The Role of Patient-Reported Outcomes in Marketing Authorization Applications and Product Labelling (2018-2020)

Abstract

BACKGROUND Little is known about the role Patient-Reported Outcomes (PROs) play in, the main clinical studies, the benefit-risk assessment, product labelling, and which determinants play a role herein. METHOD We evaluated the European Public Assessment Reports (EPARs) of new medicinal agents with a regulatory decision (approved or refused) by the EMA between January 1, 2018 and December 31, 2020. The primary and secondary endpoints of the main studies, benefit-risk balance, effect tables (results that have a strong influence on the assessment) and SmPC (for patient care decision-making) were evaluated for any mention of PROs. RESULTS A total of 149 products met our criteria. Of these, 84 (56.40%) mentioned PROs. PROs were used significantly more as a secondary endpoint and contributed significantly more to patient care decision-making by chronic treatment compared to acute and preventive treatment. Furthermore, the disease category played a significant role in PRO use. For example, by respiratory conditions, PROs weighed in on 100% of the effect tables and SmPC. In contrast to infectious disease, where PROs weighed in on 5%, and 0% of their effect tables and SmPC, respectively. Moreover, PROs from blinded studies contributed significantly more to patient care decision-making compared to open-label studies 3.836 (95% Cl 1.802-8.167, p < 0.01). CONCLUSION In conclusion, different product determinants play a role in the utilization and contribution of PROs to the assessment and patient care decision-making. Overall, more PRO claims are being approved by the EMA and a gradual increase are seen in their contribution to support drug licensing. They are weighing in more and more on the benefit-risk balance assessment.