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

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The Basic Issue• To test efficacy, need a control group.• Choices: literature control, historical controls,concurrent controls, patients as own controls.• Typical design is to recruit/assign to a treatmentgroup/observe each group and compareoutcomes.• BuBut what if you could design astudy using the same patient morethan once?
Possible Design Options• Parallel group design• Cross-over design• Factorial design• Historical controls design• Randomized withdrawal design• Early escape design• n-of-1 design• Group sequential design• Case-Control design• Prospective cohort design• Decision analysis-based design• Ranking and selection design• Adaptive design• Risk-based allocation design• Bayesian designs• Enhanced designsReduce the number ofstudy subjects neededby using enrolled subjectsmore than once.
Page 1 and 2: Clinical Trial DesignIssues and Opt
Page 3: The Issue The Implication• Few pa
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 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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