OBJECTIVES: Patient-Reported Outcomes (PROs) are increasingly used to support the development and the evaluation of health products and services. Due to the wide variety of instruments available, identifying the most suitable Patient-Reported Outcome Measures (PROMs) to use in a specific context might be complex. There is no consensus on a standardized method to choose PROs considering the scientific context and the regulatory recommendations. We developed a patient-centric, structured, replicable and generalizable method for PROs selection and development using patient-generated data.

METHODS: A patient-centric structured approach for PROs selection was developed and tested as pilots in 3 pathologies: lung cancer (n=150), breast cancer (n=250) and diabetes (n=249).

RESULTS: The four-step methodology developed starts with a literature review of patient-centered research and clinical trials to determine PRO measures frequently used in a specific disease. Outcomes and domains and sub-domains identified are put into perspective with medical experts’ perceptions and available health guidelines. As a third step, a large direct-to-patient survey aims to rank the domains from the patient perspective and highlight potential additional domains to be assessed. Finally, a gap analysis is performed to identify the most suitable PROs to use. Testing this methodology in 3 therapeutic pathologies showed that the approach allows us to highlight differences according to patients’ profile and disease, and to identify domains that are under assessed in frequently used PRO measures.

CONCLUSIONS: The increasing importance of Health-Related Quality of Life (HR-QoL) in healthcare decision-making reinforces the need to choose relevant PRO measures. This new patient-centric approach presents the advantage of considering patients’ expectations in the choice of instruments. The methodology developed in this pilot showed promising results that would require further testing and validation in other conditions.