OBJECTIVES: Benefit assessment usually requires RCT data. Orphan drugs are granted additional benefit by law, but not drugs with conditional or exceptional approval or PUMA. The objective of this study was to assess how their status is handled in benefit assessment. METHODS: All resolutions until June 2016 were analyzed whether they have been approved by EMA under these circumstances. Those which do were assessed regarding underlying evidence, extend of additional benefit and other aspects of the resolution. RESULTS: