When is an adaptive design useful in clinical dose-finding trials?

• 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.

Subject headings

NATURVETENSKAP  -- Matematik -- Sannolikhetsteori och statistik (hsv//swe)

NATURAL SCIENCES  -- Mathematics -- Probability Theory and Statistics (hsv//eng)

Keyword

Adaptive design

Clinical trial

Dose-finding

Efficiency

Fully sequential design

Interim analysis

Optimal design

Two-stage design

Medicine

medicin

Statistics

statistik

Publication and Content Type

vet (subject category)

kap (subject category)