Clinical Trial Design Issues and Options for the Study of Rare Diseases

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Sample Size Required for a Given PowerResponse rateDrug Placebo DifferenceSingle Phase Design 60% 45% 15%Total nPower70%80%90%SinglePhase Design274346462
Single Enhanced DesignRandomizeTreatmentPhase 11 2ActiveTreatmentPlaceboResultsResponderNonResponderResponderNonResponderRandomizeTreatmentPhase 23 4ActiveTreatmentPlacebo
Page 1 and 2: Clinical Trial DesignIssues and Opt
Page 3 and 4: The Issue The Implication• Few pa
Page 5 and 6: Possible Design Options• Parallel
Page 7 and 8: Crossover DesignsA1A2Difficulty get
Page 9 and 10: Treatment Effects
Page 11 and 12: Using Crossover Designs• Chronic
Page 13 and 14: Factorial DesignsCADifficulty getti
Page 15 and 16: Factorial Design Analysis
Page 17 and 18: InteractionsAABBCD
Page 19 and 20: Advantages and Limitations• Advan
Page 21 and 22: N-of-1 Randomized Trial Design• I
Page 23 and 24: Advantages and Limitations• Advan
Page 25: . Conventional designRandomizeTreat
Page 29 and 30: Comparison of Sample sizes between
Page 31 and 32: . Conventional designRandomizeTreat
Page 33 and 34: Summary• When the number of study
Page 35 and 36: References• Guyatt, GH, Sackett D

Clinical Trial Design Issues and Options for the Study of Rare Diseases

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