BILL ANALYSIS Ó ----------------------------------------------------------------- |SENATE RULES COMMITTEE | AB 1559| |Office of Senate Floor Analyses | | |1020 N Street, Suite 524 | | |(916) 651-1520 Fax: (916) | | |327-4478 | | ----------------------------------------------------------------- THIRD READING Bill No: AB 1559 Author: Pan (D), et al. Amended: 8/19/14 in Senate Vote: 21 SENATE HEALTH COMMITTEE : 8-0, 6/18/14 AYES: Hernandez, Morrell, Beall, DeSaulnier, Evans, Monning, Nielsen, Wolk NO VOTE RECORDED: De León SENATE APPROPRIATIONS COMMITTEE : 5-0, 8/14/14 AYES: De León, Hill, Lara, Padilla, Steinberg NO VOTE RECORDED: Walters, Gaines ASSEMBLY FLOOR : 79-0, 5/28/14 - See last page for vote SUBJECT : Newborn screening program SOURCE : The Myelin Project DIGEST : This bill, until January 1, 2018, requires the Department of Public Health (DPH) to expand statewide screening of newborns to include screening for adrenoleukodystrophy (ALD) as soon as possible. ANALYSIS : Existing law: 1.Requires DPH to establish a genetic disease unit to coordinate all DPH programs in the area of genetic disease that promotes a statewide program of information, testing, and counseling CONTINUED AB 1559 Page 2 services, and designates tests and regulations to be used in executing this program, and designates tests and regulations 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. Also requires screening of newborns to include screening for severe combined immunodeficiency, as soon as possible. This bill, until January 1, 2018, requires DPH to add screening for ALD to the CNSP as soon as possible. Background Adrenoleukodystrophy . ALD can cause injury to the brain, nerves, and adrenal glands. The first signs of ALD are behavioral and rapidly lead to a vegetative state and ultimately death. The childhood form is the most severe and affects boys between the ages of four and eight years old. The slightly milder adult version affects men in their 20s and 30s. Unless treated before symptoms show, children affected with ALD will die within a few months to a few years. Early detection and treatment provides dramatically better quality of life for the CONTINUED AB 1559 Page 3 affected individuals and their families. Cord blood and bone marrow transplants performed at a very early stage in the disease have proven to treat and heal the patient, enabling a healthy and long life. An August 26, 2000, article, "Long-term effect of bone-marrow transplantation for childhood-onset cerebral X-linked adrenoleukodystrophy," in the Lancet medical journal found the long-term beneficial effect of bone marrow transplantation when the procedure is done at an early stage of the disease. CNSP . According to DPH's Internet Web site, newborn screening is recognized nationally as an essential preventive health measure. All states in the nation and the District of Columbia have established newborn screening programs. Disorders screened for by the program have varying degrees of severity. If identified early, many conditions can be treated before they cause serious health problems. Prior to leaving the hospital, a few drops of blood from the newborn's heel are collected on filter paper. Parents can get the test results from the baby's doctor or clinic, which take about two weeks. If the baby needs more tests, parents will 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 clinics. Currently, the cost of the test is $111.70. Medi-Cal, health plans, and most private insurance will pay for the test. Adding new screenings . According to DPH, California, like most states, follows recommendations from the federal Health and Human Services' (HHS) Discretionary Advisory Committee on Heritable Disorders in Newborns and Children (DACHDNC) and waits for the final approval from the HHS Secretary before requiring a new screening. Because disorders are not officially part of the Recommended Uniform Screening Panel (RUSP) until they are approved by the Secretary, states do not generally implement screenings before the final approval. While DPH's Director has the authority to add new disorders administratively, in the past new disorders have been added after final federal approval through legislation in conjunction with a fee increase to CONTINUED AB 1559 Page 4 support and maintain the screening expansion. CNSP statute does not currently require screening for any disorders that are not on the RUSP. In January 2014, the DACHDNC voted to move ALD newborn screening to an external evidence review committee. Approval from the review committee is expected in early 2015, after which the HHS Secretary would need to give final approval. There have been instances in the past where the HHS Secretary rejected adding a screening to the RUSP even after the review committee gave its approval. Prior Legislation 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. This bill was held in the Assembly Appropriations Committee. 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. This bill was held in the Senate Appropriations Committee. SB 1731 (Block, Chapter 336, Statutes of 2012) established the Newborn Critical Congenital Heart Disease (CCHD) Screening Program and required hospitals, beginning July 1, 2013, to offer a pulse oximetry test for the identification of CCHD to parents of newborns prior to discharge. AB 395 (Pan, Chapter 461, Statutes of 2011) expanded statewide screening of newborns to include screening for severe combined immunodeficiency. SB 1103 (Committee on Budget and Fiscal Review) Chapter 228, Statutes of 2004, expanded statewide screening of newborns to include tandem mass spectrometry screening for fatty acid oxidation, amino acid, organic acid disorders, and congenital adrenal hyperplasia. FISCAL EFFECT : Appropriation: Yes Fiscal Com.: Yes Local: No According to the Senate Appropriations Committee: CONTINUED AB 1559 Page 5 One-time costs of about $2 million for technical and administrative activities to add ALD to the newborn screening program (Genetic Disease Testing Fund). Annual screening costs of $4.7 million per year, covered by fees (Genetic Disease Testing Fund). Adding ALD to the newborn screening program requires DPH to raise the fee charged for screening by $11 per birth. Increased costs or about $2.6 million per year to the Medi-Cal program to pay the fees charged to support the newborn screening program for births paid for by Medi-Cal (General Fund and federal funds). Increased costs of about $50,000 per year to CalPERS to pay the fees charged to support the newborn screening program for births paid for by CalPERS (various funds). Likely long-term savings due to improved clinical outcomes from early testing and treatment (various funds). According to information provided by physicians treating children with ALD, clinical outcomes can be greatly improved by early diagnosis and treatment. Without screening for ALD, a diagnosis often is not made until serious symptoms have occurred. At that point, treatment is much less effective and the child is at high risk for death or permanent disability. On the other hand, if treatment is provided at the first sign that ALD is occurring (through an MRI diagnosis, before clinical symptoms are observed) long-term outcomes are greatly improved. SUPPORT : (Verified 8/19/14) The Myelin Project (source) Adrenoleukodystrophy Foundation Be A Hero Become A Donor Foundation Brian's Hope California Professional Firefighters Croda International Fight ALD - Fighting Illness through Education International Pemphigus & Pemphigoid Foundation Moser Center for Leukodystrophies at Kennedy Krieger Institute at Johns Hopkins Medical School CONTINUED AB 1559 Page 6 Stop ALD Foundation University of Minnesota, Pediatric Blood and Marrow Transplantation AFSCME, AFL-CIO California Academy of Physician Assistants California Healthcare Institute Children's Hospital and Research Center Oakland Corpus Christi Parish Duke University Pediatric Blood and Marrow Transplant Program and Carolinas Cord Blood Bank OPPOSITION : (Verified 8/19/14) Department of Finance ARGUMENTS IN SUPPORT : Supporters argue that early detection of ALD is crucial and helps avoid undue suffering, as well as emotional and financial costs associated with treatment and care of the full onset of ALD. They further argue that newborn screening for ALD is simple, accurate, and cost-effective, using blood spots already used for screening of other diseases, and that treatment is most effective if begun before symptoms first appear. ASSEMBLY FLOOR : 79-0, 5/28/14 AYES: Achadjian, Alejo, Allen, Ammiano, Bigelow, Bloom, Bocanegra, Bonilla, Bonta, Bradford, Brown, Buchanan, Ian Calderon, Campos, Chau, Chávez, Chesbro, Conway, Cooley, Dababneh, Dahle, Daly, Dickinson, Donnelly, Eggman, Fong, Fox, Frazier, Beth Gaines, Garcia, Gatto, Gomez, Gonzalez, Gordon, Gorell, Gray, Grove, Hagman, Hall, Harkey, Roger Hernández, Holden, Jones, Jones-Sawyer, Levine, Linder, Logue, Lowenthal, Maienschein, Mansoor, Medina, Melendez, Mullin, Muratsuchi, Nazarian, Nestande, Olsen, Pan, Patterson, Perea, John A. Pérez, V. Manuel Pérez, Quirk, Quirk-Silva, Rendon, Ridley-Thomas, Rodriguez, Salas, Skinner, Stone, Ting, Wagner, Waldron, Weber, Wieckowski, Wilk, Williams, Yamada, Atkins NO VOTE RECORDED: Vacancy JL:nl 8/19/14 Senate Floor Analyses CONTINUED AB 1559 Page 7 SUPPORT/OPPOSITION: SEE ABOVE **** END **** CONTINUED