Drug repurposing for rare : progress and opportunities for the rare disease community
Copyright © 2024 Jonker, O’Connor, Cavaller-Bellaubi, Fetro, Gogou, ’T Hoen, de Kort, Stone, Valentine and Pasmooij..
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.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2024 |
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| Erschienen: |
2024 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:11 |
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| Enthalten in: |
Frontiers in medicine - 11(2024) vom: 30., Seite 1352803 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Jonker, Anneliene Hechtelt [VerfasserIn] |
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| Links: |
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| Themen: |
Drug repurposing |
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| Anmerkungen: |
Date Revised 02.02.2024 published: Electronic-eCollection Citation Status PubMed-not-MEDLINE |
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| doi: |
10.3389/fmed.2024.1352803 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
NLM367881136 |
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| 520 | |a 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 | ||
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| 700 | 1 | |a Fetro, Christine |e verfasserin |4 aut | |
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| 700 | 1 | |a de Kort, Martin |e verfasserin |4 aut | |
| 700 | 1 | |a Stone, Heather |e verfasserin |4 aut | |
| 700 | 1 | |a Valentine, Nivedita |e verfasserin |4 aut | |
| 700 | 1 | |a Pasmooij, Anna Maria Gerdina |e verfasserin |4 aut | |
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