Methodology in Anti-Emetic Trials

Abstract

The most promising anti-emetic drugs are identified during preclinical testing. Phase I and II clinical studies are used to optimize doses and schedules for the various routes of administration. Most methodological issues arise in relation to phase III studies. A prospective, randomised, double-blind, parallel-subjects design is recommended. Stratification helps balance the factors influencing emesis between the 2 arms of a randomised study. The most important of these are the strength of the emetic stimulus, prior exposure to chemotherapy, age, sex and a history of chronic alcohol consumption. The evaluation of efficacy of an anti-emetic should include its efficacy in controlling acute post-chemotherapy nausea and vomiting as well as anticipatory and delayed emesis. The side effects of an anti-emetic must also be considered since they may negate any anti-emetic advantage. Patients and observers have reported successfully evaluating these parameters using both categorical and linear-analogue scales.