GetReal: from efficacy in clinical trials to relative effectiveness in the real world

Abstract

The GetReal consortium ("incorporating real-life data into drug development") addresses the efficacy-effectiveness gap that opens between the data from well-controlled randomized trials in selected patient groups submitted to regulators and the real-world evidence on effectiveness and safety of drugs required by decision makers. Workpackage 4 of GetReal develops evidence synthesis and modelling approaches to generate the real-world evidence. In this commentary, we discuss how questions change when moving from the well-controlled randomized trial setting to real-life medical practice, the evidence required to answer these questions, the populations to which estimates will be applicable to and the methods and data sources used to produce these estimates. We then introduce the methodological reviews written by GetReal authors and published in Research Synthesis Methods on network meta-analysis, individual patient data meta-analysis and mathematical modelling to predict drug effectiveness. The critical reviews of key methods are a good starting point for the ambitious programme of work GetReal has embarked on. The different strands of work under way in GetReal have great potential to contribute to making clinical trials research as relevant as it can be to patients, caregivers and policy makers. Copyright © 2016 John Wiley & Sons, Ltd.

Cognitive-Behavioral Analysis System of Psychotherapy, Drug, or Their Combination for Persistent Depressive Disorder: Personalizing the Treatment Choice Using Individual Participant Data Network Metaregression

“…Similar analyses are warranted in the future for more studies employing other types of antidepressants and other types of psychotherapies in order to further guide their individualized treatments. Cross-methodological data synthesis from experimental and observational studies including one-arm clinical trials, cohort data, and data from registries is an emerging area of research that can bridge the gap between evidence from well-controlled randomized trials in selected patient groups and real-world evidence [57, 58]. …”

Guy’s cancer cohort – real world evidence for cancer pathways

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Anti-androgen monotherapy versus gonadotropin-releasing hormone agonists in men with advanced, non-metastatic prostate cancer: a register-based, observational study

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“…Even though RCTs are considered the gold standard for evaluating the effectiveness of interventions, observational data, also known as real-world data, is an important addition to RCTs in clinical decision making provided that confounding by indication is appropriately handled [21]. Compared to patients outside RCTs, participants in RCTs are often highly selected with smaller cancer burden, and elderly patients and those with comorbidities are frequently excluded [22,23]. Thus, additional evidence from observational studies in support of results from RCTs is needed to show the external validity of these results.…”