'Threshold crossing':a useful way to establish the counterfactual in clinical trials?

Eichler, Hans-Georg and Bloechl-Daum, Brigitte and Bauer, Peter and Bretz, Frank and Brown, Jeffrey and Hampson, Lisa Victoria and Honig, Peter and Krams, Michael and Leufkens, Hubert and Lim, Robyn and Lumpkin, Murray and Murphy, Martin and Pignatti, Francesco and Posch, Martin and Schneeweiss, Sebastian and Trusheim, Mark and Koenig, Franz (2016) 'Threshold crossing':a useful way to establish the counterfactual in clinical trials? Clinical Pharmacology & Therapeutics, 100. pp. 699-712.

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Abstract

A central question in the assessment of benefit/harm of new treatments is: how does the average outcome on the new treatment (the factual) compare to the average outcome had patients received no treatment or a different treatment known to be effective (the counterfactual)? Randomized controlled trials (RCTs) are the standard for comparing the factual with the counterfactual. Recent developments necessitate and enable a new way of determining the counterfactual for some new medicines. For select situations, we propose a new framework for evidence generation, which we call “threshold-crossing.” This framework leverages the wealth of information that is becoming available from completed RCTs and from real world data sources. Relying on formalized procedures, information gleaned from these data is used to estimate the counterfactual, enabling efficacy assessment of new drugs. We propose future (research) activities to enable “threshold-crossing” for carefully selected products and indications in which RCTs are not feasible.

Item Type:
Journal Article
Journal or Publication Title:
Clinical Pharmacology & Therapeutics
ID Code:
81848
Deposited By:
Deposited On:
14 Feb 2017 14:34
Refereed?:
Yes
Published?:
Published
Last Modified:
26 Jun 2020 02:32