First new treatment for sickle cell in 20 years
A "revolutionary" life-changing drug treatment for sickle cell disease is to be made available on the NHS – the first new treatment for the disease in 20 years.
It will initially only be made available to around 300 patients but will be extended to 450 in future years after the decision by the National Institute for Health and Care Excellence (Nice), which recommended the drug be available on the NHS.
The new drug, known as crizanlizumab, will be delivered by a transfusion drip and works by binding to a protein in the blood cells to prevent the restriction of blood and oxygen supply.
It could help as many as 5,000 people over the next three years, the NHS has said. It is hoped the drug will reduce the number of times a sickle cell patient needs to go to A&E by two fifths.
People aged over 16 who suffer from multiple sickle cell crises every year will be eligible for the treatment. It could not recommend the drug for routine use yet due to high uncertainty about the long-term effectiveness of the treatment and its associated costs. The agreement with the NHS will allow patients to access the drug while extra data is collected through clinical trials.