Abstract
According to ICH-E4, “Elucidation of the dose–response function” is a key stage in drug development. Consequently, designing a dose–response study with the desired characteristics is an important activity in drug development. Inadequate dose–response knowledge has been known to lead to a delay or denial in regulatory approvals of initial drug applications. There have also been cases when the dose initially approved for a marketed product had to be reduced subsequently. In this chapter, we focus on using the Emax model to describe a dose–response relationship, but the discussion applies equally to other dose–response models or to a collection of models. We examine in detail the three metrics introduced in Chapter 6 for assessing a dose–response study design, for both continuous and binary endpoints. We include an example of dose–response studies for an investigational medicinal product to illustrate the importance of covering an adequate dose range in dose-ranging studies.
The dose makes the poison.
—Paracelsus
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Chuang-Stein, C., Kirby, S. (2021). Designing Dose–Response Studies with Desired Characteristics. In: Quantitative Decisions in Drug Development. Springer Series in Pharmaceutical Statistics. Springer, Cham. https://doi.org/10.1007/978-3-030-79731-7_8
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DOI: https://doi.org/10.1007/978-3-030-79731-7_8
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