Senate Report 107-129 - RARE DISEASES ACT OF 2001

[Senate Report 107-129]
[From the U.S. Government Publishing Office]

Calendar No. 298
107th Congress Report
SENATE
1st Session 107-129

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RARE DISEASES ACT OF 2001

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December 18, 2001.--Ordered to be printed

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Mr. Kennedy, from the Committee on Health, Education, Labor, and
Pensions, submitted the following

R E P O R T

[To accompany S. 1379]

The Committee on Health, Education, Labor, and Pensions, to
which was referred the bill (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,
having considered the same, reports favorably thereon with an
amendment and recommends that the bill (as amended) do pass.

CONTENTS

Page
I. Purpose and summary of the bill..................................1
II. Background and need for the legislation..........................3
III. Legislative history and committee action.........................5
IV. Explanation of the legislation and committee views...............5
V. Cost estimate....................................................6
VI. Application of law to the legislative branch.....................8
VII. Regulatory impact statement......................................8
VIII.Section-by-section analysis......................................8

IX. Changes in existing law.........................................11

I. Purpose and Summary of Bill

To address a longstanding unmet need to develop new
treatments, diagnostics, and cures for rare diseases and
disorders, Congress enacted the Orphan Drug Act of 1983 (Pub.
L. 97-414). This Act created financial incentives, such as
market exclusivity, tax credits, and research grants, for the
research and production of orphan drugs, and established the
Orphan Products Board at the Food and Drug Administration
(FDA). Congress sought through the Act to encourage the
development of new ``orphan'' treatments, diagnostics, and
cures for the millions of Americans with rare diseases who did
not have access to effective medicines because prescription
drug manufacturers were unlikely to develop and market drugs
for such small groups of patients. The term ``orphan drug''
refers to a product that treats a rare disease or disorder
affecting fewer than 200,000 Americans. Although the prevalence
of each disease is rare, approximately 25 million Americans
suffer from at least one of the 6,000 known rare diseases and
disorders. Increasing our knowledge of these diseases deepens
our understanding of the human body, and serves to benefit all
Americans.
Before the Orphan Drug Act, approximately 38 drugs existed
that were considered orphan drugs. Passage and implementation
of the Orphan Drug Act has led to the development of over 220
treatments for rare diseases and disorders. However, patients
with rare diseases or disorders face continuing challenges in
receiving appropriate and adequate treatment. Despite dramatic
increases in behavioral and biomedical research funding from
Congress for the National Institutes of Health (NIH) since
1998, significant opportunities for rare disease research
remain unmet. The Rare Diseases Act of 2001 (S. 1379) is
intended to build on the successes of, and improve upon,
current law in generating urgently needed treatments,
diagnostics, and cures for rare diseases and disorders. The Act
seeks to further stimulate the research and development of
orphan drugs, by giving statutory authorization to the Office
of Rare Diseases (ORD) at the NIH, and authorizing new funds
for rare disease research centers of excellence and the FDA's
Orphan Products Grant program.

1. THE LEGISLATION AUTHORIZES THE ESTABLISHMENT OF AN OFFICE OF RARE
DISEASES

The Office of Rare Diseases (ORD) was established in 1993
within the Office of the Director at the NIH to promote
research and collaboration on orphan diseases and respond to
requests for information. The ORD also has developed and
maintains a centralized database on rare diseases. The office
serves to bridge the gap between basic and translational
research, and to stimulate new research on rare diseases.
The Rare Diseases Act of 2001 provides a statutory
authorization for this important office. It authorizes the ORD
to recommend a research agenda through the national research
institutes and centers, and provide a broad range of research
and education activities, including scientific workshops and
symposia to identify research opportunities for rare diseases.
In addition, the Director of the ORD shall promote coordination
and cooperation among thenational research institutes and
collaboration among the directors of the relevant institutes and
centers of NIH. The Act doubles the authorization for the ORD's budget
to $4 million for FY02 and such sums as may be necessary for each
subsequent fiscal year.

2. THE LEGISLATION AUTHORIZES REGIONAL CENTERS OF EXCELLENCE FOR RARE
DISEASE RESEARCH AND TRAINING

The Act authorizes the NIH to make cooperative agreements
with, and make grants to, public or private nonprofit entities
for regional centers of excellence for clinical research into,
training in, and demonstration of diagnostic, prevention,
control, and treatment methods for rare diseases. It authorizes
$20 million for FY02 and such sums as may be necessary for each
subsequent fiscal year. Support of a center may not exceed 5
years, though extensions of not more than 5 years may be
granted.

3. THE LEGISLATION INCREASES FUNDING FOR THE ORPHAN PRODUCT RESEARCH
GRANT PROGRAM

The Act doubles the funding authorization for the FDA's
Orphan Product Research Grant program from $12.5 million
currently to $25 million for fiscal year 2002 and such sums as
may be necessary for each subsequent fiscal year. This will
provide crucial additional support for clinical research on new
treatments for rare diseases and disorders.

4. THE LEGISLATION INCLUDES A TECHNICAL AMENDMENT TO THE FEDERAL FOOD,
DRUG, AND COSMETIC ACT

The Act includes a technical amendment to section 527(a) of
the Federal Food, Drug, and Cosmetic Act.

II. Background and Need for the Legislation

Prior to the passage of the Orphan Drug Act in 1983,
Americans suffering from any of the 6,000 known rare diseases
largely did not have access to effective medicines because
prescription drug manufacturers did not have adequate
incentives to develop or market drugs for such small groups of
patients. Patients with rare diseases, such as Lou Gehrig's
disease and urea cycle disorders, went without treatment or
hope for a cure. Despite the need for these medicines, they
came to be known as ``orphan drugs'' because companies would
not or could not commercialize them.
The Orphan Drug Act provided seven years of market
exclusivity and expanded tax credits to companies for the
development and marketing of orphan drugs. The Act also
established the Office of Orphan Product Development at the
Food and Drug Administration, and created the Orphan Products
Grant Program, providing financial incentives to small
companies and research grants to academic scientists to support
pivotal clinical trials, research, and production of orphan
drugs, biologics, and devices. New programs at the National
Institutes of Health (NIH) and the Food and Drug Administration
(FDA) were created to encourage clinical research and
commercial product development of products that target rare
diseases. Since 1983, the Orphan Drug Act has been amended five
times to clarify terms, improve the implementation and
effectiveness of the Act, and convene a National Commission on
Orphan Diseases to assess the activities of public and private
agencies related to rare diseases.
The Orphan Drug Act is widely recognized as having
successfully encouraged greater research and the development of
new treatments of rare diseases. This has had substantial
benefits for the health and well-being of patients, improving
their quality of life and allowing them to return to school or
work, while reducing long-term health care costs. In the decade
before 1983, less than ten orphan drugs had been marketed;
since 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 under
development.
The FDA's Orphan Products Research Grants generally support
small clinical trials at academic institutions throughout the
nation to develop the preliminary evidence that is necessary to
attract commercial sponsors. It is a model of successful
government-industry partnership, and fills a major gap between
academic research and the private sector. The grants help to
create lifesaving products that are needed throughout the
world. Drug treatments for sickle-cell anemia and narcolepsy
are on the market today only because these grants supported
some of their research.
The creation of the Office of Rare Diseases (ORD) in 1993
formalized a mechanism within the NIH to stimulate research,
bridge the gap between basic and translational research, and
encourage the exchange of ideas among investigators, voluntary
patient support groups and NIH staff. In 1995, recognizing a
need to enhance ORD's operations, Senator Mark Hatfield
introduced the Office for Rare Disease Research Act, a bill to
formally establish this office at the NIH. This bill passed
through the Senate, but was not enacted into law.
The Orphan Drug Act both laid the foundation for, and
initiated a major leap in, our understanding and treatment of
rare diseases. Yet, more needs to be done. Without statutory
authorization, the Office of Rare Diseases and its mission have
received less attention than an NIH office might otherwise
receive from the biomedical research community. While the NIH
has received a substantial increase in funding from Congress
for the purpose of expanding the national investment of the
United States in behavioral and biomedical research,
significant opportunities for rare disease research remain
unmet.
Moreover, although the Orphan Products Grant Program
continues to attract research proposals, the funding for this
program is insufficient. Today, about 100 grant applications
are received annually, but those that would otherwise be funded
are not funded because of limited resources. The investment
necessary for research and development of new drugs and devices
is large in comparison to the size of the potential market for
a rare disease. For example, only 30,000 people suffer from
cystic fibrosis, and 600 from cystinosis; yet, for each of
these people, the need for treatment is urgent. Investing in
research on rare diseases is an important health priority. When
the Orphan Drug Act passed in 1983, only a few thousand people
had been diagnosed with AIDS. According to former Health and
Human Services Secretary Louis Sullivan, the response to the
HIV/AIDS pandemic, in terms of research and drug development,
was likely enhanced by the incentives authorized by the Orphan
Drug Act.
The Rare Diseases Act of 2001, which was introduced in
conjunction with legislation to expand the orphan drug tax
credit (S. 1341), addresses current obstacles in combating rare
diseases by providing statutory authorization and increasing
funding for the ORD to promote research and develop our
understanding and treatment of rare diseases; authorizing $20
million for regional centers of excellence to further stimulate
research and diagnose diseases; and increasing funding for the
Orphan Products Research Grant program to $25 million to fund
clinical trials that translate research into treatment and
cures.

III. Legislative History and Committee Action

On August 3rd, 2001, Senator Kennedy and Senator Hatch
introduced S. 1379, the ``Rare Diseases Act of 2001.'' The bill
was referred to the Senate Committee on Health, Education,
Labor, and Pensions.
On October 16, 2001, the committee held an executive
session to consider S. 1379, and ordered the bill to be
reported favorably with an amendment in the nature of a
substitute. The committee approved S. 1379, as amended, by
unanimous voice vote.

IV. Explanation of the Legislation and Committee Views

The committee recognizes that approximately 25 million
Americans suffer from rare diseases every day. The pain and
suffering from these diseases are compounded by well-grounded
concerns that research on, and the ongoing development of, new
cures and treatments for rare diseases is limited and needs to
be accelerated.

Statutory authorization for the Office of Rare Disorders

Before the creation of the Office or Rare Diseases (ORD) in
1993, information regarding rare diseases was spread throughout
the NIH based upon disease type and classification. As many
rare diseases affect multiple systems in the human body,
funding and responsibility often was diffused across different
institutes. In 1989, the National Commission on Orphan
Diseases, recommended that Congress establish a ``Central
Office of Orphan and Rare Diseases'' at the NIH. In response to
concerns raised by the Congress and patient advocates, the
Director of the NIH created the ORD to develop a research
agenda and clinical database to promote orphan drug product
research and development.
In January 2001, the NIH issued a report on steps to expand
and coordinate rare disease research, which included
recommendations of an NIH Special Emphasis Panel (SEP) on the
coordination of this research. The SEP recommended that the NIH
``establish a permanent presence for the ORD and its activities
at the NIH.'' The committee supports this recommendation. The
Rare Diseases Act of 2001 provides the ORD with increased
funding and statutory authorization to help meet these goals.

Grants for Centers of Excellence

The NIH Special Emphasis Panel also recommended that ``the
NIH should support the establishment of Specialized Research
and Diagnostic Centers of Excellence for Rare Diseases to
stimulate research and aid in the diagnosis of rare diseases.''
The Rare Diseases Act of 2001 authorizes the creation of, and
$20 million in funding for, such regional centers of
excellence. The committee supports this provision, which was
proposed in the Administration's FY 2002 budget request.
Regional centers of excellence provide critical opportunities
for research and collaboration throughout the country, which
are of particular importance to the study and treatment of rare
diseases where researchers may not be aware of each others'
efforts. These centers would also help patients receive an
accurate diagnosis, which often takes years. In 1989, the
National Commission on Orphan Diseases estimated that only a
third of patients receive an accurate diagnosis in the 3 to 5
years after the onset of symptoms; 15 percent of the population
is not accurately diagnosed until 7 or more years after the
onset of symptoms.

Orphan Products Grant Program

The FDA's Orphan Products Grant Program plays a critical
role in securing commercial sponsors for developing drugs. The
grants fund small clinical trials at academic institutions
throughout the nation to develop preliminary evidence, and are
a model of a successful government-industry partnership.
However, the funding for this program has not met the demand
created by qualified proposals. For these reasons, the
committee supports the provision in the Rare Diseases Act of
2001 that increases the funding for the Orphan Products Grant
Program from $12.5 million to $25 million for fiscal year 2002
and such sums as may be necessary for each subsequent fiscal
year. This will provide needed additional support for clinical
research on new treatments for rare diseases and disorders.

V. Cost Estimate

U.S. Congress,
Congressional Budget Office,
Washington, DC, December 5, 2001.
Hon. Edward M. Kennedy,
Chairman, Committee on Health, Education, Labor, and Pensions, U.S.
Senate, Washington, DC.
Dear Mr. Chairman: The Congressional Budget Office has
prepared the enclosed estimate of S. 1379, the Rare Diseases
Act of 2001.
If you wish further details on this estimate, we will be
pleased to provide them. The CBO staff contact is Christopher
J. Topoleski.
Sincerely,
Barry B. Anderson
(For Dan L. Crippen, Director).
Enclosure.

CONGRESSIONAL BUDGET OFFICE COST ESTIMATE

S. 1379--Rare Diseases Act of 2001

Summary: S. 1379 would provide for a statutory
authorization of the Office of Rare Diseases (ORD) within the
Office of the Director of the National Institutes of Health
(NIH). The bill would authorize NIH to provide grants to
regional ``Centers of Excellence'' to conduct research and
training related to rare diseases and it would authorize
funding for an existing grant program administered by the Food
and Drug Administration (FDA) that sponsors clinical testing of
the safety and effectiveness of new products to treat or
diagnose rare diseases.
CBO estimates that implementing S. 1379 would cost $6
million in 2002 ad $190 million over the 2002-2006 period,
assuming the appropriation of the necessary amounts including
annual adjustments for inflation. (The five-year total would be
$181 million if such inflation adjustments are not made.) The
legislation would not affect direct spending or receipts;
therefore, pay-as-you-go procedures would not apply.
S. 1379 contains no intergovernmental or private sector
mandates as defined in the Unfunded Mandates Reform Act (UMRA).
State, local, and tribal governments could apply for and
receive grants authorized by the bill, and any costs they incur
would be voluntary.
Estimated cost to the Federal Government: The estimated
budgetary impact of S. 1379 is shown in the following table.
The costs of this legislation fall within budget function 550
(health).

----------------------------------------------------------------------------------------------------------------
By fiscal year in millions of dollars--
--------------------------------------------
2002 2003 2004 2005 2006
----------------------------------------------------------------------------------------------------------------
CHANGES IN SPENDING SUBJECT TO APPROPRIATION \1\

Estimated Authorization Level:
National Institutes of Health \2\.............................. 24 25 25 26 27
Food and Drug Administration \3\............................... 12 26 27 28 28
--------------------------------------------
Total........................................................ 36 51 52 54 55
============================================
Estimated Outlays:
National Institutes of Health.................................. 4 14 23 25 27
Food and Drug Administration................................... 2 16 26 27 26
--------------------------------------------
Total........................................................ 6 30 49 52 53
----------------------------------------------------------------------------------------------------------------
\1\ The amounts shown reflect adjustments for anticipation for those activities for which the bill would
authorize such sums as necessary. Without such inflation adjustments, the five-year changes in authorization
levels would total $232 million (instead of $248 million) and the changes in outlays would total $181 million
(instead of $190 million).
\2\ The 2002-2006 levels are CBO baseline projections, including adjustments for anticipated inflation, for the
NIH.
\3\ Estimated authorization level includes the incremental change from amounts appropriated thus far for 2002
for the FDA. The 2003-2006 levels are CBO baseline projections of the change in FDA authorization adjustments
for anticipated inflation.

Basis of estimate: The bill would provide statutory
authorization for the ORD, which was established in 1993 within
the Office of the Director at the NIH. The ORD was established
to respond to the reporting requirements of the Orphan Drug
Act, to implement the recommendations of the National
Commission on Orphan Diseases, and to respond to requests for
information on rate diseases. A rare disease is one which
typically affects fewer than 200,000 individuals in the United
States.
The bill would require ORD to promote the establishment of
a centralized clearinghouse to make data and information on
rare diseases available to the public, researchers, and
clinicians. The director of the ORD would be responsible for
preparing biennial and annual reports on the activities of the
office, its Centers for Excellence, and future research
opportunities.
The bill would authorize NIH to award grants and contracts
to public and nonprofit private entities known as Centers of
Excellence to conduct clinical research, training, diagnostic
prevention, control, and or treatment activities with respect
to rare diseases. The centers would be awarded renewable
contracts for up to five years for each contract period.
S. 1379 would authorize appropriation of $24 million for
those activities in 2002 and such sums as necessary for each
subsequent year. CBO estimates that implementing the provisions
affecting NIH would cost $2 million in 2002 and $97 million
over the 2002-2006 period, assuming appropriations of the
necessary amounts.
S. 1379 also would authorize funding for an existing grant
program administered by FDA that sponsors clinical studies on
the safety and effectiveness of new products to treat or
diagnose rare diseases. The amount appropriated thus far for
fiscal year 2002 for the current program is $13 million. The
bill would authorize the appropriation of $25 million in 2002
and such sums as necessary for each subsequent fiscal year.
Research grants awarded under the program would defray some
of the costs associated with clinical testing of certain orphan
drugs, biological, medical devices, and medical foods. An
orphan drug is a drug or biological that is used to treat or
diagnose an illness usually affecting fewer than 200,000 people
in the United States. Eligible medical devices and medical
foods include products for which there is no reasonable
expectation development without grant assistance because the
condition occurs relatively infrequently in the United States.
CBO estimates that implementing this provision for FDA
grants would cost an additional $4 million in 2002 and $93
million over the 2002-2006 period, assuming appropriation of
the necessary amounts.
Pay-as-you-go considerations: None.
Estimated impact on state, local, and tribal governments:
S. 1379 contains no intergovernmental mandates as defined in
UMRA. State, local, and tribal governments could apply for and
receive grants authorized by the bill, and any costs they incur
would be voluntary.
Estimated impact on the private sector: The bill contains
no private-sector mandates as defined in UMRA.
Estimates prepared by: Federal Costs: Christopher J.
Topoleski and Julia Christensen. Impact on State, Local, and
Tribal Governments: Leo Lex. Impact on the Private Sector:
Jennifer Bowman.
Estimate approved by: Peter H. Fontaine, Deputy Assistant
Director for Budget Analysis.

VI. Application of Law to the Legislative Branch

The Rare Diseases Act of 2001 authorizes and amends
sections of the Public Health Service Act and the Federal Food,
Drug, and Cosmetic Act. As such, it has no application to the
legislative branch.

VII. Regulatory Impact Statement

The committee has determined that this bill is unlikely to
impose any new increases in the regulatory burden of paperwork.

VIII. Section-by-Section Analysis

Section 1. Short title

This section provides the short title of the bill, the
``Rare Diseases Act of 2001.''

Section 2. Findings and purposes

Subsection (a) Findings. This subsection finds that: (1)
rare diseases affect small patient populations, typically less
than 200,000; (2) treatments for rare diseases were not
researched and developed by prescription drug manufacturers in
the past because the industry could rarely make a profit from
marketing such ``orphan drugs'' to small groups of patients;
(3) the National Organization for Rare Disorders (NORD) was
instrumental in pressing Congress for legislation to encourage
the development of orphan drugs; (4) The Orphan Drug Act and
new federal programs at NIH and FDA encouraged the development
of drugs for rare diseases; (5) since enactment of the Orphan
Drug Act, more than 220 new orphan drugs have been approved and
more than 800 other such drugs are in the research pipeline;
(6) rare diseases deserve greater emphasis and the Office of
Rare Diseases at NIH, created in 1993, lacks statutory
authorization; (7) NIH has received substantial increases in
research funding; (8) funding for rare disease at NIH has not
increased appreciably; (9) to redress this oversight, HHS has
proposed establishing a network of regional centers of
excellence for research on rare diseases; (10) the Orphan
Products Research Grants funded by FDA have led to the
development of 23 drugs and 4 medical devices for rare
diseases, yet the appropriation in FY2001 for such grants was
less than in FY1995.
Subsection (b) Purposes. This subsection states that the
purposes of the Act are to: (1) amend the Public Health Service
Act to establish an Office of Rare Diseases at NIH; and, (2)
increase the national investment in the development of
diagnostics and treatments for patients with rare diseases.

TITLE I--NATIONAL INSTITUTES OF HEALTH

Section 101. NIH Office of Rare Diseases

The act adds a new section 404E to Title IV of the Public
Health Service Act. The new section 404E establishes, within
the Office of the Director of NIH, the Office of Rare Diseases
which would be headed by a Director appointed by the Director
of NIH.
The Director of the Office of Rare Diseases would recommend
an agenda for conducting and supporting research on rare
diseases, including scientific workshops and symposia. The
Director would promote coordination and cooperation on the
topic of rare diseases research among the national research
institutes and centers and entities whose research is supported
by such institutes. The Director, in collaboration with the
directors of the other relevant institutes and centers of the
NIH, would enter into cooperative agreements with and make
grants for regional centers of excellence on rare diseases.
Sufficient allocation of NIH resources for research on rare
diseases would be promoted by the Director. A centralized
clearinghouse for rare and genetic disease information would be
established to provide information about these diseases to the
public and medical professionals. The Director would biennially
prepare a report that describes the research and education
activities on rare diseases being conducted or supported
through the national research institutes andcenters, and that
identifies particular projects or types of projects that should in the
future be conducted or supported by these institutes and centers, or
other entities in the field of research on rare diseases. An annual
report to Congress would be prepared on rare disease research conducted
or supported by NIH. The Director of the Office of Rare Diseases would
serve as the principal advisor to the Director of NIH on the topic of
rare diseases and would serve as a liaison with outside organizations.
Rare diseases are defined as any disease or condition that
affects less than 200,000 persons in the United States.
An appropriation of $4,000,000 is authorized for fiscal
year 2002 and such sums as may be necessary for subsequent
fiscal years.

Section 102. Rare Disease Regional Centers of Excellence

The act adds a new section 404F to Title IV of the Public
Health Service Act. The new section 404F establishes the Rare
Disease Regional Centers of Excellence.
The Director of the Office of Rare Diseases, along with the
Directors of other relevant NIH institutes and centers, would
enter into cooperative agreements and make grants to public and
private nonprofit entities to plan, establish, or strengthen,
and provide basic operating support for regional centers of
excellence for clinical research on rare diseases. Such
clinical research would include training in and demonstration
of diagnostic, prevention, control, and treatment methods for
rare diseases.
The Director of the Office of Rare Disease would coordinate
the activities under section 404F with similar activities
conducted by the other national research institutes, centers,
and agencies of the NIH and by the FDA to the extent that such
institutes, centers, and agencies have responsibilities that
are related to rare diseases.
Federal payments made under such cooperative agreements or
grants may be used for staffing, administrative, and other
basic operating costs including such patient care costs as are
required for research. Federal payments may also be used for
clinical training, continuing education for health
professionals, and information programs for the public on rare
diseases, as well as clinical research and demonstration
programs.
Support of such centers would not exceed a period of 5
years. The Director may extend support for additional periods
of not more than 5 years if the operations of such a center
have been peer reviewed by an appropriate technical and
scientific group established by the Director, and the group has
recommended that support should be extended.
An appropriation of $20,000,000 is authorized for fiscal
year 2002 and such sums as may be necessary for subsequent
fiscal years.

TITLE II--FOOD AND DRUG ADMINISTRATION

Section 201. Grants and contracts for the development of orphan drugs

The act amends subsection (c) of section 5 of the Orphan
Drug Act, authorizing $25,000,000 for fiscal year 2002 for
grants and contracts, and such sums as may be necessary for
subsequent fiscal years.

Section 202. Technical amendment

In section 527(a) of the Federal Food, Drug, and Cosmetic
Act, two phrases containing the word ``certification'' are
struck from the matter following paragraph (2).

IX. Changes in Existing Law

In compliance with rule XXVI paragraph 12 of the Standing
Rules of the Senate, the following provides a print of the
statute or the part or section thereof to be amended or
replaced (existing law proposed to be omitted is enclosed in
black brackets, new matter is printed in italic, existing law
in which no change is proposed is shown in roman):

PUBLIC HEALTH SERVICE ACT

* * * * * * *

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 disease 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, andsuch sums as
may be necessary for each subsequent fiscal year.

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 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.
(3) 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.

SECTION 5 OF THE ORPHAN DRUG ACT

GRANTS AND CONTRACTS FOR DEVELOPMENT OF DRUGS FOR RARE DISEASES AND
CONDITIONS

Sec. 5. [21 U.S.C. 360ee] (a) * * *

* * * * * * *

[(c) For grants and contracts under subsection (a) there
are authorized to be appropriated $10,000,000 for fiscal year
1988, $12,000,000 for fiscal year 1989, $14,000,000 for fiscal
year 1990.]
(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.

* * * * * * *

FEDERAL FOOD, DRUG, AND COSMETIC ACT

* * * * * * *

protection for drugs for rare diseases or conditions

Sec. 527. [21 U.S.C. 360cc] (a) Except as provided in
subsection (b), if the Secretary--
(1) * * *

* * * * * * *

for a drug designated under section 526 for a rare disease or
condition, the Secretary may not approve another aplication
under section 505 or issue another license under section 351 of
the Public Health Service Act for such drug for such disease or
condition for a person who is not the holder of such approved
application[, of such certification,] or of such license until
the expiration of seven years from the date of the approval of
the approved application[, the issuance of the certification,]
or the issuance of the license. Section 505(c)(2) does not
apply to the refusal to approve an application under the
preceding sentence.

* * * * * * *