Refining the Impact of Genetic Evidence on Clinical Success

The cost of drug discovery and development is driven primarily by failure, with just ~10% of clinical programs eventually receiving approval. We previously estimated that human genetic evidence doubles the success rate from clinical development to approval. In this study we leverage the growth in genetic evidence over the past decade to better understand the characteristics that distinguish clinical success and failure. Relative success (RS) between drug mechanisms with genetic support and those without varies significantly among therapy areas and development phases and improves with increasing confidence in the causal gene. RS is largely unaffected by genetic effect size, minor allele frequency, or year of discovery. Our findings suggest that we are far from reaching peak genetic insights to aid the discovery of targets for more effective drug therapies.