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Approving molecularly targeted drugs: different approval processes for cytotoxic agents

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Abstract

Background

Recently, the approval of some molecularly targeted drugs has been questioned, due to differing opinions on their risks and benefits. The approval process remains a challenge in regulatory science.

Methods

We analyzed the molecularly targeted drugs listed in the 2013 Medical Formulary. For the 21 identified drugs, 32 published Pharmaceuticals and Medical Devices Agency (PMDA) reports were open to the public. Data regarding clinical trials were extracted from these reports and assessed in order to clarify the characteristic examinations required for the approval of molecularly targeted drugs.

Results

There was no correlation between the application year and the time between application and approval (p = 0.139). The median number of clinical trials in these reports was 5 (range 1–22). Phase III studies were not included in the assessment materials for 11 reports. A survival benefit was demonstrated for six of the 32 drugs. The PMDA issued approval terms, including all-case surveillance and additional clinical trials, for 24 of these 32 drugs.

Conclusion

Molecularly targeted drugs were approved by the PMDA using a flexible process based on drug safety and efficacy. Doctors and patients who are administering or receiving these drugs should be fully informed about the lack of Japanese data assessment during these approval processes.

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Acknowledgments

This work was supported by JSPS KAKENHI Grant Number 25670240.

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Authors and Affiliations

  1. Department of Medical Oncology, Yamagata Prefectural Central Hospital, 1800 Aoyagi, Yamagata, Yamagata, Japan

    Yuriko Sasahara

  2. Cancer Prevention and Control Division, Kanagawa Cancer Center Research Institute, 2-3-2 Nakao, Asahi-ku, Yokohama, Kanagawa, 241-8515, Japan

    Hiroto Narimatsu

  3. Department of Public Health, Yamagata University Graduate School of Medicine, Yamagata, Japan

    Hiroto Narimatsu & Akira Fukao

  4. Department of Clinical Oncology, Yamagata University, Yamagata, Japan

    Takashi Yoshioka

Authors
  1. Yuriko Sasahara
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  2. Hiroto Narimatsu
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  3. Akira Fukao
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  4. Takashi Yoshioka
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Corresponding author

Correspondence to Yuriko Sasahara.

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The authors declare that they have no conflict of interest.

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Sasahara, Y., Narimatsu, H., Fukao, A. et al. Approving molecularly targeted drugs: different approval processes for cytotoxic agents. Int J Clin Oncol 21, 1004–1013 (2016). https://doi.org/10.1007/s10147-016-0980-2

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  • DOI: https://doi.org/10.1007/s10147-016-0980-2

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