Rheumatiod arthritis: Preventative treatment

Rheumatoid arthritis (sometimes called “RA”) is a common chronic inflammatory disease that affects around 1% of the population. It is 2-3 times more common in women. In most cases, patients begin having symptoms between 40 to 60 years of age, but it can begin earlier, or later in life.

RA affects the joints and causes pain, swelling, and stiffness, but also fatigue. If patients are not treated, the joints can suffer permanent damage, which subsequently can lead to disability. To date, there is no cure, which means that for the vast majority of patients treatments are long term, with the aim of controlling the inflammatory disease activity and reducing symptoms.

There is increasing research interest in the idea of treating individuals who are at an increased risk of developing RA to assess whether a relatively short course of therapy will prevent or delay the onset of RA. One such ‘at risk’ group are the siblings and children of patients with RA, who are four times more likely to develop the disease. The presence of RA-related autoantibodies in the blood increases the risk of developing the disease further.

This PREFER case study will ask siblings, children and members of the general public about their preferences for preventive treatments for RA. The case study will further look at how participant characteristics, country (UK vs Germany), population (siblings and children vs. general population), knowledge/experience with RA will impact on these treatment preferences. In addition, this case study will evaluate the similarity of two different methods to measure treatment preferences.

Publications

G Simons, K Schölin Bywall, M Englbrecht, EC Johansson, RL DiSantostefano, C Radawski, J Veldwijk, K Raza & M Falahee (2022) Exploring preferences of at-risk individuals for preventive treatments for rheumatoid arthritis, Scandinavian Journal of Rheumatology, DOI: 10.1080/03009742.2022.2116805

Simons G, Veldwijk J, DiSantostefano R, Englbrecht M, et al., Preferences for preventive treatments for rheumatoid arthritis: discrete choice survey in the UK, Germany and Romania, Rheumatology, 2022, DOI: 10.1093/rheumatology/keac397

Simons G, Caplan J, DiSantostefano R, Veldwijk J, et al., Systematic review of quantitative preference studies of treatments for rheumatoid arthritis among patients and at-risk populations, Arthritis Research & Therapy, 2022, DOI: 10.1186/s13075-021-02707-4

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

Fact sheet

Therapeutic area	Rheumatoid arthritis
Study led by	University of Birmingham University of Erlangen
PREFER leads team	Karim Raza Marie Falahee Larissa Vaylor-Mendez Jorien Veldwijk Rachael DiSantostefano
MPLC decision points of interest	Early-development and post-marketing
PREFER case study acronym	RA
Clinical objectives	Assessing the preferences of people at risk of RA for preventive treatments Evaluating the maximum acceptable risk (MAR)/Minimum acceptable benefit (MAB) Characterising preference heterogeneity and characteristics that may explain heterogeneity
Patients from	United Kingdom Germany
Methods in Qualitative study	Focus group discussion Nominal group technique
Methods in Quantitative study	DCE  Probabilistic Threshold Technique
End-date qualitative data collection	December 2019
End-date quantitative data collection	Q3 2020