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Proprietary & Confidential 1
Drug Efficacy in the WildTim Vaughan17 June 2011
Proprietary & Confidential 2
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