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UK: NICE’s Rare Disease Gatekeeping Comes Under The Microscope

 
• By Francesca Bruce

A judicial review of how health technology appraisal body NICE decided which appraisal route BioMarin’s rare disease drug Kuvan would take could have lasting implications for future treatments.

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NICE decision making under is review at the High Court

The High Court Of England And Wales is poised to hear a challenge to the way UK health technology appraisal body NICE has restricted access to the process designed for appraising rare disease treatments. If the court upholds the claim under review, more rare disease products could end up being appraised through the more “generous” Highly Specialised Technologies (HST) program designed specifically for rare and very rare disease treatments.

The case will be heard on 30 January. It is being brought to the high court on behalf of an 11-year-old patient who suffers from the rare metabolic disease phenylketonuria (PKU), and who has been unable to access treatment in the form of BioMarin’s Kuvan (sapropterin)

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