The Aetion Coalition to Advance Real-World Evidence through Randomized Controlled Trial Emulation Initiative:...

Area of Science:

• Oncology
• Health Informatics
• Biostatistics

Background:

• Recent advancements have increased the volume of real-world data (RWD) from clinical practice.
• RWD holds potential for supporting clinical and regulatory decisions in healthcare.
• In oncology, RWD offers complementary advantages to randomized controlled trials (RCTs) and enables ethically or logistically challenging investigations.

Purpose of the Study:

• To evaluate the reliability of RWD for drawing conclusions on drug effectiveness in oncology.
• To conduct 10 RWD-based studies that emulate RCTs using a systematic, protocol-driven approach.
• To inform stakeholders on RWD applications for product labeling, drug safety, and comparative effectiveness.

Main Methods:

• Emulating randomized controlled trials (RCTs) using real-world data (RWD).
• Employing a systematic, protocol-driven approach for study design and analysis.
• Addressing concerns of unmeasured confounding and bias inherent in RWD.

Main Results:

• (Study results are pending and will be reported upon completion.)
• Anticipated to provide insights into the validity of RWD for oncology research.
• Expected to demonstrate the feasibility of emulating RCTs with RWD.

Conclusions:

• RWD holds significant promise for generating clinical evidence in oncology, complementing RCTs.
• Careful study design and analysis are crucial to mitigate bias and confounding when using RWD.
• This research aims to enhance the understanding and application of RWD in oncology decision-making.