Novartis receives NHS approval for their CAR T-cell therapy to treat leukaemia in children, 10 days after receiving European Commission (EC) approval.

“It’s fantastic news for children and young people with this form of leukaemia.”
Prof Charles Swanton, Cancer Research UK’s

Dubbed as the future of cancer therapy, Kymriah, Novartis’ gene-modifying treatment is among one of the most expensive drugs of all time pricing at £282,000 per patient. It involves taking blood and engineering the patient’s own immune system T-cells to recognise and attack the cancer cells before transfusing them back into the body. The results of clinical trials have been excellent for the treatment of blood cancer with a response rate of over 80%. Kymriah has also demonstrated the ability to help beat other cancers, such as myeloma and even solid tumours.

Novartis won the NHS deal over a rival CAR-T treatment made by Gilead Sciences which is even more costly at around £290,000.

In a statement released by NHS England earlier today, Prof Charles Swanton, Cancer Research UK’s chief clinician said: “It’s fantastic news for children and young people with this form of leukaemia that CAR-T cell therapy will be made available on the NHS.”

Right now, only around 20 children with acute lymphoblastic leukaemia (ALL) who have failed a series of earlier treatments are projected to be eligible for the drug.

Kymriah has also been licensed to treat adults with the more common cancer, diffuse large B-cell lymphoma (DLBCL). A decision is yet to be made on this by the National Institute for Health and Care Excellence (Nice) on whether the NHS can afford it. With around 200 adults eligible, the therapy will run up a legendary bill for the NHS.

The NHS is confident that treatment with the drug will be able to commence very soon. “The process of producing such a treatment is immensely complex and preparations are in their final stages. The first three NHS hospitals to go through the international accreditation process for the provision of CAR-T therapy for children are in London, Manchester and Newcastle. Subject to passing accreditation requirements the first treatments could begin in a matter of weeks.”

Announcing the deal at Health Innovation Expo in Manchester, Simon Stevens, chief executive of NHS England, will say: “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.”

Read more on Kymriah here