The Novartis CAR-T cell therapy, Kymriah (tisagenlecleucel), will be made available to patients under the age of 25 via the Cancer Drugs Fund. This follows a recommendation from NICE, that the drug be made available in the treatment of patients with relapsed or refractory B-cell acute lymphoblastic leukaemia.

NICE (National Institute for Health and Care Excellence), explained that they were unable to recommend the therapy for routine use due to cost effectiveness and long-term survival concerns. Additional data will be needed to resolve these issues.

For qualifying cases, access to the treatment will be managed by a specialised NHS service, receiving an undisclosed discount on the £282,000 list price.

“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.”