Abstract

Background. A phase I drug trial is the first evaluation of a drug in humans. There are three goals in phase I drug trials: to select the dose to be used in a phase II drug trial, to explore drug toxicity in humans, and to examine the pharmacology of the drug. Phase I trials are small, usually with no more than 30 people enrolled. For this and other reasons, typically several phase I trials are conducted with a given drug before phase II trials are initiated. Phase I, then, can be thought of as a process with a single phase I trial being one of many trials in a set. ;Objectives. The objectives of this work were to examine the phase I process through a literature review investigating the adequacy of the reporting of phase I trials, and simulation studies to explore potential approaches to conducting a series of phase I trials. ;Results. The literature review indicates that generally phase I trials are inadequately reported. Aspects related to the goals of phase I trials were either absent or vague in the majority of reports included in this study. Additionally, many trials were conducted without apparent reference to use of information discovered in earlier trials were conducted without apparent reference to use of information discovered in earlier trials. Results from the simulation study show that treatment of patients and use of information is optimized if data from phase I trials are combined as they are conducted. ;Conclusion. Improvements are needed in both the way phase I trials are reported and the frequency with which they are reported. Enhanced reporting is important for increasing the quality of the phase I process as a whole