A discussion of new drug development using the example of acute myeloid leukemia (AML)—but that is applicable to a broad range of diseases— outlines the problems inherent in phase 2 clinical trials that may lead to falsepositive results, and how trial design can be improved and drug development be made more efficient and less costly (Blood. 2010;116:2420-2428).
The authors provide a number of recommendations on how AML trials might be improved, among them, increasing study sizes; using an explicitly described control group; conducting multivariate analysis to adjust for patient heterogeneity; including explicit descriptions of inclusion/exclusion criteria; using validated surrogate end points; incorporating an integrated phase 2/3 trial design so there are no delays in conducting the phase 3 trial; and discouraging early publication so that mature data are published.
