Alternative statistical methods for estimating efficacy of interferon beta-1b for multiple sclerosis clinical trials

Makiko N. Mieno, Takuhiro Yamaguchi, Yasuo Ohashi

Research output: Contribution to journalArticlepeer-review

8 Citations (Scopus)

Abstract

Background: In the randomized study of interferon beta-1b (IFN beta-1b) for multiple sclerosis (MS), it has usually been evaluated the simple annual relapse rate as the study endpoint. This study aimed to investigate the performance of various regression models using information regarding the time to each recurrent event and considering the MS specific data generation process, and to estimate the treatment effect of a MS clinical trial data. Methods. We conducted a simulation study with consideration of the pathological characteristics of MS, and applied alternative efficacy estimation methods to real clinical trial data, including 5 extended Cox regression models for time-to-event analysis, a Poisson regression model and a Poisson regression model with Generalized Estimating Equations (GEE). We adjusted for other important covariates that may have affected the outcome. Results: We compared the simulation results for each model. The hazard ratios of real data were estimated for each model including the effects of other covariates. The results (hazard ratios of high-dose to low-dose) of all models were approximately 0.7 (range, 0.613 - 0.769), whereas the annual relapse rate ratio was 0.714. Conclusions: The precision of the treatment estimation was increased by application of the alternative models. This suggests that the use of alternative models that include recurrence event data may provide better analyses.

Original languageEnglish
Article number80
JournalBMC Medical Research Methodology
Volume11
DOIs
Publication statusPublished - 2011

ASJC Scopus subject areas

  • Epidemiology
  • Health Informatics

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