Lecture 7_Barraj

Lecture 7_Barraj - Clinical Trials Design and Analysis:...

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Clinical Trials Design and Analysis: Statistical Issues Leila Barraj D.Sc. May 11, 2011 1
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References c Fundamentals of Clinical Trials. Friedman L, Furber C, DeMets D, 3rd edition, 1998, Springer-Verlag, NY c Small Clinical Trials: Issues and Challenges. Evans CH and Ildstad ST. (eds). 2001. Nat. Acad. Press ,DC. c Guidance for Industry. E9 Statistical Principles for Clinical. Trials. U.S. Department of Health and Human Services. Food and Drug Administration ttp://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/u http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/u cm073137.pdf c Statistical Guidance for Clinical Trials of Non-Diagnostic Medical Devices http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/ucm106757.htm c Guidance for the Use of Bayesian Statistics in Medical Device Clinical Trials. http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/ucm071 121.pdf 2
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Outline Definition Design Types Sample size and statistical power Blinding Planned statistical analyses Bayesian statistics 3
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Definition “… any type of planned experiment which involves patients and is designed to elucidate the most appropriate treatment of future patients under a given medical condition (Pocock, S.J. Clinical Trials: A Practical Approach (1983). John Wiley and Sons, New York) The clinical trial is a carefully, and ethically, designed experiment with the aim of answering some precisely framed question (Hill, A.B. Principles of Medical Statistics. (1967). Oxford University Press, New York) 4
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Clinical Trials - Phases Multiple types of trials Designed to address the various phases of development of new drug or medical device 5
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Phase I Clinical Trials Clinical pharmacology and toxicity Safety Small group of volunteers Narrow subgroup of patient population 6
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Phase II Clinical Trials Initial clinical investigation for treatment effect fety ND fficacy Safety AND efficacy Larger sample size 7
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Phase III Clinical Trials Full-scale evaluation Comparison with standard treatment Randomization and blinding to avoid bias Larger sample size Patients representative of larger target population 8
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Phase IV Clinical Trials Post-market surveillance Long term studies Assess performance of device/procedure performance after a few years Investigate if unforeseen side effects Typically: no controls 9
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Type of Trials Superiority: Show that the new treatment is better than the standard (or placebo) on feriority/equivalence: Non-inferiority/equivalence: Show that the new treatment is not worse than the standard by more than a certain margin 10
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Design Aims to control (or minimize) known or suspected sources of bias and other errors Bias: systematic tendency of any factors associated with the design, conduct, analysis, and
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This note was uploaded on 07/08/2011 for the course BM 501 taught by Professor Kop during the Spring '11 term at Bloomsburg.

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Lecture 7_Barraj - Clinical Trials Design and Analysis:...

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