89–006 

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 


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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).’’. 


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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 apVerDate Mar 15 2010 23:07 Oct 04, 2010 Jkt 089006 PO 00000 Frm 00003 Fmt 6659 Sfmt 6602 E:\HR\OC\HR637.XXX HR637

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proved 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. 


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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 reVerDate Mar 15 2010 03:09 Oct 05, 2010 Jkt 089010 PO 00000 Frm 00005 Fmt 6659 Sfmt 6602 E:\HR\OC\HR637.XXX HR637

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searchers 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 2012 2013 2014 2015 2011– 

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. 


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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 

* * * * * * * 


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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. 


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(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). 

* * * * * * * 

Æ 


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