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Double First At UK HTA NICE For Strimvelis, GSK’s ‘Bubble Baby’ Gene Therapy

 
• By Francesca Bruce

NICE, the HTA body for England and Wales, has for the first time recommended a treatment – GSK’s gene therapy, Strimvelis – that is currently only available in another country. Also for the first time, NICE used a new system for determining the cost-effectiveness of highly specialized technologies such as this.

Italy
Patients will travel from the UK to Italy to receive GSK's Trimvelis • Source: Shutterstock

With its final evaluation determination recommending GlaxoSmithKline PLC’s gene therapy, Strimvelis, NICE, the health technology appraisal institute for England and Wales, has for the first time recommended a treatment that is currently only available in another country. Also for the first time, NICE used higher cost-effectiveness thresholds that were introduced to its Highly Specialized Technology program last year.

Strimvelis treats the ultra-rare immune deficiency condition known as bubble baby syndrome - severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID. Around three babies are born with the condition in England each year. Treatment with the gene therapy involves the removal and modification of a patient’s bone marrow cells outside the body to produce working ADA enzyme

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