From mechanism to empiricism: the evolution of heterogeneity of treatment effects.

Abstract

Journal of Comparative Effectiveness ResearchVol. 3, No. 5 CommentaryFrom mechanism to empiricism: the evolution of heterogeneity of treatment effectsNewell E McElwee & Cynthia J GirmanNewell E McElweeUS Outcomes Research, Center for Observational & Real-world Evidence, Merck & Co., Inc., Whitehouse Station, NJ, USASearch for more papers by this author & Cynthia J GirmanData Analytics & Observational Methods, Center for Observational & Real-world Evidence, Merck & Co., Inc., Whitehouse Station, NJ, USASearch for more papers by this authorPublished Online:28 Oct 2014https://doi.org/10.2217/cer.14.49AboutSectionsView ArticleView Full TextPDF/EPUB ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareShare onFacebookTwitterLinkedInRedditEmail View articleKeywords: heterogeneity of treatment effectindividualization of drug therapypersonalized medicinesub-group analysisReferences1 Brenneman SK, Shen W, Brekke L et al. Field-testing the ENSEMBLE minimum data set: performance of an instrument to address heterogeneity of treatment effects. J. Comp. Eff. Res. 3(5), 463–472 (2014).Link, Google Scholar2 PCORI. Patient-Centered Outcomes Research Definition Revision: Response to Public Input. www.pcori.org/content/research-we-support Google Scholar3 Lesko LJ, Schmidt S. Individualization of drug therapy: history, present state, and opportunities for the future. Clin. Pharmacol. Ther. 92(4), 458–466 (2011).Google Scholar4 Goodman L, Gilman A (Eds). Chapter 1: general principles. In: The Pharmacological Basis of Therapeutics. Macmillan, NY, USA (1941).Google Scholar5 Koch-Weser J. Serum drug concentrations as therapeutic guides. N. Engl. J. Med. 287(5), 227–231 (1972).Crossref, Medline, CAS, Google Scholar6 Temple R. Enrichment of clinical study populations. Clin. Pharmacol. Ther. 88(6), 774–778 (2010).Crossref, Medline, CAS, Google Scholar7 Kravitz RL, Duan N, Braslow J. Evidence based medicine, heterogeneity of treatment effects, and the trouble with averages. Milbank Q. 82(4), 661–687 (2004).Crossref, Medline, Google Scholar8 Kent DM, Hayward RA. Limitations of applying summary results of clinical trials to individual patients: the need for risk stratification. JAMA 198(10), 1209–1212 (2007).Crossref, Google Scholar9 Gabler NB, Duan N, Liao D et al. Dealing with heterogeneity of treatment effects: is the literature up to the challenge? Trials 10, 1–12 (2009).Crossref, Medline, Google Scholar10 PCORI. The PCORI Methodology Report. www.pcori.org/content/pcori-methodology-report Google Scholar11 Kent DM, Rothwell PM, Ionnidis JPA, Altman DG, Hayward RA. Assessing and reporting heterogeneity in treatment effects in clinical trials: a proposal. Trials 11, 85 (2010).Crossref, Medline, Google Scholar12 Greenfield S, Kravitz R, Duan N, Kaplan SH. Heterogeneity of treatment effects: implications for guidelines, payment and quality assessment. Am. J. Med. 120(4 Suppl. 1), 53–59 (2007).Google ScholarFiguresReferencesRelatedDetails Vol. 3, No. 5 Follow us on social media for the latest updates Metrics Downloaded 48 times History Published online 28 October 2014 Published in print September 2014 Information© Future Medicine LtdKeywordsheterogeneity of treatment effectindividualization of drug therapypersonalized medicinesub-group analysisFinancial & competing interests disclosureNewell E McElwee and Cynthia J Girman are employees of Merck & Co., Inc and own stock in Merck & Co, Inc. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.No writing assistance was utilized in the production of this manuscript.PDF download