Quantifying hope: an EU perspective of rare disease therapeutic space and market dynamics
Rare diseases, affecting millions globally, pose a significant healthcare burden despite impacting a small population. While approximately 70% of all rare diseases are genetic and often begin in childhood, diagnosis remains slow and only 5% have approved treatments. The UN emphasizes improved access...
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2025-02-01
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| Series: | Frontiers in Public Health |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fpubh.2025.1520467/full |
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| author | Emmanuelle Cacoub Emmanuelle Cacoub Nathalie Barreto Lefebvre Dimitrije Milunov Manish Sarkar Soham Saha |
| author_facet | Emmanuelle Cacoub Emmanuelle Cacoub Nathalie Barreto Lefebvre Dimitrije Milunov Manish Sarkar Soham Saha |
| author_sort | Emmanuelle Cacoub |
| collection | DOAJ |
| description | Rare diseases, affecting millions globally, pose a significant healthcare burden despite impacting a small population. While approximately 70% of all rare diseases are genetic and often begin in childhood, diagnosis remains slow and only 5% have approved treatments. The UN emphasizes improved access to primary care (diagnostic and potentially therapeutic) for these patients and their families. Next-generation sequencing (NGS) offers hope for earlier and more accurate diagnoses, potentially leading to preventative measures and targeted therapies. In here, we explore the therapeutic landscape for rare diseases, analyzing drugs in development and those already approved by the European Medicines Agency (EMA). We differentiate between orphan drugs with market exclusivity and repurposed existing drugs, both crucial for patients. By analyzing market size, segmentation, and publicly available data, this comprehensive study aims to pave the way for improved understanding of the treatment landscape and a wider knowledge accessibility for rare disease patients. |
| format | Article |
| id | doaj-art-4da2bf3300fe474b9205b7d0a2d7f2e2 |
| institution | Kabale University |
| issn | 2296-2565 |
| language | English |
| publishDate | 2025-02-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Public Health |
| spelling | doaj-art-4da2bf3300fe474b9205b7d0a2d7f2e22025-02-03T06:33:54ZengFrontiers Media S.A.Frontiers in Public Health2296-25652025-02-011310.3389/fpubh.2025.15204671520467Quantifying hope: an EU perspective of rare disease therapeutic space and market dynamicsEmmanuelle Cacoub0Emmanuelle Cacoub1Nathalie Barreto Lefebvre2Dimitrije Milunov3Manish Sarkar4Soham Saha5Medinsights SAS, Paris, FranceESCP Business School, Paris, FranceMedinsights SAS, Paris, FranceMedinsights SAS, Paris, FranceMedinsights SAS, Paris, FranceMedinsights SAS, Paris, FranceRare diseases, affecting millions globally, pose a significant healthcare burden despite impacting a small population. While approximately 70% of all rare diseases are genetic and often begin in childhood, diagnosis remains slow and only 5% have approved treatments. The UN emphasizes improved access to primary care (diagnostic and potentially therapeutic) for these patients and their families. Next-generation sequencing (NGS) offers hope for earlier and more accurate diagnoses, potentially leading to preventative measures and targeted therapies. In here, we explore the therapeutic landscape for rare diseases, analyzing drugs in development and those already approved by the European Medicines Agency (EMA). We differentiate between orphan drugs with market exclusivity and repurposed existing drugs, both crucial for patients. By analyzing market size, segmentation, and publicly available data, this comprehensive study aims to pave the way for improved understanding of the treatment landscape and a wider knowledge accessibility for rare disease patients.https://www.frontiersin.org/articles/10.3389/fpubh.2025.1520467/fullrare diseasesorphan drug designationsEuropean Medicines Agencymarket sharerevenue modelpharmaceuticals |
| spellingShingle | Emmanuelle Cacoub Emmanuelle Cacoub Nathalie Barreto Lefebvre Dimitrije Milunov Manish Sarkar Soham Saha Quantifying hope: an EU perspective of rare disease therapeutic space and market dynamics Frontiers in Public Health rare diseases orphan drug designations European Medicines Agency market share revenue model pharmaceuticals |
| title | Quantifying hope: an EU perspective of rare disease therapeutic space and market dynamics |
| title_full | Quantifying hope: an EU perspective of rare disease therapeutic space and market dynamics |
| title_fullStr | Quantifying hope: an EU perspective of rare disease therapeutic space and market dynamics |
| title_full_unstemmed | Quantifying hope: an EU perspective of rare disease therapeutic space and market dynamics |
| title_short | Quantifying hope: an EU perspective of rare disease therapeutic space and market dynamics |
| title_sort | quantifying hope an eu perspective of rare disease therapeutic space and market dynamics |
| topic | rare diseases orphan drug designations European Medicines Agency market share revenue model pharmaceuticals |
| url | https://www.frontiersin.org/articles/10.3389/fpubh.2025.1520467/full |
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