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GetReal in mathematical modelling: a review of studies predicting drug effectiveness in the real world
Author(s) -
Panayidou Klea,
Gsteiger Sandro,
Egger Matthias,
Kilcher Gablu,
Carreras Máximo,
Efthimiou Orestis,
Debray Thomas P. A.,
Trelle Sven,
Hummel Noemi
Publication year - 2016
Publication title -
research synthesis methods
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 3.376
H-Index - 35
eISSN - 1759-2887
pISSN - 1759-2879
DOI - 10.1002/jrsm.1202
Subject(s) - medline , clinical trial , meta analysis , computer science , psychological intervention , randomized controlled trial , medicine , intensive care medicine , medical physics , psychiatry , political science , law
The performance of a drug in a clinical trial setting often does not reflect its effect in daily clinical practice. In this third of three reviews, we examine the applications that have been used in the literature to predict real‐world effectiveness from randomized controlled trial efficacy data. We searched MEDLINE, EMBASE from inception to March 2014, the Cochrane Methodology Register, and websites of key journals and organisations and reference lists. We extracted data on the type of model and predictions, data sources, validation and sensitivity analyses, disease area and software. We identified 12 articles in which four approaches were used: multi‐state models, discrete event simulation models, physiology‐based models and survival and generalized linear models. Studies predicted outcomes over longer time periods in different patient populations, including patients with lower levels of adherence or persistence to treatment or examined doses not tested in trials. Eight studies included individual patient data. Seven examined cardiovascular and metabolic diseases and three neurological conditions. Most studies included sensitivity analyses, but external validation was performed in only three studies. We conclude that mathematical modelling to predict real‐world effectiveness of drug interventions is not widely used at present and not well validated. © 2016 The Authors Research Synthesis Methods Published by John Wiley & Sons Ltd.