Ask the Scholar
Document scope · 1 page
Scholar
Ask about this object, its catalog metadata, its source description, or the page inventory.
For page-specific OCR and visual context, open one of the page chats.
Scholar Source Context
Document identity
localId
134608154
label
11/06/2002 [H.R. 4013] [771591]
core
doc
dtoType
document
citationUrl
pageCount
1
Source metadata
id
134608154
contentType
document
title
11/06/2002 [H.R. 4013] [771591]
citationUrl
collections
Records of the Office of the Executive Clerk (George W. Bush Administration)
G. Timothy (Tim) Saunders' Files
imageCount
1
hasImages
yes
source
import
hasTranscription
no
Source extras
naId
134608154
levelOfDescription
fileUnit
recordType
description
ocrSource
nara-archive
Single page context
seq
1
pageIndex
0
type
document
mediaId
c3222956dd1c8bd1
ocrText
George W. Bush Presidential Library
Collection: Executive Clerk, Office of the
Series: Saunders, G. Timothy (Tim) - Bill Files
Folder Title: 11/06/2002 [H.R. 4013] [771591]
EOP RMS - Barcode Scanning Sheet
Page 1 of 1
LAW_M
771591
FG006-01
Barcode Scanning Sheet
Collection Code: SECLOG
Staff Name:
Document Date:
Correspondent:
TIM SAUNDERS
Subject/Description:
OVERSIZE ATTACHMENT # 9469 NARA # 9360 BOX 5
FOLDER 6E BILL FILE RECEIVED FROM THE OFFICE OF
THE EXECUTIVE CLERK . NOV 05 02 ENROLLED BILL H.R.
4013 - RARE DISEASES ACT OF 2002
http://saeop82/RMS/custom/jsp/caseentry/BarcodeScanningShet.jsp
11/18/2008
UNITED THE OFFICE OF OF THE PRESIDENT
EXECUTIVE OFFICE OF THE PRESIDENT
OFFICE OF MANAGEMENT AND BUDGET
WASHINGTON, D.C. 20503
October 30, 2002
THE DIRECTOR
THE PRESIDENT HAS SEEN
MEMORANDUM FOR THE PRESIDENT
11-6-02
SUBJECT:
Enrolled Bill H.R. 4013 - Rare Diseases Act of 2002
Sponsors - Rep. Shimkus (R) IL and 54 cosponsors
Last Day for Action
November 8, 2002 - Friday
Purpose
Establishes an Office of Rare Diseases within the National Institutes of Health (NIH) and
authorizes appropriations for Rare Disease Regional Centers of Excellence.
Agency Recommendations
Office of Management and Budget
Approval
Department of Health and Human Services (HHS)
Approval
Discussion
H.R. 4013 would establish in statute an Office of Rare Diseases within the Office of the
Director of NIH. (The Office was established administratively in 1993.) A rare disease is
defined as one that afflicts fewer than 200,000 individuals in the United States. According to the
Food and Drug Administration, there are more than 6,000 rare diseases affecting 20 to 25 million
people in the United States. The Office would be responsible for recommending an agenda for
conducting and supporting research; promoting coordination and cooperation among NIH
institutes and centers with regard to rare diseases; promoting the sufficient allocation of resources
within NIH for conducting and supporting research on rare diseases; and encouraging the
establishment of a centralized clearinghouse for rare and genetic disease information for use by
the public, medical professionals, patients, and their families. H.R. 4013 would authorize
appropriations of $4 million for each of FYs 2003-2006 to carry out the responsibilities of the
Office. The FY 2003 Budget requests $11.3 million for the Office.
H.R. 4013 also would authorize appropriations of $20 million for each of FYs 2003-2006
for Rare Disease Regional Centers of Excellence. The Office of Rare Diseases, in collaboration
with other NIH institutes and centers, would enter into cooperative agreements with and make
grants to public or private nonprofit entities for regional centers of excellence that would conduct
research and training in the diagnosis, prevention, control, and treatment of rare diseases.
Federal support of the centers of excellence would be limited to five years, but could, under
certain conditions, be extended for up to an additional five years. The FY 2003 Budget does not
include funding for rare disease regional centers of excellence.
Conclusion and Recommendations
HHS recommends approval of H.R. 4013. In its views letter on the enrolled bill, the
Department states that the enrolled bill "is consistent with the Department's policies and
objectives." For example, HHS notes that "NIH has been planning to establish Specialized
Research and Diagnostic Centers of Excellence for Rare Diseases, which it considers crucial to
coordinate research on rare diseases."
We join HHS in recommending approval of H.R. 4013, which passed the House by voice
vote and the Senate by unanimous consent.
MEDanies)
Mitchell E. Daniels, Jr.
Director
Enclosures
-2-
4013
One Hundred Sebenth Congress
of the
United States of America
AT THE SECOND SESSION
Begun and held at the City of Washington on Wednesday,
the twenty-third day of January, two thousand and two
THE
OCT 2002
WHITE
An Act
To amend the Public Health Service Act to establish an Office of Rare Diseases
at the National Institutes of Health, and for other purposes.
HOUSE
Be it enacted by the Senate and House of Representatives of
the United States of America in Congress assembled,
SECTION 1. SHORT TITLE.
This Act may be cited as the "Rare Diseases Act of 2002".
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 dys-
trophy.
(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 manu-
facturers 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 pro-
vide 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.
(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 pro-
mote 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
H.R. 4013-4
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 AGREE-
MENTS 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.".
Speaker of the House of Representatives. prote -pcne.
lice President of the United States and
President of the Senate.pr tempone.
APPROVED
NOV - 6 2002
ORM
Scanning insert sheet
STOP
Remainder of case not scanned
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
ACTION FYI
ACTION FYI
VICE PRESIDENT
D
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M., THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
THANK YOU.
RESPONSE:
Harriet E. Miers
Assistant to the President
and Staff Secretary
Ext. 62702
EXECUTIVE OFFICE OF THE PRESIDENT
OFFICE OF THE PRESIDENT STATES UNITED
OFFICE OF MANAGEMENT AND BUDGET
WASHINGTON, D.C. 20503
October 30, 2002
THE DIRECTOR
MEMORANDUM FOR THE PRESIDENT
SUBJECT:
Enrolled Bill H.R. 4013 - Rare Diseases Act of 2002
Sponsors - Rep. Shimkus (R) IL and 54 cosponsors
Last Day for Action
November 8, 2002 - Friday
Purpose
Establishes an Office of Rare Diseases within the National Institutes of Health (NIH) and
authorizes appropriations for Rare Disease Regional Centers of Excellence.
Agency Recommendations
Office of Management and Budget
Approval
Department of Health and Human Services (HHS)
Approval
Discussion
H.R. 4013 would establish in statute an Office of Rare Diseases within the Office of the
Director of NIH. (The Office was established administratively in 1993.) A rare disease is
defined as one that afflicts fewer than 200,000 individuals in the United States. According to the
Food and Drug Administration, there are more than 6,000 rare diseases affecting 20 to 25 million
people in the United States. The Office would be responsible for recommending an agenda for
conducting and supporting research; promoting coordination and cooperation among NIH
institutes and centers with regard to rare diseases; promoting the sufficient allocation of resources
within NIH for conducting and supporting research on rare diseases; and encouraging the
establishment of a centralized clearinghouse for rare and genetic disease information for use by
the public, medical professionals, patients, and their families. H.R. 4013 would authorize.
appropriations of $4 million for each of FYs 2003-2006 to carry out the responsibilities of the
Office. The FY 2003 Budget requests $11.3 million for the Office.
H.R. 4013 also would authorize appropriations of $20 million for each of FYs 2003-2006
for Rare Disease Regional Centers of Excellence. The Office of Rare Diseases, in collaboration
with other NIH institutes and centers, would enter into cooperative agreements with and make
grants to public or private nonprofit entities for regional centers of excellence that would conduct
research and training in the diagnosis, prevention, control, and treatment of rare diseases.
Federal support of the centers of excellence would be limited to five years, but could, under
certain conditions, be extended for up to an additional five years. The FY 2003 Budget does not
include funding for rare disease regional centers of excellence.
Conclusion and Recommendations
HHS recommends approval of H.R. 4013. In its views letter on the enrolled bill, the
Department states that the enrolled bill "is consistent with the Department's policies and
objectives." For example, HHS notes that "NIH has been planning to establish Specialized
Research and Diagnostic Centers of Excellence for Rare Diseases, which it considers crucial to
coordinate research on rare diseases."
We join HHS in recommending approval of H.R. 4013, which passed the House by voice
vote and the Senate by unanimous consent.
MEDanies)
Mitchell E. Daniels, Jr.
Director
Enclosures
-2-
Bills Received at the White House
Date received and
notification to OMB:
10/28/2002
Last day for action:
11/8/2002
Official Title
H.R.4013
An Act to amend the Public Health Service Act to establish
an Office of Rare Diseases at the National Institutes of
Health, and for other purposes.
7
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
ACTION FYI
ACTION FYI
no obj:
VICE PRESIDENT
D
HUBBARD OK
CARD ok
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
X
BOLTEN
MARBURGER
BRIDGELAND N/C
MIERS
CALIO
OK
RICE
CONNAUGHTON N/C
RIDGE
DANIELS
ROVE NC
FLEISCHER
SPELLINGS NColy
GERSON
CLERK
x
GONZALES N/C
/
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M., THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
THANK YOU.
RESPONSE:
Harriet E. Miers
Assistant to the President
and Staff Secretary
Ext. 62702
OFFICE THE PRESIDENT STATES OF THE UNITED THE
EXECUTIVE OFFICE OF THE PRESIDENT
OFFICE OF MANAGEMENT AND BUDGET
WASHINGTON, D.C. 20503
October 30, 2002
THE DIRECTOR
MEMORANDUM FOR THE PRESIDENT
SUBJECT:
Enrolled Bill H.R. 4013 - Rare Diseases Act of 2002
Sponsors - Rep. Shimkus (R) IL and 54 cosponsors
Last Day for Action
November 8, 2002 - Friday
Purpose
Establishes an Office of Rare Diseases within the National Institutes of Health (NIH) and
authorizes appropriations for Rare Disease Regional Centers of Excellence.
Agency Recommendations
Office of Management and Budget
Approval
Department of Health and Human Services (HHS)
Approval
Discussion
H.R. 4013 would establish in statute an Office of Rare Diseases within the Office of the
Director of NIH. (The Office was established administratively in 1993.) A rare disease is
defined as one that afflicts fewer than 200,000 individuals in the United States. According to the
Food and Drug Administration, there are more than 6,000 rare diseases affecting 20 to 25 million
people in the United States. The Office would be responsible for recommending an agenda for
conducting and supporting research; promoting coordination and cooperation among NIH
institutes and centers with regard to rare diseases; promoting the sufficient allocation of resources
within NIH for conducting and supporting research on rare diseases; and encouraging the
establishment of a centralized clearinghouse for rare and genetic disease information for use by
the public, medical professionals, patients, and their families. H.R. 4013 would authorize
appropriations of $4 million for each of FYs 2003-2006 to carry out the responsibilities of the
Office. The FY 2003 Budget requests $11.3 million for the Office.
H.R. 4013 also would authorize appropriations of $20 million for each of FYs 2003-2006
for Rare Disease Regional Centers of Excellence. The Office of Rare Diseases, in collaboration
with other NIH institutes and centers, would enter into cooperative agreements with and make
grants to public or private nonprofit entities for regional centers of excellence that would conduct
research and training in the diagnosis, prevention, control, and treatment of rare diseases.
Federal support of the centers of excellence would be limited to five years, but could, under
certain conditions, be extended for up to an additional five years. The FY 2003 Budget does not
include funding for rare disease regional centers of excellence.
Conclusion and Recommendations
HHS recommends approval of H.R. 4013. In its views letter on the enrolled bill, the
Department states that the enrolled bill "is consistent with the Department's policies and
objectives." For example, HHS notes that "NIH has been planning to establish Specialized
Research and Diagnostic Centers of Excellence for Rare Diseases, which it considers crucial to
coordinate research on rare diseases."
We join HHS in recommending approval of H.R. 4013, which passed the House by voice
vote and the Senate by unanimous consent.
MEDanies)
Mitchell E. Daniels, Jr.
Director
Enclosures
-2-
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
ACTION FYI
ACTION FYI
VICE PRESIDENT
D
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M., THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
THANK YOU.
RESPONSE:
Harriet E. Miers
Assistant to the President
and Staff Secretary
Ext. 62702
EXECUTIVE OFFICE OF THE PRESIDENT
OFFICE PRESIDENT MANAGER STATES UNITED THE
OFFICE OF MANAGEMENT AND BUDGET
WASHINGTON, D.C. 20503
October 30, 2002
THE DIRECTOR
MEMORANDUM FOR THE PRESIDENT
SUBJECT:
Enrolled Bill H.R. 4013 - Rare Diseases Act of 2002
Sponsors - Rep. Shimkus (R) IL and 54 cosponsors
Last Day for Action
November 8, 2002 - Friday
Purpose
Establishes an Office of Rare Diseases within the National Institutes of Health (NIH) and
authorizes appropriations for Rare Disease Regional Centers of Excellence.
Agency Recommendations
Office of Management and Budget
Approval
Department of Health and Human Services (HHS)
Approval
Discussion
H.R. 4013 would establish in statute an Office of Rare Diseases within the Office of the
Director of NIH. (The Office was established administratively in 1993.) A rare disease is
defined as one that afflicts fewer than 200,000 individuals in the United States. According to the
Food and Drug Administration, there are more than 6,000 rare diseases affecting 20 to 25 million
people in the United States. The Office would be responsible for recommending an agenda for
conducting and supporting research; promoting coordination and cooperation among NIH
institutes and centers with regard to rare diseases; promoting the sufficient allocation of resources
within NIH for conducting and supporting research on rare diseases; and encouraging the
establishment of a centralized clearinghouse for rare and genetic disease information for use by
the public, medical professionals, patients, and their families. H.R. 4013 would authorize
appropriations of $4 million for each of FYs 2003-2006 to carry out the responsibilities of the
Office. The FY 2003 Budget requests $11.3 million for the Office.
H.R. 4013 also would authorize appropriations of $20 million for each of FYs 2003-2006
for Rare Disease Regional Centers of Excellence. The Office of Rare Diseases, in collaboration
with other NIH institutes and centers, would enter into cooperative agreements with and make
grants to public or private nonprofit entities for regional centers of excellence that would conduct
research and training in the diagnosis, prevention, control, and treatment of rare diseases.
Federal support of the centers of excellence would be limited to five years, but could, under
certain conditions, be extended for up to an additional five years. The FY 2003 Budget does not
include funding for rare disease regional centers of excellence.
Conclusion and Recommendations
HHS recommends approval of H.R. 4013. In its views letter on the enrolled bill, the
Department states that the enrolled bill "is consistent with the Department's policies and
objectives." For example, HHS notes that "NIH has been planning to establish Specialized
Research and Diagnostic Centers of Excellence for Rare Diseases, which it considers crucial to
coordinate research on rare diseases."
We join HHS in recommending approval of H.R. 4013, which passed the House by voice
vote and the Senate by unanimous consent.
Mitchell E. Daniels, Jr.
Director
Enclosures
-2-
THE SECRETARY OF HEALTH AND HUMAN SERVICES
SECURITY
WASHINGTON, D.C. 20201
USA
October 25, 2002
The Honorable Mitchell E. Daniels, Jr.
Director, Office of Management
and Budget
Washington, D.C. 20503
Dear Mr. Daniels:
This responds to your request for the views of the Department of Health and Human Services (HHS)
on H.R. 4013, an enrolled bill entitled the "Rare Diseases Act of 2002". We recommend that the
President approve the bill.
H.R. 4013 adds section 404F to the Public Health Service Act (the "Act"), to establish in statute the
NIH Office of Rare Diseases ("ORD"). Section 404F also makes the ORD director responsible for
recommending a research agenda on "rare diseases" (defined as diseases affecting fewer than 200,000
persons in the United States); promoting coordination on rare diseases within NIH; promoting sufficient
allocation of NIH resources related to rare diseases; promoting establishment of a centralized rare
diseases information clearinghouse; preparing a biennial report on rare disease research activities and
opportunities; preparing the NIH Director's annual report to Congress on rare disease research; and
serving as the NIH Director's principal advisor on rare diseases. The section authorizes appropriations
of such sums as may be necessary for FY 2002, and $4 million for each of FY 2003 through 2006.
The bill also adds section 404G to the Act, authorizing NIH to fund, through grants and cooperative
agreements, regional Centers of Excellence on rare diseases. The section authorizes appropriations of
such sums as may be necessary for FY 2002, and $20 million for each of FY 2003 through 2006.
The bill is consistent with the Department's policies and objectives. NIH established the Office of Rare
Diseases by administrative action in 1993, and it is already carrying out many of the provisions in the
bill. In particular, NIH has been planning to establish Specialized Research and Diagnostic Centers of
Excellence for Rare Diseases, which it considers crucial to coordinate research on rare diseases.
Page 2 -- Honorable Mitchell E. Daniels, Jr.
For the foregoing reasons, we recommend that the President sign the bill.
Sincerely,
Jonny S. Thoryson
Tommy G. Thompson
107TH CONGRESS
2d Session
}
HOUSE OF REPRESENTATIVES
-
REPORT
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
REPORT
[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 es-
tablish 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 In-
stitutes 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
99-006
2
the Director of the NIH with respect to rare diseases, and shall
serve to promote sufficient allocation of NIH resources to rare dis-
ease research, and promote and encourage the establishment of a
centralized clearinghouse for rare disease information for the ben-
efit 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 require-
ments 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 in-
centive for drug and biological manufacturers to invest in treat-
ments for these diseases. The Orphan Drug Act created greater in-
centives 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 Of-
fice of Rare Diseases. This action sends the strong signal of the
Congress' commitment for both this Office as well as for rare dis-
ease research generally. Also, authorizing regional centers of excel-
lence for rare disease research is a positive step, as it will enable
the NIH to select sites to concentrate on finding cures and treat-
ment methods for rare diseases. Both of these proposals are con-
tained 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, Univer-
sity 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 Representa-
tives requires the Committee to list the record votes on the motion
3
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 re-
gional 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 man-
dates 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 au-
thority for this legislation is provided in Article I, section 8, clause
3, which grants Congress the power to regulate commerce with for-
eign nations, among the several States, and with the Indian tribes.
4
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 Con-
gressional 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 co-
operation 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 pur-
poses.
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 diag-
nostic, prevention, control, and treatment methods for rare dis-
eases. 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
*
*
*
*
*
*
TITLE IV-NATIONAL RESEARCH INSTITUTES
PART A-NATIONAL INSTITUTES OF HEALTH
*
*
*
*
*
*
*
5
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 "Di-
rector"), 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 con-
ducting 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, pro-
mote coordination and cooperation among the national re-
search 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 In-
stitutes 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 con-
ducting and supporting research on rare diseases.
(E) The Director shall promote and encourage the estab-
lishment of a centralized clearinghouse for rare and genetic
disease information that will provide understandable infor-
mation about these diseases to the public, medical profes-
sionals, patients and families.
(F) The Director shall biennially prepare a report that
describes the research and education activities on rare dis-
eases being conducted or supported through the national re-
search institutes and centers, and that identifies particular
projects or types of projects that should in the future be con-
ducted or supported by the national research institutes and
centers or other entities in the field of research on rare dis-
eases.
(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 cen-
ters.
(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 na-
tional and international patient, health and scientific organiza-
tions concerned with rare diseases.
(c) DEFINITION.-For purposes of this section the term "rare dis-
ease" means any disease or condition that affects less than 200,000
persons in the United States.
6
(d) AUTHORIZATION OF APPROPRIATIONS.-For the purpose of car-
rying 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 thè 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 re-
gional 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 para-
graph (1) shall be entered into in accordance with policies es-
tablished 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 Ad-
ministration to the extent that such institutes, centers and agencies
have responsibilities that are related to rare diseases.
(c) USES FOR FEDERAL PAYMENTS UNDER COOPERATIVE AGREE-
MENTS 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 pro-
fessionals, continuing education for health professionals and al-
lied 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 cen-
ter 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 rec-
ommended to the Director that such period should be extended.
(e) AUTHORIZATION OF APPROPRIATIONS.-For the purpose of car-
rying 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.
*
*
*
H. 4013
One Hundred Sebenth Congress
of the
United States of America
AT THE SECOND SESSION
Begun and held at the City of Washington on Wednesday,
the twenty-third day of January, two thousand and two
An Act
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,
SECTION 1. SHORT TITLE.
This Act may be cited as the "Rare Diseases Act of 2002".
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 dys-
trophy.
(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 manu-
facturers 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 pro-
vide 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.
(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 pro-
mote 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
H.R. 4013-2
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 dis-
eases 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 net-
work of regional centers of excellence for research on rare
diseases.
(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.
SEC. 3. NIH OFFICE OF RARE DISEASES AT NATIONAL INSTITUTES
OF HEALTH.
Title IV of the Public Health Service Act (42 U.S.C. 281 et
seq.), as amended by Public Law 107-84, is amended by inserting
after section 404E the following:
"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 con-
ducting 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.
H.R. 4013-3
"(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 understand-
able 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 con-
ducted 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.".
SEC. 4. 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 3, is further amended by inserting
after section 404F the following:
"RARE DISEASE REGIONAL CENTERS OF EXCELLENCE
"SEC. 404G. (a) COOPERATIVE AGREEMENTS AND GRANTS.-
"(1) IN GENERAL.-The Director of the Office of Rare Dis-
eases (in this section referred to as the 'Director'), in collabora-
tion 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 activi-
ties conducted by other national research institutes, centers and
H.R. 4013-4
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 AGREE-
MENTS 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.".
Speaker of the House of Representatives.
Vice President of the United States and
President of the Senate.
518739
OFFICE OF THE OF PRESIDENT STATES UNITED THE
EXECUTIVE OFFICE OF THE PRESIDENT
OFFICE OF MANAGEMENT AND BUDGET
WASHINGTON, D.C. 20503
October 30, 2002
THE DIRECTOR
MEMORANDUM FOR THE PRESIDENT
SUBJECT:
Enrolled Bill H.R. 4013 - Rare Diseases Act of 2002
Sponsors - Rep. Shimkus (R) IL and 54 cosponsors
Last Day for Action
November 8, 2002 - Friday
Purpose
Establishes an Office of Rare Diseases within the National Institutes of Health (NIH) and
authorizes appropriations for Rare Disease Regional Centers of Excellence.
Agency Recommendations
Office of Management and Budget
Approval
Department of Health and Human Services (HHS)
Approval
Discussion
H.R. 4013 would establish in statute an Office of Rare Diseases within the Office of the
Director of NIH. (The Office was established administratively in 1993.) A rare disease is
defined as one that afflicts fewer than 200,000 individuals in the United States. According to the
Food and Drug Administration, there are more than 6,000 rare diseases affecting 20 to 25 million
people in the United States. The Office would be responsible for recommending an agenda for
conducting and supporting research; promoting coordination and cooperation among NIH
institutes and centers with regard to rare diseases; promoting the sufficient allocation of resources
within NIH for conducting and supporting research on rare diseases; and encouraging the
establishment of a centralized clearinghouse for rare and genetic disease information for use by
the public, medical professionals, patients, and their families. H.R. 4013 would authorize
appropriations of $4 million for each of FYs 2003-2006 to carry out the responsibilities of the
Office. The FY 2003 Budget requests $11.3 million for the Office.
H.R. 4013 also would authorize appropriations of $20 million for each of FYs 2003-2006
for Rare Disease Regional Centers of Excellence. The Office of Rare Diseases, in collaboration
with other NIH institutes and centers, would enter into cooperative agreements with and make
grants to public or private nonprofit entities for regional centers of excellence that would conduct
research and training in the diagnosis, prevention, control, and treatment of rare diseases.
Federal support of the centers of excellence would be limited to five years, but could, under
certain conditions, be extended for up to an additional five years. The FY 2003 Budget does not
include funding for rare disease regional centers of excellence.
Conclusion and Recommendations
HHS recommends approval of H.R. 4013. In its views letter on the enrolled bill, the
Department states that the enrolled bill "is consistent with the Department's policies and
objectives." For example, HHS notes that "NIH has been planning to establish Specialized
Research and Diagnostic Centers of Excellence for Rare Diseases, which it considers crucial to
coordinate research on rare diseases."
We join HHS in recommending approval of H.R. 4013, which passed the House by voice
vote and the Senate by unanimous consent.
MEDanies)
Mitchell E. Daniels, Jr.
Director
Enclosures
-2-
COS
DECUTIVE UNITED THE OFFICE PRESIDENT
EXECUTIVE OFFICE OF THE PRESIDENT
OFFICE OF MANAGEMENT AND BUDGET
WASHINGTON, D.C. 20503
October 30, 2002
THE DIRECTOR
MEMORANDUM FOR THE PRESIDENT
SUBJECT:
Enrolled Bill H.R. 4013 - Rare Diseases Act of 2002
Sponsors - Rep. Shimkus (R) IL and 54 cosponsors
Last Day for Action
November 8, 2002 - Friday
Purpose
Establishes an Office of Rare Diseases within the National Institutes of Health (NIH) and
authorizes appropriations for Rare Disease Regional Centers of Excellence.
Agency Recommendations
Office of Management and Budget
Approval
Department of Health and Human Services (HHS)
Approval
Discussion
H.R. 4013 would establish in statute an Office of Rare Diseases within the Office of the
Director of NIH. (The Office was established administratively in 1993.) A rare disease is
defined as one that afflicts fewer than 200,000 individuals in the United States. According to the
Food and Drug Administration, there are more than 6,000 rare diseases affecting 20 to 25 million
people in the United States. The Office would be responsible for recommending an agenda for
conducting and supporting research; promoting coordination and cooperation among NIH
institutes and centers with regard to rare diseases; promoting the sufficient allocation of resources
within NIH for conducting and supporting research on rare diseases; and encouraging the
establishment of a centralized clearinghouse for rare and genetic disease information for use by
the public, medical professionals, patients, and their families. H.R. 4013 would authorize
appropriations of $4 million for each of FYs 2003-2006 to carry out the responsibilities of the
Office. The FY 2003 Budget requests $11.3 million for the Office.
H.R. 4013 also would authorize appropriations of $20 million for each of FYs 2003-2006
for Rare Disease Regional Centers of Excellence. The Office of Rare Diseases, in collaboration
with other NIH institutes and centers, would enter into cooperative agreements with and make
grants to public or private nonprofit entities for regional centers of excellence that would conduct
research and training in the diagnosis, prevention, control, and treatment of rare diseases.
Federal support of the centers of excellence would be limited to five years, but could, under
certain conditions, be extended for up to an additional five years. The FY 2003 Budget does not
include funding for rare disease regional centers of excellence.
Conclusion and Recommendations
HHS recommends approval of H.R. 4013. In its views letter on the enrolled bill, the
Department states that the enrolled bill "is consistent with the Department's policies and
objectives." For example, HHS notes that "NIH has been planning to establish Specialized
Research and Diagnostic Centers of Excellence for Rare Diseases, which it considers crucial to
coordinate research on rare diseases."
We join HHS in recommending approval of H.R. 4013, which passed the House by voice
vote and the Senate by unanimous consent.
MEDanies)
Mitchell E. Daniels, Jr.
Director
Enclosures
-2-
Linda
OFFICE OF THE OF PRESIDENT OF UNITED THE
EXECUTIVE OFFICE OF THE PRESIDENT
OFFICE OF MANAGEMENT AND BUDGET
WASHINGTON, D.C. 20503
October 30, 2002
THE DIRECTOR
MEMORANDUM FOR THE PRESIDENT
SUBJECT:
Enrolled Bill H.R. 4013 - Rare Diseases Act of 2002
Sponsors - Rep. Shimkus (R) IL and 54 cosponsors
Last Day for Action
November 8, 2002 - Friday
Purpose
Establishes an Office of Rare Diseases within the National Institutes of Health (NIH) and
authorizes appropriations for Rare Disease Regional Centers of Excellence.
Agency Recommendations
Office of Management and Budget
Approval
Department of Health and Human Services (HHS)
Approval
Discussion
H.R. 4013 would establish in statute an Office of Rare Diseases within the Office of the
Director of NIH. (The Office was established administratively in 1993.) A rare disease is
defined as one that afflicts fewer than 200,000 individuals in the United States. According to the
Food and Drug Administration, there are more than 6,000 rare diseases affecting 20 to 25 million
people in the United States. The Office would be responsible for recommending an agenda for
conducting and supporting research; promoting coordination and cooperation among NIH
institutes and centers with regard to rare diseases; promoting the sufficient allocation of resources
within NIH for conducting and supporting research on rare diseases; and encouraging the
establishment of a centralized clearinghouse for rare and genetic disease information for use by
the public, medical professionals, patients, and their families. H.R. 4013 would authorize
appropriations of $4 million for each of FYs 2003-2006 to carry out the responsibilities of the
Office. The FY 2003 Budget requests $11.3 million for the Office.
H.R. 4013 also would authorize appropriations of $20 million for each of FYs 2003-2006
for Rare Disease Regional Centers of Excellence. The Office of Rare Diseases, in collaboration
with other NIH institutes and centers, would enter into cooperative agreements with and make
grants to public or private nonprofit entities for regional centers of excellence that would conduct
research and training in the diagnosis, prevention, control, and treatment of rare diseases.
Federal support of the centers of excellence would be limited to five years, but could, under
certain conditions, be extended for up to an additional five years. The FY 2003 Budget does not
include funding for rare disease regional centers of excellence.
Conclusion and Recommendations
HHS recommends approval of H.R. 4013. In its views letter on the enrolled bill, the
Department states that the enrolled bill "is consistent with the Department's policies and
objectives." For example, HHS notes that "NIH has been planning to establish Specialized
Research and Diagnostic Centers of Excellence for Rare Diseases, which it considers crucial to
coordinate research on rare diseases."
We join HHS in recommending approval of H.R. 4013, which passed the House by voice
vote and the Senate by unanimous consent.
MEDanies)
Mitchell E. Daniels, Jr.
Director
Enclosures
-2-
10/30/2002 WED 05:44 FAX
001
*** TX REPORT ***
TRANSMISSION OK
TX/RX NO
1141
CONNECTION TEL
66212
CONNECTION ID
ST. TIME
10/30 05:43
USAGE T
01'13
PGS. SENT
3
RESULT
OK
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
ACTION FYI
ACTION FYI
VICE PRESIDENT
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M.. THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
ACTION FYI
ACTION FYI
VICE PRESIDENT
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M., THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
THANK YOU.
RESPONSE:
Harriet E. Miers
Assistant to the President
and Staff Secretary
Ext. 62702
10/30/2002 WED 05:51 FAX
001
*** TX REPORT ***
TRANSMISSION OK
TX/RX NO
1144
CONNECTION TEL
69720
CONNECTION ID
ST. TIME
10/30 05:50
USAGE T
01'13
PGS. SENT
3
RESULT
OK
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
ACTION FYI
ACTION FYI
VICE PRESIDENT
D
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M.. THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
ACTION FYI
ACTION
FYI
VICE PRESIDENT
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M., THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
THANK YOU.
RESPONSE:
Harriet E. Miers
Assistant to the President
and Staff Secretary
Ext. 62702
10/30/2002 WED 06:20 FAX
V.
001
*** TX REPORT ***
TRANSMISSION OK
TX/RX NO
1162
CONNECTION TEL
56958
CONNECTION ID
ST. TIME
10/30 06:19
USAGE T
01'11
PGS. SENT
3
RESULT
OK
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
ACTION FYI
ACTION FYI
VICE PRESIDENT
X
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
DM THURSDAY OCTOBER 31 2002. WITH A COPY TO THE STAFF SECRETARY.
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
ACTION FYI
ACTION FYI
VICE PRESIDENT
D
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M., THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
THANK YOU.
RESPONSE:
Harriet E. Miers
Assistant to the President
and Staff Secretary
Ext. 62702
10/30/2002 WED 06:03 FAX
001
TX REPORT ***
TRANSMISSION OK
TX/RX NO
1151
CONNECTION TEL
67090
CONNECTION ID
ADMIN
ST. TIME
10/30 06:02
USAGE T
01'23
PGS. SENT
3
RESULT
OK
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
ACTION FYI
ACTION FYI
VICE PRESIDENT
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
RV P M THURSDAY. OCTOBER 31. 2002, WITH A COPY TO THE STAFF SECRETARY.
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
ACTION FYI
ACTION FYI
VICE PRESIDENT
D
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M., THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
THANK YOU.
RESPONSE:
Harriet E. Miers
Assistant to the President
and Staff Secretary
Ext. 62702
10/30/2002 WED 06:06 FAX
1
001
*** TX REPORT ***
TRANSMISSION OK
TX/RX NO
1153
CONNECTION TEL
62710
CONNECTION ID
CEQ
ST. TIME
10/30 06:05
USAGE T
01'12
PGS. SENT
3
RESULT
OK
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
ACTION FYI
ACTION FYI
VICE PRESIDENT
D
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M.. THURSDAY. OCTOBER 31. 2002. WITH A COPY TO THE STAFF SECRETARY.
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
ACTION FYI
ACTION FYI
VICE PRESIDENT
X
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M., THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
THANK YOU.
RESPONSE:
Harriet E. Miers
Assistant to the President
and Staff Secretary
Ext. 62702
10/30/2002 WED 06:11 FAX
1
001
*** TX REPORT ***
TRANSMISSION OK
TX/RX NO
1156
CONNECTION TEL
51005
CONNECTION ID
ST. TIME
10/30 06:10
USAGE T
01'11
PGS. SENT
3
RESULT
OK
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
ACTION FYI
ACTION FYI
VICE PRESIDENT
D
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M.. THURSDAY. OCTOBER 31. 2002. WITH A COPY TO THE STAFF SECRETARY.
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
ACTION FYI
ACTION FYI
VICE PRESIDENT
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M., THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
THANK YOU.
RESPONSE:
Harriet E. Miers
Assistant to the President
and Staff Secretary
Ext. 62702
10/30/2002 WED 06:30 FAX
001
*** TX REPORT ***
TRANSMISSION OK
TX/RX NO
1167
CONNECTION TEL
67090
CONNECTION ID
ADMIN
ST. TIME
10/30 06:28
USAGE T
01'14
PGS. SENT
3
RESULT
OK
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 RARE DISEASES ACT OF 2002
ACTION FYI
ACTION FYI
VICE PRESIDENT
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
21 WITH ^ TO THE STAFF SECRETARY
10/30/02 WED 19:42 FAX
1
005
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM E 30 PMS:21
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
ACTION FYI
ACTION FYI
VICE PRESIDENT
D
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
x
GONZALES
/
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M., THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
THANK YOU.
No comments
RESPONSE:
60 :9 Md OE 130 ZUL
Harriet E. Miers
H/A
Assistant to the President
525 OHNE
and Staff Secretary
Ext. 62702
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
"02UCI 30 PK/:OB
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
ACTION FYI
ACTION FYI
VICE PRESIDENT
D
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M., THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
THANK YOU.
RESPONSE:
Harriet E. Miers
Assistant to the President
and Staff Secretary
Ext. 62702
OFFICE OF THE THE OF PRESIDENT STATES UNITED THE
EXECUTIVE OFFICE OF THE PRESIDENT
OFFICE OF MANAGEMENT AND BUDGET
WASHINGTON, D.C. 20503
October 30, 2002
THE DIRECTOR
MEMORANDUM FOR THE PRESIDENT
SUBJECT:
Enrolled Bill H.R. 4013 - Rare Diseases Act of 2002
Sponsors - Rep. Shimkus (R) IL and 54 cosponsors
Last Day for Action
November 8, 2002 - Friday
Purpose
Establishes an Office of Rare Diseases within the National Institutes of Health (NIH) and
authorizes appropriations for Rare Disease Regional Centers of Excellence.
Agency Recommendations
Office of Management and Budget
Approval
Department of Health and Human Services (HHS)
Approval
Discussion
H.R. 4013 would establish in statute an Office of Rare Diseases within the Office of the
Director of NIH. (The Office was established administratively in 1993.) A rare disease is
defined as one that afflicts fewer than 200,000 individuals in the United States. According to the
Food and Drug Administration, there are more than 6,000 rare diseases affecting 20 to 25 million
people in the United States. The Office would be responsible for recommending an agenda for
conducting and supporting research; promoting coordination and cooperation among NIH
institutes and centers with regard to rare diseases; promoting the sufficient allocation of resources
within NIH for conducting and supporting research on rare diseases; and encouraging the
establishment of a centralized clearinghouse for rare and genetic disease information for use by
the public, medical professionals, patients, and their families. H.R. 4013 would authorize
appropriations of $4 million for each of FYs 2003-2006 to carry out the responsibilities of the
Office. The FY 2003 Budget requests $11.3 million for the Office.
H.R. 4013 also would authorize appropriations of $20 million for each of FYs 2003-2006
for Rare Disease Regional Centers of Excellence. The Office of Rare Diseases, in collaboration
with other NIH institutes and centers, would enter into cooperative agreements with and make
grants to public or private nonprofit entities for regional centers of excellence that would conduct
research and training in the diagnosis, prevention, control, and treatment of rare diseases.
Federal support of the centers of excellence would be limited to five years, but could, under
certain conditions, be extended for up to an additional five years. The FY 2003 Budget does not
include funding for rare disease regional centers of excellence.
Conclusion and Recommendations
HHS recommends approval of H.R. 4013. In its views letter on the enrolled bill, the
Department states that the enrolled bill "is consistent with the Department's policies and
objectives." For example, HHS notes that "NIH has been planning to establish Specialized
Research and Diagnostic Centers of Excellence for Rare Diseases, which it considers crucial to
coordinate research on rare diseases."
We join HHS in recommending approval of H.R. 4013, which passed the House by voice
vote and the Senate by unanimous consent.
Mitchell E. Daniels, Jr.
Director
Enclosures
-2-
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
702101 HOPM/107
ACTION FYI
ACTION
FYI
VICE PRESIDENT
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M., THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
THANK YOU.
RESPONSE:
6:30
AHC
10/30
Harriet E. Miers
Assistant to the President
and Staff Secretary
Ext. 62702
EXECUTIVE OFFICE OF THE PRESIDENT
SEATES MANAGER PREQUITIVE BUDGET UNITED OFFICE INPRESIDENT
OFFICE OF MANAGEMENT AND BUDGET
WASHINGTON, D.C. 20503
October 30, 2002
THE DIRECTOR
MEMORANDUM FOR THE PRESIDENT
SUBJECT:
Enrolled Bill H.R. 4013 - Rare Diseases Act of 2002
Sponsors - Rep. Shimkus (R) IL and 54 cosponsors
Last Day for Action
November 8, 2002 - Friday
Purpose
Establishes an Office of Rare Diseases within the National Institutes of Health (NIH) and
authorizes appropriations for Rare Disease Regional Centers of Excellence.
Agency Recommendations
Office of Management and Budget
Approval
Department of Health and Human Services (HHS)
Approval
Discussion
H.R. 4013 would establish in statute an Office of Rare Diseases within the Office of the
Director of NIH. (The Office was established administratively in 1993.) A rare disease is
defined as one that afflicts fewer than 200,000 individuals in the United States. According to the
Food and Drug Administration, there are more than 6,000 rare diseases affecting 20 to 25 million
people in the United States. The Office would be responsible for recommending an agenda for
conducting and supporting research; promoting coordination and cooperation among NIH
institutes and centers with regard to rare diseases; promoting the sufficient allocation of resources
within NIH for conducting and supporting research on rare diseases; and encouraging the
establishment of a centralized clearinghouse for rare and genetic disease information for use by
the public, medical professionals, patients, and their families. H.R. 4013 would authorize
appropriations of $4 million for each of FYs 2003-2006 to carry out the responsibilities of the
Office. The FY 2003 Budget requests $11.3 million for the Office.
H.R. 4013 also would authorize appropriations of $20 million for each of FYs 2003-2006
for Rare Disease Regional Centers of Excellence. The Office of Rare Diseases, in collaboration
with other NIH institutes and centers, would enter into cooperative agreements with and make
grants to public or private nonprofit entities for regional centers of excellence that would conduct
research and training in the diagnosis, prevention, control, and treatment of rare diseases.
Federal support of the centers of excellence would be limited to five years, but could, under
certain conditions, be extended for up to an additional five years. The FY 2003 Budget does not
include funding for rare disease regional centers of excellence.
Conclusion and Recommendations
HHS recommends approval of H.R. 4013. In its views letter on the enrolled bill, the
Department states that the enrolled bill "is consistent with the Department's policies and
objectives." For example, HHS notes that "NIH has been planning to establish Specialized
Research and Diagnostic Centers of Excellence for Rare Diseases, which it considers crucial to
coordinate research on rare diseases."
We join HHS in recommending approval of H.R. 4013, which passed the House by voice
vote and the Senate by unanimous consent.
Mitchell E. Daniels, Jr.
Director
Enclosures
-2-
10/31/02 THU 09:15 FAX
1
001
10/30/02 WED 18:15 FAX
X
004
SS/ RM NO.
Jen
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 M RARE DISEASES ACT OF 2002
TOTAL 31 AMY:01
ACTION FYI
ACTION FYI
VICE PRESIDENT
D
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
R
BOLTEN
MARBURGER
BRIDGELAND
/
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M., THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
THANK YOU.
cleared 10/30/02 JGN
RESPONSE:
Harriet E, Miers
Assistant to the President
and Staff Secretary
Ext. 62702
10/30/2002 WED 05:50 FAX
001
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
AMY:01
Date: 10-30-02 4:30 PM
ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
ACTION
FYI
ACTION FYI
VICE PRESIDENT
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M., THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
THANK YOU.
RESPONSE:
Harriet E. Miers
Assistant to the President
and Staff Secretary
Ext. 62702
003
10/30/2002 WED 20:21 FAX
10/31/2002 09:25 FAX 202 456 2710
CEQ
1
007
10/30/2002 WED 06:05 FAX
5
001
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
31
LAMBIOZ
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
ACTION FYI
ACTION FYI
VICE PRESIDENT
HUBBARD
8
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
X
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
/
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M., THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
THANK YOU.
RESPONSE:
No Convent
PALooney 10/30/02 CEQ
Harriet E. Miers
Assistant to the President
and Staff Secretary
Ext. 62702
10/31/02 14:56 FAX
10/30/02 17:08 FAX
STAFF ASSISTANT
5
OPD
001/001
0
004
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 P RARE DISEASES ACT OF 2002
102 0CT 31 PX3:43
ACTION FYI
ACTION FYI
VICE PRESIDENT
D
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
/
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M., THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
THANK YOU.
RESPONSE:
" Jay
Alan) philo
no
nocomments
Tevi
Harriet E. Miers
Assistant to the President
and Staff Secretary
Ext. 62702
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
102 0CT 31 PN4:05
ACTION FYI
ACTION FYI
VICE PRESIDENT
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M., THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
THANK YOU.
RESPONSE:
OK'O BY PHONE
Harriet E. Miers
10/31 4:13 p.m. p.m.
Assistant to the President
and Staff Secretary
Ext. 62702
EXECUTIVE OFFICE OF THE PRESIDENT
STATES SECUTIVE UNITED THE OFFICE OF THE PRESIDENT
OFFICE OF MANAGEMENT AND BUDGET
WASHINGTON, D.C. 20503
October 30, 2002
THE DIRECTOR
MEMORANDUM FOR THE PRESIDENT
SUBJECT:
Enrolled Bill H.R. 4013 - Rare Diseases Act of 2002
Sponsors - Rep. Shimkus (R) IL and 54 cosponsors
Last Day for Action
November 8, 2002 - Friday
Purpose
Establishes an Office of Rare Diseases within the National Institutes of Health (NIH) and
authorizes appropriations for Rare Disease Regional Centers of Excellence.
Agency Recommendations
Office of Management and Budget
Approval
Department of Health and Human Services (HHS)
Approval
Discussion
H.R. 4013 would establish in statute an Office of Rare Diseases within the Office of the
Director of NIH. (The Office was established administratively in 1993.) A rare disease is
defined as one that afflicts fewer than 200,000 individuals in the United States. According to the
Food and Drug Administration, there are more than 6,000 rare diseases affecting 20 to 25 million
people in the United States. The Office would be responsible for recommending an agenda for
conducting and supporting research; promoting coordination and cooperation among NIH
institutes and centers with regard to rare diseases; promoting the sufficient allocation of resources
within NIH for conducting and supporting research on rare diseases; and encouraging the
establishment of a centralized clearinghouse for rare and genetic disease information for use by
the public, medical professionals, patients, and their families. H.R. 4013 would authorize
appropriations of $4 million for each of FYs 2003-2006 to carry out the responsibilities of the
Office. The FY 2003 Budget requests $11.3 million for the Office.
H.R. 4013 also would authorize appropriations of $20 million for each of FYs 2003-2006
for Rare Disease Regional Centers of Excellence. The Office of Rare Diseases, in collaboration
with other NIH institutes and centers, would enter into cooperative agreements with and make
grants to public or private nonprofit entities for regional centers of excellence that would conduct
research and training in the diagnosis, prevention, control, and treatment of rare diseases.
Federal support of the centers of excellence would be limited to five years, but could, under
certain conditions, be extended for up to an additional five years. The FY 2003 Budget does not
include funding for rare disease regional centers of excellence.
Conclusion and Recommendations
HHS recommends approval of H.R. 4013. In its views letter on the enrolled bill, the
Department states that the enrolled bill "is consistent with the Department's policies and
objectives." For example, HHS notes that "NIH has been planning to establish Specialized
Research and Diagnostic Centers of Excellence for Rare Diseases, which it considers crucial to
coordinate research on rare diseases."
We join HHS in recommending approval of H.R. 4013, which passed the House by voice
vote and the Senate by unanimous consent.
Mitchell E. Daniels, Jr.
Director
Enclosures
-2-
10/30/2002 WED 06:19 FAX
SS/ RM NO.
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM
ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 * RARE DISEASES ACT OF 2002
'020CT31 M6:50
ACTION FYI
ACTION FYI
VICE PRESIDENT
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M., THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
THANK YOU.
OK- Phillip Swogel (EA
RESPONSE:
Harriet E. Miers
Assistant to the President
and Staff Secretary
Ext. 62702
P.03/04
8969 565 202
60:81 00-13-2002
OCT-31-2002 15:01
OVP
202 456 0387
P.01/03
OFFICE OF THE VICE PRESIDENT
WASHINGTON
:02 0CT 31 PM7:09
October 31, 2002
MEMORANDUM FOR JAMES JUKES
ASSISTANT DIRECTOR, OMB
FROM:
DEPUTY STAFF SECRE B TARY TO THE VICE PRESIDENT
JONATHAN BURKS
SUBJECT:
Enrolled Bill H.R. 4013 Rare Diseases Act of 2002
The Office of the Vice President has reviewed the above-referenced
draft and has no objection to the President signing the enrolled bill.
cc: Harriet Miers
Staff Secretary
SS/ RM NO.
02 NOV 1PM3:23
WHITE HOUSE STAFFING MEMORANDUM
Date: 10-30-02 4:30 PM ACTION / CONCURRENCE / COMMENT DUE BY: 10-31-02 5:00 PM
Subject: ENROLLED BILL H.R. 4013 - RARE DISEASES ACT OF 2002
ACTION FYI
ACTION FYI
VICE PRESIDENT
HUBBARD
CARD
IRASTORZA
BARTLETT
JOHNSON
BLAKEMAN
LINDSEY
BOLTEN
MARBURGER
BRIDGELAND
MIERS
CALIO
RICE
CONNAUGHTON
RIDGE
DANIELS
ROVE
FLEISCHER
SPELLINGS
GERSON
CLERK
GONZALES
HAGIN
HAWKINS
REMARKS:
PLEASE FORWARD COMMENTS DIRECTLY TO JIM JUKES, X53458, FAX X56148,
BY 5:00 P.M., THURSDAY, OCTOBER 31, 2002, WITH A COPY TO THE STAFF SECRETARY.
THANK YOU.
RESPONSE:
6x
CC Jay
Alan/ philo
Tevi
Harriet E. Miers
Assistant to the President
and Staff Secretary
Ext. 62702
OFFICE OF THE PRESIDENT STATES BUDGET OF THE UNITED THE
EXECUTIVE OFFICE OF THE PRESIDENT
OFFICE OF MANAGEMENT AND BUDGET
WASHINGTON, D.C. 20503
October 30, 2002
THE DIRECTOR
MEMORANDUM FOR THE PRESIDENT
SUBJECT:
Enrolled Bill H.R. 4013 - Rare Diseases Act of 2002
Sponsors - Rep. Shimkus (R) IL and 54 cosponsors
Last Day for Action
November 8, 2002 - Friday
Purpose
Establishes an Office of Rare Diseases within the National Institutes of Health (NIH) and
authorizes appropriations for Rare Disease Regional Centers of Excellence.
Agency Recommendations
Office of Management and Budget
Approval
Department of Health and Human Services (HHS)
Approval
Discussion
H.R. 4013 would establish in statute an Office of Rare Diseases within the Office of the
Director of NIH. (The Office was established administratively in 1993.) A rare disease is
defined as one that afflicts fewer than 200,000 individuals in the United States. According to the
Food and Drug Administration, there are more than 6,000 rare diseases affecting 20 to 25 million
people in the United States. The Office would be responsible for recommending an agenda for
conducting and supporting research; promoting coordination and cooperation among NIH
institutes and centers with regard to rare diseases; promoting the sufficient allocation of resources
within NIH for conducting and supporting research on rare diseases; and encouraging the
establishment of a centralized clearinghouse for rare and genetic disease information for use by
the public, medical professionals, patients, and their families. H.R. 4013 would authorize
appropriations of $4 million for each of FYs 2003-2006 to carry out the responsibilities of the
Office. The FY 2003 Budget requests $11.3 million for the Office.
H.R. 4013 also would authorize appropriations of $20 million for each of FYs 2003-2006
for Rare Disease Regional Centers of Excellence. The Office of Rare Diseases, in collaboration
with other NIH institutes and centers, would enter into cooperative agreements with and make
grants to public or private nonprofit entities for regional centers of excellence that would conduct
research and training in the diagnosis, prevention, control, and treatment of rare diseases.
Federal support of the centers of excellence would be limited to five years, but could, under
certain conditions, be extended for up to an additional five years. The FY 2003 Budget does not
include funding for rare disease regional centers of excellence.
Conclusion and Recommendations
HHS recommends approval of H.R. 4013. In its views letter on the enrolled bill, the
Department states that the enrolled bill "is consistent with the Department's policies and
objectives." For example, HHS notes that "NIH has been planning to establish Specialized
Research and Diagnostic Centers of Excellence for Rare Diseases, which it considers crucial to
coordinate research on rare diseases."
We join HHS in recommending approval of H.R. 4013, which passed the House by voice
vote and the Senate by unanimous consent.
MEDanies)
Mitchell E. Daniels, Jr.
Director
Enclosures
-2-