Dose Finding in Drug Development

Part of the series Statistics for Biology and Health pp 146-171

Analysis of Dose–Response Studies—Modeling Approaches

  • José C. Pinheiro
  • , Frank Bretz
  • , Michael Branson

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Identifying the correct dose is one of the most important and difficult goals in drug development. Selecting too high a dose can result in an unacceptable toxicity pro- file, while selecting too lowa dose decreases the chance of it showing effectiveness in Phase III trials and thus getting regulatory approval. Typically decisions derived from dose–response studies can be divided into two main components: establishing that the treatment has some effect on the outcome under consideration, the so called proof-of-activity (PoA), sometimes also referred to as a proof-of-concept (PoC), and selecting a dose (or doses) that appears to be efficacious and safe, for further development in Phase III, the so-called dose-finding step.