BILL ANALYSIS �
SENATE COMMITTEE ON HEALTH
Senator Ed Hernandez, O.D., Chair
BILL NO: AB 1559
AUTHOR: Pan
AMENDED: May 23, 2014
HEARING DATE: June 18, 2014
CONSULTANT: Diaz
SUBJECT : Newborn screening program.
SUMMARY : Requires the Department of Public Health to expand
statewide screening of newborns to include screening for
adrenoleukodystrophy as soon as possible.
Existing law:
1.Requires the Department of Public Health (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
Continued---
AB 1559 | Page 2
hyperplasia. Also requires screening of newborns to include
screening for severe combined immunodeficiency, as soon as
possible.
This bill: Requires DPH to add screening for
adrenoleukodystrophy (ALD) to the CNSP as soon as possible.
FISCAL EFFECT : According to the Assembly Appropriations
Committee:
1.Annual estimated screening costs to the CNSP 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 percent General Fund (GF), remainder federal funds).
Medi-Cal pays for approximately 50 percent 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.
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.
PRIOR VOTES :
Assembly Health: 19 - 0
Assembly Appropriations:17 - 0
Assembly Floor: 79 - 0
AB 1559 | Page
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COMMENTS :
1.Author's statement. According to the author, ALD is a rare
X-chromosome genetic disorder that is passed down from mother
to son. The worst form affects mostly 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. 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.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 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.
3.CNSP. According to DPH's 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
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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.
4.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 support and maintain the
screening expansion. CNSP statute does not currently require
screening for any disorders that are not on the RUSP.
According to its Charter, the purpose of the DACHDNC is to
advise the HHS Secretary about aspects of newborn and
childhood screening and technical information, which includes
development of policies and priorities. The DACHDNC is
responsible for developing a model decision-matrix for newborn
screening expansion to evaluate the potential public health
impact of such expansion, as well as implementing, monitoring,
and evaluating newborn screening activities that include
diagnosis, screening, follow-up, treatment activities, and
technology used for screening.
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
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rejected adding a screening to the RUSP even after the review
committee gave its approval.
5.Related legislation. SB 1334 (Walters) would have required
DPH, until January 1, 2020, to expand the screening of
newborns in Orange County to include Krabbe disease. This bill
died without being heard in the Senate Health Committee.
6.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.
AB 442 (Committee on Budget), Chapter 1161, Statutes of 2002,
required hospitals to collect fees associated with any tests
conducted under the CNSP.
SB 537 (Greene), Chapter 1011, Statutes of 1998, required 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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7.Support. The sponsor and 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.
8.Should California make an exception? As noted by the author,
ALD is a rare disease that, if not detected early, leads to
death once the disease is diagnosed correctly within as little
as a few months to up to a few years. However, according to
DPH, California's practice has been to wait for final approval
from HHS's Secretary before adding screening for new diseases.
Screening for ALD is still being considered by the review
committee. Though this bill requires DPH to add ALD screening
"as soon as possible," which may allow DPH to wait for final
approval from the HHS Secretary, the author may wish to
consider whether it is good public policy for the state to
break from standard practice and to add newborn screenings
before the review committee completes its process and the HHS
Secretary gives final approval.
SUPPORT AND OPPOSITION :
Support: The Myelin Project (sponsor)
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
Stop ALD Foundation
University of Minnesota, Pediatric Blood and Marrow
Transplantation
Numerous Individuals
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Oppose: None received.
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