As the end of summer approaches, we find ourselves in a period of transition, much like the evolving landscape of sickle cell disease (SCD) treatment and policy. The intense heat of Southern California is starting to ease, signaling a time of change and reflection—a perfect backdrop to National Sickle Cell Awareness Month in September.
Sickle cell disease (SCD) is an inherited blood disorder. If either or both parents have SCD or is a carrier (having the genetic trait but not the disease), their child is at risk. People born with SCD have crescent-shaped red blood cells that break down quickly and get stuck together, causing blockages. Complications of SCD include anemia; episodes of severe pain; heart, lung, and kidney damage that shortens their lifespan; serious infections; and many other issues.1
Over the years, treatments such as medications and blood transfusions have been standard, but recent advancements have introduced groundbreaking options that bring new hope. One of the most significant developments in recent times is the introduction of gene therapies like Casgevy and Lyfgenia, approved in December 2023. These therapies offer life changing possibilities, marking a new chapter in SCD treatment. However, they also come with considerable risks and side effects, emphasizing the ongoing need for comprehensive care and support to truly enhance the quality of life for those affected.2
The evolution of health policies over the last two and a half decades has mirrored this shift towards more comprehensive support for SCD patients. Starting with the introduction of the Sickle Cell Treatment Act of 2003, which sought to improve treatment and bolster research efforts.3 This was quickly followed by the 2004 American Jobs Creation Act that amended requirements that Medicaid cover primary and secondary prevention, including genetic testing and counseling for people with SCD.4
The Affordable Care Act of 2010 was a significant milestone, expanding healthcare access and ensuring that individuals with pre-existing conditions like SCD could not be denied coverage.5 In 2014, the National Heart, Lung, and Blood Institute released updated guidelines for SCD management, highlighting the importance of evidence-based care.6
In 2016, the Advancing Hope Act expanded the priority review voucher program to include treatments for SCD, encouraging the development of new therapies.7 The 2018 Sickle Cell Disease and Other Heritable Blood Disorders Research, Surveillance, Prevention, and Treatment Act reauthorized crucial programs, while the CDC's Data Collection on Sickle Cell Disease initiative aimed to enhance surveillance and understanding of SCD prevalence and outcomes.8
More recently, the Sickle Cell Disease Comprehensive Care Act of 20219 and the Sickle Cell Disease Treatment Centers Act of 202210 have focused on closing gaps in care and ensuring that patients have access to the latest treatments. These policies underscore the importance of multidisciplinary care teams, mental health services, and specialized support for those living with SCD.
However, not all policy changes have been favorable. The opioid crisis in the U.S. led to tighter regulations on opioid prescriptions, which inadvertently harmed SCD patients who rely on these medications for pain management. In 2017, the US Drug Enforcement Administration and CVS issued policies restricting the use of opioids with an exception for patients with cancer or in end-of-life care.11 In response, the Centers for Medicare & Medicaid Services issued policy recommending the exemption of Medicare beneficiaries with SCD from these restrictions in 2019, acknowledging the unique needs of this population.11
Looking ahead, the Sickle Cell Disease Comprehensive Care Act (S.996 – 118th Congress, 2023-2024) represents another crucial step forward. This bill sets aside funds for a state Medicaid demonstration project aimed at improving outpatient care for SCD patients, with a special focus on young adults and pregnant women. By selecting five to ten states to participate, the project will ensure that multidisciplinary care teams and essential services are accessible to those who need them most.12
As we near the end of summer, we are reminded that change, though challenging, often brings progress. The advancements in SCD treatment and the supportive policies now in place offer hope for a future where those affected by this disease can look forward to better health outcomes and an improved quality of life.
Author Bio:
Lisa R. Roberts, DrPH, MSN, RN
Dr. Roberts is a Professor and the Research Director at the School of Nursing, with a secondary appointment in the Division of Interdisciplinary Studies in the School of Behavioral Health. Her primary research interest concerns maternal health and coping with perinatal grief. Her research interests also include mixed methods and community-based self-help interventions, addressing issues impacting health disparities and vulnerable populations. Her clinical focus as a Family Nurse Practitioner is prevention and primary care.
GRANT SUPPORT: 1) Health Resources and Services Administration (HRSA) of the U.S. Department of Health and Human Services (HHS) under grant number UR6MC50347‐01‐00 Maternal Health Research Network (MH‐RN) for MSIs‐‐Research Awards to LOMA LINDA UNIVERSITY, Loma Linda, California.
Chanell Grismore, DrPH, MPH, MCHES
Dr. Chanell Grismore is an equity focused researcher and administrator with an emphasis on community engagement, healthcare, and environmental justice. She is an Assistant Clinical Professor in the Division of Interdisciplinary Studies in the School of Behavioral Health, leading research on Sickle Cell Disease, including a regional needs assessment and examination of care barriers and social determinants of health. Dr. Grismore employs an intersectional approach in her work, emphasizing how social identities lead to nuanced experiences of marginalized communities. As the Senior Manger of Patient Affairs at Pfizer inc., she develops community partnerships and supports advocacy for policy efforts. Previously, she was the Director of the Comprehensive Adult Sickle Cell Center at Loma Linda University Medical Center, where she shaped the adult sickle cell program.
References:
- American Society of Hematology. (2023). Understanding Sickle Cell Disease. American Society of Hematology. Retrieved June 2, 2024, from https://www.hematology.org/education/clinicians/guidelines-and-quality-care/clinical-practice-guidelines/sickle-cell-disease-guidelines
- Centers for Disease Control and Prevention. (2024). What you should know about sickle cell disease. In CDC Division of Blood Disorders and Public Health Genomics (Ed.), (Vol. CS 348718-A, pp. 2). CDC.
- Congress.gov. (2003). S.874 - Sickle Cell Treatment Act of 2003, 108th Congress. Retrieved from https://www.congress.gov/bill/108th-congress/senate-bill/874?q=%7B%22search%22%3A%22Sickle+Cell+Disease+%22%7D&s=7&r=1
- Congress.gov. (2004). H.R.4520 - American Jobs Creation Act of 2004, 108th Congress. Retrieved from https://www.congress.gov/bill/108th-congress/house-bill/4520?q=%7B%22search%22%3A%22Sickle+Cell+Disease+%22%7D&s=6&r=5
- U.S. Department of Health & Human Services. (2010). About the Affordable Care Act. Retrieved from https://www.hhs.gov/healthcare/about-the-aca/index.html
- National Heart, Lung, and Blood Institute. (2014). Evidence-based management of sickle cell disease. Retrieved from https://www.nhlbi.nih.gov/health-topics/evidence-based-management-sickle-cell-disease
- Congress.gov. (2015). S.1878 - Advancing Hope Act, 114th Congress. Retrieved from https://www.congress.gov/bill/114th-congress/senate-bill/1878?q=%7B%22search%22%3A%22Sickle+Cell+Disease+%22%7D&s=2&r=7
- Congress.gov. (2018). S.2465 - 2018 Act, 115th Congress. Retrieved from https://www.congress.gov/bill/115th-congress/senate-bill/2465?q=%7B%22search%22%3A%22Sickle+Cell+Disease+%22%7D&s=5&r=3
- Congress.gov. (2021). S.3389 - 2021 Act, 117th Congress. Retrieved from https://www.congress.gov/bill/117th-congress/senate-bill/3389?q=%7B%22search%22%3A%22Sickle+Cell+Disease+%22%7D&s=9&r=9
- Congress.gov. (2022). S.4866 - 2022 Act, 117th Congress. Retrieved from https://www.congress.gov/bill/117th-congress/senate-bill/4866?q=%7B%22search%22%3A%22Sickle+Cell+Disease+%22%7D&s=8&r=7
- Lee, L., Smith-Whitley, K., Banks, S., & Puckrein, G. (2019). Reducing health care disparities in sickle cell disease: A review. *Public Health Reports, 134*(6), 599-607.
- Congress.gov. (2023). S.996 - 2023 Act, 118th Congress. Retrieved from https://www.congress.gov/bill/118th-congress/senate-bill/996