It looks like patients with advanced cancer of the urothelial tract will not be getting access to Pierre-Fabre’s Javlor on the National Health Service, after the drug was deemed too expensive by the National Institute for Health and Clinical Excellence.

The cost watchdog has published draft guidance turning down Javlor (vinflunine) for the treatment of advanced or metastatic transitional cell carcinoma (TCC) of the urothelial tract (encompassing bladder, ureters and kidneys) following chemotherapy.

In 2006 there were 4,295 registered deaths from bladder cancer in England and Wales. Treatment options for the condition are limited; patients with advanced TCC that has invaded the bladder wall or spread to the lymph nodes typically receive surgery (radical cystectomy) and/or radiotherapy and subsequent chemotherapy, but there are currently no agents specifically licensed in the UK for second line treatment.

Hopes of an effective new treatment were raised in June 2008 when Pierre Fabre presented positive data from Phase III trials of Javlor showing that the drug is the only one to have shown a 50% improvement in survival in patients compared with best supportive care.

However, the Institute’s appraisal committee felt that the clinical evidence submitted by the drugmaker comparing failed to show conclusive evidence on its clinical effectiveness, particularly with regard to its potential survival benefits compared to best supportive care.

“Although there is some evidence to indicate that vinflunine can extend life for patients with transitional cell carcinoma, there is considerable uncertainty around the estimates provided by the manufacturer,” explained NICE chief executive Sir Andrew Dillon.

Doubts over value

“When we recommend the use of expensive treatments designed to extend life, we need, for the sake of the patients who may wish to use them, to be confident about the nature and the extent of the benefit they bring,” he added, further defending the decision.

In another stumbling block for the drug, the committee said it was concerned that the side effects caused by Javlor, including severe constipation, had not been fully taken into account in the economic evaluation.

And according to NICE’s calculations, the estimated cost per quality adjusted life year gained of Javlor is more than £120,000, and so far over the Institute’s cost effective threshold for what would normally be a cost-effective use of NHS resources.