In the realm of cell and gene therapy, scientific advancements are moving at a rapid pace, outstripping the ability to bring these innovative treatments to market. One notable example is the recent approval of the first two sickle cell disease gene therapies by the Food and Drug Administration: Vertex’s Casgevy (exagamglogene autotemcel) and bluebird bio’s Lyfgenia (lovotibeglogene autotemcel). Despite receiving marketing authorization in December 2023, uptake of these therapies has been slow, with only a small number of patients initiating treatment by August of the following year.
Recognizing the need to improve patient access to these groundbreaking therapies, the Biden Administration announced that the manufacturers of Lyfgenia and Casgevy have signed agreements with the Centers for Medicare and Medicaid Services (CMS) to participate in the Cell and Gene Therapy Access Model. This innovative model allows CMS to negotiate outcomes-based agreements on behalf of state Medicaid programs for cell and gene therapies, starting with sickle cell disease treatments. If successful, this model could serve as a blueprint for improving access to other cell and gene therapies facing similar challenges.
Sickle cell disease is a debilitating condition that affects millions of individuals worldwide, with approximately 100,000 people living with the disease in the United States alone. Characterized by abnormal red blood cells that can lead to severe pain, anemia, organ damage, and infections, sickle cell disease significantly impacts the quality of life and life expectancy of those affected, particularly individuals of sub-Saharan African descent.
While traditional treatments for sickle cell disease focus on managing symptoms and complications, such as pain medications and antibiotics, the approval of Lyfgenia and Casgevy represents a significant leap forward in potential treatment options. These novel therapies have the potential to decrease or even eliminate painful crises in patients, offering hope for improved outcomes and quality of life.
However, the high costs of these therapies, priced at $2.2 million for Casgevy and $3.1 million for Lyfgenia, present a significant barrier to access for many patients. In addition to financial challenges, cell and gene therapy manufacturers face regulatory hurdles, manufacturing complexities, and limited patient populations, all of which contribute to the difficulties in bringing these therapies to those in need.
The outcomes-based agreements established through the Cell and Gene Therapy Access Model represent a promising avenue for improving patient access to these transformative treatments. By linking payments to the actual health outcomes achieved by patients, these agreements aim to ensure that patients receive the benefits of these therapies while providing a more sustainable payment model for payers.
As the model prepares to launch next month, stakeholders are optimistic about the potential impact it could have on improving patient access to cell and gene therapies, starting with sickle cell disease treatments. By addressing the challenges of affordability, regulatory compliance, and patient access, the Cell and Gene Therapy Access Model is poised to revolutionize the landscape of healthcare delivery for these innovative treatments.
