Patient Preferences in Neoadjuvant Therapy for HER2+ Early-Stage Breast Cancer.

Purpose. Health technology assessment (HTA) focuses on overall survival (OS) as the key clinical endpoint when making oncology treatment decisions. However, capturing robust OS estimates in early-stage breast cancer (eBC) is challenging, and patients may value other endpoints. This study assessed patient preferences for treatment attributes and endpoints in neoadjuvant therapy where mature and nonconfounded OS is rarely available. Patients and Methods. An online discrete choice experiment (DCE) was developed with health care professionals and patient advisory groups and conducted in Germany, France, Italy, and Spain with patients with HER2+ eBC. Patients were presented with 15 tasks and in each asked to choose between 2 hypothetical treatment options or to opt out in the neoadjuvant setting. Treatment attributes included OS at 5-years, disease-free survival (DFS) at 5-years, pathological complete response (pCR), impact of side effects on quality of life (QoL), and ability to receive breast-conserving surgery (BCS). Data were analyzed using multinomial logit and random parameters logit models. Results. Three hundred thirty-four patients with HER2+ eBC responded. The most valued attribute was achieving pCR (no invasive cancer in the breast and lymph nodes in all patients after treatment), followed by 5-y DFS in 95% of patients. OS ranked third in importance. The ability to undergo BCS and the impact of side effects on QoL were less important. Preferences varied by hormone receptor status, time since diagnosis, cancer stage, and age. Conclusion. Our findings are in-line with regulatory reviews, which accept pCR, DFS, and OS as clinically valid endpoints. When recommending reimbursements for treatments, HTA bodies and payers should consider patient preferences for treatment attributes and endpoints in their decision making when valuing new cancer treatments. Highlights: Traditional cancer treatment assessments focus on overall survival (OS), but in early-stage breast (eBC) cancer, patients may prioritize other treatment outcomes, since robust OS data may be unavailable.A discrete choice experiment was conducted with 334 patients with HER2+ eBC in the neoadjuvant setting in Germany, France, Italy, and Spain.Patients considered achieving pathological complete response (no invasive cancer in the breast and lymph nodes in all patients after treatment) to be the most important attribute when making a treatment decision, followed by disease free-survival (DFS) and OS; the ability to undergo breast-conserving surgery and the impact of side effects on quality of life were less important.These insights highlight the need for health authorities and reimbursement bodies to consider patient-valued outcomes beyond OS in eBC treatment evaluations.