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.
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.
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).
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.||