如何设计临床试验Study Design 2008_12_18.ppt

* QOL during treatment is clean, mainly affected by treatment QOL during follow-up is complex, it may be affected by various sources that come from both disease, and protocol or non-protocol treatment. It is also easier to lose follow-up information. * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * - how much symptoms may affect QOL of a person - interested in symptoms palliation - intent of the study is not to show survival benefit but an improvement of QOL - toxicity reported by health care professionals may be different from those of patients - baseline information of QOL may contain more information as prognostic factors Blinding (cont’) HOW Allocation – Already discussed Subject – Drugs vs procedures, side effects Therapist – Placebo effects, dose adjustment Outcome – Subject, therapist, role of third party Issues Allocation procedure, drug supply Unblinding procedure Problems on Breaking Blinding Subjects on active drug might: report more favorable outcomes because they expect a benefit be more likely to stay in a study if they knew they were on active drug more sensitive to a favorable outcome or adverse event Subjects in a no-treatment group: Observers might be less likely to identify and report treatment responses Problems on Breaking Blinding (cont’) Knowledge of treatment assignment could affect: vigor of attempts to obtain on-study or follow-up data decisions about whether a subject should remain on treatment or receive concomitant medications or other ancillary therapy decisions as to whether a given subjects results should be included in an analysis choice of statistical analysis Outcome Possible Endpoints Efficacy outcome Response Survival Quality of life Clinical benefit Safety outcome Adverse events Treatment related toxicity QOL Endpoints QOL is not a straight forward concept it is not performance status, not toxicity rating, not tumor measurement, not lab values It refers to multi-dimensional construct phy