Patient Preferences for Multiple Myeloma (MM) Treatment: Interim Analysis of a Discrete Choice Experiment

• Published in: Blood 2018-11, Vol.132 (Supplement 1), p.3586-3586
• Main Authors: McKay, Caroline, Maiese, Eric M, Chiarappa, Joseph, Cambron-Mellott, M. Janelle, Maculaitis, Martine, Alunni, Nicole, Raje, Noopur
• Format: Article
• Language: English
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood-2018-99-112921

Background: The growing importance of patient preferences in treatment decision-making in oncology is evidenced by the expanding role of patient-reported experience in both regulatory and reimbursement considerations of value. While recent introduction of new treatments for multiple myeloma (MM) have demonstrated longer time to progression and improved survival, specific regimen options still vary with respect to efficacy, safety, and dosing. Therefore, patients and providers must consider the trade-offs inherent in making treatment decisions. However, a lack of evidence exists describing patient reported preferences within the context of currently available regimens. To address this gap, this interim analysis of an ongoing study was conducted to examine patient preferences for MM treatments. Methods: A sequential mixed methods design, which incorporates both qualitative and quantitative phases, was utilized for this study. The qualitative phase identified content and language, via semi-structured interviews, to elucidate how patients understand and construct treatment-related factors, such as overall survival (OS), progression free survival (PFS), dosing, and tolerability. Results from the qualitative phase were subsequently used to inform the development of the quantitative survey. The online survey was sent to adults diagnosed with MM, who had received or were currently receiving first-line therapy (FL), second-line therapy (1PL), or third-line therapy (2PL) at the time of the survey (target N=200). Patients were recruited from targeted panels, advocacy partnerships, patient communities, and physician referrals from May to June 2018. The survey utilized a Discrete Choice Experiment (DCE) methodology to assess preferences and willingness to accept trade-offs among hypothetical treatments that varied on levels of specific attributes. Treatment attributes and levels were identified through literature review, current treatment guidelines, and clinical input. In the quantitative survey, patients were asked to rate the levels of each treatment attribute (based on a 5-point Likert scale, ranging from 1=very bad to 5=very good) and select which regimen they prefer when presented with two different hypothetical regimens (fixed choice exercise - Table 1) to identify trade-offs that patients were willing to make when selecting treatments. Descriptive statistics were used to characterize the interim survey data. Full DCE results, which will model additional treatme