How DiME’s New Pediatric Rare Disease Project Could Make Clinical Trials Faster

The Digital Medicine Society (DiME) has launched a new set of digital clinical measures to accelerate the development of pediatric rare disease treatments in collaboration with partners including the FDA, BioMarin, Northwestern Medicine and Stanford Medicine.

The set of measures, released Thursday, aims to give researchers and drugmakers standardized endpoints to evaluate treatment effectiveness and speed clinical development in pediatric rare diseases, an area long challenged by small trials and inconsistent approaches to measuring outcomes.

The measures are designed to capture data from digital tools like wearable devices to provide continuous, objective insights into a patient’s health. By standardizing how outcomes are measured, DiME hopes to make trial data more efficient and comparable across studies, potentially reducing the time it takes for new therapies to reach children. 

This approach could be particularly transformative in rare diseases, where patient populations are small and every data point counts, noted DiME CEO Jen Goldsack.

“Looking at the pediatric digital medicine space, something like only 6% of investment dollars are going towards anything for pediatrics or adolescents, and there have been no successful exits in pediatric digital health. As a mission-driven organization, we just thought that was absolutely unacceptable,” she remarked.

Goldsack pointed out that rare disease trials face commercial challenges — even curative therapies often fail to be commercialized because the cost of trials cannot be recouped from tiny patient populations.

To address these gaps, DiME collaborated with its network of partners to develop a playbook outlining core domains common to pediatric rare diseases — including mobility, cognition, sleep, and seizures — and provided standardized measures for use in research and clinical trials, she explained.

Using digital endpoints in trials can shorten phase II timelines by about 3 months and reduce total study timelines, which makes trials more feasible for small patient populations, Goldsack added.

She emphasized that collaboration was key in this effort. Regulators, drugmakers, tech developers and patient advocacy groups all contributed, helping make sure the measures are practical, scientifically rigorous and aligned with the FDA’s regulatory guidance. The team also created an implementation toolkit to guide researchers and pharmaceutical companies on how to adopt these digital measures in trials.

Drugmakers and researchers can use DiME’s framework to identify the most relevant digital measures for a given pediatric rare disease, drawing on an open-access dataset of patient and caregiver insights, Goldsack explained. The framework provides standardized metrics, technical guidance and customizable tools that ensure data is collected consistently across studies, making it easier to design trials and compare results. 

“It also does the really important technical standardization — what are the common data elements? What is the measurement ontology, so that the folks developing these tools and creating registries are measuring apples and apples, and we’re actually really harmonizing the field to drive it forward?” Goldsack stated.

With this framework and toolkit, DiME aims to de-risk the pediatric rare disease market, encouraging more innovation and collaboration among researchers, drugmakers and patient groups.

Photo: The Good Brigade, Getty Images