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Analysis and comparative evaluation of expedited programs for gene therapy products: insights from the United States, the European Union, Japan, and South Korea

Gene Therapy (2024)Cite this article

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Abstract

Gene therapy products (GTPs) used for incurable diseases can be expedited for early commercialization to fulfill unmet needs. This study analyzed the expedited programs available for GTPs in the US, EU, Japan, and South Korea using their regulatory authorities’ websites, related regulations, and documents. In total, there were five expedited programs available for GTPs in the US, four in the EU, and three in both Japan and South Korea, of which four are tailored for GTPs. These programs, sharing similar objectives, can be categorized as those expediting drug development, review, and approval. However, variations are observed in eligibility criteria, specific benefits, and post-marketing study conditions across regulatory authorities. Additionally, the criteria for orphan drug designation for a rare disease differs in prevalence thresholds, incentive offered, and marketing exclusivity period. Overall, 19 GTPs were approved–13 in the US, 14 in the EU, eight in Japan, and three in South Korea–with majority obtaining regulatory approval through at least one expedited program. Therefore, future studies can analyze whether acquiring multiple expedited programs accelerates the drug development and commercialization of GTPs compared with when only one expedited program is processed. Additionally, inter-authority scientific discussion is encouraged for harmonization of expedited program requirements.

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Fig. 1: Marketing authorization procedure for gene therapy products under expedited programs in investigated regulatory authorities.
Fig. 2: Comparative analysis of the characteristics of expedited programs for gene therapy products offered by regulatory authorities.
Fig. 3: Global status of approved gene therapy products.
Fig. 4: Expedited programs implemented to approved gene therapy products in the US, EU, Japan, and South Korea.

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All data analyzed during this study are included in this published article.

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Acknowledgements

We are grateful to J.S.D. Jeehye Park (Yu Jun law) for her helpful comments about the regulations and our colleague Ms. Sua Oh for providing ideas to draw the figure.

Funding

This research was supported by a grant from the National Research Foundation of Korea (NRF), funded by the Korean government (Ministry of Science and ICT, MICT)(NRF2021R1F1A1062044), by the Basic Science Research Program through the National Research Foundation of Korea (NRF) funded by the Ministry of Education (grant number 2021R1A6A1A03044296), and by a grant (22183MFDS366) from Ministry of Food and Drug Safety of South Korea in 2022–2025, which had no role in the study design, data collection, analysis, or interpretation, the writing of the report, or in the decision to submit the paper for publication.

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Author notes

  1. These authors contributed equally: Hyeokgyo Jeong, Sujata Purja.

Authors and Affiliations

  1. Pharmaceutical Licensing, Department of Pharmaceutical Industry, Chung-Ang University, 06974, Seoul, South Korea

    Hyeokgyo Jeong & Eunyoung Kim

  2. Data Science, Evidence-Based and Clinical Research Laboratory, Department of Health, Social, and Clinical Pharmacy, College of Pharmacy, Chung-Ang University, 06974, Seoul, South Korea

    Sujata Purja & Eunyoung Kim

  3. Central Research Center of Epigenome based platform and its Application for Drug Development, College of Pharmacy, Chung-Ang University, 06974, Seoul, South Korea

    Sujata Purja & Eunyoung Kim

  4. Regulatory Science Policy, Pharmaceutical Regulatory Sciences, Chung-Ang University, 06974, Seoul, South Korea

    Eunyoung Kim

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H.J., S.P. and E.K. were responsible for study conception and design. H.J. and S.P. were responsible for acquisition of the data and data analysis. H.J., S.P., and E.K. were responsible for drafting and revision of the manuscript.

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Correspondence to Eunyoung Kim.

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Jeong, H., Purja, S. & Kim, E. Analysis and comparative evaluation of expedited programs for gene therapy products: insights from the United States, the European Union, Japan, and South Korea. Gene Ther (2024). https://doi.org/10.1038/s41434-023-00437-7

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