Elements of Clinical Study Design, Biostatistics & Research

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Contents
Foreword
Preface
	CONSENT FOR PUBLICATION
	CONFLICT OF INTEREST
Acknowledgements
Study Designs in Clinical Research
	INTRODUCTION
	OBSERVATIONAL STUDIES
		Case Series
		Case-Control Studies
		Cross-Sectional Studies, Surveys
			Methods of Survey
			By Self-Administered Questionnaire
			Interviews
			Reliability
			Validity
			Pilot Testing
		Cohort Studies
			Prospective Cohort
			Historical Cohort or Retrospective Cohort Study
			Amphispective Cohort Studies
	INTERVENTIONAL STUDIES (EXPERIMENTAL STUDIES)
		Animal Studies
		Human Studies/Clinical Trials
			Controlled Trials
			Uncontrolled Studies
	META-ANALYSIS
	CONCLUSION
		Study Designs in medical research fall into three categories:
Scales of Measurement, Descriptive Statistics & Data Presentation
	INTRODUCTION
	SCALES OF MEASUREMENT
		Nominal (Categorical Scale)
		Ordinal Scale
		Interval Scale
		Ratio Scale
	MEASUREMENT OF CENTRAL TENDENCY
		Calculation of Measures of Central Tendency
			Mean
			Median
			Mode
		Uses of Central Tendency
	GUIDELINES TO DECIDE WHICH MEASURE OF CENTRAL TENDENCY IS BEST TO BE USED
		Mean
		Median
		Mode
		Geometrical Mean
	MEASURES OF SPREAD
		The Range
		Standard Deviation
		Co-efficient of Variation (CV)
			Percentile
	GUIDELINES FOR USING DIFFERENT MEASURES OF DISPERSION
	PRESENTATION OF DATA IN TABLES AND GRAPHS
		Tables
			Simple Table
			Frequency Distribution Table
		Charts and Diagrams
			Simple Bar Chart
			Component Bar Chart
		Histogram
			Frequency Polygon
		Graphs and Line Diagram
		Pie Diagram or Pie Charts
		Pictogram
	CONCLUSION
		Scales of measurement are:
		Descriptive Statistics
		Data Presentation
Inferential Statistics
	INTRODUCTION
	PROBABILITY
		Objective Probability
		Subjective Probabilities
	EXPERIMENT AND EVENT
		Experiment
		Event
			Complementary Event
			Mutually Exclusive Event and the Rule of Addition
			Independent Events
	LIKELIHOOD
	SENSITIVITY
	SPECIFICITY
	BAYES\' THEOREM
	POPULATION AND SAMPLES
	POWER OF STUDY
	METHOD OF SAMPLE SELECTION
		Non-probabilities Sampling
		Probability Sampling
			Simple Random Sample
			Systematic Random Sample
			Stratified Sample
			Random Cluster Sample
			Properties of Good Sampling
			Random Assignment
	RANDOM VARIABLES AND PROBABILITY DISTRIBUTION
		Poisson Distribution
		Binomial Distribution
		The Normal or Gaussian Distribution
	THE CENTRAL LIMIT THEORY
	SAMPLING DISTRIBUTION
		Features of The Sampling Distribution
	SAMPLE SIZE
	APPROACHES TO STATISTICAL INFERENCES
		A confidence interval
			Estimate and Estimation:
			Point Estimate:
			Interval Estimate:
		Hypothesis Testing Steps
			Making a statement (Stating) research question in terms of statistical Hypothesis
			Making the Decision on Suitable Test Statistics
			Chi-Square Test
			Selecting the level of Significance and Determination of the Value
			Determination of the value of Significance
			Performing the Calculations
			Making Conclusions
	ERRORS IN THE HYPOTHESIS TEST
		The Type One Error
		The Type Two Error
	POWER OF STUDY
		Significance of P-Value and Alpha
		PAIRED T TEST
	INTRA-RATER RELIABILITY
	PEARSON\'S CORRELATION COEFFICIEN
	MEASUREMENT OF THE SAME VARIABLE BY TWO DIFFERENT PROCEDURES
	APPLICATION OF T-TEST
		Sample Size
	COMPARISON OF MEANS IN THREE OR MORE GROUPS
		Analysis of Variance (ANOVA)
			Example 1:
			Example 2:
			Pre-Condition (Assumptions in ANOVA)
		Multiple Comparison Procedure
			Post Hoc Comparisons
	COMPARISON OF FREQUENCIES OR PROPORTIONS IN MORE THAN TWO GROUPS
	CONCLUSION
Statistical Methods for Relationship Variables
	INTRODUCTION
	THE RELATIONSHIP BETWEEN TWO NUMERICAL OBSERVATIONS (CHARACTERISTICS)
		Correlation
			Assumptions in Correlation
			Regression
	THE RELATIONSHIP BETWEEN TWO ORDINAL CHARACTERISTICS
	THE RELATIONSHIP BETWEEN TWO NOMINAL CHARACTERISTICS
		Experimental Event Rate (EER)
		The Control Event Rate (CER)
		Relative Risk (RR)
		Relative Risk Reduction (RRR)
		Absolute Risk Reduction (ARR)
		Number Needed To Treat (NNT)
		Absolute Risk Increase (ARI)
		Number Needed to Harm (NNH)
		Odds Ratios
		Multiple Regression
	ANALYSIS OF COVARIANCE(ANCOVA)
	LOGISTIC REGRESSION
	MULTIVARIATE ANALYSIS OF VARIANCE (MANOVA)
	DIAGNOSTIC PROCEDURES
		Sensitivity
		Specificity
		Use of Sensitivity and Specificity for Revision of Probabilities:
	LIKELIHOOD RATIO
		Calculation of likelihood ratio
	BAYES\' THEOREM
		Use of Sensitivity and Specificity in Making Clinical Diagnosis (Figs. 4.2 and 4.3)
	RECEIVER OPERATING CHARACTERISTIC (ROC) CURVE
	CONCLUSION
Clinical Research
	INTRODUCTION
	CLINICAL RESEARCH
	TYPES OF CLINICAL TRIALS
	PHASES OF CLINICAL TRIALS
		Phase I
		Phase II
			Phase II(a)
			Phase II(b) (Pivotal Trial)
		Phase III(a)
			Phase-III (a) i) Superiority Clinical Trials
			Phase-III (a) ii)Equivalence Clinical Trial
			Phase-III (a) iii)Non-inferiority Clinical Trial
		Phase-III (b)
		Phase IV Clinical Trials
	RISK OF BIASES IN RANDOMIZED CONTROLLED TRIALS (RCTS)
		Cochrane Tool for Bias Risk Assessment
		CONSORT GUIDELINES
		CONSORT Checklist
	PRAGMATIC TRIALS (PRCT) (THERAPY EVALUATION)
		Main differences of (i) RCT and (ii) pRCT
	ELEMENTS OF PRAGMATIC TRIALS
		Formulation of Research Question
		Defining the patient group
		Identifying comparison group
		Defining the Treatment Protocol
		Sample Size
		Enrolment, Randomization, and Referral
		Outcome
		Analysis
		Reporting and Publication
	REVERSE PHARMACOLOGY IN DRUG DEVELOPMENT
		Reverse Pharmacology Concept
	THERE ARE FOUR STAGES OF REVERSE PHARMACOLOGY
		Stage-1:Selection of Herbal Remedy
			Literature Research
			Retrospective Treatment Outcome Study (RTO)
			Interview With Traditional Healer
		Stage-2: Dose Escalating Studies
			Dose Optimization
			Preservation of the Study Plant (Herb)
		Stage-3 Randomized Controlled Trial (RCT)
		Stage-4 Isolation And Testing Of Active Compounds
	CONCLUSION
Survey Research
	INTRODUCTION
	DESIGNING SURVEY TOOL
		The Research Question (Framing)
	ADMINISTERING THE QUESTION
		Survey Methods
		Developing Survey Questionnaire
			Scales of Measurements
			Positive and negative categories
			Balancing the responses
			Balancing the Probes
			Revised balanced probe (questionnaire)
			Using the Likert Scale
			Suggestions for Writing Probes
			Layout Of Questionnaire
	RELIABILITY AND VALIDITY OF SURVEY INSTRUMENT
		Reliability
			Test Re-Test
			Internal Consistency:
			Alternative form:
			Intra-Observer Consistency:
			Inter Observer Consistency:
		Validity
			Face Validity:
			Content Validity:
			Criterion Validity:
			Construct Validity:
	ADMINISTRATION OF THE SURVEY INSTRUMENT
		Pilot testing
		Response Rate
			Methods to Increase Response Rates
	SELECTION OF REPRESENTATIVE SAMPLE AND DETERMINATION OF SAMPLE SIZE
		Determination of Sample Size
	ANALYSIS OF SURVEY OBSERVATIONS
	CONCLUSION
Planning and Writing Research Projects
	INTRODUCTION
	PLANNING OF A RESEARCH PROJECT
	REVIEW OF LITERATURE AND IDENTIFICATION OF THE RESEARCH PROBLEM
	RESEARCH GAP ANALYSIS & FRAMING OF RESEARCH QUESTION
	CHOOSING THE APPROPRIATE STUDY DESIGN TO ANSWER THE RESEARCH QUESTION
		Experimental/Interventional studies
		Clinical trials
			Cohort Studies
			Case-Control Study Design
			Surveys & Cross-Sectional Studies
			Case Series
			Meta-Analysis
			Fishing Expedition
	LEVEL OF SIGNIFICANCE & SELECTION OF SAMPLE SIZE
		Power of the Study
		Variation in Standard Deviation
	BIASES IN THE STUDY
		Procedure Bias
		Recall Bias
		Insensitive Measurement bias
		Compliance Bias
		Selection bias
		Admission Rate Bias
	APPLICATION OF STATISTICAL PROCEDURE AND TEST OF SIGNIFICANCE
		Multiple Tests of Significance
	REPORT WRITING &DATA PRESENTATION
	SENDING RESEARCH WORK/ARTICLE FOR PUBLICATION
		Sources of guidelines for specific study design
	GUIDELINES FOR REPORTING STATISTICS
		Presentation of Results
	DISCUSSION AND CONCLUSION
		A researcher must remember that;
	CONCLUSION
APPENDIX-AETHICAL GUIDELINES FOR CLINICAL RESEARCH
	INTRODUCTION
	PRINCIPALS OF ETHICS (CORE VALUES)
		Respecting the Autonomy Of Research Subjects
			Voluntary participation
			Autonomy and research involving minors
			Autonomy and Research in Schools
			Age
			Information to subjects
			Exceptions from informed consent
		Avoiding Harm
		Privacy and Data Protection
			Protecting research data and confidentiality
			Storing/ destroying research data
			Protecting privacy in research publications
	ETHICAL REVIEW
		Ethical Review Required
		Concerns of the Institutional Ethics Committee
		Information to the research subjects
		Privacy and data protection
GLOSSARY
REFERENCES
	REFERENCES
Subject Index
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