Academic Journal
Japan's Pharmaceutical Regulatory Reform to Overcome Drug Loss Issue.
| Τίτλος: | Japan's Pharmaceutical Regulatory Reform to Overcome Drug Loss Issue. |
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| Συγγραφείς: | Nakai K; Pharmaceutical Evaluation Division, Pharmaceutical Safety Bureau, Ministry of Health, Labor and Welfare, 1-2-2, Kasumigaseki, Chiyoda-ku, Tokyo, 100-8916, Japan. nakai-kiyohito@mhlw.go.jp.; Pharmaceuticals and Medical Devices Agency, Shin-Kasumigaseki Bldg. 3-3-2 Kasumigaseki, Chiyoda-ku, Tokyo, 100-0013, Japan. nakai-kiyohito@mhlw.go.jp. |
| Πηγή: | Therapeutic innovation & regulatory science [Ther Innov Regul Sci] 2026 May; Vol. 60 (3), pp. 612-616. Date of Electronic Publication: 2026 Feb 14. |
| Τύπος έκδοσης: | Journal Article |
| Γλώσσα: | English |
| Στοιχεία περιοδικού: | Publisher: Springer International Publishing Country of Publication: Switzerland NLM ID: 101597411 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2168-4804 (Electronic) Linking ISSN: 21684790 NLM ISO Abbreviation: Ther Innov Regul Sci Subsets: MEDLINE |
| Imprint Name(s): | Publication: 2020- : Cham, Switzerland : Springer International Publishing Original Publication: Thousand Oaks, CA : Sage Publications, [2013]- |
| Ιατρικοί όροι (MeSH): | Drug Approval*/legislation & jurisprudence , Legislation, Drug*, Orphan Drug Production/legislation & jurisprudence ; Japan ; Humans |
| Περίληψη: | In recent years, there has been a significant increase in "drug loss"-the absence of drugs on the Japanese market that are already approved in countries outside of Japan-primarily due to the lack of their development in Japan. There were 86 drugs approved in Europe and the United States for which development had not yet begun in Japan. Of these, 48 (56%) were what can be termed 'venture-developed' drugs, 40 (47%) were orphan drugs, and 32 (37%) were for pediatric use. The lack of availability of these drugs in Japan is a significant issue.In an attempt to address this drug loss issue, the Japanese Ministry of Health, Labour and Welfare (MHLW) decided to focus on and review Japan's pharmaceutical regulations, which were considered an important factor in the drug loss issue. The MHLW held a series of meetings to discuss possible changes in its policies and methods as potential solutions to the drug loss problem. MHLW issued several notifications and related Question and Answer (Q&A) communications about those discussions. Furthermore, MHLW made revisions to the Pharmaceutical and Medical Device Act that were enacted in May 2025.This manuscript summarizes the major outcomes of these activities, particularly the issues related to clinical trials and the orphan designation system, in terms of how they relate to the future of pharmaceutical regulation in Japan. (© 2026. The Author(s), under exclusive licence to The Drug Information Association, Inc.) |
| Competing Interests: | Declarations. Conflict of interest: The authors declare no competing interests. |
| References: | Report of the Study Group on Pharmaceutical Regulations for Strengthening Drug Development and Ensuring Stable Supply, April 24, 2024 https://www.mhlw.go.jp/stf/newpage_39934.html. Report of the Revision of the Pharmaceutical and Medical Device Law, the Committee for Revision of the Pharmaceuticals and Medical Devices Regulation System, January 20, 2025 https://www.mhlw.go.jp/stf/shingi2/0000189913_00003.html . Partial Revision of “Guideline of Designation Drugs for Orphan Diseases Drug”, Notification from the Director of the Pharmaceutical Evaluation and the Director of the Medical Devices Evaluation Division, Pharmaceutical Safety Bureau, Ministry of Health, Labor and Welfare; Notification No. 0116–1, January 16, 2024 https://www.mhlw.go.jp/web/t_doc?dataId=00tc8207&dataType=1&pageNo=1 . Partial Revision of “Formulation of Development Plan for Pediatric Drugs during the Development of Drugs for Adults”, Notification from Director of the Pharmaceutical Evaluation Division, Pharmaceutical Safety Bureau, Ministry of Health, Labor and Welfare, March 29, 2024, Notification No. 0329–1 https://www.mhlw.go.jp/web/t_doc?dataId=00tc8406&dataType=1&pageNo=1 . Questions and Answers (Q&A) of “the Formulation of Development Plans for Pediatric Drugs during the Development of Drugs for Adults”, Pharmaceutical Evaluation Division, Pharmaceutical Affairs Bureau, Ministry of Health, Labor and Welfare, March 29, 2024 https://www.mhlw.go.jp/web/t_doc?dataId=00tc8407&dataType=1&pageNo=1 . Basic principles for conducting phase 1 studies in Japanese prior to initiating multi-regional clinical trials including Japan for drugs in which early clinical development is preceding outside Japan, Notification from Director of Pharmaceutical Evaluation Division, Pharmaceutical Safety Bureau, Ministry of Health, Labor and Welfare, Notification No. 1225–2, December 25, 2023 https://www.mhlw.go.jp/web/t_doc?dataId=00tc8153&dataType=1&pageNo=1 . English translation of “Basic principles for conducting phase 1 studies in Japanese prior to initiating multi-regional clinical trials including Japan for drugs in which early clinical development is preceding outside Japan”, Notification from Director of Pharmaceutical Evaluation Division, Pharmaceutical Safety Bureau, Ministry of Health, Labour and Welfare, Notification No. 1225–2, December 25, 2023 https://www.pmda.go.jp/files/000266727.pdf . Questions and Answers (Q&A) on “Basic principles for conducting phase 1 studies in Japanese prior to initiating multi-regional clinical trials including Japan for drugs in which early clinical development is preceding outside Japan”, Pharmaceutical Evaluation Division, Pharmaceutical Safety Bureau, Ministry of Health, Labor and Welfare, December 25, 2023 https://www.mhlw.go.jp/web/t_doc?dataId=00tc8154&dataType=1&pageNo=1 . Partial Revision of Questions and Answers (Q&A) of “Guidelines for Ensuring the Quality, Safety and Efficacy of Biosimilars”, Pharmaceutical Evaluation Division, Pharmaceutical Safety Bureau, Ministry of Health, Labor and Welfare, January 25 , 2024 http://www.nihs.go.jp/dbcb/TEXT/yakuseiyakushinnhatsu_0204_1_Q&A.pdf . Basic Principles Regarding Data on Japanese Subjects When Pivotal Clinical Trials for Orphan Drugs are Conducted Only Overseas, Notification from the Director of the Pharmaceutical Evaluation Division, Pharmaceutical Safety Bureau, Ministry of Health, Labor and Welfare, Notification No. 1023–3, October 23, 2024 https://www.mhlw.go.jp/hourei/doc/tsuchi/T241024I0020.pdf . Partial Revision of “Guideline of Conditional Approval for Pharmaceutical products”, Notification from Director of the Pharmaceutical Evaluation Division, Pharmaceutical Safety Bureau, Ministry of Health, Labor and Welfare, Notification No. 1023–2 , October 23, 2024 https://www.mhlw.go.jp/hourei/doc/tsuchi/T241024I0010.pdf . |
| Contributed Indexing: | Keywords: Clinical trial; Drug loss; Japan; Orphan drug designation; Pharmaceutical regulations |
| Entry Date(s): | Date Created: 20260214 Date Completed: 20260707 Latest Revision: 20260707 |
| Update Code: | 20260708 |
| DOI: | 10.1007/s43441-026-00928-w |
| PMID: | 41691137 |
| Βάση Δεδομένων: | MEDLINE |
| ISSN: | 2168-4804 |
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| DOI: | 10.1007/s43441-026-00928-w |