Pharmacogenomic Biomarkers in US FDA-Approved Drug Labels (2000-2020)

Pharmacogenomics (PGx) is a key subset of precision medicine that relates genomic variation to individual response to pharmacotherapy. We assessed longitudinal trends in US FDA approval of new drugs labeled with PGx information. Drug labels containing PGx information were obtained from Drugs@FDA and guidelines from PharmGKB were used to compare the actionability of PGx information in drug labels across therapeutic areas. The annual proportion of new drug approvals with PGx labeling has increased by nearly threefold from 10.3% (n = 3) in 2000 to 28.2% (n = 11) in 2020. Inclusion of PGx information in drug labels has increased for all clinical areas over the last two decades but most prominently for cancer therapies, which comprise the largest proportion (75.5%) of biomarker-drug pairs for which PGx testing is required. Clinically actionable information was more frequently observed in biomarker-drug pairs associated with cancer drugs compared to those for other therapeutic areas (n = 92 (59.7%) vs. n = 62 (40.3%), p < 0.0051). These results suggest that further evidence is needed to support the clinical adoption of pharmacogenomics in non-cancer therapeutic areas.

Automated summary

Pharmacogenomics (PGx) is a key component of precision medicine that analyzes genetic variations to tailor drug therapies. This study found that the proportion of new drugs approved by the US FDA with PGx information has been increasing, especially in the field of cancer therapies. Results show that PGx information in cancer drugs is more clinically actionable compared to other therapeutic areas, indicating the need for more evidence to support the clinical adoption of pharmacogenomics in non-cancer treatments.