The inclusion of real world evidence in clinical development planning.

Trials
R MartinaGetReal Workpackage 1

Abstract

When designing studies it is common to search the literature to investigate variability estimates to use in sample size calculations. Proprietary data of previously designed trials in a particular indication are also used to obtain estimates of variability. Estimates of treatment effects are typically obtained from randomised controlled clinical trials (RCTs). Based on the observed estimates of treatment effect, variability and the minimum clinical relevant difference to detect, the sample size for a subsequent trial is estimated. However, data from real world evidence (RWE) studies, such as observational studies and other interventional studies in patients in routine clinical practice, are not widely used in a systematic manner when designing studies. In this paper, we propose a framework for inclusion of RWE in planning of a clinical development programme. In our proposed approach, all evidence, from both RCTs and RWE (i.e. from studies in routine clinical practice), available at the time of designing of a new clinical trial is combined in a Bayesian network meta-analysis (NMA). The results can be used to inform the design of the next clinical trial in the programme. The NMA was performed at key milestones, such as at the end...Continue Reading

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Citations

Aug 29, 2020·Therapeutic Innovation & Regulatory Science·Alfredo Aram PuliniGregory Katz
Nov 20, 2019·Drugs -- Real World Outcomes·Andrew D XiaMatthew J Hirst
Mar 7, 2021·Pharmacoepidemiology and Drug Safety·Jessica M FranklinNancy A Dreyer
Apr 11, 2021·Wiener klinische Wochenschrift·Poobalan NaidooRory Leisegang
Jun 5, 2021·BMC Medical Research Methodology·Janharpreet SinghSylwia Bujkiewicz
Jun 5, 2021·JACC. Heart Failure·Muddassir Mehmood
Oct 11, 2021·BMC Medical Research Methodology·David A JenkinsSylwia Bujkiewicz
Nov 29, 2021·Clinical Pharmacology and Therapeutics·Simon DagenaisLauren Becnel

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