Roundtable Discussion: Statistically Significant vs Clinically Meaningful: Discussing the Shortfalls & Future Direction of Clinical Endpoints for Muscular Disorders
10:00 am - Wednesday 27th January 2027
- Examining the disconnect between statistical significance and meaningful patient benefit in muscular disorder trials to deliver more tangible improvements for patients
- Assessing emerging functional, digital and patient-centered endpoints alongside conventional measures to focus on treatment effects which are relevant to disease burden
- Defining a clinically meaningful change for patients, clinicians and regulators to collaboratively guide endpoint selection and increase confidence in development outcomes
