Two commonly used research methods are available to evaluate alternative health interventions: efficacy and effectiveness research. Efficacy can be defined as the performance of an intervention under ideal and controlled circumstances, whereas effectiveness refers to its performance under ‘real-world’ conditions. In comparative effectiveness research (CER), the focus is especially on effectiveness studies to maximize generalizability and acceptability of the results.
CER has been defined 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” (Institute of Medicine, Washington, DC, USA, 2009).
In recent years the relevance of CER has increased due to a broadening array of alternative healthcare options and interventions while facing growing constraints on healthcare budgets. 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.
Methods that can be used in CER (1):
- Systematic review of existing research, including meta-analysis
- Decision modelling, with or without cost information
- Retrospective analysis of existing clinical or administrative data
- Prospective observational studies, including registries
- Experimental studies, including among others pragmatic clinical trials, cluster randomized clinical trials, delayed-design trials
(1) Tunis SR, Benner J, McClellan M Comparative effectiveness research: policy context, methods development and research infrastructure. Stat in Med 2010, 29:1963-1976