DATACC BY DIME PROJECT

Defining core digital measures for
Pediatric rare disease research & care

Implementing core measures of pediatric rare diseases

Generating robust evidence for pediatric rare disease therapies faces significant barriers, such as small patient populations, data challenges, or inconsistent payor standards. As a result, therapies that appear promising in small studies tend to face prolonged evidence requirements and limited reimbursement options, forcing children to wait for treatment.

Use this guide to implement core digital measures to help reduce trial timelines, strengthen your regulatory submissions, and navigate evidence requirements.

IMPLEMENTATION GUIDE OVERVIEW

This four-part guide walks you through key areas of implementation for digital measures in clinical research and care for pediatric rare diseases. Start with the section that is most relevant to your needs.

Articulate the value of core measures for pediatric rare diseases from the perspectives of critical stakeholders.

Explore Value Framework

Plan and execute digitally-enabled clinical trials with a timeline and checklist that pinpoint clear milestones, decision gates, and practical guidance.

Build trial execution plan

Select the most appropriate regulatory pathways and interactions to accelerate time to market.

Determine Regulatory Pathways

Integrate digital measures into care workflows to enhance monitoring, engagement, and clinical decision-making.

Integrate into Care Workflows

Additional DiMe resources for strategic positioning of core measures as endpoints in disease-specific trials

Want to turn promising digital signals into decision-grade endpoints for pediatric rare disease studies? Explore additional resources that guide you through selecting, evaluating, and safely applying these measures in research and care.

Apply pediatric-specific lens

Deepen your expertise with The Playbook: Pediatric Digital Medicine. This resource details the unique requirements of working with pediatric populations, covering key topics such as human-centered design, data & ethics or planning for sustainability.

Learn More

Navigate sDHT implementation in clinical trials

To build a solid foundation for your digital measure, explore DiMe’s new How to Guide to help you answer questions such as what will we measure and why; how, where, and for whom will it be used or how will we interact with regulatory bodies.

Read the Guide

Conduct a readiness assessment

Use a structured checklist, such as those in The Playbook’s Measurement dossier, to identify gaps and define next actions before finalizing your endpoint strategy. Explore more to determine validation status, assess performance, check usability and overall experience, or ensure operational readiness.