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Trajectory for the Regulatory Approval of a Combination of Pertuzumab Plus Trastuzumab for Pre-treated HER2-positive Metastatic Colorectal Cancer Using Real-world Data

  • Author Footnotes
    † These authors contributed equally as first authors to this work
    Yasutoshi Sakamoto
    Footnotes
    † These authors contributed equally as first authors to this work
    Affiliations
    Translational Research Support Office, Division of Drug and Diagnostic Development Promotion, Department for the Promotion of Drug and Diagnostic Development, National Cancer Center Hospital East, Kashiwa, Chiba, Japan
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  • Author Footnotes
    † These authors contributed equally as first authors to this work
    Hideaki Bando
    Correspondence
    Address for correspondence: Dr. Hideaki Bando, Director, Division of Drug and Diagnostic Development Promotion, National Cancer Center Hospital East, 6-5-1 Kashiwanoha, Kashiwa, Chiba 277-8577, Japan.
    Footnotes
    † These authors contributed equally as first authors to this work
    Affiliations
    Translational Research Support Office, Division of Drug and Diagnostic Development Promotion, Department for the Promotion of Drug and Diagnostic Development, National Cancer Center Hospital East, Kashiwa, Chiba, Japan

    Department of Gastroenterology and Gastrointestinal Oncology, National Cancer Center Hospital East, Kashiwa, Chiba, Japan
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  • Yoshiaki Nakamura
    Affiliations
    Translational Research Support Office, Division of Drug and Diagnostic Development Promotion, Department for the Promotion of Drug and Diagnostic Development, National Cancer Center Hospital East, Kashiwa, Chiba, Japan

    Department of Gastroenterology and Gastrointestinal Oncology, National Cancer Center Hospital East, Kashiwa, Chiba, Japan

    International Research Promotion Office, Division of Drug and Diagnostic Development Promotion, Department for the Promotion of Drug and Diagnostic Development, National Cancer Center Hospital East, Kashiwa, Chiba, Japan
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  • Hiromi Hasegawa
    Affiliations
    Clinical Research Support Office, National Cancer Center Hospital East, Kashiwa, Chiba, Japan
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  • Takako Kuwaki
    Affiliations
    Research Audit Section, National Cancer Center Hospital East, Kashiwa, Chiba, Japan
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  • Wataru Okamoto
    Affiliations
    Cancer Treatment Center, Hiroshima University Hospital, Hiroshima, Japan
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  • Hiroya Taniguchi
    Affiliations
    Department of Clinical Oncology, Aichi Cancer Center, Nagoya, Aichi, Japan
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  • Yoshihiro Aoyagi
    Affiliations
    Information Technology Management Section, Clinical Research Management Division, Clinical Research Support Office, National Cancer Center Hospital East, Kashiwa, Chiba, Japan
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  • Izumi Miki
    Affiliations
    Translational Research Support Office, Division of Drug and Diagnostic Development Promotion, Department for the Promotion of Drug and Diagnostic Development, National Cancer Center Hospital East, Kashiwa, Chiba, Japan
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  • Hiroshi Uchigata
    Affiliations
    Translational Research Support Office, Division of Drug and Diagnostic Development Promotion, Department for the Promotion of Drug and Diagnostic Development, National Cancer Center Hospital East, Kashiwa, Chiba, Japan
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  • Naomi Kuramoto
    Affiliations
    Translational Research Support Office, Division of Drug and Diagnostic Development Promotion, Department for the Promotion of Drug and Diagnostic Development, National Cancer Center Hospital East, Kashiwa, Chiba, Japan
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  • Nozomu Fuse
    Affiliations
    Clinical Research Planning Division, Clinical Research Support Office, National Cancer Center Hospital East, Kashiwa, Chiba, Japan
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  • Takayuki Yoshino
    Affiliations
    Translational Research Support Office, Division of Drug and Diagnostic Development Promotion, Department for the Promotion of Drug and Diagnostic Development, National Cancer Center Hospital East, Kashiwa, Chiba, Japan

    Department of Gastroenterology and Gastrointestinal Oncology, National Cancer Center Hospital East, Kashiwa, Chiba, Japan
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  • Atsushi Ohtsu
    Affiliations
    National Cancer Center Hospital East, Kashiwa, Chiba, Japan
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  • Author Footnotes
    † These authors contributed equally as first authors to this work
Published:October 21, 2022DOI:https://doi.org/10.1016/j.clcc.2022.10.003

      Highlights

      • Usage of real-world data (RWD) for clinical implementation is gaining popularity.
      • Use of SCRUM-Japan Registry for the phase II TRIUMPH study was investigated
      • We've paved way for RWD approval in Japan
      • Future steps for building regulatory-grade registries and using RWD must be defined

      Abstract

      Utilizing real-world data (RWD) for effective clinical implementation is becoming more and more appealing as the cost of drug development rises, especially for patients with rare diseases and rare molecular subtypes for whom conducting randomized controlled trials is challenging. If a regulatory approval methodology based on RWD as an external control group can be established, drug development for rarer fractions can be accelerated by lowering costs and time, as well as reducing physical and emotional burdens on both patients and healthcare professionals. Since 2017, we have been prospectively collecting the clinical data of standard therapies in patients with rare molecular fractions under the SCRUM-Japan Registry platform, which is a qualified registry utilized as external control data for regulatory submission. Based on the results of the phase II TRIUMPH study (UMIN000027887) and the extracted data from the SCRUM-Japan Registry, the pharmaceutical company submitted an application for pertuzumab and trastuzumab in patients with HER2-positive metastatic colorectal cancer in April 2021. Pertuzumab and trastuzumab were approved as expanded indications on March 28, 2022, as 6 cases out of 14 extracted from the SCRUM-Japan Registry were classified and utilized as “evaluation material” under the review process of the Pharmaceuticals and Medical Devices Agency (PMDA). Through the TRIUMPH study and the SCRUM-Japan Registry, we have paved the way for regulatory approval of RWD in Japan. In future, we must define the steps for constructing regulatory-grade registries and the method/process for utilizing RWD by accumulating case experiences.

      Keywords

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