First personalised children’s cancer drug available on NHS

NHS England chief executive Simon Stevens has announced that children and young people in England will receive a ground-breaking cancer treatment, the first in what is expected to be a rapidly expanding class of personalised cancer therapies available on the NHS.

NHS England’s commercial deal with the manufacturer Novartis is the first in Europe, and comes less than 10 days after the treatment was granted its European marketing authorisation. It represents one of the fastest funding approvals in the 70 year history of the NHS.

“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”

The landmark deal means young people with a form of leukaemia are set to receive CAR-T (a chimeric antigen receptor T-cell) therapy within weeks, after the National Institute for Health and Care Excellence (NICE) also green-lighted the treatment for entry into the reformed NHS Cancer Drugs Fund.

The Tisagenlecleucel form of CAR-T, also known as Kymriah, is the first in a wave of treatments in a new era of personalised medicine and part of the NHS’s long-term plan to upgrade cancer services.

Unlike other forms of treatment, CAR-T therapy is specifically developed for each individual patient and involves reprogramming the patient’s own immune system cells which are then used to target their cancer. It has been shown in trials to cure some patients, even those with advanced cancers where other treatments have failed.

Tisagenlecleucel, which costs around £282,000 per patient at its full list price, is licenced to treat 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. Patients in trials had an average age of 11.

Simon Stevens Chief Executive of NHS England will announce the deal at Health Innovation Expo in Manchester, he said, “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. Today’s approval is proof-positive that, in our 70th year, the NHS is leading from the front on innovative new treatments. 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.”

Mari Scheiffele, Novartis Oncology General Manager, UK & Ireland, said:

“This decision to make our revolutionary CAR-T therapy available so soon after being licensed is the result of our close collaboration with NHS England and NICE. This one-time therapy, specifically custom-made for each individual patient using their own immune cells, has the potential to extend survival and improve quality of life for those children and young adults who do not respond to other treatments and whose prognosis is poor.”

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