The importance of understanding the impact of preference in clinical trials of diabetes interventions 在糖尿病干预临床试验中了解患者偏好的影响的重要性
摘要
How patients perceive their condition and associated treatment is an important consideration when making treatment decisions in self-management and progressive conditions such as diabetes. Achieving tight control of blood glucose requires that healthcare professionals (HCPs) and patients work together to address the clinical and psychosocial aspects of their illness.1, 2 Indeed the provision of care that is respectful of and responsive to individual patient preferences, needs, and values constitutes one of the core principles of evidence-based medicine.2 Patient involvement in clinical decision making increases empowerment and has been shown to improve outcomes and valuation of these outcomes.3, 4 However, clinical trials, whereby patients are randomly assigned to intervention groups, continue to provide the evidence base for marketing authorization of new interventions for diabetes. It is true that randomized clinical trials (RCTs) remain the most robust vehicle to collect the safety, efficacy and quality data required by regulatory authorities, but these designs assume clinical equipoise and remove the most important factor in how medical care is actually delivered: by choosing among alternatives. Although the process of randomization is designed to equally distribute potentially confounding prognostic variables between groups, thus maximizing the attribution of outcome to intervention, the act of randomization may unwittingly create biases in diabetes research that may threaten the validity of the findings, particularly in the modern era of comparative efficacy and relative efficacy assessment. One key bias that is often overlooked is that resulting from patient preference. Patients with strong intervention preferences may refuse randomization in clinical trials,5 particularly in diabetes, where there are effective treatments available to patients outside of a trial environment. The absence of these patients from the RCT may compromise external validity of the tr