Staying with most of the recommendations it made in a December 2007 draft, the U.K.' s National Institute for Health and Clinical Excellence recommended coverage of ranibizumab as treatment for patients with wet age-related macular degeneration, but advised against coverage of another wet AMD therapy, pegaptanib.

NICE's Aug. 26 ¹ technology appraisal guidance 155 establishes coverage for AMD by the U.K. National Health Service for patients in England and Wales. With the final guidance, NHS must make ranibizumab (Novartis/Genentech's Lucentis ) available to eligible AMD patients within three months.

Both AMD therapies previously won regulatory approval for wet AMD in the U.K. -NICE's guidance does not mean patients currently being treated with pegaptanib (Pfizer/OSI's Macugen ) must stop receiving the drug. But Macugen will not be recommended for new wet AMD patients. NICE estimates there are about 26,000 new cases of wet AMD annually in the U.K.

Following the draft guidance it issued last December, NICE specifies that Lucentis, a VEGF-A inhibitor, should be covered for patients meeting specific circumstances, but it limits NHS reimbursement to 14 injections per patient (² Also see "UK’s NICE Proposes Lucentis Dose-Capping Approach To Coverage For Wet AMD" - Pink Sheet, 14 December, 2007.). Novartis has agreed to assume costs for patients who need to continue treatment beyond 14 injections.

The December draft recommended Lucentis for patients whose best-corrected visual acuity is better than 6/60 - the final guidance instead requires patients corrected visual acuity be within a range of 6/12 and 6/96. Meanwhile, NICE ratified its earlier proposals to recommend treatment for patients with no permanent structural damage to the central fovea, lesion size less than or equal to 12 disc areas in greatest linear dimension, and recent evidence of presumed disease progression.

Treatment should continue "only in people who maintain adequate response to therapy," NICE stated, with discontinuation criteria to include persistent deterioration in visual acuity and retinal changes that indicate inadequate response. NICE also calls for a national protocol to specify criteria for cessation of Lucentis therapy.

Lucentis therapy consists of one intravitreal injection of a 0.5 mg dose monthly for three months, followed by a maintenance period involving monthly monitoring of visual acuity. If a patient experiences loss of greater than five letters in visual acuity during the maintenance period, NICE stipulates, a further dose should be given.

NICE approves the more-costly therapy

The two-year cost of Lucentis therapy, assuming eight injections during the first year and six during the second, is estimated by NICE as £10,700 ($19,600), or £761.20 per dose ($1,394).

Macugen, a pegylated modified oligonucleotide that inhibits VEGF-165, also is an injectable, administered at a 0.3 mg dose every six weeks, or roughly nine times a year. NICE determined Macugen therapy is not as effective clinically and therefore would not be cost-effective, even though its two-year cost would be £9,300 ($17,036) or £514 per injection ($942).

Novartis, which co-developed Lucentis with Genentech and holds ex-U.S. commercialization rights to the drug, noted that it provided clinical trial data from more than 7,000 patients to NICE that demonstrate the drug enabled wet AMD sufferers on average to read an addition 21 letters on an eye chart compared to untreated patients with the disease.

Lucentis is approved in more than 70 countries - it obtained FDA clearance in June 2006 - and Novartis in a release said it is committed to working with health authorities in other countries to find ways to bring the drug to as many patients as possible.

Pfizer, meanwhile, announced last month that Macugen, approved in the U.S. in 2004, has been approved in Japan as an orphan drug.

-Joseph Haas (j.haas@elsevier.com)

NICE Okays U.K. Coverage Of Lucentis With 14-Dose Cap; Macugen Not Approved

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