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

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.

• Authors: Makiko N Mieno, Takuhiro Yamaguchi, Yasuo Ohashi
• Journal: BMC medical research methodology (BMC Med Res Methodol) Vol. 11 Pg. 80 (May 26 2011) ISSN: 1471-2288 [Electronic] England
• PMID: 21612661 (Publication Type: Journal Article)

Chemical References

• Adjuvants, Immunologic
• Interferon beta-1b
• Interferon-beta

Topics

• Adjuvants, Immunologic (therapeutic use)
• Clinical Trials as Topic
• Data Interpretation, Statistical
• Female
• Humans
• Interferon beta-1b
• Interferon-beta (therapeutic use)
• Male
• Multiple Sclerosis (drug therapy)
• Poisson Distribution
• Proportional Hazards Models
• Regression Analysis
• Treatment Outcome