Research Scientist Evidera PPD, part of Thermo Fisher Scientific Lidingö, Sweden
Background: Post-approval efficacy/effectiveness studies can demonstrate or confirm the effectiveness of an authorized treatment once commercially available, especially in subpopulations under-represented or excluded from trials, such as pediatric patients. The EU PAS Register® is a publicly-available source of information on post-approval studies (PAS) .
Objectives: To provide an overview of key characteristics of finalized observational effectiveness evaluation studies in the EU PAS Register® with a focus on studies that included pediatric patients.
Methods: We have searched for observational effectiveness evaluation studies in the EU PAS Register®. The following characteristics were extracted directly from the search feature of the EU PAS Register®, for all observational studies with a reported scope of “Effectiveness evaluation”, as of 6 Feb 2023: RMP Category, age category, study status, date last updated. Manual review was conducted on a sub-set of the above studies (included pediatric patients, finalized status) to extract key characteristics including: date first registered, therapeutic area, study design, data sources, and whether the study was requested by a regulator.
Results: 768 effectiveness evaluation studies were identified. The vast majority of studies were last updated in the EU PAS Register® after 2016 (92.2%), and the proportion of studies last updated increased in each consecutive year from 2017 to 2022 by 6.4%, 10.2%, 12.0%, 13.7%, 18.4%, and 28.4%, respectively. Of the effectiveness evaluation studies, 644 (83.9%) were observational, of which 189 included pediatric patients. There were 80 finalized observational, pediatric effectiveness evaluation studies, a majority of which reported that the primary or only scope of the study was effectiveness evaluation. Approximately half had RMP category “Not Applicable”, while only 1 was registered as RMP category 2. The remaining were registered as RMP category 1 (12.5%), category 3 (16.3%), “Non-EU RMP only” (12.5%), or missing information on RMP category (10%). More than half of the studies had a year first registered before 2017. Substantial heterogeneity in terms of study design, data sources and therapeutic areas was observed.
Conclusions: Since 2017, the number of studies in the EU PAS register that include effectiveness evaluation within study scope has increased considerably. Notably, a majority of finalized observational PAS studies involving pediatrics had effectiveness evaluation as their primary or only scope. The majority were first registered prior to 2017, which may indicate the long duration needed to complete such studies. Further understanding of finalized studies may help guide design of future PAES studies.
