NICE guidance recommends treatment delivered in Italy to be funded by the NHS
National Institute for Health and Care Excellence (NICE) has released guidance recommending gene therapy for rare disease where currently the only European treatment centre is in Italy, i.e. outside of the UK.
This is a first in NICE’s assessment of treatments for use on the NHS, ruling that the health service should fund GlaxoSmithKline’s (GSK) 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.
Furthermore, and importantly, the guidance, is the first time since its introduction in April 2017, that NICE has applied its new, higher cost effectiveness limits for treatments for very rare conditions.
Costing €594,000, NICE recommends Strimvelis when no suitable matched related stem cell donor is available. The treatment is usually given once only and the effects are thought to be life-long. Strimvelis has to be given at a hospital in Milan, so people will travel to Italy to have the treatment.
Children with ADA-SCID, severe combined immunodeficiency due to adenosine deaminase deficiency, are extremely vulnerable to infection and usually live in isolation to minimise the risk, hence the nickname ‘baby in a bubble’. Up until now the only treatment has been a stem cell transplant but these are risky and it is not always possible to find a good match. Now NICE has approved a new treatment for those children who cannot find a good match. It could mean children with ADA-SCID have the chance of going to school and socialising without the constant fear of catching a simple infection that could prove life threatening.
Around 3 babies a year in England are born with ADA-SCID and if left untreated infants die before school age. Their quality of life is affected by developmental delay, chronic diarrhoea, failure to thrive and recurrent infections.
Strimvelis is only the second gene therapy for an inherited disease to be licensed anywhere in the world. The treatment involves removing bone marrow cells and modifying them outside the body to produce working ADA enzyme. The modified cells are then returned to the patient via an infusion drip into a vein.
Professor Carole Longson, director of the centre for health technology assessment at 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.”
This article was first published in Payers Insights 23rd January 2018