Clinical trial design for rare cancer

• Teramukai Satoshi

  Department of Biostatistics, Kyoto Prefectural University of Medicine Graduate School of Medical Science

Bibliographic Information

Other Title

• 希少がんに対する臨床試験デザイン

Abstract

How to manage clinical trials for rare cancer including pediatric cancer is a burning issue globally involving Japanese regulatory authorities. The conditions that can loosen limitations in study designs are as follows: (1) allowance for a one-arm trial design, (2) setting a surrogate endpoint (e.g., response rate), and (3) increasing type-I or type-II error probabilities. Also, several efficient study designs are suggested, such as study designs based on genomic information (e.g., umbrella design and basket design), N-of-1 design, adaptive designs, and Bayesian designs. The advantages of the Bayesian approach are as follows: (1) the interpretation for the probability is intuitive, (2) the flexibility that enables the probability update during the study is suitable for adaptive design, and (3) the variety of prior beliefs, which sometimes have clinically important meanings, can be explicitly integrated as a prior distribution. A trade-off exists between scientific validity and efficiency in trial designs. Therefore, the pros and cons of each design should be thoroughly discussed at the planning stage. Effective collaborations between clinicians and statisticians are necessary to improve the quality of trial design.

Journal

• The Japanese Journal of Pediatric Hematology / Oncology

  The Japanese Journal of Pediatric Hematology / Oncology 56 (5), 425-428, 2019

  The Japanese Society of Pediatric Hematology / Oncology

Keywords

• clinical trial
• rare cancer
• rare disease
• adaptive design
• Bayesian design

Details

• CRID: 1390283659850127232
• NII Article ID: 130007795826
• DOI: 10.11412/jspho.56.425
• ISSN: 21895384
• ISSN: 2187011X
• Text Lang: ja
• Data Source:
  • JaLC
  • CiNii Articles
  • KAKEN
• Abstract License Flag: Disallowed