Abstract
Background
Acute 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 methods
An 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.
Results
The 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).
Conclusion
Community-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.
Acknowledgments
The authors sincerely thank the stakeholder committees for their valuable contributions and unwavering involvement in the study. The abstract of this paper was presented at the Annual Meeting of the Society for Medical Decision Making as a poster presentation with interim findings. The poster’s abstract was published online in “Poster Abstracts” in Medical Decision Making: https://smdm.confex.com/smdm/2016bc/webprogram/Paper10491.html. This work was supported by LLS. The LLS recruited the experts and community members who participated in the stakeholder committees and completed the survey; however, the funder had no role in the interpretation of the data.
Disclosure
Ernest Voyard and Bernadette O’Donoghue are employees of the LLS. The other authors report no conflicts of interest in this work.