Accelerating Rare Disease Drug Development with Wearable Digital Health Technologies

Overview

More than 90% of rare diseases do not have any treatment, representing a substantial unmet need for patients. The development of new therapies is especially challenging given the poor understanding of rare diseases in general and the lack of established endpoints for clinical trials.

Sensor-based digital health technologies (DHTs), such as low-burden wearable devices, offer new opportunities to advance rare disease research by providing patient-centric, objective measures on how patients function in the real world.

During this 60-minute webinar, experts from ActiGraph, Bellerophon Therapeutics,  IQVIA, and Stanford University will discuss these challenges and opportunities, including a case study on how digital measures of physical activity are being used as a primary endpoint in a pivotal trial, enabling the reduction of the sample size by a factor of two.

Learning Objectives

• Identify unmet needs and challenges of developing treatment for rare disease
• Discover opportunities for wearable and sensor-based DHTs
• Hear a case study about how Bellerophon Therapeutics is using digital measures of physical activity as a primary endpoint in a pivotal trial