Temporal trends in novel drug target discovery reveal the increasing importance of human genetic data

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Abstract

Drug approvals involving novel mechanism-of-action targets currently account for about one-fifth of new FDA-approved drugs each year. Developing therapies for novel targets carries added risks, but they can significantly address areas with unmet medical needs, or current treatment limitations. The Open Targets Platform is a valuable, regularly updated, open resource for identifying and prioritising therapeutic targets, integrating diverse data sources with a user-friendly interface. However, it lacks assessment of target novelty and has minimal timestamping. In response, we implemented comprehensive timestamping across millions of biomedical data points and introduced a metric to summarise the novelty of a target in the context of disease(s) to discover novel drug targets within the Open Targets ecosystem. A retrospective analysis of novel drug target approvals since 2000 suggests that the genetic evidence for clinical progression is increasingly considered earlier in the pipeline, highlighting the benefit of assessing association evidence in the context of time.

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