25 October 2017


The NHS is to fund infants to travel to Italy to receive gene therapy for a form of severe combined immune deficiency, it was announced today.

Each treatment in Milan will cost 594,000 Euros – but should only need to be administered once, the National Institute for Health and Care Excellence said today.

The gene therapy, known as Strimvelis, treats immunodeficiency due to adenosine deaminase deficiency, a condition that affects about three babies a year born in England.

NICE said the decision was the first time that new limits on cost-effectiveness had been applied for an extremely rare condition. It said it was satisfied the therapy would give children 'near normal' lives.

Professor Carole Longson, from NICE, said: 'Strimvelis represents an important development in the treatment of ADA-SCID, offering the potential to cure the immune aspects of the condition and avoid some of the disadvantages of current treatments.

'This means that children born with ADA-SCID will now have a better chance of being able to lead as near normal a life as possible, going to school, mixing with friends, free from the constant threat of getting a potentially life-threatening infection.

'Our evaluation of Strimvelis is the first time that NICE has applied the higher cost effectiveness limits introduced earlier this year for the assessment of treatments for these extremely rare conditions.'

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