An ad hoc committee will plan and conduct a two-day public workshop to explore opportunities to improve the efficiency and validity of clinical trials for nervous system disorders. The workshop will bring together key stakeholders to consider ways to advance therapeutic development for nervous system disorders by using innovative clinical trial designs, improving patient selection, engagement, and retention, and enhancing clinical monitoring to help decrease the failure rate of drugs and devices in development.

Presentations and discussions will be designed to:

• Examine assay sensitivity challenges in clinical trials for nervous system disorders, including causes of poor signal detection and type II error.
• Explore opportunities to improve clinical trial methodology for nervous system disorders, including strategies for:
  • Guiding the selection of patient populations, such as using endophenotyping to increase the yield of responders and using genomics, proteomics, and imaging biomarkers to “stage” nervous system disorders.
  • Increasing patient engagement through all phases of the clinical trial (i.e., recruitment, screening, and post-trial) and improving adherence and retention.
  • Using patient-centric technologies (e.g., wearables) and integrating such real-world, real-time data with traditional clinical data.
  • Improving monitoring during clinical trials.
  • Leveraging recent advances in diagnostics, biomarkers, and endpoints to develop more efficient clinical trials.
  • Using novel trial designs (e.g., adaptive, enrichment, and platform design studies) for nervous system disorders, including associated regulatory challenges and opportunities.

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.