Patient-based evidence in mechanism-based drug repurposing: Challenges and recommendations.

Drug repurposing extends the therapeutic potential of existing drugs, with mechanism-based approaches increasingly being used to identify promising candidates. Decisions on their development and value should consider both clinical outcomes and patient-relevant benefits and burdens. Patient-based evidence (PBE), including patient-reported outcome measures (PROMs) and qualitative research, can provide this evidence. We explored stakeholder perspectives on the use of PBE through a cross-sectional survey informed by the Measurement Instrument for Determinants of Innovations. Stakeholders considered PROMs and qualitative methods to be relevant, particularly when collected early. Key barriers included limited knowledge, lack of organisational resources and uncertainty about acceptance of qualitative evidence. Survey findings and experiences from three repurposing trials informed practical guidance for integrating PBE into mechanism-based drug repurposing.