Final draft guidance from the National Institute for Health and Clinical Excellence (NICE) now recommends the use of Takeda's Mepact (mifamurtide) when it is made available at reduced cost to the NHS under a Patient Access Scheme (PAS).

The draft guidance backs the use of Mepact in combination with postoperative multi-agent chemotherapy as an option for treating high-grade resectable non-metastic osteosarcoma under these financial conditions. It reverses NICE's earlier draft decision, issued last October, not to recommend the drug's use because of the "exceptionally high cost the NHS is being asked to pay" for it and the "substantial uncertainty around how much extra benefit it could offer over and above currently-available treatments."

However, the Institute's independent appraisal committee has now re-examined the evidence, following a clarification from the NICE Board on how discounting should be applied in cases where treatment benefits are realised over a very long time period. The committee has concluded that Mepact, when added to the usual treatment with surgery and chemotherapy, increases the number of patients who are effectively cured of their cancer.

The members found that while the number of additional patients who would benefit in this way is small and uncertain (estimating increased overall survival by an extra seven people for every 100 treated), the treatment effects are substantial in restoring health and are sustained over a very long period. Patients who are cured continue to benefit for the rest of their lives, and the clarification from the Board provided guidance on how discount rates can be applied in this circumstance, says NICE.

In considering the evidence, the appraisal committee's view was that health-related quality of life may not have been adequately captured in the economic analysis, and the members were also "mindful of the innovative nature of the drug for a rare disease," says the Institute. Having noted the Board's clarification on discounting, and taken all other aspects into account - including the price discount that Takeda has agreed with the Department of Health - the appraisal committee concluded that Mepact could be accepted as a cost-effective use of NHS resources.

The Board's clarification, along with a revised PAS, means that the extra cost per unit of health gained that the NHS will be asked to pay for Mepact is now lower than previously determined, at a best-case probabilistic incremental cost-effectiveness ratio (ICER) of £36,000 per quality-adjusted life year (QALY) gained, compared to an earlier esteemed of £56,700 per QALY gained. However, this is still above the normal range.

The committee had "looked again very carefully in every way possible at whether there might be a case for accepting this higher than normally accepted extra cost per unit of health gained," said NICE chief executive Sir Andrew Dillon.

"This included considering the curative potential of the drug for some people, that this is a small patient population with a rare disease, and whether the change in health-related quality of life had been inadequately captured in the economic analysis," said Sir Andrew, who added that this appraisal has "raised some important methodological issues."

Although rare, osteosarcoma is the most common form of bone cancer, with around 150 new cases diagnosed each year in the UK. It is estimated that around 60 people could be treated under this draft recommendation.

The draft guidance is available for comment until September 21.