Creating and using real-world evidence to answer questions about clinical effectiveness

Area of Science:

• Health research methodologies
• Evidence-based medicine
• Data science in healthcare

Background:

• Randomized controlled trials (RCTs) provide valuable evidence but have limitations.
• There is a need for new evidence forms to complement RCTs.
• Real-World Evidence (RWE) is a promising but undefined concept.

Purpose of the Study:

• To define Real-World Evidence (RWE) as a rigorous research process.
• To outline the essential components of a robust RWE study.
• To highlight the potential of RWE in complementing RCTs.

Main Methods:

• Defining RWE through a structured research process.
• Emphasizing a priori hypothesis/research question formulation.
• Requiring critical appraisal of data sources (e.g., EHRs, big data).
• Incorporating appropriate analytical methods, including advanced analytics.
• Advocating for published protocols to ensure rigor.

Main Results:

• RWE studies are more inclusive than RCTs.
• RWE can evaluate real-world effectiveness and manage complex conditions like multimorbidity.
• Computerized medical records and big data are rich sources for RWE.
• Guidance is needed to assess RWE study rigor and validate advanced analytics.
• RCTs may increasingly focus on safety and regulatory compliance.

Conclusions:

• RWE, when rigorously conducted, can assess real-world efficacy.
• The role of RCTs may shift towards safety and regulatory aspects.
• Further development in RWE methodology and validation is crucial for its acceptance.