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. 2021 Mar 4;11(3):179.
doi: 10.3390/jpm11030179.

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

Jeeyun A Kim  1 , Rachel Ceccarelli  2 , Christine Y Lu  2
Affiliations

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

Jeeyun A Kim et al. J Pers Med. .

Abstract

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.

Keywords: US Food and Drug Administration; clinical actionability; pharmacogenomics; precision medicine.

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Conflict of interest statement

The authors declare no conflict of interest.

Figures

Figure A1
Figure A1
Trends in the number of new drug approvals with PGx labeling from 2000–2020. Data shown through July of 2020.
Figure 1
Figure 1
Therapeutic areas of new FDA drug approvals with pharmacogenomics (PGx) labeling from 2000 to 2020.
Figure 2
Figure 2
Trends in annual proportion of new drug approvals with PGx labeling and finalized regulatory guidance related to pharmacogenomics from 2000 to 2020, with data through 31 July 2020. E15, E16, and E18 Guidance were developed within the International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) and endorsed by the ICH Steering Committee at Step 4 of the ICH process [16].
Figure 3
Figure 3
Trends in the number of new biomarker–drug pairs approved per year with annual proportions by cancer vs. non-cancer from 2000 to 2020. Data shown through July of 2020.
Figure 4
Figure 4
PharmGKB PGx Levels of biomarker–drug pairs for cancer and non-cancer therapies.
See this image and copyright information in PMC

References

    1. Cacabelos R., Cacabelos N., Carril J.C. The role of pharmacogenomics in adverse drug reactions. Expert Rev. Clin. Pharmacol. 2019;12:407–442. doi: 10.1080/17512433.2019.1597706. - DOI - PubMed
    1. Burt T., Dhillon S. Pharmacogenomics in early-phase clinical development. Pharmacogenomics. 2013;14:1085–1097. doi: 10.2217/pgs.13.81. - DOI - PMC - PubMed
    1. Lauschke V.M., Milani L., Ingelman-Sundberg M. Pharmacogenomic Biomarkers for Improved Drug Therapy—Recent Progress and Future Developments. AAPS J. 2018;20:4. doi: 10.1208/s12248-017-0161-x. - DOI - PubMed
    1. Giacomini K.M., Yee K.M.G.S.W., Mushiroda T., Weinshilboum R.M., Ratain M.J., Kubo T.M.M. Genome-wide association studies of drug response and toxicity: An opportunity for genome medicine. Nat. Rev. Drug Discov. 2017;16:70. doi: 10.1038/nrd.2016.234. - DOI - PMC - PubMed
    1. Kalia M. Personalized oncology: Recent advances and future challenges. Metabolism. 2013;62:S11–S14. doi: 10.1016/j.metabol.2012.08.016. - DOI - PubMed

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