Comprehensive analysis of clinical development and

Authors: Sumimasa Nagai Keiya Ozawa

Publish Date: 2016/08/19

Volume: 34, Issue: 6, Pages: 777-791

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Abstract

To reduce the delay in marketing authorization of drugs in Japan four Japanese national projects were instituted We examined all oncologic drugs for adult patients approved or discussed through these schemes for the first time All the data are publicly available In total 197 applications/demands 181 indications and 16 dosages/uses were collected As of December 31 2015 64 indications and 10 dosages/uses were approved as offlabel drugs through these schemes without conducting additional registration trials in Japan Furthermore 46 indications and two dosages/uses were approved after registration trials in Japan requested by the national scheme councils Regarding the following 23 indications of the 197 applications/demands registration trials in Japan were commenced after the national scheme council’s request 17 hematological malignancies and six orphan solid tumors Moreover 54 indications and three dosages/uses for which demands were submitted were regarded as not a high medical priority by the national scheme council Regarding two hematological malignancy indications the dosage approved in foreign countries was intolerable for the Japanese patients in Japanese registration trials and this stopped the clinical development in Japan Our analysis showed that 110 indications and 12 dosages/uses were approved in Japan through these schemes These national projects have provided numerous therapeutic options for Japanese patients and may be meaningful for promoting clinical development and regulatory approval especially in orphan diseases in countries other than JapanDr Nagai was paid for consulting or an advisory role by Takara Bio Inc Dr Ozawa received grant or research funding from Takara Bio Inc and Sumitomo Dainippon Pharma Co Ltd Dr Ozawa was paid for consulting or an advisory role by JCR Pharmaceutical Inc and Celgene Japan

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