CAR-T Meeting 2024 | An insight into the myeloma patient perspective on PROs in CAR-T clinical trials

Myeloma Patients Europe – we support, around 50 patient groups in 30 different countries, and we receive a lot of questions around CAR-T, what it’s like to get CAR-T, what are the risks, who benefits from it, and what is the experience like? So, 3 years ago, we initiated a project, and we brought patients to focus group discussions to understand the experience they had with CAR-T. And one of the projects we had, from the discussions we had with those patients, was to understand their perspectives on patient-reported outcome collection tools that are being used in clinical trials, CAR-T clinical trials in particular.

So we had ten patients coming to the discussions in separate groups that we handled in different languages and we asked them to complete three different PRO collection tools and to tell us about their feedback around whether it was reflecting their CAR-T experience or not and what could be missing from those tools. So we asked them how long it took to complete them, if it was appropriate, if the questions were good, what kind of themes were missing, and what would they recommend to improve them in the future. Because currently, there is no CAR-T-specific PRO collection tool, and we want to make sure that the patient perspective is heard when those will be designed in the future or are currently being designed.

So it takes them around 20 minutes to complete the forms, and there’s some redundancy between the different forms. So there should be work taken on improving the flow to make sure that they don’t answer the same question several times and it also adapts to their previous answers. So if you’re able to take a long walk, of course, you’re also able to take a short walk, so maybe that question is not needed. That’s just one example.

And in terms of missing themes, people really want to know what the real impact on their lives is going to be. So, missing themes were around isolation, anxiety, but also, their fears too. Questions like, do you fear death? Well, maybe that’s not the right question. Maybe the right question is do you think a lot about death? Because someone can think about it without fearing it so much because they have accepted their diagnosis and that their life span will be shorter because of that. Around the themes that were missing, patients reported that unexpected life changes, but also isolation and boredom (especially during the hospitalization time) were missing from the questionnaires; and things about sexual functions, about not the fear of death, but whether they think about it, and also their ability to plan for the future. So it’s about not being always in the moment and having to deal with 100% myeloma-related things, but also being able to plan for the future and have long-term plans and be able to have a normal life. So that’s the things that that people reported, among other things.

And I think for us, our call to action is to make sure that the patient perspective is included when those future tools will be designed or are currently being designed. And that the themes that we identified as being missing and important to patients are included in those questionnaires. That the data that is being collected is actually being published because very often in clinical trials, we don’t see the results coming out or it’s really partial results, the ones maybe that looks good. So we really want to make sure that everything is available because, at the end of the day, this are the kind of questions that really help the patients make their treatment decision based on an informed way to assess how it’s going to affect their life.

So publish those results, but also make sure those results are included in the lay summaries of clinical trial results. Because it’s now mandatory to publish summaries and lay summaries in particular of clinical trials. So we want those PRO data to be included and make sure that, at the clinical trial site, the ‘why’ of why we are collecting that data is clearly explained to the patients because currently, the response rate is not so good. But if people were taking the time to explain to patients why we are collecting the data, how we’re going to use it, and that we are going to actually report on it, that would really help patients understand why they should care about this as much as they should care about the clinical efficacy of the medicines, because at the end of the day, they care about the quantity of life, but also about the quality of life.