Bayer Healthcare has today lodged an appeal against the National Institute for Health and Clinical Excellence’s refusal to fund the use of Nexavar on the National Health Service for patients with liver cancer.

Earlier this month, the cost regulator sparked outcry when it published a Final Appraisal Determination – which was two years in the making – barring patients in the UK with advanced hepatocellular carcinoma from access to Nexavar (sorafenib) on the NHS on the basis that it is not cost effective.

Launched in the UK in November 2007, Nexavar is the first systemic therapy to show a survival advantage for patients with advanced stage disease in 30 years, and is clinically proven to extend overall survival by 44% compared to best supportive care alone, although the prognosis for patients remains poor even with treatment.

And yet, while the Institute conceded that the drug is indeed clinically effective, it also ruled that, at a cost of £52,600 per QALY (quality-adjusted life year) gained, it simply fails to offer the NHS value for money, even when taking into account recent guidance on appraising so called end of life drugs and a patient access scheme proposed by Bayer, under which it would pick up the tab for every fourth pack of the medicine.

But Bayer is not taking NICE’s decision lying down, and says it is appealing the guidance on the basis that it is “perverse” in light of the evidence submitted and that the Institute “failed to act fairly and in accordance with its own published procedures”.

According to Nicole Farmer, Business Unit Head of Bayer Schering Pharma Oncology in the UK, the cost watchdog has “failed to adequately consider clinical need and innovation in its decision making”, and the FAD “flies in the face of the UK HCC treatment guideline, NICEs own ‘end of life’ criteria and the government’s strategy to bring cancer outcomes in line with Europe”.

The appeal contains a number of objections, including that NICE failed to take into account the follow-up research programme offered by Bayer as part of the Patient Access Scheme (PAS), which, the firm claims, would “address any residual uncertainty regarding survival”, and that it acted unfairly by not accounting for the degree of clinical need of patients.

Innovation ignored?
Bayer is also adamant that NICE has failed to place enough emphasis on innovation in reaching its decision, and has therefore not fulfilled its obligations to consider the long-term benefits of innovation to the NHS.

Its appeal against the guidance is certainly timely given that the Institute is currently holding a conference in Manchester under the banner ‘Innovation and Value’, and that Innovation Passes - which would allow patients with certain rare diseases access to new drugs on the NHS before they have been cleared by the cost watchdog, in order to give drug manufacturers sufficient time to gather a reasonable amount of clinically meaningful data before NICE carries out its cost-effectiveness test - are a step closer to reality.

Andrew Wilson Webb, chief executive of the Rarer Cancer Forum, said his organisation is standing behind Bayer’s appeal given that Nexavar offers “unique” survival and quality of life benefits to patients with liver cancer. And commenting on the Institute’s decision, he remarked: “Sadly, NICE was established to increase innovation and to ensure that effective, innovative drugs are funded by the NHS, but it seems to have instead become a gatekeeper to protect the NHS budget,” and questioned the impact that decisions such as this will have on the future of medical science in the country.