What Matters Most to Lung Cancer Patients? A Qualitative Study in Italy and Belgium to Investigate Patient Preferences

Abstract

Background: The potential value of patient preference studies has been recognized in clinical individual treatment decision-making between clinicians and patients, as well as in upstream drug decision-making. Drug developers, regulators, reimbursement and Health Technology Assessment (HTA) bodies are exploring how the use of patient preference studies could inform drug development, regulatory benefit risk-assessment and reimbursement decisions respectively. Understanding patient preferences may be especially valuable in decisions regarding Non-Small Cell Lung Cancer (NSCLC) treatment options, where a variety of treatment options with different characteristics raise uncertainty about which features are most important to NSCLC patients. As part of the Innovative Medicines Initiative PREFER project, this qualitative study aimed to identify patient-relevant lung cancer treatment characteristics.Methods: This study consisted of a scoping literature review and four focus group discussions, 2 in Italy and 2 in Belgium, with a total of 24 NSCLC patients (Stages III-IV). The focus group discussions sought to identify which treatment characteristics patients find most relevant. The discussions were analyzed thematically using a thematic inductive analysis.Results: Patients highlighted themes reflecting: 1) positive effects or expected gains from treatment such as greater life expectancy and maintenance of daily functioning, 2) negative effects or adverse events related to therapy that negatively impact patients’ daily functioning such as fatigue and 3) uncertainty regarding the duration and type of treatment effects. These overarching themes were consistent among patients from Belgium and Italy, suggesting that treatment aspects related to efficacy and safety as well as the psychological impact of lung cancer treatment are common areas of concern for patients, regardless of cultural background or country.Discussion: Our findings illustrate the value of using qualitative methods with patients to identify preferred treatment characteristics for advanced lung cancer. These could inform a subsequent quantitative preference survey that assesses patient trade-offs regarding treatment options.

Key Determinants of Health-Related Quality of Life Among Advanced Lung Cancer Patients: A Qualitative Study in Belgium and Italy

“…The variety of LC treatments, their characteristics, and the ascendency of patient-centered care require an informed decision-making by stakeholders involved in the medicinal product development, evaluation, and prescription that involves the elicitation and consideration of patient preferences ( Marzorati and Pravettoni, 2017 ). As noted in prior research, patient preferences represent a crucial consideration for both clinical decision-making by healthcare providers, as well as decision-making by pharmaceutical companies, regulators, Health Technology Assessment (HTA) bodies, payers, and across the medicinal product life cycle ( Petrocchi et al, 2021 ; The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH), 2020 ; Janssens et al, 2019a ; Janssens et al, 2019b ; Soekhai et al, 2019 ; van Overbeeke et al, 2019a ; van Overbeeke et al, 2019b ; Whichello et al, 2019 ).…”

“…In addition, the FDA emphasizes that patient preference information is especially valuable in “preference sensitive situations”, i.e., situations where: 1) multiple treatment options exist and there is no option that is clearly superior for all patients, 2) the evidence supporting one option over others is considerably uncertain or variable, and 3) patients’ views about the most important benefits and acceptable risks of a technology vary considerably within a population and may differ from those of healthcare professionals ( US Food and Drug Administration, 2016 ). Decision-making regarding the development, market approval, and reimbursement of new NSCLC treatments is therefore a preference sensitive situation, as such decision-making may depend on the preferences of patients for these diverse treatment characteristics ( Blinman et al, 2010 ; Marzorati and Pravettoni, 2017 ; Petrocchi et al, 2021 ).…”

Patient Preferences for Lung Cancer Treatments: A Study Protocol for a Preference Survey Using Discrete Choice Experiment and Swing Weighting

“…The selection of the attributes and levels of the DCE and the SW arise from a previous qualitative study ( 28 , 29 ). Through focus-group discussions conducted in Belgium and in Italy, patients highlighted themes reflecting positive effects, or expected gains of treatment, and negative effects or adverse events that negatively impacted their daily functioning.…”

“…Through focus-group discussions conducted in Belgium and in Italy, patients highlighted themes reflecting positive effects, or expected gains of treatment, and negative effects or adverse events that negatively impacted their daily functioning. Twenty-one themes emerged from those discussions, mainly consistent among patients from Belgium and Italy ( 29 ).…”

“…Results were used to derive attributes and attribute levels. From the 21 themes that emerged during the first round of discussions with patients ( 29 ), participants selected and rank-ordered the most important ones. The four most important positive characteristics were greater life expectancy, decrease in cancer growth, cancer remission, and the maintenance of daily functioning, and eight most important negative characteristics were severity of skin problems, nausea, serious infections, hair loss, and infusion reactions, gravity of fatigue, probability of renal failure, and probability of cognitive limitation.…”