Remote delivery of clinical care and validation of remote clinical outcomes in neuromuscular disorders: a response to COVID-19 and proactive planning for the future

266^th ENMC International Workshop

Location: Hoofddorp, The Netherlands

Title:  Remote delivery of clinical care and validation of remote clinical outcomes in neuromuscular disorders: a response to COVID-19 and proactive planning for the future

Date: 1 - 3 April 2022

Organisers:
Dr Linda Lowes (USA)
Meredith James (UK)
Dr Lindsay Alfano (USA)
Dr Gita Ramdharry (UK)

Participants:
Participants from 10 countries across 5 continents, 4 patient advocacy groups, 2 patient representatives
Alyssa Burns (USA) Julie Coats (USA), Jose Corderi (Argentina), Annette Costello
(Ireland), Dr Michelle Eagle (UK), Maha Elseed (UK), Elena Gargaun (France), Elin Haf Davies
(UK), Anri Human (South Africa), Alessandra Gaetta (UK), Heather Gordish Dressman (USA),
Lone Knudsen (Denmark), Jenn Levy (USA), Charlotte Lilien (UK), Maria Mancini (USA), Dr
Anna Mayhew (UK), Katlyn McGrattan (USA), Robert Muni-Lofra (UK), Allison Peck (USA),
Nathan Peck (USA), Valeria Prada (Italy), Hara Pylarinou (Cyprus), Dr Kristy Rose (Australia),
Ulla Werlauf (Denmark)

BACKGROUND AND AIMS FOR THE WORKSHOP

Standardised assessments of strength and function are used to track disease trajectory, to
prospectively advise the need for equipment, home and work modifications, and other
assistive devices in individuals with Neuromuscular disorders (NMD). Clinical outcome
assessments (COA) of strength and function are often primary or secondary endpoints for
pharmacological, rehabilitation interventions or natural history studies. It is critical that data
representing key functional endpoints are obtained via highly controlled and standardized
procedures by individuals who are qualified and adequately trained to collect them.
The COVID-19 pandemic presented unprecedented challenges to the in-person delivery of
clinical care and clinical trials. In March 2020, with several trials and natural history studies
underway, including trials that were due to collect primary endpoints during lockdowns, a
fast response, and alternative strategies to in person clinic-based assessments were required
to protect study efficacy endpoints and ensure ongoing patient evaluations.
A group of expert Physiotherapists in NMD, working across clinics, countries, and clinical
trials developed initial guidelines for the suitability and feasibility of performing COA
commonly used in clinical trials. A number of sponsors introduced amendments to enable
remote evaluations, supported by video streaming of the assessment to their local clinical
evaluators, which has raised discussions around the validity and reliability of remote
assessments and their correlation to clinic-based testing. Additionally, the model of clinical
evaluator training used was adapted to meet COVID-19 restrictions to ensure trained staff
were available for clinical evaluations.
As we progress through the pandemic, we have an opportunity to disrupt traditional trial
design paradigms. It is critical to evaluate the comparability to inform future clinical trial
design to maintain the integrity of data while also reducing the burden of trial participation,
and potentially making clinical trials accessible to a larger pool of patients.
Workshop aim and objectives
The major aim of this workshop was to assemble international experts and key stakeholders
to evaluate the utility and validity of conducting remote clinical outcomes assessments for
NMD.

Objectives:
• Evaluate the utility of remote evaluation for both clinical and research purposes to
advise best practice for continuity and access to expert clinical care and maintain the
integrity of clinical trial design
• Document experiences of conducting remote assessments from a wide range of
stakeholders (including clinicians, patients, families, sponsors, and regulatory
authorities).
• Explore utility of technology-based home assessments for NMD
• Identify limitations to using remote assessments and impact for future trial design
• Develop an action plan to address identified knowledge gaps and areas of future
research

Day 1
Friday participants workshop participants reviewed and discussed shared experiences from
around the world, benefits and limitations to conducting NMD clinical visits via telehealth to
date. Discussions focused on strategies utilised to transition to remote clinical outcome
assessment evaluations, clinical trials underway and natural history studies. The group
identified and documented current remote clinical care practices around the globe, barriers
to implementation and new initiatives with simple technology to improve access to health.
Patient and sponsor experience of remote clinical and research assessments was shared.

Day 2
Day 2 reviewed available data from prospective studies evaluating the validity and reliability
of remote testing of COA. The development, feasibility and utility of wearables, what real
world metrics are currently validated and the ongoing development of home-based
technology for the monitoring of patients with NMD was discussed. Statistical
considerations for the comparison of clinic and at home data were reviewed along with the
important current and future potential roles of patient registries in data collection and
informing care and research practices.

Day 3
The attendees reviewed the current state of standardised training of COA in clinical trials
around the globe. The group then discussed adaptions to remote delivery of health care and
lessons learned. The group highlighted the challenges in global e-health literacy capacity
building and the need for inclusivity, with embedding local knowledge to facilitate processes
and including new parts of the world while respecting and carefully navigating cultural
differences. Group discussions of future remote care and research options for patients with
NMD.

A full report will be published in Neuromuscular Disorders (pdf)