Getz Kenneth, Sethuraman Venkat, Rine Jessica, Peña Yaritza, Ramanathan Sharma, Stergiopoulos Stella
1 The Center for the Study of Drug Development, Tufts University School of Medicine, Boston, MA, USA.
2 ZS Associates, Boston, MA, USA.
Ther Innov Regul Sci. 2019 Aug 19:2168479019867284. doi: 10.1177/2168479019867284.
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.
尽管已有多项研究对实施日益复杂的方案设计时调查地点的负担进行了定量测量，但尚未开展有力的学术研究来量化患者作为临床试验参与者所面临的负担。
本文介绍了塔夫茨药物开发研究中心和ZS Associates于2019年2月至3月通过在线调查对近600名患者开展的一项横断面试点研究的结果。受访者对60种常用方案程序的感知负担进行了评分。评估了总体患者负担（通过汇总各个程序的平均感知负担评分得出）与137个单独方案的横断面样本的表现（如筛选失败率和留存率、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。
在患者负担的综合测量与特定方案的设计和表现测量之间观察到了强烈的、具有统计学意义的正相关，最显著的是高于可耐受均值的研究访视以及从首例患者首次访视到末例患者末次访视的研究实施持续时间。
研究结果提示了一种优化方案设计和提高患者参与度的新的可行方法。
