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Drug Efficacy in the Wild Tim Vaughan 17 June 2011. Contents. PatientsLikeMe What can MikeFromFinland teach us, and vice versa? Lithium delays progression of ALS?! PatientsLikeMe’s observational study Finding patients like me Results Predictive modeling / What is my outcome? - PowerPoint PPT Presentation
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Drug Efficacy in the WildTim Vaughan17 June 2011
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Contents
PatientsLikeMe What can MikeFromFinland teach us, and vice versa? Lithium delays progression of ALS?! PatientsLikeMe’s observational study Finding patients like me Results Predictive modeling / What is my outcome? Concluding remarks
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PatientsLikeMe web site
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PatientsLikeMe background – Three brothers
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Stephen Heywood (alsking101)
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What can Mike teach us, and vice versa?
Lithium
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Lithium delays progression of ALS?!
Fornai et al., PNAS 105:2052-2057 (2008)
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Timeline
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Patients track their progress
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The “kitchen sink” plot
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Random control may not be a “patient like me”
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Demographics – age
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Demographics – onset site
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Demographics – sex
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Matching algorithm
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Matching across the entire sample
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Pre-treatment progression bias reduced
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Results of lithium treatment
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Kaplan-Meier for patients & data
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Biases and other stuff that worried us
Self-selection for treatment “Recruitment bias” Data reported (vs. data opportunity) Outliers (e.g. PMA and PLS) “Optimism bias” at treatment start
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What Mike (and PatientsLikeMe) can learn
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Conclusions
Structured, self-reported patient data, despite being subject to bias (like all patient data!), has value
Think about bias, and then think about bias again (Repeat) “Pair programming” for statistics