A planning committee of the National Academies of Sciences, Engineering, and Medicine will organize and conduct a 1.5-day public workshop that brings together experts and key stakeholders from academia, industry, government, philanthropic foundations, and disease-focused non-profit organizations to discuss new genetic and neuroscience technologies and explore how they can be used to elucidate disease mechanisms and to advance the development of biomarkers and targeted therapies for people with neurological and psychiatric disorders.
Invited presentations and discussions will be designed to:

• Explore the critical need for ancestral diversity and inclusion of individuals with severe or less common disorders--in genetics and the biological specimens needed to follow up on genetics--to advance both scientific analyses and global health equity/precision medicine.

• Examine the use of genetics and other technologies to facilitate identification of genetic variation, understand the effects of both common and rare variants on disease relevant function, and gain insights into disease mechanisms and molecular pathways in order to identify biomarkers that enable patient stratification to advance therapeutic development.
  • Consider how these steps will benefit from advanced computational approaches and “big data” produced by new technologies ranging from the molecular to neural systems-level, to human phenotyping.
  • Discuss the challenges associated with identification and interpretation of common variant function.

• Highlight lessons learned from recent advances in disorders associated with rare, penetrant genetic variants, and explore how resulting lessons can be applied to more common neuropsychiatric disorders.

• Explore challenges and promising approaches to nominating and validating stratification, disease progression, and treatment biomarkers.

• Explore challenges of designing innovative clinical trials that are based on deep mechanistic understanding of diseases and coupled with target engagement strategies in patients.

• Discuss a conceptual structure and opportunities to enable advanced technologies and computational approaches to be used more broadly, effectively, and rationally for new disorders, including considering data sharing and stakeholder engagement.

The planning committee will develop the agenda for the workshop, select and invite speakers and discussants, and moderate the discussions. A proceedings of the presentations and discussions at the workshop will be prepared by a designated rapporteur in accordance with institutional guidelines.