Abstract
Purpose
Successful patient-focused drug development involves selecting and measuring outcomes in clinical trials that are important to patients. The U.S. Food & Drug Administration’s definition of clinical benefit includes how patients feel, function, or survive. Patients are considered the experts in describing how they feel and function. In cancer trials, patient-reported measures of physical function provide insight into how patients function at baseline, benefit from the interventions being studied, and the impact of treatment side effects. We conducted a qualitative study with adults diagnosed with cancer to describe facets of physical function from their perspective and to identify which facets are most important to this patient population.
Methods
Using concept elicitation and cognitive interviewing techniques, we conducted semi-structured interviews with 72 adults ≥ 22 years of age with cancer who received treatment with an anticancer drug or biologic within six months of the interview. We selected participants using purposive sampling with the aim to elicit diverse experiences regarding how they may interpret and respond to questions related to physical function. Participants were presented with patient-reported outcome (PRO) items representative of PRO measures used in cancer and general populations.
Results
Five facets of how physical function relates to activities were defined from the patient perspective: ability, difficulty, limitation, satisfaction, and completion. More than half of the participants indicated that ability was the most important facet of physical function. The next most important were satisfaction (18.3%), limitation (14.1%), difficulty (5.6%), and completion (2.8%).
Conclusion
This study demonstrates that we must be more specific about the facets of physical function that we set out to assess when we use PRO measures to describe the patient experience. These results have implications for the specificity of physical function facets when measured in cancer clinical trials.
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Data availability
Summary reports are available upon request to the corresponding author.
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Acknowledgements
The authors would like to acknowledge the patients who participated in this study, who provided important insight into the patient experience with physical function. The authors would also like to thank Liz Wing for medical editing support.
Funding
This work was supported by the U.S. Food & Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) Oncology Center of Excellence (Contract 75F40120C00069), and as part of a financial assistance award funded by FDA/HHS. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government.
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The corresponding author (TC) confirms that she had full access to all the data in the study and had final responsibility for the decision to submit for publication. Each author has contributed significantly to, and is willing to take public responsibility for, the following aspects of the study. TC: conceptualization, funding acquisition, methodology, interpretation of results, writing original draft; KP: data collection, analysis, interpretation of results, writing–review and editing; AH: data collection, analysis, interpretation of results, writing–review and editing; RF: data collection, analysis, interpretation of results, writing–review and editing; DMH: data collection, analysis, interpretation of results, writing–review and editing; CA: data collection, writing–review and editing; SLD: facilitated data collection, interpretation of results, writing–review and editing; CRG: facilitated data collection, interpretation of results, writing–review and editing; TWL: facilitated data collection, interpretation of results, writing–review and editing; VB: interpretation of results, writing–review and editing; EGH: methodology, interpretation of results, writing—review and editing; BBR: methodology, interpretation of results, writing–review and editing; KPW: conceptualization, methodology, interpretation of results, writing–review and editing.
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TC has research funding from Merck and Pfizer, a prior consulting agreement with Regenxbio, and received a speaking honorarium from ISPOR. CA was a fellow at Duke (2021–2023) funded by AstraZeneca, and is now an employee at AstraZeneca (2023-present).CRG is a consultant for Stryker and Medtronic. BBR had a consulting role with Novartis. TWL has received honoraria for consulting/advisory boards from AbbVie, Agilix, Agios/Servier, Apellis, Astellas, AstraZeneca, BlueNote, BMS/Celgene, Genentech, GSK, Lilly, Meter Health, Novartis, and Pfizer; speaking related honoraria from AbbVie, Agios, Astellas, BMS/Celgene, Incyte, and Rigel; equity interest in Dosentrx (stock options in a privately-held company); royalties from UpToDate; research funding from the AbbVie, American Cancer Society, AstraZeneca, BMS, Deverra Therapeutics, Duke University, GSK, Jazz Pharmaceuticals, and Seattle Genetics. TWL is a Scholar in Clinical Research of the Leukemia & Lymphoma Society. All other authors have no disclosures.
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Coles, T., Plyler, K., Hernandez, A. et al. What facets of physical function are most important to adults diagnosed with cancer?. Qual Life Res (2024). https://doi.org/10.1007/s11136-024-03654-8
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DOI: https://doi.org/10.1007/s11136-024-03654-8