The Importance of Patient-Reported Outcome Measures in Acne and Rosacea Clinical Trials

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In this interview, Dr John Barbieri discusses the importance of including patient-reported outcome measures (PROMs) in randomized clinical trials (RCTs) for skin conditions such as acne and rosacea. Dr Barbieri’s research is focused on conducting innovative clinical trials and epidemiology studies to identify the best approaches to care for patients with acne. He also focuses on exploring the role of patient-reported outcomes to ensure the patient’s voice and perspectives are captured.

The Dermatologist: Your findings suggest that PROMs were often not included in acne and rosacea RCTs and were rarely included as a primary outcome measure. Can you expand on this concept?

Dr Barbieri: When caring for patients with skin conditions such as acne and rosacea, there can be my assessment as the clinician about how they are doing. But ultimately, what matters for something that is not a fatal disease directly is how the patient feels about their lived experience of having acne or rosacea. If we really want to capture that patient’s experience, their own voice, their lived experience of having acne or rosacea, we want to be using things like patient-reported outcomes to get that information.

We have studied acne trials to see how often we are capturing that patient-reported outcome information. How often are we getting the patient’s voice into our key study outcomes vs just lesion counts and investigator global assessments? What we found is that many trials do not look at patient-reported outcomes. Only about half of them include a patient-reported outcome at all. Very few of them, only a couple percent, include a patient-reported outcome as the primary outcome. And often when they do use patient-reported outcomes, they tend to use more generic measures instead of dermatology-specific or disease-specific measures.

It is important to look at how well patient-reported outcomes perform. Those that are more targeted toward the population of interest tend to work better. They tend to have better content validity. Do they align with the things that the patients care about, that are relevant? And they tend to be more comprehensive covering the breadth of the lived experience of those skin conditions.

Our work highlights that even though patient-reported outcomes can be an important aspect of capturing the patient’s voice in clinical trials, they are often not recorded. And when we do use them, we probably are not using the best ones that we could. It is a real opportunity for us to create better data to guide our clinical decision-making.

When we ask clinicians how they decide to treat patients based on psoriasis trials, they say the patient-reported outcome data are just as important as the clinical outcomes. And here we are identifying that in a lot of our studies we are not capturing this critical input to decision-making.

The Dermatologist: What is the importance of capturing the patient perspective in RCTs on acne and rosacea?

Dr Barbieri: It is important to understand the patient’s lived experience and how they are benefiting from the treatments. You can imagine a treatment that makes your skin clear, but it comes with a lot of adverse reactions, or maybe it does not clear the skin in a way that is relevant to patients. Those patient-reported outcomes are going to be able to identify the nuance that if you are just counting pimples or doing an investigator global assessment, you might miss. It really adds color to our understanding of how well our treatments work and why they are so important to include in our clinical trials so we can understand the patient perspective.

The Dermatologist: How does increasing the use of PROMs in RCTs affect decision-making for dermatologists?

Dr Barbieri: I think if we increase the use of patient-reported outcomes in our clinical trials, then we will have those data about how well a treatment works in terms of improving people’s quality of life. That is ultimately what our goal is, we are trying to help improve people’s lived experience with their skin disease. We are trying to improve their quality of life. Having that direct data of how well treatment A or treatment B is doing in terms of improving the patient’s quality of life can be valuable.

In addition, if we want to think about things like cost effectiveness, these are critical input data. A fundamental input for doing cost effectiveness work is being able to estimate quality-adjusted life years that are gained from a treatment. To do that, we need to use health utility measures, and those are a form of patient-reported outcomes. So, if we want to be able to understand which treatments are providing the highest value to our patients, having patient-reported outcome data is a helpful input that can help in-form our decision-making to make sure we are doing the best we can for our patients with acne and rosacea.

The Dermatologist: What future research is needed to understand the use of PROMs in RCTs on acne and rosacea?

Dr Barbieri: There are a couple of different areas where we need more information. A helpful property of patient-reported outcomes is that health utility measures can be used to do cost effectiveness and health economic research. Unfortunately, the measures that we have available to us do not work well for skin disease. Some of the most common measures that the EQ-5D and SF-6D ask about are “Can you walk upstairs?” “How is your hearing?” Things that are just not necessarily relevant to patients with skin disease. Then when you study these measures, they do not work well. They have poor content validity. They do not align with things that are relevant and important and comprehensive to our patients’ lived experience. They do not perform, or they are not responsive. If you have a treatment that works well, on these outcomes it seems like nothing is happening because they are not measuring what matters to patients with skin disease. There is a real unmet need to develop patient-reported outcomes, and health utility measures specifically, that we can use for these health economic evaluations in dermatology.

In addition, among our quality-of-life measures, we have not fully validated a lot of them. We are missing data on key properties, such as responsiveness and content validity, where it is crucial to know that these measures perform the way we would want them to in a clinical trial. There is a real unmet need to conduct more studies to understand the measurement properties of our existing patient-reported outcomes and to develop new patient-reported outcomes to fill in gaps where we identify them.

The Dermatologist: Is there anything else you would like to share with your colleagues about PROMS in acne and rosacea RCTs?

Dr Barbieri: I think, in general, trying to use patient-reported outcomes more in clinical trials, other clinical studies, and in our clin- ical practice is an opportunity to capture patients’ voices so we can understand what treatments are working best for them and what treatments may not be meeting their needs, and so we can keep track of how successful they are in our care for these patients.

To me, patient-reported outcomes can almost be seen like the vital sign for skin disease. We do not have a blood pressure or a heart rate or something like that to measure, but our patient-reported outcomes really tell us how our patients are doing at a fundamental level with respect to their skin disease. I think there is a great opportunity to try to incorporate patient-reported outcomes more in our research and in our clinical care and dermatology.

Reference

Barbieri J. Use of patient-reported outcomes in acne vulgaris and rosacea clinical trials from 2011 to 2021: a systematic review. JAMA Dermatol. 2022;158(12):1419-1428. doi:10.1001/jamadermatol.2022.3911