Evolving regulatory perspectives on digital health technologies for medicinal product development

Autor: Seya Colloud, Thomas Metcalfe, Scott Askin, Shibeshih Belachew, Johannes Ammann, Ernst Bos, Timothy Kilchenmann, Paul Strijbos, Damien Eggenspieler, Laurent Servais, Chloé Garay, Athanasios Konstantakopoulos, Armin Ritzhaupt, Thorsten Vetter, Claudia Vincenzi, Francesca Cerreta

Publikováno v: npj Digital Medicine, Vol 6, Iss 1, Pp 1-11 (2023)

Abstract Digital health technology tools (DHTTs) present real opportunities for accelerating innovation, improving patient care, reducing clinical trial duration and minimising risk in medicines development. This review is comprised of four case stud

Externí odkaz: https://doaj.org/article/96809dd19189420b9cbff01bd079ea62

Stride Velocity 95th Centile: Insights into Gaining Regulatory Qualification of the First Wearable-Derived Digital Endpoint for use in Duchenne Muscular Dystrophy Trials

Autor: Laurent, Servais, Karl, Yen, Maitea, Guridi, Jacek, Lukawy, David, Vissière, Paul, Strijbos

Publikováno v: Journal of Neuromuscular Diseases. 9:335-346

In 2019, stride velocity 95th centile (SV95C) became the first wearable-derived digital clinical outcome assessment (COA) qualified by the European Medicines Agency (EMA) for use as a secondary endpoint in trials for Duchenne muscular dystrophy. SV95

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::71cc937a58ae1e58e6425be5dd0da700
https://doi.org/10.3233/jnd-210743

The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy.

Autor: Muntoni, Francesco^1,2 (AUTHOR), Byrne, Barry J.³ (AUTHOR), McMillan, Hugh J.⁴ (AUTHOR), Ryan, Monique M.⁵ (AUTHOR), Wong, Brenda L.⁶ (AUTHOR), Dukart, Juergen^7,8 (AUTHOR), Bansal, Amita⁹ (AUTHOR), Cosson, Valerie⁹ (AUTHOR), Dreghici, Roxana^9,10 (AUTHOR), Guridi, Maitea⁹ (AUTHOR), Rabbia, Michael¹¹ (AUTHOR), Staunton, Hannah¹² (AUTHOR), Tirucherai, Giridhar S.¹³ (AUTHOR), Yen, Karl^11,14 (AUTHOR), Yuan, Xiling¹³ (AUTHOR), Wagner, Kathryn R.^9,15 (AUTHOR) kathrynraewagner@gmail.com, the Taldefgrobep Alfa Study Group (AUTHOR), Carvajal, Irvith (AUTHOR), Chimalakonda, Anjaneya (AUTHOR), Gokemeijer, Jochem (AUTHOR)

Publikováno v: Neurology & Therapy. Feb2024, Vol. 13 Issue 1, p183-219. 37p.

Stride Velocity 95^th Centile Detects Decline in Ambulatory Function Over Shorter Intervals than the 6-Minute Walk Test or North Star Ambulatory Assessment in Duchenne Muscular Dystrophy.

Autor: Rabbia, Michael, Guridi Ormazabal, Maitea, Staunton, Hannah, Veenstra, Klaas, Eggenspieler, Damien, Annoussamy, Mélanie, Servais, Laurent, Strijbos, Paul

Publikováno v: Journal of Neuromuscular Diseases; 2024, Vol. 11 Issue 3, p701-714, 14p

Quantifying the Benefits of Digital Biomarkers and Technology-Based Study Endpoints in Clinical Trials: Project Moneyball.

Autor: Mori, Hiromasa, Wiklund, Stig Johan, Zhang, Jason Yuren

Publikováno v: Digital Biomarkers; 2022, Vol. 6 Issue 2, p36-46, 11p