Eliciting Patients' and Clinicians' Preferences on Adjuvant Treatment for HR+/HER2- Early Breast Cancer: Design of a Probabilistic Threshold Technique in Italy
Author(s)
Beatrice Canali, MSc1, Riccardo Mercati, MSc1, Francesca Fiorentino, PhD1, Rossana Berardi, MD2, Grazia Arpino, MD, PhD3, Alberto Zambelli, MD4, Matteo Basilio Suter, MD5, Diletta Valsecchi, MSc5, Chiara Vassallo, MSc1.
1IQVIA SOLUTIONS ITALY S.r.l., Milan, Italy, 2Department of Medical Oncology, Università Politecnica delle Marche, AOU delle Marche, Ancona, Italy, 3Department of Clinical Medicine and Surgery, University of Naples Federico II, Naples, Italy, 4Oncology Unit, ASST Papa Giovanni XXIII Hospital, Bergamo, Italy, 5Novartis Farma S.P.A., Milan, Italy.
1IQVIA SOLUTIONS ITALY S.r.l., Milan, Italy, 2Department of Medical Oncology, Università Politecnica delle Marche, AOU delle Marche, Ancona, Italy, 3Department of Clinical Medicine and Surgery, University of Naples Federico II, Naples, Italy, 4Oncology Unit, ASST Papa Giovanni XXIII Hospital, Bergamo, Italy, 5Novartis Farma S.P.A., Milan, Italy.
OBJECTIVES: To describe the design of a probabilistic threshold technique (PTT) aimed at quantifying the minimum additional benefit (MAB) in terms of efficacy required by Italian patients and clinicians to accept a novel oral adjuvant therapy for hormone receptor-positive/HER2-negative (HR+/HER2−) early breast cancer (eBC).
METHODS: The PTT was designed around two hypothetical treatment options, modeled on the profiles of endocrine therapy (ET) alone (reference option) and ribociclib+ET (target option). Options were described using six attributes discussed with three clinicians and three patients: 5-year invasive disease-free survival (iDFS), grade≥3 neutropenia, grade≥3 diarrhea, treatment schedule and duration, potential for dose modifications, and impact on sexual health. Attribute levels were primarily informed by data from the NATALEE trial. Where data were unavailable, specifically for 5-year iDFS and impact on sexual health, levels were extrapolated from available evidence and validated through literature and expert opinion.
RESULTS: Two identical online questionnaires were developed, one for patients and one for clinicians, with simplified clinical wording for the former. In the initial question, both options were presented with 72% 5-year iDFS, identified as the key attribute. Based on the respondent’s choice, only the 5-year iDFS of the target option was adjusted upward or downward in subsequent questions, with incremental variations from 1 to 4 percentage points. This iterative process continued until the respondent expressed indifference or changed preference, allowing estimation of the MAB in 5-year iDFS required to prefer the target option over the reference. Respondents, blinded to the real-world identity of the hypothetical treatments, could answer up to 17 questions, with target iDFS ranging from 44% to 100%, depending on individual preferences.
CONCLUSIONS: This study outlines the methodological framework for a preference elicitation exercise, useful to explore how Italian patients and clinicians weigh risk-benefit profiles when considering adjuvant treatment options in HR+/HER2− eBC and allowing comparison between the two groups.
METHODS: The PTT was designed around two hypothetical treatment options, modeled on the profiles of endocrine therapy (ET) alone (reference option) and ribociclib+ET (target option). Options were described using six attributes discussed with three clinicians and three patients: 5-year invasive disease-free survival (iDFS), grade≥3 neutropenia, grade≥3 diarrhea, treatment schedule and duration, potential for dose modifications, and impact on sexual health. Attribute levels were primarily informed by data from the NATALEE trial. Where data were unavailable, specifically for 5-year iDFS and impact on sexual health, levels were extrapolated from available evidence and validated through literature and expert opinion.
RESULTS: Two identical online questionnaires were developed, one for patients and one for clinicians, with simplified clinical wording for the former. In the initial question, both options were presented with 72% 5-year iDFS, identified as the key attribute. Based on the respondent’s choice, only the 5-year iDFS of the target option was adjusted upward or downward in subsequent questions, with incremental variations from 1 to 4 percentage points. This iterative process continued until the respondent expressed indifference or changed preference, allowing estimation of the MAB in 5-year iDFS required to prefer the target option over the reference. Respondents, blinded to the real-world identity of the hypothetical treatments, could answer up to 17 questions, with target iDFS ranging from 44% to 100%, depending on individual preferences.
CONCLUSIONS: This study outlines the methodological framework for a preference elicitation exercise, useful to explore how Italian patients and clinicians weigh risk-benefit profiles when considering adjuvant treatment options in HR+/HER2− eBC and allowing comparison between the two groups.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
MSR83
Topic
Methodological & Statistical Research, Patient-Centered Research, Study Approaches
Topic Subcategory
Survey Methods
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology