Fundamentals of Clinical Research (eBook)

Bridging Medicine, Statistics and Operations

Antonella Bacchieri, Giovanni Della Cioppa (Autoren)

eBook Download: PDF

2007 | 2007
XXV, 343 Seiten
Springer Italia (Verlag)
978-88-470-0492-4 (ISBN)

Infos

Here is a new book on methods and issues in clinical research. Its objectives can be summarized in three points. 1. Integrate medical and statistical components of clinical research. 2. Do justice to the operational and practical requirements of clinical research. 3. Give space to the ethical implications of methodological issues in clinical research. The book ends with a brief description of the drug development process and the phases of clinical development.

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Statistical methodology is an essential component of clinical (and biological) - search. Therefore it is not surprising that many textbooks aiming at "e;explaining"e; statistical methods to researchers have been published and continue to appear in print. The complexity of the basic issue, that of communication between st- isticians and researchers, is illustrated and discussed very well by the authors of this book in their Introduction. Thus it would be of no benefit to dwell further on this theme and on the difficulty of producing truly effective material. I prefer to start from a personal episode. Way back in 1959, a freshman of the faculty of Statistical Sciences, I found by chance on a stand, the book "e;Metodi statistici ad uso dei ricercatori"e;, which was the Italian translation of the famous book by RA Fisher. The textbook for the first course of statistics used that year at of our University had left me perplexed. A student like myself, with a good high school scientific education, was fascinated by the wealth of real life ex- ples, but was unable to reconstruct the thread of logical-mathematical reas- ing, especially the inductive one. The unexpected access to Fisher's legendary book raised my hopes that I could finally get to the heart of the matter.

Inhalt

1. Variability of Biological Phenomena and Measurement Errors
1.1. Phenotypic Variability
1.2. Temporal Variability.
1.3. Measurement - Related Variability
1.3.1. The Measurement
1.3.2. Measurement Errors
1.4. Variability of Diagnostic Tests
Summary
2. Distinctive Aspects of a Biomedical Study. Observational and Experimental Studies
2.1. Distinctive Features of Biomedical Studies
2.2. The Study Protocol
2.3. Observational Studies
2.4. Experimental Studies
Summary
3. Observational Studies
3.1. Basic Designs of Observational Studies
3.1.1. Prospective or Cohort Studies
3.1.2. Retrospective Studies or Control Cases
3.1.3. Sample Size
3.2. Bias and Confounding
3.2.1. Control of Bias in Epidemiology
3.2.2. Control of the Phenomenon of Confounding |
3.3. Advantages and Disadvantages of the Different Types of Observational Studies
Summary
4 Defining the Treatment Effect
4.1. From the Single Measurement to the Signal
4.2. Identification and Quantification of the End-Points (Individual Subject Level)
4.2.1. Methodological Characteristics of the End-Point
4.2.2 Discriminating Between Primary and Secondary End-Points and Between Efficacy and Safety/Tolerability End-Points 4.3. Identification and Quantification of the Signals (Group Level)
4.4. Statistical Considerations
4.5. Practical, Regulatory, Marketing and Pharmaco-Economic Considerations
4.6. Selection and Characterization of the Primary End-Point and Signal: an Example
4.6.1. Stage One: Define the Main Therapeutic Level
4.6.2. Stage Two: Define the Primary End-Point (Individual Patient Level)
4.6.3. Stages Three and Four: Define the Group Indicator, the Signal, and the Threshold of Clinical Relevance (Treatment Group and Study Levels)
4.7. More Than One Question in the Same Study: the Problem of Multiple Statistical Tests
4.8. Validation of Measurement Scales
4.9. Special Types of End-Points
Summary
5. Probability, Inference and Decision Making
5.1. Probability
5.1.1 Definitions
5.1.2. Probability Distribution and Probability Density Function
5.1.3. Normal or Gaussian Distribution
5.2. Basic Concepts of Inference
5.2.1. Hypothesis Testing and Statistical Formulation of the Medical Question
5.2.2. Statistical Estimation as the Tool for Evaluation of Clinical Relevance
5.3. Statistical Inference in the Frequentist and the Bayesian Approaches
5.4. Two Digressions: Measures of Variability and Likelihood Function
5.4.1. Measures of Variability
5.4.2. Likelihood Function
5.5. Frequentist (Classical) Analysis of a Clinical Trial
5.5.1. Hypothesis Testing: the Frequentist Solution
5.5.2. Estimation of the Effect: the Frequentist Solution
5.6. Bayesian Analysis of a Clinical Trial
5.6.1. Hypothesis Testing: the Bayesian Solution
5.6.2. Estimation of the Effect: the Bayesian Solution
5.7. Some Additional Considerations on the Frequentist and Bayesian Approaches
5.8. Parametric and Non-Parametric Inference
5.9. Statistical Decision Making in the Medical Field
5.10. Evidence-Based Medicine
Summary
6. The Choice of the Sample
6.1. Which Subjects Should Form the Sample?
6.1.1. Characteristics of the Patients to be Enrolled in the Study
6.1.2. Mechanism of Subject Selection
6.2. How Many Subjects Should Form the Sample?
6.2.1. Statistical Considerations
6.2.2. Medical and Practical Aspects
Summary
7. The Choice of Treatments
7.1. Study Treatments
7.1.1. How Many Treatments
7.1.2. What Treatments
7.1.3. Blinding of the Study Treatments
7.1.4. Packaging and Logistics
7.2. Concomitant Treatments
Summary
8. Experimental Design: Fallacy of 'Before-After' Compa

Produkt-Details

• Erscheint lt. Verlag: 29.4.2007
• Reihe/Serie: Statistics for Biology and Health
• Reihe/Serie: Statistics for Biology and Health
• Zusatzinfo: XXV, 343 p.
• Verlagsort: Milano
• Sprache: englisch
• Themenwelt: Medizin / Pharmazie ► Allgemeines / Lexika
• Themenwelt: Medizin / Pharmazie ► Medizinische Fachgebiete ► Arbeits- / Sozial- / Umweltmedizin
• Themenwelt: Medizin / Pharmazie ► Pflege
• Themenwelt: Medizin / Pharmazie ► Pharmazie ► PTA / PKA
• Themenwelt: Studium ► Querschnittsbereiche ► Epidemiologie / Med. Biometrie
• Themenwelt: Technik ► Medizintechnik
• Schlagworte: clinical medicine • Excel • likelihood • Radiologieinformationssystem • Statistics
• ISBN-10: 88-470-0492-6 / 8847004926
• ISBN-13: 978-88-470-0492-4 / 9788847004924