The support of human genetic evidence for approved drug indications

Matthew R. Nelson, Hannah Tipney, Jeffery L. Painter, Judong Shen, Paola Nicoletti, Yufeng Shen, Aris Floratos, Pak Chung Sham, Mulin Jun Li, Junwen Wang, Lon R. Cardon, John C. Whittaker, Philippe Sanseau

Research output: Contribution to journalArticlepeer-review

880 Scopus citations


Over a quarter of drugs that enter clinical development fail because they are ineffective. Growing insight into genes that influence human disease may affect how drug targets and indications are selected. However, there is little guidance about how much weight should be given to genetic evidence in making these key decisions. To answer this question, we investigated how well the current archive of genetic evidence predicts drug mechanisms. We found that, among well-studied indications, the proportion of drug mechanisms with direct genetic support increases significantly across the drug development pipeline, from 2.0% at the preclinical stage to 8.2% among mechanisms for approved drugs, and varies dramatically among disease areas. We estimate that selecting genetically supported targets could double the success rate in clinical development. Therefore, using the growing wealth of human genetic data to select the best targets and indications should have a measurable impact on the successful development of new drugs.

Original languageEnglish
Pages (from-to)856-860
Number of pages5
JournalNature Genetics
Issue number8
StatePublished - 30 Aug 2015
Externally publishedYes


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