Chapter 11. Design and Analysis of Dose-Ranging Clinical Studies

Alex Dmitrienko^[]

^[] Alex Dmitrienko is Principal Research Scientist, Global Statistical Sciences, Eli Lilly and Company, USA.

Kathleen Fritsch^[]

^[] Kathleen Fritsch is Mathematical Statistician, Division of Biometrics III, Food and Drug Administration, USA (The views presented in this chapter are those of the author and do not necessarily represent the views of the FDA. Kathleen Fritsch's contribution to this chapter was completed prior to her employment with the FDA).

Jason Hsu^[]

^[] Jason Hsu is Professor, Department of Statistics, The Ohio State University, USA.

Stephen Ruberg^[]

^[] Stephen Ruberg is Director, Global Medical Information Sciences, Eli Lilly and Company, USA.

Dose-ranging clinical studies play a very important role in early evaluation of safety and efficacy profiles of experimental drugs. The chapter reviews popular statistical methods used in dose-response analysis, including trend tests, dose-response modeling and multiple tests for identifying safe and effective doses. The testing and estimation procedures discussed in this chapter are illustrated using examples from dose-ranging clinical trials.