Accelerating Rare Disease Drug Development With Wearable Digital Health Technologies

A vast majority, exceeding 90%, of rare diseases currently lack any treatment options, underscoring a significant and urgent unmet need for patients. The intricate challenge in developing new therapies stems from the limited understanding of rare diseases as a whole and the absence of established endpoints for clinical trials.

In this hour-long webinar, the following experts from ActiGraph, Bellerophon Therapeutics, and IQVIA come together to explore the obstacles and potential breakthroughs in rare disease research:

• Christine Guo, PhD, Chief Scientific Officer, ActiGraph
• Sylvain Zorman, PhD, Director of Digital Health Science, ActiGraph
• Peter Fernandes, Chief Executive Officer, Bellerophon Therapeutics
• Salma Ajraoui, PhD, Digital Practice Lead, Patient Centered Solutions, IQVIA

As sensor-based digital health technologies (DHTs), particularly low-burden wearable devices, emerge as promising tools to propel this research forward, dive into the following learning objectives of this presentation:

1. Identify the unmet needs and challenges inherent in developing treatments for rare diseases.
2. Explore opportunities presented by wearable and sensor-based DHTs in the context of rare disease research.
3. Delve into a compelling case study from Bellerophon Therapeutics, illustrating how digital measures of physical activity serve as a primary endpoint in a pivotal trial. This innovative approach results in a remarkable reduction of the sample size by a factor of two, exemplifying the transformative potential of digital health technologies in rare disease trials.