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
| Autor: | Eric Camino, Jacek Lukawy, Laurent Servais, Aude Clement, Paul Strijbos, Craig M. McDonald, Damien Eggenspieler, Francesca Cerreta, Linda Lowes |
|---|---|
| Jazyk: | angličtina |
| Rok vydání: | 2021 |
| Předmět: |
medicine.medical_specialty
Neuromuscular disease business.industry QH301-705.5 Duchenne muscular dystrophy Perspective (graphical) Medicine (miscellaneous) Wearable computer Health Informatics Review Article neuromuscular disease medicine.disease Computer Science Applications Clinical trial Physical medicine and rehabilitation Drug development medicine Functional ability Biology (General) business Wearable technology duchenne muscular dystrophy digital endpoints |
| Zdroj: | Digital Biomarkers, Vol 5, Iss 2, Pp 183-190 (2021) Digit Biomark |
| Popis: | 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. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|