OBJECTIVES: Incorporating patients’ perspectives into drug development is increasingly recognized as crucial for understanding real-life disease impacts and treatment outcomes. However, systematic implementation in the design of clinical trials and selection of relevant clinical endpoints faces significant barriers. This study aimed to investigate current practices, barriers, and improvement opportunities regarding the implementation of patients’ perspectives into clinical trial design.

METHODS: A mixed-method approach was employed, comprising a scientific literature review with systematic search string, grey literature review, and semi-structured interviews with key stakeholders, including physicians from Belgian hospitals. This comprehensive analysis obtained an overview of reviews and guidelines on endpoint determination and selection and the integration of patient perspectives in these processes.

RESULTS: The results revealed limited guidance on the use of defined, patient-informed endpoints and varied implementation of patients’ perspectives in clinical trial design. Patient-reported outcome measures were most reported, with diverging use in different disease areas due to challenges such as lack of standardized methodologies and validated measurement tools. More research on validating patient-relevant endpoints and core outcome sets were highlighted as opportunities in clinical trial design. While there is increasing recognition of the importance of direct patient input, its incorporation in practice remains inconsistent, with limited reporting and lack of comprehensive best practices. Patient-reported experiences and patient preference data were underreported, highlighting gaps in awareness and methodological guidance. Stakeholders unanimously emphasized the value of patient perspectives in enhancing clinical trial designs and advocated for early patient involvement and multi-stakeholder collaborations.

CONCLUSIONS: Despite consensus on the benefits of integrating patient perspectives in clinical trial designs, standardized methodologies and systematic implementation are lacking. Collaborative efforts and structured approaches are essential to address the identified challenges and facilitate more effective inclusion of patients’ perspectives in clinical trial designs, thereby advancing the relevance and impact of clinical trials on patient outcomes.