OBJECTIVES: Therapeutic advances have improved overall survival (OS) for patients with multiple myeloma (MM) such that median OS may not be reached within a clinical study period, which has implications for regulatory and reimbursement decisions. Therefore, evaluation of progression-free survival (PFS) has become necessary but is not always considered in benefit assessments. This study evaluated patient and physician perspectives on the value of PFS.

METHODS: 128 participants from the United States, United Kingdom, Germany, Spain, France, Italy, Brazil, and Japan (8–12 patients and 8–12 oncologists/hematologists in each country) participated in telephone interviews in their native language between October 2023 and March 2024. The interviews focused on PFS relevance. Content and thematic analyses were performed.

RESULTS: Although physicians generally did not use the term “PFS” with patients, PFS was central to their treatment goals and selection. When discussing PFS with patients, physicians referred to the duration the cancer may be under control, get worse, and/or require new treatment. Patients reported self-monitoring their symptoms for signs of progression and tracking laboratory results with their physician. To patients, progression means their disease is advancing, causing damage, and will require new treatment, which increases their anxiety. Progression impacts their daily life, including extreme fatigue, frequent medical appointments, and the inability to work. Physicians and patients both viewed PFS as equally or more important than OS. For physicians, PFS represents disease control and maintaining patients’ quality of life (QoL), is a surrogate for OS, and is relevant across treatment lines. Patients considered PFS important since it was associated with preserving their QoL. Both physicians and patients expressed concern about developing treatment resistance or having fewer treatment options after progression.

CONCLUSIONS: PFS is an essential endpoint in MM and influences treatment selection by physicians. Remaining progression-free influences patients’ physical and emotional well-being as well as their daily lives.