This story was first published in digitalhealth.net
Revolutionary treatment for blindness in children on NHS
NHS England has announced that a revolutionary new gene therapy that restores sight will now be provided and funded by the NHS.
The life-changing treatment for children and adults – voretigene neparvovec – is the first in a new generation of gene therapies that can be directly administered to patients, in this case through an injection. Until now no treatment has been available for babies born with inherited retinal dystophies.
Patients here will now benefit from voretigene neparvovec, also known as Luxturna, after NHS England reached a NICE-endorsed deal with manufacturer Novartis to fund the drug, which costs £613,410 per patient at full price.
It is expected that the agreement will allow up to 100 patients living with a retinal dystrophy to have access to treatment for the first time.
Speaking at the Health Innovation Expo conference in Manchester, NHS chief executive Simon Stevens said: “Loss of vision can have devastating effects, particularly for children and young people, but this is truly life changing treatmecnt restores the sight of people with this rare and distressing condition. 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, tax payers and industry.”
Access is expected to be in place from January 2020, where the treatment will initially be available from three national specialist centres across the UK, with the option to roll-out the treatment to other hospitals.
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