A causal roadmap for generating high-quality real-world evidence.

Increasing emphasis on the use of real-world evidence (RWE) to support clinical policy and regulatory decision-making has led to a proliferation of guidance, advice, and frameworks from regulatory agencies, academia, professional societies, and industry. A broad spectrum of studies use real-world data (RWD) to produce RWE, ranging from randomized trials with outcomes assessed using RWD to fully observational studies. Yet, many proposals for generating RWE lack sufficient detail, and many analyses of RWD suffer from implausible assumptions, other methodological flaws, or inappropriate interpretations. The

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© The Author(s) 2023.

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The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS, the US Government, or the authors’ affiliations. LED reports tuition and stipend support from a philanthropic gift from the Novo Nordisk corporation to the University of California, Berkeley, to support the Joint Initiative for Causal Inference. IJD is the principal investigator of a research agreement between Harvard University and Sanofi and reports consulting fees from Moderna. EK is employed by Microsoft. DA is employed by Pfizer Inc. and holds stocks in Pfizer Inc. CYV and RAH are employed by Syneos Health. CYV reports that her husband is employed by Galapagos. HR and KK are employed by Novo Nordisk A/S and own stocks in Novo Nordisk A/S. RP has received the following (directed to his institution): speaker fees from Merck and Novo Nordisk; consulting fees from Bayer AG, Corcept Therapeutics Incorporated, Dexcom, Endogenex, Inc., Gasherbrum Bio, Inc., Hanmi Pharmaceutical Co., Hengrui (USA) Ltd., Lilly, Merck, Novo Nordisk, Pfizer, Rivus Pharmaceuticals Inc., Sanofi, Scohia Pharma Inc., and Sun Pharmaceutical Industries; and grants from Hanmi Pharmaceuticals Co., Metavention, Novo Nordisk, and Poxel SA. DM is employed by Moderna. CEB is co-founder of Adaptic Health Inc., Managing Director of Pivotal Strategic Consulting, LLC, and receives consulting fees from Graticule Inc. and Sophic Alliance Inc. MA is employed by Novartis Pharma AG. JBB reports contracted fees and travel support for contracted activities for consulting work paid to the University of North Carolina by Novo Nordisk; grant support by Dexcom, NovaTarg, Novo Nordisk, Sanofi, Tolerion and vTv Therapeutics; personal compensation for consultation from Alkahest, Altimmune, Anji, AstraZeneca, Bayer, Biomea Fusion Inc., Boehringer-Ingelheim, CeQur, Cirius Therapeutics Inc., Corcept Therapeutics, Eli Lilly, Fortress Biotech, GentiBio, Glycadia, Glyscend, Janssen, MannKind, Mellitus Health, Moderna, Pendulum Therapeutics, Praetego, Sanofi, Stability Health, Terns Inc., Valo and Zealand Pharma; stock/options in Glyscend, Mellitus Health, Pendulum Therapeutics, PhaseBio, Praetego, and Stability Health; and board membership of the Association of Clinical and Translational Science. MvdL and SG report that they are co-founders of the statistical software start-up company TLrevolution, Inc. MvdL and MP report personal compensation for consultation from Novo Nordisk.