OBJECTIVES:

This study investigates the consideration of patient-reported outcomes (PROs) and related measures (PROMs) in the regulatory decision-making for new drugs approval in Europe together with characteristics associated to their use.

METHODS:

All drugs for human use authorized by the European Medicines Agency (EMA) between January 2017 and December 2021 were identified and the corresponding European Public Assessment Reports (EPARs) downloaded for review. An ad hoc template was created to record systematically relevant information (e.g., therapeutic area, generic/biosimilar, orphan status, authorization date) and PROs/PROMs characteristics (e.g., primary/secondary endpoint, generic/specific measure, assessment time). The data extraction form was first piloted on a sample of 20 reports by three reviewers independently. Multivariate logistic regression was performed in Stata to identify the variables that impacted on the use of patient-reported evidence in EPARs.

RESULTS:

A total of 403 EPARs were included in the analysis; of these, 197 (48.9%) reported any use of PROs/PROMs. The use of PROMs was more common in some therapeutic areas than others (e.g., rheumatology: 94.4% vs. infectious diseases: 18.5%), and for orphan drugs (66.7%). The use of PROs/PROMs has increased over time (from 45.9% in 2017 to 55.0% in 2021), however in over 70% of cases PROs were secondary or exploratory endpoints. The most common PRO was quality of life (30.7%). Among the PROMs, 167 (25.3%) were generic. EQ-5D (9.5%), SF-36/SF-12 (6.1%) and EORTC QLQ-C30 (5.1%) were the instruments most frequently used. The average number of measures for each EPAR was 1.6 (range: 0-14). The likelihood of using PROs/PROMs was negatively affected by generic (OR=0.01, p<0.00) and biosimilar status (OR=0.46, p=0.02).

CONCLUSIONS:

In 2020, EMA launched a strategic view to reinforce patient relevance in evidence generation for pharmaceuticals. These results suggest PROs/PROMs are considered in less than half of total drug assessments. Harmonizing PROMs selection would promote their consideration.