Findings
SV95C allows continuous, objective assessment of ambulation in real-world settings, addressing biases and limitations of hospital-based assessments.
Wearable devices like ActiMyo® reduce patient and caregiver burden by enabling remote monitoring and decentralized clinical trials.
Digital endpoints like SV95C improve trial efficiency, potentially reducing required sample sizes and trial durations in rare diseases like DMD.
Regulatory qualification requires robust validation data, including comparisons with traditional measures, sensitivity to change, and precision.
Adoption of digital endpoints is dependent on stakeholder collaboration, patient engagement, and alignment with regulatory requirements.

Recommendations
Collaborate with regulatory bodies (e.g., EMA, FDA) early to align expectations for validation and qualification processes.
Focus on Patient-Centric Design: Develop wearable devices and endpoints with input from patients and caregivers to ensure usability and relevance to daily life.
Establish Robust Validation Protocols: Generate comprehensive data on precision, reliability, and sensitivity to change, including anchor-based approaches.
Provide training for patients, caregivers, and clinicians to enhance compliance and minimize missing data during trials.
Leverage Multi-Stakeholder Collaboration: Encourage partnerships among technology developers, drug developers, and patient groups to build normative datasets and refine measures.

Regulatory Considerations
Follow frameworks like the EMA qualification opinion process and FDA Drug Development Tools COA Qualification Program for validation.
Ensure validation studies demonstrate precision, reliability, and sensitivity to clinical changes, with comparisons to gold-standard assessments.
Use approaches that relate digital endpoint changes (e.g., SV95C) to meaningful clinical outcomes like loss of ambulation or other qualified measures.
Expand validation to include younger and nonambulant patients, ensuring endpoints are applicable across a broad spectrum of disease severity.
Adhere to Good Clinical Practice (GCP) and data protection regulations to ensure patient safety and trust.