Table 1.
Characteristic | RCTs | RWE |
---|---|---|
Standard of evidence | Gold standard | Complementary to RCTs |
Cost | Costly to develop and conduct | Less costly |
Patient population | Well-defined within constraints of specific inclusion criteria Results reflect outcomes in limited population |
Broader and promotes evaluation of patient populations less often studied in clinical trials Patient data derived from other sources, including insurance claims |
Sample size | Limited Requires sample size calculation to be performed in advance |
Orders of magnitude larger |
Efficacy | Randomized and blinding lead to minimized risk of data bias and confounding | Randomization and blinding may not be feasible Risk of unrecognized data bias and confounding greater |
Adverse events | Only more frequently occurring adverse events revealed | Can reveal adverse events with much lower frequency and those requiring longer exposure to occur |
Approval or clearance of new medical products | Considered the gold standard necessary for new drug approval, and when feasible for new device approval | Not generally accepted for approving new drugs but can complement RCT findings, accepted for new device indications |
Role in diabetes | Define efficacy and provide a preliminary safety profile in a well-defined and controlled population | Allows estimation of more realistic treatment effects of a wide range of diabetes interventions and evaluation of interactions such as social determinants of health and comorbidities |
Other issues | May be less useful when strong signals are available from RWE or early-phase trials | Facilitates postmarketing surveillance of adverse events and assessment of the product effectiveness Results may be less credible due when a control group is not included |
Source: Adapted from Gyawali et al.7