Economic evaluations of disease-modifying therapies for spinal muscular atrophy: a systematic literature review
The economic evaluation landscape for SMA treatments is evolving. However, challenges remain due to data gaps and methodological variability across studies. Future research should prioritise the integration of long-term real-world data into economic evaluations, consider the development of patient- and caregiver-derived utility values, and the use of transparent, standardised modelling approaches. These improvements will enhance the robustness, comparability, and policy relevance of economic evaluations in rare disease treatment funding.
Authors
- Mubarak Patel (ORCID: https://orcid.org/0000-0001-7573-1447)
- Amy Grove (ORCID: https://orcid.org/0000-0002-8027-7274)
- Janette Parr (ORCID: https://orcid.org/0000-0002-0629-1596)
- Peter Auguste (ORCID: https://orcid.org/0000-0001-5143-3218)
- Jo Parsons (ORCID: https://orcid.org/0000-0002-6542-8492)
- Anna Brown (ORCID: https://orcid.org/0000-0002-4541-6232)
- Mehdi Yousefi (ORCID: https://orcid.org/0000-0002-4791-7866)
- Furqan Butt (ORCID: https://orcid.org/0009-0002-8393-3502)
- Amin Mehrabian (ORCID: https://orcid.org/0000-0002-8879-7565)
Institutions
- Birmingham City University (GB)
- University of Birmingham (GB)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2025-12-05
- DOI
- https://doi.org/10.1186/s13023-025-04150-z
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00