NICE should appraise unlicensed drug

Age-related macular degeneration (AMD) is the biggest cause of sight loss in the western world. More than half a million people in the UK have AMD and it is becoming more common as the population ages. There is now a treatment for one form of the condition, 'wet' AMD, but it is expensive. It is called Lucentis and is a drug which is injected into the eye to stop the development of abnormal blood vessels which leak, scarring the macula and causing loss of central vision.

Lucentis is a wonderful, breakthrough drug which has saved the sight of thousands of people. However, there is a very similar cancer drug, Avastin, which is much cheaper when used in tiny eye-size doses. Lucentis costs around £900 per dose, Avastin about £100. Although not licensed for use in eyes, Avastin is widely used to treat AMD in other parts of the world including the US. Last year Lucentis accounted for more than one per cent of the entire NHS drugs budget. Now, many hard-pressed Primary Care Trusts (PCTs) want to use Avastin here, even though some clinicians say there is insufficient safety evidence to support this. We would like NICE to appraise this drug before it comes into widespread use in the NHS.

Why is it down to the government to initiate an appraisal?

NICE's remit is to appraise licensed drugs for the purpose for which they have been developed. Avastin is not licensed for use in eyes and so NICE cannot appraise it unless asked to do so by a minister.

The last government asked NICE to conduct a scoping exercise to decide if an appraisal would be feasible. That exercise was in July 2010. In December 2010 NICE said that an appraisal would be feasible if a regulatory body such as the Medicines and Healthcare products Regulatory Agency (MHRA) was prepared to help with safety and pharmacovigilance areas. However, last month health minister, Simon Burns said that the government had no plans to ask NICE to appraise Avastin, although the decision would be kept under review.

Why does the Macular Disease Society think this is important? Lots of drugs are used "off-label" without NICE appraisals.

This is a very controversial issue and there are very strong views both for and against Avastin use. Individual clinicians have legal responsibility for the off-label or unlicensed drugs they prescribe. They have conflicting messages from the bodies they look to for guidance on these matters. The Royal College of Ophthalmologists does not recommend the use of Avastin in eyes (although a newly formed committee is looking at this issue now). The General Medical Council (GMC) says that doctors should only use unlicensed drugs if licensed treatments are not available, or if it is in the patient's interests to use an unlicensed drug. They should not make prescribing decisions on the basis of cost. But PCTs are beginning to commission Avastin services to save money.

There are trials under way comparing the two drugs but even when they report we believe the controversy will continue. We think a body such as NICE has the credibility and the expertise to consider Avastin (with support from a safety body). This is the only way of ensuring that there is a coherent policy which allows doctors to make transparent decisions in the interests of their patients and in a way which does not pressurise them to act against their clinical judgement.

Not everyone agrees that NICE should appraise an unlicensed drug. Could there be damaging long-term effects on the development of new drugs?

It is quite true that the pharmaceutical industry and some others feel that it is inappropriate for NICE to appraise an unlicensed drug. They point out that NICE is not a safety regulator and that approving Avastin via NICE would be a back-door way to drug regulation which bypassed the usual safety regimes. They question why drug companies would undertake the costly trials necessary to obtain licenses if there is a cheaper, back-door route to bring drugs to market.

Under normal circumstances we would agree. But these are exceptional times. Avastin is being used in eyes and so far, nobody has regulated, approved or appraised it for use in ophthalmology.

What will happen if there is no appraisal?

We expect there to be dozens of 'mini-appraisals' in individual PCTs or PCT clusters. Some clinicians will be asked to prescribe against their clinical judgement and against the advice of their own Royal College. There will be a postcode lottery of treatment with some areas sticking to Lucentis and others using Avastin, and some using both. The argument about which is best will continue for years. If the pro-Avastin campaigners are right then the NHS will spend a lot of money on Lucentis unnecessarily. If the anti-Avastin campaigners are right then an inferior drug which is not as safe will be in use around the NHS. A credible and authoritative organisation needs to appraise this drug and make a decision. NICE is in the best position to do this.