Pilot Study Rectifies Real World Study Design To Support Regulatory Decision Making

Purpose: To evaluate the feasibility of a drug utilization study (DUS) investigating real-world use of Saxenda and Victoza in Europe. Economic and time constraints may incite researchers to avoid formalized pilot studies. Here, we report results of a pilot study which rectified the ensuing DUS protocol, thus ascertaining fit-for-purpose data availability and quality, and supporting regulatory decision making. Methods: A retrospective multicenter medical chart review in Germany and Italy, 6 months after Saxenda (liraglutide 3.0 mg, a once-daily human glucagon-like peptide-1 analog for weight management) launch, ahead of a full DUS. Collected data included: site characteristics, patient demographics, medical history, drug utilization (e.g., brand, dose, indication, weight-related comorbidities). Target study population: 100 initiators (25 Saxenda and 25 Victoza initiators in both Germany and Italy). Informed consent was obtained before medical-chart data extraction. Results: Overall, 218 sites were contacted. Fifteen sites (nine in Italy; six in Germany) enrolled initiators. There were 39 Saxenda initiators (33 in Italy; six in Germany) and 52 Victoza initiators (31 in Italy; 21 in Germany). Data were available for all Saxenda initiators and all Victoza initiators in Italy, and for 12 of 21 Victoza initiators in Germany. In nine of the 12 initiators, only target dose was recorded, with no current dose provided. Conclusions: The pilot study indicated a poor enrollment feasibility of Saxenda initiators in Germany and an unfit-for-purpose dose-related data quality. Based on these findings, refinements were implemented to the ensuing DUS protocol, thus ensuring robust real-world data to support regulatory decision making.