15 July 2016 - NICE has rejected United Therapeutics’ Unituxin (dinutuximab) in combination therapy to treat a rare cancer affecting children saying the size of the benefits from the drug remained uncertain.

The regulator said it does not recommend the drug to treat high-risk neuroblastoma in children and young people aged 1–17 years whose disease has at least partially responded to induction chemotherapy, myeloablative therapy and autologous stem cell transplant.

Evidence from the trial showed the treatment caused a number of severe side effects including low blood pressure, pain, hypersensitivity and fever - although effects stopped when treatment did, Nice said in a statement.

Dinutuximab costs £6,390 per 17.5 mg infusion, and a complete course of treatment (excluding those drugs it is given with) costs £127,800.

The regulator said the treatment did not meet the criteria to be considered for use in the Cancer Drugs Fund as it was not cost-effective, even with the patient access scheme discount proposed by the company, and the clinical uncertainties could not be addressed from further data collection within two years.

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