达拉非尼和曲美替尼——NICE指南发布——脑瘤慈善机构

正文内容

On 29th May 2024 the National Institute for Health and Care Excellence (NICE) published their final guidance for use of dabrafenib and trametinib for treatment of high and low-grade gliomas in children, meaning this treatment will be available on the NHS. What is NICE technology appraisal guidance?

NICE technology appraisal guidance represents the views of NICE – the official body which determines whether drugs are to be made available on the NHS. It dictates how a treatment they approve should be used by healthcare professionals. The appraisal guidance is written after very careful consideration of all the evidence available about a potentially new treatment. Healthcare professionals are expected to take this guidance fully into account when making treatment decisions. They must also take into account individual needs and the preferences and values of the person they are treating. What does it recommend for brain tumour treatment?

This guidance recommends that the combination of dabrafenib and trametinib is used to treat childhood high and low-grade gliomas with a BRAF V600E mutation. This treatment combination can be prescribed for children aged 1 year and over. Please speak to your treatment team if you have questions about whether this treatment is right for your or your child. What else is in the guidance?

The guidance also discusses: NICE concluded that this new treatment represents a step-change in current treatment of childhood brain tumours. Not only is this treatment more effective than current treatments, but it can be given orally at home, meaning that children can spend more time at home and less time in hospitals. How did we contribute to this outcome?

As a charity, we have played a vital role in this outcome. We know that outcomes like this take decades!

And for nearly 20 years we have been funding research that has improved the research landscape for childhood brain tumours. This includes historically funding the best researchers to study low-grade brain tumours in children and now funding The EVEREST Centre. This centre is a collaborative, international group of researchers striving to improve treatments and quality of life for children facing a low-grade brain tumour diagnosis. More recently we have also worked closely with NICE, Novartis and families personally affected by these brain tumours: Clinical effectiveness of dabrafenib and trametinib, based on clinical trial data The unmet need for children with this diagnosis We provided evidence during the NICE technical appraisal where we ensured there was a strong voice of the community represented Our Trustee Suki Sandhu also attended the appraisal. She shared how she was personally affected when her son, Raj, was diagnosed with a high-grade glioma. We’ve worked with the researchers at Great Ormond Street Hospital who set up the clinical trials to test this new treatment in high and low-grade childhood brain tumours We also worked alongside NICE to ensure that this new treatment was announced accurately and in a timely way to support the community of people who will benefit from this treatment