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 who are 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 by helping to reduce their need for blood transfusions, which can then lead to having fewer hospital visits.
The condition is more prevalent in people with an African or Caribbean family background, according to the NHS. In England, about 17,000 people are living with the disease. Of those, 4,000 are believed to be eligible for the new treatment.
The drug’s approval has been welcomed by charities and organisations that work closely with people living with sickle cell disease.
Charles Kwaku-Odoi, the chief executive of the Caribbean and African Health Network (CAHN), said: “On behalf of people living with Sickle Cell and the Black community as a whole, we are absolutely delighted that this treatment has been approved by Nice. This treatment offers significant hope of better quality of life to those that suffer from this often-debilitating condition. At CAHN, we continue to advocate and amplify patient voices when addressing systemic inequities for a community that is underserved.”
John James, the chief executive of the Sickle Cell Society, said that the decision by Nice has brought “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.”
Sickle cell disease changes the shape of blood cells into crescents, hindering blood flow. Sufferers experience severe painful episodes, which can require hospital admission. Life expectancy can be 20 to 30 years shorter than in the general population.
Helen Knight, director of medicines evaluation at Nice, said: “This is great news for people with sickle cell disease, particularly given the health inequalities experienced by people with the condition.
“Throughout our appraisal of Voxelotor the appraisal committee has always considered that it has the potential to address the need for effective treatments for sickle cell disease, as well as address Nice’s aim of reducing health inequalities.”
Prof 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.
“It is vital that we continue to get new drugs into the hands of NHS clinicians to improve the lives of people living with sickle cell disorder.”
