Towards more informative pilot studies with new anti-rheumatic drugs

No subject should be exposed to a new drug without fully studying the experience. Methodologic standards implemented in Phase I trials can improve their usefulness. Standardization of outcome measures of impairment and function to permit pooling of data; utilization of techniques to minimise measurement error; collecting information on co-variates known to affect outcome-socioeconomic status, co-morbidity, etc; definition of a meaningful clinical change in absolute and relative forms by consensus techniques. Phase I trials should also incorporate features from single subject crossover designs and sequential trials.