2022 · Orphanet journal of rare diseases · open access
What role can decentralized trial designs play to improve rare disease studies?
Moore et al.
The finding, in our words
Decentralised trial designs using digital tools such as wearables and electronic assessments can reduce patient burden and improve inclusion and data quality in rare disease studies by enabling measurements in real-world settings. This matters for decentralised diagnostics as it supports more accessible, patient-centric research with environmentally valid endpoints.
A paraphrase to the Library’s standard, never the abstract. The source is one link away and is always the authority.
A direct-to-participant, multichannel recruitment strategy enrolled 34,244 older adults into a virtual trial testing an Apple Watch based heart health programme with electrocardiogram and irregular rhythm notification features, achieving broad geographic and gender diversity but under-representing non-White ethnic groups.
Wearable-derived digital biomarkers have been accepted by regulatory agencies as endpoints in clinical trials for Duchenne muscular dystrophy, showing their potential to support decentralised diagnostics through patient-centric monitoring. This matters because it enables more sensitive and continuous assessment of disease progression outside clinical settings.
Ma et al., Neurology and therapy (paywalled) · source ↗
This study found that a decentralised approach using home-based sleep apnoea screening, heart rhythm monitoring and activity tracking in patients with atrial fibrillation is feasible, with high data completeness and low drop-out rates. The results support the use of patient-centric digital tools in decentralised trials for chronic cardiac conditions.
Hashiba et al., Danish medical journal (paywalled) · source ↗
In a Phase 1 crossover study of etrasimod, self-collected blood microsamples yielded pharmacokinetic exposures comparable to conventional venous sampling, both with and without participant practice sessions. Continuous wearable sensors concurrently enabled real-time remote monitoring of safety vital signs and electrocardiograms, demonstrating the viability of hybrid decentralised designs in early-phase clinical trials.
Malhotra et al., Clinical pharmacology and therapeutics · source ↗
A large decentralised trial investigating a wearable device for COVID-19 detection retained 80.4% of participants at six months and 68.5% until completion. Multivariable analysis identified vaccinated pensioners as a high-risk discontinuation group, and targeted telephone calls reduced their dropout rate to 11.4%, showing that personalised retention strategies can improve engagement in remote diagnostic studies.
Zwiers et al., Mayo Clinic proceedings. Digital health · source ↗