Abstract

The possibility of widespread use of a novel effective therapy for Alzheimer disease (AD) will present important clinical, policy, and financial challenges. To describe how including different patient, caregiver, and societal treatment-related factors affects estimates of the cost-effectiveness of a hypothetical disease-modifying AD treatment. In this economic evaluation, the Alzheimer Disease Archimedes Condition Event Simulator was used to simulate the prognosis of a hypothetical cohort of patients selected from the Alzheimer Disease Neuroimaging Initiative database who received the diagnosis of mild cognitive impairment (MCI). Scenario analyses that varied costs and quality of life inputs relevant to patients and caregivers were conducted. The analysis was designed and conducted from June 15, 2019, to September 30, 2020. A hypothetical drug that would delay progression to dementia in individuals with MCI compared with usual care. Incremental cost-effectiveness ratio (ICER), measured by cost per quality-adjusted life-year (QALY) gained. The model included a simulated cohort of patients who scored between 24 and 30 on the Mini-Mental State Examination and had a global Clinical Dementia Rating scale of 0.5, with a required memory box score of 0.5 or higher, at baseline. Using a health care sector perspective, which included only individual patient health care costs, the ICER for the hypothetical treatment was $192 000 per QALY gained. The result decreased to $183 000 per QALY gained in a traditional societal perspective analysis with the inclusion of patient non-health care costs. The inclusion of estimated caregiver health care costs produced almost no change in the ICER, but the inclusion of QALYs gained by caregivers led to a substantial reduction in the ICER for the hypothetical treatment, to $107 000 per QALY gained in the health sector perspective. In the societal perspective scenario, with the broadest inclusion of patient and caregiver factors, the ICER decreased to $74 000 per added QALY. The findings of this economic evaluation suggest that policy makers should be aware that efforts to estimate and include the effects of AD treatments outside those on patients themselves can affect the results of the cost-effectiveness analyses that often underpin assessments of the value of new treatments. Further research and debate on including these factors in assessments that will inform discussions on fair pricing for new treatments are needed.

Highlights

  • An estimated 6.2 million Americans currently have Alzheimer disease (AD), with that number projected to increase to 7.2 million in just a few years.[1]

  • The model included a simulated cohort of patients who scored between 24 and 30 on the Mini-Mental State Examination and had a global Clinical Dementia Rating scale of 0.5, with a required memory box score of 0.5 or higher, at baseline

  • The inclusion of estimated caregiver health care costs produced almost no change in the Incremental cost-effectiveness ratio (ICER), but the inclusion of quality-adjusted life-year (QALY) gained by caregivers led to a substantial reduction in the ICER for the hypothetical treatment, to $107 000 per QALY gained in the health sector perspective

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Summary

Introduction

An estimated 6.2 million Americans currently have Alzheimer disease (AD), with that number projected to increase to 7.2 million in just a few years.[1]. There is a need to assess the value of such treatments, and cost-effectiveness models can serve as a starting point for setting value-based prices and preparing the health care system to pay for new AD drugs.[5,6,7]. How substantial are the differences in cost-effectiveness results for an AD treatment when analyses include estimates of the impact of treatment on patients’ costs and benefits outside the health care system? How substantial is the difference in cost-effectiveness when analyses include caregiver costs and benefits along with those for patients? To help delineate some of these issues, we evaluated how different scenarios affect estimates of costeffectiveness for a hypothetical novel and disease-modifying AD treatment How substantial are the differences in cost-effectiveness results for an AD treatment when analyses include estimates of the impact of treatment on patients’ costs and benefits outside the health care system? Second, how substantial is the difference in cost-effectiveness when analyses include caregiver costs and benefits along with those for patients? what are the policy implications of the differences in cost-effectiveness results for a hypothetical 1-time AD treatment vs a repeated treatment with the same overall effectiveness? To help delineate some of these issues, we evaluated how different scenarios affect estimates of costeffectiveness for a hypothetical novel and disease-modifying AD treatment

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