Zusammenfassung
Arzneimittel für neuartige Therapien wie Zell- und Gentherapeutika oder Gewebeersatz werden in den europäischen Richtlinien unter dem Begriff „Advanced Therapy Medicinal Products“ (ATMPs) zusammengefasst. ATMPs sind in ihrer Zusammensetzung und ihren Eigenschaften hoch komplex und unterliegen sich ständig weiterentwickelnden regulatorischen Rahmenbedingungen. Die vielversprechenden Ergebnisse der Grundlagenforschung wecken hohe Erwartungen an das therapeutische Potenzial dieser Produkte. Es besteht aber das Risiko, dass sie nicht die therapeutischen Effekte zeigen, die anfangs auf Basis theoretischer Überlegungen postuliert wurden. Umso wichtiger ist daher ein effizienter präklinischer und klinischer Entwicklungsplan, um den Übergang vom Labor in die erste klinische Anwendung erfolgreich zu gestalten. Aufgrund der Komplexität der ATMPs sollte dieser Entwicklungsplan frühzeitig mit den Regulierungsbehörden abgestimmt werden, um die Besonderheiten und Herausforderungen des individuellen Produktes zu definieren. Bei der Planung und Durchführung der ersten klinischen Prüfung sind – ebenfalls aufgrund der Eigenschaften der ATMPs – besondere Anforderungen zu beachten, die in diesem Beitrag diskutiert werden.
Abstract
Novel therapies, e.g., cell and gene therapy or tissue engineering, are summarized in the European Union as advanced therapy medicinal products (ATMPs). In terms of composition and product properties, ATMPs are highly complex, and given their multiple potential actions they are subject to continuously developing regulatory requirements. Due to promising basic research findings, there are high expectations by the society toward the therapeutic potential of ATMPs. It is of utmost importance to develop a scientifically sound preclinical and clinical development plan before entering into the first clinical trial. Due to the complex features of ATMPs, this development plan should be discussed early with the regulatory authorities to define the specifics and challenges of each individual product. For planning as well as operational realization of the initial clinical trial involving ATMPs, specific requirements that need to be addressed are discussed in this paper.
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Walter, C., Rohde, B., Wicke, D. et al. Regulatorischer Rahmen für neuartige Therapien. Bundesgesundheitsbl. 54, 803–810 (2011). https://doi.org/10.1007/s00103-011-1308-z
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DOI: https://doi.org/10.1007/s00103-011-1308-z