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Gene mutation melanoma drug recommended for NHS use

5 November 2012
Appeared in BioNews 680

A drug which prolongs life in a form of skin cancer associated with a genetic mutation has been recommended for use on the NHS.

The final draft guidance published by the National Institute for Health and Clinical Excellence (NICE) recommends the drug, vemurafenib, as well as another melanoma treatment, ipilimumab, despite both medicines having been rejected as insufficiently cost-effective last year. Following this, the manufacturers submitted extra data to NICE and agreed to reduce the price of the drugs, leading to the current recommendations.

Although neither drug is a cure for melanoma, both are expected to increase the quality of life and prolong life expectancy by several months for people with advanced forms of the condition.

'Advanced melanoma can significantly affect patients' quality of life and without effective new therapies, the prognosis for advanced disease is very poor', said Professor Carole Longson of NICE. 'Vemurafenib and ipilimumab are breakthrough treatments that can potentially significantly affect prognosis for these patients'.

Vemurafenib targets a genetic mutation, called BRAF V600, present in about half of all melanoma patients. Professor Alan Ashworth of the Institute of Cancer Research, London, who was involved in research into the drug, told the Telegraph the drug was 'a brilliant example of what new-generation targeted cancer therapies can achieve'.

The Guardian says that drugs targeted against gene-specific disease variants 'were expected to transform the prospects of cancer patients' but a recent World Oncology Forum highlighted that such high expectations were not being met. 'Drugs like vemurafenib', the newspaper relates, 'cleared the cancer in an apparently miraculous fashion, but it returned within months'.

As such, vemurafenib is recommended as an end-of-life treatment, and only around 1,000 people per year are expected to be suitable to receive it. The drug originally cost £52,500 for seven months but after NICE's initial decision not to recommend the drug, Roche, the manufacturer, offered a discount. 'The size of the discount is commercial-in-confidence', a statement from NICE relates.

Professor Peter Johnson, chief clinician of Cancer Research UK told the Guardian that the negotiations were 'a good example of NICE and the pharmaceutical companies working together to ensure that effective cancer treatments get from research to the patients who need them'.

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