Understanding the Optimal Endpoints for Neurodevelopmental Disorder Symptoms & How Biomarkers Can Be Implemented to Complement Trial Design While Meeting Regulatory Standards

Time: 8:00 am - 11:00 am
day: Pre-Conference Workshop Day


Despite having seen an increase in companies entering the clinic for rare CNS and neurodevelopmental therapeutics, there are still challenges in defining objective and sensitive biological outcome measures. While these challenges remain, the development of translational and quantitative biomarkers provide promising results to support outcome measures in drug development. With more companies approaching the clinic, there has never been a better time to explore what biomarkers can tell us about molecular target engagement, treatment and pharmacodynamic response.

This workshop will cover:

  • Exploring fluid biomarker development in rare diseases
  • Leveraging emerging technologies to identify potential PD and / or
    response biomarkers
  • Determining the importance of understanding biodistribution and target efficacy, and what should be incorporated going forwards what should be incorporated going forwards
  • Utilizing functional biomarkers such as quantitative EEG, sleep and behaviour in clinical studies
  • Exploring digital technologies supporting biomarker efforts and how they have been implemented following regulatory guidance
  • Understanding what regulatory considerations need to be made when developing functional biomarkers to be used in the clinic