Poulos C, Hauber B, Mirzaei F, Dilley A, DeMuro C, Fillit HM. A study of preferences for disease-modifying treatments for Alzheimer's Disease. Poster presented at the 2020 ISPOR Virtual Conference; May 2020. [abstract] Value Health. 2020 May 1; 23(Suppl 1):S278. doi: 10.1016/j.jval.2020.04.1189.


OBJECTIVES: To address challenges affecting preference studies of people within the Alzheimer’s disease (AD) spectrum regarding disease-modifying treatments: (1) selecting screening criteria and processes to identify adults within the AD spectrum who can accurately self-report and answer preference questions; (2) identifying meaningful treatment benefits; (3) developing a preference survey that is not too cognitively burdensome.

METHODS: To address challenges affecting preference studies of people within the Alzheimer’s disease (AD) spectrum regarding disease-modifying treatments: (1) selecting screening criteria and processes to identify adults within the AD spectrum who can accurately self-report and answer preference questions; (2) identifying meaningful treatment benefits; (3) developing a preference survey that is not too cognitively burdensome.

RESULTS: Considering both sets of interviews:

Screening: Participants with mild dementia, and some with mild cognitive impairment (MCI) or subjective cognitive complaints (SCC), had difficulty following preference question instructions and many relied on interviewer support. Within-subject variation was observed in responses to repeated screening questions.

Meaningful treatment benefits: Treatment benefit concepts that were meaningful to patients and different from clinical endpoints were identified and refined in the interviews.

Survey design: Complex preference questions involving tradeoffs or ranking of multiple benefits and risks were cognitively burdensome, whereas most participants could answer questions involving pair-wise tradeoffs.

CONCLUSIONS: Based on the two-stage interview process:

Screening criteria, including a validated cognitive assessment (Fillit et al., 2003), were selected to ensure that self-reported SCC or MCI are not in more advanced stages of the AD spectrum precluding informed consent or survey completion. A two-stage screening process should be used to ensure data are reliable.

Elicit patient input on identifying and describing meaningful treatment benefits.

Use simple preference questions and interviewer-assisted survey modes to maximize data quality.

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