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Examining possible factors of orphan drug approval differences : A mixed-methods research
Velthuis, M. (2025) Examining possible factors of orphan drug approval differences : A mixed-methods research.
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| Abstract: | Orphan drugs are developed to treat rare diseases. Fewer than one-tenth of patients currently are able to get the treatment they need. This has severe impact on the quality of life of patients with rare diseases. This research, aimed to uncover factors influencing orphan drug approvals, was done by a mixed-methods approach that includes quantitative and qualitative research. A total of 100 novel orphan drugs were found to be approved by the FDA between 2021 and 2024. Of these, 75 were also approved in at least one of the other included agencies. The interviews that took place between May and June 2025 have resulted in eight factors that are affecting orphan drug approvals worldwide. A summary of both the quantitative and qualitative results, combined with earlier mentioned literature, were able to answer the research question: ‘What are the main factors causing differences in orphan drug approval within developing countries worldwide compared to developed countries?’ |
| Item Type: | Essay (Bachelor) |
| Faculty: | TNW: Science and Technology |
| Programme: | Health Sciences BSc (56553) |
| Link to this item: | https://purl.utwente.nl/essays/107549 |
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