50 Result(s)
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Christy Chuang-Stein
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Article
Rejoinder
This is a rejoinder to the preceding commentaries on the authors’ paper “Surrogate Endpoints in AIDS Drug Development: Current Status.” The authors thank Drs. Ellenberg and Gould for their thought-provoking co...
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Article
Surrogate Endpoints in Aids Drug Development: Current Status
During the past several years, significant efforts have been devoted to searching for surrogate endpoints to evaluate AIDS treatments. Efforts were made in the form of research sponsored by the government and ...
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Article
Testing for Superiority or Inferiority after Concluding Equivalence?
In this paper, we examine the issue of testing for superiority or inferiority following the equivalence conclusion. We contrast this situation with that of testing for superiority after a noninferiority conclu...
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Article
Some Issues Concerning the Normalization of Laboratory Data Based on Reference Ranges
In this paper, we examine three issues associated with normalizing lab data based on reference ranges: normalized values being negative, lab values being excluded because of missing information on reference ra...
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Article
Recent Advancements in the Analysis and Presentation of Safety Data
The importance of a favorable safety profile to the overall success of a pharmaceutical product is well known. While most of the analysis of safety data is routine and exploratory in nature, we will discuss so...
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Article
Response from the Original Author
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Article
Component Scores, or Total Scores over Components?
Patients’ responses to items on a questionnaire are frequently used to assess treatment effects in clinical trials. In this paper, we will use the Unified Parkinson’s Disease Rating Scale to address a common q...
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Chapter
Analysis of Dose–Response Relationship Based on Categorical Outcomes
In an eloquent article prepared in defense of the dichotomy, Lewis (2004) wrote that one of the most important ways in which we learned to understand the world was to describe complicated phenomena using simpl...
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Article
A Note on Comparing Two Binomial Proportions in Confirmatory Noninferiority Trials
We evaluate the performance of two commonly used methods to construct confidence intervals for noninferiority decisions when the primary end point is binary. The two methods are the classical asymptotic normal...
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Article
Sample Size Reestimation: A Review and Recommendations
Despite best efforts, some crucial information used to design a confirmatory trial may not be available, or may be available but with a high degree of uncertainty, at the design stage. When this happens, it ma...
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Article
The Probability of Observing Negative Subgroup Results When the Treatment Effect is Positive and Homogeneous across All Subgroups
Subgroup analysis is an important part in the design and analysis of clinical trials. The importance arises from the scientific and commercial implications of such analysis. The predominant concerns about subg...
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Article
Multiple Co-primary Endpoints: Medical and Statistical Solutions: A Report from the Multiple Endpoints Expert Team of the Pharmaceutical Research and Manufacturers of America
There are quite a few disorders for which regulatory agencies have required a treatment to demonstrate a statistically significant effect on multiple endpoints, each at the one-sided 2.5% level, before accepti...
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Article
An Approach to Rationalize Partitioning Sample Size into Individual Regions in a Multiregional Trial
The desire to make valuable medicines available to patients globally at approximately the same time has led many pharmaceutical sponsors to consider conducting multiregional trials. In this article, we propose...
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Article
Measures for Conducting Comparative Benefit: Risk Assessment
In this article, we review quantitative measures that have been proposed to conduct comparative benefit:risk assessment. Comparative benefit:risk assessment could be conducted at the societal level (recommendi...
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Article
Good Practices for Adaptive Clinical Trials in Pharmaceutical Product Development
This article is a summary of good adaptive practices for the planning and implementation of adaptive designs compiled from experiences gained in the pharmaceutical industry. The target audience is anyone invol...
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Reference Work Entry In depth
Biopharmaceutical Research, Statistics in
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Article
A Quantitative Approach for Making Go/No-Go Decisions in Drug Development
There are many decision points along the product development continuum. Formal clinical milestones, such as the end of phase 1, phase 2a (proof of mechanism or proof of concept), and phase 2b provide useful de...
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Chapter and Conference Paper
Quantitative Risk/Benefit Assessment: Where Are We?
Pharmaceutical sponsors use a variety of approaches to make important benefit/risk decisions about their products internally. Benefit/risk assessment is equally important when regulators evaluate a product for...
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Chapter
The Need for and the Future of Adaptive Designs in Clinical Development
There has been much progress in the development and implementation of adaptive designs over the past 20 years. A major driver for this class of novel designs is to increase the information value of clinical tr...
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Chapter
Impact of Phase 2b Strategies on Optimization of Drug Development Programs
Selecting the right dose is critical for the success of any drug development program, and for maximizing the value of a product. A well selected dose will have a better chance to demonstrate a desirable risk/b...
