NICE says “not so fast” to Vertex’s sickle cell disease CRISPR therapy

2024-03-14

基因疗法临床研究上市批准

The UK recently became the first country in the world to approve a CRISPR-based gene-editing therapy, but the National Institute for Health and Care Excellence has said it now needs more evidence before making it routinely available on the NHS.

Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy (exagamglogene autotemcel) gained clearance from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) last year for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassaemia (TDT). NICE is still assessing the therapy for TDT, while it published draft guidance in SCD on Thursday.

In its documents, NICE said that although study results suggest that Casgevy can stop vaso-occlusive crises (VOCs) in some people with SCD, the trial was limited by its small size and lack of comparator arm. The cost-effectiveness body added that “it was not clear how well it will work in the longterm.”

Along with questions on efficacy, NICE raised concerns over Casgevy’s price, which it said “is higher than what [it] normally considers to be a cost-effective use of NHS resources.” The agency added that even when taking into account the innovative nature of the technology, the cost-effectiveness estimate for Casgevy is still above its threshold.

More to come.

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