The maturation of use of real-world evidence (RWE) in decision making requires substantiation and growth in several areas including data sources and catalogs, data quality, governance, analytic methods, reporting standards, transparency, replicability, and fit-for-purpose specifications.
1, 2, 3 During the past few years, recent guidance documents from the US Food and Drug Administration, European Medicines Agency, and many other agencies and societies have begun to address these areas.
4, 5, 6 Health technology assessment (HTA) agencies or coalitions such as the National Institute for Health and Care Excellence in the United Kingdom, European Network for Health Technology Assessment, Real-World Data in Asia for Health Technology Assessment in Reimbursement, and Canadian Agency for Drugs and Technologies in Health have also issued guidance.
7, 8, 9, 10 Although the promulgation of guidance is a critical step toward framing the use of RWE in health-related innovation, regulation, and HTA, the actual use of RWE for these purposes is still very much in its nascency.