It is looking increasingly likely that PharmaMar's Yondelis will not be available on the National Health Service for patients with ovarian cancer, after cost regulators again rejected the drug on questions over efficacy.

The National Institute for Health and Clinical Excellence has republished draft guidance turning down the use of Yondelis (trabectedin), in combination with pegylated liposomal doxorubicin (PLDH), as a treatment for ovarian cancer that has returned six months or more after initial treatment with chemotherapy, including platinum for platinum-sensitive disease.

Ovarian cancer is the fifth most common cancer in women in the UK, with more than 6,500 patients diagnosed every year. In around 80% of cases the cancer will return following first-line treatment, and it is estimated that just over two fifths of these could be eligible for treatment with Yondelis, a marine-based anticancer drug derived from the sea-squirt that attacks cancer cell DNA to prevent cell growth and spread.

But the Institute's Appraisal Committee said it has serious concerns over how Yondelis' effectiveness compares to that of other treatments available on the NHS. Crucially, PharmaMar did not submit any evidence comparing its drug to platinum-based chemotherapy regimens in treating relapsed disease, despite the latter being the gold standard therapy in such cases.

"This means that we cannot be sure that [Yondelis] extends patients’ lives for longer than the most routinely used treatments," explained NICE chief executive Andrew Dillon.

Evidence 'not robust'

New evidence considered did suggest that Yondelis might be most effective in women with ‘partially platinum-sensitive’ ovarian cancer, i.e. when the disease comes back between six and 12 months after initial platinum chemotherapy. However, the Committee said it was not sure that the effectiveness of the drug in this subset of patients was "genuinely different from that seen in the wider group of patients for which the drug is licensed", and so concluded that the data were not robust enough. 

Taking all the uncertainties into account, the Committee calculated that the incremental cost-effectiveness ratio (ICER) for Yondelis could be higher than £95,000 per QALY gained for the entire eligible population, and £68,000 for the partially platinum-sensitive subgroup, despite a proposed patient access scheme under which PharmaMar offered to pick up the tag for treatment with its drug following the fifth cycle of treatment.

“Even when taking into consideration the Patient Access Scheme, through which the manufacturer limited the total cost of the drug, the committee concluded that the cost of trabectedin was too high relative to the uncertain benefits it may provide patients," Dillon said.

Meanwhile, the Institute has announced that Sir Professor Sir Michael Rawlins has been reappointed as its chair for another year's term, until March 31, 2012. 

Professor Rawlins has held the position since NICE's birth in 1999.