Description

Background Motor Neuron Disease (MND) is an incurable disease, causing progressive weakness of muscles involving the limbs, speech, and swallowing, leading to progressive disability and eventual respiratory failure. In the UK, there are approximately 5,000 people with MND (pwMND) at any one time. The average life expectancy following diagnosis is two to three years but the course of MND can vary from only a few months to over ten years. The distress and burden of the disease affects pwMND, their family, and carers, and the relentless progression of disability causes social, emotional, and financial strain.

TiM-Care To provide more responsive care for pwMND and their carers, the Telehealth in MND-Care (TiM-C) on MyPathway system was developed. TiM-C is an online system that enables pwMND and their carers to report their symptoms to healthcare professionals (HCPs) from their homes using an app or via the online website. Users are asked to complete symptom questionnaires (e.g., appetite, weight, functional rating, breathing, mental health), answers to which are securely sent to the clinical portal (see figure one).

TiM-C was co-developed with pwMND, carers, and HCPs. The system is easy to use, even for those with significant disabilities, acceptable, and low in burden, taking approximately five minutes per week to complete. 85% of pwMND would recommend TiM to others and 95% would use TiM if they could not travel to clinic. HCPs found it accurate and easy to use.

TiM-Research To provide pwMND greater access to research from the comfort of their own homes, and collect longitudinal data relating to symptoms and treatment, researchers have created Telehealth in MND-Research (TiM-R). TiM-R will send regular symptom questionnaires to pwMND, whilst also enabling invitations to participate in future projects to be sent to those who are eligible. Results from research projects can then be sent back to participants. Anonymised data from the questionnaires will be made available to researchers in a controlled database.

Aim of the project The aim of this project is to create a research database that pwMND can be invited to and submit regular data (i.e., TiM-R). Users can then be invited to research projects and notified of results. Researchers can apply to a Research Management Committee for access to anonymised data.

Project Design PwMND will be invited to TiM-R through two methods. If they are using TiM-C as part of their clinical care, a notification will appear on their account describing the purposes of TiM-R and providing the participant information sheet (PIS). They can then provide consent if they are willing to join TiM-R. If the individual does not want to join TiM-R, they can still use TiM-C to receive clinical care.

If the individual is not using TiM-C as part of their clinical care, they may still join TiM-R by responding to a study advert, which will describe the purposes of the study and a link to the PIS. Advertisements will be distributed through social media, relevant charities (e.g., MND Association), and patient support groups. If willing, the participant can follow links on the PIS to create their TiM-R account, part of which will include providing consent for the purposes of this study.

Regardless of the invitation method, once the participant has consented, they will have access to TiM-R and will start to receive regular research questionnaires (see section 9). They may also receive invitations to other research projects for which they are eligible. For each of these future projects the user can choose whether to participate.

The participant can choose to withdraw from TiM-R at any time without having to give a reason by contacting the Research Management Committee. Contact details will be available through TiM-R. Any withdrawal from TiM-R will not affect participants' ability to use TiM-C.

Researchers can contact the Research Management Committee to gain access to anonymised data collected through TiM-R. The exact process is described in section 12. Providing that the reason for this data usage falls under one of the topics covered by this generic ethical approval (see section 11), researchers will not require their own NHS ethical approval to use this data. If the Research Management Committee agrees access to anonymised data, the researcher will be provided with log in details to a Trusted Research Environment (TRE), where they can perform analyses and write up necessary reports.

Clinical Data collection Once pwMND have created their TiM-R account, they will be asked to complete regular questionnaires. Additional questionnaires may occasionally be added, either by a researcher request or to update the system in line with literature and patient reported outcome measure development. No questionnaire will ask sensitive questions relating to protected characteristics. The addition or removal of a questionnaire will be dependent on a majority decision of the Research Management Committee, who will consider user burden and justification of need for any adaptations.

PwMND who also have a TiM-C account complete regular symptom questionnaires. Researchers will ask these users to consent to the questionnaires to also be uploaded to the research database and used for research purposes, after they have been anonymised. This will be included in the PIS. The questionnaires include the SNAQ, PHQ-8, and GAD-7, however, these may change over the course of this project, depending on the needs of clinical teams using TiM-C.

Research Project Overview The database will support MND researchers inside and outside of the UK and their collaborators. Any outcomes of the research will provide information to improve the care, quality of life, and treatment of pwMND. All academic outputs will be publicly available on a dedicated website.

Only staff and researchers who have completed relevant research data security training will be granted access to TiM-R. The Research Management Committee will be responsible for ensuring that all participant-identifiable data is excluded from available datasets.