Identifying methodological uncertainties in the design and analysis of trials in people living with multiple long-term conditions

Research question What are the priority methodological uncertainties in the design and analysis of clinical trials informing the care of people living with MLTCs?

Background The number of people living with MLTCs is increasing and the age of onset is decreasing, meaning that people live with MLTCs and poor health for longer. By 2035 two-thirds of adults aged over 65 will be affected.

The prevalence of MLTCs is higher and commences earlier in the life course in those of non-white ethnic populations and in people from deprived backgrounds. Living with MLTCs is associated with high treatment burden, poor quality of life and premature mortality. Improving care for people with MLTCs has been described as a global and national priority.

There have been limited numbers of trials which have focussed on improving outcomes in people with MLTCs. Of those that have been conducted, the results have not identified clear evidence to support implementation.

Aim This project aims to improve care delivery for those with MLTCs through better design/analysis of clinical trials for this vulnerable group.

We will work towards this by prioritising methodological uncertainties in the design and analysis of clinical trials for people living with MLTCs.

This will be addressed through the following: Systematic review of completed trials in MLTCs to establish methodological approaches Consensus study to identify methodological research priorities for MLTC trials Workshop to disseminate the findings and develop working groups Methods To assess current practice and to feed into the first stage of the consensus study, a systematic review of completed MLTC trials will be undertaken.

The review will include trials of any type of intervention aiming to improve outcomes for those with MLTCs. Our consensus study will include the views of both professionals and those with lived experience. For the professional participants a 3 round Delphi study will elicit and prioritise methodological uncertainties.

On the advice of our PPI group, both face to face and virtual meetings will be held with the lived experience participants to ensure the findings of the Delphi exercise are relevant to those with MLTCs.

Finally, we will hold an online dissemination workshop including a diverse group of both professional and public contributors.

The findings of the study will be presented and a series of breakout rooms will allow attendees to begin discussions on how to address these uncertainties.

Timelines for delivery Start September 2024 for 12 months, as the findings from each element feed into the next, they will run subsequently.

Anticipated impact and dissemination The impact of this research will be realised as researchers address the uncertainties identified during this work, a workshop to disseminate our findings will accelerate this. The implementation of novel approaches in the future will improve the design and analysis of trial sin this area.

Better trial design and analysis reduces research waste and will mean that effective treatments/interventions can be shown as such and implemented into clinical practice in a timelier manner.