An Examination of the 2024 Medicaid Access and Landscape Review for Sickle Cell Disease: The Renewal of State Medicaid Programs and the Advent of Emerging Gene Therapies
Introduction
Sickle cell disease (SCD) is a group of inherited blood disorders caused by the presence of an abnormal form of hemoglobin known as Hemoglobin S. Individuals with SCD experience acute and chronic complications, including vaso-occlusive crises (VOCs), which may require multiple hospitalizations and result in organ damage. According to the Centers for Disease Control and Prevention, an estimated 100,000 Americans live with SCD, which occurs in about 1 out of every 365 Black or African American births and about 1 in every 16,300 Hispanic American births. It is unfortunate that, since SCD is predominantly among African Americans and Hispanics, the burden of managing a chronic illness while experiencing racial disparities affects access to care and health outcomes. Individuals living with SCD often have considerable unmet health needs, and with the absence of a cohesive policy to address care gaps, health inequities persist.
The field of SCD research has undergone a significant milestone, with two new cellular gene therapies now available for patients aged 12 and older. As these treatment options emerge, assessing the payer’s role and coverage policies nationwide is crucial, focusing on their contribution to ensuring equitable access to SCD therapies. Patients with SCD and their caregivers frequently encounter obstacles that hinder timely access to care and lead to substantial out-of-pocket expenses. Nearly 50% of the approximately 100,000 individuals in the U.S. living with SCD rely on Medicaid.
In line with our dedication to enhancing access and promoting advocacy opportunities, Sick Cells strives to understand which factors decision-makers consider—and disregard—when setting access criteria for SCD therapies. This knowledge contributes to a comprehensive understanding of access issues and supports state initiatives to improve health equity for SCD patients.
To provide context to these debates, Sick Cells conducts an annual Medicaid Access and Landscape Review to explore state and managed care documents that outline coverage criteria and answer three key questions:
- What is the current coverage status in Medicaid programs for available SCD therapies to manage SCD and gene therapies for a curative option?
- How are prior authorization protocols and step therapy requirements used across state Medicaid programs?
- Do Medicaid programs provide adequate clarity and transparency into coverage criteria and decision-making processes?
This full analysis includes an environmental scan of coverage criteria and utilization management under state fee-for-service programs and managed care organizations. These results can serve as a tool for assessment and as the starting point for dialogue and action to achieve improved access to therapies.
SCD Therapies Included in This Analysis
| Trade Name | Product Name | Medical or Pharmacy Benefit | FDA Approval Date |
| Adakveo® | Crizanlizumab-tmca | Medical | November 2019 |
| EndariTM | L-glutamine | Pharmacy | July 2017 |
| Casgevy® | exagamglogene autotemcel (exa-cel) | Medical* | December 2023 |
| Lyfgenia® | lovotibeglogene autotemcel (lovo-cel) | Medical* | December 2023 |
*Note: Gene therapy is restricted to medical benefit in some states. In the analysis, we included all plans with published criteria under medical or pharmacy for Casgevy and Lyfgenia.
Findings and Publications
Findings from this project are published in 4 issue briefs and a final report. Click the images below to view the publications.
- Issue Brief #1: Prior Authorization Refresh for Sickle Cell Disease Therapies in State Medicaid Programs
