Patient preferences surveyed to inform the development of acceptable new treatments

A survey of patients’ views about the benefits and risks of potential new treatments is paving the way for the patient voice to better inform the development of treatments to prevent chronic inflammatory diseases.

A new study that surveyed almost 3000 people across three European countries has established what levels of treatment related side effects are acceptable to those at risk of rheumatoid arthritis in return for a reduction in that risk.

The new paper published in the journal Rheumatology today (Wednesday 7 September) showed that effective preventive treatments were acceptable to most people who were asked to imagine they had a high risk of developing rheumatoid arthritis.

The research formed part of the IMI-PREFER research programme which has developed evidence-based recommendations on how and when patient preferences should be used to inform drug development and regulatory decision making. These recommendations received a positive qualification opinion by the European Medicines Agency in June 2022.

This latest paper contributes to a case study for the project which focused on patient preferences for preventive treatments for rheumatoid arthritis. The case study also addressed methodological research questions to inform recommendations on best practice for conducting patient preference studies to inform clinical trial design and regulatory approvals for new drugs.

Dr Marie Falahee, Lecturer in Behavioural Rheumatology at the University of Birmingham who led the study said:

“There is increasing awareness that information about what kinds of treatment are acceptable to people should inform decision making during the selection of new drug candidates, clinical trial design and approvals made by regulatory agencies and payers. This is especially important for preventive treatments, where there is uncertainty around whether, or when, the disease will develop. There have been a number of trials of preventive treatments for rheumatoid arthritis, and some have encountered difficulties recruiting people to take part. This underlines the importance of understanding what trade-offs between treatment benefits and risks are acceptable to people, to inform efficient, patient focussed drug development.”

The study showed that whilst few people would accept a risk of a very serious condition, such as cancer, people would accept higher risks of serious infections, such as pneumonia. The study also identified subgroups of people with different preferences for treatment benefits and risks, which were associated with participants’ health literacy and numeracy, as well as the extent to which they perceived themselves to be at risk of developing rheumatoid arthritis.

Karim Raza, Professor of Rheumatology at the University of Birmingham and co-investigator on the study, said:

“A preventive treatment for rheumatoid arthritis has the potential to reduce pain and disability at huge scale, as well as reducing significant healthcare costs. This study will be a useful guide for efficient development of preventive treatments that are acceptable to those at risk of getting rheumatoid arthritis. As part of our case study for IMI PREFER, this study is also having an important international impact on shaping best practice in patient centred drug development”