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A £1.7mn treatment for the rare blood disorder beta thalassemia will be given to NHS patients in England after the country’s healthcare spending watchdog approved the first therapy using Crispr gene editing technology in Europe.
The National Institute for Health and Care Excellence’s decision to recommend Casgevy, while continuing to gather data on the drug’s cost-effectiveness, shows how it is assessing innovative gene therapies that come with big upfront costs but could permanently cure patients.
Beta thalassemia limits the production of healthy red blood cells and haemoglobin, which carry oxygen around the body. It can lead to growth and bone problems. Those who suffer from it severely require regular blood transfusions.
Casgevy, developed by Vertex Pharmaceuticals and Crispr Therapeutics, works by removing a patient’s cells through a transfusion, editing them so they can produce healthy red blood cells, and re-infusing the cells back into the patient.
The treatment is based on Crispr, a gene-cutting tool whose potential to tweak and delete genetic errors responsible for health disorders was discovered by Nobel laureates Jennifer Doudna and Emmanuelle Charpentier in 2012.
The UK Medicines and Healthcare products Regulatory Agency became the world’s first regulator to approve Casgevy on grounds of safety and effectiveness for sickle cell disease and beta thalassemia in November 2023. Nice takes decision on the cost-effectiveness of drugs for the NHS.
The MHRA was closely followed by its EU and US counterparts, the European Medicines Agency and the US Food and Drug Administration.
Nice’s announcement on Thursday means up to 460 NHS patients with severe beta thalassemia will be eligible for the drug where a blood and bone marrow transplant is suitable but donors are not available. Sufferers in the UK are mainly of Pakistani, Indian and Bangladeshi ethnic origin.
In May, the spending watchdog delayed a decision on approving Casgevy for treating sickle cell disease, citing a need for more information from Vertex.
The inherited blood disorder affects about 17,000 people in England and is more common in people of Black African, Caribbean, Middle Eastern and South Asian heritage, according to NHS England.
Prescribing Casgevy will be costly: it has a list price of £1,651,000 per course of treatment, although the NHS and Vertex have a confidential commercial agreement to provide the drug at a discount.
But in clinical trials, most patients with the disease did not require further transfusions after being treated with the drug.
Helen Knight, director of medicines evaluation at Nice, said Casgevy “could represent a potential cure for some people with transfusion-dependent beta-thalassaemia, freeing them from the burden and risks of needing regular blood transfusions”.
The initiative will be funded through the Innovative Medicines Fund, a ring-fenced pot of NHS funding set up under Boris Johnson’s government to provide expensive new medicines on the NHS while gathering more data on their cost-effectiveness.
James Davies, associate professor of genomics at Oxford university, said Casgevy marked “a completely new way of treating human disease” and had “the potential to be used for treating many other genetic diseases, so this is a really important approval”.
The drug is already available in the US, while France has approved it under an early access scheme but has yet to agree pricing.
Yasmin Sheikh, head of policy and public affairs at stem cell charity Anthony Nolan, welcomed Nice’s approval of Casgevy but said the “groundbreaking therapy must also be funded for people with sickle cell, where it has huge promise and is desperately needed”.
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