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

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N-of-1 Randomized Trial DesignABBAABDrug and placebo administeredsequentially in a randomsequence, generally 3 or moredrug-placebo pairs.
N-of-1 Randomized Trial Design• If patient’s response was poor, that treatmentstopped and the next treatment in the sequencebegun immediately without breaking therandomized sequence.• At the end of the study, the mean values for allmeasures and the mean differences betweentreatments are computed.
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: Advantages and Limitations• Advan
Page 23 and 24: Advantages and Limitations• Advan
Page 25 and 26: . Conventional designRandomizeTreat
Page 27 and 28: Single Enhanced DesignRandomizeTrea
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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