Connecting real-world digital mobility assessment to clinical outcomes for regulatory and clinical endorsement–the Mobilise-D study protocol

Background: The development of optimal strategies to treat impaired mobility related to ageing and chronic disease requires better ways to detect and measure it. Digital health technology, including body worn sensors, has the potential to directly and accurately capture real-world mobility. Mobilise-D consists of 34 partners from 13 countries who are working together to jointly develop and implement a digital mobility assessment solution to demonstrate that real-world digital mobility outcomes have the potential to provide a better, safer, and quicker way to assess, monitor, and predict the efficacy of new interventions on impaired mobility. The overarching objective of the study is to establish the clinical validity of digital outcomes in patient populations impacted by mobility challenges, and to support engagement with regulatory and health technology agencies towards acceptance of digital mobility assessment in regulatory and health technology assessment decisions. Methods/design: The Mobilise-D clinical validation study is a longitudinal observational cohort study that will recruit 2400 participants from four clinical cohorts. The populations of the Innovative Medicine Initiative-Joint Undertaking represent neurodegenerative conditions (Parkinson’s Disease), respiratory disease (Chronic Obstructive Pulmonary Disease), neuro-inflammatory disorder (Multiple Sclerosis), fall-related injuries, osteoporosis, sarcopenia, and frailty (Proximal Femoral Fracture). In total, 17 clinical sites in ten countries will recruit participants who will be evaluated every six months over a period of two years. A wide range of core and cohort specific outcome measures will be collected, spanning patient-reported, observer-reported, and clinician-reported outcomes as well as performance-based outcomes (physical measures and cognitive/mental measures). Daily-living mobility and physical capacity will be assessed directly using a wearable device. These four clinical cohorts were chosen to obtain generalizable clinical findings, including diverse clinical, cultural, geographical, and age representation. The disease cohorts include a broad and heterogeneous range of subject characteristics with varying chronic care needs, and represent different trajectories of mobility disability. Discussion: The results of Mobilise-D will provide longitudinal data on the use of digital mobility outcomes to identify, stratify, and monitor disability. This will support the development of widespread, cost-effective access to optimal clinical mobility management through personalised healthcare. Further, Mobilise-D will provide evidence-based, direct measures which can be endorsed by regulatory agencies and health technology assessment bodies to quantify the impact of disease-modifying interventions on mobility. Trial registration: ISRCTN12051706

List of assessments and outcomes collected during screening, baseline assessment and every six months.

T1, Screening/Baseline; T2, 6 month assessment; T3, 12 month assessment; T4, 18 month assessment; T5, 24 month assessment; *, indicates key (primary) cohort specific outcome measure; SPPB, short physical performance battery–PFF key primary cohort specific outcome measure; † falls and fracture data are collected retrospectively, 12 month retrospective at T1 and 6 month retrospective at T2-T5; β, pre-fracture status is measured at T1, current status is measured at T3 and T5; α, only applicable to acute patients; Outcome type, type of outcome measure in accordance with FDA terminology; COA, clinical outcome measure–describes or reflects how a patient feels, functions, or survives; PRO, Patient-reported outcome; ObsRO–Observer-reported outcome; ClinRO, Clinician-reported outcome; PerfO, Performance-based outcome; PerfO-P, Performance-based outcome physical measure; PerfO-C, Performance-based outcome cognitive/mental measure; Construct, validation construct assessed; PC, predictive capacity; CV, construct validity; DC, detect change over 24 months; MID, Minimum Important Difference; MC, medical chart.</p

Proposed data flow.

Proposed data flow.</p

Inclusion and exclusion criteria.

Inclusion and exclusion criteria.</p

Study flow.

Study flow.</p

Flow chart to illustrate full recruitment process.

Flow chart to illustrate full recruitment process.</p