The New England Journal of Statistics in Data Science

The biggest risk in new drug development is either unaware of, or under-estimate the potential risks in designing clinical trials. Among all challenges in drug development, the most critical one is about finding the appropriate dose(s) for the study drug in treating patients. Designing dose finding clinical trials involves in many potential risks. In practice, most of the expensive failures in drug development originated not from “We did not know”, rather, the mistake is “We thought we knew”. In other words, greatest risks came from lack of awareness of underlying assumptions. This manuscript attempts to discuss some of these risks and make recommendations to reduce risks in the design of dose finding clinical trials. This is not a complete list of risks, but it is only starting the discussion.