ArticleScientific reports2024
Evidentiary basis of the first regulatory qualification of a digital primary efficacy endpoint.
Article in Scientific reports, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers, 2 of them syntheses that pooled it.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
17 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Basket trials in rare diseases: a systematic review of current practices, methodological challenges, and future directions.Orphanet journal of rare diseases · 2025Pooled it
- A Systematic Review of Wearable Sensors in Rett Syndrome-What Physiological Markers Are Informative for Monitoring Disease States?Sensors (Basel, Switzerland) · 2025Pooled it
- Powering Neurology Drug Development Trials to Reduce False Negative Results.Clinical and translational science · 2026Review
- Advancing Neurorehabilitation and Recovery Through Human Movement Quantification via Wearable Sensing.Neurorehabilitation and neural repair · 2026Article
- IMU-based workspace area as a promising complementary tool to assess upper limb function in Neuromuscular diseases: A one-year follow-up.Journal of neuromuscular diseases · 2026Article
- Report of the multistakeholder drug development round table meeting of the World Duchenne Organization focusing on challenges for clinical development of therapies.Journal of neuromuscular diseases · 2026Article
- Artificial Intelligence in Drug Discovery and Development: Raising Quality per Decision.Pharmacopsychiatry · 2026Review
- A framework of digital biomarkers for neurodegenerative diseases.Nature reviews bioengineering · 2026Article
- Real-world walking speed as a digital biomarker and outcome measure for clinical trials-a systematic review, regulatory status and future directions.Frontiers in digital health · 2026Review
- Lumbar Acceleration Gait Estimation: "Step-by-Step" Algorithm Updates and Improvements.Journal of medical Internet research · 2025Article
- The Integrated Approach in Patients with Spinal Muscular Atrophy in the Era of Early Diagnosis, Etiopathogenic Therapies and Multidisciplinary Standards of Care and Rehabilitation Interventions Leads to New Phenotypes.Life (Basel, Switzerland) · 2025Article
- Test-Retest Reliability of Motor Function and Myometry Outcomes From the Vamorolone Trials in Duchenne Muscular Dystrophy.Neurology. Genetics · 2025Article
- Precision psychiatry roadmap: towards a biology-informed framework for mental disorders.Molecular psychiatry · 2025Review
- Long-term, age-associated activity quantification in the DE50-MD dog model of Duchenne muscular dystrophy.Disease models & mechanisms · 2025Article
- Wearable sensors in paediatric neurology.Developmental medicine and child neurology · 2025Review
- Recent Advances in Diagnosis, Management, Treatment, and Prevention of Neuropathies in Cancer Patients.Current neurology and neuroscience reports · 2025Review
- Harnessing Fast Fourier Transform for Rapid Community Travel Distance and Step Estimation in Children with Duchenne Muscular Dystrophy.Sensors (Basel, Switzerland) · 2025Article
Corrections and comments
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Authors and funding
25 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Stride velocity 95th centile (SV95C) is a wearable-derived endpoint representing the 5% fastest strides taken during everyday living. In July 2023, SV95C received European Medicines Agency (EMA) qualification for use as a primary endpoint in trials of patients with Duchenne muscular dystrophy (DMD) aged ≥ 4 years-becoming the first digital endpoint to receive such qualification. We present the data supporting this qualification, providing insights into the evidentiary basis of qualification as a digital clinical outcome assessment. Clinical trials, natural history studies, and patient surveys (ages 5 - 14 years) showed that SV95C is accurate, valid, reliable, sensitive, and clinically meaningful. SV95C significantly correlated with traditional DMD assessments, increased rapidly after steroid initiation (0.090 m/s 3 months post-treatment), and declined steadily in patients on stable steroid regimens. Compared with traditional assessments, SV95C demonstrated earlier sensitivity to disease progression (3 vs 9 months) and greater sensitivity at 12 months. Distribution- and anchor-based approaches revealed a change of - 0.10 to - 0.20 m/s as clinically meaningful. The EMA qualification of SV95C illustrates the willingness of regulators to accept novel digital endpoints for drug approval, setting an important precedent for the evidentiary basis of regulatory digital endpoint qualification that could transform clinical development in disorders affecting movement.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.