The NHS cost watchdog for England and Wales has turned down Takeda UK’s Mepact (mifamurtide) for the treatment of children, adolescents and young adults with osteosarcoma, a rare and often fatal form of bone cancer.

According to Takeda, clinical data shows the drug - the first new treatment for the disease in over 20 years - cuts the risk of death by almost one third compared with chemotherapy alone, and has the potential to the potential to save an additional eight lives each year.

But following a review of the data the Institute’s appraisal committee concluded that, while Mepact plus postoperative multi-agent chemotherapy may be more clinically effective than the latter alone, the magnitude of this effect is uncertain, “particularly when compared with the regimen used in the NHS”.

Furthermore, the Committee agreed that, based on the evidence available, the incremental cost of the drug was at best £50,000 per QALY gained and at worse more than £100,000 per QALY gained, and therefore well above its cost-effectiveness threshold. This is despite a patient access scheme under which Mepact is provided free of charge to the NHS for the first seven doses.

“The Trust is astonished by the decision taken by NICE,” said Michael Francis, Chairman of the Bone Cancer Research Trust. “It seems perverse that we are prepared to let those young people die for the sake of £2.5 million - a sum which would dramatically allow more young people to survive”.

“If we take the decision down to its essentials, this means that NICE is prepared to sacrifice children on its self-justified altar of cost-effectiveness,” added Roger Wilson, director of Sarcoma UK.