Model-informed drug development in rare diseases

Model-informed drug development (MIDD) approaches maximise and connect information obtained on a drug during development, allowing better characterisation of its risk: benefit profile. MIDD is especially useful for rare diseases with few patients to study. Children represent more than half of patients affected by rare diseases, most of which are genetic. In recent years, submissions for rare diseases have come to rely on modelling and simulation, and regulators now expect their inclusion in dossiers. All types of regulatory documents are impacted by MIDD, from protocols to product labels. The ability to translate complicated scientific information into comprehensive text is particularly vital in MIDD due to its complex nomenclature and multifaceted data outputs.