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Chapter 10: Evaluating Health Care Systems for Improving Symptom Management: Call Outs
        

Evidence Based Medicine

Evidence-based medicine relies on rigorous scientific research to help clinicians and patients make decisions about diagnosis and care. It is also a basis for systematic changes to health care systems intended to improve the quality of health care services in ways that improve patient outcomes. Evidence-based medicine is distinguished, at least in part, by the use of:

  • Efficacy and effectiveness trials - typically of a single intervention, e.g. use of disease-modifying antirheumatic drugs (DMARDs).
  • Meta-analysis - a quantitative method of obtaining an overall estimate for effect of medical interventions (Hennekens and Buring, 1987), usually undertaken when the results from a single study are insufficient, e.g., because there is a very small number of patients involved or the study design is inadequate.

Clinical trials and health services research studies often fall into one of two categories, efficacy trials or effectiveness trials, which are compared and contrasted below.

Table 3.1

Characteristic Efficacy Trials Effectiveness Trials
Focus of StudyWhether treatment works in controlled setting Whether treatment works in real world setting
Internal validityHigherMay be lower
GeneralizabilityLower (ideal conditions, e.g. perfect compliance, difficult, if not impossible, to replicate)Higher, but intervention may be difficult to replicate

In effectiveness trials there may be less control over who receives a study drug or intervention; so the important consideration is whether the treatment does more good than harm to those who are offered and accept the treatment (Fletcher et al., 1988). Effectiveness trials may be further sub-divided into:

  • Those that assess the effectiveness of a single intervention delivered under real-world conditions (Simon, Wagner, Von Korff, 1995), versus
  • Evaluations of health care system innovations that may include a set of interventions delivered via changes in the organization and delivery of patient care (Von Korff et al, 1987).

Meta-analysis uses data from multiple studies that include the same intervention (e.g. Cox-2 inhibitors for rheumatic pain) and similar patient groups to derive an overall effect. These are useful, for example, in pain studies, which tend to include smaller numbers of patients.

Meta-analysis is only beginning to be applied to health care system changes that may consist of multi-faceted interventions. At present, it is unclear the extent to which methods of meta-analysis can be applied to evaluation of health care system change. Given that the optimal management of chronic symptoms is likely to require multi-component interventions that are fully integrated into the delivery of health care, development of methods for synthesizing evidence on the effectiveness of multi-faceted changes in health care systems is a critically important issue.

An Example of Evidence Based Medicine
The Cochrane Collaborative
http://www.cochranelibrary.com/enter
The Cochrane Collaborative is an international effort to provide and maintain up-to-date systematic reviews of all relevant randomized trials and seminal research based on medical interventions. Studies are critically appraised and include meta-analyses for each intervention using both published literature (MEDLINE, EMBASE, PSYCHLIT, etc.) and unpublished reports. The Cochrane Collaborative web site contains over 500 systematic reviews, including those for rheumatic conditions and their symptoms.

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