Oude Rengerink Katrien, Kalkman Shona, Collier Susan, Ciaglia Antonio, Worsley Sally D, Lightbourne Alison, Eckert Laurent, Groenwold Rolf H H, Grobbee Diederick E, Irving Elaine A
Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Universiteitsweg 100, Utrecht 3584 CG, The Netherlands.
Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Universiteitsweg 100, Utrecht 3584 CG, The Netherlands.
J Clin Epidemiol. 2017 Sep;89:173-180. doi: 10.1016/j.jclinepi.2016.12.021. Epub 2017 May 11.
This paper addresses challenges of identifying, enrolling, and retaining participants in a trial conducted within a routine care setting. All patients who are potential candidates for the treatments in routine clinical practice should be considered eligible for a pragmatic trial. To ensure generalizability, the recruited sample should have a similar distribution of the treatment effect modifiers as the target population. In practice, this can be best achieved by including-within the selected sites-all patients without further selection. If relevant heterogeneity between subgroups is expected, increasing the relative proportion of the subgroup of patients in the heterogeneous trial could be considered (oversampling) or a separate trial in this subgroup can be planned. Selection will nevertheless occur. Low enrollment and loss to follow-up can introduce selection and can jeopardize validity as well as generalizability. Pragmatic trials are conducted in clinical practice rather than in a dedicated research setting, which could reduce recruitment rates. However, if a trial poses a minimal burden to the physician and the patient and routine clinical practice is maximally adhered to, the participation rate may be high and loss to follow-up will not be a specific problem for pragmatic trials.
本文探讨了在常规护理环境中进行试验时，识别、招募和留住参与者所面临的挑战。在常规临床实践中，所有可能接受治疗的患者都应被视为符合实用试验的条件。为确保普遍性，招募的样本应具有与目标人群相似的治疗效果修饰因素分布。在实践中，最好的方法是在选定的地点纳入所有患者，无需进一步筛选。如果预期亚组之间存在相关异质性，可以考虑增加异质性试验中患者亚组的相对比例（过度抽样），或者可以计划在该亚组中进行单独试验。然而，选择仍会发生。低招募率和失访会引入选择偏倚，并可能危及有效性和普遍性。实用试验是在临床实践中进行的，而不是在专门的研究环境中，这可能会降低招募率。然而，如果试验给医生和患者带来的负担最小，并且最大程度地遵循常规临床实践，参与率可能会很高，失访对于实用试验来说也不会是一个特殊问题。
