Reweighting estimators to extend the external validity of clinical trials : methodological considerations
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.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2023 |
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| Erschienen: |
2023 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:33 |
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| Enthalten in: |
Journal of biopharmaceutical statistics - 33(2023), 5 vom: 03. Sept., Seite 515-543 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Kaizar, Eloise [VerfasserIn] |
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| Links: |
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| Themen: |
Diagnostic |
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| Anmerkungen: |
Date Completed 22.08.2023 Date Revised 29.08.2023 published: Print-Electronic Citation Status MEDLINE |
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| doi: |
10.1080/10543406.2022.2162067 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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NLM351993630 |
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| 520 | |a 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 | ||
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