提高效率降低成本：设计一种丰富实用的程序化的在线哮喘管理项目
2014/05/08
摘要
临床试验对于制定医疗决策至关重要，然而，在当前的范例，临床试验充满了问题，包括低登记率和高成本问题。有望提高实验效率和降低成本的选择包括，使用（1）电子方案，允许电子系统远程数据捕获（EDC）直接收集数据；（2）用电子病历（EMR）识别患者和采集数据；（3）用实用的方法进行丰富的自适应强化设计。我们描述了一个无缝的、多站点随机II/III期试验设计，以评估患有哮喘的城市青少年的哮喘管理干预。患者是随机的，被要求评估4个在线的干预对象，或者控制哮喘管理程序，之后随访一年。主要疗效终点是由哮喘控制试验（ACT）进行自我报告的哮喘控制。利用比较有效性参数的方法在降低成本的现实环境中进行试验。应用升级的电子方案来进行识别患者、评估、登记和随访。患者登记在初级保健阶段就开始了。EDC做的集中数据库用来收集CRF数据及EMR数据集成。这个II/II期试验计划样本总量为500名患者，II期已完成（n=250），中期分析包括评估干预效果，识别标志和EMR数据的可行性研究作为试验CRF数据的收集。患者招募已经开始，并在进行之中。
（林江涛 审校）
ContempClinTrials.2014Mar6.pii:S1551-7144(14)00028-7.doi:10.1016/j.cct.2014.02.008. [Epub ahead of print]
Improving efficiency and reducing costs: Design of an adaptive, seamless, and enriched pragmatic efficacy trial of an online asthma management program.
Lu M1, Ownby DR2, Zoratti E3, Roblin D4, Johnson D5, Johnson CC5, Joseph CL5.
ABSTRACT
Clinical trials are critical for medical decision-making, however, under the current paradigm, clinical trials are fraught with problems including low enrollment and high cost. Promising alternatives to increase trial efficiency and reduce costs include the use of (1) electronic initiatives that permit electronic remote data capture (EDC) for direct data collection at a site (2), electronic medical records (EMR) for patient identification and data collection, and (3) adaptive, enrichment designs with pragmatic approaches. We describe the design of a seamless, multi-site randomized Phase II/III trial to evaluate an asthma management intervention in urban adolescents with asthma. Patients are randomized, asked to access four online sessions of the intervention or control asthma management program, and are then followed for one year. The primary efficacy endpoint is self-reported asthma control as measured by the Asthma Control Test (ACT). Comparative effectiveness parametric approaches are utilized to conduct the trial in a real world setting with reduced costs. Escalated electronic initiatives are implemented for patient identification, assent, enrollment and tracking. Patient enrollment takes place during primary care visits. A centralized database with EDC is used for CRF data collection with integration of EMR data. This Phase II/II trial plans to have a total sample size of 500 patients with an interim look at the completion of Phase II (n=250), The interim analyses include an assessment of the intervention effect, marker(s) identification and the feasibility study of EMR data as the trial CRF data collection. Patient enrollment has begun and is ongoing.
ContempClinTrials.2014Mar6.pii:S1551-7144(14)00028-7.doi:10.1016/j.cct.2014.02.008. [Epub ahead of print]
