Regulation for the Translation of Gene and Cell Therapy into Medical Practice in East Asian Countries
SCIENTIFIC RELEVANCE. Currently, the Russian Federation lacks a comprehensive regulatory framework for the use of gene and cell therapy (GCT) products. There is no standard for conducting clinical trials for purposes other than marketing authorisation in Russia. In contrast, international practice s...
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| Format: | Article |
| Language: | Russian |
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NEICON ISP LLC
2024-02-01
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| Series: | Ведомости Научного центра экспертизы средств медицинского применения |
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| Online Access: | https://www.vedomostincesmp.ru/jour/article/view/615 |
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| author | E. V. Melnikova V. A. Merkulov O. V. Merkulova |
| author_facet | E. V. Melnikova V. A. Merkulov O. V. Merkulova |
| author_sort | E. V. Melnikova |
| collection | DOAJ |
| description | SCIENTIFIC RELEVANCE. Currently, the Russian Federation lacks a comprehensive regulatory framework for the use of gene and cell therapy (GCT) products. There is no standard for conducting clinical trials for purposes other than marketing authorisation in Russia. In contrast, international practice shows that, in addition to marketing authorisation, including approval based on incomplete data with post-approval commitments, there are regulatory mechanisms for the use of unregistered GCT products, such as hospital exemptions, expanded access, or compassionate use in the European Union and the USA. Relatively recently, this framework has been reformed in East Asian countries.AIM. This study aimed to analyse the regulatory mechanisms for translating GCT products into medical practice in East Asian countries and to assess the possibility of transferring elements of international experience to Russian practice.DISCUSSION. East Asian countries have adopted legislation on requirements for the manufacturing and medicinal use of GCT products. These requirements include having a mandatory license for production in accordance with Good Manufacturing Practice, consideration of the rationale for the use of GCT products by regulatory authorities or special committees, risk classification of investigational GCT products, approved registries of medical institutions authorised to use GCT products, and necessary monitoring and control of patients after GCT administration. Only cellbased innovative medicines, including genetically modified cells, are used within the framework of medical technologies (Japan, China, and Taiwan) or services (Republic of Korea), and in vivo gene therapy products can be used only in investigator-initiated clinical trials.CONCLUSIONS. The East Asian experience in translating GСT products into medical practice would be extremely useful for the Russian Federation, especially in terms of GСT use for specific indications based on accumulated clinical experience. The review suggests that it would be appropriate to establish legal provisions for investigator-initiated clinical research in Russian national legislation. |
| first_indexed | 2024-03-07T19:17:35Z |
| format | Article |
| id | doaj.art-e4c8c599afe444d8af64fbd6bb57c473 |
| institution | Directory Open Access Journal |
| issn | 1991-2919 2619-1172 |
| language | Russian |
| last_indexed | 2024-04-24T07:09:09Z |
| publishDate | 2024-02-01 |
| publisher | NEICON ISP LLC |
| record_format | Article |
| series | Ведомости Научного центра экспертизы средств медицинского применения |
| spelling | doaj.art-e4c8c599afe444d8af64fbd6bb57c4732024-04-21T11:46:35ZrusNEICON ISP LLCВедомости Научного центра экспертизы средств медицинского применения1991-29192619-11722024-02-01141294110.30895/1991-2919-2024-14-1-29-41422Regulation for the Translation of Gene and Cell Therapy into Medical Practice in East Asian CountriesE. V. Melnikova0V. A. Merkulov1O. V. Merkulova2Scientific Centre for Expert Evaluation of Medicinal ProductsScientific Centre for Expert Evaluation of Medicinal Products; I.M. Sechenov First Moscow State Medical University (Sechenov University)Scientific Centre for Expert Evaluation of Medicinal ProductsSCIENTIFIC RELEVANCE. Currently, the Russian Federation lacks a comprehensive regulatory framework for the use of gene and cell therapy (GCT) products. There is no standard for conducting clinical trials for purposes other than marketing authorisation in Russia. In contrast, international practice shows that, in addition to marketing authorisation, including approval based on incomplete data with post-approval commitments, there are regulatory mechanisms for the use of unregistered GCT products, such as hospital exemptions, expanded access, or compassionate use in the European Union and the USA. Relatively recently, this framework has been reformed in East Asian countries.AIM. This study aimed to analyse the regulatory mechanisms for translating GCT products into medical practice in East Asian countries and to assess the possibility of transferring elements of international experience to Russian practice.DISCUSSION. East Asian countries have adopted legislation on requirements for the manufacturing and medicinal use of GCT products. These requirements include having a mandatory license for production in accordance with Good Manufacturing Practice, consideration of the rationale for the use of GCT products by regulatory authorities or special committees, risk classification of investigational GCT products, approved registries of medical institutions authorised to use GCT products, and necessary monitoring and control of patients after GCT administration. Only cellbased innovative medicines, including genetically modified cells, are used within the framework of medical technologies (Japan, China, and Taiwan) or services (Republic of Korea), and in vivo gene therapy products can be used only in investigator-initiated clinical trials.CONCLUSIONS. The East Asian experience in translating GСT products into medical practice would be extremely useful for the Russian Federation, especially in terms of GСT use for specific indications based on accumulated clinical experience. The review suggests that it would be appropriate to establish legal provisions for investigator-initiated clinical research in Russian national legislation.https://www.vedomostincesmp.ru/jour/article/view/615gene and cell therapyunregistered medicinal productshospital exemptionexpanded accesscompassionate usemedical technologiesbiomedical cell product for individual medical use |
| spellingShingle | E. V. Melnikova V. A. Merkulov O. V. Merkulova Regulation for the Translation of Gene and Cell Therapy into Medical Practice in East Asian Countries Ведомости Научного центра экспертизы средств медицинского применения gene and cell therapy unregistered medicinal products hospital exemption expanded access compassionate use medical technologies biomedical cell product for individual medical use |
| title | Regulation for the Translation of Gene and Cell Therapy into Medical Practice in East Asian Countries |
| title_full | Regulation for the Translation of Gene and Cell Therapy into Medical Practice in East Asian Countries |
| title_fullStr | Regulation for the Translation of Gene and Cell Therapy into Medical Practice in East Asian Countries |
| title_full_unstemmed | Regulation for the Translation of Gene and Cell Therapy into Medical Practice in East Asian Countries |
| title_short | Regulation for the Translation of Gene and Cell Therapy into Medical Practice in East Asian Countries |
| title_sort | regulation for the translation of gene and cell therapy into medical practice in east asian countries |
| topic | gene and cell therapy unregistered medicinal products hospital exemption expanded access compassionate use medical technologies biomedical cell product for individual medical use |
| url | https://www.vedomostincesmp.ru/jour/article/view/615 |
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