Modelling Challenges in Geographic Atrophy

Author(s)

Intorcia M1, Hill S2, Longworth L3, Sarda SP1, Oluboyede Y4, Moor H2
1Apellis Pharmaceuticals, Waltham, MA, USA, 2Putnam PHMR Ltd., London, LON, UK, 3Putnam PHMR Ltd., London, UK, 4Putnam PHMR Ltd., Newcastle Upon Tyne, NT, UK

OBJECTIVES: Geographic atrophy (GA) is the advanced form of dry age-related macular degeneration (AMD) and manifests as central visual loss and impairs health-related quality-of-life. Our objective was to review existing literature for guidance on developing cost-effectiveness (CE) models for GA.

METHODS: A targeted literature review was conducted to identify clinical trial reports, commentary papers, and methodological articles on GA populations and economic models of populations with either GA or similar conditions (e.g., wet AMD; restricted to publications since 2020 in wet AMD). We searched MEDLINE, Embase, and PubMed and conducted a hand-search of health technology appraisals (HTAs) published by the National Institute for Health and Care Excellence (NICE).

RESULTS: No GA models were identified. Seventeen studies were included, among them were six wet AMD modelling studies, three NICE HTAs, and one methodological article. All included modelling studies used Markov state-transition cohort models. Several identified limitations of the wet AMD models were their lack of applicability to GA. Limitations were also related to model structure and input parameters. Intrinsic clinical differences between GA and wet AMD, such as high levels of heterogeneity in prognostic factors for visual function, a more gradual disease progression than wet AMD, and limited possibility of vision improvement, limit the applicability of existing model structures to the GA population. CE models for the GA population are needed. Given the nature of GA disease progression, key challenges of developing robust CE models include extrapolation of long-term clinical and cost outcomes beyond clinical trials and establishing and validating links between structural and visual function outcomes and health-related utilities.

CONCLUSIONS: There are no existing GA CE models. Existing wet AMD CE models have limited applicability to GA. The lack of real-world data for GA presents a daunting challenge to develop a long-term CE model.

Conference/Value in Health Info

2023-11, ISPOR Europe 2023, Copenhagen, Denmark

Value in Health, Volume 26, Issue 11, S2 (December 2023)

Code

MSR18

Topic

Methodological & Statistical Research

Disease

No Additional Disease & Conditions/Specialized Treatment Areas, Sensory System Disorders (Ear, Eye, Dental, Skin)

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