This two day meeting focused on “Evaluating RWE from Observational Studies in Regulatory Decision-Making: Lessons Learned from Trial Replication Analyses”. Preliminary results from ongoing observational trials designed to replicate the results of randomized clinical trials were presented.
The strengths and limitations of observational studies were discussed in the context of evaluating a drug product’s effectiveness for regulatory decision-making. While some of the studies successfully duplicated results based on prespecified metrics, showing that supplication is possible with the fit for purpose study designs and data, others did not.
This meeting contemplated the lessons learned from the work and potential next steps to keep advancing our understanding of how and when RWE may support regulatory decisions. An appropriate study design aimed to address an explicit question is critical and the TARGET trial approach for observational studies was provided as a good starting point and framework for reliable observational studies.