Key Statistical Concepts in Clinical Trials for Pharma [electronic resource] /
Turner, J. Rick.author.
Pharma Corporation.
This Brief discusses key statistical concepts that facilitate the inferential analysis of data collected from a group of individuals participating in a pharmaceutical clinical trial, the estimation of their clinical significance in the general population of individuals likely to be prescribed the drug if approved, and the related decision-making that occurs at both the public health level (by regulatory agencies when deciding whether or not to approve a new drug for marketing) and the individual patient level (by physicians and their patients when deciding whether or not the patient should be prescribed a drug that is on the market). These concepts include drug safety and efficacy, statistical significance, clinical significance, and benefit-risk balance.
Description based upon print version of record.
Key Statistical Concepts in Clinical Trials for Pharma; Preface; Contents; 1 Setting the Scene; Abstract; 1.1...Introduction; 1.2...The Discipline of Statistics; 1.3...Generalizing Information Gained from a Clinical Trial; 1.4...Blood Pressure; 1.4.1 The Physiology and Measurement of Blood Pressure; 1.4.2 A Cautionary Tale About Blood Pressure Measurement; 1.5...Statistical Concepts and Nomenclature; 1.5.1 Drug Safety; 1.5.2 Drug Efficacy; 1.5.3 Statistical Significance and Clinical Significance; References; 2 Analyzing Safety Data; Abstract; 2.1...Introduction
2.2...Providing Safety Data to Prescribing Physicians and Patients2.3...The Clinical Study Report; 2.4...Subject Demographics and Accountability; 2.5...Safety Parameters Measured; 2.6...Adverse Events; 2.7...From Descriptive Statistics to Inferential Statistics; 2.8...Prospective Exclusion of Unacceptable Risk; 2.8.1 Assessment of Unacceptable Cardiac Risk; 2.8.2 Assessment of Unacceptable Cardiovascular Risk; 2.8.3 A More Realistic Scenario and its Unintended Consequences; References; 3 Assessing Efficacy Data; Abstract; 3.1...Introduction; 3.2...Probability
3.3...Statistically Significant Efficacy and Hypothesis Testing3.3.1 A Straightforward Example; 3.3.2 A Quick Detour: Degrees of Freedom; 3.3.3 Returning to Our Example; 3.3.4 A Second Method of Analysis: ANOVA; 3.4...Clinically Significant Efficacy and Confidence Intervals; 3.5...Emphasizing an Earlier Point; References; 4 Confidence Intervals: Additional Commentary; Abstract; 4.1...Introduction; 4.2...The Logic of Confidence Intervals; 4.3...Differing Confidence Intervals Differ in Width; 4.4...Employment of Confidence Intervals in Both Safety and Efficacy Analyses
4.4.1 Equidistant and Non-Equidistant Confidence Intervals4.5...The Limit of Primary Interest; 4.6...Relationship Between Confidence Intervals and Probability Levels; 4.6.1 Cases in Which the Null Value is Zero; 4.6.2 Cases in Which the Null Value is Unity; 5 Meta-Methodology; Abstract; 5.1...Introduction; 5.2...Unbridled Bravado is Inappropriate When Presenting Results; 5.3...Fundamentals of Meta-Methodology; 5.4...Data Analysis: Fixed-Effect and Random-Effect Models; 5.5...Evaluating Heterogeneity; 5.6...Evaluating Robustness; 5.7...Hypothesis Generation and Hypothesis Testing; 5.8...Results and Conclusions
References6 Benefit--Risk Estimation; Abstract; 6.1...Introduction; 6.2...Drug Safety; 6.3...Decision Making; 6.4...The Subjective Nature of Many Decisions; 6.5...Determining and Enforcing Thresholds of Regulatory Concern; 6.6...Decision Analysis and Decision Making; 6.7...Qualitative and Quantitative Considerations; References; About the Author; Index
Includes bibliographical references and index.
This Brief discusses key statistical concepts that facilitate the inferential analysis of data collected from a group of individuals participating in a pharmaceutical clinical trial, the estimation of their clinical significance in the general population of individuals likely to be prescribed the drug if approved, and the related decision-making that occurs at both the public health level (by regulatory agencies when deciding whether or not to approve a new drug for marketing) and the individual patient level (by physicians and their patients when deciding whether or not the patient should be prescribed a drug that is on the market). These concepts include drug safety and efficacy, statistical significance, clinical significance, and benefit-risk balance.
Medicine.
Pharmaceutical technology.
Biomedicine, general.
Pharmaceutical Sciences/Technology.
SpringerBriefs in Pharmaceutical Science & Drug Development