Identifying Digital Biomarkers for the Self-Monitoring of Patients Living with Generalized Myasthenia gravis: a Proof of Concept
Pascal Laforet1, David Orlikowski2, Guillaume Nicolas1, Hélène Prigent1, Edouard Berling1, Clarissa Gorin3, Ana Júlia Moreira3, Juliette Narcy3, Angéline Plaud3, Emma Touré Cuq3, Martin Keller4, Brian Werneburg5, Emrah Aras5, Michael Benatar6
1Nord-Est/Ile-de-France Neuromuscular Reference Center, UMR 1179, Neurology Department, Raymond-Poincaré Hospital, Garches, and FHU PHENIX, France, 2Nord-Est/Ile-de-France Neuromuscular Reference Center, UMR 1179, CIC 1429, Neurology Department, Raymond-Poincaré Hospital, Garches and FHU PHENIX,UVSQ Paris-Saclay University, France, 3Ad Scientiam, Paris, France, 4Alexion Pharma GmbH, Giesshübelstrasse 30, 8045 Zürich, Switzerland, 5Alexion Pharmaceuticals Inc., 121 Seaport Blvd, Boston, MA 02025, USA, 6Department of Neurology, Emory University, GA, USA
Objective:
To identify clinically meaningful digital biomarkers (dBMKs) intended to track symptoms and signs among people living with generalized myasthenia gravis (gMG) and to prepare the development of a software medical device for the patients’ unsupervised digital self-assessment.
Background:
Generalized myasthenia gravis (gMG) is characterized by fluctuating manifestations and heterogeneity in fatigue and muscle group weakness. Literature pinpoints a lack of objective measurement tools for the self-assessment of gMG symptoms outside office visits, resulting in different perceptions between physicians and patients on the impact of the disease.
Design/Methods:
We used a Double Diamond methodology to identify dBMKs of interest for gMG patients and evaluate their relevance for clinicians through qualitative interviews. Unmet needs and dBMKs concepts were prioritized during a technical Proof-of-Concept (PoC) study. The acceptability of the digital device was assessed with prototype-supported interviews.
Results:
Interviews with 27 patients (France, USA) confirmed the feasibility of dBMKs to measure gMG-related neurological functions. All were interested in tracking symptoms. 9 international MG experts were interested in assessing gMG symptomology between office visits with clinically meaningful dBMKs. A PoC study on healthy subjects, performed under different conditions, confirmed the feasibility of analyzing raw data collected by the smartphone’s sensors. 5 digital active measures inspired by clinical gold standards and relying on the smartphone’s sensors were selected with their related dBMKs (ptosis, breathing, dysarthria, upper and lower extremities functions). Patient-reported outcome measures (daily activities, pain, sleep, depression, quality of life) will be incorporated as e-questionnaires.
Conclusions:
We perceived a need for an unsupervised patient self-assessment digital solution to monitor symptomatology outside office visits. In 2023, development of ME&MG, a smartphone solution, will begin. It aims to inform and improve communication between patients and their care teams. International multicenter studies comparing ME&MG to clinical gold standards including the Quantitative Myasthenia Gravis score, will be deployed.