Advancing Meaningful Remote Digital Endpoints for Rare Disease Clinical Trials

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.

Register for this webinar to hear the speakers discuss case studies and ongoing efforts toward utilizing digital endpoints in pediatric neuromuscular disease and sleep disturbance associated with epilepsy. Ultimately, to realize the full potential of this approach, the selected digital endpoints need to be seamlessly integrated into the wider clinical program. Considerations for successfully implementing this approach on a global scale will be covered.

Speakers

Leone Atkinson, MD, PhD, Executive Medical Director, Rare Disease and Pediatric Team, Labcorp Drug Development

Leone is a neurologist with a doctorate in Medical Genetics. With over 19 years of experience in the pharma, biotech and CRO industries, she has extensive rare disease drug development experience, with a focus on rare neurological and metabolic diseases.

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Aycan Palazoglu, Head of Clinical Trials, Aparito

Aycan has worked in pharma, in a large global CRO and in digital technology supporting Top 20 Pharma and small biotechs with clinical development all the way through to commercialization. Aycan has extensive knowledge in real-world evidence, clinical trial design, product launch and healthcare technology. Through her network of clients, she started to see the shift in the industry toward adopting new technologies to support better outcomes for patients and has gained a strong passion for digital health on which she has focused since 2018.

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Louise Kearney, MSc, PMP, Executive Director, Rare Disease and Pediatrics Team, Labcorp Drug Development

Louise has 20 years of experience in the operational aspects of clinical research, having worked across different therapeutic areas, phases and geographies. For the last 4 years, she has focused on rare disease and pediatric clinical trials at Labcorp, developing and implementing best practices for this unique cohort of studies.

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