Refining the impact of genetic evidence on clinical success.
other · Level V
Where this comes from
- Record sourced from PubMed, PMID 38632401.
- Also identified by DOI 10.1038/s41586-024-07316-0 and PMC identifier 11096124.
- Licence recorded as CC BY.
- The licence permits redistribution, so the abstract is shown in full and the full text is available from the publisher.
Abstract
The cost of drug discovery and development is driven primarily by failure<sup>1</sup>, with only about 10% of clinical programmes eventually receiving approval<sup>2-4</sup>. We previously estimated that human genetic evidence doubles the success rate from clinical development to approval<sup>5</sup>. 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.
Medical subject headings
- Clinical Trials as Topic
- Drug Approval
- Drug Discovery
- Treatment Outcome