Infrastructure Requirements for Secondary Data Sources in Comparative Effectiveness Research

摘要

The growing interest in comparative effectiveness research (CER) has re-ignited the debate about the inadequacy of data from randomized controlled trials (RCTs) to address patient-centered decision making. Despite their well-known internal validity and use as the gold standard for regulatory decision making, the limitations of RCTs are widely recognized. In addition to their lack of statistical power due to inadequate sample size to address certain research hypotheses, practical and ethical considerations may preclude their viability. A case in point is the ethical dilemma in conducting an RCT to establish whether a diet high in fat content may be a risk factor for dementia, which might produce useful public health information but would not be acceptable in terms of protection of human subjects. Frequently, RCTs provide substantial information regarding the efficacy of drugs and other medical interventions, yet leave large gaps in evidence that would be relevant for medical decision making. Even when RCTs are carried out with this intent, they may not necessarily reflect "real-world" experience and, therefore may not provide sufficient evidence to guide patient-centered care.