UK’s NICE supports CAR-T for young people with leukaemia

CAR-T therapy

The National Institute for Health and Care Excellence (NICE) has recommended Novartis’ Kymriah (tisagenlecleucel) for treating paediatric and young adults up to the age of 25 with B-cell acute lymphoblastic leukaemia (ALL).

It is approved in cancers that are refractory, in relapse post-transplant or in second or later relapse.

The treatment becomes only the second CAR-T cell therapy to secure routine funding for use in the NHS, and the only one for treating ALL in children and young adults under 26 years old.

Charlotte Crowley, Policy and Evidence Manager of Leukaemia Care, said: “These patients have few other options for treatment and CAR-T therapies have revolutionised the treatment of these patients already. We are really pleased that NICE has been able to recommend this treatment for the longer term, giving patients and doctors much greater clarity on what is available if they do find themselves in this position.”

The recommendation was based on longer follow-up clinical data from the ELIANA, ENSIGN and B2101J trials that were initially used at approval in 2018, as well as new data collected during its use through the Cancer Drugs Fund (CDF).

“During its time in the CDF, tisagenlecleucel has changed the way in which people with relapsed or refractory B-ALL have treatment,” said Dr Sara Ghorashian from Great Ormond Street Hospital. “It offers a chance of durable remissions and prolonged overall survival for people who often have no other option. The CDF has enabled us to build robust real-world evidence and I’m delighted that NICE has recommended that children and young adults should continue to have access to this treatment.”

Diana Spencer, Senior Digital Content Editor, DDW

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