HOUSE OF REPRESENTATIVES STAFF ANALYSIS
BILL #: CS/HB 349 Sickle Cell Care Management and Treatment Education for Physicians
SPONSOR(S): Healthcare Regulation Subcommittee, Robinson, F.
TIED BILLS: IDEN./SIM. BILLS: SB 552
REFERENCE ACTION ANALYST STAFF DIRECTOR or
BUDGET/POLICY CHIEF
1) Healthcare Regulation Subcommittee 15 Y, 0 N, As CS Osborne McElroy
2) Health Care Appropriations Subcommittee
3) Health & Human Services Committee
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.
CS/HB 349 requires specified health care practitioners to complete two hours of continuing education on the
subject of sickle care disease management as a part of every second biennial licensure or certification
renewal. The bill specifies that the course shall consist of education specific to SCD, including evidence-based
treatment protocols for patients of all ages, continuing patient and family education, periodic comprehensive
health evaluations and other disease-specific health maintenance services, psychosocial care, genetic
counseling, and pain management.
The Board of Medicine, the Board of Osteopathic Medicine, and the Board of Nursing are responsible for
implementing the provisions of the bill and approving appropriate continuing education courses. The bill
authorizes each board to adopt rules to implement the provisions of the bill.
The continuing education course required under the bill may count toward a licensee’s total number of
continuing education requirements for professionals required to complete 30 or more hours of continuing
education biennially. The bill allows a professional holding two or more licenses subject to the requirements of
the bill to satisfy such requirement through the completion of one board-approved course. Failure to comply
with the requirements of the bill constitute grounds for disciplinary action.
The bill has an insignificant, negative fiscal impact on DOH, and no fiscal impact on local government.
The bill provides an effective date of July 1, 2024.
This docum ent does not reflect the intent or official position of the bill sponsor or House of Representatives .
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 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., Tel fair, J.,
Klesges, L. M., King, A., Wun, T., Shah, N., Gibson, R. W., Hankins, J. S., & Sickle Cell Disease Impl ementation 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
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 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
cereb rovascular 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 penicil lin 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 b arriers to evidence-b ased 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. (2020). The Evolving Pharmacotherapeutic Landscape for the
Treatment of Sickle Cell Disease. Mediterranean journal of hematology and infectious diseases, 12(1), e2020010.
https://doi.org/10.4084/MJHID.2020.010
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 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 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 am ong 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
Gib b ons 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 sib ling
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.n