In 2021,
50 drugs were approved by the FDA’s Center for Drug Evaluation and Research, continuing a spell of improved productivity. Reflecting on the
past observation that drugs addressing targets supported by human genetic evidence are more likely to progress through clinical trials, we investigated the proportion of new approvals that can be retrospectively explained based on publicly available human genetic information by using a mix of systematic mapping and manual curation (see Supplementary information for details). We conclude that for 33 out of 50 (66%) of the FDA-approved new drugs in 2021, the genes coding for their primary assigned targets, or for proteins that are known to physically interact with these targets, have been previously associated in some way with the indication for which the drug is approved or a closely related phenotype (Fig. 1; Supplementary Fig. 1; Supplementary Table 1). Here, we discuss the value of the underlying genetic evidence in the context of examples from the approval list.
