Benjamin Nowell, PhD: The Importance of Patient-Reported Outcomes and Experiences


Rheumatology Network sat down for an interview with Benjamin Nowell, PhD, in partnership with CreakyJoints, to discuss his 2 most recent studies. Nowell is the Director of Patient-Centered Research at CreakyJoints, part of the Global Healthy Living Foundation.

Rheumatology Network sat down for an interview with Benjamin Nowell, PhD, in partnership with CreakyJoints, to discuss his 2 most recent studies titled, “OPEX: Development of a novel overall patient experience measure to facilitate interpretation of comparison effectiveness studies” and “Which patient-reported outcomes do rheumatology patients find important to track digitally?” Nowell is the Director of Patient-Centered Research at CreakyJoints, part of the Global Healthy Living Foundation. We discuss investigators’ initial interest in tracking outcomes and experience and how tracking these symptoms is beneficial for patients. It should be noted that the OPEX study was funded by the Rheumatology Research Foundation and the Patient reported outcomes study was funded by Eli Lilly and Company, which is a great example of how the pharmaceutical industry is interested in supporting studies that help to understand what is important to patients.

Rheumatology Network: Hi, Dr. Nowell, thank you for taking the time to speak with me today.

Benjamin Nowell, PhD: Happy to be here. Thanks for having me.

RN: In regards to the study titled "Which patient-reported outcomes do rheumatology patients find important to track digitally," what was your initial interest in tracking this behavior?

BN: So I lead a registry called ArthritisPower. And it's a patient-powered research network. And one of the underlying principles of the network is that we're conducting patient-centered outcomes research. And essentially, the principle of patient-centered outcomes research is that you're answering research questions that matter to patients. And doing so in a way that helps patients make healthcare decisions or decisions about their health care. So in this study, because identifying which symptoms or which patient reported outcomes are most relevant or meaningful to patients, is so important for their own healthcare decision-making. This was a way of contributing to our broader mission of conducting patient-centered outcomes research. Since the idea is that integration, the outcomes that they find most important that they prioritize, integrating those into research and into clinical practice in rheumatology is going to improve, at least the theory goes, improve patient's use of this kind of evidence from research into their own health decisions, and ultimately help them better manage their disease and achieve their own goals with their health care.

RN: Were you surprised by the results of this study?

BN: Well, I think some of the results that I might highlight; So essentially, what we did is we tracked patients over several months. So we asked them to select from a sort of library of patient-reported outcome measures, or symptoms, really symptom measures, which ones they wanted to track. And then we asked them again for another 3 months, once a month. So they could either continue to track the same symptoms or choose to track something different, or stop, you know, tracking measures, and they could select up to a grand total of 10. But they needed to select at least 3 that they wanted to track. A few things were actually surprising. One is that on average, patients chose to track 7 measures, which to me sounds like a lot. But that was that was what ended up being the mean number of measures that people were tracking. We also found that some things that were sort of expected, pain. There we had a few different measures for pain: 2 different ones for the PROMIS pain interference, 1 PROMIS pain intensity. Together, pain made up the most common symptom that was chosen to track by patients. Fatigue was the single measure that was selected most often across all participants. So, pain and fatigue are important. We've seen that, you know, supported by other studies as well. And then physical function PROMISE physical function was also very important, as was the duration of morning joint stiffness. So those were, you know, not all that surprising, but it just kind of reinforced some of the stuff we already knew. What was perhaps surprising was that at least 1 of the PROMIS mental health domain instruments, so basically their measures of emotional distress. Those were also very commonly selected. So at least one of those measures was selected by more than 80% of participants. So we know that those kinds of symptoms matter a lot for patients with rheumatic diseases. Well, from this study there's some evidence of that. It was also a little bit surprising that we, we included an option to select a measure of sexual function, sexual satisfaction. That was almost never chosen, almost no one chose that 1. Although we had, you know, intentionally put that on there, because we heard anecdotally that that was important for patients. But in the end, at the end of the day, not really many people chose to track that over time. The other thing that was a little bit surprising, kind of interesting, I think, is that just for patients with rheumatoid arthritis in the study, of which there were 52, so not a huge number, but 52, who selected, who basically participated throughout the whole duration of the study went from almost 80% of them choosing to track the Composite Measure, the OMERACT, as a flare measure, dropping to only about 15% tracking at the at the final month. That's a huge decline in the number of people selecting, so as I mentioned before, people could you know, at the every month mark, they could choose to select new measures, they could stop tracking other measures. So, this was one that clearly a lot of people chose initially to track flare, but most of them stopped tracking. By the end of the of the study, we speculate that that has to do with the fact that it includes several different kinds of a combination of symptoms, flare is in somewhat notoriously ill-defined. Although the OMERACT flare measure does a nice job of trying to quantify it. But yet patients were just more interested in in tracking, it seems individual measures like fatigue, you know, pain and physical function.

RN: Moving on to the OPEX study, why were you interested in tracking patients overall experience on treatment?

BN: Well, this is also intended to try to address a gap from a patient-centered outcomes research perspective. So typically, the results of randomized controlled trials. For treatments for rheumatic disease, they're usually reported as either the average improvement scores, or the percentage of patients attaining a certain response in each treatment arm. And then the number of participants in trials who experienced some kind of an adverse event, side effects specific adverse event that's reported separately from the number of patients reporting improvements. But in reality, you know, this doesn't really capture what's most important to patients. In other words, like their overall experience on treatment. So, when patients are making decisions about what treatment to start, with their doctor, in the words of a patient, to quote someone directly, who was involved in the study, “patients have no way to determine the potential net benefit for a given treatment, much less to compare across treatments.” In other words, you may experience some improvement on treatment, but patients are trying to balance that with the potential adverse event or side effect they might experience on that same treatment. So there have been other composite measures developed to try to capture this, but measures reporting the percentage of patients with those varying overall experiences have yet to be developed. But yet, that's the kind of information that patients would find really helpful to know about, because typically, what I find, at least anecdotally, with our CreakyJoints patient community and the ArthritisPower patient registry, is that patients will talk to their doctor, but then immediately, they'll want to talk to other patients and then say, you know, have you been on this treatment? What was your experience like? And this kind of some kind of a measure, like be developed here, the overall patient experience measure OPEX is a way of capturing and consolidating that information from a lot of different patients all at once, so that patients can see and compare the percentage of patients who had an overall net benefit on that treatment.

RN: Are you planning on doing any further research on this topic?

BN: We would definitely like to incorporate it into studies, but we don't have any specific plans at this time. Again, it would be useful to explore the same process that we used in this study, to other diseases. Because for this article, it describes, essentially a series of studies. So, we first had to use trajectory mapping to develop a hierarchy of the adverse events, that was the first thing we had to do to you know, from kind of the most, you know, sort of mild or no side effect at all to what's severe and irreversible. So, we first had to get that hierarchy from prospective patients developed, then we combine that with the different levels of benefit, you know, from major improvements, some improvement, or little no improvement, little or no improvement on a medication to create that sixth level hierarchy, that that makes up OPEX. And then finally, as a final step, we then applied the measure to that clinical trial as I described. So, it would be very useful to be able to do that with other groups of patients who have other conditions besides RA.

RN: Do you have anything to add before we wrap up?

BN: I don't think so. I think just that I think personally, I think this is a really exciting project in terms of advancing shared decision making, in terms of being able to communicate the results of trials that you know, that have a lot of numbers and percentages, and P values, that are often confusing to patients, you know, at a minimum or at worst, are scary or frightening to patients because they describe adverse events, even if they're extremely rare. It still can be off putting to people. So I think this is a really important area of work to be in in terms of research because it helps communicate things in a way that's more understandable to patients, and gives them information that really does help them ultimately make a decision about their own care and their own treatment.

RN: Well, Dr. Nowell, thank you so much for taking the time to speak with me today. I appreciate it.


Nowell WB, Gavigan K, Kannowski CL, et al. Which patient-reported outcomes do rheumatology patients find important to track digitally? A real-world longitudinal study in ArthritisPower. Arthritis Res Ther. 2021;23(1):53. Published 2021 Feb 10. doi:10.1186/s13075-021-02430-0

Fraenkel L, Wei Z, Ramsey C, et al. OPEX: Development of a novel overall patient experience measure to facilitate interpretation of comparison effectiveness studies. PLoS One. 2021;16(1):e0245598. Published 2021 Jan 29. doi:10.1371/journal.pone.0245598

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