The National Institute for Health and Care Excellence (NICE) has approved a chimeric antigen receptor (CAR) T-cell therapy for adults with large B-cell lymphoma. The treatment significantly delays disease progression and improves survival outcomes.
The therapy, lisocabtagene maraleucel (liso-cel; Breyanzi), is recommended as an option for patients whose cancer is refractory or relapsed within 12 months of first-line chemoimmunotherapy. Eligible conditions include:
The final draft guidance reverses NICE's rejection of the drug in October. Approval follows a confidential pricing agreement between the NHS and Bristol Myers Squibb. The list price is £297,000 per single infusion, which includes shipping, engineering, and generation of the CAR-T cells.
Liso-cel is recommended only if provided under this commercial agreement. It is limited, at the manufacturer's request, to patients eligible for an autologous stem cell transplant. Age should not determine transplant suitability.
NICE's decision was based on clinical trial evidence comparing liso-cel with standard care for relapsed or refractory large B-cell lymphoma after first-line chemoimmunotherapy, which involves salvage chemotherapy, high-dose chemotherapy, and stem cell transplantation.
Data from the TRANSFORM trial demonstrated that liso-cel more than doubled average progression-free survival compared with standard care. The event-free survival hazard ratio was 0.38. However NICE noted uncertainty about overall survival benefits. In the trial, 66.3% of patients receiving standard care crossed over to receive liso-cel as a subsequent treatment, as per the trial protocol. This crossover complicated long-term survival comparisons.
Liso-cel is available immediately through the Cancer Drugs Fund pending routine commissioning by NHS England, which must make funding available within 90 days. Up to 600 patients annually could benefit from this outpatient treatment, which reduces hospital stays and improves quality of life.
Helen Knight, director of medicines evaluation at NICE, said in a press release that the announcement offered "real hope" for patients and families as "the evidence we've seen shows remarkable results".
Josh Hill, policy officer at Blood Cancer UK, welcomed the approval. He noted in a statement that many of the existing treatments are highly toxic to human cells and that approval of liso-cel expands treatment options for blood cancer patients in England. He also highlighted the "life-limiting side effects of current treatment" and the fact that survival rates for blood cancer in the UK lag behind nations of similar wealth and health.
Diffuse large B-cell lymphoma is a common type of fast growing (high grade) non-Hodgkin lymphoma (NHL), responsible for about 40% of NHL cases in adults. Around 4300 people are diagnosed each year in England, most of them aged 65 or over, and more males than females.
Although most people are cured with first-line chemotherapy, 10%-15% have primary refractory disease, and a further 20%-30% relapse after treatment. Prognosis after relapse is poor, with 5-year survival rates dropping to around 30%.
CAR T-cell therapy is a personalised treatment where a patient's T-cells are extracted, genetically modified to specifically target and eliminate cancer cells, and then reintroduced into the body. This one-time infusion has the potential to deliver lasting therapeutic effects.
Professor Peter Johnson, NHS national director for cancer, said: "It is excellent to have another CAR T treatment available for patients who have large B-cell lymphoma which is not responding to treatment or where the disease returns early on."
The NHS in England now offers four CAR T products across five clinical indications, from which more than 1500 people have benefitted.