Dirks Abigail, Botto Emily, Smith Zachary, Getz Kenneth
Tufts Center for the Study of Drug Development, Tufts University School of Medicine, Boston, MA, USA.
Ther Innov Regul Sci. 2025 Jun 8. doi: 10.1007/s43441-025-00805-y.
During the past five years, there has been growing interest among pharmaceutical companies in incorporating patient needs and preferences early in the protocol design process. Through individual and larger group studies, the Tufts Center for the Study of Drug Development (Tufts CSDD) has collaborated with 14 pharmaceutical companies in applying a validated approach early in the protocol design process to assess patient participation burden. Data from these assessments has been aggregated to analyze trends in participation burden between 2011 and 2022.
156 phase II and III protocols were analyzed.
Overall burden for patients to participate in phase II and III clinical trials has been rising steadily since 2011. Procedures contributing most to participation burden include patient questionnaires, lab and blood work and routine procedures conducted at each planned visit. A notable increase in the average duration of each visit per protocol was observed in large part due to the volume of procedures performed per visit. A growing proportion of procedures contributing to participation burden are those supporting supplementary, tertiary and exploration endpoints. The results of this aggregate analysis demonstrate the value of assessing patient participation burden to inform protocol design optimization.
在过去五年中，制药公司越来越有兴趣在方案设计过程的早期纳入患者需求和偏好。通过个体研究和更大规模的群体研究，塔夫茨药物开发研究中心（Tufts CSDD）与14家制药公司合作，在方案设计过程的早期应用一种经过验证的方法来评估患者参与负担。这些评估的数据已汇总，以分析2011年至2022年期间参与负担的趋势。
分析了156项II期和III期方案。
自2011年以来，患者参与II期和III期临床试验的总体负担一直在稳步上升。对参与负担贡献最大的程序包括患者问卷、实验室和血液检查以及每次计划访视时进行的常规程序。每个方案每次访视的平均持续时间显著增加，这在很大程度上是由于每次访视执行的程序数量。导致参与负担的程序中，支持补充性、三级和探索性终点的程序所占比例越来越大。这项汇总分析的结果证明了评估患者参与负担对指导方案设计优化的价值。
