“RARE Hope looks for opportunities to maximize impact by advancing shared infrastructure and tools that can support multiple conditions at once. These core digital measures for pediatric rare disease were developed to be biologically and clinically appropriate for individual conditions while fitting within a shared, scalable framework. Shaped by diverse, multidisciplinary input, they enable consistent, objective, regulator-ready assessment across disorders — delivering tools and infrastructure that can ultimately improve the lives of more patients and families.”

– Nina Frost | President, RARE Hope

“Rare disease research is uniquely challenging, particularly within paediatric populations. Digital measurement holds significant promise to generate new insights into patient progression and treatment response, helping to build a deeper and more quantitative understanding of health and disease. Working with the DiMe Paediatric Rare Disease Digital Measurement Working Group was a valuable opportunity to connect with motivated, highly expert contributors from across disciplines and around the world. It was genuinely motivating to collaborate to tackle shared challenges while working to realise the potential of digital technologies for the benefit of children with rare diseases.”

– Sinead O’Connor | CEO, Ethomix | Adjunct Associate Professor, School of Medicine, Trinity College Dublin

“Pediatric rare diseases are uniquely challenging because each one affects only a few children, but together they have severe consequences for millions worldwide. DiMe’s core set of digital measures establishes a shared language and framework for a range of conditions. This not only lets us speed up individual clinical trials, but also change how we measure progress in small patient groups, making sure every child’s data can help improve care for others.”

–Amy Molten | Chief Medical Officer, Ladder Health | Chair, Section on Advances in Therapeutics & Technology, American Academy of Pediatrics

“This DiMe initiative delivers a comprehensive evidence-based framework for core digital measures to capture meaningful aspects of health for children with rare diseases. By adopting these measures, we hope to accelerate clinical trials, better understand treatment effects, and address patient needs more swiftly and efficiently than ever before, thus improving clinical care.”

– Jessica Nakhle, Loubna Chadli, Florence Keime-Guibert, Virginia Parks | Servier Team

“DiMe’s focus on Core Measures for Pediatric Rare Disease is invaluable for CURE SYNGAP1 and so many other patient advocacy groups who are heading into Clinical Trials for their gene-based medicines. We will be using this carefully curated resource to find effective endpoints and to help us show the FDA what is possible to measure based on our deepest concerns for our children’s health. This is the missing link for all the clinicians, advocates, and drug developers, who currently are looking for a cure but need each other’s expertise.”

– Kathryn Helde, PhD | Chief Scientific Officer, CURE SYNGAP1

“Pediatric rare disease trials continue to face significant hurdles: high costs, long timelines, and endpoints that often lack the sensitivity to capture clinically meaningful change. DiMe’s initiative to define a core set of high-value digital measures is an important step toward more efficient, patient-centric, and evidence-driven development. At Clouds of Care, we were proud to contribute our expertise in neurophysiological measures, particularly EEG. We see EEG as a powerful and underutilized tool that goes beyond seizure counts to provide objective, quantifiable insights into brain function. Integrating these signals alongside other digital measures can improve endpoint sensitivity, reduce trial burden, and ultimately increase the probability of success in clinical development. Supporting initiatives like this closely aligns with our mission: advancing smarter, data-driven approaches that make clinical research more meaningful for patients and more effective for sponsors.”

– Pieter van Mierlo | Chief Scientific Officer, Clouds of Care

“Pediatric rare disease is complex. Phenotypic variability both within and across clinical conditions poses significant challenges for researchers and the broader community. Through this collaboration, DiMe has advanced identification and consensus of key outcome domains for pediatric rare disease natural history studies and future clinical trials. I am particularly excited about the tool to customize core outcome measure sets that are aligned with patient-, caregiver-, and scientific priorities and tailored to the developmental characteristics most relevant to an individual study.”

– Aaron J. Kaat, PhD | Associate Professor and Director of Educational, Department of Medical Social Science, Northwestern University Initiatives Care

“We often hear ‘it takes a village’ to bring the right endpoints to clinical trials and the right measures to clinical care. DiMe Paediatric Rare Disease Project brought together this village – patients/families, clinicians, regulators, technology developers, biotechs, pharma – and brilliantly achieved the difficult task of collating diverse opinions and perspectives into a coherent set of actionable recommendations. This brings clarity, enables a more efficient use of resources, and unlocks insights and partnerships that would be difficult to achieve without this coordinated effort. We’re all working towards patient benefit, and this project has brought us closer towards that goal.”

– Mikesh Udani | Co-founder & CEO, Albus Health