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Diana Brixner PhD, 2007-2008 ISPOR President and Associate Professor and Chair of the Department of Pharmacotherapy and
Executive Director of the Pharmacotherapy Outcomes Research Center at the University of Utah College of Pharmacy, Salt Lake
City, UT, USA
Translational Research and the Value Equation
A recent commentary published in the Journal
of the American Medical Association
addressed the “Meaning of Translational
Research and Why it Matters” [1]. In light of
ISPOR related topics such as outcomes
research, comparative effectiveness, cost-effectiveness,
and pharmacoeconomics, it seems
timely to consider where these disciplines fit in
the continuum of translational research.
As Woolf acknowledges in his article, translational
research means different things to different
people. The more traditional definition of “bench
to bedside” encompasses drug discovery
(medicinal chemistry), drug formulation (pharmaceutics),
drug testing (pharmacology and
clinical development), and patient care (pharmacotherapy).
These definitions align particularly
well with the Departments in our own College of
Pharmacy at the University of Utah, and, most
likely, with various other colleges and pharmaceutical
companies across the globe. The
Institute of Medicine (IOM) Clinical Research
Roundtable has labeled this definition as “translational
block one”, or T1 [2]. The components
of T1 have been traditionally funded by individual
National Institutes of Health (NIH) institutes and
now collectively through the NIH roadmap initiative
[3] and by the launch of the Clinical and
Translational Science Award (CTSA) with a goal
of $500 million in funding across 60 academic
centers by 2012.
An alternative definition of translational research,
perhaps more relevant to the disciplines represented
by ISPOR and other population-based
organizations such as the International Society of
Pharmacoepidemiology, Academy Health,
Society for Medical Decision Making and others,
would be “translating research into practice”.
Here the disciplines of epidemiology, evidencebased
synthesis, economics, public policy,
behavioral science, and biostatistics play a much
larger role in understanding how the real world of
clinical practice, patient behaviors, and concomitant
disease can impact the predictions of the
highest quality research produced by randomized
clinical trials. The IOM Clinical Research
Roundtable labeled this as ”translational block
two” or T2 [2]. The challenges of this type of
research abound. In specific research on how a
new technology can be introduced into practice,
one must consider the nuances of a health care
system that has previously operated in the
absence of such technology. Treatment guidelines
are reviewed and revised, systematic
reviews are redone, and documentation, such as
the Academy of Managed Care Pharmacy
(AMCP) Dossier and dossiers for other global
region reimbursement agencies, is prepared and
updated. The evidence that is created to support
these documents is largely conducted in patient
populations from primary care physician practices,
integrated health care systems, and national
administrative claims databases. This “real
world” research can include assessment of the
impact of therapeutic
guidelines on the
treatment and outcomes
of disease, the
impact of disease or
medication therapy
management intervention
programs,
and health services research to evaluate the benefit
of vaccination or diagnostic screening programs.
The practice-based component of this
research is primarily conducted in community or
ambulatory settings to provide insight on the
impact and outcomes of implementing new technologies.
The practice-based research was
potentially referred to as T3 by Westfall and colleagues
[4] to more specifically acknowledge
this practice-based approach. Either way the
dynamics of funding research in the T-2 T-3
arena is far different than the funding of the more
traditional T1 translational research. The adaptation
of the NIH towards translational research had
dedicated $787 million of a $22.1 billion budget
to health services research, and the previously
mentioned resources have been directed towards
the CTSA, however, with a focus on T1. In fact
academic centers across the U.S have struggled
with how to incorporate community-based
primary care research and pharmacotherapy outcomes
research into CTSA grant applications. A
more likely source for practice-based research
has been the Agency for healthcare Research and
Quality (AHRQ) with the charge that research
results are widely disseminated and used in
healthcare decision making [5]. However, the
funding for this mission is sparse with roughly a
$300 million budget initially targeted at translational
research in practice grants with a shift
toward special project research on patient safety
and informatics.
A larger concern is that the conversation of defining
translational research largely stops here.
Although we can study the implementation of
technologies into practice, we have neither discussed
research that is focused on studying the
comparative effectiveness of different technologies
nor the associated costs. Yet these two
disciplines are the core principles of ISPOR. The
benefit and limitations of conducting practice
based research have been largely recognized and
many of these same limitations apply to real
world effectiveness studies; however, there is also acknowledged benefit of this information to
the payer. Drugs and/or other technologies
should not be expected to act the same way
when patient behaviors, disease states, and other
factors are not controlled as they are in randomized
trials, and these differences can be important
in making coverage decisions. The process
of rational health care resource allocation should
include economics, but how this information is
collected and how it is interpreted are important
considerations. These ongoing debates are also
reflected in the associated funding sources, both
for comparative and cost effectiveness, which at
this juncture are largely absent outside of those
vested in the results.
In summary, there is a continuum of research
from bench to bedside to practice to resource
allocation where the clarity of definition seems to
wane and the funding sources seem to diminish.
However, in all likelihood, this is a reflection of
the societal changes in how medicines are discovered,
applied, and paid for to prevent disease
and improve human health. If we include comparative
effectiveness and cost-effectiveness as
logical next steps in the continuum of translational
research, we should be pleased to know
there is room for many more transitional states in
the future.
References
1. Woolf SH. The meaning of translational research and why it
matters. JAMA 2008;299:211-13.
2. Sung NS, Crowley EFJr., Genel M, et al. Central Challenges
facing the national clinical research enterprise. JAMA
2003;289:1278-87.
3. http://nihroadmap.nih.gov/ Accessed January 16, 2008.
4. Westfall JM, Mold J, Fagnan L. Practice Based research -
“blue highways” on the NIH roadmap. JAMA 2007;297:403-6.
5. Agency for Healthcare Research and Quality. Budget estimates
for appropriations committees, fiscal year (FY) 2008:
performance budget submission for congressional justification.
Performance budget overview 2008
http://www.ahrq.gov/about/cj2008/cjweb08a.htm#statement
Accessed November 17, 2007
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