Pilot study rectifies real-world study design to support regulatory
decision making
Abstract
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.