Final Meeting Report and Proposed Framework Changes

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Orphan Drug Assessment: Final Meeting Report and Proposed Framework Changes

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 will be open for a 60-day public comment period, following which ICER will reflect on comments received, pursue additional feedback from stakeholders, and post a final version of adapted methods in the fall of 2017. 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.

Comments can be emailed as Word documents to and must be received by 5pm ET on September 25, 2017.


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