Novartis’ Rydapt , the first and only targeted therapy approved in Europe for FLT3-mutated acute myeloid leukaemia (AML), has been rejected for NHS use by the National Institute for Health and Care Excellence.

In draft guidelines, the cost watchdog highlighted that data from a randomised controlled trial shows people taking Rydapt (midostaurin) with chemotherapy live longer than people taking chemotherapy alone.

However, because the trial did not include people over aged over 60, the effectiveness of the drug in older people is “uncertain”, it said.

“There is also considerable uncertainty about the cost effectiveness of midostaurin because of problems with the economic model. These include not accounting properly for remission after relapse and including implausibly high long-term costs in some situations,” according to the consultation document.

The most likely cost-effectiveness estimate of Rydapt plus chemotherapy compared with chemotherapy alone was estimated to be more £62,818 per quality-adjusted life year gained, and thus too expensive for NHS use.

NICE said the drug is also not suitable for use within the Cancer Drugs Fund, as it “does not have plausible potential to be cost effective at its current price and more clinical data collection would not address the uncertainties.”