Practical considerations of utilizing propensity score methods in clinical development using real-world and...

Area of Science:

• Pharmacovigilance and Drug Development
• Biostatistics and Epidemiology
• Real-World Data Analytics

Background:

• The increasing availability of Real-World Data (RWD) and Real-World Evidence (RWE) presents new opportunities for drug clinical development and lifecycle management.
• RWD and RWE can enhance clinical trial design, planning, and execution, serving as complements or substitutes for traditional trials.
• Rigorous statistical methodologies are crucial for ensuring the robustness and reliability of analyses using RWD.

Purpose of the Study:

• To provide a systematic review and discussion of propensity score methods for the rigorous application of RWD in clinical development.
• To introduce commonly used and emerging propensity score methods, detailing their advantages and disadvantages.
• To illustrate practical considerations for utilizing propensity score methods through case studies.

Main Methods:

• Review of statistical methodologies for analyzing RWD, with a focus on propensity score methods.
• Discussion of propensity score methods, including their pros and cons.
• Presentation of three case studies demonstrating the practical application of propensity score methods in study design and evaluation using RWD and historical data.

Main Results:

• Propensity score methods are widely used for analyzing non-randomized trials with indirect comparisons.
• The paper discusses practical considerations for utilizing propensity score methods in clinical development.
• Case studies illustrate the application of these methods in real-world and historical data analysis.

Conclusions:

• Effective utilization of RWD and RWE in clinical development necessitates robust statistical methodologies like propensity score methods.
• A systematic approach, including careful consideration of patient populations, endpoints, covariates, and sensitivity analyses, is essential for successful implementation.
• This paper aims to fill the gap in systematic reviews concerning the practical application of propensity score methods in clinical development.