Charities are calling for a patient access scheme (PAS) to be agreed urgently following the National Institute for Health and Clinical Excellence (NICE)'s final rejection of the use of Novartis' Lucentis (ranibizumab) as a treatment for diabetic macular oedema (DMO).
Announcing its final decision, NICE said that while Lucentis has been shown in some clinical trials to be an effective treatment for DMO, the evidence presented by Novartis did not provide a true reflection of the drug’s cost-effectiveness compared with laser photocoagulation, the current standard treatment for DMO.
"The manufacturer's analysis produced a cost per quality-adjusted life year [QALY] gained that was at the higher end of what NICE considers to represent an effective use of NHS resources. But the committee concluded that the manufacturer's analyses were based on implausible assumptions, and that had a more plausible set of assumptions been used, the resulting cost per QALY would have exceeded this range," said NICE chief executive Sir Andrew Dillon.
"In particular, it was also noted that the manufacturer significantly underestimated the cost of treatment by not accounting in its submission the need to treat both eyes in a proportion of people with DMO," added Sir Andrew. Moreover, the appraisal committee "was also concerned that glycaemic control was much better in the trial population than it is in clinical practice, and that the evidence presented by the manufacturer suggested that the incremental cost-effectiveness ratio [ICER] would be higher in people with less well-controlled glycaemic control than observed in the trial," he said.
In July, NICE published its Final Appraisal Determination (FAD) decision rejecting Lucentis in DMO, which was appealed by Novartis and several royal colleges and charities. This appeal, which was heard in October, was rejected on all points, says NICE.
Novartis says that it is "extremely disappointed" with NICE's final guidance, and it believes the Institute did not consult sufficiently with clinical and patient experts on the additional data submitted by the company following the initial appraisal consultation document.
The charities which appealed the FAD decision - Diabetes UK, the Juvenile Diabetes Research Foundation, the Royal National Institute of Blind People (RNIB) and the Macular Disease Society (MDS) - are now calling on Novartis, the Department of Health and NICE for rapid agreement on a PAS for Lucentis as a treatment for DMO, which affects at least 50,000 people in the UK.
"As a patient organisation, we are deeply frustrated that a full review of all the available information has not been allowed," said Steve Winyard, head of policy and campaigns at the RNIB.
"We firmly believe that some of the assumptions made about the cost-effectiveness of the treatment were wrong. In particular, there is a very strong case for providing Lucentis to those patients with thicker retinas who are unable to benefit from existing laser treatments - a second consultation would have allowed this to be examined. However, we now hope that a PAS can be agreed swiftly, so that patients with DMO are not left to needlessly lose their sight," Mr Winyard added.
- Lucentis costs £742.17 per injection, with treatment being given monthly and continued until the patient's visual acuity becomes stable over three consecutive monthly assessments performed while on the drug.