CAR-T for kids: Kymriah enters under-25s market in UK
According to the agreement, patients up to 25-years-old with B cell acute lymphoblastic leukaemia that is refractory, in relapse post-transplant, or in second or later relapse, will be eligible to receive Novartis’ chimeric antigen receptor (CAR) T-cell therapy.
Kymriah (tisagenlecleucel) – made at Novartis’ Morris Plains, New Jersey facility in the US, and at Cell for Cure’s Paris site in Europe – uses a patient’s own modified T-cells to identify and destroy cells.
Just last week, Novartis announced plans to build a new facility in Stein, Switzerland, from which to manufacture cell and gene therapies, such as Kymriah, for the European market.
When announcing the Novartis deal earlier this week, England’s National Health Service (NHS) chief executive, Simon Stevens, said the “groundbreaking” personalised therapy is a “true game changer”.
“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, making them the first in Europe to have routine access to this exciting new type of immunotherapy,” agreed Cancer Research UK’s chief clinician, Charles Swanton.
Novartis Oncology general manager, UK & Ireland, Mari Scheiffele, said the firm will continue to work with the NHS to ensure physicians are appropriately “trained, supported and ready” to deliver Kymriah to patients.
Subject to passing accreditation requirements, the first three NHS hospitals to offer CAR-T therapies to children will be London, Manchester and Newcastle.
Kymriah in, Yescarta out
The deal comes just days after the European Commission approved its first two CAR T-cell therapies: Novartis’ Kymriah and Gilead’s Yescarta (axicabtagene ciloleucel).
Whereas in the US, the total cost of the Kymriah therapy can reach $1m (€860k), the treatment will cost approximately £282k ($365k) per patient at its full list price in the UK.
“It’s encouraging to see a drug company negotiate a deal with a healthcare provider that improves patient access – pending NICE approval – while receive what it feels is appropriate compensation for an innovative and clearly effective technology,” senior associate in valuations advisory at Duff & Phelps, Evelyn Warner, told us in an emailed statement.
“However, prices on the European market may be more flexible and companies more willing to accept lower profit margins than in the US. Drug makers generally expect to make the bulk of profit from the American market such that for a drug sold in the EU, value beyond recouping the cost of goods sold might be seen as additional upside,” she explained.
Yescarta, however, has been deemed too expensive by the UK’s National Institute for Health and Care Excellence (NICE), which issued a guidance recommending against its use.
According to the guidance, “The cost [of more than £50k per year of quality-adjusted life gained] is…too high for it to be considered a cost-effective use of NHS resources.”