Macugen, a new treatment for age-related macular degeneration, is launched today but primary care trusts (PCTs) are already telling patients that they will not pay for it.

A quarter of a million people in Britain suffer from “wet” AMD, the form of the disease that Macugen can treat. Every year, 21,000 more people, mostly over 60, have this form of the disease diagnosed.

But Macugen has not yet been cleared by NICE, the National Institute for Health and Clinical Excellence, and will not be for at least another year.

Specialists fear that many patients will go blind while they wait. The delay has been condemned by the Royal National Institute of the Blind (RNIB)which has given warning that many patients will lose their sight before a decision is made on approving the drug.

“We already have evidence that this drug is not getting to patients because PCTs are hiding behind NICE, saying that they can’t approve a treatment until NICE makes a ruling on its effectiveness, said Steve Winyard, head of research for the RNIB.

“If this happens across the country then we are going to see many people lose their sight and their independence to do things like drive and go shopping, so we are urging health trusts to think long and hard before they say no to patients getting Macugen.”

Ophthalmologists are excited by Macugen, the first of a new class of treatments that can attack the underlying cause of the disease. A second medicine, Lucentis, which some specialists believe will prove even more effective, is awaiting licensing in the US and Europe.

But both are relatively expensive — Macugen costs £4,000 per patient a year — and a NICE ruling on their cost-effectiveness is not expected until August next year.

Kevin Gregory-Evans, a consultant at the Western Eye Hospital in London, said of Macugen: “This really is a major step forward in fighting AMD.

“Previously we had very little to offer patients once the disease started to become active, and so severe loss of vision was an inevitable consequence of developing AMD. So to have an effective drug is absolutely marvellous.”

Both drugs are monoclonal antibodies that target VEGF (vascular endothelial growth factor), a protein that helps the formation of new blood vessels. In the eye, high levels of VEGF can cause proliferation of blood vessels and fluid leakage, the characteristics of “wet” AMD.

Sufferers begin to lose central vision, making it impossible for them to read or drive. Although they may retain sufficient vision for independent life, their quality of life is very seriously damaged.

“Unlike glaucoma or cataracts, very few people have heard of wet AMD — yet it has a major impact on patients' daily lives, depriving them of their central vision, independence and ability to complete every day activities, such as driving, shopping and cooking,” said Philip Hykin, Consultant Ophthalmologist at Moorfields Eye Hospital, London.

Macugen, made by Pfizer, is given by injections into the eye every six weeks.

In a key trial, reported in the New England Journal of Medicine, Macugen halved the risk of a severe loss of vision, while significantly more patients retained visual acuity and a few improved their sight.

While not a cure, Macugen “appears to be an effective therapy” concluded the team responsible, led by Dr Evangelos Gragoudas from the Massachusetts Eye and Ear Infirmary in Boston.

Trial results for Lucentis, made by Genentech (part of Roche) appear equally or more encouraging. Between a quarter and a third of patients improved their vision when treated with it.

While Lucentis is still awaiting a license, Macugen was licensed in February and doctors have been free to prescribe it since then. But it has been virtually unavailable on the NHS. Only patients who can pay have been given access.

AMD generally affects people over 60, and patients can go from normal vision to having lost most of their sight in just a few months.

“Anyone over 50 needs to be alert to the possibility that they could have AMD if they start having problems with their vision,” said Dr Hykin.

The precise causes of AMD are not known, although there is thought to be a genetic factor. A poor diet, high in fats and lacking vegetables and fruit, obesity and smoking are major risk factors, as is age.

Some patients have benefited in recent years from a laser treatment called photo dynamic therapy (PDT) but it can only be used for a minority of patients. Nice attracted criticism when it ruled that only patients who had gone blind in one eye first were eligible for treatment with PDF.

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