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

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n-of-1 Randomized Trial• Disorder should be chronic, i.e. relativelyunchanging.• Treatment effect rapid.• Treatment duration for optimal effect should bewell known.
Advantages and Limitations• Advantages:• Every patient receives every treatment.• Treatment is evaluated in each patient.• Limitations:• Study could take very long.• Refusal bias.• Difficult to know whether design assumptionsare met (duration of treatment foroptimal effect ).
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: N-of-1 Randomized Trial Design• I
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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