Patients & caregivers

• Shorten the time to diagnosis. Capture symptom patterns at home to point out important patterns earlier and steer genetics/referrals sooner.
• See progress in daily life. Track symptoms, function, sleep, mobility, and routines continuously at home to understand what’s changing – not just what’s visible at clinic visits.
• Catch problems earlier. Collect evidence of deterioration or safety concerns so you can act before small issues become emergencies.
• Personalize care plans. Share objective trends that help providers tailor therapies, dosing, rehab, and support to what matters most to your child.
• Catch condition-specific pre-exacerbation signatures. Surface early deterioration to start home treatments before emergency department visits.
• Show benefit when sample size is small. Use within-child baselines and dense sampling to demonstrate meaningful change where group stats are underpowered.
• Contribute to the community. Add your experience (privately through de-identified data) to research that can speed understanding and treatments for other families.
• Capture caregiver workload. Quantify night wakings, time on care tasks, and missed work/school to reflect true burden and support service eligibility. Quantify how much help a child needs for transfers, dressing, or feeding to show real gains in independence.

Sponsors (Pharma & biotech)

• Amplify signal in small-N cohorts by leveraging high-frequency, at-home measures, reducing variance.
• Characterize trajectories in early childhood, including children who are non-verbal or have developmental limitations.
• Recruit globally with decentralized/hybrid designs suited to geographically dispersed families.
• Capture caregiver-mediated outcomes (e.g., functional assistance, sleep disruption) alongside sensor data.
• Support adaptive designs with interim digital readouts that guide dose or cohort adjustments.
• Make go/no-go decisions earlier by mapping disease trajectory and treatment response early.
• Strengthen submissions by presenting objective, high-frequency evidence to regulators.
• Accelerate time to market by using more sensitive digital outcome measures that detect change earlier.

Hospital systems & providers

• Track progression and personalize care plans using continuous trends.
• Reduce in-person visits by shifting appropriate monitoring to the home.
• Improve accuracy with objective, high-resolution symptom data rather than recall.
• Coordinate and standardize care with centralized data that follows the patient across settings and visits across departments (neurology, rehab, primary care, mental health) that serve rare disease patients.
• Discuss shared data with families to co-manage goals and therapy adjustments between visits.
• Preserve scarce specialist time by focusing in-person care on high-risk signals flagged remotely.
• Triage efficiently and expose gaps in existing hospital tech stacks through interoperable digital inputs.

Digital health technology developers

• Differentiate with pediatric-focused design that addresses comfort, size, safety, assent/consent, parental dashboards.
• Translate learnings across rare diseases where pathophysiology overlaps (e.g., mobility, sleep, seizures).
• Generate real-world data via beta pilots, proof-of-concepts, and structured user feedback.
• Improve accessibility and usability by learning directly from end users and clinical workflows.
• Validate existing DHTs/digital measures in new indications and populations, bridging/equivalence and responsiveness.
• Address a market gap where many DHTs target adults; tailor algorithms to pediatric norms and specifics of rare pediatric diseases.
• Accelerate market access for new therapies by supporting pediatric endpoints in indications lacking validated tools.

Regulators

• Facilitate pediatric development when traditional endpoints are infeasible, unethical, or insensitive.
• Support extrapolation and modeling with dense, longitudinal data that augment limited sample sizes.
• Enable new endpoints that are impractical without digital technologies (e.g., continuous mobility, sleep).
• Standardize comparisons across programs with more consistent and objective measurement.
• Strengthen longitudinal understanding of disease course and treatment durability.
• Apply consistent criteria across small trials in a given rare disease to improve comparability.

Payors/HTA bodies

HTA = Health technology assessment

• Support coverage in ultra-small populations with high-frequency evidence that reduces uncertainty.
• Monitor medically complex children at home to prevent costly admissions and ER visits.
• Quantify caregiver burden (sleep disruption, time on care tasks) to reflect the total cost of care in pediatrics.
• Predict deterioration earlier to improve outcomes and avoid downstream costs.
• Operationalize value-based contracts using objective, longitudinal outcomes from routine monitoring.
• Rely on accessible digital data for more consistent, transparent assessments and fewer “flexible” endpoints.