Abstract

BackgroundThe number needed to treat (NNT) is an absolute effect measure that has been used to assess beneficial and harmful effects of medical interventions. Several methods can be used to calculate NNTs, and they should be applied depending on the different study characteristics, such as the design and type of variable used to measure outcomes. Whether or not the most recommended methods have been applied to calculate NNTs in studies published in the medical literature is yet to be determined. The aim of this study is to assess whether the methods used to calculate NNTs in studies published in medical journals are in line with basic methodological recommendations.MethodsThe top 25 high-impact factor journals in the “General and/or Internal Medicine” category were screened to identify studies assessing pharmacological interventions and reporting NNTs. Studies were categorized according to their design and the type of variables. NNTs were assessed for completeness (baseline risk, time horizon, and confidence intervals [CIs]). The methods used for calculating NNTs in selected studies were compared to basic methodological recommendations published in the literature. Data were analyzed using descriptive statistics.ResultsThe search returned 138 citations, of which 51 were selected. Most were meta-analyses (n = 23, 45.1%), followed by clinical trials (n = 17, 33.3%), cohort (n = 9, 17.6%), and case–control studies (n = 2, 3.9%). Binary variables were more common (n = 41, 80.4%) than time-to-event (n = 10, 19.6%) outcomes. Twenty-six studies (51.0%) reported only NNT to benefit (NNTB), 14 (27.5%) reported both NNTB and NNT to harm (NNTH), and 11 (21.6%) reported only NNTH. Baseline risk (n = 37, 72.5%), time horizon (n = 38, 74.5%), and CI (n = 32, 62.7%) for NNTs were not always reported. Basic methodological recommendations to calculate NNTs were not followed in 15 studies (29.4%). The proportion of studies applying non-recommended methods was particularly high for meta-analyses (n = 13, 56.5%).ConclusionsA considerable proportion of studies, particularly meta-analyses, applied methods that are not in line with basic methodological recommendations. Despite their usefulness in assisting clinical decisions, NNTs are uninterpretable if incompletely reported, and they may be misleading if calculating methods are inadequate to study designs and variables under evaluation. Further research is needed to confirm the present findings.

Highlights

  • The number needed to treat (NNT) is an absolute effect measure that has been used to assess beneficial and harmful effects of medical interventions

  • Methods recommended to calculate NNT Methodological recommendations A summary of basic and general recommendations was set up based upon the evidence reported in the Cochrane Handbook for Systematic Reviews of Interventions [31], in a thorough review performed by Bender about methods to obtain NNTs for different study designs [25], and in another review that focused on observational studies [21]

  • General characteristics of included studies The majority of studies reporting NNTs were identified from the Journal of the American Medical Association (JAMA, n = 17, 33.3%) and The Lancet (n = 14, 27.5%) (Additional file 1: Table S7)

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Summary

Introduction

The number needed to treat (NNT) is an absolute effect measure that has been used to assess beneficial and harmful effects of medical interventions. Whether or not the most recommended methods have been applied to calculate NNTs in studies published in the medical literature is yet to be determined. The use of NNT has been valuable in daily clinical practice, namely at assisting physicians in selecting therapeutic interventions [4, 5]. This metric has the potential for use as a supportive tool in benefit-risk assessments and in helping regulators make decisions on drug regulation [6,7,8]

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