Abstract 

Patient experience data (PED), including clinical outcome assessments, patient preference studies and qualitative insights, capture disease burden and meaningful post-treatment change. The aim of this review was to evaluate PED inclusion in regulatory assessments and labelling for rare disease products and examine regional acceptance. This was achieved using clinical study reports, US Food and Drug Administration and European Medicines Agency review documents and labelling negotiations for 12 rare disease products approved between 1994 and 2025.

This article describes the review carried out and summarises the findings regarding rates of PED inclusion in review documents and labelling in the EU and US.