Background: 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.
Methods: 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.
Results: 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.
Conclusions: The study results suggest a new and viable approach to optimize protocol design and improve patient engagement.
Keywords: patient participation burden; protocol design complexity; protocol design optimization; study volunteer burden.