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Orphan Drug

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Finalist

Osteosarcoma - rewriting the rule book

Takeda UK in association with Liberation Communications


Summary of work

Mepact (mifamurtide), the first therapeutic advance for more than 20 years, has been developed for osteosarcoma, a very rare form of bone cancer. It is a highly aggressive cancer with a propensity to metastasise, affecting around 150 children and young adults each year across the UK. 

Before Mepact was available a third of all children and young adults diagnosed with osteosarcoma did not survive. With Mepact it is estimated that an additional 12 children across the UK will survive osteosarcoma each year and go on to live ‘normal’ long lives. Mepact was submitted to NICE for a technical appraisal and despite a Patient Access Scheme (PAS) being proposed by Takeda, both NICE and the SMC gave initial negative decisions in July 2010. 

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 accurately assess the health economic value of Mepact, it would need to change its methodology to assess medicines that improve survival rates beyond 30 years. 

Following a fifth appraisal committee meeting a positive decision about the use of Mepact was given. The SMC also reversed its initial negative decision to recommend that Mepact could also be prescribed in Scotland. 

 

 

Executive summary as submitted


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