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Extrapolating Survival From Randomized Clinical Trial Data—Possibilities and Caution

Extrapolating Survival From Randomized Clinical Trial Data—Possibilities and Caution Extrapolating Survival From Randomized Clinical Trial Data Invited Commentary Invited Commentary Extrapolating Survival From Randomized Clinical Trial Data— Possibilities and Caution Laine Elliott Thomas, PhD; Ann Marie Navar, MD; Michael J. Pencina, PhD Many therapies in cardiovascular disease, including heart fail- lished primary event rate in DAPA-HF was 0.116 per patient year ure (HF), are studied in clinical trials that report hazard ratios of dapagliflozin and 0.156 of placebo, yielding a rate ratio of (HRs) over a follow-up period far shorter than the anticipated 0.116/0.156 = 0.74. Using an exponential distribution, the treatment duration. The HR has known limitations, and alter- expected years of event-free survival would be 8.6 years native measures, such as re- (1/0.116) in the dapagliflozin arm and 6.4 years (1/0.156) in stricted mean survival time, the placebo arm. This parametric method yields similar re- Related article have been advocated to sum- sults to those of Docherty et al but is not a preferred method, marize survival over limited follow-up. However, in chronic because the exponential model assumes that event rates are diseases, such as HF, treatment effect measures, such as re- constant over time. stricted mean survival time, that are limited to clinical trial fol- http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png JAMA Cardiology American Medical Association

Extrapolating Survival From Randomized Clinical Trial Data—Possibilities and Caution

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Publisher
American Medical Association
Copyright
Copyright 2021 American Medical Association. All Rights Reserved.
ISSN
2380-6583
eISSN
2380-6591
DOI
10.1001/jamacardio.2021.2629
Publisher site
See Article on Publisher Site

Abstract

Extrapolating Survival From Randomized Clinical Trial Data Invited Commentary Invited Commentary Extrapolating Survival From Randomized Clinical Trial Data— Possibilities and Caution Laine Elliott Thomas, PhD; Ann Marie Navar, MD; Michael J. Pencina, PhD Many therapies in cardiovascular disease, including heart fail- lished primary event rate in DAPA-HF was 0.116 per patient year ure (HF), are studied in clinical trials that report hazard ratios of dapagliflozin and 0.156 of placebo, yielding a rate ratio of (HRs) over a follow-up period far shorter than the anticipated 0.116/0.156 = 0.74. Using an exponential distribution, the treatment duration. The HR has known limitations, and alter- expected years of event-free survival would be 8.6 years native measures, such as re- (1/0.116) in the dapagliflozin arm and 6.4 years (1/0.156) in stricted mean survival time, the placebo arm. This parametric method yields similar re- Related article have been advocated to sum- sults to those of Docherty et al but is not a preferred method, marize survival over limited follow-up. However, in chronic because the exponential model assumes that event rates are diseases, such as HF, treatment effect measures, such as re- constant over time. stricted mean survival time, that are limited to clinical trial fol-

Journal

JAMA CardiologyAmerican Medical Association

Published: Nov 28, 2021

References