Pharmacological evaluation frameworks in autism spectrum disorder: Methodological and neurobiological considerations

Autism spectrum disorder (ASD) is a heterogeneous neurodevelopmental condition characterized by persistent deficits in social communication and interaction, alongside restricted and repetitive behaviors. Despite substantial advances in genetics, neurobiology and neuroimaging, no pharmacological treatment currently addresses the core symptoms of ASD. Existing medications primarily target associated behavioral disturbances such as irritability, aggression and hyperactivity. This narrative review synthesizes contemporary neurobiological findings, methodological challenges and evolving clinical trial frameworks relevant to the evaluation of emerging pharmacotherapies in ASD. Particular emphasis is placed on biomarker-informed stratification, rigorous trial design and developmental timing as critical determinants of therapeutic success. Advances in molecular genetics, systems neuroscience and multimodal biomarker integration highlight the need for precision medicine approaches that align pharmacological targets with underlying neurodevelopmental mechanisms. Strengthening methodological rigor while maintaining ethical and neurodiversity-affirming principles will be essential to advancing effective pharmacological interventions for ASD.