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Efficient design of clinical trials and epidemiological research: is it possible?

Nature Reviews Cardiology volume 14, pages 493–501 (2017)Cite this article

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Abstract

Randomized clinical trials and large-scale, cohort studies continue to have a critical role in generating evidence in cardiovascular medicine; however, the increasing concern is that ballooning costs threaten the clinical trial enterprise. In this Perspectives article, we discuss the changing landscape of clinical research, and clinical trials in particular, focusing on reasons for the increasing costs and inefficiencies. These reasons include excessively complex design, overly restrictive inclusion and exclusion criteria, burdensome regulations, excessive source-data verification, and concerns about the effect of clinical research conduct on workflow. Thought leaders have called on the clinical research community to consider alternative, transformative business models, including those models that focus on simplicity and leveraging of digital resources. We present some examples of innovative approaches by which some investigators have successfully conducted large-scale, clinical trials at relatively low cost. These examples include randomized registry trials, cluster-randomized trials, adaptive trials, and trials that are fully embedded within digital clinical care or administrative platforms.

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Figure 1: Cost trajectories for clinical trials.

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Acknowledgements

We are grateful to Iain Cockburn of Boston University, USA, for sharing the data used for Figure 1.

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  1. Michael S. Lauer is at the National Institutes of Health Office of Extramural Research, One Center Drive, Building 1, Room 144, Bethesda, Maryland 20892, USA.,

    Michael S. Lauer

  2. and Gail Pearson are at the Division of Cardiovascular Sciences of the National Heart, David Gordon, Gina Wei, Lung, and Blood Institute, 6701 Rockledge Drive, 8th Floor, Bethesda, Maryland 20892, USA.,

    David Gordon, Gina Wei & Gail Pearson

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M.S.L. researched data for the article. M.S.L. and G.W. wrote the manuscript. All the authors provided substantial contribution to the discussion of content and reviewed and edited the manuscript before submission.

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Lauer, M., Gordon, D., Wei, G. et al. Efficient design of clinical trials and epidemiological research: is it possible?. Nat Rev Cardiol 14, 493–501 (2017). https://doi.org/10.1038/nrcardio.2017.60

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