Getting the patient perspective into

clinical trials for rheumatoid arthritis

07/05/09: Tessa Sanderson, Academic Rheumatology, University of the West of England

Taken from NRAS magazine, Spring 2009

Until recently, the views of people with RA have not been considered in the testing of new treatments. There is an initiative called OMERACT (Outcome Measures for Rheumatology Clinical Trials) that aims to improve the way new drugs are assessed. In 2002, OMERACT invited people with rheumatological conditions to the bi-annual conference to give their perspective on what was important to measure. A Patient Perspective workshop was formed and the members have since been responsible for making sure that fatigue is recognised for its huge impact on people with rheumatoid arthritis (RA) and that it is always measured in future clinical trials.

The study

The Patient Perspective group at OMERACT decided that more research was needed about the priorities that people with RA have in relation to treatment. What effects would you like a new drug to have? My study, which was in three parts, has been researching this question over the last two years:

Part 1: Individual interviews with people with RA at the Bristol Royal Infirmary were conducted to produce a list of all the important changes (outcomes) they would like to see as a result of a change in treatment;

Part 2: Group meetings (nominal groups) were held to rank the most important outcomes from the list of 63 generated from the interviews in Part 1;

Part 3: A postal survey was sent out to make a final selection of people’s priorities. The survey was sent out through three NHS rheumatology outpatient departments in the South West and Wales, and to 150 members of NRAS. If you returned one of the surveys – thank you so much!

An overview of the preliminary results

254 surveys were returned. Table 1 (right) shows the characteristics of the respondents.

Three-quarters were women and nearly 90% were between the ages of 41 and 80 years. 16.5% of respondents had three other health conditions to deal with, on top of their RA.

 

 

 

 

Figure 1 (left) shows that 25.6% of people were experiencing severe pain at the time of completing the survey, 27.6% severe fatigue and 33.5% severe functional disability (difficulty in completing everyday tasks).

 From the original 63 treatment outcomes that were important to people with RA (from the interviews), eight were chosen as the most important to measure in the postal survey): pain, avoidance of joint damage, everyday activities, independence, enjoyment of life, mobility (getting around), fatigue and valued activities (Figure 2 - below). These eight were chosen on the basis of importance scores given in the postal survey. In each stage of the study, feeling well was also chosen as an extremely important outcome.

The interview data showed that feeling well depended on a combination of physical, psychological, external, and self-management factors (Figure 2). The external factors were wide-ranging: including people’s financial circumstances and accessing effective health care. The self-management factors included how people had mentally coped with their condition and were practically managing it. The data from the postal survey allowed us to test how important these influences were to the level of overall, or global, well-being. Figure 2 shows how self-management can change the relationship between the symptoms (and impact) of RA and feelings of well-being. For example, for interviewees who felt that they could control their pain or had strategies for coping with it, a reduction in pain was less important than other outcomes.

From the postal survey data it was possible to understand how much the different factors influenced people’s sense of well-being while living with RA. For example, disease symptoms (pain, fatigue and functional disability) contributed the largest influence (39%) on perceptions of global well-being. Self-management factors, such as how well respondents felt they were managing the condition, how RA had effected their ability to lead a normal life, and how confident they were that they could use different coping strategies (self-efficacy), had a smaller effect (13%). Psychological factors, including emotional well-being, and support from friends and family also had a smaller effect (11%). Therefore, the statistical tests showed that the severity of the RA at the time of completing the survey was the biggest factor in determining the level of global well-being.

In summary, respondents to the postal survey emphasised the reduction of symptoms and functional activities as the most important outcomes from treatment. I was most pleased to see enjoyment of life in the Top 8 outcomes! Many of the changes that are most important to people with RA are not currently measured in clinic or in research testing new drugs. We hope that this study will change that.

Thank you to all of the people who took part in the interviews, nominal groups and postal survey. Thank you also to the arthritis research campaign for funding this PhD studentship, to my patient research partner Pam Richards, and to my supervisors: Prof Sarah Hewlett, Prof Michael Calnan and Dr Marianne Morris.