SECTION 1. SHORT TITLE.

    This Act may be cited as the ``Diamond-Blackfan Anemia Research and 
Care Act''.

SEC. 2. FINDINGS.

    The Congress finds as follows:
            (1) Diamond-Blackfan anemia is a rare genetic bone marrow 
        failure disorder, which develops in infancy and results in 
        severe anemia due to failure to make red blood cells.
            (2) Diamond-Blackfan anemia patients have an increased risk 
        of leukemia, solid tumors, and complete bone marrow failure.
            (3) Fifty percent of patients with Diamond-Blackfan anemia 
        are born with birth defects, including abnormalities to the 
        face, head, upper arm and hand, genitourinary, and heart, and 
        21 percent of the affected patients have more than 1 defect.
            (4) Treatments for Diamond-Blackfan anemia, including the 
        use of blood transfusions and steroids such as prednisone, have 
        potential long-term side effects, including osteoporosis, iron 
        overload (because of the transfusions), and impaired growth 
        (because of the steroids).
            (5) The only cure for Diamond-Blackfan anemia is a bone 
        marrow transplant, a procedure that carries serious risks and, 
        since most patients lack an acceptable donor, is an option for 
        only about 25 percent of patients.
            (6) Because Diamond-Blackfan anemia is a genetic disorder 
        of red cell production and a cancer predisposition syndrome 
        with a high rate of congenital anomalies, the Federal 
        investment regarding Diamond-Blackfan anemia must be expanded 
        to allow the careful dissection of this disease, which will 
        provide valuable insights into the biology of blood disorders 
        and cancer predisposition and serve as an important model for 
        understanding the genetics of birth defects.

SEC. 3. DIAMOND-BLACKFAN ANEMIA.

    (a) DBA-Related Activities of NIH.--Part A of title IV of the 
Public Health Service Act is amended by inserting after section 404G 
(42 U.S.C. 283i) the following:

                       ``diamond-blackfan anemia

    ``Sec. 404H. (a) In General.--The Director of NIH, in coordination 
with the Directors of the National Heart, Lung, and Blood Institute, 
the National Institute of Diabetes and Digestive and Kidney Diseases, 
and the Office of Rare Diseases, shall expand and intensify research 
and related activities of the National Institutes of Health with regard 
to Diamond-Blackfan anemia.
    ``(b) Comprehensive Research Initiative.--
            ``(1) In general.--In carrying out this section, the 
        Director of NIH shall make grants to, or enter into contracts 
        with, public or private entities to support a comprehensive 
        research initiative to study, develop better treatments for, 
        and ultimately find a cure for Diamond-Blackfan anemia.
            ``(2) Research.--The initiative supported under this 
        subsection may include research on the following:
                    ``(A) The links of Diamond-Blackfan anemia to 
                chronic diseases.
                    ``(B) Red cell differentiation.
                    ``(C) The pathophysiology of Diamond-Blackfan 
                anemia.
                    ``(D) The relationship between Diamond-Blackfan 
                anemia and predisposition to cancer.
                    ``(E) Congenital anomalies in Diamond-Blackfan 
                anemia patients.''.
    (b) DBA-Related Activities of CDC.--Part B of title III of the 
Public Health Service Act (42 U.S.C. 243 et seq.) is amended--
            (1) by moving section 317R so that it follows section 317Q; 
        and
            (2) by inserting after section 317R the following:

``SEC. 317S. REGISTRY AND CLINICAL CARE CENTER FOR DIAMOND-BLACKFAN 
              ANEMIA.

    ``(a) Registry.--The Secretary, acting through the Director of the 
Centers for Disease Control and Prevention, shall maintain and expand 
the Diamond-Blackfan Anemia Registry (in this section referred to as 
the `Registry').
    ``(b) Comprehensive Clinical Care Center.--
            ``(1) Establishment.--The Secretary, acting through the 
        Director of the Centers for Disease Control and Prevention, 
        shall establish a comprehensive clinical care center for 
        Diamond-Blackfan anemia where a majority of the patients with 
        the disease are examined, treated, and tracked through the 
        Registry, by experts in the disease.
            ``(2) Duties.--The center established under this section 
        shall--
                    ``(A) gather and analyze extensive data on Diamond-
                Blackfan anemia to be used for public, non-profit, and 
                government research initiatives involving gene 
                discovery, ribosomal protein function, genetics of 
                birth defects, blood cell formation (recovery from 
                cancer chemotherapy), cancer predisposition, red cell 
                differentiation, and a comparison of therapeutic 
                treatments including blood transfusion, steroids, and 
                bone marrow transplants;
                    ``(B) provide thorough examinations of Diamond-
                Blackfan anemia patients by experts in the disease to 
                confirm diagnosis and provide genetic typing along with 
                a multi-system evaluation; and
                    ``(C) provide clinical care for Diamond-Blackfan 
                anemia.''.
Diamond-Blackfan Anemia Research and Care Act - Amends the Public Health Service Act to direct the Director of the National Institutes of Health, in coordination with other specified officials, to expand and intensify research and related activities of the Institute with regard to Diamond-Blackfan Anemia. States that the Director shall as part of such effort award grants to, or enter into contracts with, public or private entities to support a comprehensive research initiative.Directs the Secretary of Health and Human Services, acting through the Director of the Centers for Disease Control and Prevention, to: (1) maintain and expand the Diamond-Blackfan Anemia Registry; and (2) establish a comprehensive clinical care center for Diamond-Blackfan Anemia.
{"src": "billsum_test", "title": "To require increased activities by the National Institutes of Health and the Centers for Disease Control and Prevention regarding Diamond-Blackfan anemia, and for other purposes."}