It is currently looking like children and adolescents with neuroblastoma will not be getting routine access to United Therapeutics’ Unituxin on the National Health Service in England and Wales, after cost regulators deemed the drug too expensive. 

The National Institute for Health and Care Excellence has this morning launched a consultation on its draft guidelines ruling that Unituxin (dinutuximab) for high-risk neuroblastoma in patients aged 12 months to 17 years is not a cost-effective use of NHS resources.

The therapy won European approval in August, after clinical data showed both improved event-free and overall survival in a multicenter, open-label, randomised trial sponsored by the US National Cancer Institute and conducted by the Children's Oncology Group.

But side effects can be severe, including nerve cell irritation causing severe pain and life-threatening infusion reactions, and NICEs Committee concluded that the drug’s incremental cost is likely to be £139,600 per quality-adjusted life year (QALY) gained, thus falling well outside the range of what is considered value for money, particularly as it failed to meet short-life criteria.