Unituxin Appeal Win Suggests NICE STA Process Is Not Appropriate For Pediatric Orphan Drugs

 
• By Francesca Bruce

The UK charity Solving Kids’ Cancer has won an appeal against a NICE decision declining to recommend United Therapeutics Unituxin (dinutuximab) for treating high-risk neuroblastoma in pediatric populations. The case raises questions over whether NICE's standard technology appraisals are appropriate for medicines for rare conditions in children.

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The appeal panel of NICE, the health technology appraisal institute for England and Wales, has upheld an appeal by the charity Solving Kids’ Cancer against NICE’s decision not to recommend United Therapeutics Corp.'s Unituxin (dinutuximab) for use in a small number of children with high-risk neuroblastoma.

The panel agreed with the appellant that NICE had exceeded its powers and that its recommendation was unreasonable in the...

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