STAT 509 | Design and Analysis of Clinical Trials

About this course

Welcome to the course notes for STAT 509: Design and Analysis of Clinical Trials. These notes are designed and developed by Penn State’s Department of Statistics and offered as open educational resources. These notes are free to use under Creative Commons license CC BY-NC 4.0.

This course is part of the Online Master of Applied Statistics program offered by Penn State’s World Campus.

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If you are a current student in this course, please see Canvas for your syllabus, assignments, lesson videos, and communication from your instructor.

Lessons

Introduction and Review
Statistical Foundations
Clinical Trial Design
Regression Models
8/19/26

Overview of Clinical Trials
Clinical Trials
Research Ethics
Clinical Trial Design
Regulatory Affairs
Biostatistics
8/19/26

Overview of Clinical Trial Designs
Clinical Trial Design
Experimental Design
Study Designs
Bias and Randomization
Clinical Research Methods
8/19/26

Bias, Allocation, and Randomization
Bias and Confounding
Randomization
Treatment Allocation
Clinical Trial Design
Prognostic Factors
8/19/26

Missing Data and Intent-to-Treat
Missing Data
Intent-to-Treat Analysis
Clinical Trial Design
Data Imputation
Clinical Trial Analysis
8/19/26

Objectives, Endpoints, and Estimation
Clinical Trial Endpoints
Survival Analysis
Estimation Methods
Dose-Finding Studies
Clinical Trial Analysis
8/19/26

Early Phase Studies
Early Phase Studies
Dose-Finding Studies
Bayesian Methods
Pharmacokinetics
Correlation and Agreement
8/19/26

Sample Size and Power
Sample Size and Power
Statistical Power
Confidence Intervals
Survival Analysis
Clinical Trial Design
8/19/26

Sample Size and Power - Part II
Superiority Trials
Non-Inferiority Trials
Equivalence Trials
Active Control
Placebo-Controlled Trials
Intention-to-Treat Analysis
Protocol Analysis
Two One-Sided Tests (TOST)
Confidence Intervals
Sample Size Calculation
Statistical Power
Clinical Trial Design
Hypothesis Testing
Hazard Ratio
Binary Outcomes
Continuous Outcomes
Time-to-Event Outcomes
8/19/26

Late Phase Studies: Interim Analyses
Interim Analysis
Group Sequential Methods
Alpha Spending Functions
Data Safety Monitoring Board (DSMB)
Conditional Power
Futility Assessment
Adaptive Designs
Clinical Trial Monitoring
Type I Error Control
O'Brien-Fleming Method
Pocock Method
Haybittle-Peto Method
8/19/26

Factorial Design
Factorial Design
Treatment Interactions
Drug Combinations
Factorial Clinical Trials
Main Effects
Interaction Effects
Qualitative Interactions
Quantitative Interactions
Incomplete Factorial Design
Combination Therapy
Min Test
Experimental Design
8/19/26

Crossover Designs
Crossover Designs
Repeated Measures Design
2×2 Crossover Trials
Carryover Effects
Washout Period
Sequence Effects
Period Effects
Balanced Designs
Latin Squares
Bioequivalence
McNemar's Test
Logistic Regression
Mixed-Effects Models
Continuous Outcomes
Binary Outcomes
Time-to-Event Outcomes
8/19/26
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