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(Stroke. 2005;36:2331.)
© 2005 American Heart Association, Inc.

Editorials

The Utility of Futility

Bruce Levin, PhD

From the Department of Biostatistics, Mailman School of Public Health, Columbia University, New York, NY.

Correspondence to Bruce Levin, PhD, Department of Biostatistics, Mailman School of Public Health, Columbia University, 722 West 168th Street, Room 626a, New York, NY 10032. E-mail bruce.levin@columbia.edu

Key Words: futility studies • stroke

An extract of the first 250 words of the full text is provided, because this article has no abstract.

See related article, pages 2410–2414.

In this issue, Palesch et al¹ discuss the single-arm, phase II futility study design and illustrate how its use might have avoided 3 large (and costly) but negative phase III therapeutic trials for ischemic stroke patients. The authors offer strong arguments to support their conclusion that use of this design as a strategy in phase II development "could permit the testing of a wider array of promising treatments at a fraction of the cost of taking all treatments directly to phase III trials." In a nutshell, they argue that there is utility in futility testing.

Although common in early-phase oncology trials, the futility study (single- or double-armed) may be less familiar to readers of this journal, and careful scrutiny of the design, especially of the formulation of null and alternative hypotheses, is worthwhile. Briefly, in a futility study, the null hypothesis states that the experimental therapy is sufficiently promising to warrant definitive, phase III testing, whereas the alternative hypothesis states that the experimental therapy lacks the prespecified superiority. Thus, the futility design reverses the logical status of null and alternative hypotheses as most often formulated in the traditional efficacy design. Whereas in the latter design, sufficient evidence is required to declare a therapeutic effect statistically significant, in the futility design, there is a presumption of benefit, and sufficient evidence is required to declare a significant shortfall from that benefit, such that it would be futile to proceed to large-scale testing with the given therapy. . . . [Full Text of this Article]

Related Article:

Applying a Phase II Futility Study Design to Therapeutic Stroke Trials

Yuko Y. Palesch, Barbara C. Tilley, David L. Sackett, Karen C. Johnston, and Robert Woolson
Stroke 2005 36: 2410-2414. [Abstract] [Full Text] [PDF]

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