Despite advances in understanding and treatment of autoimmune hepatitis, important unmet clinical needs remain in both adult and paediatric patient populations. The evolving research landscape and assembly of large patient cohorts are creating unique opportunities to translate science into new therapies and care pathways, with the potential to substantially improve the lives of patients with autoimmune hepatitis. However, these areas of unmet need represent real challenges that need to be addressed if this vision is to be realised. In this Viewpoint, we outline the challenges in adult autoimmune hepatitis, particularly in relation to disease-modifying therapy, and trial design and delivery. Paediatric autoimmune hepatitis presents its own set of challenging problems.
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