Think Tank

August 28, 2020: Designing, Conducting, Monitoring, and Analyzing Data from Pragmatic Clinical Trials: Proceedings from a Multi-Stakeholder Think Tank Meeting (Trevor Lentz, PT, PhD, MHA; Lesley Curtis, PhD; Frank Rockhold, PhD)

Posted on by Gina Uhlenbrauck

Speakers

Trevor Lentz, PT, PhD, MHA
Assistant Professor in Orthopaedic Surgery
Duke Clinical Research Institute

Lesley Curtis, PhD
Chair and Professor, Department of Population Health Sciences
Duke University School of Medicine

Frank Rockhold, PhD, ScM, FASA, FSCT
Professor of Biostatistics and Bioinformatics
Duke Clinical Research Institute

Topic

Designing, Conducting, Monitoring, and Analyzing Data from Pragmatic Clinical Trials: Proceedings from a Multi-Stakeholder Think Tank Meeting

Keywords

Pragmatic clinical trials; Think tank; Risk-based monitoring; Data quality; Real-world data; Electronic health records

Key Points

  • Pragmatism in study design is not a binary concept: some trial elements are purely explanatory (to establish efficacy in ideal settings) and some elements are purely practical (to establish effectiveness in the real world). The study design must serve the research question.
  • Findings from the think tank discussions on best practices and actionable steps included:
    • Ask precise research questions, and select the appropriate degree of pragmatism.
    • Optimize data quality through study design.
    • Focus on primary endpoints in data capture to maximize likelihood of success.
    • Innovate on mechanisms for data capture.
    • Promote adherence to the study protocol.
    • Evolve trial operations staff to focus on data science and informatics.
    • Share learning experiences openly and widely.

Discussion Themes

There is a misconception that PCTs, because they pursue pragmatism, are less rigorous and conducted without proper oversight or adherence to a protocol. Quality by design and good clinical practice principles apply equally to PCTs.

Risk-based monitoring is a potentially dynamic system that could improve study safety and quality, and make better use of study resources.

There is great interest from regulators, sponsors, and the academic research community to move PCT methods forward. To achieve this, we need to see more examples of successful PCTs in a context of regulatory decision-making.

Read the proceedings from the think tank meeting published in Therapeutic Innovation & Regulatory Science.

Tags

#pctGR, @Collaboratory1