Protecting seriously ill populations during pragmatic clinical trials

Area of Science:

• Clinical Trials Methodology
• Health Services Research
• Patient-Reported Outcomes

Background:

• Explanatory randomized clinical trials (RCTs) often have limited real-world applicability.
• Pragmatic clinical trials (PCTs) aim to assess real-world effectiveness but face challenges in minimizing patient and healthcare burden.
• Current PCT practices prioritize existing data, potentially neglecting patient-reported outcomes and safety in vulnerable groups.

Purpose of the Study:

• To advocate for a risk-based data collection strategy in PCTs.
• To address the limitations of solely relying on healthcare utilization and cost measures.
• To ensure the capture of crucial patient experience and safety data, particularly for seriously ill populations.

Main Methods:

• Review of existing methodologies for judging PCT readiness.
• Analysis of the tension between minimal data collection burden and comprehensive outcome assessment.
• Proposal of a risk-based approach for incorporating additional data collection in PCTs.

Main Results:

• Standard ranking systems for PCTs may inadvertently exclude vital patient experience measures.
• A risk-based approach can systematically identify when additional data collection is necessary for safety and patient experience.
• Examples from end-of-life care interventions (Liverpool Pathway, POLST) illustrate the application of this approach.

Conclusions:

• PCTs require a nuanced approach to data collection that goes beyond minimizing burden.
• Implementing a risk-based framework enhances the ability of PCTs to detect adverse events and understand patient experiences.
• This methodology is crucial for improving the safety and relevance of PCTs for all patient populations, especially the vulnerable.