Final draft guidelines from NICE recommend gene therapy for rare immune deficiency

Written by Freya Leask

The recommendation for children with ‘bubble baby syndrome’ is the first time NICE has applied new, higher cost-effectiveness limits for very rare indications.

Newly released final draft guidelines from National Institute For Health and Clinical Excellence (NICE; UK) recommends Strimvelis, a stem cell gene therapy, for the treatment of children with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) in cases where a suitable stem cell donor cannot be found. Patients with ADA-SCID are extremely vulnerable to infection and untreated infants usually die before school age.

Professor Carole Longson, director of the Centre for health technology assessment at NICE, explained: “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.”

Strimvelis is the second gene therapy to be licensed anywhere in the world and costs €594,000. Currently, the only facility approved to manufacture is based in Milan, so patients must travel to Italy for the treatment.

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Speaking to RegMedNet, Sven Kili, VP, Head of Cell & Gene Therapy Development, GlaxoSmithKline (UK), commented: “Strimvelis, the first licensed gene therapy for children in Europe, is a great example of a medical innovation designed to address a significant unmet clinical need in ADA-SCID.

“Since its approval in Europe in 2016, we’ve seen positive responses to Strimvelis and are pleased with the number of children and families who have been able to benefit.  We’re delighted that this positive guidance from NICE now enables families in the UK who have a child with ADA-SCID and no suitable matched stem cell donor to benefit from Strimvelis.”

This guidance is now in consultation. If there are no appeals, final guidance is expected in February. With a number of cell therapy approvals coming in 2017 and the cost of such treatments even higher than Strimvelis, insurance payers and regulators will be watching publication of future guidelines closely.