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Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs
The rapid expansion of the gene therapy pipeline in recent years offers significant potential to treat diseases with great unmet medical need. However, the unique nature of these therapies poses challenges to regulating them within traditional frameworks, even when developing in a single country. Va...
Autores principales: | , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8099595/ https://www.ncbi.nlm.nih.gov/pubmed/33997101 http://dx.doi.org/10.1016/j.omtm.2021.04.001 |
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author | Drago, Daniela Foss-Campbell, Betsy Wonnacott, Keith Barrett, David Ndu, Adora |
author_facet | Drago, Daniela Foss-Campbell, Betsy Wonnacott, Keith Barrett, David Ndu, Adora |
author_sort | Drago, Daniela |
collection | PubMed |
description | The rapid expansion of the gene therapy pipeline in recent years offers significant potential to treat diseases with great unmet medical need. However, the unique nature of these therapies poses challenges to regulating them within traditional frameworks, even when developing in a single country. Various factors exacerbate the issues in commercializing products across regions, including the lack of established regulatory frameworks for developing gene therapy products in many jurisdictions. While some countries have established separate regulatory frameworks for advanced therapies/regenerative medicine products, differences exist between them. Recommended solutions to overcome these hurdles include fostering convergence among countries with separate regulatory frameworks for these products and utilizing reliance and recognition for countries without such frameworks. Additionally, regulators who choose to establish new dedicated frameworks for regulating gene therapies should consider the inclusion of key elements such as expedited regulatory pathways that offer early engagement with regulators, innovative clinical trial design, and adequate post-market confirmatory studies. Increasing the alignment of regulatory pathways across countries will be crucial to facilitating the development of, and access to, gene therapies on a global scale. |
format | Online Article Text |
id | pubmed-8099595 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | American Society of Gene & Cell Therapy |
record_format | MEDLINE/PubMed |
spelling | pubmed-80995952021-05-14 Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs Drago, Daniela Foss-Campbell, Betsy Wonnacott, Keith Barrett, David Ndu, Adora Mol Ther Methods Clin Dev Review The rapid expansion of the gene therapy pipeline in recent years offers significant potential to treat diseases with great unmet medical need. However, the unique nature of these therapies poses challenges to regulating them within traditional frameworks, even when developing in a single country. Various factors exacerbate the issues in commercializing products across regions, including the lack of established regulatory frameworks for developing gene therapy products in many jurisdictions. While some countries have established separate regulatory frameworks for advanced therapies/regenerative medicine products, differences exist between them. Recommended solutions to overcome these hurdles include fostering convergence among countries with separate regulatory frameworks for these products and utilizing reliance and recognition for countries without such frameworks. Additionally, regulators who choose to establish new dedicated frameworks for regulating gene therapies should consider the inclusion of key elements such as expedited regulatory pathways that offer early engagement with regulators, innovative clinical trial design, and adequate post-market confirmatory studies. Increasing the alignment of regulatory pathways across countries will be crucial to facilitating the development of, and access to, gene therapies on a global scale. American Society of Gene & Cell Therapy 2021-04-05 /pmc/articles/PMC8099595/ /pubmed/33997101 http://dx.doi.org/10.1016/j.omtm.2021.04.001 Text en © 2021 The Author(s) https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/). |
spellingShingle | Review Drago, Daniela Foss-Campbell, Betsy Wonnacott, Keith Barrett, David Ndu, Adora Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs |
title | Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs |
title_full | Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs |
title_fullStr | Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs |
title_full_unstemmed | Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs |
title_short | Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs |
title_sort | global regulatory progress in delivering on the promise of gene therapies for unmet medical needs |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8099595/ https://www.ncbi.nlm.nih.gov/pubmed/33997101 http://dx.doi.org/10.1016/j.omtm.2021.04.001 |
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