Approved research

Identification of patient selection biomarkers of liver safety

Lay summary

The research plan is to select a subset of Participants associated with liver diseases and identify genomic variants associated with these diseases. Variant selection will be by Formal Concept Analysis. These variants will then be validated in independent databases similar to those at the UK Biobank. Variants resulting from these analyses will be shared with the FDA and the public. In particular, these variants will be shared with the pharmaceutical companies which did clinical development for the PI3K drug class to identify those liver disease variants which can be patient selection biomarkers of liver safety. Conversion of these patient selection safety biomarkers into in vitro diagnostics will integrate their use with available patient selection efficacy biomarkers (companion diagnostics) and will encourage patient participation in risk-mitigated clinical trials. Patient selection safety biomarkers will integrate into FDA Project Optimus, by allowing higher dose levels at reduced expected adverse event levels. Patient selection safety biomarkers will also promote development of novel therapies by reducing the product safety burden on patients.