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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

Authors
  • Servais, Laurent
  • Camino, Eric
  • Clement, Aude
  • McDonald, Craig M.
  • Lukawy, Jacek
  • Lowes, Linda P.
  • Eggenspieler, Damien
  • Cerreta, Francesca
  • Strijbos, Paul
Type
Published Article
Journal
Digital Biomarkers
Publisher
S. Karger AG
Publication Date
Aug 05, 2021
Volume
5
Issue
2
Pages
183–190
Identifiers
DOI: 10.1159/000517411
Source
Karger
Keywords
Disciplines
  • Review Article
License
Green
External links

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.

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