Imagine a world where patients don’t have to leave their homes to participate in a clinical trial. Instead of burdensome trips to the clinic and time spent away from the family, patients can be monitored for disease progression and treatment response in the comfort of their home.

The concept of combining new medicines with breakthrough technology is becoming a reality -- scientists are finding new, innovative ways to collect clinical trial data in real-time that will allow regulators to make more accurate, informed decisions.

Just look at how scientists are using technology to collect data in real-time is the progress in Duchenne Muscular Dystrophy (DMD). With advances in technology, scientists have the opportunity to more accurately and precisely measure a patient’s ambulation, or the ability to walk without any assistance, during normal daily living, without needing to be in the clinic.

DMD is a rare degenerative neuromuscular genetic disorder that causes severe muscle deterioration, typically diagnosed in boys. With this rare disease, the simple tasks of walking or eating become difficult, and in the later stages, heart and breathing muscles begin to fail.

Paul Strijbos, Neurotech Innovation Leader, Product Development at Roche is one of many scientists using breakthrough technology to reduce patient burden and more accurately collect data for people living with this rare disease. This novel technology is “a real breakthrough in terms of the way we work with patients and is changing the way scientists gather accurate data in the real world.”

  • DMD is caused by a genetic mutation that interferes with the creation of a protein (dystrophin) which is essential for keeping muscle cells intact. Without dystrophin, day-to-day activities cause excessive damage to muscle cells.

  • While there is no cure for DMD, physical therapy, medications, and breakthrough technology can help control symptoms and improve quality of life.

People with DMD often are required to complete a standard “six-minute walk” test to measure their ambulation. This test is important to measure disease progression but can be extremely burdensome. The “six-minute walk” test requires a trip to the clinic where patients must complete a strenuous activity that is not always reflective of how they really move in their day-to-day home activities.

Scientists were faced with a question: is there a way for people with DMD to skip the test? With such a burdensome task, there must be an opportunity to improve this process for patients. So, they got to work and came up with a novel solution.

Stride Velocity 95th Centile (SV95C) is a first-of-its-kind digital endpoint that is captured by wearable technology that is worn on the ankle. This novel endpoint reflects an accurate and precise real-world measure of how well a patient can ambulate.

Digital endpoints are captured by a sensor, typically outside of the clinic during activities of daily living to capture clinically meaningful motion data.

SV95C was recentlyby theas a digital endpoint. This means that drug developers and clinicians alike can confidently use SV95C to monitor the ability of people with DMD remotely and determine the benefit of potential treatments. It also means that regulators will consider these data as evidence of drug benefits when they come to review new drug applications.

“With breakthrough technology developed by our collaboration partners, people with DMD have the opportunity to wear this technology at home to uniquely measure patient ambulation during normal daily living activities,” said Paul.

SV95C has been a collaborative effort across industry partners. “We believe success is a collective effort. The mix of challenge, trust, rigor, and collaborative mindset helped us go to the next level and to deliver the value of this technology to more people with DMD,” said Damien Eggenspieler, Healthcare program director at Sysnav, the partner that developed this wearable technology.

Another advantage of using the new SV95C endpoint is that drug developers will be able to conduct shorter clinical trials with a smaller number of study participants. This means that we can deliver care faster and help more patients at scale. “Conducting trials in a shorter, more efficient and less burdensome by using wearable technology, identifying early patients who respond the best to treatment is important for pharma, regulators, physicians, but even more for people with DMD,” said Laurent Servais, MD, PhD, Professor of Paediatric Neuromuscular Diseases, MDUK Oxford Neuromuscular Center, a partner in the SV95C project.

This is another example of how scientists are combining technology with novel therapies to generate real-world data to help make more accurate, informed decisions in drug development. SV95C has been received with much interest by various stakeholders in the DMD community, including patient organisations, clinicians, and Health Technology Assessors who decide on drug pricing.

Data have been submitted to a number of peer-reviewed articles, including, showing the potential this technology has for patients across different disease areas and scientists to generate more relevant and meaningful real-world data.

Paul Strijbos, Neurotech Innovation Leader, Product Development at Roche, explains more about the collaborative effort and potential of this technology

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