During the Annual SMA Conference in Washington DC this past June, the National Institutes of Health (NIH) hosted a workshop on clinical trial design in SMA for drug developers, clinical trialists, and FDA representatives.
The National Institute of Neurological Disorders and Stroke (NINDS) used the meeting as a forum to discuss the appropriate outcome measures for clinical trials, using SMA as an example population. This is critical because neurological diseases are often chronic, slowly progressive diseases with diverse populations—all of which present challenges when designing clinical trials of novel therapeutics. The three specific goals of the SO-SMART Meeting were to:
- Have an interactive discussion about the current state of knowledge in the SMA field,
- Present research data on the available outcome measures, and
- Have an interactive discussion with the patients themselves on patient-related outcomes and quality of life.
The NIH asked Cure SMA to organize and lead the panel discussion on patient and caregiver perspectives on clinical trial design. In addition, the workshop participants included scientists, patients, caregivers/parents, and representatives from Cure SMA, the SMA Foundation, the Food and Drug Administration (FDA), NINDS, and private industry.
Visit the NINDS website for a summary of this session and the entire NIH-sponsored SO-SMART Meeting.