Assessing Participation Burden in Clinical Trials: Introducing the Patient Friction Coefficient
Assessing Participation Burden in Clinical Trials: Introducing the Patient Friction Coefficient
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DOI：
10.1016/j.clinthera.2020.06.015
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发表时间：
2020-08-01
影响因子：
3.2
通讯作者：
Getz, Kenneth
中科院分区：
文献类型：
--
作者：
Cameron, David;Willoughby, Cara;Getz, Kenneth
方案设计的复杂性以及相关的研究志愿者负担会对患者招募和保留以及整体研发生产力产生负面影响。复杂的方案降低了潜在临床试验参与者的注册意愿并降低了保留率。尽管这样的评估将为优化试验设计和改善招募和保留绩效提供一个令人信服的机会，但很少有对方案设计特征的系统评估来确定研究志愿者的负担。为了发挥作用，评估需要以患者为中心，并重点关注影响整个临床试验参与的因素。与每个参与者在选择参与和留在临床试验时所做的当前治疗模式相比，这种评估还需要适应独特的成本价值权衡。本文提出了一种量化患者负担的新方法：临床试验患者摩擦系数（PFC）。提供了一个案例来说明 PFC 的实用性。设想 PFC 的许多应用：标准化患者负担评估以评估临床试验设计的可行性，阐明患者负担对临床试验经济性和性能的影响，并进行敏感性分析以确定最能减轻患者负担并提高临床试验性能和效率的因素。 (C) 2020 爱思唯尔公司
Protocol design complexity, and associated study volunteer burden, negatively impact patient recruitment and retention as well as overall research and development productivity. Complex protocols reduce the willingness of potential clinical trial participants to enroll and reduce retention rates. There have been few systematic assessments of protocol design characteristics to determine the burden placed on study volunteers, although such an assessment would offer a compelling opportunity to optimize trial designs and improve recruitment and retention performance. To be useful, an assessment would need to be patient-centric, and focused on the factors that influence participation throughout the clinical trial. Such an assessment would also need to accommodate the unique cost-value trade-off compared with current treatment patterns that each participant makes when choosing to participate and remain in a clinical trial. This article proposes a new methodology to quantify patient burden: the clinical trial patient friction coefficient (PFC). A case example is provided to illustrate the utility of the PFC. A number of applications for the PFC are envisioned: standardizing patient burden assessment to evaluate clinical trial design feasibility, shedding light on the impact of patient burden on clinical trial economics and performance, and conducting sensitivity analyses to identify factors that most reduce patient burden and improve the performance and efficiency of clinical trials. (C) 2020 Elsevier Inc.