Building on DATAcc by DiMe’s core digital measures set for pediatric rare disease, the new initiative will help life sciences companies design regulatory-compatible endpoints that reduce trial costs and speed access to therapies for children

Boston, MA — August 12 — The Digital Medicine Society (DiMe) today announced the launch of Composite and Multimodal Endpoints for Pediatric Rare Disease, a new initiative to help life sciences companies design and deploy digital endpoints that capture the full impact of complex rare conditions on a child’s life, in trials small enough where a single measure may not adequately capture clinically meaningful change.

Only 5% of rare diseases have an approved treatment, even though they affect more than 400 million people worldwide. Pediatric rare disease trials are difficult to run because patient populations are small and geographically dispersed, and the diseases themselves affect multiple parts of a child’s health and day-to-day life at once. Traditional endpoints that measure just one factor often lack the statistical power to detect a treatment effect in such small, varied populations, which drives up the cost of drug development and keeps promising therapies from reaching children who need them.

DiMe’s new initiative addresses this by bringing together life sciences companies, patient advocacy organizations, technology developers, and regulators to build shared tools for combining multiple digital measures into a single endpoint. A composite endpoint combines several measures into one score. A multimodal endpoint draws on different types of data, such as movement, speech, and sleep, to represent a single, clinically meaningful concept. Used appropriately, these approaches can capture a fuller picture of how a disease affects a child, increasing the odds a trial can detect a real treatment effect even with a small number of participants.

“DiMe has spent years bringing together patients, regulators, researchers, technology developers, and life sciences companies to develop the measurement science needed for the next generation of clinical trials,” said Jennifer Goldsack, CEO of DiMe. “Building on the measurement frameworks we’ve developed with the field in recent years, pediatric rare disease is the right place to pioneer the next generation of digital endpoints because the need is so great. I’m deeply grateful to the partners willing to tackle that challenge with us. By demonstrating how composite and multi-modal digital endpoints can work here, we’re creating a blueprint that can strengthen clinical research across many different diseases and help more patients benefit from better therapies, faster.”

The initiative builds directly on DiMe’s core digital measures set for pediatric rare disease, launched in January 2026 with more than 20 partners under DiMe’s Digital Health Measurement Collaborative Community (DATAcc). That work defined which aspects of health matter most to children with rare disease and their families, drawing on interviews with more than 70 experts and analysis of more than 1,300 patient and caregiver quotes. This new initiative uses that foundation to answer a harder question: how to combine those measures into endpoints that speed access to therapies for children.

Over the course of the project, partners will co-develop three resources: 

• a framework for designing regulatory-compatible composite and multimodal endpoints,
• an evidence library that lets sponsors assess how ready a given endpoint is for use in their own trial,
• a regulatory brief on using real-world and patient-generated data in pediatric rare disease, to be discussed directly with the FDA,
• a translational brief that maps the decisions made throughout the project to design principles for composite and multimodal endpoints across therapeutic contexts, serving as connective tissue between this project’s methodology and its application beyond pediatric rare disease.

“Greater availability of genetic testing means we’re identifying children with rare diseases earlier in their journey, at a time when we have more precision medicines than ever before to intervene,” said Andrew P. Slugg, Senior Vice-President, Global Head of Regulatory Sciences, Alnylam. “But measuring what matters to patients has not kept pace. Patients and their families don’t experience disease only during clinic visits. They live with it every day, in all the moments between. This work is about developing composite endpoints that provide evidence regulators can trust when evaluating whether new treatments are truly making a difference.”

Founding partners in the initiative include: ABPI, Alnylam, BioMarin Pharmaceutical, The Buffalo Initiative, Clouds of Care, Cure Mito Foundation, CURE SYNGAP1, CSNK2A1 Foundation, Dutch Medicines Evaluation Board, FARA, FDA, GAT Consortium, IRDiRC, Loughborough University, Neev Kolte and Brave Ronil Foundation, NORD, PMDA, Rare Care Centre (Perth Children’s Hospital), Rare Epilepsy Network, Rare Hope, RTW Foundation, Redenlab, and Trinity College Dublin.   

DiMe is welcoming additional organizations to join the initiative for a limited period following this announcement. Organizations interested in participating, please reach out.

About the Digital Medicine Society (DiMe)

The Digital Medicine Society (DiMe) is a global non-profit supporting the organizations and individuals working tirelessly to digitize health, healthcare, and health research so that it returns to the patients and builds towards a sustainable future for our industry. DiMe delivers open-access resources, multi-stakeholder collaborations, and evidence-based frameworks to accelerate the responsible digitization of healthcare. 

Media Contact: press@dimesociety.org