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Published on 8 September 2011

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NICE reverses negative opinion on osteosarcoma drug


The National Institute for Health and Clinical Excellence (NICE) has recommended Mepact® (mifamurtide)for use in children and young adults with high-grade non-metastatic osteosarcoma.

The decision represents a reversal on the negative Final Appraisal Document given by NICE on October 8, 2010, and means that Mepact® will soon be available on the National Health Service across England and Wales.

Mepact®, the first advance in osteosarcoma treatment for 20 years, has shown it can reduce the risk of death from osteosarcoma in children and young adults by almost a third (8% absolute risk reduction p=0.03).

NICE’s decision to recommend Mepact® will now be subjected to a consultation period of approximately one month. Following this period, and if no appeal is put forward, NICE will publish the final decision as official NHS guidance.

“This decision by NICE to recommend Mepact® is encouraging news for children and young adults with osteosarcoma,” said Dr Bruce Morland, Consultant Paediatric Oncologist at Birmingham Children’s Hospital, England.

“I’m really pleased NICE reversed its previous decision and has recognised the exceptional nature of its appraisal of ultra-orphan treatments for rare conditions such as osteosarcoma. This is a real triumph for clinicians and patients alike”.

Roger Paul, Chairman of the Bone Cancer Research Trust, added: “This result is thoroughly deserved and reflects the culmination of a long journey and 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”.


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