The Center for the Study of Drug Development, Tufts University School of Medicine, 75 Kneeland Street, Boston, 021111, MA, USA.
ZS Associates, Boston, MA, USA.
Ther Innov Regul Sci. 2020 May;54(3):598-604. doi: 10.1007/s43441-019-00092-4. Epub 2020 Jan 6.
Although a number of studies have quantitatively measured investigative site burden to administer increasingly complex protocol designs, robust scholarly research has not been performed to quantify the burden that patients face as participants in clinical trials.
This paper presents the results of a cross-sectional pilot study conducted by the Tufts Center for the Study of Drug Development and ZS Associates among nearly 600 patients via an online survey conducted between February and March 2019. Respondents rated the perceived burden of 60 commonly administered protocol procedures. The association and relationship between overall patient burden-derived from aggregating mean perceived burden ratings for individual procedures-and performance (eg, screen failure and retention rates, clinical trial cycle times) for a cross-sectional sample of 137 individual protocols was assessed. Descriptive statistics, significance tests, and univariate analyses were performed.
Strong positive, statistically significant associations were observed between a composite measure of patient burden and protocol-specific design and performance measures, most notably study visits above the tolerable mean and the study conduct duration from first patient first visit to last patient last visit.
The study results suggest a new and viable approach to optimize protocol design and improve patient engagement.
尽管许多研究已经对研究机构管理日益复杂的方案设计的负担进行了定量测量，但对于患者作为临床试验参与者所面临的负担，尚未进行强有力的学术研究来进行量化。
本文介绍了塔夫茨药物开发研究中心与 ZS 协会在 2019 年 2 月至 3 月期间通过在线调查，对近 600 名患者进行的一项横断面试点研究的结果。受访者对 60 种常见方案程序的感知负担进行了评分。评估了来自个体程序的平均感知负担评分的聚合的总体患者负担与表现（例如筛选失败和保留率，临床试验周期时间）之间的关联和关系。进行了描述性统计，显著性检验和单变量分析。
在患者负担的综合衡量标准与特定方案的设计和绩效指标之间观察到了强烈的正相关，统计学上具有显著意义，最显著的是高于可容忍平均值的研究访视次数和从第一个患者首次访视到最后一个患者最后一次访视的研究实施持续时间。
研究结果表明了一种优化方案设计和改善患者参与度的新的可行方法。
