Future Directions for Cardiovascular Disease Comparative Effectiveness Research Report of a Workshop Sponsored by the National Heart, Lung, and Blood Institute

Stanford University School of Medicine, Stanford, California 94305-5405, USA.
Journal of the American College of Cardiology (Impact Factor: 16.5). 06/2012; 60(7):569-80. DOI: 10.1016/j.jacc.2011.12.057
Source: PubMed


Comparative effectiveness research (CER) aims to provide decision makers with the evidence needed to evaluate the benefits and harms of alternative clinical management strategies. CER has become a national priority, with considerable new research funding allocated. Cardiovascular disease is a priority area for CER. This workshop report provides an overview of CER methods, with an emphasis on practical clinical trials and observational treatment comparisons. The report also details recommendations to the National Heart, Lung, and Blood Institute for a new framework for evidence development to foster cardiovascular CER, and specific studies to address 8 clinical issues identified by the Institute of Medicine as high priorities for cardiovascular CER.

Download full-text


Available from: Kay Dickersin, Sep 18, 2015
  • Source
    [Show abstract] [Hide abstract]
    ABSTRACT: Background: Meta-analysis of registries (comparative effectiveness research) shows that primary angioplasty and fibrinolysis have equivalent real-world survival. Yet, randomized, controlled trials consistently find primary angioplasty superior. Can unequal allocation of higher-risk patients in registries have masked primary angioplasty benefit? Methods and results: First, we constructed a model to demonstrate the potential effect of allocation bias. We then analyzed published registries (55022 patients) for allocation of higher-risk patients (Killip class ≥1) to determine whether the choice of reperfusion therapy was affected by the risk level of the patient. Meta-regression was used to examine the relationship between differences in allocation of high-risk patient to primary angioplasty or fibrinolysis and mortality. Initial modeling suggested that registry outcomes are sensitive to allocation bias of high-risk patients. Across the registries, the therapy receiving excess high-risk patients had worse mortality. Unequal distribution of high-risk status accounted for most of the between-registry variance (adjusted R(2)(meta)=83.1%). Accounting for differential allocation of higher-risk patients, primary angioplasty gave 22% lower mortality (odds ratio, 0.78; 95% confidence interval, 0.64-0.97; P=0.029). We derive a formula, called the number needed to abolish, highlighting situations in which comparative effectiveness studies are particularly vulnerable to this bias. Conclusions: In ST-segment elevation myocardial infarction, clinicians' preference for management of a few high-risk patients can shift mortality substantially. Comparative effectiveness research in any disease is vulnerable to this, especially diseases with an immediately identifiable high-risk subgroup that clinicians prefer to allocate to 1 therapy. For this reason, preliminary indications from registry-based comparative effectiveness research should be definitively tested by randomized, controlled trials.
    Full-text · Article · Nov 2012 · Circulation Cardiovascular Quality and Outcomes
  • Source

    Preview · Article · Nov 2012 · Journal of the American College of Cardiology
  • Source
    [Show abstract] [Hide abstract]
    ABSTRACT: Observational methods are evolving in response to the widespread availability of data from clinical registries, electronic health records, and administrative databases. These approaches will never eliminate the need for randomized trials, but clearly have a role in evaluating the effect of therapies in unselected populations treated in routine practice. This article reviews several approaches to the analysis of observational data that are in common use, or that may have promise even though they are not yet often applied.
    Full-text · Article · Jan 2013 · Heart Failure Clinics
Show more