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UK: NICE Wins Argument Over Appraisal Of BioMarin Rare Disease Drug

BioMarin To Re-Engage With Process But Is Doubtful Of Outcome

Executive Summary

A decision by England’s High Court to reject claims that HTA body NICE acted unlawfully when evaluating BioMarin’s PKU treatment has quashed hopes that NICE’s Highly Specialized Technologies program will become more widely used to evaluate rare disease treatments. Lawyers representing a PKU patient are planning to appeal the judgement.

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