Rare Solid Cancers: An Introduction - Slide 3 - P. Bruzzi - Methodological aspects of clinical research in rare cancers
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Rare Solid Cancers: An Introduction - Slide 3 - P. Bruzzi - Methodological aspects of clinical research in rare cancers
- 2. NCCN National Comprehensive Cancer Network Clinical Practice Guidelines in Oncolog y SOFT TISSUE SARCOMA
- 8. 2005
- 11. 2011
- 26. Scientific Method Dominating Theory
- 27. Scientific Method Dominating Theory Observations New Theory
- 28. Scientific Method Dominating Theory Observations Experiment
- 29. Scientific Method Dominating Theory Observations Experiment
- 30. Scientific Method Dominating Theory Observations Experiment New Theory
- 31. Scientific Method in Medicine Standard Therapy
- 32. Scientific Method in Medicine Standard Therapy Laboratory New standard? or trials in other diseases
- 33. Scientific Method in Medicine Standard Therapy Laboratory Clinical TRIAL or trials in other diseases
- 34. Scientific Method in Medicine Standard Therapy Laboratory Clinical TRIAL or cl. studies H0?
- 35. Scientific Method in Medicine Standard Therapy Laboratory Clinical TRIAL or cl. studies New Standard Th. H0? H1?
- 36. Scientific Method in Medicine Standard Therapy Laboratory Clinical TRIAL or cl. studies New Standard Th. H0? H1?
- 37. Scientific Method in Medicine Standard Therapy Laboratory Clinical TRIAL or cl. studies New Standard Th. H0? H1?
- 38. Scientific Method in Medicine Standard Therapy Laboratory Clinical TRIAL or cl. studies New Standard Th. H1? H0?
- 39. Scientific Method in Medicine Standard Therapy Laboratory Clinical TRIAL or cl. studies New Standard Th. H1? H0?
- 40. Scientific Method in Medicine Standard Therapy Laboratory Clinical TRIAL or cl. studies New Standard Th. H1? H0?
- 41. Scientific Method in Medicine Standard Therapy Laboratory Clinical TRIAL or cl. Studies New Standard Therapy H0?
- 46. Example Mortality Tumor X Nil vs A 15% vs 12.5% N=12000 P = 0.0007 H0 Rejected: A is effective in X
- 47. Example Mortality Tumor X Nil vs A 15% vs 12.5% N=12000 P = 0.0007 Tumor Y Nil vs A 15% vs 7.5% N= 240 P=0.066 H0 not rejected: A not shown effective in y
- 52. How large? – Needed number of events =0.05, power = 80%
- 55. Example: Adjuvant therapy in N- triple negative premenopausal BC
- 73. Consequence In rare tumors/conditions, it is not possible to evaluate the effectiveness of a (new) treatment (?!?)
- 74. What can be done?
- 82. Test of significance Mortality Tumor X Nil vs A P = 0.0007 Tumor Y Nil vs A P=0.066
- 83. Test of significance vs Estimates of effect Mortality Tumor X Nil vs A 15% vs 12.5% N=12000 P = 0.0007 Tumor Y Nil vs A 15% vs 7.5% N= 240 P=0.066
- 84. Estimates of effect + Prior Evidence Tumor X 5yrs mortality Trial 1 Nil vs A 15% vs 12.5% (advanced disease) N=12000 P = 0.0007 Trial 2 Nil vs A 15% vs 7.5% (early disease) N= 240 P=0.066
- 85. What if A has a molecular taget present both in X and Y? Mortality Tumor X Nil vs A 15% vs 12.5% N=12000 P = 0.0007 Tumor Y Nil vs A 15% vs 7.5% N= 240 P=0.066
- 86. Interpretation of the two trials CONVENTIONAL Tumor X: P = 0.0007 Tumor Y : P= 0.066 Efficacy of treatment A is proven in X, undemonstrated in Y
- 87. Interpretation of the two trials CONVENTIONAL Efficacy of treatment A is proven in X, undemonstrated in Y BAYESIAN (Posterior) Probability that treatment A significantly (HR<0.9) lowers mortality in tumor X: 80% in tumor Y: 90%
- 101. Once the available evidence has been summarised, it is possible to estimate the probability that the new treatment, when compared to the standard is: a) Definitely worse: Stop b) Much better: RCT not ethical, confirmatory uncontrolled trials (e.g. GIST) c) Neither : RCT necessary and ethically justified
- 110. Adaptive trials
- 121. I-SPY2 Design
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