Co-Chairs: Douglas E. Feltner, MD; Aidan Power, MD

Precision medicines target treatment to a diseased population using a specifc genetic or biologic marker that can be assessed with a diagnostic test. Precision medicine (also known as personalized or stratified  medicine) has become a standard approach to the development of new anti-cancer therapeutics. The adoption of precision medicine approaches in neuroscience therapeutics has lagged that of oncology and other therapy areas.

In response to the increasing number of precision medicines being developed, regulatory agencies have recently drafted several guidances related to the qualification and use of biomarkers in clinical trials. These include the recent FDA guidance on drug development tools, ICH guidance on the format of genomic biomarker qualification submissions, FDA standards for clinical trial imaging endpoints, FDA guidance on clinical pharmacogenomics, and FDA guidance on in vitro companion diagnostic devices.

This workshop will seek to identify the opportunities and barriers for developing precision medicines in neuroscience indications. Alzheimer’s disease, schizophrenia, and major depressive disorder will be used as examples to highlight the issues. Both scientific and practical opportunities and challenges will be addressed, including state of the science, sponsor organizational challenges/opportunities, regulatory interactions, and payor and implementation opportunities/challenges. A second goal of this workshop will be to identify those individuals interested in advancing precision medicines for neuroscience indications and assessing whether a precision medicine workgroup should be formed to develop precision medicine expertise and knowledge within ISCTM, and to advance the goal of developing methods for the evaluation of precision medicines in neuroscience clinical trials.