Clinical Trial Design Issues and Options for the Study of Rare Diseases
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Issues• 2 chances to receive the active agent.• Study takes at least twice as long.• Efficiency depends upon response rate of thosereceiving placebo (placebo effect)• Savings in sample size since all patients areused once and some are used twice.
. Conventional designRandomizeTreatmentPhase 11 2ActiveTreatmentPlaceboResultsResponderNonResponderResponderNonResponder
- 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 and 26: . Conventional designRandomizeTreat
- Page 27 and 28: Single Enhanced DesignRandomizeTrea
- Page 29: Comparison of Sample sizes between
- Page 33 and 34: Summary• When the number of study
- Page 35 and 36: References• Guyatt, GH, Sackett D
<strong>Issues</strong>• 2 chances to receive <strong>the</strong> active agent.• <strong>Study</strong> takes at least twice as long.• Efficiency depends upon response rate <strong>of</strong> thosereceiving placebo (placebo effect)• Savings in sample size since all patients areused once <strong>and</strong> some are used twice.
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