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

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Published

Standard

'Threshold crossing': a useful way to establish the counterfactual in clinical trials? / Eichler, Hans-Georg; Bloechl-Daum, Brigitte; Bauer, Peter et al.
In: Clinical Pharmacology & Therapeutics, Vol. 100, 12.2016, p. 699-712.

Research output: Contribution to Journal/Magazine › Journal article › peer-review

Harvard

Eichler, H-G, Bloechl-Daum, B, Bauer, P, Bretz, F, Brown, J, Hampson, LV, Honig, P, Krams, M, Leufkens, H, Lim, R, Lumpkin, M, Murphy, M, Pignatti, F, Posch, M, Schneeweiss, S, Trusheim, M & Koenig, F 2016, ''Threshold crossing': a useful way to establish the counterfactual in clinical trials?', Clinical Pharmacology & Therapeutics, vol. 100, pp. 699-712.

APA

Eichler, H-G., Bloechl-Daum, B., Bauer, P., Bretz, F., Brown, J., Hampson, L. V., Honig, P., Krams, M., Leufkens, H., Lim, R., Lumpkin, M., Murphy, M., Pignatti, F., Posch, M., Schneeweiss, S., Trusheim, M., & Koenig, F. (2016). 'Threshold crossing': a useful way to establish the counterfactual in clinical trials? Clinical Pharmacology & Therapeutics, 100, 699-712.

Vancouver

Eichler H-G, Bloechl-Daum B, Bauer P, Bretz F, Brown J, Hampson LV et al. 'Threshold crossing': a useful way to establish the counterfactual in clinical trials? Clinical Pharmacology & Therapeutics. 2016 Dec;100:699-712. Epub 2016 Oct 19.

Author

Eichler, Hans-Georg ; Bloechl-Daum, Brigitte ; Bauer, Peter et al. / 'Threshold crossing' : a useful way to establish the counterfactual in clinical trials?. In: Clinical Pharmacology & Therapeutics. 2016 ; Vol. 100. pp. 699-712.

Bibtex

@article{7fde7b9b7cc5436dbaf0f127f98b7c4b,
title = "'Threshold crossing': a useful way to establish the counterfactual in clinical trials?",
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.",
author = "Hans-Georg Eichler and Brigitte Bloechl-Daum and Peter Bauer and Frank Bretz and Jeffrey Brown and Hampson, {Lisa Victoria} and Peter Honig and Michael Krams and Hubert Leufkens and Robyn Lim and Murray Lumpkin and Martin Murphy and Francesco Pignatti and Martin Posch and Sebastian Schneeweiss and Mark Trusheim and Franz Koenig",
year = "2016",
month = dec,
language = "English",
volume = "100",
pages = "699--712",
journal = "Clinical Pharmacology & Therapeutics",

}

RIS

TY - JOUR

T1 - 'Threshold crossing'

T2 - a useful way to establish the counterfactual in clinical trials?

AU - Eichler, Hans-Georg

AU - Bloechl-Daum, Brigitte

AU - Bauer, Peter

AU - Bretz, Frank

AU - Brown, Jeffrey

AU - Hampson, Lisa Victoria

AU - Honig, Peter

AU - Krams, Michael

AU - Leufkens, Hubert

AU - Lim, Robyn

AU - Lumpkin, Murray

AU - Murphy, Martin

AU - Pignatti, Francesco

AU - Posch, Martin

AU - Schneeweiss, Sebastian

AU - Trusheim, Mark

AU - Koenig, Franz

PY - 2016/12

Y1 - 2016/12

N2 - 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.

AB - 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.

M3 - Journal article

VL - 100

SP - 699

EP - 712

JO - Clinical Pharmacology & Therapeutics

JF - Clinical Pharmacology & Therapeutics

ER -