NHS approves 'transformative' sickle cell treatment in England
Four thousand people diagnosed with sickle cell disease in England may now access 'life-changing' treatment following its approval for NHS use
Four thousand people diagnosed with sickle cell disease in England may now access "life-changing" treatment following its approval for NHS use.
This treatment, known as voxelotor or oxbryta, and manufactured by Pfizer, is hoped to enhance the quality of life for those affected by the condition.
People with sickle cell disease produce unusually shaped red blood cells which can cause problems because the blood cells do not survive long as healthy blood cells and can block blood vessels.
The drug works by helping haemoglobin (a protein in red blood cells) hold on to more oxygen and prevents red blood cells from becoming misshapen.
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Around 17,500 people in England have sickle cell disease. The condition can cause patients multiple periods of intense pain a year – known as a crisis – due to blood clotting in their arteries.
The condition is more common among people with an African or Caribbean family background.
The new treatment helps a crucial protein in red blood cells to retain more oxygen, which prevents the deformation of red blood cells.
It was initially rejected for widespread NHS use but the National Institute for Health and Care Excellence (Nice) has approved the drug in final draft guidance after a reduction in its price.
Patients have told the society that the drug has helped to reduce pain and improve emergency levels.
The Sickle Cell Society welcomed the news, saying the drug has “transformed” the lives of people with the condition with improvements found in both physical and mental health.
John James, chief executive of the Sickle Cell Society, said: “Users of voxelotor have shared with us remarkable stories of improved quality of life, reduced pain and substantial increases in energy levels.
"This has allowed them to work, connect with friends and family, improve their physical and mental health – and generally achieve a quality of life the rest of us often take for granted. “We have seen decades of underinvestment in better care and safe effective treatments for sickle cell."
Initially, voxelotor faced NHS rejection due to cost concerns.
But the National Institute for Health and Care Excellence (Nice) approved the drug in final draft guidance after a reduction in its price.
Amanda Pritchard, chief executive of NHS England, said the service had “worked hard to make this life-changing treatment available".
She added: “Sickle cell disease can have a huge impact on people’s lives, and this is one of the biggest breakthroughs in treatment in recent decades.
"I know how much this announcement will mean for thousands of people across the country.”
Health Minister Andrew Stephenson said: “This is hugely welcome news that will help tackle longstanding health inequalities experienced by this community.”
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