University of Miami's Clinical and Translational Sciences Institute (CTSI) runs a bootcamp course for medical residents & fellows and postdocs. This is my 2012 version of "Questions for knowledge creators" lecture.
1. Questions for knowledge creators
richard j. bookman
rbookman@miami.edu
2012 Bootcamp Course
university of miami
12 September 2012
2. disclosures
• I have no financial interest in any of the
content presented today.
• I have no direct experience in translational
or clinical research
36. Abstract
Patients with serious diseases may experiment with drugs
that have not received regulatory approval. Online patient
communities structured around quantitative outcome data
have the potential to provide an observational environment
to monitor such drug usage and its consequences. Here
we describe an analysis of data reported on the website
PatientsLikeMe by patients with amyotrophic lateral sclerosis (ALS)
who experimented with lithium carbonate treatment.
To reduce potential bias owing to lack of randomization, we
developed an algorithm to match 149 treated patients to multiple
controls (447 total) based on the progression of their disease course.
At 12 months after treatment, we found no effect of lithium on
disease progression. Although observational studies using unblinded
data are not a substitute for double- blind randomized control trials,
this study reached the same conclusion as subsequent randomized
trials,
http://www.nature.com/nbt/journal/v29/n5/pdf/nbt.1837.pdf
40. “Patient engagement is the
blockbuster drug of the 21st
century”
Leonard Kish
http://www.hl7standards.com/blog/2012/08/28/drug-of-the-century/
41. summary
What problem do you want to work on?
How does your own brain limit your ability to reason?
Have you fallen in love with your own ideas?
Are you looking for black swans?
In a digitally enabled world, can you think of better ways to
create useful knowledge?
How can you empower patients so as to accelerate discovery?
Editor's Notes
Image is Gerhard Richter painting.http://www.gerhardrichterpainting.com/wp-content/uploads/2012/01/gerhardrichter_photo3.jpg
Translation is supposed to get us across the valley of death…
But something Is amiss….
This course will probably not turn you all into riders of death valley…
Scientific method is part of how we try to overcome the limits….
falling in love with your own ideas2,4,6 gameblack swansrole of discovery
JvN: “I am thinking about something much more important than bombs. I am thinking about computers.”The IAS computer in 1952.Numbers that mean things vs. numbers that do things.Code that can modify code.
