IPECAD MODELING WORKSHOP 2023 CROSS COMPARISON CHALLENGE ON COST-EFFECTIVENESS MODELS IN ALZHEIMER'S DISEASE AND RELATED DEMENTIAS

Autoři	HANDELS Ron HERRING William L. KAMGAR Farzam AYE Sandar TATE Ashley GREEN Colin GUSTAVSSON Anders WIMO Anders WINBLAD Bengt SKÖLDUNGER Anders RAKET Lars Lau BEDREJO STELLICK Chelsea SPACKMAN Eldon HLÁVKA Jakub WEI Yifan MAR Javier SOTO-GORDOA Myriam DE KOK Inge BRÜCK Chiara ANDERSON Robert PEMBERTON-ROSS Peter URBICH Michael JÖNSSON Linus
Rok publikování	2023
Druh	Článek v odborném periodiku
Časopis / Zdroj	VALUE IN HEALTH
Fakulta / Pracoviště MU	Ekonomicko-správní fakulta
Citace
Doi	http://dx.doi.org/10.1016/j.jval.2024.09.006
Klíčová slova	Alzheimer’s disease; decision-analytic modeling; health-economic evaluation; cross-validation
Popis	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 (CDR-SB, CDR-global, MMSE, FAQ) and analysis method (observed severity transitions, change from baseline, progression hazard ratio, or calibration to these). Predicted mean time in MCI ranged from 2.6-5.2 years for control strategy, and from 0.1-1.0 years for difference between intervention and control strategies. Predicted quality-adjusted life-year gains ranged from 0.0-0.6 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.