Back to Agenda
Endotype Approach for Drug Development
Session Chair(s)
Dominik Hartl, MD
TA Head ATI, Biomarker Development
Novartis Pharma AG , Switzerland
The science and practice of drug development continues to evolve rapidly and it is critical to the future of sustainable drug development that regulatory science continues to evolve in tandem, particularly with the expanding understanding of the pathophysiology and genetics of disease. The guidance ‘Providing Clinical Evidence of Effectiveness for Human Drug and Biologicals Products’ (1998) recognized that as a result of advances in the understanding of pathogenesis and disease staging, clinical studies of drugs would likely focus on a more specific disease stage or clinically distinct subpopulation. This approach is validated through the recent approvals of highly targeted cancer therapies to a patient’s specific genetic mutation or biomarker (e.g. PI3Ka mutant cancers). Traditionally, clinical trials and product labelling identify patients based upon presence of a collection of signs and symptoms considered indicative of a particular disease state. This led to challenges of sub-optimal response due to inclusion of heterogenous populations and/or use of insensitive tools. Evolution of the understanding of the underlying pathophysiology of certain diseases has allowed molecular endotypes to be identified based upon characteristics such as presence of germline or somatic variants, genetic signatures, serum markers or presence of autoantibodies, which allow a more precise diagnosis of disease. In this session a short review will be given on this topic.
Have an account?