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NHS to offer ‘life-changing’ treatment for sickle cell disease

The new drug could reduce the need for blood transfusions and ensure a ‘higher quality of life’ for patients with sickle cell disease

NHS England is set to roll out a new treatment for sickle cell disease which could be ‘life-changing’ for people living with the condition.

The drug, Voxelotor, is the first sickle cell treatment approved by the National Institute for Health and Care Excellence (NICE) for routine use in the NHS. It is administered as a daily tablet to people with the disease aged 12 and over and is said to reduce their need for blood transfusions, leading to fewer hospital visits.

NHS chief executive Amanda Pritchard called this one of the ‘biggest breakthroughs in treatment,’ which could benefit thousands of people living with the condition.

‘By improving the quality of life and reducing the need for hospital care, this new treatment option also has the potential to free up doctors, nurses and other clinicians to better support other patients, so we are acting fast to get it to the frontline immediately through our Innovative Medicines Fund (IMF),’ she said.

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Sickle cell disease arises when healthy, oxygenated, O-shaped red blood cells lose oxygen via an abnormal haemoglobin protein, causing the cells to turn C-shaped, resembling a sickle.

The cells then become hard and sticky and clog up blood vessels. This can cause severe pain and lead to frequent hospitalisation, delayed growth, strokes and lung problems.

In England, there are around 17,000 people living with sickle cell disease. It is more common in people of Black African, Caribbean, Middle Eastern and South Asian heritage.

Healthcare leaders have welcomed the availability of the new drug in significantly improving the lives of people living with the disease.  

Professor Bola Owolabi, director of the National Healthcare Inequalities Improvement Programme at NHS England, said: ‘We are proud to make this new treatment available on the NHS – it could help thousands of patients living with sickle cell disease across the country to have a higher quality of life and experience fewer side effects, and represents a significant step forward in addressing the healthcare inequalities experienced by some of our communities.’

John James, the chief executive of the Sickle Cell Society, said that the decision by NICE has ‘brought a great deal of hope, and we are profoundly grateful that this day has arrived. It is a deeply life changing and celebrated moment for people living with the condition.’