OBJECTIVES: Patient-Reported Outcomes (PROs) are critical to support medical product development and guide healthcare decision-making. Nevertheless, due to the wide variety of instruments available, identifying the most suitable PROs to use in a specific context might be complex. This research aimed to identify current methods used for PROs selection and propose a more patient-centric methodology.

METHODS: This research consisted of a literature review to determine methods used for PRO identification. A more patient-centric approach for PROs selection was developed and applied as a pilot in lung cancer (n=1000 respondents, 9 countries).

RESULTS: The literature review showed that there is no standardized method to select PROs. The four-step methodology developed starts with a literature review of patient-centered research and clinical trials to determine PROs frequently used in a given therapeutic area. PROs domains identified are then assessed by a board of medical experts and a review of available health technology assessment guidelines. As a third step, a large direct-to-patient survey aims to rank the domains from the patient perspective and highlight potential additional fields. Finally, a gap analysis is performed to identify the most suitable PROs to use. Testing this methodology in lung cancer revealed that the most important domains vary according to patients’ profile and domains such as end-of-life feelings and caregivers’ lives are crucial for patients but under assessed in frequently used PROs.

CONCLUSIONS: The increasing importance of health-related quality of life in healthcare decision-making, reinforces the need to choose relevant PROs instruments. This new patient-centric approach presents the advantage of considering patients’ expectations in the choice of tools. The methodology developed in this pilot showed promising results that would require further testing and validation in other therapeutic areas.