PATIENT PREFERENCES FOR BENEFIT-RISK TRADE-OFFS AND EVIDENCE UNCERTAINTY OF ANTI-AMYLOID THERAPIES IN EARLY ALZHEIMER'S DISEASE: A DISCRETE CHOICE EXPERIMENT
Author(s)
Hye-In Jung, PharmD1, YEBIN YOON, PharmD2, Sun-Kyeong Park, PhD2, Ha-Jun Song, PharmD1, GaHee Choi, Bachelor's degree1, Myeongseog Kim, Bachelor's degree1, Ji-Hyeon Namgung, Bachelor's degree1, Mi-Hai Park, PhD1, EUI-KYUNG LEE, PhD1.
1Sungkyunkwan University, Suwon, Korea, Republic of, 2The Catholic University of Korea, Bucheon, Korea, Republic of.
1Sungkyunkwan University, Suwon, Korea, Republic of, 2The Catholic University of Korea, Bucheon, Korea, Republic of.
OBJECTIVES: This study aimed to investigate patient preferences regarding the benefit-risk profile and evidence uncertainty associated with anti-amyloid therapies among individuals with mild cognitive impairment (MCI) or mild dementia due to Alzheimer’s disease.
METHODS: A discrete choice experiment (DCE) was conducted to elicit patient preferences for anti-amyloid therapies. Attributes and levels were identified based on pivotal clinical trial data and regulatory evidence surrounding currently available anti-amyloid agents. The selected attributes represented three key domains: (1) benefit, including delay in disease progression over 18 months and the possibility of treatment discontinuation following amyloid clearance; (2) risk, including symptomatic ARIA-edema/effusions (ARIA-E), severe ARIA-microhemorrhages and hemosiderin deposition (ARIA-H), and discontinuation due to infusion-related reactions; and (3) dosing interval and evidence uncertainty, including the level of certainty regarding long-term clinical benefits beyond the trial period. Choice tasks were generated using a D-efficient experimental design. Preference data were analyzed using a mixed logit model to estimate utility coefficients and relative attribute importance while accounting for preference heterogeneity among respondents.
RESULTS: A total of 220 individuals with MCI or mild dementia due to Alzheimer’s disease were recruited. Treatment efficacy, particularly the magnitude of delay in disease progression, is a major driver of patient preferences. Respondents also appeared sensitive to treatment-related safety risks. Furthermore, evidence availability regarding the durability of long-term clinical benefits emerged as an important consideration in treatment decision-making.
CONCLUSIONS: This study provides novel evidence on how patients with early Alzheimer’s disease value therapeutic benefits, safety risks, and uncertainty surrounding long-term treatment outcomes. The findings may inform patient-centered benefit-risk assessments and support the integration of patient preference evidence into regulatory and health technology assessment decision-making for therapies approved on the basis of evolving clinical evidence.
METHODS: A discrete choice experiment (DCE) was conducted to elicit patient preferences for anti-amyloid therapies. Attributes and levels were identified based on pivotal clinical trial data and regulatory evidence surrounding currently available anti-amyloid agents. The selected attributes represented three key domains: (1) benefit, including delay in disease progression over 18 months and the possibility of treatment discontinuation following amyloid clearance; (2) risk, including symptomatic ARIA-edema/effusions (ARIA-E), severe ARIA-microhemorrhages and hemosiderin deposition (ARIA-H), and discontinuation due to infusion-related reactions; and (3) dosing interval and evidence uncertainty, including the level of certainty regarding long-term clinical benefits beyond the trial period. Choice tasks were generated using a D-efficient experimental design. Preference data were analyzed using a mixed logit model to estimate utility coefficients and relative attribute importance while accounting for preference heterogeneity among respondents.
RESULTS: A total of 220 individuals with MCI or mild dementia due to Alzheimer’s disease were recruited. Treatment efficacy, particularly the magnitude of delay in disease progression, is a major driver of patient preferences. Respondents also appeared sensitive to treatment-related safety risks. Furthermore, evidence availability regarding the durability of long-term clinical benefits emerged as an important consideration in treatment decision-making.
CONCLUSIONS: This study provides novel evidence on how patients with early Alzheimer’s disease value therapeutic benefits, safety risks, and uncertainty surrounding long-term treatment outcomes. The findings may inform patient-centered benefit-risk assessments and support the integration of patient preference evidence into regulatory and health technology assessment decision-making for therapies approved on the basis of evolving clinical evidence.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PCR124
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Neurological Disorders