Precise and Agile Biomarker Approaches in Pharmaceutical R&D. How to Avoid Ending up with a Stamp Collection?

The decision-making process for any pharmaceutical R&D project is a balancing act between extrapolation and producing interpretable meaningful (translatable) data. Gathering the right information at the right time in right quantities with a constant challenge of the available resources is not trivial. Particularly for any innovative first-in-class approach, in diseases where mostly the molecular pathophysiology is poorly understood or fragmentary and where diagnosis is mostly based on medical symptoms assessments. It is imperative to choose approaches that reverse translate clinical information into the early projects and as projects mature, develop key decision biomarkers for clinical trials. Implementing the right biomarker strategy from early discovery to early clinical development is key to any successful precision medicine strategy. Through the “context of use” and “fit-for-purpose” principles we will discuss a few case studies to demonstrate how Idorsia outside of oncology is obtaining precision information on disease and treatment effects, from preclinical animal models to the clinical stage. The agile use of modern technologies allows to obtain critical information that is focused on decisions making not information collection. From rare diseases such as lysosomal storage disorders all the way to more common diseases such as Asthma.