2000 
[DOCID: f:s1379rs.txt]





                                                       Calendar No. 298
107th CONGRESS
  1st Session
                                S. 1379

                          [Report No. 107-129]

 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, Mr. Hatch,  Mr. Hollings, Mr. Bingaman, Mr. 
 Durbin, Mr. Jeffords, Mrs. Clinton, Mr. Smith of Oregon, Ms. Collins, 
and Mr. Kerry) introduced the following bill; which was read twice and 
  referred to the Committee on Health, Education, Labor, and Pensions

                           December 18, 2001

               Reported by Mr. Kennedy, with an amendment
 [Strike out all after the enacting clause and insert the part printed 
                               in italic]

_______________________________________________________________________

                                 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.

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

<DELETED>SECTION 1. SHORT TITLE.</DELETED>

<DELETED>    This Act may be cited as the ``Rare Diseases Act of 
2001''.</DELETED>

<DELETED>SEC. 2. FINDINGS AND PURPOSES.</DELETED>

<DELETED>    (a) Findings.--Congress makes the following 
findings:</DELETED>
        <DELETED>    (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.</DELETED>
        <DELETED>    (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.</DELETED>
        <DELETED>    (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.</DELETED>
        <DELETED>    (4) The Orphan Drug Act 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.</DELETED>
        <DELETED>    (5) Before 1983, some 38 orphan drugs had been 
        developed. Since the enactment of the Orphan Drug Act, 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.</DELETED>
        <DELETED>    (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.</DELETED>
        <DELETED>    (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.</DELETED>
        <DELETED>    (8) Notwithstanding such increases, funding for 
        rare diseases and disorders at the National Institutes of 
        Health has not increased appreciably.</DELETED>
        <DELETED>    (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.</DELETED>
        <DELETED>    (10) 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.</DELETED>
<DELETED>    (b) Purposes.--The purposes of this Act are to--</DELETED>
        <DELETED>    (1) amend the Public Health Service Act to 
        establish an Office of Rare Diseases at the National Institutes 
        of Health; and</DELETED>
        <DELETED>    (2) increase the national investment in the 
        development of diagnostics and treatments for patients with 
        rare diseases and disorders.</DELETED>

       <DELETED>TITLE I--NATIONAL INSTITUTES OF HEALTH</DELETED>

<DELETED>SEC. 101. NIH OFFICE OF RARE DISEASES.</DELETED>

<DELETED>    Title IV of the Public Health Service Act (42 U.S.C. 281 
et seq.) is amended by inserting after section 404D the 
following:</DELETED>

              <DELETED>``office of rare diseases</DELETED>

<DELETED>    ``Sec. 404E. (a) Establishment.--There is established 
within the Office of the Director of NIH an office to be known as the 
Office of Rare Diseases (in this section referred to as the `Office'), 
which shall be headed by a Director (in this section referred to as the 
`Director'), appointed by the Director of NIH.</DELETED>
<DELETED>    ``(b) Duties.--</DELETED>
        <DELETED>    ``(1) In general.--The Director of the Office 
        shall carry out the following:</DELETED>
                <DELETED>    ``(A) The Director shall recommend an 
                agenda for conducting and supporting research on rare 
                diseases through the national research institutes and 
                centers. The agenda shall provide for a broad range of 
                research and education activities, including scientific 
                workshops and symposia to identify research 
                opportunities for rare diseases.</DELETED>
                <DELETED>    ``(B) The Director shall, with respect to 
                rare diseases, promote coordination and cooperation 
                among the national research institutes and centers and 
                entities whose research is supported by such 
                institutes.</DELETED>
                <DELETED>    ``(C) The Director shall enter into 
                cooperative agreements with and make grants for 
                regional centers of excellence on rare diseases in 
                accordance with section 404F.</DELETED>
                <DELETED>    ``(D) The Director shall promote the 

2000
                sufficient allocation of the resources of the National 
                Institutes of Health for conducting and supporting 
                research on rare diseases.</DELETED>
                <DELETED>    ``(E) The Director shall promote and 
                encourage the establishment of a centralized 
                clearinghouse for rare and genetic disease information 
                that will provide understandable information about 
                these diseases to the public, medical professionals, 
                patients and families.</DELETED>
                <DELETED>    ``(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.</DELETED>
                <DELETED>    ``(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.</DELETED>
        <DELETED>    ``(2) Principal advisor regarding orphan 
        diseases.--With respect to rare diseases, the Director shall 
        serve as the principal advisor to the Director of NIH and shall 
        provide advice to other relevant agencies. The Director shall 
        provide liaison with national and international patient, health 
        and scientific organizations concerned with rare 
        diseases.</DELETED>
<DELETED>    ``(c) Definition.--For purposes of this section, the term 
`rare disease' means any disease or condition that affects less than 
200,000 persons in the United States.</DELETED>
<DELETED>    ``(d) Authorization of Appropriations.--For the purpose of 
carrying out this section, there are authorized to be appropriated 
$4,000,000 for fiscal year 2002, and such sums as may be necessary for 
each subsequent fiscal year.''.</DELETED>

<DELETED>SEC. 102. RARE DISEASE REGIONAL CENTERS OF 
              EXCELLENCE.</DELETED>

<DELETED>    Title IV of the Public Health Service Act (42 U.S.C. 281 
et seq.), as amended by section 101, is further amended by inserting 
after section 404E the following:</DELETED>

    <DELETED>``rare disease regional centers of excellence</DELETED>

<DELETED>    ``Sec. 404F. (a) Cooperative Agreements and Grants.--
</DELETED>
        <DELETED>    ``(1) In general.--The Director of the Office of 
        Rare Diseases (in this section referred to as the `Director') 
        shall enter into cooperative agreements with and make grants to 
        public or private nonprofit entities to pay all or part of the 
        cost of planning, establishing, or strengthening, and providing 
        basic operating support for regional centers of excellence for 
        clinical research into, training in, and demonstration of 
        diagnostic, prevention, control, and treatment methods for rare 
        diseases.</DELETED>
        <DELETED>    ``(2) Policies.--A cooperative agreement or grant 
        under paragraph (1) shall be entered into in accordance with 
        policies established by the Director of NIH.</DELETED>
<DELETED>    ``(b) Coordination With Other Institutes.--The Director 
shall coordinate the activities under this section with similar 
activities conducted by other national research institutes, centers and 
agencies of the National Institutes of Health and by the Food and Drug 
Administration to the extent that such institutes, centers and agencies 
have responsibilities that are related to rare diseases.</DELETED>
<DELETED>    ``(c) Uses for Federal Payments Under Cooperative 
Agreements or Grants.--Federal payments made under a cooperative 
agreement or grant under subsection (a) may be used for--</DELETED>
        <DELETED>    ``(1) staffing, administrative, and other basic 
        operating costs, including such patient care costs as are 
        required for research;</DELETED>
        <DELETED>    ``(2) clinical training, including training for 
        allied health professionals, continuing education for health 
        professionals and allied health professions personnel, and 
        information programs for the public with respect to rare 
        diseases; and</DELETED>
        <DELETED>    ``(3) clinical research and demonstration 
        programs.</DELETED>
<DELETED>    ``(d) Period of Support; Additional Periods.--Support of a 
center under subsection (a) may be for a period of not to exceed 5 
years. Such period may be extended by the Director for additional 
periods of not more than 5 years if the operations of such center have 
been reviewed by an appropriate technical and scientific peer review 
group established by the Director and if such group has recommended to 
the Director that such period should be extended.</DELETED>
<DELETED>    ``(e) Authorization of Appropriations.--For the purpose of 
carrying out this section, there are authorized to be appropriated 
$20,000,000 for fiscal year 2002, and such sums as may be necessary for 
each subsequent fiscal year.''.</DELETED>

       <DELETED>TITLE II--FOOD AND DRUG ADMINISTRATION</DELETED>

<DELETED>SEC. 201. GRANTS AND CONTRACTS FOR THE DEVELOPMENT OF ORPHAN 
              DRUGS.</DELETED>

<DELETED>    Subsection (c) of section 5 of the Orphan Drug Act (21 
U.S.C. 360ee(c)) is amended to read as follows:</DELETED>
<DELETED>    ``(c) For grants and contracts under subsection (a) there 
are authorized to be appropriated $25,000,000 for fiscal year 2002, and 
such sums as may be necessary for each subsequent fiscal 
year.''.</DELETED>

<DELETED>SEC. 202. TECHNICAL AMENDMENT.</DELETED>

<DELETED>    Section 527(a) of the Federal Food, Drug, and Cosmetic Act 
(21 U.S.C.360cc(a)) is amended in the matter following paragraph (2)--
</DELETED>
        <DELETED>    (1) by striking ``, of such certification,''; 
        and</DELETED>
        <DELETED>    (2) by striking ``, the issuance of the 
        certification,''.</DELETED>

SECTION 1. SHORT TITLE.

    This Act may be cited as the ``Rare Diseases Act of 2001''.

SEC. 2. FINDINGS AND PURPOSES.

    (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 instru
2000
mental in pressing Congress for 
        legislation to encourage the development of orphan drugs.
            (4) The Orphan Drug Act 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, 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.
            (10) 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 purposes of this Act are to--
            (1) amend the Public Health Service Act 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.

                 TITLE I--NATIONAL INSTITUTES OF HEALTH

SEC. 101. NIH OFFICE OF RARE DISEASES.

    Title IV of the Public Health Service Act (42 U.S.C. 281 et seq.) 
is amended by inserting after section 404D the following:

                       ``office of rare diseases

    ``Sec. 404E. (a) Establishment.--There is established within the 
Office of the Director of NIH an office to be known as the Office of 
Rare Diseases (in this section referred to as the `Office'), which 
shall be headed by a Director (in this section referred to as the 
`Director'), appointed by the Director of NIH.
    ``(b) Duties.--
            ``(1) In general.--The Director of the Office shall carry 
        out the following:
                    ``(A) The Director shall recommend an agenda for 
                conducting and supporting research on rare diseases 
                through the national research institutes and centers. 
                The agenda shall provide for a broad range of research 
                and education activities, including scientific 
                workshops and symposia to identify research 
opportunities for rare diseases.
                    ``(B) The Director shall, with respect to rare 
                diseases, promote coordination and cooperation among 
                the national research institutes and centers and 
                entities whose research is supported by such 
                institutes.
                    ``(C) The Director, in collaboration with the 
                directors of the other relevant institutes and centers 
                of the National Institutes of Health, shall enter into 
                cooperative agreements with and make grants for 
                regional centers of excellence on rare diseases in 
                accordance with section 404F.
                    ``(D) The Director shall promote the sufficient 
                allocation of the resources of the National Institutes 
                of Health for conducting and supporting research on 
                rare diseases.
                    ``(E) The Director shall promote and encourage the 
                establishment of a centralized clearinghouse for rare 
                and genetic disease information that will provide 
                understandable information about these diseases to the 
                public, medical professionals, patients and families.
                    ``(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.
            ``(2) Principal advisor regarding orphan diseases.--With 
        respect to rare diseases, the Director shall serve as the 
        principal advisor to the Director of NIH and shall provide 
        advice to other relevant agencies. The Director shall provide 
        liaison with national and international patient, health and 
        scientific organizations concerned with rare diseases.
    ``(c) Definition.--For purposes of this section, the term `rare 
disease' means any disease or condition that affects less than 200,000 
persons in the United States.
    ``(d) Authorization of Appropriations.--For the purpose of carrying 
out this section, there are authorized to be appropriated $4,000,000 
for fiscal year 2002, and such sums as may be necessary for each 
subsequent fiscal year.''.

SEC. 102. RARE DISEASE REGIONAL CENTERS OF EXCELLENCE.

    Title IV of the Public Health Service Act (42 U.S.C. 281 et seq.), 
as amended by section 101, is further amended by inserting after 
section 404E the following:

             ``rare disease regional centers of excellence

    ``Sec. 404F. (a) Cooperative Agreements and Grants.--
            ``(1) In general.--The Director of the Office of Rare 
        Diseases (in this section referred to as the `Director') shall, 
        in collaboration with the directors of the other relevant 
        institutes and centers of the National Institutes of Health, 
        enter into cooperative agreements with and make grants to 
        public or private nonprofit entities to pay all or part of the 
        cost of planning, establishing, or strengthening, and providing 
        basic operating support for regional centers of excellence for 
        clinical research into, training in, and demonstration of 
        diagnostic, prevention, control, and treatment methods for rare 
        diseases.
            ``(2) Policies.--A cooperative agreement or grant under 
        paragraph (1) shall be entered into in accordance with policies 
        established by the Director of NIH.
    ``(b) Coordination With Other Institutes.--The Director shall 
coordinate the activities under this section with similar activities 
conducted by other n
bb3
ational research institutes, centers and agencies 
of the National Institutes of Health and by the Food and Drug 
Administration to the extent that such institutes, centers and agencies 
have responsibilities that are related to rare diseases.
    ``(c) Uses for Federal Payments Under Cooperative Agreements or 
Grants.--Federal payments made under a cooperative agreement or grant 
under subsection (a) may be used for--
            ``(1) staffing, administrative, and other basic operating 
        costs, including such patient care costs as are required for 
        research;
            ``(2) clinical training, including training for allied 
        health professionals, continuing education for health 
        professionals and allied health professions personnel, and 
        information programs for the public with respect to rare 
        diseases; and
            ``(3) clinical research and demonstration programs.
    ``(d) Period of Support; Additional Periods.--Support of a center 
under subsection (a) may be for a period of not to exceed 5 years. Such 
period may be extended by the Director for additional periods of not 
more than 5 years if the operations of such center have been reviewed 
by an appropriate technical and scientific peer review group 
established by the Director and if such group has recommended to the 
Director that such period should be extended.
    ``(e) Authorization of Appropriations.--For the purpose of carrying 
out this section, there are authorized to be appropriated $20,000,000 
for fiscal year 2002, and such sums as may be necessary for each 
subsequent fiscal year.''.

                 TITLE II--FOOD AND DRUG ADMINISTRATION

SEC. 201. GRANTS AND CONTRACTS FOR THE DEVELOPMENT OF ORPHAN DRUGS.

    Subsection (c) of section 5 of the Orphan Drug Act (21 U.S.C. 
360ee(c)) is amended to read as follows:
    ``(c) For grants and contracts under subsection (a) there are 
authorized to be appropriated $25,000,000 for fiscal year 2002, and 
such sums as may be necessary for each subsequent fiscal year.''.

SEC. 202. TECHNICAL AMENDMENT.

    Section 527(a) of the Federal Food, Drug, and Cosmetic Act (21 
U.S.C.360cc(a)) is amended in the matter following paragraph (2)--
            (1) by striking ``, of such certification,''; and
            (2) by striking ``, the issuance of the certification,''.




                                                       Calendar No. 298

107th CONGRESS

  1st Session

                                S. 1379

                          [Report No. 107-129]

_______________________________________________________________________

                                 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.

_______________________________________________________________________

                           December 18, 2001

                       Reported with an amendment

0