Richard J. Bookman's presentation at the University of Miami boot camp discusses the importance of problem selection in research and the evolving landscape of clinical research, particularly with the rise of patient engagement and online communities. The document highlights a study examining the effects of lithium carbonate on ALS patients, concluding no significant impact on disease progression despite the observational nature of the research. It emphasizes the need for researchers to reevaluate their approach in a digital age and the empowerment of patients in accelerating scientific discovery.

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

3. objectives
leave you with questions

7. How do you pick a problem?

9. Research is a human activity

10. http://www.stanford.edu/group/vista/cgi-bin/FOV/wp-content/uploads/2012/02/tables1.png

12. Which offer do you want?

13. Which offer do you want?

14. results
68% 16%
32% 84%
Dan Ariely, Predictably Irrational

16. Hypothesis-driven vs.
discovery-driven
research

17. Let’s play a game

19. basic applied

20. fundamental
useful
?
D.E. Stokes, Pasteur’s Quadrant, 1997

21. fundamental
useful

23. From G. Dyson, Turing’s Cathedral, 2012

24. the internet of things
big bang

25. every
the internet of things
^
big data

26. http://doi.ieeecomputersociety.org/10.1109/MIS.2009.36

27. research 3.0

31. how are these trends
playing out in clinical
research ??

32. http://www.patientslikeme.com

33. http://www.pnas.org/content/105/6/2052

34. Methods
http://www.pnas.org/content/105/6/2052

35. http://www.nature.com/nbt/journal/v29/n5/full/nbt.1837.html

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

37. http://artisopensource.net/cure/

38. Salvatore Iaconesi
Founder, Art is open source

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?