(OA-18) The Value of Progression-Free Survival (PFS) from the Perspectives of Patients with Multiple Myeloma (MM) and Treating Physicians: Quantitative Research Findings from Eight Countries

Advances in MM treatments (Tx) have improved overall survival (OS); yet, relying on mature OS data for regulatory and health authority approval is challenging, emphasizing the need to better understand alternative endpoints, like PFS. We evaluated how patients (pts) and physicians value PFS in Tx decision-making and how progression impacts pts’ quality of life (QoL) in the US, UK, Spain, France, Germany, Italy, Japan, and Brazil.

Methods:

Hematologists/oncologists with ≥10 pts with MM and 3–30 y of experience, and pts with MM diagnosed ≤10 y ago, completed an online questionnaire. Questionnaire content was informed by physician and pt qualitative interviews (Kissling A, et al. Value Health 2024;27:S543–544), and used structured questions and best-worst scaling (BWS). Questions focused on understanding of PFS, progression-related symptoms/impacts, and the role of PFS in Tx decisions. Descriptive statistics were used for analyzing study variables; BWS was analyzed using hierarchical Bayes estimation.

Results:

Physicians had a mean practice experience of 15.6 y (standard deviation [SD], 7.0) and treated a median of 88.9 pts (SD, 119.5) in the past 6 months. Pts (N=237) had a mean age of 68.8 y (SD, 7.3), 63.7% were male, and mean time since diagnosis was 3.3 y (SD,1.6). Pts were undergoing 1st line (1L; 47.3%; n=112), 2nd line (2L; 19.8%; n=47), or 3rd line or later (32.9%; n=78) Tx.

Around half of pts (49.3%) and physicians (55.0%) considered PFS equal or more important than OS; and over two-thirds of physicians for the following pt groups: frail (74.6%), those with pain (73.4%), elderly (71.5%), symptomatic disease (71.5%), comorbid (70.4%), and 4th line (68.9%). When asked why PFS was important in Tx selection, 52.4% of physicians selected that PFS was a surrogate for OS when OS data were unavailable and >95% supported its use in healthcare access and coverage decisions regardless of OS data availability.

Among pts who experienced progression (52.7%), 97.6% had ≥1 of the following symptoms: general pain (63.2%), fatigue (50.4%), muscle weakness (48.0%), anemia (32.8%), bone pain (32.0%), fragile bones, breaks, and fractures (32.0%), and kidney problems and/or renal failure (11.2%). Other symptoms affecting QoL included numbness (53.6%), anxiety (40.8%), and depression (27.2%).

In the BWS, physicians valued PFS and OS most across Tx lines. QoL attributes were valued more by physicians in 2L Tx decision-making and achieving measurable residual disease negativity when selecting 1L Tx. OS was the most important attribute among pts. Other attributes, including multiple QoL factors and PFS, were similarly valued by pts across Tx lines.

Conclusions:

OS and PFS are important to physicians and pts in Tx-related decisions. Around half of pts and, for certain groups, two-thirds of physicians valued OS over PFS. Nearly all physicians support the use of PFS in healthcare access and coverage decisions. Experiencing progression significantly impacts pts' QoL.