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NICE has issued draft guidance recommending against funding vorasidenib for NHS use in England. This is NOT a final decision. The guidance is currently under consultation, and all stakeholders have until 4 November 2025 to submit their responses.

A second NICE committee meeting will take place on 20 November 2025, when a final decision will be made. The outcome is expected shortly before or after Christmas 2025.We think it is important that the community who is living with this type of LGG understands the facts that sit around the NICE process. There has been considerable noise around decision announced last week, some of which is incorrect. We recognise that discussions about access to new treatments are deeply emotive, particularly for people living with brain tumours, which includes their families. However, we believe it is important that patients and their caregivers understand the facts about the NICE appraisal process and what happens next, the current status of vorasidenib, and what the draft guidance means. The information provided here is to help you navigate this uncertain time with clarity about what is happening and what may happen next. One fact – NICE awarded positive recommendations to 79% of oncology indications it appraised between 2009 and 2013.

What Is vorasidenib? Vorasidenib is a targeted oral therapy designed for people aged 12 and over with grade 2 (low-grade) gliomas that have mutations in the IDH1 or IDH2 genes. These IDH-mutant low-grade gliomas, which include astrocytomas and oligodendrogliomas, are among the most common brain tumours in adults under 50, affecting approximately 300 people annually in England who would be eligible for this treatment. The drug works by crossing the blood-brain barrier and inhibiting the abnormal IDH1 and IDH2 enzymes that drive tumour growth. It is taken once daily as a tablet and has been approved by the FDA in the United States and the MHRA in the UK for private use, but it is not yet available on the NHS.

What is the evidence? The approval was based on the Phase 3 INDIGO clinical trial, which involved 331 patients from 10 countries. The trial demonstrated that vorasidenib:

-Significantly slowed tumour progression: The median progression-free survival was 27.7 months for patients taking vorasidenib compared to 11.1 months for those receiving a placebo.
-Delayed the need for further treatment: The drug extended the time to next intervention, postponing the need for radiotherapy or chemotherapy.
– Had a predominantly low-grade safety profile: Side effects were generally manageable.

So why has NICE issued draft guidance against funding? NICE’s draft decision to decline funding is based on four key concerns about the evidence:
1. Immature data
The primary concern is that insufficient time has passed since the trial ended to understand fully the long-term outcomes for patients. While the trial showed that vorasidenib delays disease progression, NICE requires more mature data to assess the treatment’s full impact. More modelling of the existing data is being done and will be shared with NICE.
2. Uncertainty around overall survival
Clinical trial evidence demonstrates that vorasidenib increases the time before the tumour progresses. However, there is not enough evidence to show whether vorasidenib extends how long people with the condition live. This is a critical factor in NICE’s assessment. It means that whilst there may be data on progression free survival, we don’t yet have data on overall survival.
3. Uncertainty about time to next intervention
There is uncertainty surrounding exactly how long vorasidenib delays the need for subsequent treatments like chemotherapy or radiotherapy, and how this translates into real-world clinical benefit. It is important to remember that vorasidenib is not a cure. Low-grade gliomas with IDH mutations continue to grow over time. Vorasidenib is designed to slow progression of the tumour and delay the need for more toxic treatments, which may preserve
quality of life for longer. This has played a factor in the decision. It is not a cure.
4. Cost effectiveness concerns
Because of these uncertainties in the economic model, NICE has decided that it cannot establish whether vorasidenib represents value for money for the NHS at this time. So it has asked for more evidence. What’s next? The consultation process is an important opportunity for additional evidence and patient perspectives to influence the final decision.

What happens now? 

1. Consultation period: Open until 4 November 2025 for stakeholders to submit responses
2. Second NICE Committee Meeting: 20 November 2025
3. Final decision: Expected around Christmas 2025
4. Implementation: If approved, vorasidenib would typically become available on the NHS
within three months of the final approval date.

Why This Matters.

The draft guidance means that, for now, vorasidenib will not be routinely available on the NHS. However, the decision is not final, and the consultation process allows patient groups, clinicians, and the drug manufacturer to provide additional evidence that may address NICE’s concerns. If you would like to add your voice to the second consultation you have until 4 November. You can leave your comments on the NICE website. 

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Introduction

The Brain Tumour Data Dashboard lets you explore up -to-date, population level data about the brain tumours diagnosed in England between 2013 and 2015. Using the drop down menus on the left you can select different groups of patients to view in the charts below. In these charts the number of patients for every 100 diagnoses is displayed as images of people. Patients have been grouped by date of diagnosis, type of tumour, age, gender, and region in England.

For each group of patients you can explore the different routes to diagnosis, the proportion of those who received chemotherapy or radiotherapy, as well as the survival of the patients within each group. For more information about what these metrics mean please see the glossary.

How to use

  1. Select the year of diagnosis using the drop down menu.
  2. Select your patient group of interest from the four drop down menus in the following order:
    1. Tumour group
    2. Age at diagnosis
    3. Region of England
    4. Gender of patient
  3. To view a second chart to compare different groups of patients, click the ‘compare’ button.The second chart will appear below the first chart.

*Note that the tool is best used on a laptop or tablet rather than a mobile phone*

Unavailable data

Some of the data in these charts is not available.There are two main reasons for this:

  1. How the data has been grouped

If you cannot select a patient group from the drop down menus, the data is unavailable because of how the data has been organised.

Public Health England has grouped the data like a branching tree. The bottom of the tree contains all the patients with brain tumours, and then each branch divides the data by a certain characteristics, like age, or location of tumour. But the data is divided in an order, starting with location of the tumour (endocrine or brain), then by age, region, and gender. Age is at the start because it makes a bigger difference to survival rates and treatment rates than gender or region. Sometimes, after the data has been split by type of tumour and age, there is not enough data to be split again. This is because to protect patient confidentiality groups cannot contain less than 100 patients. Because some groups cannot be split further, you cannot create ‘totals’ for everyone by region or gender. For example, you cannot see results for all ages by region, or all brain tumours by gender. If these totals were calculated and released, it might be possible to identify patients, which is why Public Health England cannot release this data.

  1. Statistical reasons and data availability

If you can select a patient group from the chart menus, but the chart does not display, the data is unavailable for one of several reasons:

  1. Data is not yet available for the selected year from Public Health England.
  2. Data is not available because the data quality is too poor to release this statistic.
  3. Data is not available as the statistic is not appropriate for this group.
  4. Data is not available because the standard error of the estimate was greater than 20% and so the estimate has been supressed.

Up to date brain tumour data

Brain tumour data may influence the decisions you make about your care. Data also helps you understand the bigger picture, or landscape, in which you find yourself.

Brain tumour data and statistics influence the focus, and work of organisations like brainstrust. The information helps us to understand the scale and impact of the problems we are setting out to solve.

This tool helps you understand the landscape in which you find yourself having been diagnosed with a brain tumour. This landscape can be particularly tricky to navigate as there are many different types of brain tumour, all of which have a different impact.

The information you see represents the most up-to-date, official, population level brain tumour data available for England. Over time we will be adding to the brain tumour data available and publishing reports, with recommendations, as a result of what we learn from this data.

The data behind this content has come from Public Health England’s National Cancer Registration and Analysis Service (NCRAS) and is a direct result of the ‘Get Data Out’ project.

This project provides anonymised population level brain tumour data for public use in the form of standard output tables, accessible here: http://cancerdata.nhs.uk/standardoutput

Incidence

The number or rate (per head of population) of new cases of a disease diagnosed in a given population during a specified time period (usually a calendar year). The crude rate is the total number of cases divided by the mid-year population, usually expressed per 100,000 population.

Malignant

Malignant tumours which grow by invasion into surrounding tissues and have the ability to metastasise to distant sites

Mortality

The number or rate (per head of population) of deaths in a given population during a specified time period (usually a calendar year). The crude rate is the total number of deaths divided by the mid-year population, usually expressed per 100,000 population.

Non-malignant

Not cancerous. Non-malignant tumours may grow larger but do not spread to other parts of the body.

Survival

The length of time from the date of diagnosis for a disease, such as cancer, that patients diagnosed with the disease are still alive. In a clinical trial, measuring the survival is one way to see how well a new treatment works. Also called ‘overall survival’ or ‘OS’.

Routes to Diagnosis

Under the ‘Routes to Diagnosis’ tab in the Brain Tumour Data Dashboard, you can explore the ways patients have been diagnosed with brain tumours. There are many ways, or routes, for cancers to be diagnosed in the NHS. A ‘route to diagnosis’ is the series of events between a patient and the healthcare system that leads to a diagnosis of cancer. The routes include:

  1. Two Week Wait

Patients are urgently referred by their GP for suspected cancer via the Two Week Wait system and are seen by a specialist within 2 weeks where they are diagnosed.

  1. GP referral

Diagnosis via a GP referral includes routine and urgent referrals where the patient was not referred under the Two Week Wait system.

  1. Emergency Presentation

Cancers can be diagnosed via emergency situations such as via A&E, emergency GP referral, emergency transfer or emergency admission.

  1. Outpatient

Outpatient cancer diagnoses include diagnoses via an elective route which started with an outpatient appointment that is either a self-referral or consultant to consultant referral. (It does not include those under the Two Week Wait referral system).

  1. Inpatient elective

Diagnosis via an inpatient elective route is where diagnosis occurs after the patient has been admitted into secondary care from a waiting list, or where the admission is booked or planned.

  1. Death Certificate Only

Diagnoses made by Death Certificate Only are made where there is no more information about the cancer diagnosis other than the cancer related death notifications. The date of diagnosis is the same as that of the date of death.

  1. Unknown

For some patients with a cancer diagnosis, there is no relevant data available to understand the route to diagnosis.

 

More information

If any of the statistical terms in this section of the brainstrust website are hard to understand, we recommend looking them up here:

Cancer Research UK’s Cancer Statistics Explained

http://www.cancerresearchuk.org/health-professional/cancer-statistics/cancer-stats-explained/statistics-terminology-explained#heading-Seven

If you are looking for help understanding terms relating specifically to brain tumours, and treatment, then the brainstrust glossary is available here:

https://www.brainstrust.org.uk/advice-glossary.php