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According to a new report, the ongoing battle against sickle cell disease may soon be over thanks to a newly-approved drug treatment that is to be made available on the NHS.
The drug, known as crizanlizumab, is the first new treatment for the disease in 20 years after the National Institute for Health and Care Excellence (Nice) made the recommendation. It will only be made available to around 300 patients to start with, but will be extended to 450 in future years.
Sickle cell patients over the age of 16 will be eligible for treatment, although Nice said they could not recommend the drug for routine use yet because of uncertainty about long-term effectiveness, side-effects and the associated costs. As the drug begins to roll-out, Nice have pledged to continue to collect more data through clinical trials.
A lifelong condition, sickle cell disease disproportionately affects people with an African or Caribbean background and causes severe pain and organ failure, often keeping sufferers in and out of hospital throughout their lives. Up until now, the only option was to manage symptoms with drugs like hydroxycarbamide, but according to the NHS, the new drug could help as many as 5,000 people over the next three years.
It’s also said that it will help reduce the number of hospital admissions in sickle cell patients by as many as two fifths. Crizanlizumab will be administered via transfusion drip and works by binding to a protein in the blood cells to prevent the restriction of blood and oxygen supply.
“This is a historic moment for people with sickle cell disease who will be given their first new treatment in over two decades,” said Amanda Pritchard, NHS England’s chief executive. “This revolutionary treatment will help to save lives, allow patients to have a better quality of life and reduce trips to A&E by almost half. The NHS has agreed a deal for this drug, so we are able to provide the latest and best possible treatments for patients at a price that is affordable for taxpayers.”
Sickle Cell Society chair Kye Gbangbola added: “A new treatment brings new hope for people living with sickle cell disorder, the world’s most common genetic blood condition. SCD is a medical emergency, it causes excruciating pain, this new treatment will reduce the number of agonising pain episodes we have to endure. The hope is improved quality of life for many living with the condition and their families.”