Clinical Studies Leveraging Real-World Data Using Propensity Score-based Methods

Heng Li () and Lilly Q. Yue
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Heng Li: Center for Devices and Radiological Health, US Food and Drug Administration, Division of Biostatistics
Lilly Q. Yue: Center for Devices and Radiological Health, US Food and Drug Administration, Division of Biostatistics

A chapter in Real-World Evidence in Medical Product Development, 2023, pp 167-192 from Springer

Abstract: Abstract This chapter discusses the design and analysis of three types of hybrid studies. A hybrid study is defined as a study whose data come from two sources: (1) a traditional clinical study to be designed and conducted and (2) RWD, which may or may not be in existence at the planning stage. What motivates a hybrid study are often ethical or practical considerations. The incorporation of RWD may save time and reduce cost. A fundamental issue in designing a hybrid study is the potential systematic difference between the patients in the traditional clinical study and in RWD. This issue is addressed by using the propensity score methodology. In implementing the propensity score methodology, it is critical to safeguard the integrity and the objectivity of study design and interpretability of study results. To that end, a two-stage design framework is proposed. To ensure that the information contributed by the RWD does not overwhelm the information contributed by the traditional clinical study, a method of discounting or down-weighting may be used, such as power prior for Bayesian inference and composite likelihood for frequentist inference.

Keywords: Propensity score; Power prior; Composite likelihood (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_10

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DOI: 10.1007/978-3-031-26328-6_10

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