Gene therapy for ‘bubble baby’ syndrome approved on NHS

The NHS will fund gene therapy for the first time after the UK’s healthcare cost watchdog approved treatment for the so-called “bubble baby” syndrome, despite a price tag of more than £500,000.

The treatment is used against adenosine deaminase deficiency, or ADA-SCID, which disables the immune system and means that children with the illness have to be kept in isolation to avoid infection – hence the “bubble baby” name.

The National Institute for Health and Care Excellence (Nice) said in draft guidance published on Friday that GlaxoSmithKline’s Strimvelis gene therapy for the condition improves overall survival compared with standard stem cell transplant therapy.

Gene therapy is designed to deliver a one-off cure for the patient, and drug-makers are typically asking a hefty price that is comparable to the combined costs of alternative life-long treatment. The Strimvelis therapy is so specialised that it is only offered by one hospital, which is in Italy.

“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,” Nice said.

“Costing €594,000 [£530,000], the treatment is usually given once only and the effects are thought to be life-long,” it added. It said that the treatment would give children a better chance of being able to live a more normal life, going to school and mixing with friends without fear of a life-threatening infection.

The draft guidance marks the first time Nice has applied its new cost-effectiveness limits for treatments for very rare conditions.

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