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NICE denies children life saving treatment for bone cancer


Takeda UK announces that in its final appraisal the National Institute for Health and Clinical Excellence (NICE) does not recommend the use of Mepact for the treatment of bone cancer (osteosarcoma) in children, adolescents and young adults. This comes despite strong support for Mepact from physicians and patient groups.

Professor of Cancer Studies at St James University Hospital, Leeds, Ian Lewis said of the verdict, ‘I am saddened and very disappointed by this NICE decision because it means it will be much harder for children with this rare form of cancer to receive Mepact. Osteosarcoma is a rare bone cancer so patient numbers in trials are never going to be huge. The largest trial ever completed in osteosarcoma has been done with Mepact. This showed an improvement in the most important endpoint of all, overall survival. This means that more children and young adults, who otherwise would not have survived their cancer, could now live a full life just like everyone else.‘

Osteosarcoma is a rare and often fatal form of bone cancer, with approximately 100–150 children and young adults being diagnosed in the UK each year. The average age of an osteosarcoma patient is fifteen, although children as young as two years old have been diagnosed with it. Typically it is a highly aggressive disease which in about a fifth of sufferers spreads to other parts of the body, usually the lungs. For up to a third of newly diagnosed children and young adults, the cancer will return.

Mepact is known as an ultra orphan drug which means that it will only ever be used to treat osteosarcoma and, unlike other cancer drugs, it will not be used to treat other forms of cancer. According to its own selection guidelines NICE should not assess ultra orphan medicines (for very rare diseases). The committee responsible for this final appraisal determination (FAD) acknowledges that they had not received direction from the Department of Health on the appraisal of treatments for rare conditions.

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Director of Sarcoma UK, Roger Wilson said, ‘NICE is hiding behind rules and regulations to justify its negative verdict.  It should do the right thing and withdraw Mepact from the whole appraisal process because it should never have been there in the first place.  NICE should stop holding things up and instead think about the children and families who could benefit from being able to have access to Mepact.‘

There have not been any advances in the treatment of osteosarcoma for more than 20 years. Current standard treatment is chemotherapy given before and after the tumour has been surgically removed (resection). Between a third to half of all children and young adults receiving this treatment will not survive beyond five years. Mepact improves chances of survival by almost a third meaning that an extra eight young lives could be saved each year in England.

Commenting on NICE’s decision, Chairman of the Bone Cancer Research Trust, Michael Francis who lost his own son to osteosarcoma said, ‘This is tragic for all families living with osteosarcoma. The facts are clear, Mepact offers a better chance of survival than current treatment. It mystifies me how NICE can justify this decision to take that chance away from us. These are real lives and real children who could go on to achieve great things but they’re not going to be given that opportunity.‘

Mepact’s Manufacturer, Takeda has announced today that it will continue to fight to get Mepact available on the NHS so that all eligible patients could be treated with it. The company plans to look at all options, including appealing against the decision, to ensure that children and young adults are given access to Mepact and offered a chance to fulfil their potential.


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