Title: HTA46 Outcomes of Benefit Assessments of Orphan Drugs Without Orphan Designation in Germany
Abstract: Orphan drugs with orphan designation (OD) are granted an added benefit by law in the German HTA process. However, some drugs indicated for rare diseases do not obtain or lose OD, leading to regular benefit assessments without orphan privilege. The objective of the current study is to investigate the outcomes of benefit assessments of orphan drugs without OD in Germany compared to non-orphan drugs and orphan drugs with OD.