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The promise of futility trials in neurological diseases

Nature Reviews Neurology volume 11, pages 300–305 (2015)Cite this article

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Abstract

Double-blinded randomized controlled trials (RCTs) have contributed much important evidence to guide treatment decisions in neurology. RCTs are relatively straightforward to conduct, provided that they investigate common diseases, have clearly defined outcome measures, and are of short duration. In neurology, however, many diseases are uncommon, have no consensus outcome measures, and develop over decades. Basic research into neurological diseases continues to identify candidate therapies faster than they can be tested for their clinical utility, leading to a 'translational gap'. Futility trials were initially developed in oncology to efficiently test candidate therapies in phase II trials. As single-arm unblinded studies, futility trials are relatively easy to conduct, and they generally require smaller sample sizes than RCTs. In this article, we discuss futility models, highlighting their advantages as well as challenges to their application in several neurological diseases, including Parkinson disease, stroke and multiple sclerosis.

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Authors and Affiliations

  1. Department of Clinical Neurosciences, Foothills Hospital, University of Calgary, 1403 29th Street NW, Calgary, T2N 2T9, AB, Canada

    Marcus W. Koch, Lawrence Korngut, David G. Patry, Yahya Agha-Khani, Christopher White, Justyna R. Sarna, Michael Yeung, V. Wee Yong & Luanne Metz

  2. Department of Community Health Sciences, TRW Building, University of Calgary, 3280 Hospital Drive NW, Calgary, T2N 4Z6, AB, Canada

    Marcus W. Koch

  3. Department of Oncology, Tom Baker Cancer Centre, 1331 29th Street NW, Calgary, T2N 4N2, AB, Canada

    Daniel Y. C. Heng

  4. Department of Biostatistics, School of Public Health, University of Alabama at Birmingham, 1665 University Boulevard, Birmingham, 35294-0022, AL, USA

    Gary Cutter

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M.W.K. researched data for the article. All the authors made substantial contributions to the discussion of content, to writing the article, and to revising and editing the article before submission.

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Correspondence to Marcus W. Koch.

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Koch, M., Korngut, L., Patry, D. et al. The promise of futility trials in neurological diseases. Nat Rev Neurol 11, 300–305 (2015). https://doi.org/10.1038/nrneurol.2015.34

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