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Outcomes

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Behavioral Clinical Trials for Chronic Diseases

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Abstract

The objective of all confirmatory Phase II and Phase III behavioral trials is to estimate the likelihood that the intervention(s) under study will improve a future health outcome. This future health outcome is the primary outcome for the trial. It is a decision that is made early in the design phase and drives many subsequent design decisions. This chapter focuses on considerations that guide the selection of a primary outcome, ways to avoid biased assessment, and the balance between multiple secondary outcomes and the risk of unintended consequences from them. Simplicity, objectivity, pre-specification, and clinical relevance are characteristics of outcome assessment in confirmatory behavioral trials. Exploration of moderators, mediators, and mechanisms are more appropriate for earlier stages of behavioral treatment development.

“Truth is ever to be found in simplicity, and not in the multiplicity and confusion of things.”

Isaac Newton

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Authors and Affiliations

  1. Department of Preventive Medicine, Rush University Medical Center, Chicago, IL, USA

    Lynda H. Powell

  2. College of Nursing, Villanova University, Villanova, PA, USA

    Peter G. Kaufmann

  3. Department of Psychiatry, Washington University in St. Louis, St. Louis, MO, USA

    Kenneth E. Freedland

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  2. Peter G. Kaufmann
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Powell, L.H., Kaufmann, P.G., Freedland, K.E. (2021). Outcomes. In: Behavioral Clinical Trials for Chronic Diseases. Springer, Cham. https://doi.org/10.1007/978-3-030-39330-4_9

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