BILL ANALYSIS                                                                                                                                                                                                    �



                                                                            



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                                    THIRD READING


          Bill No:  SB 224
          Author:   Walters (R)
          Amended:  5/28/13
          Vote:     21


           SENATE HEALTH COMMITTEE  :  9-0, 4/3/13
          AYES:  Hernandez, Anderson, Beall, De Le�n, DeSaulnier, Monning,  
            Nielsen, Pavley, Wolk

           SENATE APPROPRIATIONS COMMITTEE  :  7-0, 5/23/13
          AYES:  De Le�n, Walters, Gaines, Hill, Lara, Padilla, Steinberg


           SUBJECT  :    Newborn screening program

           SOURCE  :     Hunter's Hope Foundation


           DIGEST  :    This bill requires the Department of Public Health  
          (DPH), until January 1, 2019, to expand the screening of  
          newborns in Orange County to include screening for Krabbe  
          disease, and exempts the amendment of contracts for this purpose  
          from existing provisions that establish standards for contracts,  
          as specified.

           ANALYSIS  :    

          Existing law:

          1.Requires DPH to develop a genetic disease testing program.   
            Requires newborn screening for preventable heritable or  
            congenital disorders leading to physical defects or  
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            intellectual disabilities.  Requires newborn screening for  
            sickle cell anemia, phenylketonuria, and severe combined  
            immunodeficiency (SCID).  Also requires tandem mass  
            spectrometry (TMS) screening in newborns, as defined.

          2.Requires DPH to establish a genetic disease unit to coordinate  
            programs in the area of genetic disease and evaluate and  
            prepare recommendations on the implementation of tests for the  
            detection of certain hereditary and congenital diseases.   
            Requires DPH to provide genetic screening and follow-up  
            services.

          3.Requires DPH to charge a fee for newborn screening and  
            follow-up services, and requires the amount of the fee to be  
            established pursuant to regulation and periodically adjusted.

          4.Requires that any fee charged for screening and follow-up  
            services provided to Medi-Cal- eligible persons, health care  
            service plan enrollees, or persons covered by disability  
            insurance policies are to be paid directly to the Genetic  
            Disease Testing Fund, and are subject to the terms and  
            conditions of the health care service plan or insurance  
            coverage.

          5.Requires, under regulation, testing for hereditary  
            hemoglobinopathies, primary congenital hypothyroidism, and  
            galactosemia.

          This bill:

          1.Declares that this act shall be known, and may be cited, as  
            Jacquelyn's Law.

          2.Requires DPH to establish a pilot program to expand in Orange  
            County the screening program established in existing law to  
            include the screening of newborns in that county for Krabbe  
            disease.

          3.Requires the expenditure of funds from the Genetic Disease  
            Testing Fund to be authorized for implementation of this act  
            through the amendment of the Genetic Disease Branch Screening  
            Information System contracts, as defined.  Exempts the  
            contracts from existing policies, procedures, regulations or  
            manuals, as specified.

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          4.Requires Jacquelyn's Law to remain in effect only until  
            January 1, 2019, unless a later enacted statute, that is  
            enacted before January 1, 2019, deletes or extends that date.



           Background
           
          Currently, all babies in California are screened for over 75  
          diseases at birth through newborn screening.  These diseases are  
          not otherwise clinically recognized at birth but if untreated  
          can cause irreversible physical and mental damage to children,  
          with many resulting in death  For these diseases, early  
          detection is essential to avoid such devastating outcomes.   
          Likewise, early detection through newborn screening for Krabbe  
          disease is crucial to give these children a chance at a healthy  
          life.  While treatment options are available, these treatments  
          are only effective when given before symptoms are present.  Most  
          often children are not diagnosed in time and, therefore, are not  
          able to receive these life-saving treatments.

           Newborn Screening Program  .  Newborn screening is recognized  
          nationally as an essential preventive health measure and an  
          important state-based public health program.  All states in the  
          nation and the District of Columbia mandate newborn screening of  
          all infants born within their jurisdiction for certain treatable  
          disorders that may not otherwise be detected before  
          developmental disability or death occurs.  Newborns with these  
          genetically screened disorders typically appear normal at birth.  
           The testing and follow-up services of newborn screening  
          programs are designed to provide early diagnosis and treatment  
          before significant, irreversible damage occurs.

          According to DPH, positive test results are immediately  
          telephoned to a follow-up coordinator at one of the Newborn  
          Screening Area Service Centers throughout the state.  The  
          coordinator contacts the newborn's physician to arrange for  
          repeat testing.  If repeat testing determines that the baby has  
          a disorder, the coordinator will supply the latest clinical  
          information on diagnosis and treatment and assist with referrals  
          to special care.  In 2011-12, approximately 492,000 newborns  
          were screened for 80 genetic disorders.  Approximately 9,250, or  
          under 2%, were classified as positive or questionable and were  

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          referred for follow-up testing or services.

           Krabbe Disease  .  According to the National Institute of  
          Neurological Disorders and Stroke, Krabbe disease is a rare,  
          inherited degenerative disorder of the central and peripheral  
          nervous systems.  Krabbe disease occurs in about 1 out of  
          100,000 individuals in the United States.  The symptoms of  
          Krabbe disease usually begin before the age of one year (the  
          infantile form).  As the disease progresses, muscles continue to  
          weaken, affecting the infant's ability to move, chew, swallow,  
          and breathe.  Less commonly, onset of Krabbe disease can occur  
          in childhood, adolescence, or adulthood (late-onset forms).
          There is no cure for Krabbe disease.  Infantile Krabbe disease  
          is generally fatal before age two.  Results of a very small  
          clinical trial of patients with infantile Krabbe disease found  
          that children who receive cord-blood stem cells from unrelated  
          donors prior to symptom onset developed with little neurological  
          impairment.  Results also showed that disease progression  
          stabilized faster in patients who receive cord blood compared to  
          those who receive adult bone marrow.

           Uniform screening panel  .  The Secretary's Advisory Committee on  
          Heritable Disorders in Newborns and Children (SACHDNC) was  
          chartered in February 2003 to advise the Secretary of the U.S.  
          Department of Health and Human Services (HHS) regarding the most  
          appropriate application of universal newborn screening tests,  
          technologies, policies, guidelines and standards for effectively  
          reducing morbidity and mortality in newborns and children  
          having, or at risk for, heritable disorders.  In 2010, the HHS  
          Secretary adopted the recommendation of SACHDNC for a uniform  
          screening panel.  Lysosomal storage disorders, such as Krabbe  
          desease although considered, was not included in these  
          recommendations.

           Methodology for adding new conditions  .  According to DPH, there  
          is no national standard process to add a disorder to a state's  
          screening program.  Some states will add a disorder once it is  
          recommended by SACHDNC but prior to official acceptance by the  
          HHS Secretary, while other states wait for the official  
          acceptance by the HHS Secretary.  In California, disorders have  
          been added through the budget process or through legislation  
          directing DPH to add a specific disorder to the screening  
          program.


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           Prior Legislation
           
          SB 1072 (Strickland, 2012), would have required DPH, until  
          January 1, 2018, to expand statewide screening of newborns to  
          include screening for Hurler syndrome and Krabbe disease.  SB  
          1072 died in the Senate Appropriations Committee.

          SB 1731 (Block, Chapter 336, Statutes of 2012), establishes the  
          Newborn Critical Congenital Heart Disease (CCHD) Screening  
          Program and requires hospitals, beginning July 1, 2013, to offer  
          a pulse oximetry test for the identification of CCHD to parents  
          of newborns prior to discharge.

          SB 395 (Pan, Chapter 461, Statutes of 2011), expands statewide  
          screening of newborns to include screening for SCID.

          SB 1103 (Senate Budget and Fiscal Review, Chapter 228, Statutes  
          of 2004), expanded statewide screening of newborns to include  
          TMS screening for fatty acid oxidation, amino acid, organic acid  
          disorders, and congenital adrenal hyperplasia.

          AB 442 (Committee on Budget, Chapter 1161, Statutes of 2002),  
          requires hospitals to collect fees associated with any tests  
          conducted under the state's Newborn Screening Program.

          SB 537 (Greene, Chapter 1011, Statutes of 1998), requires DPH to  
          establish a program to provide extended newborn genetic  
          screening services for persons who elect to have, and pay for,  
          the additional screening.

           FISCAL EFFECT  :    Appropriation:  Yes   Fiscal Com.:  Yes    
          Local:  No

          According to the Senate Appropriations Committee:

                 One-time costs to DPH to upgrade information technology  
               systems and adopt regulations of about $210,000 (Genetic  
               Disease Testing Fund).

                 Ongoing costs to expand newborn screening of about  
               $880,000 per year, covered by fees (Genetic Disease Testing  
               Fund).

                 Increased costs to Medi-Cal of $400,000 per year for  

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               testing fees (50% General Fund, 50% federal funds).

                 Earlier diagnosis of infants covered by public health  
               care programs with one of these conditions may result in  
               increased costs, to the extent that ongoing treatments such  
               as enzyme replacement therapy begin earlier than they would  
               without screening for these conditions.  The extent to  
               which this will actually increase costs is unknown.

                 Whether early screening and diagnosis will improve  
               medical outcomes and reduce future treatment costs to  
               public healthcare programs is unknown, given the limited  
               information available on treatment outcomes.

           SUPPORT  :   (Verified  5/23/13)

          Hunter's Hope Foundation (source)



           OPPOSITION  :    (Verified  5/23/13)

          California Hospital Association

           ARGUMENTS IN SUPPORT  :    According to the source, Hunter's Hope  
          Foundation, early detection through newborn screening is crucial  
          for those afflicted with Krabbe disease to benefit from the  
          potentially life-saving available treatments.  The sponsor  
          maintains that should California begin screening, significant  
          reduction in costs can be anticipated due to less costly care of  
          children with Krabbe disease when they are functional and do not  
          require continuous nursing care.

           ARGUMENTS IN OPPOSITION  :    The California Hospital Association  
          (CHA) writes in opposition to this bill that in California there  
          are more than 500,000 babies born in hospitals each year.  For  
          each of those births, hospitals administer a newborn screening  
          for more than 75 genetic diseases and congenital disorders that  
          are preventable or remediable by early intervention testing.   
          CHA maintains that mandating new screenings will impose new  
          costs on the newborns parents, the program, the payers  
          (including Medi-Cal) and the providers.  CHA argues that any  
          screening expansion must be evidence-based and implemented only  
          once standard protocols have been tested and it is established  

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          that actual benefits, in terms of improved patient outcomes,  
          will result.  Currently, CHA asserts, there are insufficient  
          clinical research and evidence-based reviews of these rare  
          conditions to provide an opinion on the extent to which  
          lysosomal storage disorders meet the available and efficacy of  
          treatment thresholds.


          JL:ej  5/28/13   Senate Floor Analyses 

                           SUPPORT/OPPOSITION:  SEE ABOVE

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