Understanding and use of real world evidence (RWE) is on the increase in the medical community. While evidence from randomised controlled trials (RCTs) is still considered to be the gold standard for measuring treatment efficacy and safety, RWE studies are increasingly being recognised as providing important additional evidence. Notably, the FDA is considering the role of RWE in the evaluation of pharmaceutical products, having recently issued guidelines regarding the use of RWE in medical device regulatory decisions. An article published in Drug Discovery and Development last month outlines the uses and limitations of RWE and asks, “why do we need RWE and how can we use it?”
The author, Dr Shafrin, describes how the information garnered from RWE studies becomes very important when RCTs are not possible or when populations or conditions in RCTs do not reflect real world practice. For example, treatment efficacy may be high in an RCT where adherence is good, but may be affected in real world practice if adherence is poor. RWE may also be key in the drug approval process for certain rare diseases, where the sample size may be too low for an RCT to be conducted. Moreover, RWE can be used to assess long-term efficacy and safety, beyond the time-frame possible in a standard RCT. However, Dr Shafrin emphasises that robust study design is important for RWE studies, as is careful selection of relevant data sources, as outlined in a recent report from Duke University.
Dr Shafrin concludes that RWE has an important role to play in providing healthcare stakeholders with evidence to make better regulatory, policy, coverage, and treatment decisions. This is in line with reports earlier this year that RWE should be used earlier in the drug development pathway and no longer seen as merely a supplement to RCTs.