Design of Clinical Research Protocols .pptVIP

Study Design and Hypothesis Testing in Clinical Research Jonathan J. Shuster, Ph.D jshuster@biostat.ufl.edu Research Professor of Biostatistics Univ. of Florida, College of Medicine Take-home Messages Rely on Evidence-Based Medicine. Conventional wisdom can easily lead us astray. The objective of Statistics is to make informed inferences about a population, based on a sample. It is imperative to quantify the uncertainty. The P-value is a quantity that allows us to infer something about whether a scientific hypothesis is false. Non-significant results are inconclusive Randomization and intent-to-treat are vital components in sound clinical research Topics 1. Motivating Evidence-Based Clinical Studies 2. Objective of Statistics 3. Hypothesis testing and P-values 4. Real Examples and their lessons 1. Motivating Evidence-Based Medicine A coin is “loaded”, with a 70% chance of landing heads. One player picks a three outcome sequence e.g. HTH , then the other picks a different sequence. Whoever’s sequence comes up first is the winner. Do you want to choose first, and if so, what sequence to you select? Evidence-Based Medicine So you decided to go first and pick HHH, right? OK, I pick THH. HHH can only occur before THH if it is on the first three flips. If the first time HHH occurs is flips 6,7,8 then flip 5 is T, so flips 5,6,7 are THH, I win. I make your first 2, my last 2, so I tend to stay ahead. Your chance of winning .73 .343 34.3% Evidence-Based Medicine Lesson from this example. Things are not always what they seem. You need to be a healthy skeptic. Reference: Shuster, J. A two-player coin game paradox in the classroom. American Statistician, 2006 Feb , vol 60, pp 68-70. 2. Objective of Statistics To make an inference about a defined target population from a representative sample. That is, for us, to start from a medical hypothesis about a medical condition, help design a study that can collect data to test the question, and draw conclus