BILL ANALYSIS Ó
SENATE HEALTH
COMMITTEE ANALYSIS
Senator Ed Hernandez, O.D., Chair
BILL NO: AB 395
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AUTHOR: Pan
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AMENDED: May 27, 2011
HEARING DATE: July 6, 2011
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CONSULTANT:
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Trueworthy
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SUBJECT
Newborn screening program
SUMMARY
Requires the California Department of Public Health (DPH)
to expand statewide screening of newborns to include
screening for severe combined immunodeficiency (SCID) and
other T-cell lymphopenias detectable as a result of SCID.
CHANGES TO EXISTING LAW
Existing law:
Declares the intent of the Legislature that the state's
hereditary disorders program activities are to be fully
supported by fees collected for services provided by the
program, unless otherwise provided.
Requires DPH to establish a genetic disease unit to
coordinate programs in the area of genetic disease and
evaluate and prepare recommendations on the implementation
of tests for the detection of certain hereditary and
congenital diseases.
Requires DPH to charge a fee for newborn screening and
follow-up services, and requires the amount of the fee to
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be established pursuant to regulation and periodically
adjusted.
Requires that any fee charged for screening and follow-up
services provided to Medi-Cal eligible persons, health care
service plan enrollees, or persons covered by disability
insurance policies are to be paid directly to the Genetic
Disease Testing Fund, and are subject to the terms and
conditions of the health care service plan or insurance
coverage.
This bill:
Requires DPH to expand statewide screening of newborns to
include screening for severe SCID and other T-cell
lymphopenias detectable as a result of SCID.
FISCAL IMPACT
According to the Assembly Appropriations committee
analysis, AB 395 has the following potential costs:
1. Annual estimated screening costs to the DPH Newborn
Screening (NBS) Program statewide of $5 million
(special fund).
2. Annual estimated screening costs to the Medi-Cal
program of $2.2 million (50 percent General Fund
(GF)). Medi-Cal pays for approximately 45 percent of
the births in the state, and reimburses the NBS
program for screening costs associated with Medi-Cal
births.
3. Annual treatment costs to the Medi-Cal program for
babies diagnosed with SCID are estimated to be in the
range of $250,000 annually (50 percent GF).
4. Annual treatment cost savings to the Medi-Cal
program from early detection of SCID, potentially in
the range of $1-2 million annually (50 percent GF).
In the absence of early detection, treatment costs for
newborns in the Medi-Cal program with SCID annually
are highly variable and can be significant, depending
on the time of diagnosis, whether they are diagnosed
before succumbing to lethal opportunistic infections,
and whether and when treatment is attempted.
BACKGROUND AND DISCUSSION
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According to the author, AB 395 expands the California NBS
Program to adopt SCID as the 30th "core" disorder screened
for in the program. The author argues that this bill is
needed to implement the recommendations of the federal
Health and Human Services (HHS) Secretary's Advisory
Committee on Heritable Disorders in Newborns and Children
(SACHDNC) and bring the NBS Program into alignment with the
most up-to-date research, technology, laboratory, and
public health standards and practices. The author states
that SCID is the most serious primary immunodeficiency
disorder and leads to extreme susceptibility to serious
illness. The author argues that unless these defects are
corrected early on in the child's life the child will die
of opportunistic infections before their first or second
birthday.
SCID
The defining characteristic of SCID is the absence of
T-cells and, as a result, the lack of B-cell functions.
B-cells are specialized white blood cells made in the bone
marrow that fight infection. These genetic defects lead to
extreme susceptibility to serious illness. Unless these
defects are corrected, children are vulnerable to
opportunistic infections and will likely die before their
first or second birthday. In the past, children with this
disorder were kept in strict isolation, sometime in a
plastic isolator or "bubble." Now treatments are available
to significantly enhance the health outcomes of infants
with SCID who are presyptomatic or early symptomatic.
Based on national studies, stem cells from either umbilical
cord blood or bone marrow appears to be effective in
significantly decreasing the morbidity and mortality for
children with a type of SCID caused by a mutated gene on
the X chromosome. According to the genetic disease
screening program, if the treatment is provided within 3.5
months of life, the long-term survival rate is 95 percent
and after 3.5 months it is 60 to 70 percent. When left
untreated, children rarely survive past the age of two.
Pilot project
Beginning in August of 2010, DPH has been participating in
a pilot project to test for SCID sponsored by the Jeffrey
Modell Foundation and the National Institutes of Health.
According to the March of Dimes, all babies born in
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California since the pilot began have been screened. Out
of 235,686 babies screened, seven have been identified as
SCID babies. The pilot has also lead to the identification
of four other babies with T-cell lymphopenia. Current data
as a result of the pilot has shown the rate of SCID to be 1
in 35,000 in California while it was previously estimated
to be 1 in 100,000.
Methodology for adding new conditions
According to DPH, there is no national standard process to
add a disorder to a state's screening program. Some states
will add a disorder once it is recommended by SACHDNC but
prior to official acceptance by the HHS Secretary, while
other states wait for the official acceptance by the HHS
Secretary. In California disorders have been added through
the budget process or through legislation directing the
department to add a specific disorder to the screening
program.
In May 2010, Secretary Sebelius of the HHS adopted the
SACHDNC recommendation of the Uniform Screening Panel to
adopt the recommendation to add SCID as a core condition
and the related T-cell lymphocyte deficiencies to the list
of secondary targets.
Prior legislation
SB 142 (Alpert), Chapter 687, Statutes of 2004, extends the
date by which DPH would be required to obtain screening
from laboratories by competitive bid from July 1, 2005 to
August 1, 2005.
SB 1103 (Senate Budget and Fiscal Review), Chapter 228,
Statutes of 2004, expands statewide screening of newborns
to include mass spectrometry screening (TMS) screening for
fatty acid oxidation, amino acid, organic acid disorders,
and congenital adrenal hyperplasia and provides that if DPH
is unable to provide this screening by July 1, 2005,
requires screening for these disorders to be obtained from
one or more laboratories. Provides specified flexibility
to amend contracts for implementation of the TMS screening.
AB 442 (Committee on Budget), Chapter 1161, Statutes of
2002, requires hospitals to collect fees associated with
any tests conducted under the state's NBS Program, and
makes an outreach and community awareness process for this
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program voluntary, versus mandatory.
AB 2427 (Kuehl), Chapter 803, Statutes of 2000, makes
various changes to existing law relating to the genetic
disease testing program, as specified, and states
legislative intent that unless otherwise specified, the
program carried out is to be fully supported from fees
collected for services provided by the program.
SB 148 (Alpert), Chapter 541, Statutes of 1999, requires
health care service plans and specified disability insurers
to provide coverage for the testing and treatment of
phenylketonuria (PKU), a genetic disorder.
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, and would require
the department to charge a fee not to exceed the costs of
these additional screenings, that would be deposited into
the Genetic Disease Testing Fund.
Arguments in support
The March of Dimes, the sponsor of the bill, writes that,
any disease affecting newborns that can be detected and
treated should be, and it is critical that SCID be added to
the California NBS Program. The March of Dimes argues that
the health benefits of screening newborns for SCID are
apparent, but screening would also likely lead to economic
relief in California as well. The Children's Advocacy
Institute writes that AB 395 would make possible the
identification of SCID shortly after birth, allowing for
timely, cost-effective treatment and could save the lives
of children who are born with this condition.
The American Academy of Pediatrics writes that early
screening for SCID is critically important as data shows
that SCID infants who receive a related donor bone marrow
transplant within the first 14 weeks of life are
significantly more likely to survive and have fewer
problems over time than those who receive transplants later
in infancy or who have already developed an infection.
PRIOR ACTIONS
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Assembly Health: 15- 1
Assembly Appropriations:12- 5
Assembly Floor: 71- 4
POSITIONS
Support: March of Dimes (sponsor)
American Academy of Pediatrics, California
California Medical Association
Children's Advocacy Institute
Talecris Biotherapeutics
The American College of Obstetricians and
Gynecologists, California
Oppose: None on file.
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