[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).

           *       *       *       *       *       *       *


                                  
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