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Dealing with Uncertainty and Accounting for Social Value Judgments in Assessments of Orphan Drugs: Evidence from Four European Countries

Elena Nicod, Karen Berg Brigham, Isabelle Durand-Zaleski, Panos Kanavos
May 2017

Orphan medicinal products (OMPs) are often characterised by lower quality evidence than more conventional medicines. To address this, health technology assessment (HTA) bodies have implemented different strategies in their evaluation process. In order to understand the variation in HTA recommendation outcomes for OMPs across countries, this study aimed to elicit the views of HTA bodies about their approaches to OMP evaluation. A series of semi-structured interviews with HTA representatives from England, Scotland, Sweden, and France were conducted. Interviews were transcribed and analysed using qualitative thematic data analysis. Results highlight general agreement around evidentiary requirements and preferences for OMPs between HTA bodies. Differences were identified around how uncertainty should be considered in terms of, for example, the extent to which it is acceptable or the extent to which key concepts (e.g., unmet need, innovation, or disease severity) have a weight in decisions.

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