[House Report 111-637]
[From the U.S. Government Publishing Office]
111th Congress Report
HOUSE OF REPRESENTATIVES
2d Session 111-637
======================================================================
ACQUIRED BONE MARROW FAILURE DISEASE RESEARCH AND TREATMENT ACT OF 2010
_______
September 28, 2010.--Committed to the Committee of the Whole House on
the State of the Union and ordered to be printed
_______
Mr. Waxman, from the Committee on Energy and Commerce, submitted the
following
R E P O R T
[To accompany H.R. 1230]
[Including cost estimate of the Congressional Budget Office]
The Committee on Energy and Commerce, to whom was referred
the bill (H.R. 1230) to amend the Public Health Service Act to
provide for the establishment of a National Acquired Bone
Marrow Failure Disease Registry, to authorize research on
acquired bone marrow failure diseases, and for other purposes,
having considered the same, report favorably thereon with
amendments and recommend that the bill as amended do pass.
CONTENTS
Page
Amendment........................................................ 2
Purpose and Summary.............................................. 3
Background and Need for Legislation.............................. 3
Committee Consideration.......................................... 3
Committee Votes.................................................. 4
Committee Oversight Findings and Recommendations................. 4
New Budget Authority, Entitlement Authority, and Tax Expenditures 4
Statement of General Performance Goals and Objectives............ 4
Constitutional Authority Statement............................... 4
Earmarks and Tax and Tariff Benefits............................. 4
Federal Advisory Committee Statement............................. 4
Applicability of Law to the Legislative Branch................... 5
Federal Mandates Statement....................................... 5
Committee Cost Estimate.......................................... 5
Congressional Budget Office Cost Estimate........................ 5
Section-by-Section Analysis of the Legislation................... 7
Explanation of Amendment......................................... 7
Changes in Existing Law Made by the Bill, as Reported............ 7
Amendment
The amendment is as follows:
Strike all after the enacting clause and insert the
following:
SECTION 1. SHORT TITLE.
This Act may be cited as the ``Acquired Bone Marrow Failure Disease
Research and Treatment Act of 2010''.
SEC. 2. ACQUIRED BONE MARROW FAILURE DISEASE RESEARCH.
Part B of title III of the Public Health Service Act (42 U.S.C. 243
et seq.) is amended by inserting after section 317T the following:
``SEC. 317U. ACQUIRED BONE MARROW FAILURE DISEASE RESEARCH.
``(a) In General.--The Secretary may conduct research on acquired
bone marrow failure diseases. Such research may address factors
including--
``(1) trends in the characteristics of individuals who are
diagnosed with acquired bone marrow failure diseases, including
age, race and ethnicity, general geographic location, sex,
family history, and any other characteristics determined
appropriate by the Secretary;
``(2) the genetic and environmental factors, including
exposure to toxins, that may be associated with developing
acquired bone marrow failure diseases;
``(3) approaches to treating acquired bone marrow failure
diseases;
``(4) outcomes for individuals treated for acquired bone
marrow failure diseases, including outcomes for recipients of
stem cell therapeutic products; and
``(5) any other factors pertaining to acquired bone marrow
failure diseases determined appropriate by the Secretary.
``(b) Collaboration With the Radiation Injury Treatment Network.--In
carrying out subsection (a), the Secretary may collaborate with the
Radiation Injury Treatment Network of the C.W. Bill Young Cell
Transplantation Program established pursuant to section 379 to--
``(1) augment data for the studies under such subsection;
``(2) access technical assistance that may be provided by the
Radiation Injury Treatment Network; or
``(3) perform joint research projects.
``(c) Definition.--In this section, the term `acquired bone marrow
failure disease' means--
``(1) myelodysplastic syndromes (MDS);
``(2) aplastic anemia;
``(3) paroxysmal nocturnal hemoglobinuria (PNH);
``(4) pure red cell aplasia;
``(5) acute myeloid leukemia that has progressed from
myelodysplastic syndromes;
``(6) large granular lymphocytic leukemia; or
``(7) any other bone marrow failure disease specified by the
Secretary, to the extent such disease is acquired and not
inherited, as determined by the Secretary.''.
SEC. 3. MINORITY-FOCUSED PROGRAMS ON ACQUIRED BONE MARROW FAILURE
DISEASES.
Title XVII of the Public Health Service Act (42 U.S.C. 300u et seq.)
is amended by inserting after section 1707A the following:
``SEC. 1707B. MINORITY-FOCUSED PROGRAMS ON ACQUIRED BONE MARROW FAILURE
DISEASES.
``(a) Information and Referral Services.--
``(1) In general.--The Secretary may establish and coordinate
outreach and informational programs targeted to minority
populations, including Hispanic, Asian-American, Native
Hawaiian, and Pacific Islander populations, that are affected
by acquired bone marrow failure diseases.
``(2) Program activities.--Programs under subsection (a) may
carry out activities that include--
``(A) making information about treatment options and
clinical trials for acquired bone marrow failure
diseases publicly available; and
``(B) providing referral services for treatment
options and clinical trials.
``(b) Definition.--In this section, the term `acquired bone marrow
failure disease' has the meaning given such term in section 317U(c).''.
SEC. 4. BEST PRACTICES FOR DIAGNOSIS OF AND CARE FOR INDIVIDUALS WITH
ACQUIRED BONE MARROW FAILURE DISEASES.
Part B of title III of the Public Health Service Act (42 U.S.C. 243
et seq.), as amended by section 2, is further amended by inserting
after section 317U the following:
``SEC. 317V. BEST PRACTICES FOR DIAGNOSIS OF AND CARE FOR INDIVIDUALS
WITH ACQUIRED BONE MARROW FAILURE DISEASES.
``(a) Grants.--The Secretary, acting through the Director of the
Agency for Healthcare Research and Quality, may award grants to
researchers to study best practices with respect to diagnosing acquired
bone marrow failure diseases and providing care to individuals with
such diseases.
``(b) Definition.--In this section, the term `acquired bone marrow
failure disease' has the meaning given such term in section 317U(c).''.
Amend the title so as to read:
A bill to amend the Public Health Service Act to provide
for research on acquired bone marrow failure diseases,
minority-focused programs on such diseases, and the development
of best practices for diagnosis of and care for individuals
with such diseases.
Purpose and Summary
H.R. 1230, the ``Acquired Bone Marrow Failure Disease
Research and Treatment Act of 2010'', was introduced on
February 26, 2009, by Rep. Doris O. Matsui (D-CA) and referred
to the Committee on Energy and Commerce.
The goal of H.R. 1230 is to support research on acquired
bone marrow failure diseases, establish and coordinate outreach
and informational programs on such diseases targeted to
minority populations, and promote best practices for the
diagnosis of and care for, individuals with these diseases.
Background and Need for Legislation
Bone marrow produces the blood cells that circulate in the
human body. A bone marrow failure disease occurs when bone
marrow stops functioning or begins to produce abnormal blood
cells; it can be either inherited or acquired. These diseases,
while rare, affect thousands of adults and children every year.
They include aplastic anemia, myelodysplastic syndromes,
paroxysmal nocturnal hemoglobinuria, pure red cell aplasias,
and sideroblastic anemia.
While federal research is ongoing on both inherited and
acquired bone marrow failure diseases, further efforts are
needed to study these conditions.
Committee Consideration
H.R. 1230, the ``Acquired Bone Marrow Failure Disease
Research and Treatment Act of 2010'', was introduced by Ms.
Matsui of California on February 26, 2009, and referred to the
Committee on Energy and Commerce. The bill was subsequently
referred to the Subcommittee on Health on March 6, 2010. On
September 15, 2010, the Subcommittee held a legislative hearing
on the bill. The Subcommittee met in open markup session to
consider H.R. 1230 on September 16, 2010. An amendment in the
nature of a substitute (manager's amendment) by Mr. Pallone of
New Jersey was adopted by a voice vote. Subsequently, H.R. 1230
was favorably forwarded to the full Committee, amended, by a
voice vote.
On September 23, 2010, the Committee on Energy and Commerce
met in open markup session and considered H.R. 1230 as approved
by the Subcommittee. There were no amendments offered in full
Committee and subsequently the Committee ordered H.R. 1230
favorably reported to the House, as amended by the Subcommittee
on Health, by a voice vote.
Committee Votes
Clause 3(b) of rule XIII of the Rules of the House of
Representatives requires the Committee to list each record vote
on the motion to report legislation and amendments thereto. A
motion by Mr. Pallone ordering H.R. 1230 reported to the House,
as amended, was approved by a voice vote. There were no record
votes taken during consideration of this bill.
Committee Oversight Findings and Recommendations
In compliance with clause 3(c)(1) of rule XIII and clause
(2)(b)(1) of rule X of the Rules of the House of
Representatives, the oversight findings and recommendations of
the Committee are reflected in the descriptive portions of this
report, including the finding that further efforts are needed
to study acquired bone marrow failure diseases.
New Budget Authority, Entitlement Authority, and Tax Expenditures
Regarding compliance with clause 3(c)(2) of rule XIII of
the Rules of the House of Representatives, the Committee finds
that H.R. 1230 would result in no new budget authority,
entitlement authority, or tax expenditures or revenues.
Statement of General Performance Goals and Objectives
In accordance with clause 3(c)(4) of rule XIII of the Rules
of the House of Representatives, the performance goals and
objectives of the Committee are reflected in the descriptive
portions of this report, including the goal of supporting
efforts to research acquired bone marrow failure diseases.
Constitutional Authority Statement
Pursuant to clause 3(d)(1) of rule XIII of the Rules of the
House of Representatives, the Committee finds that the
constitutional authority for H.R. 1230 is provided under
article I, section 8, clauses 3 and 18 of the Constitution of
the United States.
Earmarks and Tax and Tariff Benefits
H.R. 1230 does not contain any congressional earmarks,
limited tax benefits, or limited tariff benefits as defined in
clause 9 of rule XXI of the Rules of the House of
Representatives.
Federal Advisory Committee Statement
The Committee finds that the legislation does not establish
or authorize the establishment of an advisory committee within
the definition of 5 U.S.C. App., section 5(b) of the Federal
Advisory Committee Act.
Applicability of Law to the Legislative Branch
Section 102(b)(3) of Public Law 104-1 requires a
description of the application of this bill to the legislative
branch where the bill relates to terms and conditions of
employment or access to public services and accommodations.
H.R. 1230 contains no such provisions.
Federal Mandates Statement
Section 423 of the Congressional Budget and Impoundment
Control Act of 1974 (as amended by section 101(a)(2) of the
Unfunded Mandates Reform Act, Public Law 104-4) requires a
statement on whether the provisions of the report include
unfunded mandates. In compliance with this requirement the
Committee adopts as its own the analysis of federal mandates
prepared by the Director of the Congressional Budget Office
regarding H.R. 1230.
Committee Cost Estimate
Pursuant to clause 3(d)(2) of rule XIII of the Rules of the
House of Representatives, the Committee adopts as its own the
cost estimate of H.R. 1230 prepared by the Director of the
Congressional Budget Office pursuant to section 402 of the
Congressional Budget Act of 1974.
Congressional Budget Office Cost Estimate
With respect to the requirements of clause (3)(c)(3) of
rule XIII of the Rules of the House of Representatives and
section 402 of the Congressional Budget Act of 1974, the
Committee has received the following cost estimate for H.R.
1230 from the Director of Congressional Budget Office:
U.S. Congress,
Congressional Budget Office,
Washington, DC, September 27, 2010.
Hon. Henry A. Waxman,
Chairman, Committee on Energy and Commerce,
U.S. House of Representatives, Washington, DC.
Dear Mr. Chairman: The Congressional Budget Office has
prepared the enclosed cost estimate for H.R. 1230, the Acquired
Bone Marrow Failure Disease Research and Treatment Act of 2010.
If you wish further details on this estimate, we will be
pleased to provide them. The CBO staff contact is Lisa Ramirez-
Branum.
Sincerely,
Douglas W. Elmendorf.
Enclosure.
H.R. 1230--Acquired Bone Marrow Failure Disease Research and Treatment
Act of 2010
Summary: H.R. 1230 would amend the Public Health Service
Act to authorize the Secretary of the Department of Health and
Human Services (HHS) to conduct research and outreach
activities related to diseases involving the failure of
acquired bone marrow. Additionally, the bill would authorize
the Director of the Agency for Healthcare Research and Quality
(AHRQ) to award grants to researchers to study best practices
with respect to diagnosing and providing care to individuals
with such diseases.
Assuming the appropriation of necessary amounts, CBO
estimates that implementing the bill would cost $2 million in
2011 and $26 million over the 2011-2015 period. Enacting H.R.
1230 would not affect direct spending or revenues; therefore,
pay-as-you-go procedures do not apply.
H.R. 1230 contains no intergovernmental or private-sector
mandates as defined in the Unfunded Mandates Reform Act (UMRA).
Estimated cost to the Federal Government: The estimated
budgetary impact of H.R. 1230 is shown in the following table.
The costs of this legislation fall within budget function 550
(health).
----------------------------------------------------------------------------------------------------------------
By fiscal year, in millions of dollars--
-----------------------------------------------------------
2011-
2011 2012 2013 2014 2015 2015
----------------------------------------------------------------------------------------------------------------
CHANGES IN SPENDING SUBJECT TO APPROPRIATION
Estimated Authorization Level....................... 6 6 6 7 7 32
Estimated Outlays................................... 2 5 6 6 6 26
----------------------------------------------------------------------------------------------------------------
Note: Components may not sum to totals because of rounding.
Basis of estimate: For this estimate, CBO assumes that the
legislation will be enacted near the beginning of fiscal year
2011 and that the necessary amounts will be appropriated for
each year. Estimated outlays are based on historical spending
patterns for similar federal programs and on information
provided by HHS, AHRQ, and the Office of Minority Health (OMH).
H.R. 1230 would authorize the Secretary of HHS to conduct
research on diseases involving the failure of acquired bone
marrow in collaboration with the department's Radiation Injury
Treatment Network. CBO does not anticipate that any costs would
be incurred under this provision beyond what the National
Institutes of Health will spend under current law to conduct
similar research over the next several years.
H.R. 1230 also would authorize the Secretary to establish
and coordinate outreach and informational programs targeted to
minority populations that are affected by diseases involving
the failure of acquired bone marrow. Based on information from
OMH, CBO estimates that this provision would require
appropriations totalling $16 million over the 2011-2015 period.
Assuming appropriation of those amounts, CBO estimates that
implementing those programs would cost $13 million over the
2011-2015 period.
The bill also would authorize AHRQ to award grants to
researchers to study best practices with respect to diagnosing
and providing care to individuals with diseases involving the
failure of acquired bone marrow. Based on spending by AHRQ for
similar activities, CBO estimates that implementing this
provision would require appropriations totalling $17 million
over the 2011-2015 period. Assuming appropriation of those
amounts, CBO estimates that implementing that grant program
would cost $13 million over the 2011-2015 period.
Intergovernmental and private-sector impact: H.R. 1230
contains no intergovernmental or private-sector mandates as
defined in UMRA and would impose no costs on the budgets of
state, local, or tribal governments.
Estimate prepared by: Federal Costs: Jamease Kowalczyk and
Lisa Ramirez-Branum; Impact on State, Local, and Tribal
Governments: Lisa Ramirez-Branum; Impact on the Private Sector:
Sarah Axeen.
Estimate approved by: Holly Harvey, Deputy Assistant
Director for Budget Analysis.
Section-by-Section Analysis of the Legislation
Section 1. Short title
Section 1 designates that the short title may be cited as
the ``Acquired Bone Marrow Failure Disease Research and
Treatment Act of 2010''.
Section 2. Acquired bone marrow failure disease research
Section 2 authorizes the Secretary of Health and Human
Services (HHS) to support research on acquired bone marrow
failure diseases and specifies factors that such research may
address. In supporting this research, the Secretary may
collaborate with the Radiation Injury Treatment Network of the
C.W. Bill Young Cell Transplantation Program (established under
Section 379 of the Public Health Service Act).
Section 3. Minority-focused programs on acquired bone marrow failure
diseases
Section 3 provides authority for the Secretary to establish
outreach and information programs targeted to minority
populations affected by acquired bone marrow failure disease
and provide referral services for treatment options and
clinical services.
Section 4. Best practices for diagnosis of and care for individuals
with acquired bone marrow failure diseases
Section 4 provides authority for the Secretary, acting
through the Director of the Agency for Healthcare Research and
Quality (AHRQ), to award grants to researchers to study best
practices with regard to the diagnosis and treatment of
individuals with acquired bone marrow diseases.
Explanation of Amendment
During the Subcommittee on Health markup of H.R. 1230, Mr.
Pallone of New Jersey offered an amendment in the nature of a
substitute (manager's amendment), which was adopted by a voice
vote. The substance of the substitute amendment is reflected in
the section-by-section analysis contained in this report.
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 III--GENERAL POWERS AND DUTIES OF PUBLIC HEALTH SERVICE
* * * * * * *
Part B--Federal-State Cooperation
* * * * * * *
SEC. 317U. ACQUIRED BONE MARROW FAILURE DISEASE RESEARCH.
(a) In General.--The Secretary may conduct research on
acquired bone marrow failure diseases. Such research may
address factors including--
(1) trends in the characteristics of individuals who
are diagnosed with acquired bone marrow failure
diseases, including age, race and ethnicity, general
geographic location, sex, family history, and any other
characteristics determined appropriate by the
Secretary;
(2) the genetic and environmental factors, including
exposure to toxins, that may be associated with
developing acquired bone marrow failure diseases;
(3) approaches to treating acquired bone marrow
failure diseases;
(4) outcomes for individuals treated for acquired
bone marrow failure diseases, including outcomes for
recipients of stem cell therapeutic products; and
(5) any other factors pertaining to acquired bone
marrow failure diseases determined appropriate by the
Secretary.
(b) Collaboration With the Radiation Injury Treatment
Network.--In carrying out subsection (a), the Secretary may
collaborate with the Radiation Injury Treatment Network of the
C.W. Bill Young Cell Transplantation Program established
pursuant to section 379 to--
(1) augment data for the studies under such
subsection;
(2) access technical assistance that may be provided
by the Radiation Injury Treatment Network; or
(3) perform joint research projects.
(c) Definition.--In this section, the term ``acquired bone
marrow failure disease'' means--
(1) myelodysplastic syndromes (MDS);
(2) aplastic anemia;
(3) paroxysmal nocturnal hemoglobinuria (PNH);
(4) pure red cell aplasia;
(5) acute myeloid leukemia that has progressed from
myelodysplastic syndromes;
(6) large granular lymphocytic leukemia; or
(7) any other bone marrow failure disease specified
by the Secretary, to the extent such disease is
acquired and not inherited, as determined by the
Secretary.
SEC. 317V. BEST PRACTICES FOR DIAGNOSIS OF AND CARE FOR INDIVIDUALS
WITH ACQUIRED BONE MARROW FAILURE DISEASES.
(a) Grants.--The Secretary, acting through the Director of
the Agency for Healthcare Research and Quality, may award
grants to researchers to study best practices with respect to
diagnosing acquired bone marrow failure diseases and providing
care to individuals with such diseases.
(b) Definition.--In this section, the term ``acquired bone
marrow failure disease'' has the meaning given such term in
section 317U(c).
* * * * * * *
TITLE XVII--HEALTH INFORMATION AND HEALTH PROMOTION
* * * * * * *
SEC. 1707B. MINORITY-FOCUSED PROGRAMS ON ACQUIRED BONE MARROW FAILURE
DISEASES.
(a) Information and Referral Services.--
(1) In general.--The Secretary may establish and
coordinate outreach and informational programs targeted
to minority populations, including Hispanic, Asian-
American, Native Hawaiian, and Pacific Islander
populations, that are affected by acquired bone marrow
failure diseases.
(2) Program activities.--Programs under subsection
(a) may carry out activities that include--
(A) making information about treatment
options and clinical trials for acquired bone
marrow failure diseases publicly available; and
(B) providing referral services for treatment
options and clinical trials.
(b) Definition.--In this section, the term ``acquired bone
marrow failure disease'' has the meaning given such term in
section 317U(c).
* * * * * * *