Table 2.

Strengths and limitations of real‐world evidence about treatment efficacy

	Strengths of RWE	Limitations of RWE
Description of treatment efficacy in “clinical practice” heterogeneous population	Lower selection bias in study population compared with RCTs	The absence of a control group does not allow the accurate estimation of the efficacy compared with previous/alternative standard Quality of data sources, collection, and verification could be lower compared with clinical trials
Description of treatment efficacy in special patient populations	Potential focus on efficacy in special patient populations, often under‐represented in (or excluded from) RCTs
Evaluation of efficacy in settings where an RCT has not been performed (e.g., rare subpopulations)	Production of evidence in a setting suffering from the absence of an RCT
Nonrandomized comparison of patients receiving different treatments for the same condition	Production of evidence in a setting suffering from the absence of a direct comparison	Selection bias is inherent in nonrandomized groups and cannot be avoided even with statistical techniques (e.g., propensity score, multivariate analysis) Quality of data sources, collection, and verification could be lower compared with clinical trials
Use of treatments within specific geographic and/or economic contexts	Production of evidence in patients with different characteristics compared with RCTs (due to ethnicity, characteristics of disease, other treatments) Production of pharmaco‐economic data within a specific country or a specific health system	Results produced within a specific geographic and/or economic context cannot be applied to different contexts

Abbreviations: RCT, randomized controlled trial; RWE, real‐world evidence.