Premarket assessment of molecular alterations in drug targets: a case study of 2020 drug approvals: Supplementary data

Aim: Molecular alterations in drug targets may result in differential drug activity. Therefore, the authors

aimed to characterize how molecular alterations in drug targets were assessed during drug development.

Materials & methods: The authors analyzed nonclinical and clinical study reports submitted to the US FDA

for novel drugs approved in 2020 to determine if in vitro studies, animal models or clinical studies assessed

molecular alterations in the drug target. Results & conclusion: Assessment of the impact of molecular

alterations in drug targets on drug activity varies considerably depending on the type of assessment and

therapeutic area. Premarket assessment of drug target molecular alterations is common in the oncology

setting, less frequent in the genetic disease setting and rare for other diseases.