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Meeting on the use and development of wearables and other digital outcomes for Duchenne

On July 15, the World Duchenne Organization organized a meeting about the use of wearables, and other digital outcome measures, in Duchenne and Becker Muscular Dystrophy (DMD and BMD). The online meeting aimed to share and improve knowledge with various stakeholders about the current and future use of wearables and other digital outcomes for Duchenne muscular dystrophy. In total, 90 representatives from different backgrounds including, patient organizations, Biotech and pharma companies,  enterprises developing digital outcome measures, research groups and regulators joined the meeting and discussions. 

Monitoring health

Wearable devices such as smart watches and sensors can be used to monitor people’s health and wellbeing. In the field of DMD and BMD, wearables could play a role in daily care and management. The data that is collected through wearables and other digital technologies could potentially support drug development and reimbursement or be used to monitor treatment and interventions. 

As was mentioned in the meeting, the time for using wearables to gather digital outcomes is now. Duchenne could and should be at the forefront. We are seeing the drive towards wearables in clinical trials. As no or very few hospital visits are needed, you have a potentially wider patient pool. Better trial retention can lead to needing smaller sample groups. It might provide for more precise and real-time data, and the data capturing can be a lot more efficient. However these are still assumptions, and this is why we need developers to talk to patients.

Gather digital outcomes

Elizabeth Vroom, chair of the World Duchenne Organization stated: “Several activities regarding the use of digital outcome measures are ongoing in the Duchenne space. By collaborating and exchanging information the field can move forward faster. The World Duchenne Organization has a longstanding interest in this subject. For example, we are a partner in Trials@Home which focuses on remote decentralized clinical trials and how they can be conducted.”

Paulien Klap, Lead Product Engineer at Yumen Bionics, emphasizes the importance of creating with the people using the end product. In this case: people living with Duchenne. “You have to design with the user, not for the user. Including people living with Duchenne in the design of a wearable device ensures you are taking the right decisions, which can improve compliance and thus, data quality.”

Wearables in clinical trials

The first company has started using wearables in a Duchenne trial. Others are considering the use of wearables in future trials. This online meeting aimed to learn from experiences from the broader wearables community. During the meeting, various initiatives and updates were presented and discussed. Topics like qualification of the digital outcomes by regulatory agencies, and how to make the data derived  from wearables FAIR at source, are relevant to almost all groups who are working, or planning to work, with wearables and other digital outcomes.

Damien Eggenspieler presented his lessons learnt from the EMA qualification and deployment of wearables in clinical trials. The healthcare director at Sysnav is hopeful yet critical. “At the moment, commercially available wearables are easily tricked. In a personal encounter, my smartwatch is registering hand movement as taking steps. This is fine for my Sunday run, but we cannot afford this confusion when measuring efficacy of an intervention in clinical trials. We need to increase the level of precision and our expectation for validation for the wearable tools used in clinical trials, if we want to make them worthwhile.”

Meeting report

To summarize the meeting and its discussions, a scientific report will be created, that will be submitted to in a scientific journal. For more information on the meeting on the development of wearables and other digital outcomes for Duchenne MD, please contact Suzie-Ann Bakker at [email protected].