Quantitative systems pharmacology (QSP) approaches integrate mathematical modelling and experimental data to examine the mechanistic links between molecular mechanisms of drugs, disease biology, and clinical outcomes. Introduced in 2011, the QSP discipline has significantly impacted pharmaceutical development, particularly in oncology and inflammation. QSP models have supported >150 FDA submissions in the last decade. However, QSP’s application in neurodegenerative diseases is still in its infancy and has been identified as a priority area (a recent survey conducted across 50 major pharmaceutical industries).