The National Institute for Health and Care Excellence has turned down NHS funding for use of Novartis’ CAR-T therapy Kymriah in adults with relapsed or refractory diffuse large B-cell lymphoma (DLBCL).
The draft guidance recommends against its use for adults after two or more lines of systemic therapy, concluding that it is not cost effective for routine funding or use within the Cancer Drugs Fund (CDF).
A key sticking point is the fact that there is no data comparing Kymriah (tisagenlecleucel-T) to salvage chemotherapy, the most common treatment currently used, making it difficult to determine the exact benefits of the therapy, NICE said.
The committee also noted the degree of side effects a person may experience from Kymriah and the need for extensive monitoring by trained specialists.
These were also key factors behind the Institute’s earlier rejection of Gilead’s CAR-T therapy Yescarta (axicabtagene ciloleucel) for diffuse large B-cell lymphoma and primary mediastinal B-cell lymphoma.
“Although we could not recommend tisagenlecleucel-T for adults with lymphoma, we welcome further discussions around the cost-effectiveness of the treatment and engagement with stakeholders,” said Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE.
“The company had offered a confidential discount on the list price of £282,000, however, the cost-effectiveness estimates were still above the range that NICE considers an acceptable use of NHS resources,” she added.
In a statement, a spokesperson for Novartis said: "While we are disappointed with this preliminary decision, we recognize that this one-time therapy is an innovative and radically different treatment approach with curative intent, and it may be challenging to adequately compare Kymriah to current treatments for patients with this type of blood cancer who have limited options.
"We were surprised that NICE have not supported the “end of life” criteria for this population of DLBCL patients and strongly disagree with this decision. This relates to individuals who have already failed two previous therapies, and for the vast majority of this population, life expectancy can be counted in months rather than years. This is in contrast to a recent review for the same class of treatment and population of patients.
"We are working together with NICE to define the most relevant comparator studies and reconsider the “end of life” decision. We will continue to collaborate closely to support the adoption of this medical innovation for patients who have limited treatment options."
Earlier this month NICE backed CDF funding for Kymriah when used to treat patients up to 25 years old with B cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse to receive the therapy within weeks.
“CAR-T therapies are expensive and while there have been hugely promising early results in clinical trials, long-term survival rates are not known for certain because it is such a new technology," said Dr Alasdair Rankin, director of Research and Patient Experience at blood cancer charity Bloodwise.
“By approving CAR-T therapy to treat childhood leukaemia on the Cancer Drugs Fund earlier this month and investing in the infrastructure needed to make it available, the NHS has made it clear that it is willing to take a chance on these life-saving cancer treatments if enough evidence is available and a price can be agreed.
“People with diffuse large B-cell lymphoma who relapse after multiple treatments have a very poor outlook if they are treated again with chemotherapy. We hope the concerns and questions around CAR-T can be addressed so that these patients can be given access to the chance of a cure.”