Oxfordshire woman with sickle cell disorder 'devastated' after new treatment refused for use on NHS
ITV News Meridian's Natalie Verney reports.
A draft decision by the National Institute for Health and Care Excellence (NICE), to reject the use of a gene therapy as a treatment for sickle cell disorder, has been branded disappointing by campaigners.
A new treatment, branded exa-cel, involves modifying a patient’s own stem cells to treat sickle cell.
It was approved by the medical regulator MHRA in November for use in the UK as a treatment for sickle cell, followed by FDA approval in the US in December.
However, NICE says it cannot recommend the new treatment as it needs further evidence to prove its effectiveness.
For patients like Funmi Dasaolu from Oxfordshire, the news is disappointing. She currently suffers from a range of symptoms caused by the disorder.
“From simple things like trying to hold down a job, to relationships, to work, social activities, everything is impacted”, she said.
“I think because the condition is so unpredictable, so you can wake up in the morning feeling fine and then midday you're like actually my leg's on fire and I need to be admitted.
“Ache, stabbing, dull, crushing, it's just indescribable. Even when you go to the hospital they will ask you what's your pain out of ten, normally it's off the charts, like, I don't know, twelve or fifteen out of ten.
“They might give you morphine, morphine doesn't really work.”
Watch: Funmi Dasaolu tells describes the effect sickle cell disorder has on her life.
Sickle cell disease is an inherited blood disorder that causes red blood cells in the body to be an unusual crescent shape.
They have a shorter lifespan compared to normal red blood cells. That unusual shape means they don’t move smoothly through blood vessels and can actually block them and cause major problems.
The condition mainly affects people of African and Caribbean descent. Until recently, only a stem cell transplant could be used as a potential cure for a patient, but, like Funmi, very few people are eligible.
Anthony Nolan and the Sickle Cell Society are urging NICE and pharmaceutical company Vertex to find a solution, so this provisional decision can be reversed without delay.Caitlin Farrow, Director of Strategy and Influencing at Anthony Nolan, said: “We are deeply disappointed with this decision. Sickle cell is one of the most common genetic conditions impacting people in England from predominantly African and African-Caribbean backgrounds, and this community has been waiting years for a new, effective treatment.
“There is a longstanding history of the experiences of people with sickle cell not being taken seriously.
"It’s not fair that the community is being asked yet again to explain the severely negative impact this disease has on their lives.
"We urge NICE and Vertex to find a solution to deliver this much-needed treatment to patients as soon as possible.”
In response, NICE told ITV Meridian it would like Vertex to further consider how it could collection additional data to address clinical uncertainties.
Helen Knight, director of medicines evaluation at NICE, said: “The appraisal committee considered that exa-cel could represent a potential cure for some people with SCD, freeing people from the burden of VOCs as well as addressing NICE’s aim of reducing health inequalities associated with the condition.
“As such, and acknowledging the limitations with the evidence, NICE is willing to accept higher than the usual maximum for assessing cost-effectiveness.
“NICE and Vertex are ready to work together to address the issues highlighted in this draft guidance and in particular explore how the uncertainty in the evidence could be addressed through an updated managed access proposal.
"This would allow people to have treatment with exa-cel while more evidence is collected.”
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