An ad hoc committee will plan and conduct a 1.5-day public workshop to explore opportunities to accelerate early stages of drug development for nervous system disorders in the absence of animal models that reflect disease and predict efficacy. The workshop will bring together key stakeholders to discuss scientific, regulatory, and business challenges and to identify potential opportunities in this domain to accelerate therapeutic development to address unmet medical needs.

Invited presentations and discussions will be designed to:

• Explore the utility of novel approaches from genetic, stem cell, and molecular imaging research for target identification and validation (e.g., establishing predictive validity in proof-of-concept studies), and to identify biomarkers.
  
• Discuss additional evidence and future technological developments that would facilitate bringing compounds that appear to be safe into human dose finding and efficacy trials, even if an animal model of the human disease is not achievable.
  
• Discuss the regulatory landscape and what would be needed for regulatory agencies to consider these approaches.
  
• Explore the private sector environment for proceeding with drug development approaches in situations that lack animal models to predict drug efficacy.
  
• Consider ethical issues, including for exploratory trials in pediatric populations.

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