Temporal trends in evidence supporting novel drug target discovery
Maria J. Falaguera, Ellen M. McDonagh, David Ochoa, Polina V. Rusina, Juan María Roldán‐Romero, David G. Hulcoop, Andrew R. Leach, Ian Dunham · Nature Communications · 2025
Over the past decade, about one-fifth of FDA-approved drugs each year involve novel mechanism-of-action human targets. Although riskier than modulating well-known targets, these therapies address unmet needs and strengthen sector innovation. The Open Targets Platform is a valuable open resource for identifying novel targets, integrating diverse datasets with regular updates and a user-friendly interface. To expand its capabilities, we implement comprehensive timestamping across millions of biomedical data points and introduce a target novelty metric for disease contexts, enabling discovery of novel targets within the ecosystem. We also present a retrospective analysis of novel drug target approvals over two decades, revealing a shift around 2015: supportive biomedical evidence (e.g., human genetics, literature-derived insights, differential expression, and pathway data) increasingly appears before rather than after the approval year. These findings underscore the importance of time-based evidence assessments for earlier identification of novel clinical opportunities and offer guidance for future target selection trends.