Demonstrating Heterogeneity of Treatment Effects Among Patients: An Overlooked but Important Step Toward Precision Medicine

Jennifer S. Gewandter*, Michael P. McDermott, Hua He, Shan Gao, Xueya Cai, John T. Farrar, Nathaniel P. Katz, John D. Markman, Stephen Senn, Dennis C. Turk, Robert H. Dworkin

*Corresponding author for this work

    Research output: Contribution to journalArticleResearchpeer-review

    19 Citations (Scopus)

    Abstract

    Although heterogeneity in the observed outcomes in clinical trials is often assumed to reflect a true heterogeneous response, it could actually be due to random variability. This retrospective analysis of four randomized, double-blind, placebo-controlled multiperiod (i.e., episode) crossover trials of fentanyl for breakthrough cancer pain illustrates the use of multiperiod crossover trials to examine heterogeneity of treatment response. A mixed-effects model, including fixed effects for treatment and episode and random effects for patient and treatment-by-patient interaction, was used to assess the heterogeneity in patients’ responses to treatment during each episode. A significant treatment-by-patient interaction was found for three of four trials (P < 0.05), suggesting heterogeneity of the effect of fentanyl among different patients in each trial. Similar analyses in other therapeutic areas could identify conditions and therapies that should be investigated further for predictors of treatment response in efforts to maximize the efficiency of developing precision medicine strategies.

    Original languageEnglish
    Pages (from-to)204-210
    Number of pages7
    JournalClinical Pharmacology and Therapeutics
    Volume106
    Issue number1
    DOIs
    Publication statusPublished - Jul 2019

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