Selecting the right dose is a critical step in pharmaceutical drug development. The basic difficulty is the trade-off between wanted and un-wanted effects: Selecting too high a dose may result in unacceptable safety problems, while selecting too low a dose may make the drug ineffective. In the past, dose finding studies were often designed using a small number of doses and a narrow dose-range. This often led to the selection and use of an overly high dose with dose-related adverse drug reactions emerging only after the new medicine had been introduced and used in large numbers of patients. Only in more recent years has there been a significant shift towards investigating the full dose-response relationship with larger interest in the minimum effective dose.

Traditional dose ranging designs do not provide explicit dose response characterization, and often lack precision to differentiate between active doses. In contrast, the MCP-Mod methodology provides a structured approach to model-based design and analysis of Phase II dose finding studies under model uncertainty while adjusting for multiplicity and is designed to increase the likelihood that a new medicine will progress through the final stages of development and onto the market. In this presentation we share our experiences of developing, implementing and fostering the use of MCP-Mod as an efficient statistical methodology for dose finding studies which has recently received the CHMP Qualification Opinion.