Clinical and Translational Science Seminar Series12/12/12

Richard J. Bookmanrbookman@miami.edu

@rbookman

^

but it will be tweeted

research

“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 ofTransformation and Opportunity: The Future of the U.S. Research Enterprise, by the President’s Council of Advisors on Science and Technology (PCAST)November 30, 2012http://www.whitehouse.gov/sites/default/files/microsites/ostp/pcast_future_research_enterprise_20121130.pdf

NIH Purchasing Power – 2003-2013

2003 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 Senate

$18

$20

$22

$24

$26

$28

$30

$32

$27.1$27.9 $28.5 $28.5

$29.0 $29.3

$30.5$31.0 $30.7 $30.6 $30.7

$26.9 $26.4

$25.3$24.8

$23.9 $24.2$23.9

$23.0$22.4

$21.9

Current Constant (BRDPI)

Bill

ion

s

Labor-HHS BA only

Sources: NIH Budget Office; House and Senate Appropriations Committees

NIH Purchasing Power – 2003-2013

Labor-HHS BA onlySources: NIH Budget Office; House and Senate Appropriations Committees

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

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.

our challenge

“Institutions seek to preserve the problem for which they are the solution.”

- Clay Shirky

From G. Dyson, Turing’s Cathedral, 2012

John von Neumann at IAS, Princeton, 1945

2012: a tipping point year?

50% of all US book* sales are e-books

Online movie views surpass in-theatre views

3-D printers fall below $1000

Majority of computers sold are smartphones

* non-textbook sales

& self-replicate!

the internet of things

big data

^

every

\

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

disintermediation

reintermediation

open platforms:weatherGPSgoogle maps

re-useful is the new useful

Research 3.0who’s doing research?

how is research organized?

how is research communicated?

who’s paying for research?

open platforms for

collaborative discovery

Terms of UseSee this page for info, including copyright

how are these trends playing out in clinical research ??

Randomized controlled trial

vs.

Observational trial

open platforms for

collaborative discovery

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

Methods

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

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

AbstractPatients with serious diseases may experiment with drugsthat have not received regulatory approval. Online patient communities structured around quantitative outcome datahave the potential to provide an observational environmentto monitor such drug usage and its consequences. Herewe 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

http://artisopensource.net/cure/

“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/

Research 3.0who’s doing research?

how is research organized?

how is research communicated?

who’s paying for research?

design

spe

c

^

but it will be tweeted

research