Characteristics of Clinical Trials for the US Food and Drug Administration Accelerated Approval and Subsequent Confirmatory Trials: Implications for Drug Approval in Japan

Author:

Mamiya Hiroaki1,Narukawa Mamoru2

Affiliation:

1. Department of Pharmaceutical Sciences, School of Pharmacy at Narita, International University of Health and Welfare

2. Department of Clinical Medicine (Pharmaceutical Medicine), Kitasato University Graduate School of Pharmaceutical Sciences

Publisher

Pharmaceutical Society of Japan

Reference19 articles.

1. 1) FDA. “21 CFR 314.510 -- Approval based on a surrogate endpoint or on an effect on a clinical endpoint other than survival or irreversible morbidity”: ‹https://www.ecfr.gov/current/title-21/chapter-I/subchapter-D/part-314/subpart-H/section-314.510›, accessed 25 January, 2024.

2. 2) “21 CFR 601.41 -- Approval based on a surrogate endpoint or on an effect on a clinical endpoint other than survival or irreversible morbidity”: ‹https://www.ecfr.gov/current/title-21/chapter-I/subchapter-F/part-601/subpart-E/section-601.41›, accessed 25 January, 2024.

3. 3) US Food and Drug Administration. “Center for Drug Evaluation. Research. Expedited programs for serious conditions––drugs and biologics (2022).”: ‹https://www.fda.gov/regulatory-information/search-fda-guidance-documents/expedited-programs-serious-conditions-drugs-and-biologics›, accessed 25 January, 2024.

4. 4) Gyawali B, Hey SP, Kesselheim AS. Assessment of the clinical benefit of cancer drugs receiving accelerated approval. JAMA Intern. Med., 179, 906–913 (2019).

5. 5) Beaver JA, Howie LJ, Pelosof L, Kim T, Liu J, Goldberg KB, Sridhara R, Blumenthal GM, Farrell AT, Keegan P, Pazdur R, Kluetz PG. A 25-year experience of US Food and Drug Administration accelerated approval of malignant hematology and oncology drugs and biologics: a review. JAMA Oncol., 4, 849–856 (2018).

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