The cost of drug discovery and development is driven primarily by failure¹, with only about 10% of clinical programmes eventually receiving approval^2,3,4. We previously estimated that human genetic evidence doubles the success rate from clinical development to approval⁵. 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.

药物发现和开发的成本主要是由失败造成的¹，只有约 10% 的临床项目最终获得批准^2,3,4。我们之前估计，人类遗传证据可以使从临床开发到批准的成功率翻倍⁵。在这项研究中，我们利用过去十年遗传证据的增长来更好地了解区分临床成功和失败的特征。我们估计有遗传支持的药物机制成功的概率是没有遗传支持的药物机制的 2.6 倍。这种相对成功因治疗领域和开发阶段而异，并随着对因果基因的信心的增加而改善，但在很大程度上不受遗传效应大小、次要等位基因频率或发现年份的影响。这些结果表明，我们远未达到遗传学洞察力的顶峰，无法帮助发现更有效的药物靶标。