Developing and piloting an instrument to prioritize the worries of patients with acute myeloid leukemia

Abstract

BackgroundAcute myeloid leukemia (AML) is a rapidly progressing blood cancer for which new treatments are needed. We sought to promote patient-focused drug development (PFDD) for AML by developing and piloting an instrument to prioritize the worries of patients with AML.Patients and methodsAn innovative community-centered approach was used to engage expert and community stakeholders in the development, pretesting, pilot testing, and dissemination of a novel best–worst scaling instrument. Patient worries were identified through individual interviews (n=15) and group calls. The instrument was developed through rigorous pretesting (n=13) and then piloted among patients and caregivers engaged in this study (n=25). Priorities were assessed using best–worst scores (spanning from +1 to −1) representing the relative number of times that items were endorsed as the most and the least worrying. All findings were presented at a PFDD meeting at the US Food and Drug Administration (FDA) that was attended by >80 stakeholders.ResultsThe final instrument included 13 worries spanning issues such as decision making, treatment delivery, physical impacts, and psychosocial effects. Patients and caregivers most prioritized worries about dying from their disease (best minus worst [BW] score=0.73), long-term side effects (BW=0.28), and time in hospital (BW=0.25).ConclusionCommunity-centered approaches are valuable in designing and executing PFDD meetings and associated quantitative surveys to document the experience of patients. Expert and community stakeholders welcomed the opportunity to share their experiences with the FDA and strongly endorsed implementing this survey nationally.

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“…This study was designed with the Leukemia & Lymphoma Society (LLS) to quantify the patient experience in AML to inform patient‐focused drug development (PFDD) 4 ,. 18–21 Study governance included an executive committee of researchers and LLS staff, and two different stakeholder groups: a committee of AML experts and a community committee comprised of AML patients and caregivers. Study design was organized across five predetermined stages: engagement, development, pretesting, pilot testing, and presentation resulting in a national survey of AML patients using an AML‐specific best–worst scaling (BWS) instrument.…”

“…We designed a BWS instrument to elicit patient worries from patients and their caregivers based on the lived experience of patients and the current clinical environment. A detailed description of instrument development has been published 20 . Development and all revisions, including determination of worry domains and items, were informed by engagement with the stakeholders.…”