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SB 1095 Page 1 Date of Hearing: June 21, 2016 ASSEMBLY COMMITTEE ON HEALTH Jim Wood, Chair SB 1095 (Pan) – As Amended May 31, 2016 SENATE VOTE: 39-0 SUBJECT: Newborn screening program. SUMMARY: Requires the Department of Public Health (DPH) to expand statewide screening of newborns to include screening for any disease that is detectable in blood samples as soon as the disease is adopted by the federal Recommended Uniform Screening Panel (RUSP). EXISTING LAW: 1) Requires DPH to establish a genetic disease unit to coordinate all DPH programs in the area of genetic disease that will promote a statewide program of information, testing, and counseling services and to have the responsibility of designating tests and regulations to be used in executing this program and to have the responsibility of designating tests and regulations to be used in executing the California Newborn Screening Program (CNSP). 2) Requires DPH to provide genetic screening and follow-up services. Allows DPH to provide laboratory (lab) testing facilities or work with qualified outside labs to conduct testing. 3) Requires DPH to charge a fee for newborn screening and follow-up services, and requires the amount of the fee to be periodically adjusted in order to meet the costs of CNSP. 4) Requires DPH to evaluate and prepare recommendations on the implementation of tests for the detection of hereditary and congenital diseases, including, but not limited to, biotinidase deficiency and cystic fibrosis. Requires DPH to also evaluate and prepare recommendations on the availability and effectiveness of preventative follow-up interventions, including the use of specialized medically necessary dietary products. 5) Requires statewide screening of newborns to include tandem mass spectrometry screening for fatty acid oxidation, amino acid, and organic acid disorders and congenital adrenal hyperplasia. Requires screening of newborns to include screening for severe combined immunodeficiency, as soon as possible. 6) Requires DPH to expand statewide screening of newborns to include screening for adrenoleukodystrophy (ALD) as soon as ALD is adopted by the federal RUSP. FISCAL EFFECT: According to the Senate Appropriations Committee: 1) One-time costs of $2.4 million and ongoing costs of $4.3 million per year to screen for two diseases (MPS-1 and Pompe disease) that have already been approved for inclusion in the federal RUSP (Genetic Disease Testing Fund). The ongoing costs above would cover initial screening tests, follow up tests for positive results, and initial case management for confirmed diagnoses. Adding these two conditions would require an increase in the existing SB 1095 Page 2 $113 fee charged for screening by about $9. Most health insurance, including Medi-Cal, cover the costs of the screening fee. 2) Ongoing costs of about $2.5 million per year for coverage of the increased screening fee by the Medi-Cal program (General Fund and federal funds). Medi-Cal covers the cost of the screening exam and Medi-Cal pays for about 50% of the births in the state. 3) Unknown future costs to include additional diseases in the state’s newborn screening program as they are added to the federal RUSP (Genetic Disease Testing Fund). The costs to include additional diseases will vary depending on the specific costs for testing of that disease. In recent years, anticipated costs to include additional diseases in the state’s newborn screening program have generally been in the low millions per condition, per year. 4) Likely long-term savings due to improved clinical outcomes from early testing and treatment (various funds). When considering whether to include additional diseases in the RUSP, the federal advisory committee considers issues such as the reliability of the screening test, the availability of treatments, the benefits of early diagnosis, and the anticipated impact on health outcomes from additional screening. Specific information about the long-term impacts on health outcomes or avoided health care costs are often not available, because the diseases that are considered for inclusion are rare and treatments are evolving rapidly. However, new diseases are added when the advisory committee finds that there are benefits from screening, primarily the ability to avoid long-term health consequences with early intervention. Therefore, it is reasonable to believe that including additional diseases that have been approved by the federal government will reduce state health care spending in the long-run. COMMENTS: 1) PURPOSE OF THIS BILL. According to the author, newborn screening for rare diseases is a critical, cost-effective public health intervention, as diagnosing diseases at birth leads to earlier medical intervention. In turn, early treatment saves and improves children’s and families’ lives and reduces medical costs by eliminating or ameliorating the devastating consequences of rare and treatable diseases. California’s current approach to reviewing and approving screening for additional diseases has created a lag behind current medical recommendations. A study in the journal Pediatrics found that California saves $9.32 in health care costs for every dollar spent on newborn screening. 2) BACKGROUND. In 1966 California began its CNSP with the testing of phenylketonuria. Since its creation, the CNSP has been expanded several times as new discoveries are made and tests developed and now screens for more than 70 disorders. Diseases have been continually added through regulation and legislation. The last disease added by statute was ALD in 2014. ALD was added to the RUSP in February of 2016. a) RUSP. The Advisory Committee on Heritable Disorders in Newborns and Children (HDN Committee) is established by federal law for the purpose of making recommendations and changes to the RUSP. The mission of the HDN Committee is to reduce morbidity and mortality in newborns and children who have, or are at risk for, heritable disorders. The HDN Committee advises the Secretary of the U.S. Department of Health and Human Services (HHS) on the most appropriate application of universal newborn screening tests, technologies, policies, guidelines, and standards. The HDN SB 1095 Page 3 Committee recommends that every newborn screening program include a Uniform Screening Panel that screens for 32 core disorders and 26 secondary disorders; the disorders' selection was determined using available scientific evidence, availability of a screening test, presence of an efficacious treatment, adequate understanding of the natural history of the condition, and whether the condition was either part of the differential diagnosis of another condition or whether the screening test results related to a clinically significant condition. b) CNSP. Prior to leaving the hospital, a few drops of blood from the newborn’s heel are collected on filter paper. The newborn screening test should be done when the baby is at least 12 hours of age but before six days of age. The ideal time to do the test is when the baby is between 24 and 48 hours of age. Blood collected before 12 hours of age is not always reliable for some metabolic diseases. The sample is sent to one of eight regional labs that contract with the DPH for testing. The results are sent to DPH for data collection and quality control. Parents obtain the test results from the baby’s doctor or clinic. It takes about two weeks for the doctor to receive the written results. If the baby needs more tests, parents get a letter or a phone call a few days after discharge from the hospital. 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 2009-10, approximately 520,000 newborns were screened for 75 genetic disorders. Approximately 9,200 or under 2% were classified as positive or questionable and were referred for follow-up testing or services. Disorders screened for by the CNSP have varying degrees of severity. If identified early many of these conditions can be treated before they cause serious health problems. Treatments may include medication, dietary supplements, avoidance of fasting and/or special diet and comprehensive care to reduce morbidity and mortality. The test screens for specific diseases in the following groups: i) ii) iii) iv) v) vi) Metabolic: chemical reactions in the body to create energy and build tissue; Endocrine: hormones that affect body functions; Hemoglobin: red blood cells that carry oxygen; Other genetic diseases; Cystic Fibrosis; and, Severe Combined Immunodeficiency. CNSP disorders cause delays in development, neurological damage, dehydration, incorrect sex assignment, mental retardation, and death if not treated at an early newborn age. In California about one out of every 600 babies tested will have one of these conditions or diseases. 3) SUPPORT. The California Hospital Association states that the bill provides a forward thinking approach to properly and thoughtfully expanding newborn screening by providing that California screen for diseases that are detectable in blood samples and listed on the RUSP as certified by the HHS Secretary. The RUSP list is periodically updated through a thorough, deliberative review process involving a committee of experts in newborn SB 1095 Page 4 screening. By allowing California to take advantage of the work done by these medical experts, we can remove the obstacles to needed testing and minimize the suffering that comes from untreated diseases. The EveryLife Foundation for Rare Diseases (EveryLife) states that the bill provides a much needed, practical, science-based approach to improving public health for all of California's Children. The bill allows for the earliest diagnosis of disease and access to potentially lifesaving and life-altering treatments for babies. Early treatment is vital in ensuring the best possible life-altering health outcomes for children. In many cases, early detection can avert costly and risky medical procedures later in life. A study in the journal Pediatrics found that California saves $9.32 in health care costs for every dollar spent on newborn screenings. EveryLife states that this bill will ensure that babies born in California, particularly those that will be affected by debilitating and life-threatening diseases, are diagnosed and treated at the earliest age possible and without unconscionable delay. This approach is critical for improving health outcomes for children and providing the brightest future for all babies born in California. 4) OPPOSITION. The California Right to Life, Inc. argues that this bill does not correct the issue of the limited ability to opt out of the screening program for religious beliefs or practices and may even bring discrimination against parents or guardians who do opt out. 5) OPPOSE UNLESS AMENDED. DPH states that if it is required to expand the current NBS program to include other diseases as soon as a disease is adopted by the RUSP, it will need additional time to plan and implement a quality screening program that addresses all of the issues addressed in the public health feasibility and readiness assessments provided to the Secretary of Health and Human Services as part of the approval process for new RUSP diseases. 6) RELATED LEGISLATION. AB 170 (Gatto), would require DPH to provide information about the genetic disease screening tests and obtain a signed informational acknowledgment form for the receipt of information by the parent or guardian of a newborn child regarding the storage, retention, and use of the newborn child’s blood sample for medical research. AB 170 is pending in the Senate Health Committee. 7) PREVIOUS LEGISLATION. a) AB 1559 (Pan), Chapter 565, Statutes of 2014, requires DPH to expand statewide screening of newborns to include screening for ALD. b) SB 224 (Walters) of 2013 would have required DPH, until January 1, 2019, to expand the screening of newborns in Orange County to include screening for Krabbe disease. SB 224 was held in the Assembly Appropriations Committee. c) SB 1072 (Strickland) of 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 was held in the Senate Appropriations Committee. d) SB 1731 (Block), Chapter 336, Statutes of 2012, establishes the Newborn Critical Congenital Heart Disease (CCHD) Screening Program and requires hospitals, beginning SB 1095 Page 5 July 1, 2013, to offer a pulse oximetry test for the identification of CCHD to parents of newborns prior to discharge. e) AB 395 (Pan), Chapter 461, Statutes of 2011, expands statewide screening of newborns to include screening for severe combined immunodeficiency. f) SB 1103 (Committee on Budget and Fiscal Review), Chapter 228, Statutes of 2004, expands statewide screening of newborns to include tandem mass spectrometry screening for fatty acid oxidation, amino acid, organic acid disorders, and congenital adrenal hyperplasia. g) AB 442 (Committee on Budget), Chapter 1161, Statutes of 2002, requires hospitals to collect fees associated with any tests conducted under CNSP. h) 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. REGISTERED SUPPORT / OPPOSITION: Support EveryLife Foundation for Rare Diseases (sponsor) Acid Maltase Deficiency Association (AMDA) Aidan Jack Seeger Foundation ALD Connect, Inc. American Behcet's Disease Association Amour Fund - Alpha Epsilon Omega Foundation Angels for Life Foundation Batten Disease Support & Research Association Biocom Brian’s Hope Bridge the Gap – SYNGAP Education and Research Foundation California Hospital Association California Academy of Physicians Assistants Children's PKU Network Chronic Granulomatous Disease Association Coalition Duchenne County Health Executives Association of California Cure Sanfilippo Foundation DiMedio Foundation for Children Drew’sHope Research Foundation Engage Health, Inc. Fabry Support & Information Group Friedreich’s Ataxia Research Alliance Gene Giraffe Project Gene Spotlight, Inc. Global Genes Grace Science Foundation Hannah’s Hope Fund Hope4Bridget Hunter Syndrome Foundation Immune Deficiency Foundation International Pemphigus & Pemphigoid Foundation International Society for Mannosidosis and Related Diseases International Waldenstrom's Macroglobulinemia Foundation Jeffrey Modell Foundation Jett Foundation Jonah’s Just Begun Foundation to Cure Sanfillipio LAL Solace, Inc. Leukemia Lymphoma Society Little Miss Hannah Foundation Lung Transplant Foundation Lupus and Allied Diseases Association Lymphatic Malformation Institute SB 1095 Page 6 Lysosomal Disease Network March of Dimes MPS 6CESS Foundation Muscular Dystrophy Association National Leiomyosarcoma Foundation National Lymphedema Network National Organization for Rare Disorders National PKU Alliance National Tay-Sachs & Allied Diseases Association Nicholas Conor Institute Noah’s Hope NTM Info & Research, Inc. Olivia’s Heart Project Organic Acidemia Association Oxalosis and Hyperoxaluria Foundation Parent Project Muscular Dystrophy Pediatric Hydrocephalus Foundation Phoenix Fox Foundation Pulmonary Fibrosis Advocates Rare & Undiagnosed Network Rare Disease United Foundation Reflex Sympathetic Dystrophy Syndrome Association Sanfilippo Foundation for Children Save Babies Through Screening Foundation Saving Case & Friends, a Hunter Syndrome research & advocacy foundation Stop ALD Foundation Taylor’s Tale Team Niemann-Pick Type C The Mastocystosis Society The MLD Foundation The Myelin Project The National MPS Society The Orphan Disease Pathway Project The OsteoPETrosis Society The RASopathies Network The Ryan Foundation The XLH Network, Inc. United Leukodystrophy Foundation Vanishing White Matter Disease Wired4Life Zeqing for a Cure Opposition California Right to Life Committee, Inc. Analysis Prepared by: Paula Villescaz / HEALTH / (916) 319-2097