Work Packages

Work Package 5 - Rare diseases technologies and value assessment

Leader Panos Kanavos-London School of Economics and Political Sciences, United Kingdom



  • to understand the criteria used by competent authorities in selected Member States in assessing the value of orphan medicines relying on evidence-based medicine (EBM) and cost-effectiveness analysis (CEA) in each jurisdiction
  • by drawing on a selection of orphan drug appraisals in a number of EU countries over the January 2006 to December 2012 period and across therapeutic classes, to determine the criteria used to evaluate these drugs and  the way clinical practice guidelines are developed across the selected sample of drugs; and
  • to explore BP based on the evidence collected and share these across EU Member States.



D5.1-Identification of best practices (M22)