'Miracle' jab which cures rare form of child blindness to be offered by the NHS, Simon Stevens to announce
Previously no treatment has been available for infants born with inherited retinal dystrophies disorders, which cause poor vision and often result in complete blindness by childhood.
Mr. Stevens, chief executive of the NHS, is expected to announce the revolutionary gene therapy treatment, which costs over £600,000 per patient, at the Health Innovation Expo conference in Manchester.
“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," he is expected to say.
“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.”
Retinal dystrophy is not a single condition but comprises a wide range of disorders such as Retinitis Pigmentosa. Most retinal dystrophies are genetic, caused by a mistake in a patient's genes.
Today’s announcement provides treatment to those with a specific faulty gene, RPE65, which blocks the messages that make proteins in the eye which are essential for normal vision.
It is estimated there are currently up to 100 patients, including children and adults, living with the retinal dystrophy who will be eligible for the new treatment.
Around 3 to 5 babies are expected to be born every year suffering with the condition. In order to undergo the treatment, patients will be tested for the faulty gene and if they test positive they will be eligible.
NHS England reached a deal with manufacturer Novartis to fund the drug, which costs £613,410 per patient at full price.
The treatment, voretigene neparvovec, also known as Luxturna, is administered by a single injection to the back of each eye under the retina, which recreates the protein-making process.
Access is expected to be in place from January 2020, and treatment will initially be available from three national specialist centers 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."
The announcement is part of the NHS Long Term Plan to invest in cutting edge technologies and treatments.
Last year, cancer patients in England were among the first in the world to benefit from “revolutionary” CAR-T therapy.
Mr. Stevens then announced the NHS would offer cancer patients a drug that programs the body to attack rogue cells.
The treatment was hailed by scientists as one of the most significant breakthroughs in cancer for decades.
The Health Innovation Expo conference is the largest NHS-led event of the year, where around 5,000 people from the NHS, social care, local government and the third sector come together to showcase innovative solutions for the nation's healthcare.