The approval of two groundbreaking gene therapies to treat sickle cell disease has brought hope to patients suffering from this debilitating condition. However, the high cost and logistical challenges associated with these treatments are raising concerns about equitable access for all patients. In this article, we will explore the challenges of affordability for these gene therapies and the efforts being made to ensure access for those in need.
Sickle cell disease predominantly affects Black people and people of color, making it a pressing issue of racial health disparities. Patients like Michael Goodwin have experienced the unpredictable and painful episodes caused by this genetic blood disorder, forcing them to leave their jobs and spend significant time in the hospital. Goodwin, like many others, is hesitant to pursue the new gene therapies due to the intensive medical preparation required and the high cost of treatment.
One gene therapy, Casgevy by Vertex Pharmaceuticals, lists at $2.2 million, while another treatment called Lyfgenia by Bluebird Bio is priced at $3.1 million. While insurance coverage may alleviate some of the financial burden, patients like Goodwin already face substantial medical bills and are wary of taking on additional costs.
Dr. Julie Kanter, director of the Adult Sickle Clinic at the University of Alabama at Birmingham, explains that even if access to these therapies were opened up to all patients, only a small percentage would likely opt for the treatment. The lack of infrastructure and capacity to administer the therapies on a large scale is a significant hurdle that needs to be addressed.
According to estimates from the Centers for Disease Control and Prevention, over 100,000 Americans are living with sickle cell disease, with 50% to 60% of them covered by Medicaid. State and federal officials are now faced with the challenge of providing access to these costly treatments through the Medicaid safety net program. Kate McEvoy, executive director of the National Association of Medicaid Directors, points out that state budgets alone cannot manage the high cost of these therapies.
A study conducted by the University of Washington suggests that a price point closer to $1 million would ensure greater access to the gene therapies while offsetting the lifetime medical and quality-of-life costs for patients with acute sickle cell disease. To address the cost issue, the Biden administration is launching negotiations with Vertex and Bluebird Bio to obtain discounts for state Medicaid plans. These negotiations will be linked to patient health outcomes, ensuring that payment is tied to the effectiveness of the treatments.
The Biden administration’s Cell and Gene Therapy Access Model aims to make high-priced treatments more accessible by implementing a new payment demonstration program starting in January 2025. This program was initially planned for a later date but was moved forward due to the approval of the sickle cell gene therapies. McEvoy emphasizes the importance of developing strategies to support Medicaid programs’ capacity to cover these treatments, especially considering the numerous therapies in the pipeline awaiting FDA approval.
Despite ongoing discussions regarding payments, pharmaceutical companies like Vertex and Bluebird Bio are taking steps to educate doctors and patient communities about the benefits of their gene therapies. They are working towards ensuring wide access and addressing long-standing inequities in care for the sickle cell disease community. Vertex is confident about the negotiation process, expressing its commitment to securing access for Medicaid-covered patients.
The challenges of affordability are not limited to government programs like Medicaid. Private employer health plans are also facing difficulties in covering the rising costs of these novel treatments. Smaller companies, in particular, may struggle to afford specialty treatments with seven-figure price tags. Morgan Health, a company specializing in workplace health programs, is exploring new risk-sharing payment models to help businesses cover these costs.
For patients like Goodwin, the decision to pursue these gene therapies is not an easy one. Despite the high cost and intensive medical preparation required, he would be willing to undergo treatment if guaranteed a cure for his sickle cell disease. While efforts are being made to address the challenges of affordability and access, there is still a long way to go in ensuring that groundbreaking gene therapies are available to all who need them.
In conclusion, the approval of gene therapies for sickle cell disease has brought hope to patients, but the challenges of affordability and access remain significant hurdles. Efforts are underway to negotiate discounts for Medicaid plans and develop payment models based on patient outcomes. However, the high cost of these treatments poses challenges for both government programs and private employer health plans. While progress is being made, there is a need for continued efforts to ensure that these groundbreaking therapies are accessible to all patients in need.
