As the debate over off-label use is reignited following the publication of data on Roche's cancer blockbuster Avastin as a treatment for wet age-related macular degeneration, the European Alliance for Access to Safe Medicines is calling for urgent action to clarify when drugs can be used outside their approved status.

The patient group's concerns follow the publication a week ago of data from the two-year CATT study funded by the US government which revealed that Avastin (bevacizumab) and Roche/Novartis' eye drug Lucentis (ranibizumab) are equivalent in treating wet AMD, though the former is not approved for that indication. That was followed by a second, UK-based study, called IVAN, which also found that the drugs were comparably effective and claimed that the National Health Service could save more than £84 million per year if Avastin was used instead of Lucentis.

However, the EAASM notes comments from the chief executive of the UK's Macular Disease Society, Helen Jackman who welcomed the studies but noted that "they cannot provide definitive safety information and the overall question of the safety of Avastin is, unfortunately, not answered to everyone's satisfaction by these trials". She adds that the use of Avastin "will remain a matter of judgement for clinicians, with some satisfied with the evidence and others not".

Reacting to the studies, EAASM chair Jim Thomson, said "it is a complex issue, with hard-pressed budgets involved, but that should not cloud the fact that patients deserve to be given the choice of the licensed medicine. We are talking about an elderly population and these patients should be fully informed and give written consent if they are to be treated with an unlicensed medicine".

Mr Thomson added that "the very idea that a healthcare professional could prescribe an unlicensed medicine to a patient without his or her fully informed consent, is unthinkable". He noted that the EAASM met with the Medicines and Healthcare products Regulatory Agency recently and "they assured us that they plan to strengthen the adverse drug reaction reporting requirements in this area. That is extremely welcome".

Tip of patient safety iceberg

He went on to say "the Lucentis/Avastin debate is just the tip of a barely visible patient safety iceberg and whilst any savings at all might seem on the surface to be attractive in the current economic climate, that isn’t in itself reason enough to disregard two important factors". Those two factors are the regulatory process and the importance of keeping patients fully informed so they, "together with their healthcare professional, make choices that are based on the best treatment option, rather than the cheapest".

The EAASM statement comes days after the General Medical Council postponed plans to allow doctors, for the first time, to prescribe cheaper off-label alternatives to licensed medicines.