Every Patient Matters: Bespoke Digital Endpoint Strategies for Rare Disease Drug Development Webinar Replay

Rare disease drug development presents unique challenges: patient populations are often minimal, clinical endpoints may not be well-established and traditional approaches to evidence generation cannot always be applied. To succeed, sponsors need flexible, carefully tailored digital endpoint strategies that can adapt to the realities of rare disease trials.

In this webinar, the featured speakers illustrate how integrated solutions can help sponsors design endpoints that are both meaningful and reliable when every datapoint counts, by:

• Highlighting how eCOA strengthens the validity of clinical scales, reduces variability and safeguards endpoint quality through structured site engagement and rater training
• Exploring the complexities of implementing imaging in rare disease populations, including special considerations for paediatric and other unique populations
• Examining the role of continuous ECG monitoring and cardiac imaging in detecting disease progression, as well as the regulatory expectations for QT assessments

Watch today to gain insights on how to select, operationalize and protect endpoints that stand up to regulatory scrutiny and support confident decision-making in rare disease drug development.