BILL ANALYSIS �
AB 1559
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Date of Hearing: April 9, 2014
ASSEMBLY COMMITTEE ON APPROPRIATIONS
Mike Gatto, Chair
AB 1559 (Pan) - As Amended: March 19, 2014
Policy Committee: HealthVote:19-0
Urgency: No State Mandated Local Program:
No Reimbursable: No
SUMMARY
This bill requires the Department of Public Health (DPH) to
expand statewide screening of newborns to include screening for
adrenoleukodystrophy (ALD).
FISCAL EFFECT
1)Annual estimated screening costs to the DPH Newborn Screening
(NBS) Program statewide of $4.5 million (Genetic Disease
Testing Fund). This test is estimated to cost $9 per child.
2)Annual estimated costs to the Medi-Cal program of $2.3 million
(45% General Fund (GF), remainder federal funds). Medi-Cal
pays for approximately 50% of the births in the state, and
reimburses the NBS Program for screening costs associated with
Medi-Cal births.
3)Because ALD is a condition covered by the California
Children's Services (CCS) program, care costs for the majority
of children diagnosed with ALD are expected to be borne by the
state. About 15 boys per year will be diagnosed and
monitored, and of these, about 5 would likely receive bone
marrow transplants at some point during their childhood.
Extremely small numbers make it difficult to predict any state
cost impacts with certainty. However, given that boys who
manifest severe symptoms are likely to be covered by CCS, the
fiscal question is the costs of treating these boys earlier
versus later. Early identification through NBS allows for
monitoring and treatment when the disease has barely
progressed. Later identification does not impose monitoring
costs, but imposes greater treatment costs, as the disease has
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progressed further and medical risks are greater.
Furthermore, children treated after symptoms manifest are
likely to be experience permanent physical and cognitive
disability, which may make impose costs for other state
services, such as services provided through regional centers
or long-term care facilities.
It appears likely that with the imposition of the screening
test, monitoring of ALD-diagnosed children who otherwise would
not have been monitored will increase costs by a small amount,
likely less than $100,000 (GF/ federal) annually. Treatment
costs in CCS associated with ALD would likely be similar to or
somewhat less than what CCS pays currently, with treatment
being provided to slightly more children, but with lower
costs, fewer risks, and better outcomes.
To the extent successful treatment avoids permanent disability
and reduces the number of children receiving services the
state would otherwise pay for, such as regional center or
long-term care services, unknown but potentially significant
annual GF cost savings are possible.
COMMENTS
1)Purpose . According to the author, the purpose of this bill is
to add ALD to the NBS Program. The author contends that
although a federal advisory panel has not yet recommended all
states add ALD to their NBS panels, data from an ongoing pilot
study appears to support newborn screening as a mechanism for
early identification of ALD. ALD is a rare disease that causes
damage to the myelin sheath, an insulating membrane that
surrounds nerve cells in the brain. The most severe symptoms
primarily affect males, and prevalence is estimated at 1 in
17,000 males.
2)Newborn Screening . All states have newborn screening programs
for congenital diseases that benefit from early detection and
treatment. California began its NBS Program in 1966 with
testing for phenylketonuria (PKU). Since then, numerous
conditions have been added to the programs as advancements in
medical science have produced screening and treatment options
for more diseases. Screening tests have been added through
the budget process as well as through legislation directing
the department to add a specific disorder. DPH is also
authorized to add conditions to the panel.
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3)Recommended Uniform Screening Panel (RUSP). The RUSP is a list
of conditions every state is advised to include in their NBS
programs, including core conditions and secondary conditions.
The list is generated by the Discretionary Advisory Committee
on Heritable Disorders in Newborns and Children, a federal
expert advisory body that evaluates and makes recommendations
on the application of universal genetic screening tests. This
federal committee conducts comprehensive evidence-based
assessments of conditions nominated to the RUSP. For each
condition nominated, the committee reviews the available
evidence and data regarding the benefits and harms of
screening for the nominated condition. The committee makes a
determination about the addition of the condition to the RUSP
on the basis of the magnitude and certainty of net benefit to
the population of screened newborns that could result from
testing and treatment.
California currently screens for all of 31 core conditions on
the RUSP. Legislation in both 2011 and 2012 has added
conditions that are on the RUSP (see Prior Legislation).
ALD has been nominated for addition to the RUSP, but the
committee has not added it, citing insufficiency of data and
evidence. However, the committee indicates ALD is "a
medically important disorder that deserves serious
consideration, possessing a well-established case definition
as well as screening, diagnostic, and treatment protocols."
The committee indicates it wishes to conduct an expedited
review once a large screening trial is complete, meaning the
disease may be added to the RUSP in the coming years if the
committee finds the results of the trial support the addition
of ALD.
4)Prior Legislation . AB 1731 (Block), Chapter 336, Statutes of
2012 required birthing hospitals to develop a screening
program for critical congenital heart disease (CCHD) and to
offer parents of a newborn a test for CCHD prior to the
newborn's discharge.
AB 395 (Pan), Chapter 461, Statutes of 2011 required DPH to
expand screening of newborns to include screening for severe
combined immunodeficiency (SCID) and, insofar as it does not
require additional costs, other T-cell lymphopenias detectable
as a result of screening for SCID.
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Analysis Prepared by : Lisa Murawski / APPR. / (916) 319-2081