Reweighting estimators to extend the external validity of clinical trials: methodological considerations.
- Resource Type
- Article
- Authors
- Kaizar, Eloise; Lin, Chen-Yen; Faries, Douglas; Johnston, Joseph
- Source
- Journal of Biopharmaceutical Statistics. 2023, Vol. 33 Issue 5, p515-543. 29p. 1 Diagram, 3 Charts, 2 Graphs.
- Subject
- *VALIDITY of statistics
*CLINICAL trials
*ALZHEIMER'S disease
*RANDOMIZED controlled trials
- Language
- ISSN
- 1054-3406
Methods to extend the strong internal validity of randomized controlled trials to reliably estimate treatment effects in target populations are gaining attention. This paper enumerates steps recommended for undertaking such extended inference, discusses currently viable choices for each one, and provides recommendations. We demonstrate a complete extended inference from a clinical trial studying a pharmaceutical treatment for Alzheimer's disease (AD) to a realistic target population of European residents diagnosed with AD. This case study highlights approaches to overcoming practical difficulties and demonstrates limitations of reliably extending inference from a trial to a real-world population. [ABSTRACT FROM AUTHOR]