‘Life-changing’ drug for sickle cell disease is being offered by NHS in England
A new treatment for sickle cell disease has been recommended by the health watchdog, in a move described as life-changing for people living with the condition in England.
The drug, Voxelotor, has been recommended for use on the NHS in England by the National Institute for Health and Care Excellence (Nice) in people with the disease aged 12 and over.
The drug, administered as a daily tablet, is said to significantly improve the quality of life for people with sickle cell disease by helping to reduce their need for blood transfusions, which could then lead to fewer hospital visits.
The condition is more common in people with an African or Caribbean family background, according to the NS. About 17,000 people in England are living with the disease. Of these, 4,000 would be eligible for the new treatment.
The drug’s approval has been welcomed by charities and organizations that work closely with people with sickle cell disease.
Charles Kwaku-Odoi, the CEO of the Caribbean and African Health Network (CAHN), said: “On behalf of people living with sickle cell disease and the Black community as a whole, we are absolutely delighted that this treatment has been approved by Nice. . This treatment offers significant hope for a better quality of life for those suffering from this often debilitating condition. At CAHN, we continue to advocate and amplify patient voices in addressing systemic inequities for an underserved community.”
John James, the CEO of the Sickle Cell Society, said the Nice decision “has brought a lot of hope, and we are extremely grateful that this day has arrived. It is a profoundly life-changing and celebrated moment for people living with the condition.”
Sickle cell disease changes the shape of blood cells into crescents, impeding blood flow. Patients experience severe painful episodes, which may require hospitalization. Life expectancy may be 20 to 30 years shorter than in the general population.
Helen Knight, director of drug evaluation in Nice, said: “This is great news for people with sickle cell disease, especially given the health inequalities experienced by people with the condition.
“Throughout our review of Voxelotor, the review committee consistently considered that it has the potential to meet the need for effective treatments for sickle cell disease, and to meet Nice’s goal of reducing health inequalities.”
Prof. Bola Owolabi, Director of the National Healthcare Inequalities Improvement Program at NHS England, said: “We are proud to be making this new treatment available on the NHS – it could help thousands of patients with sickle cell disease across the country to get a to have a higher risk. quality of life and experience fewer side effects, and represents an important step forward in addressing the healthcare inequities faced by some of our communities.
“It is vital that we continue to get new medicines into the hands of NHS clinicians to improve the lives of people with sickle cell disease.”