Lecture given to the Clinical and Translational Sciences Seminar course at the University of Miami, December, 2012.

1. research
^
but it will be tweeted
Clinical and Translational Science Seminar Series
12/12/12
Richard J. Bookman
rbookman@miami.edu
@rbookman

2. “The United States is in the midst of
a profound reorganization of how
research is done, where it is done,
who does it, and how its results find
their way to the marketplace.”
opening sentence of
Transformation and Opportunity: The Future of the U.S. Research Enterprise, by the
President’s Council of Advisors on Science and Technology (PCAST)
November 30, 2012
http://www.whitehouse.gov/sites/default/files/microsites/ostp/pcast_future_research_enterprise_20121130.pdf

4. NIH Purchasing Power – 2003-2013
NIH Purchasing Power – 2003-2013
$32 $31.0
$30.5 $30.7 $30.6 $30.7
$30 $29.3
$29.0
$27.9 $28.5 $28.5
$27.1
$28 $26.9 $26.4
Billions
$25.3
$26 $24.8
$23.9 $24.2 $23.9
$24
$23.0
$22.4
$21.9
$22
$20
$18
2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013
Current Constant (BRDPI) Senate
Labor-HHS BA only
Labor-HHS NIHonly
Sources: BA Budget Office; House and Senate Appropriations Committees
Sources: NIH Budget Office; House and Senate Appropriations Committees

6. Nature 477, 526-528 (2011) | doi:10.1038/477526a

8. Over the past decade, before pursuing a particular line of research, scientists (including
C.G.B.) in the haematology and oncology department at the biotechnology firm Amgen in
Thousand Oaks, California, tried to confirm published findings related to that work. Fifty-
three papers were deemed 'landmark' studies. It was acknowledged from the outset that
some of the data might not hold up, because papers were deliberately selected that
described something completely new, such as fresh approaches to targeting cancers or
alternative clinical uses for existing therapeutics. Nevertheless, scientific findings were
confirmed in only 6 (11%) cases. Even knowing the limitations of preclinical research, this
was a shocking result.

9. our challenge

11. “Institutions seek to
preserve the problem for
which they are the
solution.”
- Clay Shirky

13. John von Neumann at IAS, Princeton, 1945
From G. Dyson, Turing’s Cathedral, 2012

14. 2012: a tipping point year?
Majority of computers sold are smartphones
50% of all US book* sales are e-books
Online movie views surpass in-theatre views
3-D printers fall below $1000 & self-replicate!
* non-textbook sales

15. every
the internet of things
^
big data

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

17. disintermediation
reintermediation

18. open platforms:
weather
GPS
google maps

19. re-useful is the new useful

20. Research 3.0
who’s doing research?
who’s paying for research?
how is research organized?
how is research communicated?

21. open platforms
for
collaborative discovery

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

27. Randomized controlled trial
vs.
Observational trial

29. open platforms
for
collaborative discovery

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

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

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

34. 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

36. http://artisopensource.net/cure/

38. “If patient engagement were a
drug, it would be the blockbuster
drug of the century and
malpractice not to use it.”
Leonard Kish
http://www.hl7standards.com/blog/2012/08/28/drug-of-the-century/

41. Research 3.0
who’s doing research?
who’s paying for research?
how is research organized?
how is research communicated?

42. research
^
but it will be tweeted