Title: Better outcomes for patients living with motor neuron disease
Funder: Department of Health and Social Care

Abstract: Research questions: Can highly specialised personalised evidence-based multidisciplinary care for people living with MND be delivered effectively close to home? Can technology and patient centred outcome measures reduce burden and increase participation in clinical trials? Background: Motor neuron disease (MND) is a devastating neurodegenerative disease with high-unmet needs. The pace of change in MND can be fast and unpredictable with patients and care teams constantly playing catch up with the decisions that need to be taken and the provision of care interventions is often too late. Good MDT care prolongs life in MND but access to this is inequitable and burdensome. Only 10% of patients with MND enter clinical trials, in which trial burden can be considerable in part due to non-patient centred outcome measures.Aims and objectives: I aim to transform how care is delivered and how clinical trials are conducted. I will develop and evaluate a low burden evidence-based personalised care pathway for people living with MND to support decision making, the timely delivery of care interventions to improve quality of life and survival, and increased participation in clinical trials. I will develop a care prediction model to predict the optimal timing of care interventions and use this to develop and enhance decision aids for patients. I will develop a new combined function and HRQoL preference-based patient reported outcome measure (PROM). I will define outcomes that are relevant and patient centric and evaluate technologies that enable their low burden remote collection in trials. I will develop further our Telehealth in MND (TiM) platform adding new remote monitoring capabilities and evaluating impact on care. I will develop the evidence for secretion management.Methods: I propose four work packages (WPs) that employ a broad range of qualitative and quantitative methodologies with co-design a consistent thread throughout. NICE Evidence standards framework for digital health technologies and the MRC framework for evaluating complex interventions will be adhered to. Timelines for delivery:WP1: Within 12 months the personalised prediction model will be developed. By the end of year 3 this will be integrated into decision aids.WP2: In 2 years a new PROM will be developed and ready for incorporation into clinical trials. By end of year 3 three remote technologies will be ready to be deployed in low burden clinical trials of MND interventions. By year 5 report performance of PROM and remote technologies in clinical trials.WP3: Within 3 years successful iteration of TiM implemented across the MND network with evaluation of impact completed and reported by end of year 5. WP4: Within year 1 co-design clinical trial and submit application to HTA aiming to start clinical trial in year 3. Anticipated Impact and dissemination: The key impact from this work will be the introduction of timely personalised high-quality care for those living with MND and the opportunity for all to participate in low burden clinical trials, ultimately bringing closer the day when we have an effective treatment for MND. I will disseminate findings through publication, social media, and existing partnerships.

Lay abstract: AimsWe can improve the quality of life of those living with motor neuron disease (MND) now by tailoring care to an individual and find a cure by doing better trials. I aim to transform how care is delivered and how clinical trials are conducted. BackgroundAcross all UK communities 1 in every 300 people develop MND. They lose the ability to walk, use their arms and hands, speak, eat and breathe. These can occur in any order making it difficult to plan timely care and with no effective treatment most people die within 3 years. Good care for people with MND can improve survival but accessing specialist care can be difficult particularly in the context of a pandemic when vulnerable people with MND are shielding. Improving care nowThe lack of reliable information about their MND and their likely disease trajectory means that decisions are often delayed with serious consequences as most interventions have an optimal window for introduction. I will examine existing databases of over 15000 patient journeys to develop computer models that can predict the optimal timing of interventions for people with MND and develop new resources to support patients to make decisions about the care they want to receive. People with MND have to make arduous trips to receive specialised care so instead they often receive non-specialised care from community teams who may not have the knowledge of MND. I have developed 'TiM' - a telehealth system which enables patients to communicate with their MND experts. With patients I will develop this further to remotely monitor other important aspects of MND care that currently require hospital trips and to improve communication between different care teams involved in the care of a person with MND. I will roll this system out across the UK and demonstrate its impact on people living with MND and their care.Currently treatment is focused on managing day-to-day problems to improve quality of life. One problem which remains difficult to treat is uncontrolled drooling from the mouth. People with MND have indicated this is a distressing problem. With patients I will plan a clinical trial to determine the best way to treat drooling. Finding the cure fasterThere is a desperate need for a cure for MND but progress has been slow. This is partly due to the way treatments are tested in burdensome trials in which as little as 10% of patients with MND participate. This means trials take much longer and their findings may not be true for the other 90%. Everyone with MND should have the opportunity to participate in clinical trials that are evaluating outcomes that are important to them and are designed to minimise the burden of being in a trial. To that end I will develop with people with MND a new patient reported outcome measure and demonstrate how remote technologies can be used to make participation easier.I have presented my research ideas to 20 patients and carers from around the UK and the national MND charity. They felt my plans addressed important areas. In particular the need to improve access to care for those living with MND and to be able to increase participation in clinical studies. I will work with patients and charities to ensure that the information produced from this work is accessible to them. I will publicise the results in medical journals and at meetings to reach teams who look after MND patients and design clinical trials. I will use the findings to influence NHS care through my experience with NICE and my contacts with the commissioners of NHS MND services.

Research activity codes: 7.2 END OF LIFE CARE;7.3 MANAGEMENT AND DECISION MAKING;8.4 RESEARCH DESIGN AND METHODOLOGIES (HEALTH SERVICES)
Health categories: NEUROLOGICAL