Propensity score-integrated power prior approach for incorporating real-world evidence in single-arm clinical studies

Chenguang Wang, Heng Li, Wei Chen Chen, Nelson Lu, Ram Tiwari, Yunling Xu, Lilly Q. Yue

Research output: Contribution to journalArticle

Abstract

We are now at an amazing time for medical product development in drugs, biological products and medical devices. As a result of dramatic recent advances in biomedical science, information technology and engineering, ``big data'' from health care in the real-world have become available. Although big data may not necessarily be attuned to provide the preponderance of evidence to a clinical study, high-quality real-world data can be transformed into scientific evidence for regulatory and healthcare decision-making using proven analytical methods and techniques, such as propensity score methodology and Bayesian inference. In this paper, we extend the Bayesian power prior approach for a single-arm study (the current study) to leverage external real-world data. We use propensity score methodology to pre-select a subset of real-world data containing patients that are similar to those in the current study in terms of covariates, and to stratify the selected patients together with those in the current study into more homogeneous strata. The power prior approach is then applied in each stratum to obtain stratum-specific posterior distributions, which are combined to complete the Bayesian inference for the parameters of interest. We evaluate the performance of the proposed method as compared to that of the ordinary power prior approach by simulation and illustrate its implementation using a hypothetical example, based on our regulatory review experience.

Original languageEnglish (US)
Pages (from-to)731-748
Number of pages18
JournalJournal of biopharmaceutical statistics
Volume29
Issue number5
DOIs
StatePublished - Jan 1 2019

Fingerprint

Propensity Score
Information Science
Delivery of Health Care
Bayesian inference
Healthcare
Biological Products
Decision Making
Methodology
Technology
Equipment and Supplies
Product Development
Posterior distribution
Information Technology
Analytical Methods
Leverage
Covariates
Drugs
Pharmaceutical Preparations
Evidence
Power (Psychology)

Keywords

  • Covariate balance
  • overlapping coefficient
  • power prior
  • propensity score
  • real-world data
  • real-world evidence

ASJC Scopus subject areas

  • Statistics and Probability
  • Pharmacology
  • Pharmacology (medical)

Cite this

Propensity score-integrated power prior approach for incorporating real-world evidence in single-arm clinical studies. / Wang, Chenguang; Li, Heng; Chen, Wei Chen; Lu, Nelson; Tiwari, Ram; Xu, Yunling; Yue, Lilly Q.

In: Journal of biopharmaceutical statistics, Vol. 29, No. 5, 01.01.2019, p. 731-748.

Research output: Contribution to journalArticle

Wang, Chenguang ; Li, Heng ; Chen, Wei Chen ; Lu, Nelson ; Tiwari, Ram ; Xu, Yunling ; Yue, Lilly Q. / Propensity score-integrated power prior approach for incorporating real-world evidence in single-arm clinical studies. In: Journal of biopharmaceutical statistics. 2019 ; Vol. 29, No. 5. pp. 731-748.
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