This docum ent does not reflect the intent or official position of the bill sponsor or House of Representatives.
STORAGE NAME: h1481e.HHS
DATE: 4/24/2023
HOUSE OF REPRESENTATIVES STAFF ANALYSIS
BILL #: CS/CS/CS/HB 1481 Review and Report on Sickle Cell Disease Medications, Treatments, and
Services for Medicaid Recipients
SPONSOR(S): Health & Human Services Committee, Health Care Appropriations Subcommittee, Healthcare
Regulation Subcommittee, Driskell
TIED BILLS: IDEN./SIM. BILLS:
REFERENCE ACTION ANALYST STAFF DIRECTOR or
BUDGET/POLICY CHIEF
1) Healthcare Regulation Subcommittee 16 Y, 0 N, As CS Guzzo McElroy
2) Health Care Appropriations Subcommittee 13 Y, 0 N, As CS Aderibigbe Clark
3) Health & Human Services Committee 20 Y, 0 N, As CS Guzzo Calamas
SUMMARY ANALYSIS
Sickle cell disease (SCD) is an inherited disease caused by abnormal hemoglobin in red blood cells, which causes
the red blood cells to become hard and sticky making them prone to clogging the flow of blood. This can lead to
episodes of pain or other health problems such as strokes, organ failure, eye problems, and infections. It affects
100,000 people nationwide, 90 percent of which are people of African descent.
In 2022, the General Appropriations Act (GAA) included nonrecurring funds for the Agency for Health Care
Administration (AHCA) to conduct a review and publish a report of its findings on the availability and utilization of
specific services for Medicaid enrollees diagnosed with SCD.
The report, which was published on February 1, 2023, used information from 2018 through 2021 on Medicaid
enrollees with SCD to produce the following results:
On average, 7,328 Medicaid enrollees have SCD, which is twice as high as the national average;
Enrollees are predominantly female (58%), young (median age 18), and black (63%);
85 percent were evaluated or treated in an outpatient clinic setting; 61 percent were evaluated in an
emergency room; and 52 percent were admitted for inpatient care in a hospital;
Stroke screening for children with transcranial doppler ultrasound was only performed at half the rate
recommended by national guidelines; and
72 clinical treatment programs contract with managed care plans to provide care to individuals with SCD.
CS/CS/HB 1481 requires AHCA to biannually review and report the same information it was required to report by the
Fiscal Year 2022-2023 GAA. Additionally, it requires AHCA to assess their existing Medicaid payment methodologies
for approved SCD treatments and medications in the inpatient setting and whether such payment methodologies
result in barriers to access. If barriers to access are identified, AHCA must assess whether the payment
methodologies may be modified or improved.
The bill requires the Department of Health (DOH) to contract with a community-based sickle cell disease medical
treatment and research center to establish and maintain a registry to track outcome measures of newborns who are
identified as carrying a sickle cell hemoglobin variant. The bill requires a screening provider to notify the primary care
physician of an infant who tests positive for sickle cell hemoglobin variant and to submit the results of the screening to
DOH for inclusion in the sickle cell registry. The primary care physician must provide information on the availability
and benefits of genetic counseling to the parent or guardian of the newborn.
The bill appropriates $1,060,804 in recurring funds and $21,355 in nonrecurring funds to DOH. The bill has no fiscal
impact on local government.
The bill provides an effective date of July 1, 2023. STORAGE NAME: h1481e.HHS PAGE: 2
DATE: 4/24/2023
FULL ANALYSIS
I. SUBSTANTIVE ANALYSIS
A. EFFECT OF PROPOSED CHANGES:
Background
Sickle Cell Disease
Sickle cell disease (SCD) is the most common inherited blood disorder in the United States—
approximately 100,000 Americans have SCD.
1
It is caused by a mutation in the hemoglobin-Beta gene
called HbS. When a child inherits HbS from only one parent, the person may have sickle cell trait,
which means they are a carrier of SCD, thus increasing the chance of having a child with SCD if they
have a child with another carrier.
2
When both parents have SCD, there is a 25 percent chance their
child will have SCD. A child from those same parents also has a 50 percent chance of inheriting sickle
cell trait and a 25 percent chance of no sickle cell inheritance.
3
Hemoglobin transports oxygen from the lungs to other parts of the body. Red blood cells with normal
hemoglobin are smooth and round and glide through blood vessels. In people with SCD, abnormal
hemoglobin molecules stick to one another and form long, rod-like structures. These structures cause
red blood cells to become stiff, assuming a sickle shape, which causes them to pile up and create
blockages that can damage vital organs and tissue.
4
SCD can also cause fatigue, which is why it is
sometimes called sickle cell anemia.
5
Diagnosis and Treatment
All newborns in Florida are screened within 1 week after birth for SCD.
6
It can also be diagnosed
prenatally by using diagnostic tests that identify chromosomal or genetic abnormalities.
7
SCD is a disease that gets worse over time, but treatments exist that can prevent complications and
lengthen the lives of people with SCD. Oral penicillin is the standard of care for children with SCD
because chronic damage to the spleen increases the risk of life-threatening pneumococcal bacterial
infection.
8
The only therapy approved by the FDA that may be able to cure SCD is a bone marrow or
stem cell transplant.
9
These treatments are estimated to cost $1 million to $2 million per person and are
only available at a limited number of transplant centers.
10
AHCA Report on Sickle Cell Disease
In 2022, the Legislature appropriated $250,000 in nonrecurring funds from the General Revenue Fund
to the Agency for Health Care Administration (AHCA), in consultation with the Florida Medical Schools
Quality Network and a dedicated SCD medical treatment and research center that maintains a sickle
cell patient database and tracks SCD outcome measures, to conduct a review and publish a report of
1
National Heart, Lung, and Blood Institute, What is Sickle Cell Disease?, available at https://www.nhlbi.nih.gov/health/sickle-cell-
disease (last visited March 20, 2023).
2
AHCA, Florida Medicaid Study of Enrollees with Sickle Cell Disease, February 1, 2023, available at
https://ahca.myflorida.com/content/download/20771/file/Florida_Medicaid_Study_of_Enrollees_with_Sickle_Cell_Disease.pdf (last
viewed Apr. 10, 2023).
3
Id.
4
CDC, What is Sickle Cell Disease?, available at https://www.cdc.gov/ncbddd/sicklecell/facts.html (last visited March 20, 2023).
5
Id.
6
S. 383.14(2), F.S., and rule 64C-7.002, F.A.C.
7
Supra note 4.
8
Supra note 2, at 10-12. Hydroxyurea is the standard of care for people as young as 9 months old; L-Glutamine is recommended for
adults and children age 5 and older; Voxelotor is recommended for adults and children age 4 and older; Crizanlizumab is recommended
for people age 16 and older; and opioids and iron chelating agents are also often used.
9
Supra note 4.
10
Supra note 2. STORAGE NAME: h1481e.HHS PAGE: 3
DATE: 4/24/2023
its findings on the availability and utilization of specific services for Medicaid enrollees diagnosed with
SCD.
11
Specifically, the report was required to include detailed information on:
Age and population demographics;
Utilization patterns and expenditures for all medical and pharmaceutical services provided; and
The number of clinical treatment programs who contract with managed care plans and are
specifically designed or certified to provide care to individuals with SCD.
The report was published on February 1, 2023, and the results were based on data gathered between
2018 through 2021 on Medicaid enrollees with SCD.
12
Age and Population Demographics
The average number of Medicaid enrollees with SCD is 7,328, which is twice as high as the national
average. Florida and New York have the highest number of Medicaid recipients with SCD. According to
the report, the SCD population is predominantly female (58%), young (median age 18), and black
(63%). Geographically, the highest number of Medicaid enrollees with SCD live in Central and South
Florida.
Utilization of Medical and Pharmaceutical Services
Nearly all enrollees with SCD were evaluated by a physician at least once and approximately 85
percent were evaluated or treated in an outpatient clinic setting at least once. Sixty-one percent were
evaluated and treated in an emergency room at least once, and those with an emergency room visit
had an average of 4.5 visits from 2018 to 2021. Fifty-two percent of enrollees with SCD were admitted
for inpatient care in a hospital, and those enrollees had an average of 2.9 inpatient admissions from
2018 to 2021.
National guidelines recommend that children with SCD aged 2-16 should have an annual transcranial
doppler ultrasound (TCD) to screen for stroke risk.
13
Florida Medicaid child enrollees had an average of
two TCDs over the four-year reporting period, which was below the recommended four TCDs for that
period.
Data on medication utilization indicated that 77 percent of Medicaid enrollees with SCD had a
pharmacy expenditure for a sickle cell disease-relevant medication, including disease-modifying
treatments, prophylactic antibiotics, opioids, or iron chelating agents.
Medication Utilization by Eligible Individuals with SCD
14
Medication
Number Eligible to
Receive Medication
Number of Patients
Taking Medication
Percent of Eligible
Population Taking
Medication
Oral Penicillin 2,054 1,197 58%
Hydroxyurea 7,613 1,691 22%
L-glutamine 6,840 154 2%
Voxelotor 5,629 8 0.1%
Crizanlizumab 4, 956 4 0.8%
Managed Care Clinical Treatment Programs for SCD
11
Ch. 2022-156, Laws of Fla.
12
Supra note 2.
13
National Heart, Lung, and Blood Institute, Evidence-Based Management of Sickle Cell Disease: Expert Panel Report, 2014, available
at https://www.nhlbi.nih.gov/health-topics/evidence-based-management-sickle-cell-disease (last visited March 20, 2023).
14
Supra note 2, at 10. STORAGE NAME: h1481e.HHS PAGE: 4
DATE: 4/24/2023
The report identified 72 clinical treatment programs who contract with managed care plans to provide
care to individuals with SCD. They include as little as one treatment program offered by a managed
care plan to as many as 23. According to the report, in addition to these clinical treatment programs,
hematopoietic cell transplant programs at the University of Miami, H. Lee Moffitt Cancer Center, the
University of Florida, and others have transplanted autologous or allogeneic adult blood stem cells to
patients with SCD to introduce a new blood system free of sickle shaped red blood cells. Further, in
2023, these transplant centers may also offer new FDA approved gene therapies for people with SCD
that have the potential to cure SCD. These treatments are estimated to cost $1 million to $2 million per
person and are only available at a limited number of transplant centers.
15
Effect of the Bill
CS/CS/HB 1481 requires AHCA, in consultation with the Florida Medical Schools Quality Network and
a dedicated SCD medical treatment and research center that maintains a sickle cell patient database
and tracks SCD outcome measures, to biannually review and report the same information it was
required to report by the Fiscal Year 2022-2023 GAA. Additionally, it requires AHCA to assess their
existing Medicaid payment methodologies for approved SCD treatments and medications in the
inpatient setting and whether such payment methodologies result in barriers to access. If barriers to
access are identified, AHCA must assess whether the payment methodologies may be modified or
improved.
The bill authorizes $250,000 in nonrecurring funds from the General Revenue Fund to AHCA to
conduct the review and develop the report.
The bill requires the Department of Health (DOH) to contract with a community-based sickle cell
disease medical treatment and research center to establish and maintain a registry to track outcome
measures of newborns who are identified as carrying a sickle cell hemoglobin variant. The bill requires
a screening provider to notify the primary care physician of an infant who tests positive for sickle cell
hemoglobin variant and to submit the results of the screening to DOH for inclusion in the sickle cell
registry. The primary care physician must provide information on the availability and benefits of genetic
counseling to the parent or guardian of the newborn.
The bill requires DOH to establish a system to ensure that the sickle cell research center notifies the
parent or guardian of a child included in the registry that a follow-up consultation with a physician is
recommended, at least once during early adolescence and once during late adolescence. The bill also
requires DOH to provide individuals who are 18 years of age and that are included in the registry with
information regarding available educational services, genetic counseling, and other beneficial
resources.
The bill provides rulemaking authority to DOH to create the registry and requires DOH to adopt rules to
establish a process for removing individuals from the registry.
The bill provides an effective date of July 1, 2023.
B. SECTION DIRECTORY:
Section 1: Creates s. 383.147, F.S., relating to newborn and infant screenings for sickle cell
hemoglobin variants; registry.
Section 2: Creates s. 409.91235, F.S., relating to agency review and report on medications,
treatments, and services for sickle cell disease.
Section 3: Provides an appropriation to implement the bill.
Section 4: Provides an appropriation to implement the bill.
Section 5: Provides an effective date of July 1, 2023.
II. FISCAL ANALYSIS & ECONOMIC IMPACT STATEMENT
15
Supra note 2, at 17. STORAGE NAME: h1481e.HHS PAGE: 5
DATE: 4/24/2023
A. FISCAL IMPACT ON STATE GOVERNMENT:
1. Revenues:
None.
2. Expenditures:
The bill provides $250,000 in nonrecurring funds from the General Revenue Fund to AHCA to
conduct the review and develop the report. This is the same amount that AHCA received in 2022,
which it used to contract for the completion of the report. The bill requires the report to be
conducted biannually. If AHCA were to contract for the completion of the biannual report, it would
cost $1.25 million every ten years.
The bill provides $450,000 in recurring funds from the General Revenue Fund to DOH to contract
with a community-based sickle cell disease medical treatment and research center to establish and
maintain a registry to track outcome measure.
The bill provides $150,000 in recurring funds from the General Revenue Fund to educate providers
and consumers throughout the state about the sickle cell registry.
Finally, the bill appropriates $460,804 in recurring funds from the General Revenue Fund for five
full-time equivalent positions along with the associated salary rate to implement the provisions of
the bill.
B. FISCAL IMPACT ON LOCAL GOVERNMENTS:
1. Revenues:
None.
2. Expenditures:
None.
C. DIRECT ECONOMIC IMPACT ON PRIVATE SECTOR:
None.
D. FISCAL COMMENTS:
None.
III. COMMENTS
A. CONSTITUTIONAL ISSUES:
1. Applicability of Municipality/County Mandates Provision:
Not applicable. The bill does not appear to affect county or municipal governments.
2. Other:
None.
B. RULE-MAKING AUTHORITY:
The bill provides sufficient rule-making authority to DOH to implement the provisions of the bill.
C. DRAFTING ISSUES OR OTHER COMMENTS:
None.
STORAGE NAME: h1481e.HHS PAGE: 6
DATE: 4/24/2023
IV. AMENDMENTS/COMMITTEE SUBSTITUTE CHANGES
On March 22, 2023, the Healthcare Regulation Subcommittee adopted an amendment and reported the bill
favorably as a committee substitute. The amendment:
Required the Department of Health (DOH) to contract with a community-based sickle cell disease
medical treatment and research center to establish and maintain a registry for newborns who are
identified as carrying a sickle cell hemoglobin variant.
Required the registry to track sickle cell disease outcome measures.
Required DOH to adopt a rule to create a process for a parent or guardian of a newborn to request
to have their child removed from the registry.
Required a screening provider to notify the primary care physician of an infant who tests positive for
sickle cell hemoglobin variant and to submit the results of the screening to DOH for inclusion in the
sickle cell registry.
Required DOH to establish a system to ensure that the sickle cell research center notifies the
parent or guardian of a child included in the registry that a follow-up consultation with a physician is
recommended, at least once during early adolescence and once during late adolescence.
Required DOH to make every reasonable effort to notify individuals who are 18 years of age that
they may be removed from the registry, and requires DOH to adopt a rule to create a process for
such notification.
Required DOH to provide individuals who are 18 years of age and that are included in the registry
with information regarding available educational services, genetic counseling, and other beneficial
resources.
Provided rulemaking authority to DOH.
On April 12, 2023, the Health Care Appropriations Subcommittee adopted an amendment and reported the
bill favorably as a committee substitute. The amendment appropriates 5.0 full-time equivalent positions and
the associated salary rate and budget to the DOH for the implementation of this bill.
On April 24, 2023, the Health & Human Services Committee adopted an amendment and reported the bill
favorably as a committee substitute. The amendment removed duplicative provisions from the bill.
This analysis is drafted to the committee substitute as passed by the Health & Human Services Committee.