Harold C. Sox

Can and should good citizenship in the research community be a criterion for promotion?

Cancer care is costly for individuals and in the aggregate. In one study, insured patients with advanced cancer reported median monthly out-of-pocket treatment expenditures of $592.¹ In the aggregate, US expenditures for cancer care were $127 billion in 2013, which was 7% of total health care expenditures.² These costs are projected to be at least $158 billion in 2020.³ Cancer care contributed 6.5% of the growth in health expenditures from 2000 to 2013. Circulatory diseases provide an informative contrast: disability-adjusted life-years have shrunk nearly 20% from 2005 to 2010 while annual expenditures have increased 7%. For cancer, the same figures are 12% and 26%, respectively.²

T he subject of this Point / Counterpoint is the future of clinical practice guidelines. Does it still make sense to devote resources to creating them? Poor adherence is the principal argument against guidelines. Two articles in this issue show low rates of adherence to guidelines for statins and asthma, respectively. 1,2 While its premise is true, the poor adherence argument ignores the good that guidelines do. First, they represent a profession's best efforts to define its standard of practice. Second, health insurance companies use guidelines to help decide their coverage policies. Third, guidelines are the basis for practice measures, which do influence clinical practice. 3 Fourth, the systematic reviews that serve as the basis for practice guidelines are a public good. For these reasons, although practice guidelines have a bright future, they need fixing. I address 3 questions. How can we increase public trust in guidelines? How can we make guidelines more transparent to the public? What is the role of practice guidelines in the era of shared decision making? What is a practice guideline? The 2010 Institute of Medicine (IOM) study entitled "Practice Guidelines That We Can Trust" 4 defined them as follows: "Clinical practice guidelines are statements that include recommendations intended to optimize patient care that are informed by a systematic review of evidence and an assessment of the benefits and harms of alternative care options."

The article by James and colleagues published in JAMA contains the long-awaited guideline for hypertension issued by the panel members appointed by the National

In Reply: The purpose of our Viewpoint was to describe the unique contributions of the IOM standards for developing practice guidelines and the additional steps needed to increase their usefulness for appraising the trustworthiness of practice guidelines. We contrasted them with AGREE because AGREE has been widely used and cited, and we based our comments on the 2009 AGREE II standards,¹ which we did not state clearly in the Viewpoint. The 2003 AGREE² and AGREE II¹ standards are quite similar except for a new item on evaluating evidence.

The Affordable Care Act created the Patient-Centered Outcomes Research Institute (PCORI) to help patients, clinicians, and policy makers make well-informed decisions about health care. Because its funding expires in 2019, the institute has little time in which to produce timely, practice-changing results that will build public support for comparative effectiveness research. PCORI should plan its research agenda strategically, so that it addresses research questions that comparative effectiveness research could answer quickly and decisively. To date, the institute has not chosen this path. In January 2012 PCORI's first research agenda described broad research priorities rather than specific clinical questions. The institute must drive the burgeoning discipline of comparative effectiveness research forward, starting with a research project agenda that conveys a sense of urgency and strategic direction.

Chest pain is a common complaint in primary care, with coronary heart disease (CHD) being the most concerning of many potential causes. Systematic reviews on the sensitivity and specificity of symptoms and signs summarize the evidence about which of them are most useful in making a diagnosis. Previous meta-analyses are dominated by studies of patients referred to specialists. Moreover, as the analysis is typically based on study-level data, the statistical analyses in these reviews are limited while meta-analyses based on individual patient data can provide additional information. Our patient-level meta-analysis has three unique aims. First, we strive to determine the diagnostic accuracy of symptoms and signs for myocardial ischemia in primary care. Second, we investigate associations between study- or patient-level characteristics and measures of diagnostic accuracy. Third, we aim to validate existing clinical prediction rules for diagnosing myocardial ischemia in primary care. This article describes the methods of our study and six prospective studies of primary care patients with chest pain. Later articles will describe the main results.

This review describes methods used in comparative effectiveness research (CER). The aim of CER is to improve decisions that affect medical care at the levels of both policy and the individual. The key elements of CER are (a) head-to-head comparisons of active treatments, (b) study populations typical of day-to-day clinical practice, and (c) a focus on evidence to inform care tailored to the characteristics of individual patients. These requirements will stress the principal methods of CER: observational research, randomized trials, and decision analysis. Observational studies are especially vulnerable because they use data that directly reflect the decisions made in usual practice. CER will challenge researchers and policy makers to think deeply about how to extract more actionable information from the vast enterprise of the daily practice of medicine. Fortunately, the methods are largely applicable to research in the public health system, which should therefore benefit from the intense interest in CER.

The focus of comparative effectiveness research (CER) is decision making by patients, physicians, and policy makers. The key elements of CER are head-to-head comparisons of active treatments, study patients who are representative of typical practice, and the search for patient characteristics that predict differences in response to tests or treatment. The four pillars of CER are research, human and scientific capital, data infrastructure, and translation (of evidence into clinical practice). These are also among the fundamental attributes of an academic health center (AHC). This congruity of structure and purpose should mean that AHCs are well positioned to gain from conducting CER and from translating CER results into practice. This commentary discusses the fit between the missions of AHCs and the purposes of CER, using the four pillars as an organizing framework. Aside from the opportunity to do research, AHCs will see the most gains from CER if they hold themselves accountable for using the best available evidence in patient care. Conversely, practice in community settings will gain from CER only if AHCs do a much better job of teaching medical students and residents to become expert in using evidence to make decisions.