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| Verfasst von: | Stacchiotti, Silvia [VerfasserIn]  |
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| | Bouche, Gauthier [VerfasserIn] |
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| | Herold, Ralf [VerfasserIn] |
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| | Pantziarka, Pan [VerfasserIn] |
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| | Schuster, Kathrin [VerfasserIn] |
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| | Wilson, Roger [VerfasserIn] |
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| | Pignatti, Francesco [VerfasserIn] |
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| | Kasper, Bernd [VerfasserIn] |
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| Titel: | How to develop new systemic treatments in ultra-rare cancers with high unmet needs? |
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| Titelzusatz: | The case of alveolar soft-part sarcoma |
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| Verf.angabe: | Silvia Stacchiotti, Gauthier Bouche, Ralf Herold, Pan Pantziarka, Kathrin Schuster, Roger Wilson, Francesco Pignatti, Bernd Kasper |
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| E-Jahr: | 2024 |
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| Jahr: | May 2024 |
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| Umfang: | 5 S. |
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| Fussnoten: | Gesehen am 11.09.2024 |
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| Titel Quelle: | Enthalten in: European journal of cancer |
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| Ort Quelle: | Amsterdam [u.a.] : Elsevier, 1992 |
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| Jahr Quelle: | 2024 |
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| Band/Heft Quelle: | 202(2024) vom: Mai, Artikel-ID 114003, Seite [1]-5 |
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| ISSN Quelle: | 1879-0852 |
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| Abstract: | Developing new drugs or generating evidence for existing drugs in new indications for ultra-rare cancers is complex and carries a high-risk of failure. This gets even harder in ultra-rare tumours, which have an annual incidence of 1 per 1,000,000 population or less. Here, we illustrate the problem of adequate evidence generation in ultra-rare tumours, using Alveolar Soft-Part Sarcomas (ASPS) - an ultra-rare sarcoma newly diagnosed in approximately 60 persons a year in the European Union - as an exemplar case showing challenges in development despite being potentially relevant for classes of agents. We discuss some possible approaches for addressing such challenges, especially focussing on constructive collaboration between academic groups, patients and advocates, drug manufacturers, and regulators to optimise drug development in ultra-rare cancers. This article, written by various European stakeholders, proposes a way forward to ultimately get better options for patients with ultra-rare cancers. |
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| DOI: | doi:10.1016/j.ejca.2024.114003 |
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| URL: | Bitte beachten Sie: Dies ist ein Bibliographieeintrag. Ein Volltextzugriff für Mitglieder der Universität besteht hier nur, falls für die entsprechende Zeitschrift/den entsprechenden Sammelband ein Abonnement besteht oder es sich um einen OpenAccess-Titel handelt.
Volltext: https://doi.org/10.1016/j.ejca.2024.114003 |
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| | Volltext: https://www.sciencedirect.com/science/article/pii/S095980492400159X |
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| | DOI: https://doi.org/10.1016/j.ejca.2024.114003 |
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| Datenträger: | Online-Ressource |
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| Sprache: | eng |
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| Sach-SW: | Clinical trials |
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| | Drug development |
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| | Sarcomas |
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| | Ultra-rare cancers |
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| K10plus-PPN: | 1902322231 |
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| Verknüpfungen: | → Zeitschrift |
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How to develop new systemic treatments in ultra-rare cancers with high unmet needs? / Stacchiotti, Silvia [VerfasserIn]; May 2024 (Online-Ressource)
