Analysis of physicians' perspectives versus patients' preferences: direct assessment and discrete choice experiments in the therapy of multiple myeloma

Abstract

Background: Against the background of patient involvement, understanding patients' preferences for treatments is crucial: Do physicians have the same or a different perception of the patients' preferences? As there is currently no cure for patients with multiple myeloma, primary objectives of treatment are to extend survival at the best possible quality of life. In this study, physicians' beliefs about patients' preferences regarding the treatment of multiple myeloma (MM) were explored in a direct assessment and a discrete choice experiment (DCE), and were compared to the previously explored patients' views. How much do physicians know about their patients' preferences?

Methods: In a preceding study with German multiple myeloma patients, relevant attributes of an ideal multiple myeloma treatment were collected by reviewing the literature and by conducting a qualitative study with focus groups. The attributes were analyzed using both a direct measurement (16 items on a five-point Likert scale) and a DCE (eight pairs with eight characteristics). For the present study, 30 German physicians reviewed the treatment attributes from the previous study for completeness. A total of 243 physicians participated in the study (including the 30 participants in the pre-test). The direct assessment and the DCE covered four major preference dimensions that both the literature review and the focus groups revealed: Aspects of medical effectiveness (including prolonged life expectancy, effectiveness and long duration of effect), side effects, quality of life (including social, physical and emotional quality of life) and flexibility (breaks in therapy and further treatment options).

Results: In the direct measurement of patients' preferences, physicians rated physical quality of life (specified as "reduced mobility or good mobility"), rare side effects and effectiveness aspects (duration of effect, maximal prolonged life expectancy and effectiveness) as the most important attributes from the patients' perspective, followed by emotional quality of life (specified as "Not always think of the disease") and therapy-free intervals. Especially further treatment options and dosage were more important to patients than physicians believed. In this case, the physicians had quite obviously underestimated the importance of these attributes from the perspective of those affected. Physicians ranked prolonged life expectancy as relatively the most important and significantly more important than all other treatment attributes. Further treatment options were the second most important attribute and significant compared to the attributes breaks in therapy and physical quality of life, whereas the patients ordered these two attributes in reverse order. Similarly, the patients gave the opposite relative importance to the next two priorities: self-application of treatment and emotional quality of life.

Conclusions: Asking patients or physicians about the multiple myeloma patients' treatment preferences, the combination of direct assessment and DCE proves to be a valid survey technique. Over a broad range of treatment attributes, the physicians' perceptions of preferences were very close to those of multiple myeloma patients. Both the direct assessment of importance in order to rank the patient perceptions and the DCE provide important insights into the preference structure of patients with multiple myeloma. The findings can subsequently be used as a basis for tailoring health care services for multiple myeloma patients in reference to their preferences.