Evaluating the Use of Nonrandomized Real-World Data Analyses for Regulatory Decision Making

Area of Science:

• Health Services Research
• Biostatistics
• Regulatory Science

Background:

• Real-world evidence (RWE) from longitudinal healthcare data is increasingly utilized outside of traditional clinical trials.
• Analysis of healthcare databases is a key focus for RWE generation.

Purpose of the Study:

• To discuss the role of RWE in drug regulation, focusing on healthcare database analyses.
• To present current and potential uses of RWE in supporting regulatory decisions.
• To propose a structured process for RWE study implementation and evaluation.

Main Methods:

• Review of existing literature and case examples of RWE application in drug regulation.
• Evaluation of the empirical evidence base for validity, transparency, and reproducibility of RWE from healthcare databases.
• Discussion of key considerations for designing and assessing RWE studies.

Main Results:

• RWE is already being used in specific regulatory contexts and has potential for broader application.
• Key issues for investigators and regulators in RWE study design and evaluation were identified.
• An evidence base for RWE validity, transparency, and reproducibility is developing, but further work is required.

Conclusions:

• RWE from healthcare databases holds significant promise for informing drug regulatory decisions.
• Ensuring the validity, transparency, and reproducibility of RWE analyses is crucial for regulatory acceptance.
• Continued development and standardization of RWE methodologies are necessary to provide decision-ready evidence on treatment effectiveness and safety.