First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases

Laurent Servais, Eric Camino, Aude Clement, Craig M. McDonald, Jacek Lukawy, Linda P. Lowes, Damien Eggenspieler, Francesca Cerreta, Paul Strijbos

Research output: Contribution to journalReview articlepeer-review

1 Scopus citations

Abstract

Background: Functional outcome measures used to assess efficacy in clinical trials of investigational treatments for rare neuromuscular diseases like Duchenne muscular dystrophy (DMD) are performance-based tasks completed by the patient during hospital visits. These are prone to bias and may not reflect motor abilities in real-world settings. Digital tools, such as wearable devices and other remote sensors, provide the opportunity for continuous, objective, and sensitive measurements of functional ability during daily life. Maintaining ambulation is of key importance to individuals with DMD. Stride velocity 95th centile (SV95C) is the first wearable acquired digital endpoint to receive qualification from the European Medicines Agency (EMA) to quantify the ambulation ability of ambulant DMD patients aged ≥5 years in drug therapeutic studies; it is also currently under review for the US Food and Drug Administration (FDA) qualification. Summary: Focusing on SV95C as a key example, we describe perspectives of multiple stakeholders on the promise of novel digital endpoints in neuromuscular disease drug development.

Original languageEnglish (US)
Pages (from-to)183-190
Number of pages8
JournalDigital Biomarkers
Volume5
Issue number2
DOIs
StatePublished - Aug 5 2021

Keywords

  • Digital endpoints
  • Duchenne muscular dystrophy
  • Neuromuscular disease

ASJC Scopus subject areas

  • Computer Science Applications
  • Medicine (miscellaneous)
  • Health Informatics

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