Analysis of Patient Preferences in Lung Cancer – Estimating Acceptable Tradeoffs Between Treatment Benefit and Side Effects
Received 24 October 2019
Accepted for publication 28 April 2020
Published 3 June 2020 Volume 2020:14 Pages 927—937
Checked for plagiarism Yes
Review by Single anonymous peer review
Peer reviewer comments 2
Editor who approved publication: Dr Johnny Chen
Ellen M Janssen,1 Sydney M Dy,2 Alexa S Meara,3 Peter J Kneuertz,4 Carolyn J Presley,5 John FP Bridges6,7
1Center for Medical Technology Policy, Baltimore, MD, USA; 2Department of Health Policy and Management, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA; 3Department of Internal Medicine Division Of Rheumatology, The Ohio State University, College of Medicine, Columbus, OH, USA; 4Thoracic Surgery Division, Department of Surgery, The Ohio State University Wexner Medical Center, Columbus, OH, USA; 5Division of Medical Oncology, Department of Internal Medicine, The Ohio State University College of Medicine, Columbus, OH, USA; 6Department of Biomedical Informatics, The Ohio State University College of Medicine, Columbus, OH, USA; 7Department of Surgery, The Ohio State University College of Medicine, Columbus, OH, USA
Correspondence: Ellen M Janssen
Research Director, Center for Medical Technology Policy, 401 East Pratt Street, Suite 631, Baltimore, MD 21202, USA
Tel +1 443-222-8775
Objective: Increased treatment options and longer survival for lung cancer have generated increased interest in patient preferences. Previous studies of patient preferences in lung cancer have not fully explored preference heterogeneity. We demonstrate a method to explore preference heterogeneity in the willingness of patients with lung cancer and caregivers to trade progression-free survival (PFS) with side effects.
Patients and Methods: Patients and caregivers attending a national lung cancer meeting completed a discrete-choice experiment (DCE) designed through a collaboration with patients. Participants answered 13 choice tasks described across PFS, short-term side effects, and four long-term side effects. Side effects were coded as a one-level change in severity (none-mild, mild-moderate, or moderate-severe). A mixed logit model in willingness-to-pay space estimated preference heterogeneity in acceptable tradeoffs (time equivalents) between PFS and side effects. The study was reported following quality indicators from the United States Food and Drug Administration’s patient preference guidance.
Results: A total of 87 patients and 24 caregivers participated in the DCE. Participants would trade 3.7 month PFS (95% CI (CI): 3.3– 4.1) for less severe functional long-term treatment side effects, 2.3 months for less severe physical long-term effects (CI: 1.9– 2.8) and cognitive long-term effects (CI: 1.8– 2.8), 0.9 months (CI: 0.4– 1.4) for less severe emotional long-term effects, and 1.8 months (CI: 1.4– 2.3) for less severe short-term side effects. Most participants (90%) would accept treatment with more severe functional long-term effects for 8.4 additional month PFS.
Conclusion: Participants would trade PFS for changes in short-term side effects and long-term side effects, although preference heterogeneity existed. Lung cancer treatments that offer less PFS but also less severe side effects might be acceptable to some patients.
Keywords: non-small cell lung cancer, patient preferences, discrete choice experiment, heterogeneity, long-term side effects
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