Developing Real-world Evidence-Ready Datasets: Time for Clinician Engagement

Abstract

Purpose of review: Real-world data (RWD) applications in healthcare that support learning health systems and pragmatic clinical trials are gaining momentum, largely due to legislation supporting real-world evidence (RWE) for drug approvals. Clinical notes are thought to be the cornerstone of RWD applications, particularly for conditions with limited effective treatments, extrapolation of treatments from other conditions, or heterogenous disease biology and clinical phenotypes.

Recent findings: Here, we discuss current issues in applying RWD captured at the point-of-care and provide a framework for clinicians to engage in RWD collection. To achieve clinically meaningful results, RWD must be reliably captured using consistent terminology in the description of our patients. RWD complements traditional clinical trials and research by informing the generalizability of results, generating new hypotheses, and creating a large data network for scientific discovery. Effective clinician engagement in the development of RWD applications is necessary for continued progress in the field.

Keywords: Big data; Bioinformatics real-world data; CDE; Common data elements; Electronic health record; Learning health systems; Neuro-oncology; Neuroinformatics; Point-of-care; Pragmatic clinical trials; Precision medicine; RWD; RWE; Real-world evidence.

Fig. 1

Real-world evidence (RWE) is derived from real-world data (RWD) systematically obtained from a multitude of sources loosely defined as patient information obtained outside of traditional research settings. RWE can expand the impact of prospective interventional clinical trials through evidence generation of the patient experience in conditions most similar to everyday healthcare situations (i.e., patients with additional comorbidities treated in routine clinical environments). Pragmatic clinical trials are a study type aimed at showing impact of an intervention in broad patient populations with minimal deviation in standard practice and are thus well-suited to RWE-based analysis. Classical clinical trials, RWE, and pragmatic trials inform learning healthcare systems