ESH CML/MPN 2025 | The importance of timely reporting of patient-reported outcomes in clinical trials

This is a very important aspect because it’s not uncommon to see many trials that, for example, you assess quality of life as a secondary point of your trial. And of course, it makes sense that you, of course, publish the first results on the efficacy and safety of the drug. But then you have to consider also to give enough space to describe in terms of methodology, in terms of outcome reporting, or what you have done in terms of quality of assessment. Because this has to be done in a rigorous way. 

So there are a number of aspects that you need to document about your assessment. You need to describe the way you administer the questionnaire, the amount of missing data, what kind of statistical approaches you have used. So if you do not report all this information, I mean, it is very unlikely that the quality of life results will have an impact in real-world practice because you don’t know the robustness based on which the information that you collected is actually robust. 

So you need to publish this with extensive details, and you also have to do this in a timely manner. Because, for example, what might happen is that you publish the efficacy results this year, and then you might not publish the quality of life results, or you might publish the quality of life results after, you know, six years, five years, you know. So it is important that you need to publish this data in a timely manner. So people can actually understand the overall results, both in terms of efficacy and also the results from the patient perspective. 

With regard to the methodology, there are important international guidelines with regard to what kind of aspect you need to report in the trial publication. And I’m referring to specifically to the CONSORT patient-reported outcome guidance. So, we know very well what aspects are to be documented in a trial report. And this is very important that investigators adhere with this high-quality recommendation. 

In terms of timely reporting, this actually really depends on the study team. There might be a situation in which the study team might publish the results after one year or in conjunction with the clinical paper, or they might report this as an appendix to the efficacy results. So there are different options. As long as the scientific community is provided with this data in a timely manner, it’s good. 

And I have to say that we, in some areas, for example, we are talking about specific, let’s say, hematology, we have seen outstanding progress. And, for example, what’s happening in MPN, it is pretty outstanding. I mean, the example that we have seen of the recent approval of momelotinib, which used the patient-reported outcomes as a primary point, is an excellent example of how patient-reported outcomes can actually be helpful in clinical research.

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