National Health Service patients with advanced soft tissue carcinoma in England and Wales will not be getting access to Spanish drugmaker Zeltia’s cancer agent Yondelis if current National Institute for Health and Clinical Excellence recommendations remain unchanged.

The Institute has published an appraisal consultation document rejecting the use of the drug for this indication as it claims it fails to offer the cash-strapped health service value for money.

Yondelis (trabectedin) is an alkylating agent that acts on cancer cells by damaging their DNA, and is licensed in the UK to treat advanced soft tissue carcinoma in patients who are unresponsive or unable to tolerate therapy with anthracyclines and ifosfamide.

In England and Wales around 500-600 patients are affected by the condition. There have been no major changes in the treatment of advanced soft tissue sarcoma in the past 20 years, and patients and experts consider treatment with trabectedin an option for those patients who would otherwise have no licensed treatment options.

Cost-effective?
According to Zeltia unit PharmaMar’s own economic analysis submitted to NICE, the base-case scenario cost of Yondelis, which does not take into account additional expenses for adverse events or patient monitoring, for example, is £61,000 per QALY (quality adjusted life year) gained compared to best supportive care. However, while the Institute’s Expert Review Group agreed with the calculation, it stressed that “in view of the weak evidence base” there remains “considerable uncertainty” around this estimate.

And although the Committee agreed that Yondelis meets the criteria for being a life-extending, end-of-life treatment, with an overall survival benefit of an three months compared to current care, “the magnitude of additional weight that would need to be assigned to the original QALY benefits in this patient group for the cost effectiveness of the drug to fall within the current threshold range would be too great”, it said, and so concluded that the drug would not be a cost-effective use of NHS resources.

The closing date for any comments on this consultation is July 21, and a second appraisal committee meeting is scheduled for August 4.