Multiple myeloma is a complex, incurable disease, and patients will relapse and become refractory to therapy. Treatment of patients who have responded inadequately to ≥2 prior lines of therapy is particularly challenging, and outcomes tend to be poor. Many new classes of therapies and novel agents have been developed for the treatment of patients with relapsed/refractory multiple myeloma (RRMM), which can complicate treatment decisions for prescribers.
Clear recommendations have yet to be developed for patients in their third line of therapy and beyond, and many factors can influence treatment decisions. Variables include patient characteristics such as age, comorbidities, performance status, and disease factors; in addition, relevant treatment factors include the number of prior lines of therapy, resistance to therapy, potential for adverse reactions, and depth of response to previous treatments. Because of the high number of variables, it is important to understand physician drivers for treatment decisions for patients with RRMM in later-line settings.
To better understand these decision drivers, researchers assessed treatment preferences of US-based oncologists for patients with RRMM at third line of therapy and beyond. The study consisted of a qualitative phase to understand decision factors and associated levels and a subsequent quantitative phase, which assessed the decision-making process of physicians using a discrete-choice experiment (DCE) applying the factors identified in the qualitative phase. The qualitative phase started with a targeted literature review to identify relevant treatment decision factors for current treatments and the processes and language used by physicians when making treatment decisions. These factors were then used to develop the attributes and associated levels in the DCE. Decision factors included progression-free survival (PFS), overall response rate (ORR), and overall survival (OS); physicians also weighed adverse events, prior adverse events, and mode of administration.
Vignettes using the identified factors were developed as hypothetical patient profiles and incorporated into the DCE. A total of 227 physicians were presented with the vignettes in an online survey. Preferred choices and relative importance of the factors were collected for each physician. After completing the DCE survey, physicians ranked 10 factors in order of importance. Additional items collected in the DCE survey were difficulties in making choices, understanding of scenarios, and interpreting the factors described.
Of the 227 physicians participating, 83 completed the survey for third-line patients, 73 for fourth-line, and 71 for fifth-line treatment in the theoretical patient profiles. Sociodemographic and clinical variables of the physicians were consistent. The primary outcome was to assess the factors that determine physician preferences and understand the trade-offs between these attributes. Overall, efficacy was determined to be the decision factor with the highest mean relative importance when treating patients with RRMM at third line of therapy and beyond. Specifically, OS was seen as most important among physicians when making treatment decisions for patients at third-line and fourth-line and beyond. PFS and ORR were also relevant factors. In cumulative preference scores considering all rankings received for each factor, PFS was found to be more important in third-line, and ORR and PFS were equally important in fourth-line and beyond.
In all groups, adverse events were less important than efficacy. Overall, physicians preferred treatments with lower incidence of keratopathy and thrombocytopenia, both of which have similar relative importance in later-line treatment. Mode of administration, drug regimen frequency, and use of steroids were less important factors in all groups.
Overall, this survey evaluated factors important to physicians when making a treatment decision in later lines in patients with RRMM and identified the most important efficacy outcomes. Additional understanding of physician preferences may help to facilitate adoption of a wider variety of new treatment options.
Batchelder L, Stephanie Philpott S, Divino V, et al. Physician treatment preferences for relapsed/refractory multiple myeloma: a discrete choice experiment. Future Oncol. 2022;18(25):2843-2856.