Abstract
In this chapter, we compare successive trials designed and conducted to assess the efficacy of a new drug to a series of diagnostic tests. The condition to diagnose is whether the new drug has a clinically meaningful efficacious effect. This comparison offers us the opportunity to apply properties pertaining to diagnostic tests discussed in Chap. 3 to clinical trials. Building on the results in Chap. 3, we discuss why replication is such a critically important concept in drug development and show why replication is not as easy as some might have hoped. The difference between replicability and reproducibility is briefly discussed. We end the chapter by highlighting the difference between statistical power and the probability of a positive trial. This last point becomes more important as a new drug moves through the various development stages as will be illustrated in Chap. 9.
Using preliminary research to approve new treatments has high costs in morbidity and healthcare dollars.
—British Medical Journal, Nov 23 2015
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Chuang-Stein, C., Kirby, S. (2021). The Parallel Between Clinical Trials and Diagnostic Tests. In: Quantitative Decisions in Drug Development. Springer Series in Pharmaceutical Statistics. Springer, Cham. https://doi.org/10.1007/978-3-030-79731-7_4
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DOI: https://doi.org/10.1007/978-3-030-79731-7_4
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