A new gene therapy which restores sight will be provided by the NHS, chief executive Simon Stevens has announced.
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Babies born with inherited retinal dystrophies disorder have poor sight which swiftly deteriorates, with most losing their vision completely in childhood. Until now no treatment has been available.
The 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.
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 treatment 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, taxpayers and industry.”
Patients in England were amongst the first to benefit from CAR-T therapy, which uses their own immune system to fight cancers, after a deal was done in time to allow fast access.
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.
The agreement was reached after Novartis entered into a licensing and supply agreement with US company Spark Therapeutics, making the therapy available to markets outside of the US.
It is expected that this will allow up to 100 patients living with a retinal dystrophy to have access to treatment for the first time.
The condition blocks messages for making proteins in the eye that are essential for normal vision the drug works by recreating these processes like that in a healthy functioning eye.
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.Sue Sharp, deputy chief executive at the Royal Society for Blind Children, (RSBC) 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."
Investment in technologies and treatments is at the heart of the NHS Long Term Plan. The agreement is the latest in a string of ‘smart deals’ struck to drive value for the taxpayer and benefits for patients.
These include:
- a deal to make ocrelizumab, an innovative multiple sclerosis drug available;
- a deal which could eliminate Hepatitis C;
- a £300 million saving after negotiating deals with five manufacturers on low cost versions of adalimumab;
- striking a full access deal in Europe for CAR-T therapy via the Cancer Drugs Fund, and;
- reaching a deal to make the life-extending lung cancer drug pembrolizumab available for routine use on the NHS
- a deal to make nusinersen available - which targets the underlying cause of spinal muscular atrophy