NHS England is to pay for a cutting-edge genetic treatment that will save children with a rare inherited disorder from going blind.
A one-off injection will prevent babies born with poor sight because of an inherited retinal dystrophies disorder from losing their vision entirely, which usually happens during childhood.
The list price is high, at £613,410 per patient, but the NHS has done a deal with Novartis, the UK supplier of voretigene neparvovec, also known as Luxturna, and the numbers needing treatment are low. The company estimates that 86 people in England could benefit now, and about three to five babies with the genetic mutation are born every year.
“Loss of vision can have devastating effects, particularly for children and young people, but this truly life-changing treatment restores the sight of people with this rare and distressing condition,” said NHS chief executive, Simon Stevens, making the announcement at the Health Innovation Expo conference in Manchester.
“For previous generations, curing blindness would literally have been seen as a miracle. Now modern medicine is making that a reality for our patients. Once again the NHS is at the forefront of the genomic revolution with patients in England among the first to benefit from this revolutionary new form of treatment.
“This latest deal reinforces the benefits for companies willing to engage with us and be pragmatic with their pricing – which is good news for patients, taxpayers and industry.”
The National Institute for Healthcare Excellence (Nice) announced it had approved the treatment within 20 weeks instead of the average 38 weeks through its highly specialised technologies programme and with the cooperation of the company.
“This is a highly innovative treatment for a very distressing, inherited condition so we are pleased the company has worked with us and NHS England and NHS Improvement to allow us to offer these families hope for the future,” said Meindert Boysen, director of Nice’s Centre for Health Technology Evaluation.
The treatment is expected to become available in January at three specialist eye centres in the UK.
Stevens has pushed for NHS access to a number of innovative treatments, such as CAR-T therapy for blood cancers where other drugs have failed and the lung cancer drug pembrolizumab. The NHS can afford expensive new drugs for rarer conditions through tough deals and saving money on the more routine medicines, it says, and has cut the annual bill by £700m.
Sue Sharp, deputy chief executive at the Royal Society for Blind Children, said: “We see first hand the devastating effect of childhood sight loss, and so we welcome news of this breakthrough therapy and its impact on the lives of children with inherited retinal dystrophies disorder.”