OBJECTIVES: Decision-analytic models assessing the value of emerging Alzheimer's disease (AD) treatments are challenged by limited evidence on short-term trial outcomes and uncertainty in extrapolating long-term patient-relevant outcomes. To improve understanding and foster transparency and credibility in modeling methods, we cross-compared AD decision models in a hypothetical context of disease-modifying treatment for mild cognitive impairment (MCI) due to AD. METHODS: A benchmark scenario (US setting) was used with target population MCI due to AD and a set of synthetically generated hypothetical trial efficacy estimates. Treatment costs were excluded. Model predictions (10-year horizon) were assessed and discussed during a 2-day workshop. RESULTS: Nine modeling groups provided model predictions. Implementation of treatment effectiveness varied across models based on trial efficacy outcome selection (clinical dementia rating - sum of boxes, clinical dementia rating - global, mini-mental state examination, functional activities questionnaire) and analysis method (observed severity transitions, change from baseline, progression hazard ratio, or calibration to these). Predicted mean time in MCI ranged from 2.6 to 5.2 years for control strategy and from 0.1 to 1.0 years for difference between intervention and control strategies. Predicted quality-adjusted life-year gains ranged from 0.0 to 0.6 and incremental costs (excluding treatment costs) from -US$66 897 to US$11 896. CONCLUSIONS: Trial data can be implemented in different ways across health-economic models leading to large variation in model predictions. We recommend (1) addressing the choice of outcome measure and treatment effectiveness assumptions in sensitivity analysis, (2) a standardized reporting table for model predictions, and (3) exploring the use of registries for future AD treatments measuring long-term disease progression to reduce uncertainty of extrapolating short-term trial results by health-economic models.

Alzheimer Centre Limburg Faculty of Health Medicine and Life Sciences School for Mental Health and Neuroscience Department of Psychiatry and Neuropsychology Maastricht University Maastricht The Netherlands; Division of Neurogeriatrics Department of Neurobiology Care Sciences and Society Karolinska Institutet Solna Sweden

Basque Health Service Debagoiena Integrated Healthcare Organisation Research Unit Arrasate Mondragón Spain; Biogipuzkoa Health Research Institute Donostia San Sebastián Spain; Biosistemak Institute for Health Service Research Barakaldo Spain

Biogen International GmbH Baar Switzerland

Care Policy and Evaluation Centre London School of Economics London England UK

Clinical Memory Research Unit Department of Clinical Sciences Lund University Lund Sweden

Community Health Sciences and O'Brien Institute of Public Health University of Calgary Calgary Alberta Canada

Department of Public Health Erasmus MC University Medical Center Rotterdam Rotterdam The Netherlands

Division of Neurogeriatrics Department of Neurobiology Care Sciences and Society Karolinska Institutet Solna Sweden

Division of Neurogeriatrics Department of Neurobiology Care Sciences and Society Karolinska Institutet Solna Sweden; Biogen Idec Ltd Maidenhead England UK

Division of Neurogeriatrics Department of Neurobiology Care Sciences and Society Karolinska Institutet Solna Sweden; Health Economics RTI Health Solutions Research Triangle Park NC USA

Division of Neurogeriatrics Department of Neurobiology Care Sciences and Society Karolinska Institutet Solna Sweden; Quantify Research Stockholm Sweden

Division of Neurogeriatrics Department of Neurobiology Care Sciences and Society Karolinska Institutet Solna Sweden; Theme Inflammation and Aging Karolinska University Hospital Huddinge Sweden

Faculty of Engineering Electronics and Computing Department Mondragon Unibertsitatea Mondragon Gipuzkoa Spain

Health Economics Policy and Innovation Institute Masaryk University Brno Czech Republic; USC Price School of Public Policy and Schaeffer Center for Health Policy and Economics Los Angeles CA USA

Health Economics RTI Health Solutions Research Triangle Park NC USA

USC Price School of Public Policy and Schaeffer Center for Health Policy and Economics Los Angeles CA USA

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