The first advance in the treatment of bone cancer in children and young people in two decades has been recommended in final guidance by NICE.
Takeda’s Mepact (mifamurtide), in combination with postoperative multi-agent chemotherapy, has been made available to the NHS at a reduced cost under an agreed Patient Access Scheme (PAS).
Sir Andrew Dillon, NICE Chief Executive, says for those treated with Mepact “the health benefits continue over the rest of their lives, effectively being a cure”.
The appraisal saw NICE for only the second time in its history clarify its ‘methods guide’ for treatments which can restore health over a long period, and together with the PAS, reduced the cost per QALY of Mepact.
“Following a clarification from the NICE Board on this issue, and in addition to a revised patient access scheme from the manufacturer, the extra cost per unit of health gained that the NHS will pay for mifamurtide is now lower than previously determined, but still above the normal range that is usually accepted,” said Sir Andrew.
“After having looked again very carefully at a number of factors (such the curative potential of the drug for some people and the small patient population) and taking these factors into account, the Committee was able to recommend mifamurtide for osteosarcoma as a cost-effective use of NHS resources.
“Today’s recommendation of mifamurtide will help children and young people with this very painful and distressing disease, as well as providing some for hope for them, and the people caring for them.”
NICE initially failed to recommend the treatment in an FAD published in October last year. However, after the revision to its methods guide and the proposed PAS, Yasuhiro Fukutomi, Managing Director of Takeda UK, says the company is delighted with the outcome.
“We are grateful to all those who have been involved in this long process including those at NICE, the Department of Health, physicians and patient organisations,” he said. “It has taken everyone’s collaboration to lead us to this successful conclusion for osteosarcoma patients today.”