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

Abstract

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.

Keywords: Acute myeloid leukemia (AML); Community engagement; Instrument development; Stated preference.

Figure 1.

Governance structure and stakeholder engagement process.

Figure 2.

Timeline of the study.

Figure 3.

Preference weights for acute myeloid leukemia (AML) treatment attributes (A) and relative attribute importance (B). (A) Preference weights for AML treatment attributes (n = 26). Preference weights are rescaled to set the lowest level of each attribute to zero. The y-axis represents how much participants value the attribute: a positive preference weight indicates that participants preferred the attribute, a negative preference weight indicates that participants avoided the attribute. (B) Relative attribute importance is calculated by taking the difference in the preference estimate of the most preferred level of an attribute and the least preferred level of an attribute. The RAIs were rescaled so that the largest RAI (for complete remission) was equal to 10. The vertical bars surrounding each RAI denote the 95% confidence interval about the point estimate.