NHS Rolls Out £1.7 Million Gene Therapy for Sickle Cell Disease
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The one-time gene-editing therapy Casgevy, developed by Vertex Pharmaceuticals Inc. and Crispr Therapeutics AG, has a list price of £1.7 million ($2.1 million), but England's drug cost regulator said it has negotiated a lower, undisclosed price.
About 1,700 people could be eligible for the therapy, which aims to repair genetic code, although it's likely only about 50 patients a year will receive it given the complexity of the treatment.
Sickle cell disease affects the red blood cells and causes extreme pain, organ damage and can shorten a patient's life. Only some patients with the disease will be eligible to receive Casgevy.
The treatment is designed to work by editing the faulty gene in a patient's bone marrow stem cells. The cells are taken out of the bone marrow, edited in a laboratory and infused back into the patient — after which the results have the potential to be life-long. Since it's discovery more than a decade ago, Crispr technology has been expected to bring about a revolution in medicine.
The decision announced Friday reverses an earlier rejection in draft guidance from the regulator. The treatment will be rolled out under a so-called managed access program, where more data is collected to help the regulator — the National Institute for Health and Care Excellence — understand the longer-term benefits before it re-assesses the drug.
England has a stringent process to assess the cost-effectiveness of new medicines, and NICE decides which drugs will be available free to patients on the NHS. With the health service under growing financial pressure, the regulator has faced recent criticism over its refusal to accept some cancer medicines.
Still, the UK was the first country to approve Casgevy in 2023 and the treatment is already available to a smaller group of patients with a serious form of beta-thalassemia, a hereditary blood disorder that requires repeated transfusions.
The treatment for sickle cell requires chemotherapy, meaning it's likely only patients with the most severe and progressive problems will benefit, said David Rees, professor of pediatric sickle cell disease at King's College London. Despite his concerns, he said it 'offers a really important new treatment option.'
'Excitingly, advances in gene editing are happening very rapidly at the moment and it seems likely that cheaper, safer and more effective forms of gene editing will emerge for sickle cell disease over the coming years,' he said.
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