By Clinical Research News Staff 

March 12, 2026 | Efforts to improve clinical trials for children with rare diseases must focus on ensuring flexibility while capturing the best data. Florence Mowlem, chief scientific officer at uMotif, joins the most recent episode of The Scope of Things to discuss how thoughtful study design—not just advanced technology—is essential for ensuring reliable data and strong patient participation.  

Mowlem specializes in electronic clinical outcome assessments (eCOA), which capture how patients feel, function, or survive using digital tools such as mobile devices, wearable sensors, and interactive voice systems. These systems are increasingly used to collect patient-reported or caregiver-reported outcomes in clinical trials. While eCOA can improve efficiency and compliance, Mowlem said technology alone cannot fix fundamental flaws in study design.  

Pediatric rare disease trials pose particular challenges. Researchers must balance regulatory requirements and site workloads with the everyday realities faced by children and their families. As the number of studies targeting rare pediatric conditions grows, sponsors must rethink how data are collected and how digital tools are deployed. 

“A one-size-fits-all approach is definitely not going to work across all trials,” says Mowlem. 

One major issue arises when sponsors attempt to measure too many outcomes. Because rare disease trials often involve small patient populations, sponsors may feel pressure to collect as much data as possible in a single study. But that strategy can backfire, leading to long clinic visits and an overwhelming number of questionnaires for families already coping with significant stress. 

Many outcome measures, especially those completed by caregivers, can be lengthy. When multiple questionnaires are stacked together, fatigue can set in, potentially affecting both compliance and data quality. Instead, Mowlem advises sponsors to begin with a clear understanding of the trial’s ultimate goals. Researchers should define the primary endpoints and focus measurement strategies around those outcomes. Attempting to capture every possible symptom or behavior often results in collecting irrelevant data that may frustrate participants and dilute the insights that truly matter. 

To learn more about data consistency and data quality concerns in pediatric rare disease trials, as well as a probiotic preventing immune system disorders in babies, GLP-1s potentially benefitting the brain, and more, listen to The Scope of Things podcast.