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.
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