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Another insight is obvious to those who engage in
biomedical research, but the message is likely
underappreciated by the American public.
Biomedical research provides the basis for
progress in health and health care.
Nabel EG. J Clin Invest 2009;119:2858
3
4 Science 1965:149:1394-5
5
6
“Whether or not the correction of these abnormalities once
they are discovered will favorably alter the risk of
development of disease, while reasonable to contemplate
and perhaps attempt, remains to be demonstrated…”
Annals Internal Med 1961;55:33-50
7 JAMA 1979;242:2562-71; JAMA 1991;265:3255-3264
8 N Engl J Med 2010;362:1575-85
BHAT 1981
ALLHAT 2002
Primary and Secondary Prevention
NHBPEP
1972
CPPT 1984
HDFP 1979
NCEP 1985
Risk Factors defined
1961 Risk Factors
CASS
1983
TIMI 1985
Physical Intervention
1980
Year
400
300
200
100
0
Deaths/100,000 Population
500
600
1970 1960 1950 1990 2000 2010
9
10 Cutler D et al. N Engl J Med 2006;355:920-7
11
Editorial
Damage From Brain Stents Published: September 8, 2011
“…. The stents had been approved for humanitarian use by the FDA in 2005.
That approval was based on a 45-person trial that lacked a control group.
Optimistic surgeons have since inserted the devices in thousands of people.
Now [a] rigorous controlled study of some 450 patients has shown that those
who simply had treatment with drugs and lifestyle changes fared better than
those who got the stents as well. This case…clearly shows the value of
conducting rigorous controlled studies with enough patients to provide
meaningful results. This is just the kind of ‘comparative effectiveness’
research that the national health care reforms seek to promote.”
12 Chimowitz MI, et al. N Engl J Med 2011;365:993-1003
13
“It seems reasonable to recommend antiarrhythmic treatment as a
means of reducing postinfarction risk. However, no study has been done
to show that antiarrhythmic drug treatment can reduce mortality after
infarction. Our results do encourage the pursuit of further studies.”
Bigger JT et al. Circulation 1984;69:250-8
14 Hine LK et al. Arch Intern Med 1989;149:1524-7
“The late 1960s and 1970s witnessed a flurry of reports on
arrhythmias and sudden death and it became recognized
that patients with ventricular ectopy are at increased risk for
premature mortality… Antiarrhythmic agents were prescribed
with the hope that suppressing ventricular ectopy would
improve survival of these patients with heart disease.”
15
“The suppression of
VPCs alone in this
population is not an
adequate indication
that a drug will be
helpful in prolonging
survival…This study
emphasizes the
need for … trials
…with mortality
endpoints.”
N Engl J Med 1989;321:406-12
16 Bardy G et al. N Engl J Med 2005;352:225-37
17
“…We believe that confirmation of these results in a
prospective randomized trial is important before this therapy
can be accepted for widespread use. Many new therapies,
initially promising, fizzle. This treatment should only be
offered at major centers…and, whenever possible, [into]
randomized comparative trials…”
Peters WP, et al. J Clinical Oncology 1993;11:1132-43
18
“… By the time Peters had organized his trial, few women
wanted to participate…[It] meant running the risk of not
getting high-dose chemo, and many had read newspaper
accounts that convinced them that the treatment was their
only chance for survival. Their doctors often agreed. One
transplanter pulled out a copy of Peters' 1993 paper. ‘I don't
see how it's even ethical to do a randomized trial,’ he said.”
Brownlee S. Discover Magazine 2002.
19
“… From the moment Peters first administered high-dose chemotherapy
until the first clinical trials were concluded, nearly 20 years passed.
During that time, hundreds of physicians practiced the unproven
treatment. An estimated 30,000 breast cancer patients suffered through
high-dose chemotherapy, only a fraction of them as part of a clinical
trial. All told, the nation spent around $3 billion paying for it, while an
estimated 4,000 to 9,000 women died not from their cancer but from the
treatment…”
Brownlee S. Discover Magazine 2002.
Peters WP et al. J Clinical Oncology 2005;23:2191-2200
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22 Tricoci P et al. JAMA 2009;301:831-41
Nearly 50% of recommendations are based on expert opinion.
Only 11% are based on multiple randomized trials.
23
“It's hard not to scream when you see how many physicians,
pharmaceutical companies, [and] medical-device
makers…seem to hate science, or at best ignore it. These
days the science that inspires fear and loathing is
‘comparative-effectiveness research.’”
Sharon Begley, Newsweek, Feb 28 2009
24 Sharon Begley, Newsweek, Feb 28 2009
25 Thank you to Andrew Epstein
26 Smith GCS, Pell JP. BMJ 2003;327:1459-61
27
28 Kahneman D. Thinking Fast and Slow. FSG 2011
29 Tversky A, Kahneman D. Science 1974;85:1124-31
30
31 Akerlof GA. Q J Economics 1970;84:488-500
32
33 Wheelan C. W. W. Norton, 2010
“The National Marfan Foundation does not recommend switching
from a beta blocker to losartan as a way to manage Marfan syndrome
until the trial is completed. This is because we do not know
whether losartan is clearly better than atenolol for taking care of people
with Marfan syndrome.”
http://www.marfan.org/marfan/2408/Atenolol-vs.-Losartan-Clinical-Trial
36 Wheelan C. W. W. Norton, 2010
37
“Biomedical knowledge is a public good, available to any
individual even if that individual does not contribute to it.
Participation in research is a critical way to support an
important public good. Consequently, all have a duty to
participate. The public goods argument implies that
individuals should participate unless they have a good
reason not to. Such a shift would be of great aid to the
progress of biomedical research, eventually making society
significantly healthier.”
Schaefer GO, Emanuel EJ, Wertheimer A. JAMA 2009;302:67-72
38
29%
47%
19%
5% Very likely
Somewhat likely
Not likely
Would not participate
Please consider your own possible participation as a volunteer in clinical
research. How likely would you be to participate in a clinical research
study?
Thanks to Mary Wooley (Research America)
39
Has your doctor ever suggested that you participate in a clinical
research study?
6%
94%
Yes
No
Thanks to Mary Wooley (Research America)
40
41 http://media-1.web.britannica.com/eb-media/20/144220-004-3E7E60A9.jpg
42 Van Helmont JA. Oriatrike. London: Lodowick-Loyd, 1662, p.526
43 Van Helmont JA. Oriatrike. London: Lodowick-Loyd, 1662, p.526
