Patient representatives have reacted with dismay at new draft guidance from the National Institute for Health and Clinical Excellence (NICE) which does not recommend Roche Products' skin cancer drug Zelboraf (vemurafenib).

This assessment by NICE concerns Zelboraf's use in the treatment of unresectable locally-advanced or metastatic BRAF V600 mutation-positive melanoma. The data for this use submitted by Roche came primarily from the BRIM3 study, which compared its product with a current treatment, dacarbazine. The results showed Zelboraf to be effective for patients with melanoma with the BRAF V600 mutation.

However, according to NICE, the drug's longer-term effect on survival was uncertain because many of the patients taking dacarbazine were moved on to other treatments such as Zelboraf or Bristol-Myers Squibb's Yervoy (ipilimumab) when their disease progressed, making comparison difficult.

Treatment with Zelboraf costs around £1,750 per week. Roche has agreed a patient access scheme (PAS) with the Department of Health for a discount on the drug's list price, the size of which is commercial-in-confidence.

NICE's independent appraisal committee agreed that the most plausible cost per Quality-Adjusted Life Year (QALY) for Zelboraf was highly uncertain, due to the limitations of the available evidence, but was likely to be considerably higher than £50,000 per QALY gained, taking the PAS into account. Roche estimated that it would £56,400 per QALY.

NICE appraisal committees will normally recommend treatments that cost around £30,000 per QALY or less. If, like Zelboraf, a drug meets the criteria to be considered under NICE's supplementary advice for end-of-life treatments, then a higher cost per QALY may be accepted, There is no set threshold cost per QALY for end-of-life drugs but the highest cost per QALY of such a drug recommended by NICE is £50,000.

Commenting on the draft recommendation, NICE chief executive Sir Andrew Dillon said: “we need to be sure that new treatments provide sufficient benefit to patients to justify the significant cost the NHS is being asked to pay. Vemurafenib is an expensive drug and its long-term befits are difficult to quantify."

The draft guidance has now been issued for consultation. The manufacturer, healthcare professionals and members of the public are able to comment on it, said Sir Andrew, adding: "Roche can also provide further comment on the evidence it provided and have the opportunity to submit a further patient access scheme, if they wish."

Patient support group Factor 50 immediately called on NICE to reconsider its preliminary decision, pointing out that, with its provisional rejection of Yervoy last October, this is the second time in less than a year that a melanoma drug "has been refused NHS access."

Zelboraf "has proved beyond doubt in clinical trials to prolong the lives of many sufferers with advance-stage metastatic melanoma," said Factor 50, and the group's chief executive, Gill Nuttall, described NICE's draft guidance as “another truly devastating blow to all melanoma patients and their families, many of whom are very young and with young families."

"Standard treatments that have been available since the 1970s are ineffective and to deny this drug to patients is tantamount to passing them a death sentence," she said.