TECHNOLOGY & DATA MANAGEMENT C


linical trials have traditionally relied on ecacy outcomes measured in te clinic suc as moility tests and


clinician assessments o disease activity. ut researchers are now devising digital endoints to collect data remotely in real time oering a more comlete icture o a atients ealt.


Digital endpoints refer to clinical outcomes gathered outside of the clinical setting, usually through devices such as wearable sensors. These can range from mobility measures for patients with amyotrophic lateral sclerosis (ALS) to smartphone-based cough detection for children with asthma. Despite the high potential of digital endpoints, their uptake remains relatively low among most major pharma players, says Adam Cohen, a clinical pharmacologist researching digital endpoints at Leiden University, Netherlands. Complex regulatory barriers and inadeate fnding pose significant implementation challenges. “The opportunities for digital endpoints are enormous,” Cohen says. “Unfortunately, the barriers are almost greater.”


Measuring mobility In the central nervous system (CNS) disease space, there is a growing emphasis on using activity trackers to measure patients’ real- world mobility, says Dr Laurent Servais, a neuroscientist at the University of Oxford. Activity trackers, such as wearable sensors and pedometers, can capture the daily impact of disease better than many traditional outcomes in clinical trials. The actual development of a digital endpoint


can tae p to five years, however, and many trial sponsors are reluctant to adopt a novel approach, Servais says. Over the past decade, the uptake of activity trackers in CNS clinical trials has only increased slowly, according to GlobalData’s Clinical Trials database. Regulators have shown slightly more


willingness to accept digital endpoints, which could spur increased uptake in the coming years. Servais successfully developed the digital endpoint Stride Velocity 95th Centile (SV956), which received EMA approval as a secondary endpoint for trials


of Duchenne muscular dystrophy in 2019. SV956 uses a wearable device to measure the speed of the fastest strides taken over a period of 180 hours. Meanwhile, most trials for Duchenne muscular dystrophy currently use the outcome 6-Minute Walk Test (6MWT), which measures the distance a patient can cover in six minutes in a clinical setting. Uncontrollable factors, such as fatigue from


travelling to the clinic, could influence how a patient performs in the 6MWT. A digital endpoint, such as SV956, however, could better capture a patient’s mobility in their daily lives by measuring data over a longer period of time in a real-world setting. Digital endpoints such as 9V956 are still


mostly relegated to secondary or exploratory outcomes in trials. “There’s not enough confidence to se digital endpoints as primary outcomes in pivotal studies, but that confidence cold come with eperience, Servais says.


Overcoming regulatory barriers Most patients support digital endpoints as tools for capturing and monitoring their own health better, explains Dr Ruben van Eijk, a biostatistician at University Medical Center Utrecht, Netherlands. However, digital endpoints still face significant delays and even resistance from regulatory bodies. “Until digital endpoints are accepted by regulators, I think the uptake will always remain low,” van Eijk says. For example, van Eijk worked with a pharma


company to incorporate a device that assesses lung function in clinical trials, both remotely and in the clinic. Although European regulators supported using the device during site visits, they would not accept data recorded remotely. Regulators said they could not guarantee that the patient – and not a family member or caregiver – was blowing into the device. Digital endpoint researchers need to show


regulators that data collected with devices both inside and outside the clinic have a high correlation without systemic biases, van Eijk says. Unfortunately, regulators in the US and


Europe have key operational differences that can complicate the approval process. The EMA ass researchers to collect significant data


Outsourcing In Clinical Trials | 39


Page 1  |  Page 2  |  Page 3  |  Page 4  |  Page 5  |  Page 6  |  Page 7  |  Page 8  |  Page 9  |  Page 10  |  Page 11  |  Page 12  |  Page 13  |  Page 14  |  Page 15  |  Page 16  |  Page 17  |  Page 18  |  Page 19  |  Page 20  |  Page 21  |  Page 22  |  Page 23  |  Page 24  |  Page 25  |  Page 26  |  Page 27  |  Page 28  |  Page 29  |  Page 30  |  Page 31  |  Page 32  |  Page 33  |  Page 34  |  Page 35  |  Page 36  |  Page 37  |  Page 38  |  Page 39  |  Page 40  |  Page 41  |  Page 42  |  Page 43  |  Page 44  |  Page 45  |  Page 46  |  Page 47  |  Page 48  |  Page 49  |  Page 50  |  Page 51  |  Page 52  |  Page 53  |  Page 54  |  Page 55  |  Page 56  |  Page 57  |  Page 58  |  Page 59  |  Page 60  |  Page 61  |  Page 62  |  Page 63  |  Page 64  |  Page 65  |  Page 66  |  Page 67  |  Page 68  |  Page 69  |  Page 70  |  Page 71  |  Page 72