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News | 25 November 2020

Webinar | Digital Endpoints: Implementing Innovative Technologies to Improve Clinical Trials for Rare Diseases

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Interested in discovering how the use of new technologies and digital endpoints can improve clinical trials for rare diseases? Watch our latest webinar, featuring expert speakers from industry and patient organisations, to learn more about the role of wearables and digital endpoints in advancing rare disease research.

Clinical trials often fail due to poor design and / or endpoint selection. These challenges are often amplified in the rare disease field, where substantial patient heterogeneity exists.

In this webinar we will explain the evolution of wearable technologies and digital endpoints, outlining their potential advantages over conventional measurements and monitoring of patients at clinical trial sites.

We will provide examples of such endpoints that have been used in rare disease, including describing the process to successful acceptance by the regulatory authorities to provide a roadmap for developing novel outcome measures and using digital endpoints to demonstrate clinical efficacy in trials.

During the 60-minute session our speakers from industry, academia and patient groups discuss the following:

  • Describe wearable technologies for continuous monitoring and digital endpoints and provide examples of how these have been implemented in rare diseases
  • Outline potential advantages and challenges of using digital endpoints
  • Explain how digital endpoints can be utilised to develop clinical trials that more accurately reflect disease progression and patient benefit
  • How to develop robust evidence to support the utility of digital endpoints
  • Lessons learned from the experience of taking digital endpoints to regulatory authorities

Expert speakers include:

You can watch the webinar recording below. We did our best to answer as many questions as possible during the session, but if you have any additional ones for us please take a look at our FAQs section or submit them via email to accelerate@healx.io

Digital Endpoints: Implementing Innovative Technologies to Improve Clinical Trials for Rare Diseases from Healx on Vimeo.

Just to note – Mindy Leffler from Casimir would like to acknowledge the invaluable contributions from the team at Nationwide Children’s in the development of the DVA. Their logo was inadvertently left off the final slide of her presentation!

About our speakers

Laurent Servais, PhD | Professor of Paediatric Neuromuscular Diseases at MDUK Oxford Neuromuscular Centre

After graduating from Louvain Medical School, Brussels, Belgium in 1999, Laurent completed a PhD in Neuroscience from Free University of Brussels, Belgium, followed by residencies in child neurology at the Free University of Brussels and Robert Debré Hospital, Paris. Laurent was subsequently appointed Head of Clinical Trials and Database Services. Most recently, he served as Head of the Institute of Myology’s I-Motion (Institute of Muscle-Oriented Translational Innovation), and Head of the Neuromuscular Centre in Liège, Belgium.

Laurent joined the MDUK Oxford Neuromuscular Center and the University of Oxford in September 2019. Laurent has been involved as principal investigator in numerous clinical trials to test treatments for Duchenne muscular dystrophy and spinal muscular atrophy (SMA). His main research expertise covers the development of innovative outcome measures, including connected devices for real-life evaluation of patients.

Mindy Leffler | President at Casimir

The frustration over her son’s clinical trial experience led Mindy to leave a 20-year career in software development to start Casimir, a research organisation dedicated to capturing the caregiver and patient perception of change during clinical trial participation. Casimir shapes an outcome measure development and data capture strategy around the experience of the patient to ensure that the lived experience of the disease becomes part of the totality of the data. Casimir’s novel outcome measure, the Neuromuscular Video Assessment, quantifies ease of movement in everyday tasks for patients with neuromuscular disease.

Daniel Lewi | Head of Business Development at Aparito and Co-founder and CEO of the CATS Foundation

Daniel is Head of Business Development at Aparito, an innovative digital health technology company that has built a platform that reduces the burden of collecting patient data for both patients and physicians. He has spent the majority of his career in business development in the financial and health sectors.

Daniel has first-hand experience of caring for a family member with a rare disease after his daughter was diagnosed with Tay-Sachs in 2011. He is also the Co-Founder and Chief Executive of the only Tay-Sachs and Sandhoff disease specific charity in the UK, the CATS Foundation. Through this role, Daniel was appointed the chairman of the European Tay-Sachs and Sandhoff Charity Consortium (ETSCC), which has brought together all the Tay-Sachs and Sandhoff charities in Europe. This has enabled the community to work together and support research on a regional, national and international level.