Assessing cost‐effectiveness of early intervention in Alzheimer's disease: An open‐source modeling framework

Green, Colin; Handels, Ron; Gustavsson, Anders; Wimo, Anders; Winblad, Bengt; Sköldunger, Anders; Jönsson, Linus

doi:10.1016/j.jalz.2019.05.004

Cited by 48 publications

(57 citation statements)

References 47 publications

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“…AD Alzheimer's disease, IPECAD International Pharmaco-Economic Collaboration on Alzheimer's Disease, LY life-year, MCI mild cognitive impairment, SveDem Swedish Dementia registry. Sources: Kansal et al [54]; Green et al [51]; Vermunt et al [53]; Wimo et al [30]; IPECAD Modeling Group [52] economic, and societal value of aducanumab for the treatment of patients with early AD.…”

Section: Discussionmentioning

confidence: 99%

Predicted Lifetime Health Outcomes for Aducanumab in Patients with Early Alzheimer’s Disease

et al. 2021

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Introduction: Alzheimer's disease (AD) is a chronic and progressive neurodegenerative disease that places a substantial burden on patients and caregivers. Aducanumab is the first AD therapy approved by the US Food and Drug Administration to reduce a defining pathophysiological feature of the disease, brain amyloid plaques. In the phase 3 clinical trial EMERGE (NCT02484547), aducanumab reduced clinical decline in patients with mild cognitive impairment (MCI) due to AD and mild AD dementia and confirmed amyloid pathology. Methods: We used a Markov modeling approach to predict the long-term clinical benefits of aducanumab for patients with early AD based on EMERGE efficacy data. In the model, patients could transition between AD severity levels (MCI due to AD; mild, moderate, and severe AD dementia) and care settings (community vs. institution) or transition to death. The intervention was aducanumab added to standard of care (SOC), and the comparator was SOC alone. Data sources for base-case and scenario analyses included EMERGE, published National Alzheimer's Coordinating Center analyses, and other published literature. Results: Per patient over a lifetime horizon, aducanumab treatment corresponded to 0.65 incremental patient quality-adjusted life-years (QALYs) and 0.09 fewer caregiver QALYs lost compared with patients treated with SOC. Aducanumab treatment translated to a lower lifetime probability of transitioning to AD dementia, a lower lifetime probability of transitioning to institutionalization (25.2% vs. 29.4%), delays in the median time to transition to AD dementia (7.50 vs. 4.92 years from MCI to moderate AD dementia or worse), and an incremental median time in the community of 1.32 years compared with SOC.

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Section: Discussionmentioning

confidence: 99%

Predicted Lifetime Health Outcomes for Aducanumab in Patients with Early Alzheimer’s Disease

et al. 2021

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“…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 …”

Section: Introductionmentioning

confidence: 99%

Evaluation of the Cost-effectiveness of Drug Treatment for Alzheimer Disease in a Simulation Model That Includes Caregiver and Societal Factors

et al. 2021

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IMPORTANCEThe possibility of widespread use of a novel effective therapy for Alzheimer disease (AD) will present important clinical, policy, and financial challenges. OBJECTIVE 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. DESIGN, SETTING, AND PARTICIPANTS 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. EXPOSURES A hypothetical drug that would delay progression to dementia in individuals with MCI compared with usual care. MAIN OUTCOMES AND MEASURES Incremental cost-effectiveness ratio (ICER), measured by cost per quality-adjusted life-year (QALY) gained. RESULTSThe 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. CONCLUSIONS AND RELEVANCEThe 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.

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“…Our model only included the progression according to MMSE and CDR-SB scores. A strength of our approach is that the mathematical functions used to reproduce patient trajectories were derived from the same clinical trial, but we recognise that other models have incorporated more dimensions [ 32 , 33 ]. Comprehensive models include three dimensions (cognitive, functional and behavioural).…”

Section: Discussionmentioning

confidence: 99%

Economic evaluation of supplementing the diet with Souvenaid in patients with prodromal Alzheimer’s disease

et al. 2020

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Background The LipiDiDiet trial showed that Souvenaid, a medical food, might delay progression to dementia in prodromal Alzheimer’s disease (AD). The objective of this study was to assess the cost-utility of Souvenaid compared to placebo in patients with prodromal AD under the conditions applied in that trial. Methods A discrete event simulation model was developed based on the LipiDiDiet trial and a literature review to assess the cost-utility of Souvenaid from a societal perspective considering direct and indirect costs. For both intervention and control groups, patient trajectories in terms of functional decline on the Clinical Dementia Rating Sum of Boxes (CDR-SB) scale in LipiDiDiet were reproduced statistically with mixed models by assigning time until events to simulated patients. From the societal perspective, four scenarios were analysed by combining different options for treatment duration and diagnostic test cost. Univariate sensitivity analysis assessed parameter uncertainties. Results Validation results at year 2 of disease progression fit with CDR-SB progression in LipiDiDiet. The incremental cost-utility ratio (ICUR) in the baseline case was €22,743/quality-adjusted life year (QALY). All scenarios rendered an ICUR lower than €25,000/QALY (the societal threshold). Moreover, the treatment option was cost-saving and increased health benefits when diagnostic costs were not considered and treatment was only administered during the prodromal stage. Conclusions Treating prodromal AD with Souvenaid is a cost-effective intervention in all scenarios analysed. The LipiDiDiet trial showed a modest improvement in disease course but as the social costs of AD are very high, the intervention was efficient. Assessing small benefits at specific stages of AD is relevant because it is reasonable to expect that no effective, safe and affordable disease-modifying therapies will become available in the short to medium term.

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Assessing cost‐effectiveness of early intervention in Alzheimer's disease: An open‐source modeling framework

Cited by 48 publications

References 47 publications

Predicted Lifetime Health Outcomes for Aducanumab in Patients with Early Alzheimer’s Disease

Predicted Lifetime Health Outcomes for Aducanumab in Patients with Early Alzheimer’s Disease

Evaluation of the Cost-effectiveness of Drug Treatment for Alzheimer Disease in a Simulation Model That Includes Caregiver and Societal Factors

Economic evaluation of supplementing the diet with Souvenaid in patients with prodromal Alzheimer’s disease

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