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

Executive Summary

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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