PRM211 - Making Pragmatic Trials Work

Abstract

Designers of pragmatic trials need to be aware of the consequences of their design choices for a study, as they balance implications on validity, precision, generalizability to routine practice, acceptability to stakeholders and operational feasibility. However, comprehensive tools that focus on the design of pragmatic trials and assist in this balancing exercise are lacking. Therefore, our objectives were 1) to identify the key design elements, their implications, and operational challenges in pragmatic trials 2) to develop a decision support tool for pragmatic trial design. Based on a literature review enhanced with stakeholder interviews we identified design choices, their implications and operational challenges for pragmatic trials and grouped them into key domains. Experts from academia, industry, patient groups, health care providers, regulatory bodies and HTA agencies were consulted. Findings were integrated into an decision support tool, in collaboration with a company specialized in serious gaming. We identified seven key domains influencing pragmatic trial design and conduct: site, participants, intervention/comparators, outcomes, data collection, safety and monitoring. We mapped the complex interplay between design choices, generalizability, validity & precision of trial results, as well as acceptability to stakeholders and operational feasibility of performing the study. A web based software tool was developed to visualize this interplay between design choices, implications and operational challenges and guide trial designers through the consequences of design choices for conduct and interpretation of their study. The decision support tool captures most relevant design choices, their implications and operational challenges encountered when designing a pragmatic trial and enables a more explicit and transparent trial design process. Our work hopes to provide trial designers with the insights and tools to make investments in more pragmatic trials return optimal value for companies, regulators, HTA bodies and patients.