Scientific and Social Value Judgments for Orphan Drugs in Health Technology Assessment
Elena Nicod, Panos Kanavos
Orphan medicinal products (OMPs) are often characterised by uncertain evidence and high incremental cost effectiveness ratios (ICERs). In such cases, health technology assessment (HTA) bodies may factor in “other considerations” beyond more routine measures when judging their relative value. Looking across different publicly available HTA reports, this study sought to better understand the dimensions of value used across countries by systemically identifying the scientific and social value judgements made in England, Scotland, Sweden, and France. The analysis focused on ten orphan drugs, and identified and classified these “other considerations”. Better defining and grouping these value considerations and determinants can help improve HTA transparency, accountability, and consistency across OMP decisions.