Non-inferiority (NI) trials have gained recognition as an effective tool with which to search for substitutes for a standard treatment that is associated with certain undesirable features, such as adverse side-effects, an exorbitant cost, or an extremely complicated therapeutic regimen. Statistical methods have been developed for NI studies with multiple experimental treatments. However, experimental treatments sometimes represent a new treatment with various dose levels, and in such circumstances, identification of the minimum non-inferior dose is a reasonable objective of the trial. Thus, we examine several potential testing procedures with respect to this testing objective. A simulation study is conducted to evaluate their performance, and determination of the sample size is discussed. Two clinical examples are provided to illustrate the proposed method.
All Science Journal Classification (ASJC) codes
- Statistics and Probability