NICE Rejects Cenegermin for Rare Eye Disease, Citing Cost Concerns
Key Insights
NICE has issued a draft rejection for Dompé (search)'s cenegermin, a novel eye drop therapy for neurotrophic keratitis (search), deeming its £14,500 per course cost exceeding NHS value thresholds.
Clinical trials showed promising results with low recurrence rates of 0-5% at 56 weeks in the European REPARO study, though NICE noted concerns about long-term efficacy data.
Without access to cenegermin, patients may need to resort to tarsorrhaphy, a disfiguring procedure where eyelids are partially sewn together to protect the cornea.
The UK's National Institute for Health and Care Excellence (NICE) has delivered a preliminary rejection for cenegermin, an innovative treatment for neurotrophic keratitis (search), citing concerns over its cost-effectiveness for the National Health Service (NHS).
The decision impacts treatment options for patients with neurotrophic keratitis (search), a rare degenerative corneal disease affecting approximately five in 10,000 Europeans. Without effective treatment, patients face a high risk of permanent vision loss due to fibrotic scarring.
Treatment Mechanism and Administration
Cenegermin, developed by Italian pharmaceutical company Dompé (search), represents a novel therapeutic approach as an artificial nerve growth factor. The treatment is administered as eye drops six times daily at two-hour intervals, offering a non-surgical intervention option for patients.
Clinical Evidence and Outcomes
The drug's efficacy was demonstrated through two significant clinical trials. The European REPARO study, involving 156 patients, showed remarkably low recurrence rates of 0-3% at 32 weeks, extending to 3-5% at 56 weeks. The smaller US 0214 trial, with 48 participants, reported recurrence rates ranging from 0% to 14% across different trial arms.
Cost and Access Barriers
At £14,500 for an eight-week course, NICE determined that cenegermin exceeds their cost-effectiveness threshold of £30,000 per Quality Adjusted Life Year (QALY). The assessment was complicated by what NICE described as an inadequate cost-effectiveness analysis from the manufacturer, despite available clinical trial data.
Current Treatment Landscape
Without access to cenegermin, patients may be forced to undergo tarsorrhaphy, a surgical procedure where the eyelids are partially sutured together to protect the cornea. While less expensive, this alternative can result in significant disfigurement and impact quality of life.
Next Steps
The draft guidance is currently under consultation, with opportunities for further negotiations before a second draft is released later this year. Clinical experts consulted by NICE acknowledged the challenging nature of treating neurotrophic keratitis (search) and the limited effectiveness of many existing treatments, highlighting the unmet need in this therapeutic area.