Temporal trends in evidence supporting novel drug target discovery
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.
Authors
- Ian Dunham (ORCID: https://orcid.org/0000-0003-2525-5598)
- David G. Hulcoop (ORCID: https://orcid.org/0000-0003-1323-1759)
- Polina Rusina (ORCID: https://orcid.org/0000-0001-7917-3120)
- Andrew R. Leach (ORCID: https://orcid.org/0000-0001-8178-0253)
- Juan María Roldán‐Romero (ORCID: https://orcid.org/0000-0002-5651-4313)
- Ellen M. McDonagh (ORCID: https://orcid.org/0000-0001-5806-6174)
- Maria J. Falaguera (ORCID: https://orcid.org/0000-0002-6635-4982)
- David Ochoa (ORCID: https://orcid.org/0000-0003-1857-278X)
Institutions
- European Bioinformatics Institute (GB)
- Wellcome Sanger Institute (GB)
- Open Targets (GB)
Publication Details
- Journal
- Nature Communications
- Published
- 2025-12-07
- DOI
- https://doi.org/10.1038/s41467-025-67180-y
- Citations
- 1
- Primary Topic
- Computational Drug Discovery Methods
- Type
- article
- Field-Weighted Citation Impact
- 1.50
Funders
- Bristol-Myers Squibb
- Pfizer
- Sanofi
- European Bioinformatics Institute
- European Molecular Biology Laboratory
- Genentech