This docum ent does not reflect the intent or official position of the bill sponsor or House of Representatives.
STORAGE NAME: h7085.HHS
DATE: 2/22/2024
HOUSE OF REPRESENTATIVES STAFF ANALYSIS
BILL #: HB 7085 PCB HHS 24-04 Sickle Cell Disease
SPONSOR(S): Health & Human Services Committee, Skidmore
TIED BILLS: IDEN./SIM. BILLS:
REFERENCE ACTION ANALYST STAFF DIRECTOR or
BUDGET/POLICY CHIEF
Orig. Comm.: Health & Human Services
Committee
19 Y, 0 N Osborne Calamas
SUMMARY ANALYSIS
Sickle cell disease (SCD) is the most common inherited blood disorder in the United States, affecting
approximately 100,000 Americans. SCD affects mostly, but not exclusively, Americans of African ancestry.
SCD is a group of inherited disorders in which abnormal hemoglobin cause red blood cells to buckle into the
iconic sickle shape; the deformed red blood cells damage blood vessels and over time contribute to a cascade
of negative health effects beginning in infancy, such as intense vaso-occlusive pain episodes, strokes, organ
failure, and recurrent infections. The severity of complications generally worsens as people age, but treatment
and prevention strategies can mitigate complications and lengthen the lives of people with SCD.
Treatment for SCD has improved significantly in recent decades. Appropriate pharmaceutical treatments and
evidence-based management protocols have the capacity to significantly improve the quality of life for people
with SCD. In spite of the improvements in treatments for SCD, there significant underutilization among
patients, due in part to gaps in understanding of the disease and its treatments among health care
practitioners.
In 2023, the Legislature directed the Department of Health (DOH) to partner 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. Adults identified as carrying a
sickle cell hemoglobin variant are not eligible to participate in the registry.
HB 7085 creates the Sickle Cell Disease Research and Treatment Grant Program (Program) within DOH. The
Program will award grants to community-based sickle cell disease treatment and research centers to fund the
operation of Centers of Excellence for the treatment of sickle cell disease and the development of a health care
workforce that is prepared to address the unique needs of patients with sickle cell disease.
The bill expands the existing sickle cell registry to allow adults with sickle cell disease to, at their discretion, opt
into the registry.
The bill has a significant, negative fiscal impact on state government. See Fiscal Comments. The bill has no
fiscal impact on local government.
The bill provides an effective date of July 1, 2024.
STORAGE NAME: h7085.HHS PAGE: 2
DATE: 2/22/2024
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, affecting
approximately 100,000 Americans.
1
SCD affects mostly, but not exclusively, Americans of African
ancestry.
2
SCD is a group of inherited disorders in which abnormal hemoglobin cause red blood cells to
buckle into the iconic sickle shape; the deformed red blood cells damage blood vessels and over time
contribute to a cascade of negative health effects beginning in infancy, such as intense vaso-occlusive
pain episodes, strokes, organ failure, and recurrent infections.
3
The severity of complications from SCD generally worsen as people age, but treatment and prevention
strategies can mitigate complications and lengthen the lives of people with SCD.
4
SCD was historically
perceived as a childhood disease due to high rates of childhood mortality, however, more than 90
percent of those living with the disease today are expected to survive into adulthood.
5
Roughly 60
percent of individuals with SCD in the US today are adults, but the life expectancy of individuals with
SCD remains approximately 22 years shorter than the general population.
6
Management of SCD
SCD management primarily focuses on treating and preventing complications caused by the disease
such as acute pain episodes, infection, stroke, vision loss, and severe anemia. The most well-
researched treatments for SCD relate to mitigating the risk of infection and stroke in children. There is a
lack of research-driven data specific to adult populations with SCD.
7
Stroke is one of the most common and devastating complications of SCD.
8
Blood transfusions may be
used to treat acute episodes of elevated stroke risk, or through chronic transfusion therapy which
reduces a person’s overall stroke risk as well as preventing painful vaso-occlusive events.
9
Chronic
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 January 30, 2024).
2
Centers for Disease Control and Prevention, Data & Statistics on Sickle Cell Disease. Available at
https://www.cdc.gov/ncbddd/sicklecell/data.html (last visited January 30, 2024).
3
Centers for Disease Control and Prevention, What is Sickle Cell Disease? Available at
https://www.cdc.gov/ncbddd/sicklecell/facts.html (last visited January 24, 2024). See also, AHCA (2023) Florida Medicaid Study of
Enrollees with Sickle Cell Disease. Available at
https://ahca.myflorida.com/content/download/20771/file/Florida_Medicaid_Study_of_Enrollees_with_Sickle_Cell_Disease.pdf (last
visited January 24, 2024).
4
Centers for Disease Control and Prevention, Complications of Sickle Cell Disease. Available at
https://www.cdc.gov/ncbddd/sicklecell/complications.html (last visited January 24, 2024).
5
DiMartino, L. D., Baumann, A. A., Hsu, L. L., Kanter, J., Gordeuk, V. R., Glassberg, J., Treadwell, M. J., Melvin, C. L., Telfair, J.,
Klesges, L. M., King, A., Wun, T., Shah, N., Gibson, R. W., Hankins, J. S., & Sickle Cell Disease Implementation Consortium (2018).
The sickle cell disease implementation consortium: Translating evidence-based guidelines into practice for sickle cell disease.
American journal of hematology, 93(12), E391–E395. https://doi.org/10.1002/ajh.25282.
6
Lubeck D, Agodoa I, Bhakta N, et al. (2019) Estimated Life Expectancy and Income of Patients With Sickle Cell Disease Compared
With Those Without Sickle Cell Disease. JAMA Netw Open. 2019;2(11):e1915374. doi:10.1001/jamanetworkopen.2019.15374.
Available at https://jamanetwork.com/journals/jamanetworkopen/article-abstract/2755485 (last visited January 30, 2024).
7
Adams-Graves, P. & Bronte-James, L. Recent Treatment Guidelines for Managing Adult Patients with Sickle Cell Disease: Challenges
in Access to Care, Social Issues, and Adherence. (2016). Expert Review of Hematology, 9:6, 511-614.
http://dx.doi.org/10.1080/17474086.2016.1180242
8
U.S. Department of Health and Human Services, 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 January 31, 2024).
9
Brandow, A.M., Panepinto, J.A. (2010). Hydroxyurea Use in Sickle Cell Disease: The Battle with Low Prescription Rates, Poor Patient
Compliance, and Fears of Toxicities. Expert Reviews: Hematology. DOI: 10.1586/EHM.10.22 STORAGE NAME: h7085.HHS PAGE: 3
DATE: 2/22/2024
transfusion therapy has been shown to improve health-related quality of life in children with SCD.
10
There are, however, risks associated with frequent blood transfusions and chronic transfusion therapy
can be logistically and financially difficult for caregivers to manage.
11
A transcranial Doppler ultrasound
(TCD), is a specialized ultrasound device capable of detecting elevated stroke risk.
12
For children ages
2-16 with SCD who have a heightened risk of stroke, annual TCD screening is recommended by the
American Society of Hematology to monitor stroke risk and prevent stroke.
13
People with SCD are generally at a higher risk of severe bacterial infections due to poor spleen
function, but fatality is especially high among young children and infants who lack the immune response
necessary to combat infection. Defective or reduced spleen function begins early in the first year of life
for infants with SCD.
14
To protect against life-threatening pneumococcal bacterial infection, daily oral
penicillin is the standard of care for children from infancy through age five.
15
In addition to daily oral penicillin and routine screening to monitor stroke risk in children, there are other
pharmaceutical treatments available to manage the symptoms of SCD, reduce the long-term health
impacts of the disease, and improve quality of life for children and adults with SCD. Hydroxyurea is an
oral medication taken once daily which has been proven to be effective at reducing a person’s pain
episodes, mitigating stroke risk, and preventing organ damage.
16
Hydroxyurea is generally safe for both
children and adults and is recommended for patients with certain forms of SCD experiencing “frequent
pain episodes” or acute chest syndrome.
17
Opioids are commonly used to treat the severe acute pain that results from vaso-occlusive episodes.
Opioids are not recommended for treatment of the chronic pain that is associated with SCD due to the
significant risks of overdose and addiction associated with frequent opioid use. Opioids are, however,
very effective for managing acute severe pain in acute settings and as such the National Heart Lung
and Blood Institute recommends rapid initiation of opioids for patients visiting the emergency
department for a vaso-occlusive pain episode.
18
More recent pharmaceutical developments for the treatment of SCD include L-glutamine, Voxelotor,
and Crizanlizumab. L-glutamine in an essential amino acid which was approved by the FDA in 2017 for
the treatment of SCD in adults and children over five years of age. The mechanism of action of L-
glutamine is not well understood, however, it has been shown to reduce a patient’s number of sickle
cell crisis episodes.
19
Voxelotor and Crizanlizumab are two disease modifying drugs approved by the
FDA in 2019. The drugs may be beneficial for different subgroups of SCD patients for whom other
treatments have proven insufficient or ineffective. Voxelotor and Crizanlizumab act through different
10
Beverung, L.M., Strouse, J.J., Hulbert, M.L. (2015) Health-related Quality of Life in Children with Sickle Cell Anemia: Impact of Blood
Transfusion Therapy. American Journal of Hematology. http://doi.org/10/1002/ajh.2387
11
Supra, note 12.
12
Runge, A., Brazel, D., Pakbaz, Z. (2022). Stroke in Sickle Cell Disease and the Promise of Recent Disease Modifying Agents.
Journal of the Neurological Sciences. http://doi.org/10.1016/j.jns.2022.120412
13
DeBaun, M., et al. American Society of Hematology 2020 guidelines for sickle cell disease: prevention, diagnosis, and treatment of
cerebrovascular disease in children and adults. (2020). Blood Advances; 4 (8): 1554–1588.
doi: https://doi.org/10.1182/bloodadvances.2019001142
14
U.S. Department of Health and Human Services, 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 January 31, 2024).
15
AHCA (2023) Florida Medicaid Study of Enrollees with Sickle Cell Disease. Available at
https://ahca.myflorida.com/content/download/20771/file/Florida_Medicaid_Study_of_Enrollees_with_Sickle_Cell_Disease.pdf (last
visited January 24, 2024). Amoxicillin may also be prescribed for this purpose. In patients with a known or suspected penicillin allergy,
erythromycin is prescribed.
16
Id.
17
U.S. Department of Health and Human Services, 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 January 31, 2024).
18
Id. See also, Smeltzer, M.P., Howell, K.E., Treadwell, M. (2021). Identifying barriers to evidence-based care for sickle cell disease:
results from the Sickle Cell Disease Implementation Consortium cross-sectional survey of healthcare providers in the USA. BMJ Open
2021. DOI: 10.1136/bmjopen-2021-050880
19
Quinn C. T. (2018). l-Glutamine for sickle cell anemia: more questions than answers. Blood, 132(7), 689–693.
https://doi.org/10.1182/blood-2018-03-834440. See also, Ballas S. K. The Evolving Pharmacotherapeutic Landscape for the Treatment
of Sickle Cell Disease (2020). Mediterranean Journal of Hematology and Infectious Diseases, 12(1), e2020010.
https://doi.org/10.4084/MJHID.2020.010 STORAGE NAME: h7085.HHS PAGE: 4
DATE: 2/22/2024
mechanisms, but both mitigate the harmful effects of damaged red blood cells in the body. There is
ongoing research into their impact on other SCD morbidities.
20
Curative Treatments for SCD
On December 8, 2023, the FDA approved the first two gene therapies for the treatment of SCD. The
products, Casgevy and Lyfgenia, are cell-based gene therapies approved for the treatment of SCD in
patients 12 years of age or older. Both products are made from the patients’ own blood stem cells,
which are modified, and administered to the patient as a one-time, single-dose infusion as part of a
hematopoietic (blood) stem cell transplant. Prior to treatment, a patients’ stem cells are collected, and
then the patient must undergo high-dose chemotherapy, a process that removes cells from the bone
marrow so they can be replaced with the modified cells.
21
The recently FDA-approved gene therapies have not reached full market availability, but the costs are
anticipated to be as high as $2 to million per patient.
22
It is yet to be determined how insurance
companies or Medicaid will cover the treatment.
23
Prior to the approval of these gene therapy treatments, the only treatment for SCD with curative
potential was a matched/related hematopoietic stem cell transplant (HSCT). HSCT has been shown to
be highly effective as a cure, though outcomes are more favorable when the transplant is performed
before age 16 and with a matched sibling donor.
24
While highly curative, HSCT poses significant risks
including transplant rejection that can result in the patient’s death.
25
The procedure is infrequently
performed due to the high cost,
26
the limited number of capable transplant centers, the strenuous
preparation regimen and significant risks,
27
and the need for a genetically matched donor.
28
Barriers to Care for SCD
While SCD is the most common inherited blood disorder in the US and is often diagnosed at birth
through newborn screening programs,
29
patients with SCD often experience significant barriers to
accessing appropriate care. Barriers to care include lack of insurance, unmet transportation needs, and
provider inexperience and lack of knowledge about SCD. There is a limited number of knowledgeable
health care professionals with expertise in the management of SCD, and mistrust among patients and
bias among providers continue to affect access to and quality of care.
30
Recent decades have brought major scientific advancements in understanding the biological
mechanisms of SCD, the development of new pharmaceutical treatments, the establishment of
evidence-based treatment protocols, and methods for mitigating the risk of catastrophic
20
Supra, note 12.
21
US Food & Drug Administration, FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease (2023). Available at
https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease (last visited
January 30, 2024).
22
National Heart, Lung, and Blood Institute. FDA approval of gene therapies for sickle cell disease: Q&A with NHLBI Director Dr. Gary
Gibbons and NHLBI’s Division of Blood Diseases and Resources Director Dr. Julie Panepinto (2023). Available at
https://www.nhlbi.nih.gov/news/2023/fda-approval-gene-therapies-sickle-cell-disease-dr-gibbons-dr-panepinto (last visited January 30,
2024).
23
MacMillan, C., Casgevy and Lyfgenia: Two Gene Therapies Approved for Sickle Cell Disease (2023). Yale Medicine. Available at
https://www.yalemedicine.org/news/gene-therapies-sickle-cell-disease (last visited January 30, 2023).
24
Gluckman, E., Cappelli, B., Bernaudin, F., et al. (2017). Sickle cell disease: an international survey of results of HLA-identical sibling
hematopoietic stem cell transplantation. Blood, 129(11), 1548–1556. https://doi.org/10.1182/blood-2016-10-745711
25
Ashorobi D, Bhatt R. Bone Marrow Transplantation in Sickle Cell Disease. (2022). In: StatPearls. Treasure Island (FL): StatPearls
Publishing. Available at https://www.ncbi.nlm.nih.gov/books/NBK538515/ (last visited January 31, 2024).
26
Supra, note 17. HSCT is estimated to cost approximately $1 million to $2 million per person.
27
Supra, note 17.
28
Salcedo, J., Bulovic, J., & Young, C. Cost-effectiveness of a Hypothetical Cell or Gene Therapy Cure for Sickle Cell Disease (2021).
Scientific Reports. https://doi.org/10.1038/s41598-021-90405-1
29
Centers for Disease Control and Prevention. Newborn Screening (NBS) Data (2023). Available at
https://www.cdc.gov/ncbddd/hemoglobinopathies/scdc-state-data/newborn-
screening/index.html#:~:text=Newborn%20screening%20(NBS)%20for%20sickle,SCD%20living%20in%20a%20state. (last visited
January 20, 2024).
30
Sickle Cell Disease Coalition, State of Sickle Cell Disease: 2020 Report Card (2020). Available at
http://www.scdcoalition.org/pdfs/SCD%20Report%20Card%202020.pdf (last visited January 31, 2024). STORAGE NAME: h7085.HHS PAGE: 5
DATE: 2/22/2024
complications.
31
Collectively, these advancements provide the means for significantly improving the
quality of life for many patients with SCD; however, few of these interventions are utilized to their full
potential.
The nature of SCD inherently leads to a greater use of health care services compared to the general
population, but gaps in access to appropriate care are common and lead to unmitigated health crises
and a greater consumption of costly emergency medical services.
32
Health care practitioners who have
not specialized in the treatment of SCD express discomfort in prescribing essential treatments for
SCD,
33
and a lack of knowledge regarding recent treatment developments.
34
Access to adequate care is especially challenging for young adults transitioning from pediatric to adult
care settings.
35
While SCD has historically been associated with childhood mortality, more than 90
percent of those living with the disease are expected to survive into adulthood today.
36
The system of
care for SCD has developed with a focus on pediatric patients; as a result, patients with SCD are more
likely to receive well-managed preventative care as children through specialized pediatric programs.
Patients aging out of pediatric care and transitioning into adult care are less likely to have access to
consistent and appropriate SCD care, which leads to higher rates of emergency department reliance
than other age groups.
37
SCD care in emergency settings presents additional challenges. Patients with SCD who present to
emergency care settings in the midst of vaso-occusive pain crises may have their behavior perceived
as drug seeking and have their pain severity doubted and undertreated.
38
Educational gaps and biases
among providers, staff, and patients create barriers to communication and trust, and erode the
provider–patient relationship, which can result in inadequate or inappropriate treatment of patients.
39
Florida’s Medicaid SCD Population
In 2022, the Legislature directed the Agency for Health Care Administration (AHCA) to conduct a study
assessing Florida’s population of Medicaid enrollees with SCD and their utilization of specific health
care services.
40
The Florida Medicaid Study of Enrollees with Sickle Cell Disease (the study) analyzed
data from 2018 through 2021 and found that Florida’s rate of Medicaid enrollees with SCD was twice
31
American Society of Hematology. ASH Sickle Cell Disease Initiative: Sickle Cell Disease Timeline. Available at
https://www.hematology.org/advocacy/sickle-cell-disease-initiative/scd-timeline (last visited January 30, 2024).
32
DiMartino, L. D., Baumann, A. A., Hsu, L. L., Kanter, J., Gordeuk, V. R., Glassberg, J., Treadwell, M. J., Melvin, C. L., Telfair, J.,
Klesges, L. M., King, A., Wun, T., Shah, N., Gibson, R. W., Hankins, J. S., & Sickle Cell Disease Implementation Consortium (2018).
The sickle cell disease implementation consortium: Translating evidence-based guidelines into practice for sickle cell disease.
American Journal of Hematology, 93(12), E391–E395. https://doi.org/10.1002/ajh.25282. See also, Brousseau, D.C., Owens, P.L.,
Mosso, A.L., Panepinto, J.A., Steiner, C.A. Acute Care Utilization and Rehospitalizations for Sickle Cell Disease (2010). JAMA.
2010;303(13):1288–1294. doi:10.1001/jama.2010.378
33
Lanzkron S, Haywood C Jr, Hassell KL, Rand C. Provider barriers to hydroxyurea use in adults with sickle cell disease: a survey of
the sickle cell disease adult provider network. (2008) Journal of the National Medical Association. 100(8): 968-973.
https://doi.org/10.1016/S0027-9684(15)31419-X
34
Robinson, K., Esgro, R., Cooper, S., LoPresti, M., & Carson, B. Identifying and Addressing Knowledge and Confidence Gaps
Regarding the Management of Patients with Sickle Cell Disease Via Engaging Continuing Medical Education. (2023). Blood, 142
(Supplement 1): 7228. doi: https://doi.org/10.1182/blood-2023-177576
35
Hemker, B., Brouseau, D., Yan, K., Hoffmann, R., & Panepinto. When Children with Sickle Cell Disease Become Adults: Lack of
Outpatient Care Leads to Increased Use of the Emergency Department (2011). American Journal of Hematology. 86:10, 863-865.
https://doi.org/10.1002/ajh.22106
36
Id.
37
Blinder, M. A., Duh, M. S., Sasane, M., Trahey, A., Paley, C., & Vekeman, F. Age-Related Emergency Department Reliance in
Patients with Sickle Cell Disease (2015). The Journal of Emergency Medicine, 49(4), 513–522.e1.
https://doi.org/10.1016/j.jemermed.2014.12.080
38
DiMartino, L. D., Baumann, A. A., Hsu, L. L., Kanter, J., Gordeuk, V. R., Glassberg, J., Treadwell, M. J., Melvin, C. L., Telfair, J.,
Klesges, L. M., King, A., Wun, T., Shah, N., Gibson, R. W., & Hankins, J. S. The sickle cell disease implementation consortium:
Translating evidence-based guidelines into practice for sickle cell disease (2018). American Journal of Hematology, 93(12), E391–
E395. https://doi.org/10.1002/ajh.25282
39
Glassberg, G., Improving Emergency Department-Based Care of Sickle Cell Pain (2017). Hematology. American Society of
Hematology. Education Program, 2017(1), 412–417. https://doi.org/10.1182/asheducation-2017.1.412
40
AHCA (2023) Florida Medicaid Study of Enrollees with Sickle Cell Disease. Available at
https://ahca.myflorida.com/content/download/20771/file/Florida_Medicaid_Study_of_Enrollees_with_Sickle_Cell_Disease.pdf (last
visited January 30, 2024). STORAGE NAME: h7085.HHS PAGE: 6
DATE: 2/22/2024
that of the national average,
41
with approximately 7,328 Medicaid enrollees with SCD per year. The
study found that Florida’s Medicaid SCD population was predominantly female (58%), young (median
age 18), and Black (63%).
The study showed that nearly all of the Medicaid SCD population received treatment from a physician
at least once during the study period. 85 percent of Medicaid SCD patients were evaluated or treated in
an outpatient clinic setting, 61 percent were treated in an emergency room (ER) at least once, and 52
percent were admitted for inpatient care in a hospital. Individuals who received treatment in an ER had
an average of 4.5 visits to the ER during the four-year study period.
The study showed that routine screenings and preventative treatments were broadly underutilized by
the Medicaid SCD population. Only 41 percent of children in the Medicaid SCD population had at least
one TCD screening for stroke risk during the four-year study period; this is significantly less than the
recommended annual screening for children with SCD.
42
Data on blood transfusions, which are
commonly used to reduce stroke risk when elevated risk is detected by TCD, were not included in the
study.
The study showed that penicillin was the most commonly prescribed medication for Medicaid SCD
patients. The study showed that 58 percent of eligible individuals were being prescribed penicillin, but
there remains a persistent gap between use and recommended care. Other medications for treating
SCD symptoms and complications were prescribed with even less frequency. Hydroxyurea
43
and L-
glutamine were prescribed to only 22 percent and 2 percent of eligible SCD Medicaid patients
respectively. The newer disease-modifying drugs, Voxelotor and Crizanlizumab were each prescribed
to less than 1 percent of the eligible Medicaid SCD population.
Sickle Cell Disease Registry
Sickle cell disease presents unique challenges to medical researchers; patient data is scare, small
sample sizes limit research possibilities, and mistrust of the medical establishment is common among
patients with sickle cell disease leads. Patient registries are a means of overcoming some of the
research limitations that exist due to the nature of rare diseases generally and sickle cell disease
specifically. Patient registries are organized systems that allow for the use of observational study
methods to collect uniform data and evaluate specified outcomes for a population defined by a
particular disease.
44
In 2023, the Legislature directed the Department of Health (DOH) to partner 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.
45
DOH
has since contracted with the Foundation for Sickle Cell Research for the implementation of the
registry.
46
Under current law, only newborns who have been detected as carrying a sickle cell
hemoglobin variant through the Newborn Screening Program are included in the registry. Parents may
choose to have their child removed from the registry by submitting a form provided by DOH.
47
There is
not a mechanism under current law for adults with SCD to be included in the registry.
Current law also directs the newborn’s primary care physician to provide the parent or guardian of the
newborn with information regarding the availability and benefits of genetic counseling.
41
Centers for Medicare and Medicaid Services (2021), Medicaid and CHIP Sickle Cell Disease Report, T-MSIS Analytic Files (TAF)
2017. Available at https://www.medicaid.gov/medicaid/quality-of-care/downloads/scd-rpt-jan-2021.pdf (last visited January 31, 2024).
42
Supra, note 17.
43
AHCA cites high-cost as a potential barrier to the utilization of hydroxyurea by patients, however, hydroxyurea is on Florida’s
preferred drug list for patients with SCD, which significantly reduces the cost for Medicaid patients.
44
Hageman, I.C., van Rooij, I.A., de Blaauw, I., et al. A systematic overview of rare disease patient registries: challenges in design,
quality management, and maintenance (2023). Orphanet Journal of Rare Diseases 18, 106. https://doi.org/10.1186/s13023-023-02719-
0
45
S. 383.147, F.S.
46
Department of Health. Contract Summary: Contract # CMO28. On file with the Healthcare Regulation Subcommittee.
47
S. 383.147, F.S. STORAGE NAME: h7085.HHS PAGE: 7
DATE: 2/22/2024
Effect of the Bill
Sickle Cell Disease Research and Treatment Grant Program
HB 7085 creates the Sickle Cell Disease Research and Treatment Grant Program (Program) within
DOH. The Program will fund projects that improve the quality and accessibility of health care that is
available to people living with SCD in Florida. The Program seeks to:
Improve the health outcomes and quality of life for Floridians with SCD;
Expand access to high-quality, specialized care for SCD; and
Improve awareness and understanding among health care practitioners of current best
practices for the treatment and management of SCD.
The Office of Minority Health and Health Equity, within DOH, will award grants to community-based
sickle cell disease medical treatment and research centers for projects which support the cultivation of
a health care workforce that is educated and familiar with the unique needs of patients with SCD, and
the growth and development of SCD Centers of Excellence.
Under the bill, a SCD Center of Excellence is a health care facility which is dedicated to the treatment
of patients with sickle cell disease. A Center of Excellence provides evidence-based, comprehensive,
patient-centered coordinated care for SCD patients.
The bill requires DOH to:
Publicize the availability of funds and establish an application process for grant proposals;
Initiate a call for applications no later than July 15, 2024;
Develop uniform data reporting requirements in order to evaluate the performance of grant
recipients and the improvement of health outcomes; and
Develop a monitoring process to evaluate progress toward meeting grant objectives.
DOH must also submit an annual report to the Governor, the President of the Senate, the Speaker of
the House of Representatives, and the State Surgeon General by March 1 of each year. The report
must include the status and progress of each project supported by the Program in the previous
calendar year, and include the following components for each project:
A summary of the project and the project outcomes or expected project outcomes
The status of the project, including whether it has concluded or the estimated date of
completion;
The amount of the grant awarded and the estimated or actual cost of the project;
The source and amount of any federal, state, or local government grants or donations or private
grants or donations funding the project; and
A list of all entities involved in the project.
The bill specifies that no more that 5 percent of grant funds may be used by a grant recipient toward
administrative expenses. The bill also grants that the balance of any appropriation from the General
Revenue Fund for the program which has not been disbursed, but which is obligated under a contract
or committed to be expended June 30
th
of the Fiscal Year, may be carried forward for up to five years
after the effective date of the original appropriation.
The bill authorizes DOH to adopt rules as necessary to implement the provisions of the bill.
Sickle Cell Disease Registry
The bill revises the registry to clarify the role of screening providers, DOH, and primary care physicians
in the processes in current law. The bill transfers the responsibility of informing parents of the
availability and benefits of genetic counseling from the infant’s primary care physician to DOH.
STORAGE NAME: h7085.HHS PAGE: 8
DATE: 2/22/2024
The bill also creates a process by which adults with SCD who are Florida residents to choose to be
included in the registry. The bill directs DOH to prescribe by rule the process for an adult to opt into the
registry.
The bill provides an effective date of July 1, 2024.
B. SECTION DIRECTORY:
Section 1: Creates s. 381.814, F.S., relating to the Sickle Cell Disease Research and Treatment
Grant Program.
Section 2: Amends s. 383.147, F.S., relating to newborn and infant screenings for sickle cell
hemoglobin variants; registry.
Section 3: Provides an effective date of July 1, 2024.
II. FISCAL ANALYSIS & ECONOMIC IMPACT STATEMENT
A. FISCAL IMPACT ON STATE GOVERNMENT:
1. Revenues:
None.
2. Expenditures:
The bill will have a significant, negative impact on state government. The House of Representatives
has allocated $10 million in the 2024-2025 General Appropriations Act to fund sickle cell treatment
and research.
48
B. FISCAL IMPACT ON LOCAL GOVERNMENTS:
1. Revenues:
None.
2. Expenditures:
None.
C. DIRECT ECONOMIC IMPACT ON PRIVATE S ECTOR:
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:
48
HB 5001 (2024), Section 3 430A, pg. 102. Available at
https://www.myfloridahouse.gov/Sections/Documents/loaddoc.aspx?FileName=Orig_GAA.pdf&DocumentType=Bill&BillN
umber=5001&Session=2024 (last visited February 19, 2024). STORAGE NAME: h7085.HHS PAGE: 9
DATE: 2/22/2024
None.
B. RULE-MAKING AUTHORITY:
The bill provides sufficient rule-making authority to implement the provisions of the bill.
C. DRAFTING ISSUES OR OTHER COMMENTS:
None.
IV. AMENDMENTS/COMMITTEE SUBSTITUTE CHANGES