SW-1F - Integrating Real-World Data with Randomized Controlled Trials: Advancing Evidence Generation

Background: Combining data from Randomized Controlled Trials (RCTs) and Real-World Data (RWD) can enhance research and regulatory insights. While RCTs are often considered the gold standard, they have limitations such as narrow generalizability, high costs, and long timelines. RWD complements RCTs by capturing diverse patient populations and real-world treatment outcomes. Integrating these data sources, researchers can generate deeper insights into treatment effectiveness, expand findings to broader populations, and enhance decision-making.
This approach aligns with recent FDA guidance documents, including the Real-World Evidence Framework, guidance on External Control Arms, and draft guidance on Integrating RCTs into Routine Clinical Practice, highlighting FDA’s recognition of RWD’s potential improve the applicability of clinical findings to more diverse patient populations.

Objectives: • Review methods for integrating RWD with RCT data to overcome trial limitations.
• Provide practical insights into using combined data sources to generate evidence.
• Showcase global applicability of integrated data through real-world case studies.
• Engage researchers, regulators, and industry professionals in leveraging integrated data for pharmacoepidemiology research.

Description: This workshop will provide a clear and practical overview of how integrating RWD with RCT data can enhance evidence generation. Through real-world use cases, we will illustrate how RWE and RCTs are integrated in practice, aiming to equip participants with actionable insights.
Key takeaways include methodologies for combining data, exploring global datasets, and implementation insights. Presentations include:
• Ms. Grip and Dr. Geale: Creating external control arms to generate additional evidence: a cytarabine trial in acute myeloid leukemia.
• Dr. Christian: A gated approach for ensuring relevance and reliability of your external control arm to the intervention population
• Dr. Young: Integrating RWD with RCTs to extend trial results to relevant target populations: evaluating anticoagulation in percutaneous coronary intervention
• Dr. Ross: Methodological considerations for extending inference when trial activities affect treatment adherence
The workshop will conclude with a 30-minute interactive discussion and Q&A led by Dr. Ritchey, featuring live interactive polling to capture audience perspectives and prioritize topics in real time. Key discussion questions include:
• What are the most promising areas for integrating RCTs and RWD?
• What strategies can accelerate this integration for better decision making?
• What challenges are slowing progress, and how can we overcome them?