Journal of Comparative Effectiveness Research Vol. Epub ahead of print No. | Special Report

Replication of randomized clinical trial results using real-world data: paving the way for effectiveness decisions


The US FDA is preparing guidance about using real-world evidence (RWE) to support decisions about product effectiveness and guidance regarding whether and how observational study designs may generate evidence to support decisions related to product effectiveness is expected by the end of 2021. Several ongoing efforts aim to replicate randomized clinical trial (RCT) results using RWE, with the intent of identifying circumstances and methods that provide valid evidence of drug effects.

Lack of concordance between RCT and RWE results may not be due to faulty methods, but rather to the challenges with emulating RCTs, differences in healthcare settings and patient populations, differences in effect measures and data analysis, bias, and/or the efficacy–effectiveness gap. In fact, for some decisions, RWE may lead to better understanding of how treatments work in usual care settings than a more constrained view from RCTs. Efforts to reconcile the role and opportunities for generating complementary evidence from RWE and RCTs will advance regulatory science.

In this Special Report, investigators from Eli Lilly & Company (IN, USA) and IQVIA (MA, USA) discuss the complementary nature of data obtained from randomized clinical trials and observational studies, and how use of the latter to support regulatory decisions could yield considerable gains.

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