Trialblazers: “Build-a-Trial” workshops for people with psoriatic arthritis

Abstract

Caroline Struthers of the Enhancing the QUAlity and Transparency Of health Research (EQUATOR) Centre of the Nuffield Department of Orthopaedics, Rheumatology, and Musculoskeletal Sciences (NDORMS) at the University of Oxford in the UK developed and ran a series of ‘Build-a-Trial’ workshops for patients and carers. The workshops were designed to allow participants to have input into key design decisions informing plans and a funding application for a clinical trial. This case study involved people with mild psoriatic arthritis. Participants provided input into who should be included in the trial; what treatment or management strategies should be tested; what was the most important thing to measure to indicate if a treatment or management strategy was working or not; how this could be measured during a trial in the most efficient, convenient, and least invasive way; what was the best way to recruit people to join the trial; and what was the best way to keep people engaged with the trial. Struthers’ aim was to develop a generalizable method of involving patients and carers in trial design in advance of a funding decision.

Introduction

Struthers won a grant in April 2023 to undertake preparatory work with a patient community to support an application for a Trialblazers project on the feasibility of involving patients and carers in the development of trial protocols to guide funders. This was to be followed in June 2024 by an application to the Medical Research Council (MRC) funding stream for a grant in understanding public involvement in non-clinical research.

In collaboration with the NDORMS’ public-involvement group, Open Arms, and led by clinician researcher Laura Coates, the team developed and ran Build-a-Trial workshops with patients to design this much needed trial from scratch. Struthers wrote a report for participants which gives more detail of what was done and what was learned, and was invited by Rachel Taylor of the National Institute for Health and Care Research (NIHR) at the Oxford Biomedical Research Centre (BRC) to present the Build-a-Trial project as a case study during a session on the impact and benefits of public and community involvement and engagement in health research at the Oxford Inside Out Event. 

Who should benefit?

Despite its stated commitment to patient and public involvement and engagement (PPIE), the academic research culture and infrastructure can create an intimidating environment for all but the most confident and scientifically literate patients. The commitment required to contribute to a research project can be a burden for people who are, by definition, less likely to be able to bear it.

Starting with trials, Trialblazers would address the need for a range of low-commitment and high-impact ways for patients and carers to influence the direction and design of trials at the earliest possible stage. Common mistakes include primary outcomes being chosen without any input from patients, a lack of a clear and justifiable statement of what is considered by patients to be the smallest important effect size, and a reliance on subjective outcome measures (such as surveys and questionnaires) when trials cannot be placebo controlled or masked (for example, in studies of psychological therapies).

These mistakes and others could be avoided by bringing teams of patients, carers, clinicians, and methodological experts together to conduct robust critical appraisals of previous and ongoing treatment trials that are relevant to a particular patient population, and to work together to design detailed plans or protocols for future trials of that treatment which avoid the mistakes of the past.

An added benefit of this method of designing trials would be to avoid factors which can cause significant bias in the way trials are designed, conducted, and reported, such as financial and non-financial conflicts of interest. If generated by funders seeking researchers in a high priority area, these ‘pre-protocols’ could be made openly available as a resource of pre-approved clinical trial designs which funders could put out to tender. If generated by researchers seeking funding, the pre-protocols generated by patients and carers would demonstrate the researchers’ commitment to meaningful public involvement at the earliest possible stage of their research.

Shaun Treweek, a professor of Health Services Research at the University of Aberdeen and the director of Trial Forge, stated that “Your idea is excellent and is the sort of design improvement innovation that is lacking [...] Having methodologically sound, patient-relevant protocols would be a good step towards better commissioning and reducing research waste.”

Engagement

The main support for this work was the department’s Open Arms patient-involvement group. They initially allowed Struthers a spot at their regular ‘Meet the Researcher’ event to talk directly to patients about the Trialblazers project.

A survey was created to recruit patients and carers to the Build-a-Trial workshops, and Coates helped with recruitment via her own patient contacts and colleagues. Struthers liaised closely with participants in the weeks leading up to the workshops and sent them participant information, a fun edible incentive, and details of payment for their contribution in line with the National Institute for Health and Care Research (NIHR) guidelines. The information was presented in an informal and engaging way and was sent to participants in advance of the in-person and online workshops.

After the workshops, Struthers kept in touch with all participants, asking for further feedback and whether they wanted any further involvement. A report of the workshops was sent to all participants and they will be acknowledged in any planned journal article.

One of the in-person workshop participants contributed to the presentation at Oxford University’s Inside Out Event in September 2023.

Research

Struthers’ first initiative to try and involve patients at the earliest possible stages of designing a trial was to develop and run three Build-a-Trial workshops. Struthers recruited a total of 26 people and facilitated their input into a much wider range of decisions on trial design than is usually offered by research teams. The crucial difference between this approach and conventional involvement opportunities is that the engagement takes place before a detailed trial protocol has been drafted or the trial has been funded.

The pilot workshops were run with a specific patient group: people with psoriatic arthritis. The intention is to develop this approach into a method which can be applied to any clinical trial of already licensed treatments or established therapeutic approaches in need of a stronger evidence base which can only be provided by trials.

This project was triggered by a wish to try out elements of a patient-led approach to trial design to inform a funding application to develop and standardize the approach so it can be adapted for trials in other patient populations.

The main challenge was a lack of expertise and training in facilitating workshops. Limitations of the funding made it impossible to recruit any individuals who are experts both in trial methodology and in patient involvement to help design and run the workshops more efficiently and effectively. We believe this is the first time this approach to trial design has been attempted.

Translating into impact

It has not yet been possible to objectively measure or evaluate the wider impact of this work. However, it is hoped that the impact will include a successful application for Coates and her colleagues to win funding for a trial with patients with mild psoriatic arthritis. Another possibility is that some of the first Build-a-Trial workshop participants could be recruited to the funded trial-steering committee to keep the trajectories on the straight and narrow.

Struthers hopes that the work will also lead to a successful funding application for the Trialblazers project. Both the NIHR and the James Lind Alliance have expressed interest in the idea, and it is hoped that this will translate into support or collaboration for the funding application. The NIHR has a standard rolling call for research studies addressing James Lind Alliance priorities, and there may be potential to extend the scope of the James Lind Alliance priority-setting partnerships to include Build-a-Trial workshops to design trials indicated by one or more of the top-ten research priorities.

It may also be possible to publish the results of Build-a-Trial workshops and explicitly link them to the NIHR rolling call. In advance of the funding application in June 2024, Struthers made efforts to advocate for this approach, for example by joining the National Health Service (NHS) Health Research Authority (HRA) Public Involvement Working Group on Clinical Trials. After a second working group meeting, two participants independently contacted Struthers wanting more information about Trialblazers. It is hoped that the HRA might provide support or collaboration for funding applications. At the time of writing, it was too early to try and implement this approach more widely in the EQUATOR Centre of the University of Oxford. However, if funding is secured, Struthers intends to develop and test this approach in more patient groups. The hope is that this idea can become a standard way of initiating and designing more pragmatic, relevant, and adequately powered clinical trials which will benefit larger numbers of patients.