Refining the impact of genetic evidence on clinical success.

Autor: Minikel EV; Stanley Center for Psychiatric Research, Broad Institute, Cambridge, MA, USA., Painter JL; JiveCast, Raleigh, NC, USA.; GlaxoSmithKline, Research Triangle Park, NC, USA., Dong CC; Deerfield Management Company LP, New York, NY, USA., Nelson MR; Deerfield Management Company LP, New York, NY, USA. mnelson@genscience.com.; Genscience LLC, New York, NY, USA. mnelson@genscience.com.
Jazyk: angličtina
Zdroj: Nature [Nature] 2024 May; Vol. 629 (8012), pp. 624-629. Date of Electronic Publication: 2024 Apr 17.
DOI: 10.1038/s41586-024-07316-0
Abstrakt: The cost of drug discovery and development is driven primarily by failure 1 , with only about 10% of clinical programmes eventually receiving approval 2-4 . We previously estimated that human genetic evidence doubles the success rate from clinical development to approval 5 . 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. We estimate the probability of success for drug mechanisms with genetic support is 2.6 times greater than those without. This relative success varies among therapy areas and development phases, and improves with increasing confidence in the causal gene, but is largely unaffected by genetic effect size, minor allele frequency or year of discovery. These results indicate we are far from reaching peak genetic insights to aid the discovery of targets for more effective drugs.
(© 2024. The Author(s).)
Databáze: MEDLINE
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