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ESH CML/MPN 2025 | The importance of integrating quality of life assessment into clinical trials and routine practice
Fabio Efficace, PhD, Italian Group for Adult Hematologic Diseases (GIMEMA) Data Center and Health Outcomes Research Unit, Rome, Italy, discusses the importance of assessing quality of life (QoL) in clinical trials and routine practice, highlighting the need for patient-reported outcomes (PROs) to better understand treatment effectiveness and inform treatment decisions. Dr Efficace notes that quality of life assessment can provide unique information on the impact of treatments on patients’ lives and that understanding symptom burden is crucial for optimizing adherence to therapy. This interview took place at the European School of Haematology (ESH) 4th How to Diagnose and Treat: CML/MPN meeting in Vienna, Austria.
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Transcript
It was a talk about the importance of assessing quality of life in clinical trials, and also the importance of assessing quality of life and symptoms, which we call patient-reported outcomes, in routine practice. So, these are two different scenarios.
So, one is that of using quality of life as a treatment outcome measure...
It was a talk about the importance of assessing quality of life in clinical trials, and also the importance of assessing quality of life and symptoms, which we call patient-reported outcomes, in routine practice. So, these are two different scenarios.
So, one is that of using quality of life as a treatment outcome measure. So when we assess the efficacy of a new drug, we also want to assess the impact on patients’ quality of life. And the only way of doing this is just asking patients themselves by using validated questionnaires. And we can actually get unique information, and this type of information is crucial to better understand overall treatment effectiveness and to make more informed treatment decisions.
The other area where we actually use quality of life might be in routine practice settings. So, on a regular basis, we want to assess the actual symptom burden experienced by patients. Because, for example, with regard to oral chronic therapies, this might be associated with adherence to therapy, and adherence to therapy is so important to maximize clinical effectiveness. So there is a complex interplay between quality of life, adherence to therapy, and let’s say efficacy. So it is important that we understand better what is the burden of the treatment from the patient perspective. And of course, we have to do this in a robust way from a scientific perspective.
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Disclosures
Consultancy/advisory role: AbbVie, Incyte, Novartis, JAZZ Pharmaceuticals; Research support (institution): Daiichi Sankyo. All disclosures are outside the submitted work.
