Therapeutic Development in the Absence of Predictive Animal Models of Nervous System Disorders: Proceedings of a Workshop


Note: Proceedings contain the opinion of the presenters, but do NOT reflect the conclusions of the Health and Medicine Division or the National Academies. Learn more about the differences between Reports and Proceedings.

Despite the high prevalence and burden of nervous system disorders, development of new therapeutics lags behind other disease areas. Gaps in understanding the underlying pathophysiology, a dearth of biomarkers, and limitations in the capacity of animal models to predict drug efficacy for human brain disorders have contributed to a high rate of late stage failures in drug development and decreased investment in neuroscience research programs at pharmaceutical companies. On September 12-13, 2016, the National Academies of Sciences, Engineering, and Medicine’s Forum on Neuroscience and Nervous System Disorders hosted a public workshop to explore opportunities to accelerate drug development absent predictive animal models. Experts from academia, government, industry, and the non-profit sectors discussed novel approaches to target validation and biomarker development, such as stem cell technologies and human brain organoids, and the evidence needed to bring compounds that appear to be safe into human efficacy trials. The workshop further explored important ethical, regulatory, and business considerations associated with therapeutic development when an animal model of the human disease is not achievable.