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The first treatment for sickle cell disease in over 20 years will be rolled out to thousands of patients in England with life-saving benefits, the head of the NHS announced today.
People with the condition endure severe pain during a ‘sickle cell crisis’ that can occur multiple times per year, often requiring hospital admission so they can be given morphine to control the pain and prevent organ failure which can be fatal.
Known as Crizanlizumab, the new drug will be delivered by a transfusion drip and works by binding to a protein in the blood cells to prevent the restriction of blood and oxygen supply that lead to a sickle cell crisis.
Announcing the new treatment, Amanda Pritchard, NHS chief executive, said the drug deal, struck by the NHS, would help as many as 5,000 people over the next three years to have a much better quality of life.
Patients with sickle cell suffer from monthly episodes, making it difficult for people to continue in their jobs or other everyday activities.
The hereditary condition is much more prevalent among people from African or African-Caribbean origin.
The drug will also reduce the number of times a sickle cell patient needs to go to A&E by two fifths.
Last year, NHS England set up ten new dedicated centres to treat sickle cell disease across the country and patients will be able to access the new treatment through their consultant at one of these clinics.
The NHS agreed a deal to make Crizanlizumab available for patients earlier than would have otherwise been possible at a price that is fair for taxpayers.
It is the latest in a line of deals secured by the NHS, including a new cholesterol-busting jab and lung cancer drug last month as well as the world’s most expensive drug which treats spinal muscular atrophy in infants, earlier in the year.
NHS chief executive Amanda Pritchard said: “This is a historic moment for people with sickle cell disease who will be given their first new treatment in over two decades.
“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”.
This announcement paves the way for the National Institute for Health and Care Excellence (NICE) to publish final guidance on Crizanlizumab which will take into consideration the data that will be collected as part of the agreement.
People aged 16 and over who suffer from multiple sickle cell crises, or vaso-occlusive crises as they are scientifically known, per year will be eligible for the treatment.
Toks Odesanmi, a sickle cell patient at Cambridge University Hospitals Trust, said: “Sickle Cell Disease has defined me, defined my body and made a big dent to my dreams.
“No matter how hard I fight it continues to defeat me. A new treatment brings hope and might make dreaming possible again”.
The disease is characterised by unusually shaped red blood cells that are produced which can cause serious health issues across the body, sending organs into crisis and causing extreme levels of pain.
Chair of the Sickle Cell Society, Kye Gbangbola MBA, said: “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”.
The NHS continues to tackle health inequalities and has put in place more than £1 billion of funding into local areas where health inequalities are highest as part of the Long Term Plan.
As well as tackling inequalities in areas such as maternity services and mental health provision, the NHS has also overhauled the way it treats sickle cell disease.
Health and Social Care Secretary Sajid Javid said: “It is fantastic news that this ground-breaking new treatment for sickle cell disease will soon be available in England where it will make a difference to thousands of people’s lives.
“The UK has proven itself to be a world leader in rolling out innovative, life-saving treatments to ensure every NHS patient gets access to the medicines they deserve.
“This is a historic day for those living with this condition and I want to extend my thanks to the National Institute for Health and Care Excellence, NHS England and Novartis for their work to provide this vital treatment to the patients who need it”.
Novartis Oncology President, Susanne Schaffert, said: “Today’s decision is reflective of the new approach to partnerships with healthcare systems that Novartis is taking around the world, to drive equity of access for people living with potentially debilitating conditions such as sickle cell.
“We are pleased that this close partnership with NICE, NHS England and the sickle cell community will enable access to crizanlizumab for those in need of an alternative treatment to help manage their condition and demonstrates the commitment of UK life sciences to ensure access to innovation”.