Perceived impact and actionability of barriers to drug repurposing: Insights from a multi-stakeholder policy survey

Résumé fourni par la source

Background: Exploring new therapeutic uses for existing medicines can generate important clinical and societal impact. Although drug repurposing (DR) provides a faster and more affordable complement to de novo drug development, barriers to successful DR persist across the DR ecosystem. A previous systematic literature review undertaken by the REMEDi4ALL Horizon Europe project identified 33 policy barriers hindering the development and access of repurposed medicines (RMs). This study aimed to prioritize these barriers and examine differences in their perceived importance across stakeholder groups. Methods: An online policy survey was conducted among impacted stakeholders. Participants rated the perceived impact and actionability of each barrier using 5-point ordinal scales. Barriers were prioritized using a composite score that weighted impact twice as much as actionability. The 20 highest-ranked barriers overall were supplemented with highly ranked barriers from individual stakeholder groups to ensure that distinctive stakeholder perspectives were retained. Differences between stakeholder groups were assessed descriptively by comparing barrier rankings, with stakeholder-specific preferences visualized using a heatmap. Results: 60 responses were included in the analysis, resulting in a final shortlist of 22 barriers. The highest-prioritized barriers concerned pricing mechanisms for off-patent RMs, market and data exclusivity, incentives to convert off-label use to on-label authorization, and the business case for repurposing off-patent medicines. Stakeholder groups differed in the relative priority assigned to individual barriers, with patient representatives showing the greatest divergence from the priorities of other groups. Conclusion: Beyond confirming previously recognized barriers, the findings reveal important differences in how stakeholder groups perceive policy barriers to DR. Prioritizing barriers can facilitate the development of targeted solutions by focusing on the most critical challenges, while recognizing differing stakeholder perspectives can support the development of shared policy recommendations. The findings provide a structured basis for the subsequent steps in policy research, including linking priority barriers with potential facilitators, existing good practices, and R&D incentive mechanisms, followed by stakeholder validation and assessment of their transferability across European contexts. Multi-stakeholder engagement will be essential to ensure that resulting policy solutions are relevant to the differing priorities across the DR ecosystem.

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