Finalist
Osteosarcoma – rewriting the rule book
Summary of work
Mepact (mifamurtide) is the first advance in osteosarcoma treatment for more than 20 years. A very rare form of bone cancer, osteosarcoma is highly aggressive with a propensity to metastasise, affecting around 150 children and young adults each year across the UK.
Before Mepact a third of all children and young adults diagnosed with osteosarcoma did not survive. With Mepact up to an estimated additional 12 children each year across the UK will survive osteosarcoma and go on to live “normal‟ long lives.
In July 2010, NICE gave initial guidance, which did not recommend Mepact for use on the NHS in England despite a Patient Access Scheme (PAS) being proposed by Takeda. The SMC also gave a negative decision.
Takeda worked with the Department of Health, NICE and the SMC to find a solution. After three further appraisal committee meetings, NICE acknowledged that to evaluate the health economic value of Mepact accurately, it would need to change its methodology to assess medicines, which improve survival rates beyond 30 years.
Following a fifth appraisal committee meeting a positive decision for the use of Mepact was given. The SMC also reversed its initial negative decision to recommend the use of Mepact in Scotland.
Executive summary as submitted
Judges’ comments
"Takeda brought about definite change as a result of taking a practical stance on payer challenges."
