Takeda's Mepact (mifamurtide) has been recommended by the National Institute for Health and Clinical Excellence (NICE) as a treatment for young patients with osteosarcoma, when it is made available to the NHS at reduced cost under a patient access scheme (PAS).
In a Final Appraisal Determination (FAD) which reverses the negative FAD published by NICE a year ago, the Institute recommends Mepact in combination with post-operative multi-agent chemotherapy as an option for treating high-grade resectable non-metastatic osteosarcoma in children, adolescents and young adults under a PAS. The evidence indicates that, when added to the usual treatment with survey and chemotherapy, the product increases the number of patients who are effectively cured of their cancer, it says.
Around 150 new cases of osteosarcoma are diagnosed in the UK every year, and while rare, it is the most common form of bone cancer. NICE points out that the number of additional patients who would benefit from Mepact is small and uncertain, but the treatment effects are both substantial in restoring health and sustained over a very long period.
"For the small number of patients who benefit from mifamurtide, the health benefits continue over the rest of their lives, effectively being a cure," said NICE chief executive Sir Andrew Dillon.
He also noted that this situation had presented specific methodological issues for NICE's independent appraisal committee regarding the way in which health benefits are valued and calculated over a very long period of time. Following a clarification from the NICE Board on how discounting is applied, and in addition to a revised PAS from the manufacturer, the extra cost per unit of health gained that the NHS will pay for Mepact was reduced from £67,000 to £36,000, which is still above the normal range that is usually accepted but considered by NICE to be cost-effective.
NICE's decision to reverse its previous negative FAD has been widely welcomed, with Dr Bruce Morland, consultant paediatric oncologist at Birmingham Children's Hospital, describing it as "a real triumph for clinicians and patients alike." Roger Paul, chairman of the Bone Cancer Research Trust, said the result is "thoroughly deserved" and reflects "the tireless campaigning by many of our members who have contributed to the appraisal process with their personal experiences."
"This will give further hope to the patients and their parents as they battle with this terrible disease," said Mr Paul.
Takeda points out that Mepact is the first advance in the treatment of osteosarcoma for 20 years, and that the new decision represents only the second time in NICE's 11-year history that it has clarified its methods guide for undertaking appraisal for treatments which can restore health over a long period, normally at least 30 years.
"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," said Yasuhiro Fukutomi, managing director of Takeda UK. "It has taken everyone's collaboration to lead us to this successful conclusion for osteosarcoma patents today," he added.
- The new NICE guidance includes additional recommendation supporting further research into the size of the effect of Mepact when combined with the type of chemotherapy typically used in UK clinical practice, as well as further collection of quality-of-life data from people who are cured.