NICE is not planning to recommend Casgevy for public reimbursement
UK authorities' decision expected to be finalized May 8
Authorities in the U.K. have preliminarily recommended that Casgevy (exagamglogene autotemcel) not be reimbursed for eligible patients with sickle cell disease (SCD) under its national public health program based on uncertainties about the gene therapy’s cost-effectiveness.
The drafted decision from the National Institute for Health and Care Excellence (NICE) has sparked concern in the SCD communities, who hope it can be reversed before it’s finalized. With the intended decision published, a consultation period will follow where stakeholders and others can submit comments for consideration when NICE convenes on May 8 to finalize its verdict.
The Sickle Cell Society and Anthony Nolan Charity are calling on SCD patients and healthcare professionals to respond to the decision. They’ve provided a fact sheet that explains the decision and includes links where comments can be sent, which must be submitted by April 11. The organizations are also urging one of Casgevy’s developers, Vertex Pharmaceuticals, to work with NICE toward a solution to reverse the decision.
“We aim to make sure the sickle cell community voice is heard, and that NICE is left in no doubt as to the huge impact that sickle cell has on people’s lives,” the organization stated in a press release.
Previously called exa-cel, Casgevy is a one-time gene-editing therapy designed to boost the production of a version of hemoglobin, the protein that’s faulty in SCD, offering a possible cure for the blood disorder. Vertex developed Casgevy with CRISPR Therapeutics.
What goes into a NICE recommendation?
U.K. regulators were the first to approve the therapy last November for SCD patients, ages 12 and older, who have recurrent and painful vaso-occlusive crises (VOCs), and are eligible for a stem cell transplant, but don’t have a suitable donor.
Once a therapy is approved in the U.K., it’s considered for listing through the National Health Services (NHS), a public health program. Listed medications are available at little to no cost for eligible patients. NICE is responsible for recommending if a therapy will be listed through NHS England and if so, who will be eligible for it. That decision is typically also accepted in Wales and Northern Ireland.
As part of its call, NICE considers a treatment’s effectiveness against its cost, which is negotiated between the U.K. government and a therapy’s developer. Like other gene therapies, Casgevy was evaluated by NICE’s highly specialized technologies evaluation committee, which has a higher threshold for determining cost-effectiveness.
The clinical benefits of Casgevy were demonstrated in the Phase 2/3 CLIMB-121 trial (NCT03745287), where nearly all the participants were entirely free of severe VOCs and VOC-related hospitalizations for at least 12 consecutive months, with reported quality of life benefits.
In its draft guidance, however, NICE noted Casgevy’s benefits weren’t certain, since it wasn’t compared with any other treatment in the open-label trial and the analysis only involved 30 treated patients. It also noted that it isn’t yet clear how well it will work long term and if its effects will wane. Plus, economic analyses indicate the price of Casgevy is higher than what NICE normally deems cost-effective for routine use.
While the negotiated price of the gene therapy in the U.K. hasn’t been disclosed, it’s among the most expensive therapies in the world, with a list price in the U.S. hovering around $2.2 million.
Vertex has indicated Casgevy still offers great value by reducing patients’ other lifetime costs associated with managing the disease.
Nevertheless, the committee notes it “requires more information to address uncertainties in the clinical and economic evidence,” before recommending Casgevy be listed through the NHS. For the decision to be overturned, Vertex will need to use the feedback period to provide NICE with additional information.
At the time of its clearance for SCD, Casgevy was also approved for transfusion-dependent beta-thalassemia, a related blood disorder. NICE is considering the therapy for that indication separately.
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