When is an adaptive design useful in clinical dose-finding trials?
2015 (English)In: Festschrift in Honor of Hans Nyquist on the occasion of his 65th birthday / [ed] Ellinor Fackle-Fornius, Stockholm: Stockholms universitets förlag, 2015, 28-43 p.Chapter in book (Other academic)
During the development process for new drugs, dose-finding trials have to be conducted and the choice of their design is an important issue. Traditionally, the standard design is a balanced design where equally large groups of patients are treated with different doses of the new drug or with a control. However, it has been identified that other innovative designs might be more efficient: Optimal designs which use non-balanced allocation to dose, and adaptive designs where the allocation to the doses can be changed during the study based on results collected earlier in the study. In a simulation study we will compare efficiencies of balanced non-adaptive, optimal non-adaptive, adaptive two-stage and fully sequential adaptive designs. In all situations considered one can gain from applying optimal design theory. However, when moving from the optimal non-adaptive design to an adaptive design, there are situations where the design is improved and other situations where there is only a minor or no gain. Based on our considered situations, we generalize our observations to answer when an adaptive design is useful.
Place, publisher, year, edition, pages
Stockholm: Stockholms universitets förlag, 2015. 28-43 p.
Adaptive design; Clinical trial; Dose-finding; Efficiency; Fully sequential design; Interim analysis; Optimal design; Two-stage design
Probability Theory and Statistics
Research subject Medicine; Statistics
IdentifiersURN: urn:nbn:se:su:diva-124588OAI: oai:DiVA.org:su-124588DiVA: diva2:890162