BILL ANALYSIS Ó
SB 1095
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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
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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
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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 $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.
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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
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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
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
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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) Metabolic: chemical reactions in the body to create
energy and build tissue;
ii) Endocrine: hormones that affect body functions;
iii) Hemoglobin: red blood cells that carry oxygen;
iv) Other genetic diseases;
v) Cystic Fibrosis; and,
vi) 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
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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 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 life-saving 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
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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
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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
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.
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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
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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
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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
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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
Lysosomal Disease Network
March of Dimes
MPS 6CESS Foundation
Muscular Dystrophy Association
National Leiomyosarcoma Foundation
National Lymphedema Network
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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
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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
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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.
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Analysis Prepared by:Paula Villescaz / HEALTH / (916)
319-2097