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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