The National Institute for Health and Clinical Excellence is looking at the possibility of appraising Roche/Genentech’s Avastin to treat wet age-related macular degeneration, despite the drug not being licensed for this indication.

In an unusual move, ministers have asked that the Institute explore with stakeholders the potential value of assessing the clinical and cost effectiveness of the cancer drug Avastin (bevacizumab) on the National Health Service as an alternative to Novartis/Genentech’s Lucentis (ranibizumab), which specifically holds a license to treat the disease.

The long-running, global saga over the off-label use of Avastin in wet AMD, which remains the leading cause of blindness in the UK, stems from the fact that the drug has a very similar mode of action to Lucentis, but is significantly cheaper, making it an attractive proposition particularly for a cash-strapped NHS.

Wet AMD is caused by the abnormal growth of blood vessels around the eye, and both drugs work by preventing the formation of new blood vessels by targeting a natural protein called vascular endothelial growth factor. However, unlike Lucentis Avastin, which is approved in several cancer indications, has not been specifically designed or formulated for injection into the eye.

Nevertheless, Lucentis comes with a heavy price tag, and only made it through the Institute’s rigorous appraisals process by the skin of its teeth. Under NICE’s initial plans, only around 20% of patients with wet AMD would have had access to the drug, which costs around £10,000 per eye treated. But public outcry at the time, and a patient access scheme offered by Novartis under which it pays for any treatment beyond 14 injections, helped change its mind and issue a wider recommendation for the drug’s use on the NHS.

Although the Institute does not normally assess drugs outside of their licensed indications, it is able to do so on request by the Department of Health. And it seems that Ministers are keen to offer doctors a cheaper alternative to Lucentis, particularly as the number of patients being treated with the drug is reportedly greater than anticipated, further swelling costs.

The government is already funding two current trials pitting the two drugs head-to-head to compare their safety and effectiveness as treatments for wet AMD, and results are late next year and early 2012, which should help give a clearer picture of whether Avastin can have a more secure place in the NHS as a therapy for the condition.

Novartis 'disappointed'
Understandably, Novartis seems a little peeved at the development. A spokeswoman for the firm told PharmaTimes UK News that the company is disappointed by the direction given by Ministers. “It is very unusual for Ministers to ask NICE to develop technology appraisal guidance on the use of a drug outside its licensed indication. This is particularly so when there is a licensed and NICE recommended alternative already available on the market”, she said.

More than 7,500 patients have been enrolled into Lucentis wet AMD trials and several large, randomised controlled trials assessing the drug in new indications such as diabetic macular oedema and retinal vein occlusion have recently reported, while Avastin, in contrast, was formulated and developed for the intravenous treatment of cancers and not for administration in the eye, she explained.

“The legislation governing the development and licensing of medicines exists primarily to safeguard patient safety, [and] Novartis is very concerned that that this instruction could undermine this framework and place patients at unnecessary risk,” she stressed.