These open-access resources will accelerate the development of life-saving therapies, improve prioritization in drug pipelines, and reduce development costs
BOSTON, Jan. 29, 2026 /PRNewswire/ — The Digital Health Measurement Collaborative Community (DATAcc) by the Digital Medicine Society (DiMe) today announced the launch of the first core set of digital clinical measures for pediatric rare diseases, a toolkit created to address the stark reality that 95% of rare diseases have no disease modifying treatment or cure, leaving most children with rare and ultra-rare conditions without disease-modifying therapies and their families without hope.
While advances in genomics are enabling more timely and accurate diagnoses for children with rare diseases, drug development is not keeping pace. Because each condition affects so few children, traditional trials are often long, costly, and burdensome for families, frequently requiring many global sites to enroll only a handful of patients. The result is an evidence model that struggles to produce data that is both meaningful for children and economically viable for sponsors, leaving even promising breakthroughs to languish on the shelf instead of being fully developed to reach the children who need them.
Higher-resolution measures that support decentralized trial designs offer a path forward. By reducing sample size requirements, shortening timelines, lowering development costs, and decreasing burden on families, these approaches make pediatric rare disease research more feasible and create a more realistic path to bringing new therapies to children. DiMe’s new core digital measures set is designed to enable this shift by standardizing how meaningful health data can be captured across rare pediatric conditions.
“Children with rare diseases are dying or living a poor quality of life, while we continue to rely on trial designs that are scientifically insufficient, often slow and unsustainable,” said Lucy Cesnakova, Program Lead at DiMe. “We now have the tools and the digital measurement capabilities to capture meaningful health data – what we have lacked is a coordinated approach to translate those capabilities into a viable framework for disease research and drug development. DiMe’s core measures close that gap and make pediatric rare diseases a market that actually works for the children and families who need it most.”
The global market for rare disease therapies is expected to exceed $490 billion by 2033, driven by precision medicine advances and supportive policy environments. Research previously conducted through DATAcc by DiMe shows the financial and operational benefits of using digital endpoints in clinical trials, including shorter development timelines and reduced risk. DiMe’s new core measures set make digital clinical capabilities more accessible and support their use at scale across the rare pediatric disease community.
“Families affected by rare diseases know that time is not on their side. Every delay in research translates to lives lost and childhoods stolen,” said Nasha Fitter, Co-Founder, Chief Business Officer, Citizen Health. “This core measures set represents a fundamental shift in how we approach pediatric rare disease research. By standardizing digital measurement tools across conditions, we can finally build the evidence base these children deserve while making trials faster, more accessible, and economically viable. At Citizen Health, we’ve seen firsthand how patient-driven data can unlock breakthroughs, and we are thrilled to be a part of this effort to give families a way to contribute meaningfully to research while ensuring their children’s experiences are captured with scientific rigor.”
These new, open-access resources reflect input from the full pediatric rare disease ecosystem, including pharmaceutical and technology developers, children’s hospitals, global research networks, patient advocacy organizations, and the families of children living with rare conditions. Together, these partners created:
- A Conceptual model defining meaningful aspects of health, and for identifying high-frequency themes meaningful to patients
- A defined core set of digital clinical measures, which can be customized to the respective needs of a population or an individual
- A patient & caregiver perspective dataset with over 1,300 quotes
- A catalogue of measurement ontologies
- An implementation toolkit designed to shape operational best practices for deploying digital measures across research and care.
Now publicly available as open-access resources, these tools are the first step in ongoing efforts through DATAcc by DiMe to work with partners across the ecosystem to further develop and apply digital measures that can unlock new therapies for children with rare diseases.
The full core measures set and implementation resources are available here.
DATAcc by DiMe convened partners including Citizen Health (Impact Sponsor), ActivInsights, Albus Health, Alexion, American Academy of Pediatrics, BioMarin, Boston Children’s Hospital, Clouds of Care, CURE SYNGAP1, Dyne Therapeutics, Friedreich’s Ataxia Research Alliance, FDA, FOXG1 Research Foundation, Stanley Manne Children’s Research Institute, International Rare Diseases Research Consortium, Medicine Evaluation Board, Neev Kolte & Brave Ronil Foundation, National Organization for Rare Disorders, Northwestern Medicine School of Medicine Feinberg School of Medicine, Rare Care Centre, Perth Children’s Hospital, RARE Hope, Rett Syndrome Research Trust, RTW Foundation, Servier, Stanford Medicine School of Medicine, Department of Pediatrics, Strados Labs, Sysnav Healthcare, Trinity College Dublin, and Vivosense.
This work builds on DATAcc by DiMe’s broader efforts to optimize digital clinical measures across therapeutic areas for medical product development and patient care, and comes at a pivotal moment for rare disease innovation. Regulatory agencies are advancing supportive infrastructure, including the FDA’s Rare Disease Innovation Hub and the NIH’s Collaborative Data Networks, signaling growing momentum for modern, data-driven approaches to evidence generation.
About the Digital Medicine Society (DiMe)
The Digital Medicine Society (DiMe) is a global nonprofit organization dedicated to advancing the safe, effective, equitable, and ethical use of digital technologies to redefine healthcare and improve lives. DiMe delivers open-access resources, multi-stakeholder collaborations, and evidence-based frameworks to accelerate the responsible digitization of healthcare. Learn more at www.dimesociety.org.
About DATAcc by DiMe
The Digital Health Measurement Collaborative Community (DATAcc) by the Digital Medicine Society (DiMe) is a collaborative community with the FDA’s Center for Devices and Radiological Health. DATAcc provides a forum for collaboration where partners and experts from across the digital health field work to advance the use of digital health measures in research and care to improve lives.
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SOURCE Digital Medicine Society (DiMe)
