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NICE recommends first overseas treatment for use on NHS with GSK 'bubble baby' gene therapy

Published on 04/01/18 at 10:59am

NICE has chalked up a new first in its assessment of treatments for use on the NHS, ruling that the health service should fund GlaxoSmithKline’s gene therapy Strimvelis for the treatment of adenosine deaminase deficiency (ADA-SCID) – also known as ‘bubble baby syndrome’ – marking the first time the body has recommended a treatment which is currently only available in another country.

ADA-SCID is a very serious disease of the immune system which can kill those affected before they reach school age if left untreated and causes a range of issues including recurrent infections, development delay and chronic diarrhoea. It is so rare that only around three babies a year are born in the UK with the condition. Those who remain untreated are required to remain in isolation to avoid infection, hence the ‘bubble baby’ moniker.

The disease has in the past been treated with stem cell transplant, which carries an overall survival rate of just 67% from matched unrelated donors and 71% from matched related or unrelated donors.

By comparison, Strimvelis reported no deaths in clinical trials, though patient numbers were small. It is currently only available in Milan, but NICE’s assessment found that, despite its £526,000 price tag including travel to Italy, the treatment cost less than £100,000 per Quality Adjusted Life Year (QALY) compared to stem cell therapy, meeting its cost-effectiveness guidelines for rare diseases.

“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 disadvantages of current treatments,” commented Professor Carole Longson, Director of the Centre for Health Technology at NICE. “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 last year for the assessment of treatments for these extremely rare conditions.”

Matt Fellows

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