Abstract
To evaluate and characterize the usage of patient-reported outcome (PRO) claims in orphan drug approvals from the European Medicines Agency (EMA) dated between 1/1/2012 and 31/12/2016. Orphan drug approval documentation was obtained from the EMA website. PRO-related language was extracted from the Summaries of Product Characteristics (SmPCs). Among 60 approvals that met the inclusion criteria, 12 products approved by the EMA for 13 (21.7%) orphan indications contained PRO language in the Clinical Studies section of the SmPC. Twelve SmPCs contained PRO instruments based on symptoms, of which five also concerned patient functioning. Eight approvals included PRO claims related to quality of life (QoL); most commonly for cancer treatment. The rate of PRO claims was lower for orphan drugs specifically than for all drug approvals by the EMA. Further, the EMA was particularly inclined to grant PRO claims concerning Health-Related QoL; mostly for cancer treatments.
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