Which clinical trial designs and statistical approaches have been used in assessments of orphan maintenance by the European Medicines Agency between 2012 and 2022? A cross-sectional study.

Objectives In the European Union, a new orphan medicinal product must demonstrate ‘significant benefit’ over approved medicinal products targeting the same indication. To demonstrate a significant benefit, comparisons between the new product and the already approved medicinal products—either directl...

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Bibliographic Details
Main Authors: Fabian Windfuhr, Kristina Larsson, Theodor Framke, Florian Lasch
Format: Article
Language:English
Published: BMJ Publishing Group 2024-12-01
Series:BMJ Open
Online Access:https://bmjopen.bmj.com/content/14/12/e086171.full
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