Patients with brain cancer express disappointment after drug rejected for NHS

Patients with brain cancer have expressed disappointment after a drug that slows – or even stops – tumours growing was rejected for use on the NHS.

The National Institute for Health and Care Excellence (Nice) has published draft guidance turning down vorasidenib over uncertainties in the economic data and lack of clarity on whether the drug improves overall survival.

Vorasidenib is for people aged 12 or over who have had surgery for types of low-grade glioma with either the IDH1 or IDH2 genetic mutations.

Patients who have taken the drug in clinical trials report remarkable results, including their tumours shrinking.

IDH-mutant low-grade gliomas are one of the most common primary brain tumours diagnosed in people under 50 and around 300 people in England would have been eligible for vorasidenib.

Current treatment includes surgery, followed by radiotherapy or chemotherapy.

Patients are then monitored for tumour re-growth before further treatment is considered.

Vorasidenib is a daily pill that works by inhibiting the abnormal enzymes caused by the IDH mutation, which drive tumour growth.

A clinical trial on 331 patients from 10 countries found that vorasidenib slowed or even stopped tumour growth in patients with grade 2 IDH-mutant glioma.

The drug also delayed the time before patients needed another intervention.

Taylor Pepper, 35, from Peterborough, was diagnosed with an oligodendroglioma brain tumour in 2024 following a routine eye test which detected swelling behind her right eye.

Mrs Pepper, who is married and has a six-year-old daughter, had to stop running her business and give up her driving licence.

Surgeons at Addenbrooke’s Hospital in Cambridge removed most of her tumour but could not take it all because of its location.

Mrs Pepper joined a clinical trial and now takes vorasidenib daily and will stay on it. The drug has shrunk her tumour.

She said: “As much as I keep positive, it is very scary … but with the treatment I know I’m in a good place.

“Being able to take this drug has given me a lot more benefits. I’m able to live a normal life and make memories with my little girl.

“It’s devastating that this is a no (from Nice) because brain tumour patients have had to wait a long time for decisions on treatment anyway so having to wait longer for a decision on this will cause a lot of stress and worry.

“They’ve managed to find a drug that’s not as harsh a treatment as chemo and radiotherapy, meaning we’re able to do so much more.

“Having a brain tumour is hard enough, especially when you’re told it’s incurable and that chemo and radiotherapy would only make it worse. You feel desperate.

“My last MRI scan showed a small decrease in size which is incredible so I want to stay on this drug as it’s allowed me to carry on as normal as I can.

“Anyone facing this diagnosis should be eligible for this drug.”

Dawn Emerton, a trustee of Astro Brain Tumour Fund (ABTF) whose son Shay received the drug, said vorasidenib is “first new treatment for low-grade gliomas in decades, offering real hope to patients”.

She added: “I am dismayed that Nice has not made it available on the NHS.

“If Nice reverses this decision, eligible patients could experience improved quality of life, fewer seizures, and delayed treatment with harsher therapies.

“It would also demonstrate the UK’s commitment to innovation and provide all patients with hope for progress in brain tumour treatment.”

Dr Simon Newman, chief scientific officer at The Brain Tumour Charity, said: “This draft decision is very disappointing as it means more patients will have chemotherapy and radiotherapy earlier than necessary, which can be effective, but which can have significant long-term side effects.

“We are urgently asking our community to help turn this draft ‘no’ from Nice into a ‘yes’.

“Outcomes for brain tumour patients remain among the poorest of any cancer, so we need to work with all stakeholders to ensure great science is translated into medicines that are available on the NHS as quickly as possible.”

A Nice spokesperson said the guidance was open to consultation, adding: “We are of course aware of the impact that a decision not to recommend a treatment can have…

“However, we also need to consider the evidence of a treatment’s clinical and cost-effectiveness in our decision-making.

“Clinical trial evidence ​shows​ that vorasidenib increases how long people have before their cancer gets worse compared with a placebo. But there is not enough evidence to show if vorasidenib extends how long people with the condition live.”