Scotland’s healthcare system has rejected the use of Novartis‘ immunotherapy Kymriah (tisagenlecleucel) for adults with aggressive diffuse large B-cell lymphoma (DLBCL) who failed at least two prior lines of therapy.
The Scottish Medicines Consortium (SMC) denied routine access to Kymriah through NHS Scotland based on “uncertainties in the company’s evidence around its long-term benefits,” according to a Cancer Research UK report.
SMC states that Novartis’ justification for the therapy’s cost in relation to its health benefits was not sufficient. Moreover, the company “did not present a sufficiently robust economic analysis to gain acceptance by SMC.”
This information is available in a public summary published by the agency.
The key evidence supporting the use of Kymriah for DLCBL comes from the open-label, single-arm, JULIET Phase 2 trial (NCT02445248), which served as the basis for Kymriah’s approval in the U.S.
The treatment yielded an overall response rate of 53%, meaning that about half of the treated patients achieved some degree of tumor shrinkage. For four in 10 patients, the disease completely disappeared three months after treatment.
However, Kymriah was also seen to cause major side effects, including severe neurological problems and serious infections.
Moreover, patients were not followed up for enough time to draw meaningful conclusions about the relevance of treatment responses, SMC stated in a detailed report.
The decision follows the approval of Kymriah for England’s NHS to treat the same group of patients in February 2019, by the National Institute for Health and Care Excellence (NICE).
Initially, NICE had rejected the therapy, due to its cost and the lack of studies supporting its long-term effectiveness. But after additional negotiations with Novartis about Kymriah’s cost, NICE changed its stance and recommended that Kymriah be offered through the Cancer Drugs Fund, which is a source of funding for cancer medications in England.
Kymriah is a form of immunotherapy called CAR T-cell therapy. It involves taking a patient’s own immune T-cells and genetically engineering them in the lab to express a chimeric antigen receptor (CAR).
With Kymriah, the CAR recognizes, on the surface of B-cells, a protein called CD19, and primes T-cells to identify and kill malignant B-cells, such as those growing in DLBLC. The engineered T-cells are then infused back into the patient.
Gregor McNie, head of external affairs in Scotland for Cancer Research UK, considers the SMC’s decision “disappointing.”
“If approved, Kymriah would have provided a new treatment option for patients with this aggressive type of blood cancer who might have few other options available to them,” he said, hopeful that SMC will eventually reach an agreement with Novartis to make Kymriah routinely available to more patients in Scotland.
Despite SMC’s decision, individual patients in Scotland can still apply for financial support to get Kymriah, if their doctors claim they would benefit from it.
