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Are drug targets with genetic support twice as likely to be approved? Revised estimates of the impact of genetic support for drug mechanisms on the probability of drug approval

Fig 1

Estimated effect of evidence from human genetic studies on the probability of advancing in clinical development.

A: Partitioning Pharmaprojects, OMIM, and GWAS Catalog into training data available to Nelson et al. 2015 and validation sets. We use validation set Pipeline Progression, consisting of target-indication pairs assigned a clinical phase in 2013, to determine whether gene target-indication pairs with genetic evidence were more likely to advance to the next pipeline phase from 2013-2018. Pharmaprojects target-indication pairs absent from or assigned an unknown clinical phase in the Nelson et al. dataset form the New Pipeline replication set. Pharmaprojects target-indication pairs approved prior to 2013 or with unknown phase in our dataset are not part of any replication set. B: Our estimates of the effect of genetic evidence on gene target-indication pair progression compared to values reported by Nelson et al. 2015 [3] in validation sets New Pipeline (drugs and indications > 2013, 2013 inactive drugs) New Genetic (only new genetic information > 2013) Pipeline Progression, and in the full updated dataset (Full Data). Estimates falling close to the identity line (shown in black) are consistent between the two analyses.

Fig 1

doi: https://doi.org/10.1371/journal.pgen.1008489.g001