Hospitals are gearing up to offer Novartis’ ground-breaking CAR-T therapy Kymriah to children with advanced leukaemia, after the drugmaker reached a deal with NHS England over its provision.

NHS England says the commercial deal with Novartis is the first in Europe, and comes less than 10 days after Kymriah (tisagenlecleucel) won marketing authorisation, marking one of the fastest funding approvals in the NHS 70-year history.

The “landmark deal” paves the way for patients up to 25 years old with B cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse to receive the therapy within weeks, after NICE approved the treatment for entry into the reformed NHS Cancer Drugs Fund.

“CAR-T therapy is a true game changer, and NHS cancer patients are now going to be amongst the first in the world to benefit,” said NHS England chief executive Simon Stevens.

“This constructive fast-track negotiation also shows how responsible and flexible life sciences companies can succeed – in partnership with the NHS – to make revolutionary treatments available to patients.”

“This decision to make our revolutionary CAR-T therapy Kymriah available so soon after being licensed is the result of our close collaboration with NHS England and NICE, with flexibility shown by all parties to ensure young patients can access this life-saving treatment as quickly as possible," said Mari Scheiffele, Novartis Oncology general manager, UK & Ireland.

"We trust that the NHS will continue this collaboration and flexibility in granting tisagenlecleucel access to adult patients with diffuse large B cell lymphoma (DLBCL), the most common non-Hodgkin’s lymphoma, where there is also a high unmet need for patients who have relapsed and have no other treatment options."

Kymriah works by harnessing the patient’s own immune system to fight certain types of blood cancer. During the process, T cells are drawn from a patient's blood and reprogrammed in the lab to create T cells that are genetically coded to hunt the patient's cancer cells.

In clinical trials, the therapy showed an 83 percent overall remission rate in this patient population with limited treatment options and historically poor outcomes.

“CAR-T cell therapy is the most exciting advances in treatment for childhood leukaemia for decades,” noted Dr Alasdair Rankin, director of research at the blood cancer charity Bloodwise.

“Intensive chemotherapy can now cure the vast majority of children but a significant number still tragically die every year because they do not respond to treatment. CAR-T cell therapy offers the genuine chance of a long-term cure for children who otherwise would have no other hope.”

Kymriah costs around £282,000 per patient at its full list price, and like other CAR-T therapies, is extremely complex to administer.

As such, hospitals wishing to provide CAR-T therapy must complete an international accreditation process. In England, the first hospitals to undergo this process are in London, Manchester and Newcastle. Subject to obtaining accreditation, these hospitals could offer the first treatments in a matter of weeks, NHS England said.