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“Challenges in Providing Equitable Access to Costly Gene Therapies for Sickle Cell Disease”

Challenges in Providing Equitable Access to Costly Gene Therapies for Sickle Cell Disease

The approval of two gene therapies to treat sickle cell disease has brought hope to patients suffering from this debilitating disease, which disproportionately affects Black people and people of color. However, health officials now face the challenge of ensuring equitable access to these costly treatments. The high cost and lack of infrastructure to administer the therapies have raised questions about how many people will actually benefit from them.

Living with sickle cell disease is a constant struggle for patients like Michael Goodwin. The unpredictable episodes of pain caused by this genetic blood disorder have forced Goodwin to leave his job and spend a significant amount of time in the hospital. As a result, he is hesitant to try the new gene therapies, which require months of intensive medical preparation and come with a hefty price tag. Vertex Pharmaceuticals’ Casgevy therapy costs $2.2 million, while Bluebird Bio’s Lyfgenia treatment is priced at $3.1 million.

Goodwin’s concerns are shared by many patients and healthcare professionals. Dr. Julie Kanter, director of the Adult Sickle Clinic at the University of Alabama at Birmingham, believes that even if the therapies were made available to everyone, only a small percentage of those affected by sickle cell would choose to undergo the treatment. The lack of infrastructure and high cost make it difficult to manage the demand for these therapies.

According to the Centers for Disease Control and Prevention, more than 100,000 Americans have sickle cell disease, with 50% to 60% of them covered by Medicaid. As state and federal officials grapple with providing access to these costly treatments, they are also exploring new payment models. The Biden administration is set to negotiate with Vertex and Bluebird Bio to obtain discounts for state Medicaid plans, with payments linked to patient health outcomes. This initiative aims to make high-priced treatments more accessible.

The negotiations with the drugmakers come at a time when large pharmaceutical companies are suing the Biden administration over Medicare price negotiations. However, Vertex executives express confidence in the negotiation process for sickle cell treatments. They believe that discussions with individual state Medicaid agencies will ensure wide access and address long-standing inequities in care for the sickle cell disease community.

Private employer health plans are also facing challenges in covering the rising costs of these novel treatments. Many employers are considering carving out cell and gene therapies from their benefits due to their high costs. Morgan Health, a firm specializing in workplace health programs, is exploring new risk-sharing payment models to help small- and medium-sized businesses cover these expenses.

While the approval of gene therapies for sickle cell disease brings hope to patients, the challenges of providing equitable access and managing the high costs remain. Efforts are being made to negotiate lower prices and develop payment models that ensure wider access to these life-changing treatments. As discussions continue, patients like Michael Goodwin hope for a future where they can receive these therapies without the burden of high costs and limited access.


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