This story was first published in digitalhealth.net
NICE approves therapy for ‘bubble baby syndrome’
NICE has approved strimvelis, a treatment for a rare inherited immune deficiency condition that has been termed ‘bubble baby syndrome’, in draft guidance.
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 ‘baby in a bubble’.
The only treatment has been a stem cell transplant up until now, but these are risky and it is not always possible to find a good match.
NICE has now approved a new treatment for those children who cannot find a good match. It could mean children with the deficiency have the chance of going to school and socialising without the constant fear of catching a simple infection that could be life threatening.
The draft guidance, which is the first time NICE has applied its new, higher cost effectiveness limits for treatments for very rare conditions, recommends strimvelis when no suitable matched related stem cell donor is available.
Strimvelis is the second gene therapy for an inherited disease to be licensed anywhere in the world. Costing €594,000, the treatment is usually given once only and the effects are thought to be life-strong. Strimvelis has to be given at a hospital in Milan, so people will have to travel to have the treatment.
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.”
You can read the draft guidance here - https://www.nice.org.uk/guidance/indevelopment/gid-hst10005/documents
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