On Monday 27 August, the first two CAR-T therapies were authorised for use in the European Union, following EMA approval: Novartis’ Kymriah and Kite Pharma’s Yescarta.
Kymriah (tisagenlecleucel) has been authorised for the treatment of paediatric and young 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, and for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy.
Yescarta (axicabtagene ciloleucel) has been approved for the treatment of DLBCL and for primary mediastinal large B-cell lymphoma, after two or more lines of systemic therapy.
Both CAR-T therapies have already received approval in the US from the FDA.
Having received centralised European marketing authorisations, the companies must now liaise with national bodies to agree on the pricing and reimbursement of these treatments. They must also ensure that the necessary infrastructure is in place and training/accreditation completed to enable these therapies to be used.
In the UK, as is typical with new cancer therapies, NICE had begun evaluating both therapies several months earlier.
On Tuesday 28 August, NICE published draft guidelines stating that the cost per patient set by Kite in relation to Yescarta is currently too high for it to be considered a cost-effective use of NHS resources. NICE acknowledged the positive response rates, recognised that it represents a step-change in treatment and meets NICE’s criteria to be considered a life-extending treatment at the end of life. However, it requested further data as without this it could not calculate the size of the benefit of Yescarta over the current standard treatment (salvage chemotherapy). NICE also considered making Yescarta available on an interim basis via the Cancer Drugs Fund, while further evidence of its longer term benefits is collected. However, it deemed again that it does not meet the necessary hurdle of having the “plausible potential” to be cost effective. Kite and any other interested parties (including the general public) now have the opportunity to provide comments and further evidence. The consultation period runs until 18 September 2018 and the final publication is expected on 19 December 2018.
It is an almost identical story for Kymriah when it comes to treating DLBCL. Once again NICE decided that, despite good response rates and overall survival, there was insufficient evidence for an effect comparison with salvage chemotherapy and the cost was too high. NICE also ruled that Kymriah did not meet the criteria for the Cancer Drugs Fund. As with Yescarta, there is to be further consultation until 10 October 2018, with a final publication expected on 23 January 2019.
However it was not all bad news for Novartis. On Wednesday 5 September, fewer than 10 days after marketing authorisation grant, NHS England and Novartis announced that they had reached an agreement in relation to the use of Kymriah to treat paediatric and young patients (a subset of the approved uses). This was made possible by NICE agreeing that funding could be used from the Cancer Drugs Fund. Once accreditation processes have completed at specific NHS centres in London, Manchester and Newcastle, Kymriah will become available for paediatric and young patients at those centres. This is expected to happen in a matter of weeks.