Communications for Statistical Applications and Methods

  • 제31권6호
  • /
  • Pages.613-625
  • /
  • 2024
  • /
  • 2287-7843(pISSN)
  • /
  • 2383-4757(eISSN)
한국통계학회 (The Korean Statistical Society)
권호 표지
DOI QR코드

DOI QR Code

A comparison of testing methods in non-inferiority clinical trials

  • Jieun Park (Department of Statistics, Korea University) ;
  • Jae Won Lee (Department of Statistics, Korea University)
  • 투고 : 2024.04.18
  • 심사 : 2024.10.02
  • 발행 : 2024.11.30
PDF 다운로드
⟨ 이전 논문 다음 논문 ⟩

초록

A general non-inferiority (NI) clinical trial is typically conducted using parametric testing methods with large samples. However, patient recruitment challenges often hinder rare disease trials, leading to enrollment failures. In this study, we introduce current parametric and nonparametric NI trial testing methods and propose modifications to enhance the performance of the nonparametric approach. Through a comprehensive simulation study with various sample sizes, data distributions, and sample ratios, we compare empirical levels and statistical powers as criteria for evaluating performance. Our findings indicate that the modified nonparametric methods outperformed the existing methods, particularly under conditions of small sample sizes and non-normal distributions, offering valuable insights for improving the reliability and sensitivity of NI trials in the context of rare diseases.

키워드

과제정보

This work was supported by the National Research Foundation of Korea (NRF) grant funded by the Korea government (MSIT) (No. RS-2023-00208882, No. NRF-2022M3J6A1063595). This research was also supported and funded by the Korean National Police Agency [Project Name: Advancing the Appraisal Techniques of Forensic Entomology / Project Number: PR10-04-000-22].

참고문헌

  1. Brunner E, Bathke AC, and Konietschke F (2018). Rank and Pseudo-rank Procedures for Independent Observations in Factorial Designs, Springer International Publishing, Cham, Switzerland.
  2. Brunner E, Konietschke F, Bathke AC, and Pauly M (2021). Ranks and Pseudo-ranks-Surprising results of certain rank tests in unbalanced designs, International Statistical Review, 89, 349-366.
  3. Brunner E and Puri ML (1996). 19 nonparametric methods in design and analysis of experiments, Handbook of Statistics, 13, 631-703.
  4. Domhof S (2001). Nichtparametrische relative Effekte (Ph D. thesis), University of Gottingen, Gotting en.
  5. Guideline IHT (2000). Choice of control group and related issues in clinical trials E10. Choice. E, 10, Available from: www.ich.org/fileadmin/Public Web Site/ICH Products/Guidelines/Efficacy/E10/Step4/E10 Guideline.pdf
  6. Hida E and Tango T (2011). On the three-arm non-inferiority trial including a placebo with a prespecified margin, Statistics in Medicine, 30, 224-231.
  7. Huang LC, Wen MJ, and Cheung SH (2015). Noninferiority studies with multiple new treatments and heterogeneous variances, Journal of Biopharmaceutical Statistics, 25, 958-971.
  8. Munzel U (2009). Nonparametric non-inferiority analyses in the three-arm design with active control and placebo, Statistics in Medicine, 28, 3643-3656.
  9. Park S and Kim D (2014). Nonparametric method for a non-inferiority test using confidence interval, The Korean Journal of Applied Statistics, 27, 833-842.
  10. U.S. Food and Drug Administration (2016). Non-inferiority clinical trials to establish effectiveness - guidance for industry, FDA guidance for industry, Available from: www.fda.gov/regulatoryinformation/search-fda-guidancedocuments/non-inferiority-clinical-trials-establish-effectivenessguidance-industry