Mistakes by both sides led to Vidaza NICE rejection, says Celgene

Celgene has said it will fight to ensure patients get access to its blood cancer drug Vidaza (azacitadine) following its rejection by NICE, saying that mistakes by both organisations led to a huge difference in cost-effectiveness estimates.

The company told pharmaphorum it will challenge a NICE cost-effectiveness analysis in first draft guidance that said the drug would cost the system much more than the manufacturer estimated.

NICE last week said Vidaza should not be routinely funded in adults aged 65 years or older not eligible for haematopoietic stem cell transplant with acute myeloid leukaemia, with more than 30% marrow blasts.

Wim Souverijns, vice president and general manager at Celgene UK & Ireland, told pharmaphorum at a briefing in Switzerland that Celgene had made mistakes in cost modelling that led to the company estimating Vidaza's would cost around £20,000 per quality adjusted life year (QALY).

But he added that an evidence review group that estimated a cost per QALY in the region of £240,000 had also made errors.

Souverijns said: "We are disappointed we don't have a resolution," adding that if the company had thought the cost was so high "it would have taken a different approach."

"There is a mistake in the model, there are a lot of confounding factors – but they also made some mistakes."

But he said that there were still doubts that following the negotiations the drug would even fall below a £50,000 per QALY cost effectiveness threshold that NICE allows if the drug is considered an end of life treatment.

Celgene will continue to work with NICE in a consultation on the first draft document. A second draft is due in the coming months ahead of final guidance due to be published in July.

Should the drug be rejected by NICE, the newly remodelled Cancer Drugs Fund may provide funding. Instead of previous arrangements where the CDF funded cancer drugs rejected by NICE, the cost-effectiveness body will manage which drugs enter the scheme.

These are then trialled for two years while data are gathered – and NICE will then make a decision based on the updated evidence.

But Souverijns said NICE's assessments need to be changed as they often fail to show the value of drugs in very old patients towards the end of their lives, and where drugs provide incremental improvements when standard of care is already high.

There is hope that the Accelerated Access Review, which is delayed because of the UK's European Union membership referendum, could provide an answer.

"We don't need wholesale reform, we just need some flexibility," he said. Souverijns' views reflect those of the Association of the British Pharmaceutical Industry trade body, which has said new CDF arrangements will lead to many cancer drugs still being rejected.

England's revised Cancer Drugs Fund to begin in April