The National Institute for Health and Clinical Excellence has sparked widespread outrage with a decision that would bar patients with liver cancer from access to Bayer’s Nexavar on the National Health Service.

Nexavar (sorafenib) is the first systemic treatment to show a survival advantage for patients with advanced liver cancer, and is proven to extend overall survival by 44% compared to best supportive care alone. In addition, while still considered a rarer form of cancer its incidence is on the rise and the disease now kills more than 3,000 people a year in the UK, highlighting the need for new effective therapies.

But despite concluding that Nexavar is a clinically effective treatment for this patient group, at a cost of £52,600 per QALY (quality-adjusted life year) gained the drug failed to fall under the Institute’s cost-effectiveness threshold and it was concluded that even under new guidance for appraising end-of-life therapies the drug does simply not offer the NHS value for money.

“We were disappointed not to have been able to recommend the use of sorafenib, but after carefully considering all the evidence, including the proposed ‘patient access scheme’ in which the manufacturer offered to provide every fourth pack free, sorafenib does not provide enough benefit to patients to justify its high cost,” said Andrew Dillon, Chief Executive of NICE, explaining the draft guidance.

Bayer has previously said the decision “directly conflicts with current UK and global guidelines for recommended treatment of HCC”, because recent treatment guidelines by the Hepatocellular UK Group “clearly state that sorafenib is the standard of care for patients with advanced HCC for whom no potential curative option is available”.

And Mike Hobday, Head of Campaigns at Macmillan Cancer Support, also voiced his disappointment at the rejection. “It is a scandal that the only licensed drug proven to significantly prolong the lives of people with this devastating disease has been rejected, leaving them with no treatment options”, he said, and called for a “rethink” of the way new cancer drugs are assessed to ensure fairer access for patients with rarer cancers.

'Devastating' news
Drug access pressure group The Pamela Northcott Fund said Nexavar’s rejection “is devastating to thousands of UK patients who will be left with no treatment option at all”, and founder Kate Spall added: “When are the government going to wake up and realise we want clinicians to decide on our treatments – not accountants and statisticians?”

In addition, the group said the decision means that the UK is now “the only country in the world to refuse patients access to Nexavar, despite it being widely available for both kidney and liver cancer in the US and every single other country it is licensed in, from Germany and France to Poland and Romania”.

This is not the first unpopular decision the cost watchdog has made on Nexavar, after rejecting an appeal back in August to make the drug, as well as Roche’s Avastin and Wyeth's Torisel, available on the NHS for patients with kidney cancer.

Bayer and other parties now have three weeks to lodge an appeal against the proposed guidance.