A “revolutionary” life-changing drug treatment for sickle cell disease is to be made available on the NHS – the first new treatment for the disease in 20 years.
It will initially only be made available to around 300 patients but will be extended to 450 in future years after the decision by the National Institute for Health and Care Excellence (Nice), which recommended the drug be available on the NHS.
Sickle cell disease – which is particularly common in people with an African or Caribbean background – is a serious and lifelong health condition causing severe pain and organ failure often requiring hospital admissions.
The new drug, known as crizanlizumab, 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.
It could help as many as 5,000 people over the next three years, the NHS has said. It is hoped the drug will reduce the number of times a sickle cell patient needs to go to A&E by two fifths.
People aged over 16 who suffer from multiple sickle cell crises every year will be eligible for the treatment.
Nice said it could not recommend the drug for routine use yet due to high uncertainty about the long-term effectiveness of the treatment and its associated costs. The agreement with the NHS will allow patients to access the drug while extra data is collected through clinical trials.
Sufferer Adeola Omoyele, aged 28, from London, told The Independent from hospital: “It’s a Catch-22 situation. It’s good that at least some people, at least, will have the extra treatment. But it’s quite sad that it’s been 20 years since a new treatment drug for sickle cell and now only a few people will benefit from it. More needs to be done for the benefit of all.
“When you look at the disparities between other illnesses, such as cancer, more research goes into those than sickle cell which predominantly affects black people. Issues around lack of awareness around sickle cell, shortage of funding and resources and poor care for patients need to be addressed.”
Laurel Brumant, aged 57, from London, was diagnosed with sickle cell disease at the age of three.
The children’s author told The Independent: “The fact that it’s something new for sickle cell sufferers and it will benefit someone out there who really needs it is positive. There are a lot of people with sickle cell disease out there who are going to want access to this treatment. I wonder how doctors will make the decision of who to give it to given that so many people have the condition?”
Her symptoms were managed with another drug called hydroxycarbamide for 10 years – before then she would suffer frequent crises accompanied by pain she described as “out of this world”. She said she hoped the new drug would provide relief for those who need it.
“If you’d offered me any medication that could stop it, and give me better quality of life, I would have taken it,” she said.
Hayley King, founder of Cianna’s Smile, a charity which supports and educates families about sickle cell anaemia in the UK, said: “We are extremely excited and relieved that finally a new treatment has been developed and will be available on the NHS for those affected by sickle cell in the UK.
“Seeing developments in treatment is extremely reassuring. Sickle cell is the fastest growing genetic condition in the world and this seems long overdue. We hope the other treatments that have already been approved by the FDA [United States Food and Drug Administration] will also be approved by Nice and become available on the NHS as soon as possible.
“This will give people options and the opportunity to reduce the complications and symptoms they experience and the life challenges many people with sickle cell face.”
NHS England’s 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.”
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.”