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Optimal planning of phase II/III programs for clinical trials with multiple endpoints

Owing to increased costs and competition pressure, drug development becomes more and more challenging. Therefore, there is a strong need for improving efficiency of clinical research by developing and applying methods for quantitative decision making. In this context, the integrated planning for pha...

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Hauptverfasser: Kieser, Meinhard (VerfasserIn) , Kirchner, Marietta (VerfasserIn) , Dölger, Eva (VerfasserIn) , Götte, Heiko (VerfasserIn)
Dokumenttyp: Article (Journal)
Sprache:Englisch
Veröffentlicht: 26 April 2018
In: Pharmaceutical statistics
Year: 2018, Jahrgang: 17, Heft: 5, Pages: 437-457
ISSN:1539-1612
DOI:10.1002/pst.1861
Online-Zugang:Verlag, Volltext: https://doi.org/10.1002/pst.1861
Verlag, Volltext: https://onlinelibrary.wiley.com/doi/abs/10.1002/pst.1861
Volltext
Verfasserangaben:Meinhard Kieser, Marietta Kirchner, Eva Dölger, Heiko Götte
Beschreibung
Zusammenfassung:Owing to increased costs and competition pressure, drug development becomes more and more challenging. Therefore, there is a strong need for improving efficiency of clinical research by developing and applying methods for quantitative decision making. In this context, the integrated planning for phase II/III programs plays an important role as numerous quantities can be varied that are crucial for cost, benefit, and program success. Recently, a utility-based framework has been proposed for an optimal planning of phase II/III programs that puts the choice of decision boundaries and phase II sample sizes on a quantitative basis. However, this method is restricted to studies with a single time-to-event endpoint. We generalize this procedure to the setting of clinical trials with multiple endpoints and (asymptotically) normally distributed test statistics. Optimal phase II sample sizes and go/no-go decision rules are provided for both the “all-or-none” and “at-least-one” win criteria. Application of the proposed method is illustrated by drug development programs in the fields of Alzheimer disease and oncology.
Beschreibung:Gesehen am 21.10.2019
Beschreibung:Online Resource
ISSN:1539-1612
DOI:10.1002/pst.1861

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