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On the clinical trial design and statistical analysis in development of orphan drug for rare diseases

Research Project

Project/Area Number 16K00049
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeMulti-year Fund
Section一般
Research Field Statistical science
Research InstitutionOsaka University (2017-2018)
Hiroshima University (2016)

Principal Investigator

Hida Eisuke  大阪大学, 医学系研究科, 特任教授(常勤) (30469952)

Research Collaborator TANGO toshiro  
Project Period (FY) 2016-04-01 – 2019-03-31
Project Status Completed (Fiscal Year 2018)
Budget Amount *help
¥4,550,000 (Direct Cost: ¥3,500,000、Indirect Cost: ¥1,050,000)
Fiscal Year 2018: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
Fiscal Year 2017: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2016: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Keywordssmall clinical trial / 臨床試験デザイン / 分析感度 / 非劣性試験 / Network Meta-Analysis / 検証的試験 / Small Clinical Trials / 医学統計学 / 医薬品開発 / 希少疾病
Outline of Final Research Achievements

As a result of this research, a new trial design and evaluation method for SCTs focusing on non-inferiority trials was proposed. In particular, assay sensitivity in non-inferiority trials must be evaluated from external information, because there is no internal control that can be evaluated directly. Therefore, we proposed two methods that can evaluate non-inferiority with the assay sensitivity. One is the proposal of the analysis method and the sample size calculation for the 3-arm non-inferiority trial including the placebo group. The results were published in Pharmaceutical Statistics. The other is the consideration of the method to evaluate the analysis sensitivity indirectly using Network Meta-Analysis, we reported the results at domestic and international conferences.

Academic Significance and Societal Importance of the Research Achievements

本研究は,SCTsという適切な規模での臨床試験の実施が困難な場合における問題点を解決する新たな臨床試験デザインを提案することが目的である.非劣性試験はSCTsにおいても重要な比較デザインであり,非劣性試験が有する問題点である分析感度の保証に関しての新たなアプローチの提案は,SCTsに限定されることなく全ての疾患領域において,新治療薬に対する有効性を適切に評価することが可能なアプローチである.したがって,本研究により提案法を利用することで,画期的な治療の開発期間が短縮でき,有用な治療が早期承認されることに繋がることが予想される.

Report

(4 results)
  • 2018 Annual Research Report   Final Research Report ( PDF )
  • 2017 Research-status Report
  • 2016 Research-status Report
  • Research Products

    (13 results)

All 2019 2018 2017

All Journal Article (7 results) (of which Int'l Joint Research: 3 results,  Peer Reviewed: 7 results,  Open Access: 7 results) Presentation (6 results) (of which Int'l Joint Research: 4 results)

  • [Journal Article] A Novel Smoking Cessation Smartphone App Integrated With a Mobile Carbon Monoxide Checker for Smoking Cessation Treatment: Protocol for a Randomized Controlled Trial2019

    • Author(s)
      Nomura Akihiro、Tateno Hiroki、Masaki Katsunori、Muto Tomoyasu、Suzuki Shin、Satake Kohta、Hida Eisuke、Fukunaga Koichi
    • Journal Title

      JMIR Research Protocols

      Volume: 8 Issue: 2 Pages: e12252-e12252

    • DOI

      10.2196/12252

    • Related Report
      2018 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Design and analysis of a 3-arm noninferiority trial with a prespecified margin for the hazard ratio2018

    • Author(s)
      Hida Eisuke、Tango Toshiro
    • Journal Title

      Pharmaceutical Statistics

      Volume: 17 Issue: 5 Pages: 489-503

    • DOI

      10.1002/pst.1875

    • Related Report
      2018 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Endothelial dysfunction, abnormal vascular structure and lower urinary tract symptoms in men and women2018

    • Author(s)
      Matsui Shogo、Kajikawa Masato、Maruhashi Tatsuya、Iwamoto Yumiko、Oda Nozomu、Kishimoto Shinji、Hashimoto Haruki、Hidaka Takayuki、Kihara Yasuki、Chayama Kazuaki、Hida Eisuke、Goto Chikara、Aibara Yoshiki、Nakashima Ayumu、Yusoff Farina Mohamad、Noma Kensuke、Kuwahara Yoshitaka、Matsubara Akio、Higashi Yukihito
    • Journal Title

      International Journal of Cardiology

      Volume: 261 Pages: 196-203

    • DOI

      10.1016/j.ijcard.2018.02.041

    • Related Report
      2018 Annual Research Report
    • Peer Reviewed / Open Access / Int'l Joint Research
  • [Journal Article] Interrelationships Among Flow‐Mediated Vasodilation, Nitroglycerine‐Induced Vasodilation, Baseline Brachial Artery Diameter, Hyperemic Shear Stress, and Cardiovascular Risk Factors2018

    • Author(s)
      Tatsuya Maruhashi, Yumiko Iwamoto, Masato Kajikawa, Nozomu Oda, Shinji Kishimoto, Shogo Matsui, Haruki Hashimoto, Yoshiki Aibara, Farina Mohamad Yusoff, Takayuki Hidaka, Yasuki Kihara, Kazuaki Chayama, Kensuke Noma, Ayumu Nakashima, Chikara Goto, Eisuke Hida, Yukihito Higashi
    • Journal Title

      Journal of the American Heart Association

      Volume: 7 Issue: 1 Pages: 1-9

    • DOI

      10.1161/jaha.117.006797

    • Related Report
      2017 Research-status Report
    • Peer Reviewed / Open Access / Int'l Joint Research
  • [Journal Article] Segmentectomy Versus Lobectomy for Radiologically Pure Solid Clinical T1a-bN0M0 Lung Cancer2018

    • Author(s)
      Tsubokawa Norifumi、Tsutani Yasuhiro、Miyata Yoshihiro、Handa Yoshinori、Misumi Keizo、Hanaki Hideaki、Hida Eisuke、Okada Morihito
    • Journal Title

      World Journal of Surgery

      Volume: Feb. Issue: 8 Pages: 1-9

    • DOI

      10.1007/s00268-018-4514-0

    • Related Report
      2017 Research-status Report
    • Peer Reviewed / Open Access
  • [Journal Article] Optimal Target Level of Low-density Lipoprotein Cholesterol for Vascular Function in Statin Naive Individuals2017

    • Author(s)
      Matsui Shogo、Kajikawa Masato、Hida Eisuke、Maruhashi Tatsuya、Iwamoto Yumiko、Iwamoto Akimichi、Oda Nozomu、Kishimoto Shinji、Hidaka Takayuki、Kihara Yasuki、Chayama Kazuaki、Goto Chikara、Aibara Yoshiki、Nakashima Ayumu、Yusoff Farina Binti Mohamad、Noma Kensuke、Higashi Yukihito
    • Journal Title

      Scientific Reports

      Volume: 7 Issue: 1 Pages: 1-10

    • DOI

      10.1038/s41598-017-09043-1

    • Related Report
      2017 Research-status Report
    • Peer Reviewed / Open Access / Int'l Joint Research
  • [Journal Article] Temporal Processing Instability with Millisecond Accuracy is a Cardinal Feature of Sensorimotor Impairments in Autism Spectrum Disorder: Analysis Using the Synchronized Finger-Tapping Task2017

    • Author(s)
      Morimoto Chie、Hida Eisuke、Shima Keisuke、Okamura Hitoshi
    • Journal Title

      Journal of Autism and Developmental Disorders

      Volume: 48 Issue: 2 Pages: 351-360

    • DOI

      10.1007/s10803-017-3334-7

    • Related Report
      2017 Research-status Report
    • Peer Reviewed / Open Access
  • [Presentation] RWDを用いた非劣性の検証について2019

    • Author(s)
      飛田英祐
    • Organizer
      RIMS共同研究 最尤法とベイズ法
    • Related Report
      2018 Annual Research Report
  • [Presentation] One approach to the assurance of assay sensitivity in non-inferiority trials with network meta-analysis2018

    • Author(s)
      Hida E, Tango T
    • Organizer
      The Joint International Society for Clinical Biostatistics and Australian Statistical Conference
    • Related Report
      2018 Annual Research Report
    • Int'l Joint Research
  • [Presentation] Consideration of three-arm non-inferiority trial design and analysis with a prespecified margin for a survival endpoint2018

    • Author(s)
      Hida E, Tango T
    • Organizer
      XXIXth International Biometric Conference
    • Related Report
      2018 Annual Research Report
    • Int'l Joint Research
  • [Presentation] Consideration of hybrid non-inferiority trials design using Network-Meta-Analysis2017

    • Author(s)
      Eisuke Hida
    • Organizer
      38th Annual Conference of the International Society for Clinical Biostatistics
    • Related Report
      2017 Research-status Report
    • Int'l Joint Research
  • [Presentation] The design of non-inferiority trial with assay sensitivity using Network Meta-Analysis2017

    • Author(s)
      Eisuke Hida
    • Organizer
      Joint Conference on Biometrics & Biopharmaceutical Statistics
    • Related Report
      2017 Research-status Report
    • Int'l Joint Research
  • [Presentation] Network-Meta-Analysis を利用した非劣性試験デザインの検討2017

    • Author(s)
      飛田英祐
    • Organizer
      2017年度日本計量生物学会年会
    • Place of Presentation
      中央大学後楽園キャンパス
    • Related Report
      2016 Research-status Report

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Published: 2016-04-21   Modified: 2020-03-30  

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