Secretary and the Congress.’’.