Small Clinical Trials Issues and Challenges (2001) / Chapter Skim
Currently Skimming:
1 Introduction
1 Introduction
Pages 12-19
The Chapter Skim interface presents what we've algorithmically identified as the most significant single chunk of text within every page in the chapter.
Select key terms on the right to highlight them within pages of the chapter.
From page 12... ...
In addition, in the past 10 years clinical trials of drugs and other interventions have become more than a process required to judge the safeties and efficacies of potential treatments.
|
From page 13... ...
For example, a study focused on assessing the effects of microgravity on bone mineral density loss during space missions would have to rely on data for a few individuals (see Box 1-21. This report defines this research situation as a small clinical trial and explores the various design and analytical strategies one might consider to approach a small clinical trial.
|
From page 14... ...
Nonetheless, the general requirements for small clinical trials are no different than those for adequately powered "large clinical" trials; that is, they must be sufficiently designed and appropriately analyzed to provide a reasonable measure of the effect of an intervention. They should be designed to have an outcome measure for determination of success, a baseline measure that can be used to determine changes, and a means to monitor the changes (Meinert, 20001.
|
From page 15... ...
In some cases, however, properly designed small clinical trials can contribute to substantial evidence of efficacy; however, those conclusions may require the use of assumptions and inferences given the paucity of data (Siegel, 20001. Small clinical trials may successfully be used to study diseases or conditions with a well-described natural history with little variation; when sensitive pharmacodynamic effects are directly related to pathophysiology; when good nonhuman models are available; and when the intervention has a large effect on efficacy, produces a predictable relationship between measurable drug levels and effects, and has been applied to a related condition (Siegel, 2000)
|
From page 16... ...
For example, a possible alternative is to assess the therapeutic results in a single treated population by sequentially measuring whether the intervention results in outcomes that
|
From page 17... ...
A major question, however, for this and other approaches is whether the science base of alternative methods alone or in combination is sufficiently developed for these nonrandomized clinical trials to be effective in demonstrating efficacy in studies with small sample size. It has been recognized for some time that RCTs although highly desirable are neither practical nor feasible as a means of answering all clinical research questions.
|
From page 18... ...
other alternatives to RCTs, such as assessments of therapeutic results for a single treated population by sequentially measuring whether the outcomes from the intervention fall above or below a preestablished probability outcome range and meet predesigned specifications as opposed to incremental improvements. · Convene a 1-day conference during which participants from federal research and regulatory agencies, industry, academia, and other areas of clinical research and practice will discuss the progress being made in the strategies and the state of the science in the design, conduct, and evaluation of clinical trials of drugs, biologics, devices, and other medical interventions in populations with small numbers of individuals.
|
From page 19... ...
Chapter 2 addresses the fundamental tenets of clinical trial design and how they are challenged and possibly addressed by studies with small numbers of participants. Chapter 3 focuses on several statistical approaches that can be used to analyze small clinical trials.
|
Key Terms
This material may be derived from roughly machine-read images, and so is provided only to facilitate research.
More
information on Chapter Skim is available.