Cost-Effectiveness Modelling of Multiple System Atrophy for Early Health Technology Assessment
Literature searches for disease analogues proved useful for early modelling in multiple system atrophy, for example, to inform model structures, but surrogate data could only be used when sufficient granularity was available (e.g., micro costing). These findings suggest that early conceptual modelling can help identify data gaps and guide future evidence generation, such as selecting appropriate endpoints for natural history of disease studies. Cost-effectiveness results can be highly uncertain for rare diseases at these early stages and should be considered as part of an iterative modelling framework.
Authors
- Tobias Sydendal Grand (ORCID: https://orcid.org/0000-0002-7058-916X)
- Daniel Oudin Åström (ORCID: https://orcid.org/0000-0003-4742-417X)
- Shijie Ren (ORCID: https://orcid.org/0000-0003-3568-7124)
- Praveen Thokala (ORCID: https://orcid.org/0000-0003-4122-2366)
- Stefano Zanigni (ORCID: https://orcid.org/0000-0003-4071-0886)
- Florian Krismer (ORCID: https://orcid.org/0000-0002-4493-5073)
- Werner Poewe (ORCID: https://orcid.org/0000-0003-4367-0971)
- Günter U. Höglinger (ORCID: https://orcid.org/0000-0001-7587-6187)
- Stéphane A. Régnier (ORCID: https://orcid.org/0000-0002-1994-4648)
- Aroussi Bidani (ORCID: https://orcid.org/0009-0000-8319-9163)
Institutions
- Lundbeck (Denmark) (DK)
- Innsbruck Medical University (AT)
- German Center for Neurodegenerative Diseases (DE)
- Munich Cluster for Systems Neurology (DE)
- Ludwig-Maximilians-Universität München (DE)
- Ashland (United States) (US)
- University of Sheffield (GB)
Publication Details
- Journal
- PharmacoEconomics - Open
- Published
- 2025-10-31
- DOI
- https://doi.org/10.1007/s41669-025-00609-8
- Primary Topic
- Parkinson's Disease Mechanisms and Treatments
- Type
- article
- Field-Weighted Citation Impact
- 0.00