Basics of Study Design - Boston University Medical Campus ：研究设计-波士顿大学医学校区基础.pptVIP

Basics of Study Design Janice Weinberg ScD Assistant Professor of Biostatistics Boston University School of Public Health Basics of Study Design Bias and variability Randomization: why and how? Blinding: why and how? General study designs Bias and Variability The clinical trial is considered to be the “gold standard” in clinical research Clinical trials provide the ability to reduce bias and variability that can obscure the true effects of treatment Bias ? affects accuracy Variability ? affects precision Bias: any influence which acts to make the observed results non-representative of the true effect of therapy Examples: healthier patients given treatment A, sicker patients given treatment B treatment A is “new and exciting” so both the physician and the patient expect better results on A Many potential sources of bias Variability: high variability makes it more difficult to discern treatment differences Some sources of variability Measurement instrument observer Biologic within individuals between individuals Can not always control for all sources (and may not want to) Fundamental principle in comparing treatment groups: Groups must be alike in all important aspects and only differ in the treatment each group receives In practical terms, “comparable treatment groups” means “alike on the average” Why is this important? If there is a group imbalance for an important factor then an observed treatment difference may be due to the imbalance rather than the effect of treatment Example: Drug X versus placebo for osteoporosis Age is a risk factor for osteoporosis Older subjects are enrolled in Drug X group Treatment group comparison will be biased due to imbalance on age How can we ensure comparability of treatment groups? We can not ensure comparability but randomization helps to balance all factors between treatment groups If randomization “works” then groups will be similar in all aspects except for the treatment received Randomization A