Dose response studies. I. Some design considerations

A critical aspect of biomedical research is the characterization of the dose response relationship of a compound. This is true in laboratory experiments and clinical trials and pertains to efficacy, safety, and the resulting benefit/risk ratio. Presented here is Part I of this article, which deals with some clinical trial design issues surrounding dose response studies. Some additional comments are made about trials for identifying the minimum effective dose, randomized concentration controlled trials, and the use of one-sided hypotheses in designing such trials. Part II is a separate paper reviewing some analysis strategies for dose response studies.