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107th Congress                                                   Report
                        HOUSE OF REPRESENTATIVES
 2d Session                                                     107-543

======================================================================



 
                       RARE DISEASES ACT OF 2002

                                _______
                                

 June 26, 2002.--Committed to the Committee of the Whole House on the 
              State of the Union and ordered to be printed

                                _______
                                

 Mr. Tauzin, from the Committee on Energy and Commerce, submitted the 
                               following

                              R E P O R T

                        [To accompany H.R. 4013]

  The Committee on Energy and Commerce, to whom was referred 
the bill (H.R. 4013) to amend the Public Health Service Act to 
establish an Office of Rare Diseases at the National Institutes 
of Health, and for other purposes, having considered the same, 
report favorably thereon without amendment and recommend that 
the bill do pass.

                                CONTENTS

                                                                   Page
Purpose and Summary..............................................     1
Background and Need for Legislation..............................     2
Hearings.........................................................     2
Committee Consideration..........................................     2
Committee Votes..................................................     2
Committee Oversight Findings.....................................     3
Statement of General Performance Goals and Objectives............     3
New Budget Authority, Entitlement Authority, and Tax Expenditures     3
Committee Cost Estimate..........................................     3
Congressional Budget Office Estimate.............................     3
Federal Mandates Statement.......................................     3
Advisory Committee Statement.....................................     3
Constitutional Authority Statement...............................     3
Applicability to Legislative Branch..............................     4
Section-by-Section Analysis of the Legislation...................     4
Changes in Existing Law Made by the Bill, as Reported............     4

                          Purpose and Summary

    The purpose of H.R. 4013, the Rare Diseases Act of 2002, is 
to authorize in statute the Office of Rare Diseases at the 
National Institutes of Health (``NIH''). Rare diseases are 
diseases affecting fewer than 200,000 individuals in the United 
States. The Director of the Office of Rare Diseases will serve 
as the principal advisor to the Director of the NIH with 
respect to rare diseases, and shall serve to promote sufficient 
allocation of NIH resources to rare disease research, and 
promote and encourage the establishment of a centralized 
clearinghouse for rare disease information for the benefit of 
the public, medical professionals, patients, and families.

                  Background and Need for Legislation

    In 1993, the NIH created an Office of Rare Diseases within 
the Office of the Director of NIH, to respond to the reporting 
requirements of the Orphan Drug Act of 1983. The Office of Rare 
Diseases implements the recommendations of the National 
Commission on Orphan Diseases, and responds to requests for 
information on rare diseases.
    Rare, or Orphan, Diseases affect fewer than 200,000 
individuals in America. Presently, more than 6,000 rare 
diseases affecting more than 25 million Americans are known. 
Prior to the enactment of the Orphan Disease Act of 1983, there 
was not enough of an incentive for drug and biological 
manufacturers to invest in treatments for these diseases. The 
Orphan Drug Act created greater incentives through increased 
exclusivity (7 years) for drugs and biologicals which treat 
Orphan Diseases, among other things.
    While the Orphan Drug Act has achieved dramatic increases 
in research into, and treatments for, rare diseases, more still 
needs to be done. One positive step includes authorizing in 
statute the Office of Rare Diseases. This action sends the 
strong signal of the Congress' commitment for both this Office 
as well as for rare disease research generally. Also, 
authorizing regional centers of excellence for rare disease 
research is a positive step, as it will enable the NIH to 
select sites to concentrate on finding cures and treatment 
methods for rare diseases. Both of these proposals are 
contained within H.R. 4013.

                                Hearings

    The Subcommittee on Health held a hearing on ``The National 
Institutes of Health: Investing in Research to Prevent and Cure 
Disease'' on June 6, 2002. The Subcommittee received testimony 
from Claude Lenfant, M.D., Director, National Heart, Lung, and 
Blood Institute; Audrey S. Penn, M.D., Acting Director, 
National Institute of Neurological Disorders and Stroke; Robert 
O. Bonow, M.D., President-elect, American Heart Association; 
Eric Hargis, President and CEO, The Epilepsy Foundation; Edward 
Sanchez, M.D., M.P.H., Commissioner, Texas Department of 
Health; Daniel Jones, M.D., Vice Chancellor, University Medical 
Center, University of Mississippi.

                        Committee Consideration

    On Wednesday, June 19, 2002, the Full Committee met in open 
markup session and favorably ordered reported H.R. 4013, the 
Rare Diseases Act of 2002, by voice vote, a quorum being 
present.

                            Committee Votes

    Clause 3(b) of rule XIII of the Rules of the House of 
Representatives requires the Committee to list the record votes 
on the motion to report legislation and amendments thereto. 
There were no record votes taken in connection with ordering 
H.R. 4013 reported. A motion by Mr. Tauzin to order H.R. 4013 
reported to the House, without amendment, was agreed to by a 
voice vote.

                      Committee Oversight Findings

    Pursuant to clause 3(c)(1) of rule XIII of the Rules of the 
House of Representatives, the Committee held an oversight 
hearing and made findings that are reflected in this report.

         Statement of General Performance Goals and Objectives

    The objective of this legislation is to increase support 
for rare disease research within and without the National 
Institutes of Health through the statutory authorization of the 
Office of Rare Diseases and by allowing the Secretary to 
establish rare disease regional centers of excellence.

   New Budget Authority, Entitlement Authority, and Tax Expenditures

    In compliance with clause 3(c)(2) of rule XIII of the Rules 
of the House of Representatives, the Committee finds that H.R. 
4103, would result in no new or increased budget authority, 
entitlement authority, or tax expenditures or revenues.

                        Committee Cost Estimate

    The Committee adopts as its own the cost estimate prepared 
by the Director of the Congressional Budget Office pursuant to 
section 402 of the Congressional Budget Act of 1974, which is 
included in the report to accompany H.R. 4984.

                  Congressional Budget Office Estimate

    Pursuant to clause 3(c)(3) of rule XIII of the Rules of the 
House of Representatives, the cost estimate provided by the 
Congressional Budget Office pursuant to section 402 of the 
Congressional Budget Act of 1974 is included in the report to 
accompany H.R. 4984.

                       Federal Mandates Statement

    The Committee adopts as its own the estimate of Federal 
mandates prepared by the Director of the Congressional Budget 
Office pursuant to section 423 of the Unfunded Mandates Reform 
Act. The estimate is included in the report to accompany H.R. 
4984.

                      Advisory Committee Statement

    No advisory committees within the meaning of section 5(b) 
of the Federal Advisory Committee Act were created by this 
legislation.

                   Constitutional Authority Statement

    Pursuant to clause 3(d)(1) of rule XIII of the Rules of the 
House of Representatives, the Committee finds that the 
Constitutional authority for this legislation is provided in 
Article I, section 8, clause 3, which grants Congress the power 
to regulate commerce with foreign nations, among the several 
States, and with the Indian tribes.

                  Applicability to Legislative Branch

    The Committee finds that the legislation does not relate to 
the terms and conditions of employment or access to public 
services or accommodations within the meaning of section 
102(b)(3) of the Congressional Accountability Act.

             Section-by-Section Analysis of the Legislation


Section 1. Short Title

    Section 1 establishes that the short title for this 
legislation is the Rare Diseases Act of 2002.

Section 2. Findings and Purposes

    Section 2 contains Congressional findings.

Section 3. NIH Office of Rare Diseases at National Institutes of Health

    Section 3 establishes within the Office of the Director of 
the NIH an office to be known as the Office of Rare Diseases, 
and ensures that the Director of the Office of Rare Diseases 
will (1) recommend an agenda for conducting and supporting 
research on rare diseases; (2) promote, with respect to rare 
diseases, coordination and cooperation among NIH institutes and 
centers; and, (3) promote the sufficient allocation of the 
resources of the NIH for conducting and supporting research on 
rare diseases. The section also authorizes $4 million in each 
of Fiscal Years 2003 through 2006 for these purposes.

Section 4. Rare Disease Regional Centers of Excellence

    Section 4 empowers the Director of the Office of Rare 
Diseases, in collaboration with the directors of the other 
relevant institutes and centers of the NIH, to enter into 
cooperative agreements and make grants for the establishment of 
regional centers of excellence for clinical research into, 
training in, and demonstration of diagnostic, prevention, 
control, and treatment methods for rare diseases. This section 
authorizes $20 million in each of Fiscal Years 2003 through 
2006 for these purposes.

         Changes in Existing Law Made by the Bill, as Reported

  In compliance with clause 3(e) of rule XIII of the Rules of 
the House of Representatives, changes in existing law made by 
the bill, as reported, are shown as follows (new matter is 
printed in italic and existing law in which no change is 
proposed is shown in roman):

                      PUBLIC HEALTH SERVICE ACT

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                 TITLE IV--NATIONAL RESEARCH INSTITUTES


                 Part A--National Institutes of Health

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                        OFFICE OF RARE DISEASES


  Sec. 404F. (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, 
                may enter into cooperative agreements with and 
                make grants for regional centers of excellence 
                on rare diseases in accordance with section 
                404G.
                  (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 such sums as already have been appropriated for 
fiscal year 2002, and $4,000,000 for each of the fiscal years 
2003 through 2006.


              rare disease regional centers of excellence


  Sec. 404G. (a) Cooperative Agreements and Grants.--
          (1) In general.--The Director of the Office of Rare 
        Diseases (in this section referred to as the 
        ``Director''), in collaboration with the directors of 
        the other relevant institutes and centers of the 
        National Institutes of Health, may 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 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.
  (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 such sums as already have been appropriated for 
fiscal year 2002, and $20,000,000 for each of the fiscal years 
2003 through 2006.

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