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Randomized Controlled Trials and the Efficacy of Psychotropic Medications

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NeuroPsychopharmacotherapy

Abstract

Randomized controlled trials (RCTs) are the most important component of the evidence base justifying the use of psychiatric medications in clinical practice. Establishing efficacy of an investigational new drug through RCTs requires careful consideration of factors related to trial design, patient enrollment, study conduct, and data analysis. Each disorder has unique characteristics that require tailoring of RCT designs in terms of duration, assessment, and treatment setting. Placebo effects and patient nonadherence to study medication present significant challenges to identifying truly efficacious drugs. Although RCTs have been criticized for being vulnerable to various forms of bias, they remain the best means for identifying treatments to relieve mental suffering. Mental health practitioners can benefit from a deeper understanding of clinical research procedures and the standards used for evaluating a medication’s efficacy, safety, and tolerability. This understanding can inform clinicians’ judgment regarding the generalizability of RCT results to their clinical practice.

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Authors and Affiliations

  1. Department of Psychiatry and Behavioral Sciences, Emory University School of Medicine, Atlanta, GA, USA

    Boadie Waid Dunlop & Carolina Medeiros Da Frota Ribeiro

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  1. Boadie Waid Dunlop
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  2. Carolina Medeiros Da Frota Ribeiro
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Correspondence to Boadie Waid Dunlop .

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Editors and Affiliations

  1. Clinic and Policlinic for Psychiatry, Psychosomatics and Psychotherapy, University Hospital Wuerzburg, Wuerzburg, Germany

    Peter Riederer

  2. Institute of Psychological Medicine (IPM), Soyen, Germany

    Gerd Laux

  3. Centre for Research Promotion and Support, Fujita Health University, Toyoake, Aichi, Japan

    Toshiharu Nagatsu

  4. Center Translational Res Neurol Diseases, Dalian Med Uni Shanghai Inst Health Sc Center Translational Res Neurol Diseases, Dalian, China

    Weidong Le

  5. Würzburg, Germany

    Christian Riederer

Section Editor information

  1. University Hospital, Center for Mental Health, Clinic and Policlinic for Psychiatry, Psychosomatics and Psychotherapy, University Wuerzburg, Wuerzburg, Germany

    Peter Riederer

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Dunlop, B.W., Medeiros Da Frota Ribeiro, C. (2022). Randomized Controlled Trials and the Efficacy of Psychotropic Medications. In: Riederer, P., Laux, G., Nagatsu, T., Le, W., Riederer, C. (eds) NeuroPsychopharmacotherapy. Springer, Cham. https://doi.org/10.1007/978-3-030-62059-2_4

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