NHS to fund revolutionary treatment for blindness in children

A revolutionary new gene therapy which restores sight will be provided by the NHS, chief executive Simon Stevens has announced today.

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 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.

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, tax payers and industry.”

Patients in England were amongst the first in the world 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 of the announcement:  “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 world-class, cutting edge 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 ground-breaking deal which could eliminate Hepatitis C;
  • a £300 million saving after negotiating deals with five manufacturers on low cost versions of the health service’s most costly drug, adalimumab;
  • striking the first full access deal in Europe for CAR-T therapy via the Cancer Drugs Fund, which can potentially cure some children and adults with blood cancers where other treatments have failed, 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 – the first treatment that targets the underlying cause of spinal muscular atrophy

The NHS is already one of the most efficient health services in the world but the Long Term Plan committed to go even further in freeing up funds for frontline care and cutting edge treatments.

The NHS has already saved more than £700 million from the annual medicines bill to reinvest in new treatments, such as voretigene neparvovec.

An NHS-wide campaign has supported patients and doctors to maximise the use of ‘generic’ and best value ‘biologic’ treatments to treat conditions including arthritis and cancer.

New figures show that the uptake of best value medicines lowered costs to taxpayers by £294 million last year alone, coming on top of the £413 million saved from the annual medicines bill in the previous two years, meaning the NHS is on track to meet its ambitious target of a further £400 million annual savings by 2021.