*Important Note: This is a no cost in person workshop

Real-world data (RWD) offers an opportunity to inform regulatory decision-making, particularly in rare diseases. However, variability in data sources and inconsistent data quality often limit the utility of RWD for regulatory decision-making. A central challenge is determining whether a given RWD source is fit-for-purpose before substantial analytical investment.

This free workshop will address the challenge by focusing on the evaluation of RWD rather than downstream analyses. Anchored in published FDA guidance and gaps identified in the LEADER 3D Public Report, the session introduces a structured framework for assessing RWD across four core dimensions: (1) relevance (availability of required data elements), (2) observability (continuity of care and longitudinal follow-up), (3) structural comparability (ability to define populations and comparators), and (4) reliability (data capture, consistency, missingness, and provenance), with explicit consideration of the underlying data generating process and its implications. In this interactive session, participants will work in small groups to apply this framework to evaluate theoretical RWD sources. Data strengths and deficiencies will be identified and assessments made as to whether the RWD sets are fit-for-purpose, supportive, or unsuitable for regulatory use. The session will conclude with a synthesis of data limitations and identification of priority areas for tool development and standardization.

Participants: the workshop is intended for stakeholders across industry, academia, patient organizations, and regulatory bodies seeking to advance the generation and use of RWD to support regulatory decision-making. Outcomes include a shared approach to early feasibility assessment and actionable insights to guide future RWD collection and curation strategies.

*Agenda: TBA