LONDONBritain's health costs watchdog NICE has decided to back Japanese drugmaker Takeda's bone cancer drug Mepact after re-assessing the impact of a discount on the medicine for patients in the state-funded National Health Service (NHS).
The National Institute for Health and Clinical Excellence (NICE) had said in October that while there was evidence Mepact, known generically as mifamurtide, could be more effective when combined with chemotherapy than chemotherapy alone, there was uncertainty about its cost-benefit balance.
Takeda proposed a price reduction scheme under which it would make the drug available free to the NHS for the first seven doses, but NICE was at first unconvinced.
In a re-appraisal issued on Wednesday, NICE said a new evaluation on how discounting should be applied meant the drug's cost-benefit profile had improved.
"This clarification, along with a revised patient-access scheme, means that the extra cost per unit of health gained that the NHS will be asked to pay for mifamurtide is now lower than previously determined, but still above the normal range," Andrew Dillon, NICE's chief executive, said.
"After further careful consideration ... the committee was able to recommend mifamurtide for osteosarcoma."
NICE said this was draft guidance, and consultees and commentators have until September 21 to appeal it.
Although rare, osteosarcoma is the most common form of bone cancer with around 150 new cases diagnosed each year in Britain. The average age of an osteosarcoma patient is 15, with children as young as two having been diagnosed with it.
Tumors can grow anywhere in the skeleton, with the most common places being the legs or upper arm.
Treatment for osteosarcoma involves chemotherapy first to shrink the tumor, then surgery to remove the part of the bone or limb affected, followed by further chemotherapy after surgery to destroy any remaining cancer cells.
Mepact has a marketing authorization for use after surgery, in combination with post-operative chemotherapy drugs.
Bruce Morland, a consultant pediatric oncologist at Birmingham Children's Hospital, said NICE's decision was "a real triumph for clinicians and patients alike," while Roger Paul of the Bone Cancer Research Trust said it would give "further hope to the patients and their parents as they battle with this terrible disease."