Remote Delivery of Motor Function Assessment and Training for Clinical Trials in Neuromuscular Disease: A Response to the COVID-19 Global Pandemic

Abstract

Clinical outcome assessments of function or strength, assessed by physical therapists, are commonly used as primary endpoints in clinical trials, natural history studies and within clinics for individuals with neuromuscular disorders. These evaluations not only inform the efficacy of investigational agents in clinical trials, but also importantly track disease trajectory to prospectively advise need for equipment, home and work modifications, and other assistive devices. The COVID-19 pandemic had a global impact on the safety and feasibility of in-person visits and assessments, necessitating rapid development of mitigation strategies to ensure ongoing collection of key clinical trial endpoints and access to expert clinical care despite travel restrictions. Physical therapists who are expert in neuromuscular disorders working across clinics, countries, and clinical trials developed initial guidelines and methods for the suitability and feasibility of performing remote evaluations. A number of Sponsors introduced amendments to their study protocols to enable remote evaluations, supported by live video streaming of the assessment to their local clinical evaluators. Similarly, application of these techniques to clinical telemedicine enabled objective evaluations for use in payer discussions, equipment procurement, and general access to expert physical therapy services. Here we report on our methodology for adapting current practices to remote testing and considerations for remote evaluations.

Reliable virtual clinical assessment in spino-bulbar muscular atrophy (SBMA)

“…The pandemic has greatly accelerated the development of telehealth in the remote clinical evaluation of patients with neuromuscular disorders,11 and the administration of outcome measures remotely has been performed or advised particularly for myopathies, spinal muscle atrophy and myasthenia gravis 12 13. No similar attempt has been reported for SBMA, although we believe SBMA is particularly suitable for such approach because it is not characterised by predominant distal muscle weakness and sensory loss, among the most difficult impairment components to evaluate remotely.…”

A Comprehensive Review of Neuromuscular Manifestations of COVID-19 and Management of Pre-Existing Neuromuscular Disorders in Children

“…The guidelines address location of the patient, PT, clinical evaluator (CE), and involvement of a caregiver as assistant for the remote exam. Physical safety, data protection, and validity of clinical assessment were all addressed, with the authors reporting success in introducing remote visits to seven research trials over 20 trial sites [4].…”

“…Reliance on telemedicine has increased, which was easier for follow-up visits than for new diagnoses. Centralized clinical trials carried increased risk, and efforts were made to modify and continue the trials via remote administration [4].…”

Validity of remote live stream video evaluation of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy

“…The need to travel to distant clinical trial assessment sites can require missed time at work or school, resulting in lost wages in addition to the base cost of travel to the site; this may skew trial participation towards more affluent families [ 20 ]. Moreover, the difficulties of in-clinic assessments were exacerbated by the restrictions and risks associated with the COVID-19 pandemic for patients, caregivers, and clinicians alike [ 21 ]. As a result, there exists a strong need for a complementary mode of reliably assessing motor function.…”