Ground-breaking CAR-T therapy approved in UK

Hundreds of people with an aggressive form of lymphoma are set to benefit from the first personalised immunotherapy treatment to be recommended for routine use in the NHS. Axicabtagene ciloleucel (Yescarta, Kite) is a CAR-T therapy for adults with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL) who have had two or more lines of systemic therapy. NICE has issued final draft guidance recommending the treatment be made routinely available on the NHS for suitable patients. It was previously only available through the Cancer Drugs Fund (CDF) and was commissioned routinely to patients in England after the NHS Commercial Medicines Directorate struck a confidential commercial deal with the company. NICE’s independent appraisal committee considered new evidence, including data from a clinical trial and from people who received axicabtagene ciloleucel through the CDF, before making the recommendation. Helen Knight, Director of Medicines Evaluation, NICE, said: “I am delighted that we have been able to recommend this pioneering treatment for people. The evidence from its use in the CDF and clinical trials shows it can offer an effective treatment, helping people live longer and with a better quality of life.” New clinical evidence A total of 318 people received treatment through the CDF between December 2018 and October 2021 in England. Overall survival rates in the CDF, with data collected over 36 months, show the median overall survival of people having axicabtagene ciloleucel was 28.5 months and 45% of people were alive after three years. The company estimates that the median overall survival on best supportive care, which is salvage chemotherapy, is around 6.4 months. There is currently no standard treatment for relapsed or refractory diffuse large B-cell lymphoma or primary mediastinal large B-cell lymphoma after two or more systemic therapies. Best supportive care usually includes salvage chemotherapy, which is given after the cancer has not responded to other treatments. Around 5,500 people are diagnosed with DLBCL each year in England. It is an aggressive disease which progresses quickly and is the most common type of high-grade non-Hodgkin lymphoma (NHL). It can develop at any age, but most people diagnosed with DLBCL are 65 or over. The company estimates that just over 450 people in England will be eligible to receive this new treatment.