IMPLEMENTING PERSONALIZED ENDPOINTS TO SUPPORT REGULATORY AND REIMBURSEMENT DECISION-MAKING: A CASE OF GOAL ATTAINMENT SCALING (GAS) IN ALZHEIMER’S DISEASE
Author(s)
Gunes Sevinc, PhD1, Chere Chapman, MBA, MPH2, Christopher Barnum, PhD3, Judith Jaeger, PhD3, Kenneth Rockwood, MD4.
1Director of Patient-Centered Outcomes, Ardea Outcomes, Halifax, NS, Canada, 2Ardea Outcomes, Halifax, NS, Canada, 3INmune Bio, Inc, Boca Raton, FL, USA, 4Dalhousie University, Halifax, NS, Canada.
1Director of Patient-Centered Outcomes, Ardea Outcomes, Halifax, NS, Canada, 2Ardea Outcomes, Halifax, NS, Canada, 3INmune Bio, Inc, Boca Raton, FL, USA, 4Dalhousie University, Halifax, NS, Canada.
OBJECTIVES: Personalized endpoints are increasingly used to capture outcomes that are meaningful to patients and caregivers, particularly in conditions with substantial heterogeneity in symptom expression and progression, such as Alzheimer’s disease (AD). Traditional outcome measures may fail to detect subtle but clinically meaningful benefits, including stabilization or changes in daily functioning. Goal Attainment Scaling (GAS) offers a structured, patient-centered approach to quantifying such individualized change; however, to support regulatory and reimbursement decision-making, GAS requires rigorous and standardized implementation. The objective of this study was to describe a standardized strategy for implementing GAS in a Phase 2 AD clinical trial.
METHODS: GAS was implemented in a global, six-month, randomized, double-blind, placebo-controlled Phase 2 trial of pegipanermin (XPro1595) in 201 patients with early AD and biomarkers of inflammation. GAS standardization strategies included: (1) a disease expert-informed goal inventory aligned with the product’s proposed mechanism of action, covering cognition, behavior, daily function, executive function, and physical manifestation domains; (2) structured GAS orientation for patients and study partners; (3) standardized GAS interviewer training and certification along with realtime goal scale reviews and ongoing interviewer support; (4) structured goal-setting and follow-up and centralized data monitoring through a GAS-focused electronic clinical outcome assessment platform (GoalNav®). Consistency of implementation was assessed by tracking protocol deviations and other sources of variation (e.g., changes in GAS interviewers).
RESULTS: The standardized GAS framework was feasible and enabled consistent elicitation and quantification of patient- and caregiver-prioritized goals across multiple sites and geographic locations, producing reliable, interpretable GAS data. Analysis of sources of variation provided insights into methodological and operational gaps, as well as areas for improvement.
CONCLUSIONS: With rigorous standardization, GAS can enhance the evidentiary value of clinical trials by quantifying outcomes that are inherently meaningful to patients and caregivers. This case demonstrates how GAS can support drug development in Alzheimer’s disease.
METHODS: GAS was implemented in a global, six-month, randomized, double-blind, placebo-controlled Phase 2 trial of pegipanermin (XPro1595) in 201 patients with early AD and biomarkers of inflammation. GAS standardization strategies included: (1) a disease expert-informed goal inventory aligned with the product’s proposed mechanism of action, covering cognition, behavior, daily function, executive function, and physical manifestation domains; (2) structured GAS orientation for patients and study partners; (3) standardized GAS interviewer training and certification along with realtime goal scale reviews and ongoing interviewer support; (4) structured goal-setting and follow-up and centralized data monitoring through a GAS-focused electronic clinical outcome assessment platform (GoalNav®). Consistency of implementation was assessed by tracking protocol deviations and other sources of variation (e.g., changes in GAS interviewers).
RESULTS: The standardized GAS framework was feasible and enabled consistent elicitation and quantification of patient- and caregiver-prioritized goals across multiple sites and geographic locations, producing reliable, interpretable GAS data. Analysis of sources of variation provided insights into methodological and operational gaps, as well as areas for improvement.
CONCLUSIONS: With rigorous standardization, GAS can enhance the evidentiary value of clinical trials by quantifying outcomes that are inherently meaningful to patients and caregivers. This case demonstrates how GAS can support drug development in Alzheimer’s disease.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
PCR11
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
SDC: Geriatrics, SDC: Neurological Disorders