OBJECTIVES: To compare the usage of patient-reported outcome (PRO) claims in orphan drug approvals from the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) dated between 1/1/2012 and 31/12/2016.

METHODS: Orphan drug approval documentation was obtained from the EMA and FDA websites. PRO-related language was extracted from the Summaries of Product Characteristics (SmPCs) and Product Inserts (PIs) respectively.

RESULTS: Among 60 approvals by the EMA, 12 products for 13 (21.7%) orphan indications contained PRO language in their labels. In contrast, among 178 orphan drugs approved by the FDA only 16 products approved for 16 (9%) orphan indications contained PRO language. Twelve EMA labels contained PRO instruments based on symptoms, of which five also concerned patient functioning. Eight EMA approvals included PRO claims related to quality of life (QoL); most commonly for cancer treatment. However, all PRO instruments in FDA approvals concerned disease symptoms, and two also referred to patient functioning. The most common PRO instrument approved by FDA was a bleed-specific rating scale for four products for the treatment or prevention of bleeding episodes in patients with genetic bleeding disorders.

CONCLUSIONS: Orphan drugs approved by the EMA had higher PRO rates than those approved by the FDA. The EMA is also more inclined to grant HRQoL claims than the FDA and to allow such claims for oncology products.