BILL ANALYSIS �
AB 1559
Page 1
Date of Hearing: April 1, 2014
ASSEMBLY COMMITTEE ON HEALTH
Richard Pan, Chair
AB 1559 (Pan) - As Amended: March 19, 2014
SUBJECT : Newborn screening program.
SUMMARY : Requires the Department of Public Health (DPH) to
expand statewide screening of newborns to include screening for
adrenoleukodystrophy (ALD).
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. Also requires screening of newborns to include
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screening for severe combined immunodeficiency, as soon as
possible.
FISCAL EFFECT : This bill has not been analyzed by a fiscal
committee.
COMMENTS :
1)PURPOSE OF THIS BILL . According to the author ALD, is an
X-chromosome genetic disorder that is passed down from mother
to son. The worst form affects young boys. Once symptoms
show, it progresses quickly and it is usually too late to do
anything meaningful to save the boy. Correct diagnosis is
difficult due to the rarity of the disease and the nature of
the early symptoms - which are behavioral and are often
misdiagnosed as Attention Deficit Hyperactivity Disorder, low
IQ, depression, and even Multiple Sclerosis in the adult form.
The author further states that, our knowledge about and
ability to combat the disease has increased greatly in the
last couple decades, including treatments that can cure
patients if caught early enough and this makes it imperative,
medically and morally, that we catch the disease as early as
humanly possible - which makes ALD the perfect candidate for
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 added through regulation and legislation.
Currently only New York and Connecticut screen newborns for ALD.
However, on January 16, 2014, the federal Discretionary
Advisory Committee on Heritable Disorders in Newborns and
Children, which 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, approved
ALD for review to add the disease as a core condition for
recommended screening of all newborns in the U.S. The review
process should take approximately two years before a final
decision is made.
DPH is currently evaluating the addition of ALD to the newborn
testing panel and expects to have cost estimates in the Spring
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of 2014. Those estimates will include initial and
confirmatory testing, provider and patient education,
modifications to the Screening Information System computer
system, changes to the test results, and follow-up on
presumptive positives. The testing method developed for ALD
uses tandem mass spectrometry which California already employs
to test for multiple different metabolic disorders. According
to DPH, their testing vendor is developing a test for ALD
using the current testing equipment.
According to the California Department of Health Care
Services, between 2004 and 2013, the cost of treating 22
children with the disease in the California Children's
Services program was slightly more than $100,000 per child,
during that 9 year period.
3)ALD . ALD is a rare X-chromosome genetic disorder (which
affects mostly boys) that can cause injury to the brain,
nerves, and adrenal glands. People with ALD do not generate
enough of the protein that breaks down very long chain fatty
acids (VLCFA). The myelin sheath, which acts as insulation
around the nerves, is made up of these fatty acids; buildup of
the VLCFA prevents the brain from communicating with the body.
The first signs of ALD are behavioral and it is often
misdiagnosed as attention deficit disorder, but it rapidly
leads 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 20's and 30's. 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
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. The article "Long-term effect of
bone-marrow transplantation for childhood-onset cerebral
X-linked adrenoleukodystrophy," in medical journal the Lancet,
August 26, 2000; Volume 356, found the long-term beneficial
effect of bone marrow transplantation when the procedure is
done at an early stage of the disease.
4)SUPPORT . The sponsor of the bill, The Myelin Project writes
that an accurate test for ALD has recently been developed and
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proven to work. However, the treatment, bone marrow or cord
blood transplants, can only work before symptoms show, usually
around age five. This makes newborn screening of ALD
imperative.
Stanford University Medical Center, Department of Neurology and
Neurological Sciences writes in support of this bill that
newborn screening for ALD will allow physicians and affected
families to apply proven surveillance strategies that allow
timely implementation of life saving treatments that reduce
disability, save lives, and lower healthcare costs.
Children's Hospital Los Angeles supports this bill and states
that newborn screening for ALD will allow early identification
of children with the disease, allow appropriate care and
screening in the asymptomatic phase, and allow for close
monitoring and rapid progression to bone marrow transplant
once indicated.
5)RELATED LEGISLATION . SB 1334 (Walters) would require DPH,
until January 1, 2020, to expand the screening of newborns in
Orange County to include screening for Krabbe disease. SB
1334 is currently pending in the Senate Health Committee.
6)PREVIOUS LEGISLATION .
a) 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.
b) 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.
c) 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.
d) AB 395 (Pan), Chapter 461, Statutes of 2011, expands
statewide screening of newborns to include screening for
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severe combined immunodeficiency.
e) 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.
f) AB 442 (Committee on Budget), Chapter 1161, Statutes of
2002, requires hospitals to collect fees associated with
any tests conducted under CNSP.
g) 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.
7)SCREENING LEGISLATION . According to the World Health
Organization, screening, in medicine, is a strategy used in a
population to identify an unrecognized disease in individuals
who may not have signs or symptoms. As such, screening tests
are unique in that they are performed on persons apparently in
good health. As a matter of public policy, when contemplating
mandating a specific type of screening, the Legislature should
consider several things. Is the proposed screening test
effective in accurately identifying a disease? What are the
consequences of a false positive result? Is there an
effective treatment for the disease? What is the cost of the
screening? Is there access to treatment for California's
diverse population? The Legislature should also look to
information from other organizations, such as the U.S.
Preventative Task Force and the HHS Secretary's Advisory
Committee on Heritable Disorders in Newborns and Children.
These organizations are expert bodies that evaluate available
screening processes to ensure that there is evidence that the
proposed screen is effective for widespread use. It is not
necessarily good public policy to mandate a screening that has
not been recommended for widespread use.
8)POLICY COMMENT . ALD appears to meet the criteria necessary
for inclusion in CNSP. There is a cost-effective screening
test with a low false-positive rate, and when identified early
there is an effective treatment.
REGISTERED SUPPORT / OPPOSITION :
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Support
The Myelin Project (sponsor)
Adrenoleukodystrophy Foundation
Be A Hero Become A Donor Foundation
Brian's Hope
California Healthcare Institute
California Professional Firefighters
Children's Hospital Los Angeles
Corpus Christi Parish
Croda International Pic
Harvard Medical School
International Pemphigus & Pemphigoid Foundation
Lucile Packard Children's Hospital at Stanford
Massachusetts General Hospital
The Stop ALD Foundation
University of California, San Francisco
University of Minnesota Medical Center
Numerous individuals
Opposition
None on file.
Analysis Prepared by : Lara Flynn / HEALTH / (916) 319-2097