Intervention TrialsPrinciples of study design.ppt

Intervention TrialsPrinciples of study design James B. Spies M.D.MPH Professor of Radiology Georgetown University School of Medicine Washington D. C. Categories of StudiesUsers Guide to Medical Literature Studies answer one of 4 types of questions: Therapy (efficacy) Randomized trials, other comparative studies Harm Cohort or case control studies Diagnosis Diagnostic studies comparing new study to reference standard Prognosis Cohort studies Efficacy Trials What type of study design? Case reports Case series (+/- historic controls) Retrospective or prospective Prospective comparative studies w/o randomization. Randomized comparative studies Non-randomized designCase Series- Weaknesses Selection bias The investigator controls recruitment and may enroll those who are better candidates for the intervention compared to the typical patient. Does not allow for control of any known or unknown confounders in subjects. Often done retrospectively, in which incomplete medical records are abstracted. Does not allow for clear cut definition and recording of baseline characteristics, technical aspects of therapy, or outcome. Non-randomized designCase Series No controls provides no means of assessing study impact. Before and after within subject control does not allow assessment of placebo effect (if not blinded) and any improvement that may occur without intervention. Historical controls subject to even greater selection bias Research staff collecting data are typically not blinded and significant assessment bias may be introduced. Non-randomized designCase Series Investigators not blinded Researchers may have vested financial interest in positive outcome. Investigators may be on company speaker’s bureaus, be consultants, or on medical advisory board. This is why investigators required to report conflicts at presentations and in publications. Red Flag. May have a personal vested interest The study is usually done with the intent and the hope that the new therap