Peterborough woman 'devastated' after brain cancer drug rejected for NHS use
Health bosses ruled it out over doubts on cost and survival rates
A Peterborough woman living with brain cancer says it’s "devastating" a promising new drug won’t be offered on the NHS.
Taylor Pepper’s tumour got smaller after taking vorasidenib, but she and other patients now face more intense treatments after health bosses ruled it out over cost and doubts it would improve survival rates.
"As much as I keep positive, it is very scary, but with the treatment, I know I'm in a good place," Taylor said.
"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."
Doubts over drug
The National Institute for Health and Care Excellence (NICE) has published draft guidance turning down vorasidenib over uncertainties in the economic data and lack of clarity on whether the drug improves overall survival.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.
Wait for decision causes 'stress and worry'
Taylor was diagnosed with an oligodendroglioma brain tumour last year following a routine eye test which detected swelling behind her right eye.
The 35-year-old, 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.
Taylor joined a clinical trial and now takes vorasidenib daily and will stay on it. The drug has shrunk her tumour.
"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," she said.
"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."
Taylor said her last MRI scan showed a small decrease in tumour size, and believes anyone that has the same diagnosis as her should be eligible to take the drug.
"I want to stay on this drug as it's allowed me to carry on as normal as I can," she added.
Decision not final
Dr Simon Newman, chief scientific officer at The Brain Tumour Charity, said he is disappointed by the decision and is urging NICE to reverse its decision.
"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," he said.
A NICE spokesperson said the views of people affected by its recommendations for a treatment "are important in the development of our guidance.
"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."
The spokesperson added that this is not NICE's final decision on vorasidenib and its draft guidance is open for public consultation.
NICE has also asked the company behind the drug for more information on key issues such as overall survival, with feedback to be considered by an independent committee at its next meeting.