Harnessing the Power of Real-World Evidence (RWE): A Checklist to Ensure Regulatory-Grade Data Quality

Abstract

The role of real-world evidence (RWE) in regulatory, drug development, and healthcare decision-making is rapidly expanding. Recent advances have increased the complexity of cancer care and widened the gap between randomized clinical trial (RCT) results and the evidence needed for real-world clinical decisions. Instead of remaining invisible, data from the >95% of cancer patients treated outside of clinical trials can help fill this void.

Figure 1

The journey from data to evidence. Real‐world data (RWD) are data that are routinely collected in the form of electronic health records (EHRs), patient disease registries, wearables, genomic datasets, medical claims registries, and others. These data can be aggregated, linked, and processed to produce key conclusions in the form of real‐world evidence (RWE). The proposed checklist can be used to assess if the quality of the RWD is regulatory‐grade.

Figure 2

Use cases for RWE in the drug development cycle. RWE can be valuable across the lifecycle of drug development. In a drug's predevelopment stages, RWE helps inform clinical development strategies and study planning. During clinical development stages, RWE may inform trial design and feasibility (e.g., patient enrollment, inclusion and exclusion criteria), and serve as a synthetic control arm. Following drug approval, RWE may help fulfill postmarketing commitments and potentially support label expansions. Finally, in clinical practice, RWE may assist personalization of treatment by offering clinicians and patients a deeper understanding of the nuances of drug performance in the real world.