December 2024 brought a major breakthrough in increasing access to gene therapy. The Biden-Harris administration announced that two drug manufacturers “have entered into agreements with the Centers for Medicare & Medicaid Services (CMS) to participate in the Cell and Gene Therapy Access Model.” This initiative, which involves LYFGENIA and CASGEVY, has the potential to break down barriers that currently limit access for people with sickle cell to life-changing treatments. Particularly, this event marks a pivotal moment in the management of sickle cell disease, by providing a potentially curative option. The Cell and Gene Therapy Access Model is projected to launch in January 2025, aiming to expand access and reduce costs for patients who have Medicaid.
Sickle Cell Disease is a debilitating blood disorder that is statistically known to disproportionately affect Black and Hispanic Americans. With a reduced life expectancy of nearly 20 years, and the increased potential for strokes and chronic organ damage, addressing this condition is absolutely critical. SCD impacts over 100,000 people in the US, making the need to address the issues associated with this disease that much more dire. Currently, patients who undergo treatment for this must endure frequent hospitalizations and significant healthcare costs, leading to insurmountable debt. Furthermore, current treatments are extremely limited, leaving many without effective solutions. However, two recently FDA-approved gene therapies, LYFGENIA and CASGEVY, represent a revolutionary leap forward for effective treatment options. These therapies, which address the root cause of SCD by altering a patient’s genes to produce healthy red blood cells, offer a novel approach to the pool of options that patients were once limited to and the possibility of a cure. Unfortunately, the high costs associated with these treatments pose serious challenges to access, especially for Medicaid patients, who comprise 50%- 60% of the SCD population.
This is where the role of the Cell and Gene Therapy Access model comes into play. This model, led by the Centers for Medicaid and Medicaid Services, utilizes outcomes-based agreements (OBAs) as a way to address these cost and access challenges. This system works by tying costs for SCD treatment to improvements in patient health outcomes. In doing so, this model incentivizes manufacturers to focus on making their therapies effective, while also helping Medicaid programs to better predict and manage budgets, reducing the states’ financial risk. The collaboration between CMS and these states involves many moving parts, including the negotiation of OBAs, additional pricing rebates, and putting forth access policies, CMS will assist in implementing and streamlining this model in the states that opt in to the program.
Looking forward into the near future, the access model provides a springboard for establishing Cell and Gene Therapies as the standard of care, providing renewed hope for the SCD community, and laying the path for similarly gene-editing-based treatment of other life-threatening diseases under development. The inclusion of LYFGENIA and CASGEVY into the CGT access model lays the groundwork for a system that removes financial and logistical barriers. This initiative could ultimately transform lives, giving those who suffered from SCD access to therapies that were once out of their reach.
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