New Brain Cancer Drug Rejected for NHS Use, Sparking Patient Outcry
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The National Institute for health and Care Excellence (NICE) has issued draft guidance rejecting the use of vorasidenib, a new drug shown to slow or halt the growth of certain brain tumors, leaving patients and advocates dismayed. The decision hinges on uncertainties surrounding the economic data and a lack of conclusive evidence demonstrating improved overall survival rates, according to the agency’s preliminary assessment.
The drug offers hope for individuals aged 12 and over diagnosed with low-grade glioma – a type of brain tumor – who have undergone surgery and carry either the IDH1 or IDH2 genetic mutations. Current standard treatment typically involves surgery followed by radiotherapy or chemotherapy, often with significant side effects. Clinical trials of vorasidenib, a daily pill, revealed promising results, including tumor shrinkage and a delay in the need for further intervention.
A Mother’s Fight: the Impact of the Decision
The rejection is especially devastating for patients like Taylor Pepper, 35, of Peterborough, who was diagnosed with an oligodendroglioma brain tumor in 2024 after a routine eye exam revealed swelling behind her right eye. forced to abandon her business and driving privileges, Pepper underwent surgery at Addenbrooke’s Hospital in Cambridge, but surgeons were unable to remove the entire tumor due to its location.
“As much as I keep positive, it is indeed very scary,” Pepper stated. “But with the treatment,I know I’m in a good place.” She credits vorasidenib with allowing her to live a more normal life and cherish time with her six-year-old daughter. “Being able to take this drug has given me a lot more benefits…It’s devastating that this is a no (from NICE),as 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 emphasized the benefit of a less harsh treatment option, stating, “Having a brain tumour is hard enough…you feel desperate.” Recent scans showed a small decrease in tumor size, reinforcing her desire to continue the treatment.
Prevalence and Clinical Trial Data
IDH-mutant low-grade gliomas represent one of the most frequently diagnosed primary brain tumors in individuals under 50. Approximately 300 people in England would have been eligible for vorasidenib had the drug been approved. A clinical trial involving 331 patients across ten countries demonstrated that vorasidenib effectively slowed or even stopped tumor growth in patients with grade 2 IDH-mutant glioma.
Advocates Express Dismay
Dawn emerton, a trustee of the Astro Brain Tumour Fund, whose son Shay benefited from the drug, expressed her disappointment. “I am dismayed that NICE has not made it available on the NHS,” she said.”If NICE reverses this decision, eligible patients could experience improved quality of life, fewer seizures and delayed treatment with harsher therapies.” She further argued that approval would signal the UK’s commitment to medical innovation and offer renewed hope to brain tumor patients.
Dr. Simon Newman, chief scientific officer at The Brain Tumour Charity, echoed these concerns. “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,” he noted. He urged the community to advocate for a reversal of the decision, emphasizing that “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.”
