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.

• Key points for decision makers

• The Leukemia and Lymphoma Society (LLS) initiated an independent effort to promote patient-focused drug development (PFDD). This study presents the development and piloting of a preference study as a first step in this initiative.

• Results of this pilot study were used to guide a PFDD meeting to discuss the lived experience of patients and caregivers affected by AML.

• Productive engagement by all patients, caregivers, and stakeholders throughout the process resulted in strong endorsement of the project’s approach and recognition of the need to conduct a national study.