Last month, the National Institute for Health and Clinical Excellence’s proposal to restrict blindness drugs to patients who have already gone blind in one eye caused widespread uproar, and now a review by the Drugs and Therapeutics Bulletin, published in the British Medical Journal, concludes that the ruling is indeed “unacceptable.”
Wet age-related macular degeneration - the third leading cause of blindness worldwide of which there are 26,000 new cases in the UK each - is caused by the growth of new blood vessels beneath the retina, a process stimulated by vascular endothelial growth factor.
Genentech/Novartis’ Lucentis (ranibizumab) and Pfizer/OSI Pharmaceuticals’ Macugen (pegaptanib) block the effects of VEGF and, therefore, the formation of new blood vessels, as does Roche’s cancer drug Avastin (bevacizumab), which is not licensed for the condition but is sometime used ‘off-label’ as it is a cheaper alternative.
After a review of published clinical trial data, the DTB concludes that there is good evidence that both Lucentis and Macugen prevent further deterioration in vision in most patients, and Lucentis even improves vision in a third.
Lucentis restricted, Macugen rejected
But in its draft guidance, NICE recommended the use on the National Health Service of Lucentis for treating 20% of patients with wet age-related macular degeneration and then only when both eyes are affected, and Pfizer and OSI Pharmaceuticals' AMD product Macugen was rejected outright.
Explaining NICE’s decision at the time, Andrew Dillon, the agency’s chief executive, said: “when treatments are very expensive, we have to use them where they give most benefit to patients. Most people with AMD only seek help once the disease is beginning to affect their second eye.” Because of this, “our independent advisory committee believes the right thing to do is to treat and try to save as much sight as possible in the better-seeing eye,” he added.
The move, however, attracted severe criticism from all sides of the industry. The Royal National Institute of Blind People, for one, said that the decision will condemn 20,000 people each year in the UK to blindness, saying it is “outraged” at the recommendations to “offer treatment to just one in five 'lucky' patients - but only after they've gone blind in one eye.”
But a spokeswoman for NICE told PharmaTimes UK News that “the idea that people have to go blind in one eye before getting treatment is misleading. Firstly, AMD does not cause complete sight loss, and secondly, we are recommending that people should have AMD in both eyes and that the better seeing eye should be treated, not that they have to have severely affected sight before receiving treatment.”
However, speaking to PharmaTimes UK News, a spokeswoman for RNIB said that AMD does lead to blindness, and pointed out that it accounts for 56% of people registered as blind, with 90% of these having wet forms of the condition. She said patients should be given access to treatment as soon as they show any symptoms of the condition, and that the current proposals are gambling with people’s sight.
The DTB says: “We believe that policies that dictate that patients must go blind in one eye before being given treatment for the other are unacceptable.” It argues that Lucentis and Macugen should be available for use throughout the NHS as, it points out, is the case in Scotland. “In the absence of such national arrangements elsewhere in the UK, NHS commissioners need to work with local clinicians to ensure that the benefits of these drugs are made available to as many patients as possible.”
Final guidance from NICE is due in September.