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RNA-based drugs and regulation: Toward a necessary evolution of the definitions issued from the European union legislation

Many RNA-based drugs, both vaccines and non-vaccines, are under development or even approved. They include coding mRNAs and non-coding (nc) RNAs among them antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), micro-RNAs (miRNAs), small activating RNAs (saRNAs), RNA aptamers and RNA gu...

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Detalles Bibliográficos
Autores principales: Guerriaud, Mathieu, Kohli, Evelyne
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9618588/
https://www.ncbi.nlm.nih.gov/pubmed/36325384
http://dx.doi.org/10.3389/fmed.2022.1012497
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author Guerriaud, Mathieu
Kohli, Evelyne
author_facet Guerriaud, Mathieu
Kohli, Evelyne
author_sort Guerriaud, Mathieu
collection PubMed
description Many RNA-based drugs, both vaccines and non-vaccines, are under development or even approved. They include coding mRNAs and non-coding (nc) RNAs among them antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), micro-RNAs (miRNAs), small activating RNAs (saRNAs), RNA aptamers and RNA guides. According to the European Union (EU) legislation, these products can be currently categorized into different regulatory statuses, depending, for vaccines, on their target (infectious disease or not) and, for other drugs, on how they are obtained (chemically or biologically). This classification is fundamental to the type of marketing authorization (MA), and therefore to the controls to be performed, from preclinical stages through clinical trials to pharmacovigilance, to meet the safety requirements for patients. However, the current rules raise several problems, in particular the risk, because technology is evolving, to have similar RNA drugs being covered by very different legal statuses and the lack of international harmonization. The objectives of this study are (i) to review how RNA medicinal products are currently legally categorized in the EU and especially whether they fall under the status of gene therapy medicinal products (GTMP), a regulatory status belonging to advanced therapy medicinal products (ATMP), (ii) to discuss the issues generated by this classification, with a focus on the heterogeneity of statuses of these products, the differences with the American and ICH definitions and the potential impact on the safety requirements.
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spelling pubmed-96185882022-11-01 RNA-based drugs and regulation: Toward a necessary evolution of the definitions issued from the European union legislation Guerriaud, Mathieu Kohli, Evelyne Front Med (Lausanne) Medicine Many RNA-based drugs, both vaccines and non-vaccines, are under development or even approved. They include coding mRNAs and non-coding (nc) RNAs among them antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), micro-RNAs (miRNAs), small activating RNAs (saRNAs), RNA aptamers and RNA guides. According to the European Union (EU) legislation, these products can be currently categorized into different regulatory statuses, depending, for vaccines, on their target (infectious disease or not) and, for other drugs, on how they are obtained (chemically or biologically). This classification is fundamental to the type of marketing authorization (MA), and therefore to the controls to be performed, from preclinical stages through clinical trials to pharmacovigilance, to meet the safety requirements for patients. However, the current rules raise several problems, in particular the risk, because technology is evolving, to have similar RNA drugs being covered by very different legal statuses and the lack of international harmonization. The objectives of this study are (i) to review how RNA medicinal products are currently legally categorized in the EU and especially whether they fall under the status of gene therapy medicinal products (GTMP), a regulatory status belonging to advanced therapy medicinal products (ATMP), (ii) to discuss the issues generated by this classification, with a focus on the heterogeneity of statuses of these products, the differences with the American and ICH definitions and the potential impact on the safety requirements. Frontiers Media S.A. 2022-10-17 /pmc/articles/PMC9618588/ /pubmed/36325384 http://dx.doi.org/10.3389/fmed.2022.1012497 Text en Copyright © 2022 Guerriaud and Kohli. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Medicine
Guerriaud, Mathieu
Kohli, Evelyne
RNA-based drugs and regulation: Toward a necessary evolution of the definitions issued from the European union legislation
title RNA-based drugs and regulation: Toward a necessary evolution of the definitions issued from the European union legislation
title_full RNA-based drugs and regulation: Toward a necessary evolution of the definitions issued from the European union legislation
title_fullStr RNA-based drugs and regulation: Toward a necessary evolution of the definitions issued from the European union legislation
title_full_unstemmed RNA-based drugs and regulation: Toward a necessary evolution of the definitions issued from the European union legislation
title_short RNA-based drugs and regulation: Toward a necessary evolution of the definitions issued from the European union legislation
title_sort rna-based drugs and regulation: toward a necessary evolution of the definitions issued from the european union legislation
topic Medicine
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9618588/
https://www.ncbi.nlm.nih.gov/pubmed/36325384
http://dx.doi.org/10.3389/fmed.2022.1012497
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