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Advancing Meaningful Remote Digital Endpoints for Rare Disease Clinical Trials

November 3, 2021
Looking to better assess treatment benefit, drug developers for rare diseases are eager to incorporate remote monitoring solutions to complement the "snapshot" data collected at clinical visits and to collect richer data on how patients function at home. Remote digital endpoints allow for objective data capture in combination with real-time patient feedback and provide a path to more meaningful data and improved patient access to much-needed treatments. Making this a reality has proven more difficult and time-consuming than hoped, and more than a few clinical development plans have been derailed by challenges with validation and interpretation of digital endpoints. It is important to work closely with rare disease patient communities to identify the disease hallmarks that are important to that community, address any gaps and identify the best way to measure and capture those hallmarks.