Choosing outcomes for clinical trials: a pragmatic perspective

Area of Science:

• Critical care medicine
• Clinical trial design
• Evidence-based medicine

Background:

• The rise of evidence-based medicine has increased randomized clinical trials in critical care.
• Many trials lack significant findings due to issues with primary outcome selection.
• This has sparked debate on the optimal design of clinical trials.

Purpose of the Study:

• To synthesize recommendations for selecting primary outcomes in clinical trials.
• To ensure trials yield data that informs patient-centered decision-making.
• To guide the design of more impactful clinical research.

Main Methods:

• Review and synthesis of pragmatic recommendations for primary outcome selection.
• Integration of principles from rigorous statistical practice.
• Consideration of patient-centered decision-making needs.

Main Results:

• Six key principles for selecting primary outcomes are proposed.
• Principles include plausibility, audience relevance, patient relevance, efficiency, reliability, and use of continuous scales.
• Guidelines for selecting secondary outcomes are also presented.

Conclusions:

• Emphasizing these principles can improve the quality and impact of critical care trials.
• Preliminary evidence supporting adherence to these principles should be presented when proposing trials.
• Better outcome selection will enhance the utility of trial data for clinical practice and patient care.