Drug Efficacy in the Wild Tim Vaughan 8-Sep-2011
Feb 23, 2016
Proprietary & Confidential 1
Drug Efficacy in the WildTim Vaughan8-Sep-2011
Proprietary & Confidential 2
PatientsLikeMe – Three brothers’ story
Proprietary & Confidential 3
ALS − Rare neurologic disease
Proprietary & Confidential 4
ALS − Time is of the essence
Proprietary & Confidential 6
PatientsLikeMe web site
Proprietary & Confidential 7
Stephen Heywood (alsking101)
Proprietary & Confidential 8
Data collection
Proprietary & Confidential 9
Why is Mike taking lithium?
Lithium
Proprietary & Confidential 10
Lithium delays progression of ALS?!
Fornai et al., PNAS 105:2052-2057 (2008)
Proprietary & Confidential 11
The observational study germinates
Proprietary & Confidential 12
Timeline
Proprietary & Confidential 13
Patients track their progress
Proprietary & Confidential 14
The “kitchen sink” plot
Proprietary & Confidential 15
Random control may not be a “patient like me”
Proprietary & Confidential 16
Demographics – age
Proprietary & Confidential 17
Demographics – onset site
Proprietary & Confidential 18
Demographics – sex
Proprietary & Confidential 19
Matching algorithm
Proprietary & Confidential 21
Pre-treatment progression bias reduced
Proprietary & Confidential 22
Results of lithium treatment
Proprietary & Confidential 23
Kaplan-Meier for patients & data
Proprietary & Confidential 24
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
Proprietary & Confidential 25
What Mike (and PatientsLikeMe) can learn
Proprietary & Confidential 26
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!)