More research is needed—but what type?

Despite all the attention to (and contention about) comparative
effectiveness research in the US, much of it very well summarized in the
recent paper by Tunis and Pearson [1], it is striking that most
publications and websites fail to provide a comprehensive yet boundary-
setting definition of comparative effectiveness research (including,
unfortunately, the Tunis and Pearson paper). Instead, one is likely to
find statements about the drivers, the methods, the intended results, and
beneficiary stakeholders. To paraphrase Godlee [2], this is baffling
indeed, not only because the need for comparative effectiveness research
is so obvious, but also because significant resources will be committed to
an enterprise (much needed) that will shape health care decision-making
from the patient to the policy levels.

For instance, the Federal Coordinating Council defines comparative
effectiveness research as "the conduct and synthesis of research comparing
the benefits and harms of various interventions and strategies for
preventing, diagnosing, treating, and monitoring health conditions in real
-world settings" [3]. The Agency for Health Care Research and Quality
refers to comparative effectiveness research as "a type of health care
research that compares the results of one approach for managing a disease
to the results of other approaches. Comparative effectiveness usually
compares two or more types of treatment, such as different drugs, for the
same disease. Comparative effectiveness also can compare types of surgery
or other kinds of medical procedures and tests. The results often are
summarized in a systematic review." [4] In its Congressionally-mandated
task to identify priorities for comparative effectiveness research, the
Institute of Medicine defined comparative effectiveness research as "the
generation and synthesis of evidence that compares the benefits and harms
of alternative methods to prevent, diagnose, treat and monitor a clinical
condition, or to improve the delivery of care. The purpose of [comparative
effectiveness research] is to assist consumers, clinicians, purchasers,
and policy makers to make informed decisions that will improve health care
at both the individual and population levels."[5]

We faced the challenge of defining comparative effectiveness research
in our efforts to develop an interprofessional postdoctoral fellowship
program in clinical outcomes and comparative effectiveness research in
rural primary care. This program will bring together doctorally prepared
practitioners from nursing, medicine, pharmacy, and public health for
advanced research (80%) and clinical (20%) training. Taking the best from
these definitions yet also aiming to provide more specificity in terms of
foci and methodologies and setting boundaries to both concept and
enterprise, we built on the Institute of Medicine definition but extended
it significantly. Hence we propose the following definition:

"Comparative effectiveness research is the generation and synthesis
of evidence generated through prospective and retrospective studies with
either primary or secondary data sources by:

- Comparing the benefits and harms of alternative methods to prevent,
diagnose, treat and monitor a clinical condition, or to improve the
delivery of care;

- Comparing the same method(s) between different patient groups;

- Comparing the same method(s) between different clinical
environments; or

- Comparing one or more methods across combinations of treatments,
patient groups, and/or environments.

At the clinical level, comparative effectiveness research
investigates methods, already shown to be efficacious in randomized
controlled trials, in real-world settings; i.e., under ordinary and
variable conditions, when prescribed by licensed clinicians with varying
degrees of expertise and practicing across the spectrum of health care
settings, to treat a heterogeneity of patients.

Comparative effectiveness research aims to discover the best methods
for personalizing care to individual patients by broadening the evidence
base, and by providing more, better, and detailed information with which
to craft a management strategy for each individual patient.

The ultimate purpose of comparative effectiveness research is to
assist consumers, clinicians, purchasers, and policy makers to make
informed decisions that will improve health care at both the individual
and population levels."

It is indeed important to adopt a broad but also well-delineated
definition of comparative effects. This, we believe, will facilitate the
mission of the Patient-Centered Outcomes Research Institute and enable it
to fulfill its scientific and public service mandates.

References

[1] Tunis SR, Pearson SD. US moves to improve health decisions. BMJ
2010;341:c4336.

[2] Godlee F. More research is needed - but what type? BMJ
2010;341:c4662.

[3] Conway PH, Clancy C. Comparative-effectiveness research -
implications of the Federal Coordinating Council's Report. N Engl J Med
2009;361:328-330.

[4] See http://effectivehealthcare.ahrq.gov/index.cfm/glossary-of-
terms/. Last accessed 9 September 2010.

[5] Institute of Medicine. Initial national priorities for
comparative effectiveness research. Washington, DC: National Academies
Press, 2009.

Corresponding author: Ivo Abraham, College of Pharmacy, The
University of Arizona, 1295 N. Martin Avenue, Room B-211G, Tucson, AZ
85716, USA. Email: abraham@pharmacy.arizona.edu.