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

Area of Science:

• Oncology
• Health Economics
• Patient-Reported Outcomes

Background:

• Health technology assessment (HTA) traditionally prioritizes overall survival (OS) for oncology treatment decisions.
• Robust OS data is often unavailable in early-stage breast cancer (eBC) neoadjuvant settings.
• Patients may value other treatment endpoints beyond OS.

Purpose of the Study:

• To assess patient preferences for treatment attributes and endpoints in neoadjuvant therapy for HER2+ eBC.
• To understand patient priorities when mature OS data is not yet available.

Main Methods:

• An online discrete choice experiment (DCE) was conducted with 334 patients with HER2+ eBC across Germany, France, Italy, and Spain.
• Patients evaluated hypothetical treatment options based on attributes including 5-year OS, 5-year DFS, pCR, side effect impact on quality of life (QoL), and breast-conserving surgery (BCS) possibility.
• Data were analyzed using multinomial logit and random parameters logit models.

Main Results:

• Pathological complete response (pCR) was the most valued attribute, followed by 5-year DFS, and then OS.
• The ability to undergo BCS and the impact of side effects on QoL were less prioritized by patients.
• Patient preferences showed variation based on hormone receptor status, time since diagnosis, cancer stage, and age.

Conclusions:

• Patient preferences indicate that pCR and DFS are highly valued endpoints in eBC neoadjuvant treatment.
• HTA bodies and payers should incorporate patient-valued outcomes, beyond OS, into reimbursement decisions for novel cancer therapies.
• Findings align with regulatory acceptance of pCR and DFS as clinically valid endpoints.