Developing an instrument to assess patient preferences for benefits and risks of treating acute myeloid leukemia to promote patient-focused drug development

Objective Acute myeloid leukemia (AML) is a progressive blood cancer with few effective treatment options. As part of a patient-focused drug development (PFDD) initiative led by the Leukemia and Lymphoma Society (LLS), this study sought to use a community-centered approach to develop and pilot an instrument to measure patient preferences for the benefits and risks of treating AML. Methods Instrument development was informed by a literature review, engagement with expert stakeholders (n = 12), engagement with community stakeholders, and pre-testing. A discrete-choice experiment (DCE), in which participants made choices between 16 pairs of hypothetical treatments, was developed with five attributes: event-free survival, complete remission, time in hospital, short-term side-effects, and long-term side-effects. A pilot test was conducted and analyzed using conditional logistic regression. Results are presented using relative attribute importance (RAI) scores. Results Patients with AML and caregivers were engaged in developing (n = 15), pre-testing (n = 13), and pilot testing (n = 26) the instrument. The pilot included patients with AML (n = 18) and caregivers of living or deceased patients with AML (n = 8). Participants had a mean age of 50 years (range =24-81), and were mostly college educated (n = 22), privately insured (n = 21), and employed (n = 13). Based on the DCE, complete remission was identified as the most important attribute (RAI =10), followed by event-free survival (3.7), time in hospital (2.8), long-term side-effects (2.3), and short-term side-effects (2.1). Conclusion The mixed-methods approach to PFDD was welcomed by all stakeholders and there was strong endorsement to implement this DCE as part of a national survey.