Analyzing and Reporting Efficacy Data
When faced with the myriad challenges in drug development, the discipline of Statistics (recognized by the use of an upper case “S”) is “the knight in shining armor” that rides to our assistance and facilitates the collection, analysis, and interpretation
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Analyzing and Reporting Efficacy Data Our interest in the discipline of Statistics is a pragmatic one since it provides the best way currently available to conduct clinical development programs (Turner 2010).
4.1
Introduction
When faced with the myriad challenges in drug development, the discipline of Statistics (recognized by the use of an upper case “S”) is “the knight in shining armor” that rides to our assistance and facilitates the collection, analysis, and interpretation of optimal-quality data as the basis for rational decision-making at all stages of the process (Durham and Turner 2008). In this and the following two chapters, we have resisted the temptation to provide exhaustive discussion of subtle nuances of statistical analysis: our interest in the discipline of Statistics is a pragmatic one since it provides the best way currently available to conduct clinical development programs. We noted in Chap. 1 that, since no biologically active drug is free from the possibility of causing adverse reactions in certain individuals who are genetically and/ or environmentally susceptible, regulators have to balance a drug’s therapeutic benefit with its toxicity to assess the drug’s benefit–risk balance. Therefore, it is important to have a fundamental understanding of assessments of benefit, which is called efficacy in clinical trials. Quantitative information concerning both efficacy and safety provides the rational basis for evidence-based decision-making, both by sponsors throughout their drug development programs and by regulators when sponsors apply for marketing approval. When planning clinical trials, two considerations are of critical importance. First, the statistical analyses that will eventually be conducted must be planned at the design stage of the study. Second, the desired goal, i.e., approval of a new drug by regulatory agencies, is known from the outset. Regulatory agencies provide enormous amounts of detailed guidance for the conduct and reporting of drug development research. Guidance documents should therefore be studied before starting the drug development program.
© Springer International Publishing Switzerland 2017 J.R. Turner et al., Cardiovascular Safety in Drug Development and Therapeutic Use, DOI 10.1007/978-3-319-40347-2_4
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4.2
4 Analyzing and Reporting Efficacy Data
Categorization of Clinical Trials
Clinical trials are often categorized into four phases, with a given trial being identified as belonging to one of them. Traditional descriptions are as follows: • Phase I: These are pharmacologically oriented studies that typically look for the best dose(s) to employ in subsequent trials. Comparison with other treatments is not the first priority, although a small control group is often included in trial designs. The focus here is on safety. • Phase II: These trials are usually performed in individuals with the clinical condition of interest, and look for evidence of biological activity, efficacy, and safety. • Phase III: Comparison with another treatment (which can be a pla
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