Curriculum in CardiologyLeveraging observational registries to inform comparative effectiveness research
Section snippets
Evolution of observational CER efforts
Current observational databases, most of which are now electronic, were borne out of business process need (eg, claims databases); however, provider-led databases focusing specific diseases and/or patients have also emerged, with the earliest examples being the Duke Databank for Cardiovascular Disease established in 196911 and later the Coronary Artery Surgery Study started in 1975 to assist the National Heart, Blood, and Lung Institute to develop a program to support research investigations.12
Beyond RCTs—opportunity for observational CER
The RCT is the “criterion standard” methodology providing the highest level of evidence to inform efficacy of a particular intervention or therapy.1 Through randomization and, in most cases, treatment blinding, RCTs control for unmeasured selection and treatment biases, as well as potentially other biases, to determine the relative risk and benefits of a particular treatment. Evidence from these studies can be used for regulatory approval, to support clinical guidelines, and to inform policy
Observational data sources
Database sources for observational studies that currently exist are heterogeneous in terms of the type of patient population followed, data elements collected, geography, and funding mechanism. Table I demonstrates some of the potential sources of a variety of observation databases. Claims databases have the advantage of being large and capturing almost every interaction with the health care delivery system. However, because the data are used for claims and billing purposes, they lack detailed
Role of observational data to inform CER
To fill the evidence void, the growing availability of electronic databases allows the ability to answer questions that cannot be answered by traditional RCTs. These observational data have the potential to rapidly and effectively fill many of the gaps in therapeutic decision making. It is hoped that the insights gained from CER using these data will be leveraged to improve our understanding of therapeutic options as well as to reduce health care expenditures by identifying efficacious and
Limitations of observational CER
Comparative effectiveness research analyses are subject to 4 key biases that limit the use of observational data sources: selection, detection, performance, and attrition.39 (1) Selection bias refers to differences that may exist among comparator groups that arise from physician treatment selection, patient treatment choices, or treatment assignments due to patient characteristics, such as gender, age, income, education, race, etc. (2) Detection bias occurs when a difference occurs in the
Evidence generation and dissemination
Comparative effectiveness research is a key component in the cycle of therapeutic development and evidence generation (Figure 1).43 In this cycle, key to the success of observational CER investigations is asking the right questions. As posited by the Agency for Healthcare Research and Quality6 and the Federal Coordinating Council for Comparative Effectiveness Research,44 CER investigations should address important and specific questions where there is uncertainty for clinical decision making by
Conclusions
The ARRA 2009 has accelerated CER efforts and provided the resources to further develop and build new clinical electronic repositories for observational research. Comparative effectiveness research offers the opportunity to provide insights into the outcomes and costs of existing and even new therapeutics by leveraging observational databases to inform decision making. The recognition that evidence for many therapies used in routine clinical practice and associated outcomes is lacking endorses
References (44)
- et al.
ACC 2009 advocacy position statement: principles for comparative effectiveness research
J Am Coll Cardiol
(2009) - et al.
The price of innovation: new estimates of drug development costs
J Health Econ
(2003) - et al.
ACC/AHA key data elements and definitions for measuring the clinical management and outcomes of patients with atrial fibrillation: a report of the American College of Cardiology/American Heart Association Task Force on Clinical Data Standards (Writing Commitee to Develop Data Standards on Atrial Fibrillation)
J Am Coll Cardiol
(2004) - et al.
American College of Cardiology key data elements and definitions for measuring the clinical management and outcomes of patients with acute coronary syndromes: a report of the American College of Cardiology Task Force on Clinical Data Standards (Acute Coronary Syndromes Writing Committee) endorsed by the American Association of Cardiovascular and Pulmonary Rehabilitation, American College of Emergency Physicians, American Heart Association, Cardiac Society of Australia and New Zealand, National Heart Foundation of Australia, Society for Cardiac Angiography and Interventions, and the Taiwan Society of Cardiology
J Am Coll Cardiol
(2001) - et al.
Trends in quality of care for patients with acute myocardial infarction in the National Registry of Myocardial Infarction from 1990 to 2006
Am Heart J
(2008) - et al.
From controlled trials to clinical practice: monitoring transmyocardial revascularization use and outcomes
J Am Coll Cardiol
(2003) - et al.
Integrating quality into the cycle of therapeutic development
J Am Coll Cardiol
(2002) - et al.
The American Heart Association's principles for comparative effectiveness research. a policy statement from the American Heart Association
Circulation
(2009) - American College of Physicians. Improved availability of comparative effectiveness information: an essential feature...
- Institute of Medicine, Roundtable on Evidence-based Mediciner. July 27, 2006. Available at:...
Creating a center for evidence-based medicine
AHRQ's research efforts in comparative effectiveness: statement before the U.S. House of Representatives Committee on Ways and Means SoHJ
Government-funded comparative effectiveness research
Principles for government-supported health outcomes research on medical technologies and services
A clinical research strategy to support shared decision making
Health Aff
Report to the President and the Congress on comparative effectiveness research
Percutaneous intervention, surgery, and medical therapy: a perspective from the Duke Databank for Cardiovascular Diseases
Semin Thorac Cardiovasc Surg
Coronary artery surgery study (CASS): a randomized trial of coronary artery bypass surgery. Survival data
Circulation
The American Recovery and Reinvestment Act of 2009. H.R.1.
Scientific evidence underlying the ACC/AHA clinical practice guidelines
JAMA
Representation of elderly persons and women in published randomized trials of acute coronary syndromes
JAMA
Beyond the randomized clinical trial: the role of effectiveness studies in evaluating cardiovascular therapies
Circulation
Cited by (31)
Large Clinical Trials and Registries-Clinical Research Institutes
2018, Principles and Practice of Clinical ResearchCreating National Practice Standards Through Collaboration
2018, Quality and Safety in NeurosurgeryImproving Guideline Compliance in Australia With a National Percutaneous Coronary Intervention Outcomes Registry
2017, Heart Lung and CirculationCitation Excerpt :Identifying gaps between randomised trial evidence, guidelines and clinical practice will allow development of strategies to improve patient outcomes. The GenesisCare Cardiovascular Outcomes Registry has been designed within a Comparative Effectiveness Research (CER) structure to collect and report data from hospitals located in geographically diverse regions of Australia [1,10]. The Institute of Medicine in the United States has estimated that less than 50% of current treatments are supported by evidence and 30% of health care expenditure reflects care that is of uncertain value [10].
Ethical and regulatory considerations in the design of traumatic brain injury clinical studies
2015, Handbook of Clinical NeurologyCitation Excerpt :In addition, restrictions on inclusion and exclusion often limit the generalizability of the data from randomized controlled clinical trials (Lang, 2011). Prospective and retrospective observational studies may provide an alternative approach for determining which treatments are most effective (Shah et al., 2010; Marko and Weil, 2011; Maas et al., 2012). It may be possible to determine the causal effects of a treatment by comparing outcomes between patients with the same propensity scores, defined as “a patient's probability of being treated versus control as a function of all relevant observed covariates” (Rubin, 2010).
The Design and Rationale of the Australian Cooperative National Registry of Acute Coronary care, Guideline Adherence and Clinical Events (CONCORDANCE)
2013, Heart Lung and CirculationCitation Excerpt :Securing adequate funding and developing a business model is a singularly influential factor in initiating and sustaining a clinical registry [27]. Funding for the CONCORDANCE Registry has been provided by pharmaceutical and non-government agencies [28]. These funds have been distributed between project coordination and database management and provision to the sites with per patient payments.
Large Clinical Trials and Registries-Clinical Research Institutes
2012, Principles and Practice of Clinical Research