Breaking Down the Cost of Sickle Cell Disease Gene Therapies
As CMS attempts to navigate the astronomical costs of new sickle cell disease treatments, including gene therapy, it is critical to break down treatment costs.
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- Earlier this week, the Biden–Harris administration announced its plans to address the expensive price tag of sickle cell disease, looking for a way to widen access despite the cost. In an announcement by the United States Centers for Medicare and Medicaid Services (CMS), the organization noted that sickle cell disease will be the first focus of the Cell and Gene Therapy (CGT) Access Model.
“Medical advancements are bringing us closer to a cure for sickle cell disease (SCD). Yet, many of the more than 100,000 Americans with SCD today face difficulty accessing effective health care and groundbreaking treatments,” said HHS Secretary Xavier Becerra in the press release. “Our CGT Access Model gives us a chance to streamline a cumbersome process and put transformative cell and gene therapies within reach for Americans with sickle cell disease.”
According to the American Academy of Family Physicians, the list price of Casgevy, the first and only CRISPR-based gene editing therapy for SCD and transfusion-dependent beta thalassemia (TDT), is approximately $2.2 million.
Comparatively, the lifetime cost of treating SCD is roughly $1.7 million, and the cost of treating TDT is approximately $5–5.7 million.
Theoretically, a one-time gene editing treatment helps alleviate some of the long-term financial burdens, including the cost of treatment, travel, and lost time or productivity for treatment. Additionally, a singular treatment may reduce the treatment burden on patients.
However, the cost of singular treatment deters many eligible patients from pursuing this therapy.
This barrier is not exclusive to Casgevy; many other cell and gene therapies have an unaffordable price tag, limiting patient access. For example, Hemgenix, a gene therapy for hemophilia B, is listed at $3.5 million. Additionally, Skysona, which treats active cerebral adrenoleukodystrophy, is listed at $3 million.
These examples prove that the high cost of treatment impacts nearly all cell and gene therapies. Without sufficient insurance coverage for these treatments, patients are left pursuing less effective treatment options.
As more cell and gene therapies come to market, developing feasible payment models should be a priority to ensure all patients, not just those with millions of dollars in their pockets, can access care.
“Gene therapies for sickle cell disease have the potential to treat this devastating condition and transform people’s lives, offering them a chance to live healthier and potentially avoid associated health issues,” added CMS Administrator Chiquita Brooks-LaSure. “Increasing access to these promising therapies will not only help keep people healthy, but it can also lead to savings for states and taxpayers as the long-term costs of treating sickle cell disease may be avoided.”
