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

More documents

Recommendations

Info

Comparison of Sample Size Required fora Given PowerResponse rateDrug Placebo DifferenceSingle Phase Design or SPCD Phase 1 60% 45% 15%SPCD Phase 2 50% 25% 25%1.6XPower70%80%90%Total nSinglePhase Design274346462SPCD157199266
Comparison of Sample sizes between Parallel and SPDdesigns for three scenarios with low placebo response rate.Stage 1 Stage 2 Parallel SPDDrug Pbo Drug Pbo n nScenario 1 .40 .20 .30 .10 164 101Scenario 2 .30 .15 .20 .05 242 134Scenario 3 .25 .10 .20 .05 200 116All calculations are based on two-tailed alpha = 0.05, 80% power. Forthe SPD, the estimates are based on a 2:1 initial allocation ofplacebo:drug, and assumed retention of placebo patients from Stage 1to Stage 2 = 90%.
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: Single Enhanced DesignRandomizeTrea
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

You also want an ePaper? Increase the reach of your titles

Delete template?

Save as template?