I 

111TH CONGRESS 

1ST SESSION 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. 

IN THE HOUSE OF REPRESENTATIVES 

FEBRUARY 26, 2009 

Ms. MATSUI (for herself, Mr. BISHOP of Georgia, Mr. MURTHA, Mr. 

MCDERMOTT, Mrs. EMERSON, Mr. GENE GREEN of Texas, Mr. ABERCROMBIE, Ms. SCHWARTZ, Mr. CONYERS, Ms. HIRONO, Ms. BORDALLO, 

Mr. LEWIS of Georgia, Mr. FRANK of Massachusetts, Mr. WOLF, Mr. 

KING of New York, Mr. VAN HOLLEN, Mr. MCGOVERN, Ms. DEGETTE, 

Mr. ARCURI, and Ms. ESHOO) introduced the following bill; which was 

referred to the Committee on Energy and Commerce 

A BILL 

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. 

1 Be it enacted by the Senate and House of Representa2 tives of the United States of America in Congress assembled, 

3 SECTION 1. SHORT TITLE. 

4 This Act may be cited as the ‘‘Bone Marrow Failure 

5 Disease Research and Treatment Act of 2009’’. 


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1 SEC. 2. FINDINGS. 

2 The Congress finds the following: 

3 (1) Between 20,000 and 30,000 Americans are 

4 diagnosed each year with myelodysplastic syndromes, 

5 aplastic anemia, paroxysmal nocturnal hemo6 globinuria, and other acquired bone marrow failure 

7 diseases. 

8 (2) Acquired bone marrow failure diseases have 

9 a debilitating and often fatal impact on those diag10 nosed with these disease. 

11 (3) While some treatments for acquired bone 

12 marrow failure diseases can prolong and improve the 

13 quality of patients’ lives, there is no single cure for 

14 these diseases. 

15 (4) The prevalence of acquired bone marrow 

16 failure diseases in the United States will continue to 

17 grow as the general public ages. 

18 (5) Evidence exists suggesting that acquired 

19 bone marrow failure diseases occur more often in 

20 minority populations, particularly in Asian-American 

21 and Hispanic/Latino populations. 

22 (6) The National Heart, Lung, and Blood Insti23 tute and the National Cancer Institute have con24 ducted important research into the causes of and 

25 treatments for acquired bone marrow failure dis26 eases. 


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1 (7) The National Marrow Donor Program Reg2 istry has made significant contributions to the fight 

3 against bone marrow failure diseases by connecting 

4 millions of potential marrow donors with individuals 

5 and families suffering from these conditions. 

6 (8) Despite these advances, a more comprehen7 sive Federal strategic effort among numerous Fed8 eral agencies is needed to discover a cure for ac9 quired bone marrow failure disorders. 

10 (9) Greater Federal surveillance of acquired 

11 bone marrow failure diseases is needed to gain a bet12 ter understanding of the causes of acquired bone 

13 marrow failure diseases. 

14 (10) The Federal Government should increase 

15 its research support for and engage with public and 

16 private organizations in developing a comprehensive 

17 approach to combat and cure acquired bone marrow 

18 failure diseases. 

19 SEC. 3. NATIONAL ACQUIRED BONE MARROW FAILURE DIS20 EASE REGISTRY. 

21 Part B of the Public Health Service Act (42 U.S.C. 

22 311 et seq.) is amended by inserting after section 317T 

23 the following: 


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1 ‘‘SEC. 317U. NATIONAL ACQUIRED BONE MARROW FAILURE 

2 DISEASE REGISTRY. 

3 ‘‘(a) ESTABLISHMENT OF REGISTRY.— 

4 ‘‘(1) IN GENERAL.—Not later than 6 months 

5 after the date of the enactment of this section, the 

6 Secretary, acting through the Director of the Cen7 ters for Disease Control and Prevention, shall— 

8 ‘‘(A) develop a system to collect data on 

9 acquired bone marrow failure diseases; and 

10 ‘‘(B) establish and maintain a national and 

11 publicly available registry, to be known as the 

12 National Acquired Bone Marrow Failure Dis13 ease Registry, in accordance with paragraph 

14 (3). 

15 ‘‘(2) RECOMMENDATIONS OF ADVISORY COM16 MITTEE.—In carrying out this subsection, the Sec17 retary shall take into consideration the recommenda18 tions of the Advisory Committee on Acquired Bone 

19 Marrow Failure Diseases established under sub20 section (b). 

21 ‘‘(3) PURPOSES OF REGISTRY.—The National 

22 Acquired Bone Marrow Failure Disease Registry— 

23 ‘‘(A) shall identify the incidence and preva24 lence of acquired bone marrow failure diseases 

25 in the United States; 


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1 ‘‘(B) shall be used to collect and store data 

2 on acquired bone marrow failure diseases, in3 cluding data concerning— 

4 ‘‘(i) the age, race or ethnicity, general 

5 geographic location, sex, and family history 

6 of individuals who are diagnosed with ac7 quired bone marrow failure diseases, and 

8 any other characteristics of such individ9 uals determined appropriate by the Sec10 retary; 

11 ‘‘(ii) the genetic and environmental 

12 factors that may be associated with devel13 oping acquired bone marrow failure dis14 eases; 

15 ‘‘(iii) treatment approaches for deal16 ing with acquired bone marrow failure dis17 eases; 

18 ‘‘(iv) outcomes for individuals treated 

19 for acquired bone marrow failure diseases, 

20 including outcomes for recipients of stem 

21 cell therapeutic products as contained in 

22 the database established pursuant to sec23 tion 379A; and 


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1 ‘‘(v) any other factors pertaining to 

2 acquired bone marrow failure diseases de3 termined appropriate by the Secretary; and 

4 ‘‘(C) shall be made available— 

5 ‘‘(i) to the general public; and 

6 ‘‘(ii) to researchers to facilitate fur7 ther research into the causes of, and treat8 ments for, acquired bone marrow failure 

9 diseases in accordance with standard prac10 tices of the Centers for Disease Control 

11 and Preventions. 

12 ‘‘(b) ADVISORY COMMITTEE.— 

13 ‘‘(1) ESTABLISHMENT.—Not later than 6 

14 months after the date of the enactment of this sec15 tion, the Secretary, acting through the Director of 

16 the Centers for Disease Control and Prevention, 

17 shall establish an advisory committee, to be known 

18 as the Advisory Committee on Acquired Bone Mar19 row Failure Diseases. 

20 ‘‘(2) MEMBERS.—The members of the Advisory 

21 Committee on Acquired Bone Marrow Failure Dis22 eases shall be appointed by the Secretary, acting 

23 through the Director of the Centers for Disease 

24 Control and Prevention, and shall include at least 

25 one representative from each of the following: 


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1 ‘‘(A) A national patient advocacy organiza2 tion with experience advocating on behalf of pa3 tients suffering from acquired bone marrow 

4 failure diseases. 

5 ‘‘(B) The National Institutes of Health, in6 cluding at least one representative from each 

7 of— 

8 ‘‘(i) the National Cancer Institute; 

9 ‘‘(ii) the National Heart, Lung, and 

10 Blood Institute; and 

11 ‘‘(iii) the Office of Rare Diseases. 

12 ‘‘(C) The Centers for Disease Control and 

13 Prevention. 

14 ‘‘(D) Clinicians with experience in— 

15 ‘‘(i) diagnosing or treating acquired 

16 bone marrow failure diseases; and 

17 ‘‘(ii) medical data registries. 

18 ‘‘(E) Epidemiologists who have experience 

19 with data registries. 

20 ‘‘(F) Publicly or privately funded research21 ers who have experience researching acquired 

22 bone marrow failure diseases. 

23 ‘‘(G) The entity operating the C.W. Bill 

24 Young Cell Transplantation Program estab25 lished pursuant to section 379 and the entity 


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1 operating the C.W. Bill Young Cell Transplan2 tation Program Outcomes Database. 

3 ‘‘(3) RESPONSIBILITIES.—The Advisory Com4 mittee on Acquired Bone Marrow Failure Diseases 

5 shall provide recommendations to the Secretary on 

6 the establishment and maintenance of the National 

7 Acquired Bone Marrow Failure Disease Registry, in8 cluding recommendations on the collection, mainte9 nance, and dissemination of data. 

10 ‘‘(4) PUBLIC AVAILABILITY.—The Secretary 

11 shall make the recommendations of the Advisory 

12 Committee on Acquired Bone Marrow Failure Dis13 ease publicly available. 

14 ‘‘(c) GRANTS.—The Secretary, acting through the 

15 Director of the Centers for Disease Control and Preven16 tion, may award grants to, and enter into contracts and 

17 cooperative agreements with, public or private nonprofit 

18 entities for the management of, as well as the collection, 

19 analysis, and reporting of data to be included in, the Na20 tional Acquired Bone Marrow Failure Disease Registry. 

21 ‘‘(d) DEFINITION.—In this section, the term ‘ac22 quired bone marrow failure disease’ means— 

23 ‘‘(1) myelodysplastic syndromes (MDS); 

24 ‘‘(2) aplastic anemia; 


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1 ‘‘(3) paroxysmal nocturnal hemoglobinuria 

2 (PNH); 

3 ‘‘(4) pure red cell aplasia; 

4 ‘‘(5) acute myeloid leukemia that has pro5 gressed from myelodysplastic syndromes; or 

6 ‘‘(6) large granular lymphocytic leukemia. 

7 ‘‘(e) AUTHORIZATION OF APPROPRIATIONS.—There 

8 is authorized to be appropriated to carry out this section 

9 $3,000,000 for each of fiscal years 2010 through 2014.’’. 

10 SEC. 4. PILOT STUDIES THROUGH THE AGENCY FOR TOXIC 

11 SUBSTANCES AND DISEASE REGISTRY. 

12 (a) PILOT STUDIES.—The Secretary of Health and 

13 Human Services, acting through the Administrator of the 

14 Agency for Toxic Substances and Disease Registry, shall 

15 conduct pilot studies to determine which environmental 

16 factors, including exposure to toxins, may cause acquired 

17 bone marrow failure diseases. 

18 (b) COLLABORATION WITH THE RADIATION INJURY

19 TREATMENT NETWORK.—In carrying out the directives of 

20 this section, the Secretary may collaborate with the Radi21 ation Injury Treatment Network of the C.W. Bill Young 

22 Cell Transplantation Program established pursuant to sec23 tion 379 of the Public Health Service Act (42 U.S.C. 

24 274j) to— 


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1 (1) augment data for the pilot studies author2 ized by this section; 

3 (2) access technical assistance that may be pro4 vided by the Radiation Injury Treatment Network; 

5 or 

6 (3) perform joint research projects. 

7 (c) AUTHORIZATION OF APPROPRIATIONS.—There is 

8 authorized to be appropriated to carry out this section 

9 $1,000,000 for each of fiscal years 2010 through 2014. 

10 SEC. 5. MINORITY-FOCUSED PROGRAMS ON ACQUIRED 

11 BONE MARROW FAILURE DISEASES. 

12 Title XVII of the Public Health Service Act (42 

13 U.S.C. 300u et seq.) is amended by inserting after section 

14 1707 the following: 

15 ‘‘MINORITY-FOCUSED PROGRAMS ON ACQUIRED BONE

16 MARROW FAILURE DISEASES

17 ‘‘SEC. 1707A. (a) INFORMATION AND REFERRAL

18 SERVICES.— 

19 ‘‘(1) IN GENERAL.—Not later than 6 months 

20 after the date of the enactment of this section, the 

21 Secretary, acting through the Deputy Assistant Sec22 retary for Minority Health, shall establish and co23 ordinate outreach and informational programs tar24 geted to minority populations affected by acquired 

25 bone marrow failure diseases. 


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1 ‘‘(2) PROGRAM REQUIREMENTS.—Minority-fo2 cused outreach and informational programs author3 ized by this section— 

4 ‘‘(A) shall make information about treat5 ment options and clinical trials for acquired 

6 bone marrow failure diseases publicly available, 

7 and 

8 ‘‘(B) shall provide referral services for 

9 treatment options and clinical trials, 

10 at the national minority health resource center sup11 ported under section 1707(b)(8) (including by means 

12 of the center’s website, through appropriate loca13 tions such as the center’s knowledge center, and 

14 through appropriate programs such as the center’s 

15 resource persons network) and through minority 

16 health consultants located at each Department of 

17 Health and Human Services regional office. 

18 ‘‘(b) HISPANIC AND ASIAN-AMERICAN AND PACIFIC

19 ISLANDER OUTREACH.— 

20 ‘‘(1) IN GENERAL.—The Secretary, acting 

21 through the Deputy Assistant Secretary for Minority 

22 Health, shall undertake a coordinated outreach ef23 fort to connect Hispanic, Asian-American, and Pa24 cific Islander communities with comprehensive servVerDate Nov 24 2008 00:59 Feb 28, 2009 Jkt 079200 PO 00000 Frm 00011 Fmt 6652 Sfmt 6201 E:\BILLS\H1230.IH H1230

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1 ices focused on treatment of, and information about, 

2 acquired bone marrow failure diseases. 

3 ‘‘(2) COLLABORATION.—In carrying out this 

4 subsection, the Secretary may collaborate with public 

5 health agencies, nonprofit organizations, community 

6 groups, and online entities to disseminate informa7 tion about treatment options and clinical trials for 

8 acquired bone marrow failure diseases. 

9 ‘‘(c) GRANTS AND COOPERATIVE AGREEMENTS.— 

10 ‘‘(1) IN GENERAL.—Not later than 6 months 

11 after the date of the enactment of this section, the 

12 Secretary, acting through the Deputy Assistant Sec13 retary for Minority Health, shall award grants to, or 

14 enter into cooperative agreements with, entities to 

15 perform research on acquired bone marrow failure 

16 diseases. 

17 ‘‘(2) REQUIREMENT.—Grants and cooperative 

18 agreements authorized by this subsection shall be 

19 awarded or entered into on a competitive, peer-re20 viewed basis. 

21 ‘‘(3) SCOPE OF RESEARCH.—Research funded 

22 under this section shall examine factors affecting the 

23 incidence of acquired bone marrow failure diseases 

24 in minority populations. 


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1 ‘‘(d) DEFINITION.—In this section, the term ‘ac2 quired bone marrow failure disease’ has the meaning given 

3 to such term in section 317U(d). 

4 ‘‘(e) AUTHORIZATION OF APPROPRIATIONS.—There 

5 is authorized to be appropriated to carry out this section 

6 $2,000,000 for each of fiscal years 2010 through 2014.’’. 

7 SEC. 6. DIAGNOSIS AND QUALITY OF CARE FOR ACQUIRED 

8 BONE MARROW FAILURE DISEASES. 

9 (a) GRANTS.—The Secretary of Health and Human 

10 Services, acting through the Director of the Agency for 

11 Healthcare Research and Quality, shall award grants to 

12 entities to improve diagnostic practices and quality of care 

13 with respect to patients with acquired bone marrow failure 

14 diseases. 

15 (b) AUTHORIZATION OF APPROPRIATIONS.—There is 

16 authorized to be appropriated to carry out this section 

17 $2,000,000 for each of fiscal years 2010 through 2014. 

18 SEC. 7. DEFINITION. 

19 In this Act, the term ‘‘acquired bone marrow failure 

20 disease’’ means— 

21 (1) myelodysplastic syndromes (MDS); 

22 (2) aplastic anemia; 

23 (3) paroxysmal nocturnal hemoglobinuria 

24 (PNH); 

25 (4) pure red cell aplasia; 


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1 (5) acute myeloid leukemia that progressed 

2 from myelodysplastic syndromes; or 

3 (6) large granular lymphocytic leukemia. 

Æ 


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