Comparative Effectiveness Research: Challenges for Medical Journals
Authors: Harold C. Sox, Mark Helfand, Jeremy Grimshaw, Kay Dickersin, David Tovey, J. André Knottnerus, Peter Tugwell
Journal: PLoS Medicine (2010)

In order to optimize health outcomes within the constraints of inevitably limited resources, low- and high-income countries alike require unbiased means of assessing health care interventions for their relative effectiveness. Such interventions include diagnostic tests and treatments (both established and newly developed) and implementation of health policy [1]. Likewise, health care professionals and patients need better information to inform health care decisions that require weighing benefits and risks in light of the patient's medical history and personal preferences.

Some countries and international organizations have recognized the need for such evidence and are already allocating funds for research to provide it [2]. The WHO Ministerial Summit in Mexico called for the establishment of support for a substantive and sustainable program of health systems research aligned with countries' priority needs and aimed at achieving internationally agreed-upon health-related development goals, including those contained in the United Nations Millennium Declaration [3]. The UK has established the National Institute for Health Research to commission and disseminate research that supports decision making by professionals, policy makers and patients and to ensure that the UK's health system, the National Health Service, has access to the best possible evidence to inform decisions and choices [4].

The US is now addressing similar goals with an initiative known as comparative effectiveness research (CER). In 2008, a report by the US Institute of Medicine (IOM) noted that patient care “should be based on the conscientious, explicit, and judicious use of current best evidence” [1]. In legislation that allocated US $1.1 billion in the US for CER on health care practices in 2009, the US Congress mandated that the IOM set national priorities for CER clinical topics. The IOM defined CER 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” [5]. The definition further stated that “The purpose of CER 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.”

To the authors and endorsers of the present Editorial, the potential value of research with these characteristics is self-evident. The challenge will be to realize the full potential of such research to improve health. Doing so will require assessing a heterogeneous body of evidence consisting of prospective randomized trials—including pragmatic trials—and observational research using data obtained in the course of regular practice. Hence, medical journals must use rigorous approaches, including but not limited to peer review by independent experts, to assess the limitations inherent in such research, such as missing data, incomplete follow-up, unmeasured biases, the potential role of chance, competing interests, and selective reporting of results.

Drawing on many years of collective experience in assessing these issues in the course of evaluating health research through peer review, we support the following principles and standards for CER.