Drug repurposing for rare: progress and opportunities for the rare disease community
Repurposing is one of the key opportunities to address the unmet rare diseases therapeutic need. Based on cases of drug repurposing in small population conditions, and previous work in drug repurposing, we analyzed the most important lessons learned, such as the sharing of clinical observations, rea...
| Main Authors: | , , , , , , , , , |
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2024-01-01
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| Series: | Frontiers in Medicine |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fmed.2024.1352803/full |
| _version_ | 1797353627987214336 |
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| author | Anneliene Hechtelt Jonker Anneliene Hechtelt Jonker Daniel O’Connor Daniel O’Connor Maria Cavaller-Bellaubi Maria Cavaller-Bellaubi Christine Fetro Christine Fetro Maria Gogou Maria Gogou Peter A. C. ’T Hoen Peter A. C. ’T Hoen Martin de Kort Martin de Kort Heather Stone Heather Stone Nivedita Valentine Nivedita Valentine Anna Maria Gerdina Pasmooij Anna Maria Gerdina Pasmooij |
| author_facet | Anneliene Hechtelt Jonker Anneliene Hechtelt Jonker Daniel O’Connor Daniel O’Connor Maria Cavaller-Bellaubi Maria Cavaller-Bellaubi Christine Fetro Christine Fetro Maria Gogou Maria Gogou Peter A. C. ’T Hoen Peter A. C. ’T Hoen Martin de Kort Martin de Kort Heather Stone Heather Stone Nivedita Valentine Nivedita Valentine Anna Maria Gerdina Pasmooij Anna Maria Gerdina Pasmooij |
| author_sort | Anneliene Hechtelt Jonker |
| collection | DOAJ |
| description | Repurposing is one of the key opportunities to address the unmet rare diseases therapeutic need. Based on cases of drug repurposing in small population conditions, and previous work in drug repurposing, we analyzed the most important lessons learned, such as the sharing of clinical observations, reaching out to regulatory scientific advice at an early stage, and public-private collaboration. In addition, current upcoming trends in the field of drug repurposing in rare diseases were analyzed, including the role these trends could play in the rare diseases’ ecosystem. Specifically, we cover the opportunities of innovation platforms, the use of real-world data, the use of artificial intelligence, regulatory initiatives in repurposing, and patient engagement throughout the repurposing project. The outcomes from these emerging activities will help progress the field of drug repurposing for the benefit of patients, public health and medicines development. |
| first_indexed | 2024-03-08T13:33:46Z |
| format | Article |
| id | doaj.art-cc6f95ef5fa542e780aabb40d1b8199f |
| institution | Directory Open Access Journal |
| issn | 2296-858X |
| language | English |
| last_indexed | 2024-03-08T13:33:46Z |
| publishDate | 2024-01-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Medicine |
| spelling | doaj.art-cc6f95ef5fa542e780aabb40d1b8199f2024-01-17T04:33:01ZengFrontiers Media S.A.Frontiers in Medicine2296-858X2024-01-011110.3389/fmed.2024.13528031352803Drug repurposing for rare: progress and opportunities for the rare disease communityAnneliene Hechtelt Jonker0Anneliene Hechtelt Jonker1Daniel O’Connor2Daniel O’Connor3Maria Cavaller-Bellaubi4Maria Cavaller-Bellaubi5Christine Fetro6Christine Fetro7Maria Gogou8Maria Gogou9Peter A. C. ’T Hoen10Peter A. C. ’T Hoen11Martin de Kort12Martin de Kort13Heather Stone14Heather Stone15Nivedita Valentine16Nivedita Valentine17Anna Maria Gerdina Pasmooij18Anna Maria Gerdina Pasmooij19Health Technology and Services Research Department, Technical Medical Centre, University of Twente, Enschede, NetherlandsInternational Rare Diseases Research Consortium, Paris, FranceInternational Rare Diseases Research Consortium, Paris, FranceABPI, London, United KingdomInternational Rare Diseases Research Consortium, Paris, FranceEURORDIS-Rare Diseases Europe, Paris, FranceInternational Rare Diseases Research Consortium, Paris, FranceFondation Maladies Rares, Paris, FranceInternational Rare Diseases Research Consortium, Paris, FranceDepartment of Neurology, Great Ormond Street Hospital for Children, London, United KingdomInternational Rare Diseases Research Consortium, Paris, FranceDepartment of Medical BioSciences, Radboud University Medical Center, Nijmegen, NetherlandsInternational Rare Diseases Research Consortium, Paris, FranceEATRIS ERIC, European Infrastructure for Translational Medicine, Amsterdam, NetherlandsInternational Rare Diseases Research Consortium, Paris, FranceCURE ID, Office of Medical Policy, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MA, United StatesInternational Rare Diseases Research Consortium, Paris, France0Global Product Innovation, Pharmanovia, Value Added Medicines Committee, Medicines for Europe, Basildon, United KingdomInternational Rare Diseases Research Consortium, Paris, France1Dutch Medicines Evaluation Board, Utrecht, NetherlandsRepurposing is one of the key opportunities to address the unmet rare diseases therapeutic need. Based on cases of drug repurposing in small population conditions, and previous work in drug repurposing, we analyzed the most important lessons learned, such as the sharing of clinical observations, reaching out to regulatory scientific advice at an early stage, and public-private collaboration. In addition, current upcoming trends in the field of drug repurposing in rare diseases were analyzed, including the role these trends could play in the rare diseases’ ecosystem. Specifically, we cover the opportunities of innovation platforms, the use of real-world data, the use of artificial intelligence, regulatory initiatives in repurposing, and patient engagement throughout the repurposing project. The outcomes from these emerging activities will help progress the field of drug repurposing for the benefit of patients, public health and medicines development.https://www.frontiersin.org/articles/10.3389/fmed.2024.1352803/fulldrug repurposingrare diseasesorphan drugsunmet needinnovationtherapy development |
| spellingShingle | Anneliene Hechtelt Jonker Anneliene Hechtelt Jonker Daniel O’Connor Daniel O’Connor Maria Cavaller-Bellaubi Maria Cavaller-Bellaubi Christine Fetro Christine Fetro Maria Gogou Maria Gogou Peter A. C. ’T Hoen Peter A. C. ’T Hoen Martin de Kort Martin de Kort Heather Stone Heather Stone Nivedita Valentine Nivedita Valentine Anna Maria Gerdina Pasmooij Anna Maria Gerdina Pasmooij Drug repurposing for rare: progress and opportunities for the rare disease community Frontiers in Medicine drug repurposing rare diseases orphan drugs unmet need innovation therapy development |
| title | Drug repurposing for rare: progress and opportunities for the rare disease community |
| title_full | Drug repurposing for rare: progress and opportunities for the rare disease community |
| title_fullStr | Drug repurposing for rare: progress and opportunities for the rare disease community |
| title_full_unstemmed | Drug repurposing for rare: progress and opportunities for the rare disease community |
| title_short | Drug repurposing for rare: progress and opportunities for the rare disease community |
| title_sort | drug repurposing for rare progress and opportunities for the rare disease community |
| topic | drug repurposing rare diseases orphan drugs unmet need innovation therapy development |
| url | https://www.frontiersin.org/articles/10.3389/fmed.2024.1352803/full |
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