Innovative Hybrid Designs and Analytical Approaches Leveraging Real-World Data and Clinical Trial Data

Lisa V. Hampson () and Rima Izem
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Lisa V. Hampson: Statistical Methodology, Novartis Pharma AG
Rima Izem: Statistical Methodology, Novartis Pharma AG

A chapter in Real-World Evidence in Medical Product Development, 2023, pp 211-232 from Springer

Abstract: Abstract This chapter will review prospectively planned hybrid approaches that incorporate real-world data (RWD) and clinical trial data in the evaluation of treatment effectiveness and safety. RWD can serve to augment the trial data in a variety of ways including the analysis population, the treatments considered, the variables on each subject, or the follow-up time. These approaches include using RWD as external controls to complement single arms trials, approaches using RWD to augment the control arm of a randomized controlled trial (RCT), and approaches using RWD in lieu of clinical measurements in pragmatic randomized trials and decentralized clinical trials. These designs can also complement variables collected concurrently, preceding or following the clinical trials with those collected in other linked data sources. Finally, the approaches include analytical methods to help extrapolate clinical trial results to a population captured in RWD. We present considerations for implementation and use of these approaches, advantages and potential limitations and illustrate them with examples.

Keywords: Pragmatic; Point of care; Decentralized; Linkage; Tokenization; External controls; Dynamic borrowing; Generalization (search for similar items in EconPapers)
Date: 2023
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Persistent link: https://EconPapers.repec.org/RePEc:spr:sprchp:978-3-031-26328-6_12

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DOI: 10.1007/978-3-031-26328-6_12

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