NICE appraisal
Macugen and Lucentis have been going through the National Institute for Health and Clinical Excellence (NICE) appraisal process since August 2006. This process is now in its final stages following the publication of the so-called Final Appraisal Determination (FAD). This document contains NICE's guidance for the two drugs Lucentis and Macugen. While it does not approve Macugen as treatment for wet AMD on the NHS, it rules that Lucentis should be made available to almost all patients. Put in simple terms patients are eligible if:
and have been going through the National Institute for Health and Clinical Excellence (NICE) appraisal process since August 2006. This process is now in its final stages following the publication of the so-called Final Appraisal Determination (FAD). This document contains NICE's guidance for the two drugs Lucentis and Macugen. While it does not approve Macugen as treatment for wet AMD on the NHS, it rules that Lucentis should be made available to almost all patients. Put in simple terms patients are eligible if:
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Their sight falls between two measurements for visual acuity (how well they see while wearing appropriate glasses or contact lenses). The first cut-off point is that they will have reached the level of sight where they would normally no longer be able to drive (so-called visual acuity of 6/12). In practice, it is not likely that consultants will be held to this threshold by their PCTs since it would be difficult to ensure that patients were treated as soon as they hit this threshold. A second cut-off point (visual acuity of less than 6/96) means that patients who have lost too much sight will not be eligible for treatment. Whereas this cut-off point was originally above the level when patients were eligible to be registered as partially sighted it is now well below that level.
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There is no permanent structural damage at the centre of the retina.
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The bleeding is limited in its extent.
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There is evidence of recent deterioration.
Significantly, NICE has decided that it will only allow treatment with Lucentis on the NHS under the condition that the manufacturer of the drug (Novartis) pay for the cost of treatment beyond 14 injections in the eye that is being treated. A payment system will be put in place to ensure that this decision does not cause any delay in treatment once a patient has received the 14th injection. While this decision is important in terms of ensuring that the NHS can afford expensive new treatments it will not impact on the patient in anyway.
The Royal College of Ophthalmologists will develop criteria to decide when treatment should be discontinued and patients who are currently receiving Macugen will be allowed to continue treatment with the drug.
Next steps
Consultees in the process will now have the opportunity to appeal against this decision. If this happens final guidance, which is currently expected in June, will be delayed. RNIB will continue to lobby Primary Care Trusts and Local Health Boards to implement this guidance now rather than wait until final guidance is confirmed and an implementation deadline is set.
History of the NICE appraisal of anti-VEGF treatments
The Appraisal Committee initially took a negative view of the cost-effectiveness of both Macugen and Lucentis. As a result it rejected the use of Macugen on the NHS in its first preliminary guidance (issued on 14 June 2007). Most importantly, it also recommended restricting the use of Lucentis to people who are currently eligible for PDT (20% of all patients) and who have lost their sight in one eye.
All main parties involved in the consultation were opposed to this restrictive guidance since they felt that the Appraisal Committee had misinterpreted the available evidence. RNIB and the Macular Disease Society submitted a joint response to the first ACD and joined the Royal College of Ophthalmologists in calling on NICE to issue another ACD before taking its final decision.
In addition, an RNIB advert in the Daily Mail called on the general public to express their outrage at the draft guidance to NICE and RNIB, the Macular Disease Society and the AMD Alliance International mobilised their supporters to generate the greatest response to any NICE recommendations ever. As a result NICE received more than 13,000 letters, emails and signed petitions.
In response to the overwhelmingly negative reaction to its initial recommendations, NICE commissioned further analysis of the cost-effectiveness arguments for and against the new treatments. It has now issued a new set of recommendations which constitute a major u-turn.
The new recommendations, if adopted in its final guidance will mean that the vast majority of patients will receive treatment whether they have the condition in the first or the second eye and irrespective of the sub-type of AMD they have developed.
There are a few issues that still need to be resolved, including the threshold when treatment will no longer be made available. At present NICE recommends that a patient's visual acuity will have to be better than 6/60 assuming that 6/60 is the threshold for being registered as blind. However, 6/60 is the threshold for being registered as partially sighted and the Royal College recommends a cut-off point of visual acuity of 6/96 or 4/60 instead. The cut-off point matters because patients may see some of their sight restored if the treatment is effective and that is particularly important for people who have already lost significant amounts of their sight.
Another issue is the renewed decision not to allow the use of Macugen on the NHS. RNIB continues to support a clinician's choice to opt for the treatment that is best for each individual patient. This is only possible if Macugen is made available. Doctors in Scotland have the option to prescribe Macugen where this is clinically indicated and so should their colleagues in England and Wales.
RNIB and the Macular Disease Society submitted another joint response to NICE. The NICE Appraisal Committee then met on 13 February 2008 to discuss the submissions received from consultees.
Scotland
Macugen was approved for use on the NHS by the Scottish Medicines Consortium in August 2006 and Lucentis followed suit in June 2007. This means that patients in Scotland have access to treatment whereas at present patients in other parts of the UK generally have to go through case-by-case assessments by PCTs and Local Health Boards and are often denied funding.