New draft guidance from the National Institute for Health and Clinical Excellence does not recommend the use of Takeda’s “exceptionally high-cost” bone cancer drug Mepact in combination with post-operative chemotherapy drugs for the treatment of high-grade non-metastatic, surgically treatable osteosarcoma.

Patient group the Bone Cancer Research Trust (BCRT) says that as a result of this decision, a number of young patients who would have survived if they had received the drug will not now do so, and it will launch an appeal.

NICE says that the evidence presented to its independent advisory committee indicated that Mepact (mifamurtide) could become a valuable new treatment for osteosarcoma, when used in combination with post-operative chemotherapy, showing a statistically significant increase in overall survival. However, “the evidence presented by the manufacturer highlighted substantial uncertainty around the size of the treatment effect relative to standard UK clinical practice,” said NICE chief executive Sir Andrew Dillon, who also highlighted the drug’s “exceptionally high cost.”

According to Takeda’s submission, Takeda costs £2,375 for one dose and £114,000 for a full treatment course of 48 doses. Under a patient access scheme proposed by the firm to reduce the cost of the drug, it would be available at no charge to the to the National Health Service (NHS) for the first seven doses.

Professor Ian Lewis, chair of the BCRT’s scientific advisory panel, described NICE’s decision as “immensely disappointing” and said it meant that a number of young patients who would have survived if they received Mepact will not now do so.

“This drug is now available throughout Europe and once again is likely to mean that cancer survival in the UK will be worse than elsewhere. Survival rates for osteosarcoma have not improved in the last 20 years and more treatments options are desperately needed. The largest clinical trial ever carried out in osteosarcoma showed that mifamurtide significantly improved overall survival,” said Prof Lewis.

Mike Francis, chair of the charity’s trustees, added that the Trust had been “fighting for access to this important treatment for over a year and will appeal the decision.”

Although rare, osteosarcoma is the most common form of primary bone cancer, with around 150 new cases diagnosed each year in the UK, most frequently between the ages of 10-24. Approximately 55% of all patients treated in the UK will live for at least five years after treatment. The exact causes of osteosarcoma are unknown.

- Consultees have until October 22 to appeal against its recommendations, says NICE, which goes on to point out that it has not yet issued final guidance on Mepact in the treatment of osteosarcoma, and that this is expected to be published later this year. “Until then, NHS bodies should make decisions locally on the funding of specific treatments,” says the Institute.