Using qualitative interviews to identify patient-reported clinical trial endpoints and analyses that are the most meaningful to patients with advanced breast cancer

• Published in: PloS one Vol. 18; no. 1; p. e0280259
• Main Authors: Flood, Emuella, Krasnow, Anna, Orbegoso, Cecilia, Karantzoulis, Stella, Bailey, Julie, Bayet, Solène, Elghouayel, Arthur, Foxley, Andrew, Sommavilla, Roberto, Schiavon, Gaia
• Format: Journal Article
• Language: English
• Published: United States Public Library of Science 17-01-2023; Public Library of Science (PLoS)
• Subjects: Adult; Antineoplastic Combined Chemotherapy Protocols - therapeutic use; Biology and Life Sciences; Breast cancer; Breast Neoplasms - drug therapy; Breast Neoplasms - metabolism; Cancer therapies; Chemotherapy; Clinical outcomes; Clinical trials; Data collection; Decision making; Drug therapy; Endocrine therapy; Epidermal growth factor; ErbB-2 protein; Female; Graphical representations; Growth factors; Humans; Interviewing; Interviews; Mammography; Medical prognosis; Medical research; Medicine and Health Sciences; Metastasis; Methods; Outcome and process assessment (Health Care); Pain; Patient Reported Outcome Measures; Patients; Qualitative analysis; Qualitative research; Quality of Life; Receptor, ErbB-2 - metabolism; Receptors; Research and Analysis Methods; Research methodology; Therapy; Toxicity; Treatment Outcome; Triple Negative Breast Neoplasms - drug therapy; United States; United Kingdom > UK; United States > US
• ISSN: 1932-6203; 1932-6203
• DOI: 10.1371/journal.pone.0280259

Designing clinical trials with the emphasis on the patient-centered approach and focusing on clinical outcomes that are meaningful to patients is viewed as a priority by drug developers, regulatory agencies, payers, clinicians, and patients. This study aimed to capture information on clinical trial endpoints that would be most important and relevant for patients with advanced breast cancer, based on patient-reported outcomes.
Patients with either advanced triple-negative breast cancer [TNBC] and a maximum of two lines of systemic therapy or hormone receptor-positive/human epidermal growth factor receptor 2-negative [HR+/HER2-] breast cancer and a maximum of three lines of systemic therapy, participated in semi-structured concept elicitation interviews. Concept saturation was assessed. A sign, symptom, or impact was defined as "salient" if mentioned by ≥ 60% of participants, with an average bother rating of ≥ 5 (0-10 Scale). Participants were also asked about treatment priorities and to evaluate hypothetical scenarios showing different health-related functioning and quality-of-life treatment outcomes, using graphical representations.
Thirty-two participants (97% women; aged 29+ years) with TNBC (n = 17) or HR+/HER2- breast cancer (n = 15) provided generally similar reports on symptom experience, with fatigue and pain being most salient, though importance of certain treatment-related symptoms varied between the two groups. Patients reported consistent perspectives on the importance of treatment outcomes: when considering a new treatment, they prioritized efficacy of the therapy, acceptable tolerability, stability, predictability of symptoms over time, and the duration of preserved health-related quality of life and physical functioning. The meaningful difference in preserved physical functioning was 2-3 months for 46% of participants with TNBC, whereas for most participants with HR+/HER2- breast cancer it started from 6-7 months. Both groups of participants found it easier to accept some toxicity at the beginning of therapy if it was followed by improvement, as opposed to improvement followed by deterioration.
The results may help to inform the design of patient-centered clinical trials, to interpret health-related quality of life and/or patient-reported outcomes, and to optimize care for patients with advanced breast cancer.