According to the National Institute for Health and Care Excellence (NICE) draft guidance, cost-effectiveness estimates for Novartis’ chimeric antigen receptor (CAR) T-cell therapy, Kymriah (tisagenlecleucel-T), are “above the range that NICE considers an acceptable use of NHS [National Health Service] resources.”
Kymriah received European approval last month for the treatment of certain patients with relapsed or refractory diffuse large B-cell lymphoma ((DLBCL), and Novartis has since signed a commercial deal with the NHS to supply children and young people in the UK.
However, the firm’s confidential discount on the UK list price of £282,000 ($372,000) was not enough for NICE to recommend the treatment for adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy.
According to NICE, another primary concern is the lack of data comparing Kymriah to salvage chemotherapy – the most common treatment currently used for this type of cancer: “This makes it difficult to determine the exact benefits of tisagenlecleucel-T and the committee concluded that in this case, tisagenlecleucel-T is not cost-effective for routine funding or use within the Cancer Drugs Fund.”
While the appraisal is not final – NICE’s committee will consider comments and additional evidence on October 23, 2018 – Novartis says it is “disappointed with this preliminary decision”.
“We recognise 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,” the firm said in an emailed statement.
Novartis also expressed concern regarding NICE’s ‘end of life’ criteria judgement.
“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.
“We are working together with NICE to define the most relevant comparator studies and reconsider the “end of life” decision,” the firm added.
Novartis is not the only CAR-T firm to have experienced regulatory resistance in the UK. Gilead’s CAR T-cell therapy, Yescarta (axicabtagene ciloleucel), received European approval the same day as Kymriah, however was immediately rejected by NICE in draft guidance citing cost-effectiveness concerns.
In response to NICE’s most recent decision, senior associate in valuations advisory at Duff & Phelps, Evelyn Warner, referenced research published in Social Science and Medicine, titled ‘Randomized Controlled Trials and Evidence-based Policy: A Multidisciplinary Dialogue’.
“NICE’s main reason for rejecting Kymriah was effectively that its clinical studies are accepted as the gold standard in clinical development.
“The results [Randomized Controlled Trials and Evidence-based Policy] presented make a powerful argument that a direct comparison between a one-size-fits-all drug like chemotherapy to a personalised treatment like Kymriah is unhelpful: a treatment designed for population health is antithetical to one tailored for a specific individual, and other potentially more appropriate methods of comparison need to be explored for the new wave of personalised medicine,” said Warner.