Liana Fraenkel, MD, MPH, study author and primary investigator, Professor Adjunct, Yale University School of Medicine, Rheumatology, Berkshire Health Systems, and W. Benjamin Nowell, PhD, study author, Director of Patient-Centered Research at CreakyJoints, and principal investigator of ArthritisPower, discuss a study that evaluated the use of the Overall Patient EXperience measure (OPEX) to compare the desirability of treatments for patients with rheumatoid arthritis (RA). The results of Drs. Fraenkel and Nowell’s study were published in PLOS One.

DocWire News: What prompted you to undertake this study?

Dr. Fraenkel: It was hearing from patients that really prompted us to think about this study. I’ve been very interested in shared decision-making and helping patients make complex medical decisions for most of my career. And when we do that, we often have to compare different treatment options.

And the best data we have for that are from randomized controlled trials. But randomized controlled trials present benefits and risks separately. So there’s a table telling you how well people did. And then there’s another table telling you the percentage of people who develop specific side effects on these treatments.

My patients, basically, when they’re thinking about two treatment options, they kind of want to know, “What’s my quality of life going to be overall on this medication? How will I feel on medication A compared to medication B?” And there are really no outcome measures that report the global patient experience on a medication. And so that’s what prompted us to try to figure out how to measure that.

DocWire News: What were the key takeaways from the study?

Dr. Fraenkel: The key takeaways are that one, patients can compare a lot of different side effects. So, right now the ways that side effects are classified are fairly complicated and they list a lot of different side effects, some which are meaningful to patients and some which are not. And the first part of this study was to figure out, “Can we group and clump side effects together that have similar impact on a patient’s quality of life so that they can use that information more effectively in their decision-making?” And we did find that. So we created an initial hierarchy of different classes of side effects, ranging from no side effects to really severe side effects with long lasting consequences. And so, not surprisingly, things that cause minor discomfort and are easily reversible tend to cluster together. Things that potentially cause some irreversible changes or that require more risky treatment or a hospitalization, obviously running towards the more severe end. So that was the initial step. And then we combined that with benefits to create global outcomes.

DocWire News: Did any of the study’s findings come as a surprise to you?

Dr. Nowell: I think what was most surprising about the study is that this is such an important area for patients and that this is a relatively new way of looking at the results of trials. So patients, we know anecdotally … I mean, I’m situated with a Patient Advocacy Organization, Global Healthy Living Foundation, and CreakyJoints. And we know that patients will talk to their doctor. Doctors may recommend a treatment or a change in treatment. And patients, what they want to do is hear from other patients what that experience was on a particular treatment. And so ultimately the information that can be generated from this kind of an OPEX to measure that would be developed here is that you can get a lot of information from a lot of different patients about what the expected experience is on a particular treatment. And you can see that by the different percentages, having different types of overall experience on a particular treatment. So for me, that was what was most surprising, was that something that’s so crucial, so fundamental to patient decision making has not been fully fleshed out or developed yet.

Dr. Fraenkel: We really expected that patients would strongly value no side effects. That would really trump a lot. And we were absolutely wrong. Patients, basically clumped no side effects together with mild side effects. Even in some cases, some moderate side effects. And when we went back and we interviewed patients, “Why?” it was basically, they assume that all medications will have some risk of side effects and they expect that and they can deal with that. So for them, the lowest category, the most mild category is mild reversible side effects that are not going to cause me any significant harm or any long lasting harm. And those were all clumped together. So patients really don’t expect a medication to have no side effects and they think they’re very comfortable clumping all those together, which was a surprise to us actually.

DocWire News: What limitations did the study have?

Dr. Nowell: So, a couple of limtiations were one, that we, in the ultimate measure, the OPEX measure, is a six-level hierarchy. So of course when you’re only limiting things to six levels, you may miss some of the nuances. So, that’s one potential limitation. The other limitation was that in our study, we focused specifically on rheumatoid arthritis (RA). So this measure right now can only be applied to RA. So, I think obviously there’s only so much it can tell us, and only for that specific group of patients,

DocWire News: Do you have any future research plans pertaining to this area?

Dr. Nowell: Well, to address the limitation I just mentioned. It would be great if we could explore this same process in other diseases, other rheumatic and musculoskeletal diseases, so that we can develop a similar measure, understand also the overall patient experience for different diseases on the treatments that are relevant for those diseases. And I think at this time we don’t have any specific future plans scheduled, but we would like to incorporate this into other studies.

DocWire News: Any final comments you’d like to share?

Dr. Nowell: Only that this is a very important step forward in terms of shared decision making, where patients are able to really understand the decisions that they’re making about their treatment and have information that’s useful to them, that’s relevant to them, that’s meaningful to them to be able to, in a sense, hear from what other patients are experiencing on particular treatments so that they can actually make a reasonable comparison that is understandable to the patient.

Dr. Fraenkel: This is a good example of how important it is for us to listen to our patients to measure what’s important to them. It’s sometimes outside of the framework of things we’re used to measuring, but this really pushed us to try to figure out sort of a new approach to measuring outcomes in randomized trials that we wouldn’t have thought about really had we not heard our patients.

Funding for the project was provided by the Rheumatology Research Foundation Innovative Research Award.