Roche's cancer drug Avastin is a cheap, effective treatment for preventing blindness and should be used routinely in countries that cannot afford the far more costly product Lucentis, researchers said.
Avastin is not licensed for use in the eyes, but it is very similar to Lucentis — another Roche drug — and the tiny amount needed for an eye injection costs only around $50, against $1,950 for Lucentis.
Both drugs work by stopping the growth of blood vessels that can leak in the retina in a condition known as wet age-related macular degeneration (AMD), the leading cause of blindness in older people.
Avastin — currently approved for bowel and other cancers — is already used "off-label" for wet AMD by some doctors, but clinical evidence about its role in the eye has been sketchy.
Now a small randomized clinical trial conducted in Britain has endorsed both its safety and effectiveness.
"In countries where ranibizumab (Lucentis) is not affordable or available, we should use this drug immediately," lead investigator Adnan Tufail of London's Moorfields Eye Hospital said in an interview.
"We did this study because many people have been using this drug (Avastin) willy-nilly, and we've worried about whether it is safe and if it works. The answer is: absolutely."
Tufail and colleagues reported in the British Medical Journal (BMJ) that Avastin worked far better than older treatments, including OSI Pharmaceuticals' Macugen, and had few serious side effects.
Nearly a third of patients in the 131-patient study who were given Avastin injections gained the ability to read letters off a chart, and the group had far less eyesight deterioration.
However, the study did not look specifically at Lucentis, as the newer drug was not available when the trial started.
More information on how Avastin stacks up directly against Lucentis is expected to be available next year. It will be keenly followed by both medical experts and payers.
Roche has resisted using Avastin as an eye treatment, but that hasn't stopped independent groups from studying Avastin and Lucentis in head-to-head studies. Most attention is focused on a 1,200-patient U.S. National Eye Institute study that could report interim results around mid-2011.
Tufail said he expected the two medicines to show very similar results in head-to-head tests — an outcome that could trigger wholesale switching to Avastin as insurers and governments back the cheaper option.
The British government recently asked the National Institute for Health and Clinical Excellence (NICE) to consider whether it should evaluate the cost-effectiveness of Avastin in wet AMD, in a move that could pave just such a move to widespread use.
Some experts, however, remain cautious.
Usha Chakravarthy of Belfast's Royal Victoria Hospital said the latest British trial filled a gap in the evidence by showing Avastin was clearly better than older treatments, but did not prove it was as good as Lucentis.
"The off-label use of bevacizumab (Avastin) should not be encouraged until the large randomized trials comparing it with ranibizumab report their findings," she wrote in an editorial in the BMJ.
A Roche spokesman said the company continued to believe Lucentis was the most appropriate treatment for wet AMD, since it was engineered from the outset to work in the eye and was designed to bind more tightly to its target than Avastin.
Novartis, which sells Lucentis outside the United States, said the strength of evidence from the British trial was limited, given its small size and the fact that Lucentis was not included.
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