DEVELOPING A BEST-WORST SCALING INSTRUMENT TO PRIORITIZE THE WORRIES OF PATIENTS WITH ACUTE MYELOID LEUKEMIA (AML)

Purpose: Acute myeloid leukemia (AML) is a rapidly progressing blood cancer for which treatment strategies have remained unchanged for the past three decades. Patient-focused drug development (PFDD) in this disease area has been hindered by a lack of studies detailing the patient experience. We sought to identify key patient worries and develop an instrument to prioritize them. 

Method: In partnership with the Leukemia and Lymphoma Society (LLS), the largest health organization devoted to blood cancers, two stakeholder committees were formed to guide this research; the “community” stakeholder committee included patients and caregivers and the “expert” stakeholder committee consisted of clinicians and industry representatives. Throughout five months of engagement, both stakeholder groups were actively involved in all stages of instrument development. This process included item identification, instrument pretesting, and online piloting. In the pilot, a best-worst scaling (BWS) instrument was presented in which respondents viewed 13 subsets of 4 worries and selected the most and least worrying items. These efforts culminated in a well-attended meeting at the FDA where the identified worries were discussed and the preliminary pilot priorities were presented.

Result: Patients and caregivers (n=15) initially identified 12 worries that fell into four experience domains (decision-making, treatment delivery, physical impacts, psychosocial effects). A draft survey was pretested (n=13) using cognitive debriefing, leading to substantive revisions to 3 worries and the addition of 1 new object (“long term side effects”). The final list of 13 worries was incorporated into a pilot BWS survey and administered to an expanded community stakeholder committee (n=25). Dying from my disease was prioritized as most worrisome and received a standardized score of 100. This was followed by long term side effects (73) and time in the hospital (70). Communication with doctors (23) and the overall financial costs of AML (41) were the two least prioritized worries. These findings where confirmed and explained by the patients and caregivers (and other stakeholders) in attendance at the FDA meeting.

Conclusion: Rigorous engagement of patients, caregivers, and other stakeholders is important in describing and quantifying disease experience. Our approach of combining survey development with traditional patient testimony was considered an important advancement in PFDD and expanding this research to incorporate a national study of patients and caregivers received strong endorsement by all involved.