drug efficacy in the wild tim vaughan 8-sep-2011
DESCRIPTION
Drug Efficacy in the Wild Tim Vaughan 8-Sep-2011. PatientsLikeMe – Three brothers’ story . ALS − Rare neurologic disease. ALS − Time is of the essence. PatientsLikeMe web site. Stephen Heywood (alsking101). Data collection. Why is Mike taking lithium?. Lithium. - PowerPoint PPT PresentationTRANSCRIPT
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Drug Efficacy in the WildTim Vaughan8-Sep-2011
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PatientsLikeMe – Three brothers’ story
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ALS − Rare neurologic disease
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ALS − Time is of the essence
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PatientsLikeMe web site
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Stephen Heywood (alsking101)
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Data collection
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Why is Mike taking lithium?
Lithium
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Lithium delays progression of ALS?!
Fornai et al., PNAS 105:2052-2057 (2008)
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The observational study germinates
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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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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
Savvy patients are using the internet in creative ways to understand and improve their health
Structured, self-reported patient data has profound value, despite being subject to bias (like all patient data!)