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Kymriah to be made available in UK for paediatric leukaemia patients via Cancer Drugs Fund

Published on 15/11/18 at 12:47pm

NICE has announced its recommendation that Novartis’ CAR-T cell therapy Kymriah (tisagenlecleucel) be made available in the treatment of relapsed or refractory B-cell acute lymphoblastic leukaemia (ALL) for patients under the age of 25 via the Cancer Drugs Fund.

Eligible patients within this age range will have proven unresponsive to current treatment – blinatumomab or chemotherapy – or relapsed following stem cell transplant, so it has been estimated that around 25 to 30 people will benefit from the decision in England each year.

While the institute noted that based on current analysis it could not recommend the therapy for routine use due to uncertainties concerning long-term survival and cost-effectiveness, it added that additional data would be likely to resolve these issues.  

A specialised NHS service will be established to facilitate and manage access to the treatment, which itself will be made available over the coming weeks. While the treatment boasts a list price of £282,000, a confidential discount has been agreed.

“NHS cancer patients will now be amongst the first in the world to benefit from this game changing therapy,” commented John Stewart, NHS England’s Director of Specialised Commissioning. “Constructive engagement with NICE and fast-track negotiations with NHS England shows how responsible and flexible life science companies can succeed – in partnership with the NHS – to make revolutionary treatments available to patients”

Dr Alasdair Rankin, Director of Research at the charity Bloodwise, added: “CART-cell therapy can give children with leukaemia the real possibility of long-term survival if they do not respond to standard treatments. Today’s announcement will come as a huge relief for a number of worried families. We hope that people will be able to access the therapy as soon as possible.”

Matt Fellows

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