Optimal designs for phase II/III drug development programs including methods for discounting of phase II results

• Verfasst von: Erdmann, Stella [VerfasserIn]
• Verfasst von: Kirchner, Marietta [VerfasserIn]
• Verfasst von: Götte, Heiko [VerfasserIn]
• Verfasst von: Kieser, Meinhard [VerfasserIn]
• Titel: Optimal designs for phase II/III drug development programs including methods for discounting of phase II results
• Verf.angabe: Stella Erdmann, Marietta Kirchner, Heiko Götte, Meinhard Kieser
• E-Jahr: 2020
• Jahr: 09 October 2020
• Fussnoten: Gesehen am 04.11.2020
• Titel Quelle: Enthalten in: BMC medical research methodology
• Ort Quelle: London : BioMed Central, 2001
• Jahr Quelle: 2020
• Band/Heft Quelle: 20(2020) Artikel-Nummer 253, 14 Seiten
• ISSN Quelle: 1471-2288
• DOI: doi:10.1186/s12874-020-01093-w
• Datenträger: Online-Ressource
• Sprache: eng
• K10plus-PPN: 1737749599
• K10plus-PPN:
  Universitätsbibliothek
• Lokale URL UB: Zum Volltext

Abstract

Go/no-go decisions after phase II and sample size chosen for phase III are usually based on phase II results (e.g., the treatment effect estimate of phase II). Due to the decision rule (only promising phase II results lead to phase III), treatment effect estimates from phase II that initiate a phase III trial commonly overestimate the true treatment effect. Underpowered phase III trials are the consequence. Optimistic findings may then not be reproduced, leading to the failure of potentially expensive drug development programs. For some disease areas these failure rates are described to be quite high: 62.5%.