Working together to develop a preference study for treatments to reduce risk of developing RA


Figuring out what patients prefer can help inform decision making during drug development. But getting useful results requires rigorous preparation. An international team of PREFER researchers including academics, clinicians, pharmaceutical industry representatives and patient research partners have worked together to develop a preference study capable of informing decision making and also answering questions for the PREFER recommendations about the methods used to study patient preferences. In a recent BMJ Open publication, they describe how they designed their study to be informative for a wide range of stakeholders. 

Rheumatoid arthritis (RA) mainly affects the joints and causes pain, swelling, stiffness and fatigue. If patients are not treated, the joints can suffer permanent damage, which can lead to disability. Treatment of RA is usually required in the long term and is associated with serious side effects. There is now increasing interest in the idea of treating people for a short time before the onset of RA, to reduce their risk of developing this serious condition. Therefore, it’s important to understand how people at risk of RA would weigh up the risks and benefits of this kind of preventive treatment to inform research and development of new treatments.

First degree relatives have an increased risk of developing RA. This is the first study involving a large sample of confirmed, rather than self-reported, first degree relatives of patients with a diagnosis of RA. It also involves large samples of the general population across three European countries.

“Deciding whether to take a drug treatment for a disease you haven’t yet got, and might not ever get is complex. Yet preventive treatment is widely accepted for other chronic conditions, such as heart disease. This study will help us to understand what balance of treatment risks and benefits are acceptable to people at risk of developing RA” says Marie Falahee, one of the authors.

When designing their preference study, the multi-stakeholder team had both clinical and methodological research objectives in mind. For example, this study compares two different methods of collecting information about treatment preferences. Once the results from this PREFER core case study are in, they will contribute to developing efficient prevention strategies for RA as well as best practice for integrating patient preferences in drug development. The results will feed into the project’s recommendations for when and how to listen to the patient voice.

Explore PREFER publications 

Falahee M, Simons G, DiSantostefano R, Valor Méndez L et al., Treatment preferences for preventive interventions for rheumatoid arthritis: protocol of a mixed methods case study for the Innovative Medicines Initiative PREFER project, BMJ Open, 2021;11:e045851 DOI: 10.1136/bmjopen-2020-045851

By Anna Holm