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Potential new paediatric glioma treatment on the horizon

We will be working with NICE as they assess a new paediatric glioma treatment and ensuring the community has its voice heard as part of this process.

a hand holds a magnifying glass up signifying new paediatric treatments on the horizon

When writing our new strategy 2023 – 2030 you told us that you wanted to have more effective treatments and that brain tumours felt like the forgotten cancer. As such, we are committed to working with stakeholders to ensure that there is a transparent, fair and fast system that will bring new treatments to patients as soon as is appropriate. In the spirit of this, we have been working with NICE on the assessment of a new paediatric glioma treatment.

Working with NICE on paediatric glioma treatment

This new treatment is designed for treating BRAF V600E mutation-positive glioma in children and young people aged between 1 and 17. It could be used when they have either a low-grade glioma that requires systemic treatment or a high-grade glioma that has relapsed, progressed or failed to respond to previous systemic treatment.

This is a combination therapy called dabrafenib with trametinib and we have been involved in the scoping exercise. This includes submitting evidence to NICE and attending a scoping workshop with them and other key stakeholders.

What NICE will assess

NICE will now look at the clinical and cost effectiveness of this treatment and to do that they will examine the following:

  • Clinical evidence – which demonstrates how well the medicine or treatment works.
  • Economic evidence – which demonstrates how well the medicine or treatment works in relation to how much it costs the NHS ie. does it represent value for money?

This treatment has been shown in a clinical trial to improve progression-free survival and response rates compared with chemotherapy in patients with low grade glioma. In high-grade glioma it has also demonstrated an improvement in terms of response rate, progression-free survival and overall survival compared with historical cohorts. It could also place less strain on healthcare resource as it can be administered orally at home.

The next steps in the paediatric glioma treatment assessment

The next steps are that stakeholders are invited to submit evidence ahead of a meeting in March 2024, with expected publication date of the guidance being May 2024.

We are in the process of collating and preparing that evidence and it will help NICE and those who sit on the appraisal committee to understand the experience of people affected by a paediatric brain tumour diagnosis, from both a patient and carer point of view.

As this is indicated in children and young people, it will be essential that the parent’s perspectives are considered in a meaningful way. This will focus on quality of life issues and what the advantages and disadvantages of the new treatment may be.  It is not our role to comment on the clinical or cost effectiveness, which will be the purview of other stakeholders. We will also enable families to share their stories as part of this.

Your role

The patient and patient organisation perspective is a meaningful part of these proceedings and will be carefully considered. As part of the scoping exercise we made the point of explicitly asking that health related quality of life applying to both patients and carer was called out as a consideration, which was subsequently updated to include. This is especially important given we know the impact of a childhood brain tumour diagnosis on the entire family.

As part of this we will be drawing on the information we currently collect in our paediatric Improving Brain Tumour Care Survey and so your voice will be truly represented as part of this decision making process.

Ultimately, this is a positive step, and we want to see new treatments coming through. It is essential that they are then assessed fairly, and that the system ensures that the patient perspective is part of this decision making process. We take this responsibility seriously and will continue to update our community when we can.

If you would like to learn more about what we’re doing to change things for those affected by brain tumours, click the link below: