Patient-Centric Pragmatic Clinical Trials: Opening the DOOR.

Area of Science:

• Clinical Trial Methodology
• Biostatistics
• Medical Decision-Making

Background:

• Randomized clinical trials (RCTs) are crucial but often lack evidence for clinical practice.
• Traditional RCTs struggle with complex patient outcomes and benefit:risk assessments.
• Existing methods like major adverse cardiovascular events (MACE) have limitations in evaluating multiple events and patient-centricity.

Purpose of the Study:

• Introduce the Desirability Of Outcome Ranking (DOOR) paradigm.
• Address limitations in current clinical trial design, analysis, and reporting.
• Advance clinical trial science through comprehensive patient-centric benefit:risk evaluation.

Main Methods:

• The DOOR paradigm integrates patient-centric benefit:risk evaluation throughout the trial lifecycle.
• It addresses the cumulative nature of multiple outcomes and competing risks.
• DOOR facilitates the design, monitoring, analysis, interpretation, and reporting of clinical trials.

Main Results:

• The DOOR paradigm provides a framework for comprehensive benefit:risk evaluation.
• It enhances the ability to incorporate patient-centric outcomes and multiple events.
• The methodology is illustrated with an example and supported by freely available online tools.

Conclusions:

• The Desirability Of Outcome Ranking (DOOR) paradigm offers a novel approach to clinical trial methodology.
• DOOR improves the evaluation of treatment benefits and harms by focusing on patient-centric outcomes.
• This paradigm advances clinical trial science and supports informed medical decision-making.