How DiME’s new pediatric rare disease program is speeding up clinical trials
The Digital Medicine Initiative (DiME), working with partners including the FDA, BioMarin, Northwestern Medicine, and Stanford Medicine, has launched a new set of digital clinical measures to improve pediatric rare disease care.
The set of measures released Thursday aims to provide researchers and drugmakers with standardized endpoints to assess treatment effectiveness and accelerate clinical development for rare pediatric diseases, a field that has long been challenged by small trials and inconsistent outcome measures.
These measures aim to capture data from digital tools such as wearable devices to provide a continuous understanding of patient health. By standardizing how outcomes are measured, DiME hopes to make trial data more valid and comparable across studies, potentially reducing the time it takes for new treatments to reach children.
DiME CEO Jen Goldsack noted that this approach is particularly transformative for rare diseases, where the patient population is small and every data point matters.
“Looking at the pediatric digital health space, only about 6% of investment dollars are in pediatrics or adolescents, and there have been no successful exits in pediatric digital health. As a mission-driven organization, we believe this is absolutely unacceptable,” she commented.
Goldsack noted that rare disease trials face commercial challenges—even curative therapies often fail to be commercialized because trial costs cannot be recouped from extremely small patient populations.
To address these gaps, DiME worked with its network of partners to develop a manual that outlines core areas common to pediatric rare diseases, including mobility, cognition, sleep and seizures, and provides standardized measures for use in research and clinical trials, she explained.
Goldsack added that using digital endpoints in trials can shorten phase II by about three months and reduce overall study time, making trials more feasible for small patient populations.
She emphasized that collaboration is key to this effort. Regulators, drug manufacturers, technology developers and patient advocacy groups have all contributed to help ensure these measures are practical, scientifically rigorous and consistent with FDA regulatory guidance. The team also created an implementation toolkit to guide researchers and pharmaceutical companies on how to adopt these digital measures in trials.
Goldsack explained that drugmakers and researchers can leverage DiME's framework to identify the most relevant digital measurements for specific pediatric rare diseases using open-access datasets of patient and caregiver insights. The framework provides standardized metrics, technical guidance, and customizable tools to ensure consistent data collection across studies, making it easier to design trials and compare results.
“It also standardizes the technology that’s really important — what are the common data elements? What is the measurement ontology, so the people developing these tools and creating the registries are measuring apples and apples, and we’re actually coordinating the field to move it forward?” Goldsack said.
Through this framework and toolkit, DiME aims to de-risk the pediatric rare disease market and encourage more innovation and collaboration between researchers, drugmakers and patient groups.
Photo: Good Travel, Getty Images
