Final Meeting Report and Proposed Framework Changes

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Orphan Drug Assessment: Final Meeting Report and Proposed Framework Changes

This is an outdated version of the proposed adaptations of “ultra rare” conditions. To see the most recent version, click here.


This paper presents a set of proposed adaptations to the ICER value assessment framework when the topic under review is a treatment for an “ultra-rare” condition. These proposals were open for a 60-day public comment period, after which time a final set of adaptations were published. In the future, the formal update to these methods will be performed simultaneously with the updates to the overall value assessment framework every two years.



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