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The Predictive Approaches to Treatment effect Heterogeneity (PATH) Statement: Explanation and Elaboration.

Publication ,  Journal Article
Kent, DM; van Klaveren, D; Paulus, JK; D'Agostino, R; Goodman, S; Hayward, R; Ioannidis, JPA; Patrick-Lake, B; Morton, S; Pencina, M; Raman, G ...
Published in: Ann Intern Med
January 7, 2020

The PATH (Predictive Approaches to Treatment effect Heterogeneity) Statement was developed to promote the conduct of, and provide guidance for, predictive analyses of heterogeneity of treatment effects (HTE) in clinical trials. The goal of predictive HTE analysis is to provide patient-centered estimates of outcome risk with versus without the intervention, taking into account all relevant patient attributes simultaneously, to support more personalized clinical decision making than can be made on the basis of only an overall average treatment effect. The authors distinguished 2 categories of predictive HTE approaches (a "risk-modeling" and an "effect-modeling" approach) and developed 4 sets of guidance statements: criteria to determine when risk-modeling approaches are likely to identify clinically meaningful HTE, methodological aspects of risk-modeling methods, considerations for translation to clinical practice, and considerations and caveats in the use of effect-modeling approaches. They discuss limitations of these methods and enumerate research priorities for advancing methods designed to generate more personalized evidence. This explanation and elaboration document describes the intent and rationale of each recommendation and discusses related analytic considerations, caveats, and reservations.

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Published In

Ann Intern Med

DOI

EISSN

1539-3704

Publication Date

January 7, 2020

Volume

172

Issue

1

Start / End Page

W1 / W25

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Risk Assessment
  • Randomized Controlled Trials as Topic
  • Models, Statistical
  • Individuality
  • Humans
  • General & Internal Medicine
  • Evidence-Based Medicine
  • Clinical Decision-Making
  • Clinical Decision Rules
 

Citation

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Kent, D. M., van Klaveren, D., Paulus, J. K., D’Agostino, R., Goodman, S., Hayward, R., … Steyerberg, E. W. (2020). The Predictive Approaches to Treatment effect Heterogeneity (PATH) Statement: Explanation and Elaboration. Ann Intern Med, 172(1), W1–W25. https://doi.org/10.7326/M18-3668
Kent, David M., David van Klaveren, Jessica K. Paulus, Ralph D’Agostino, Steve Goodman, Rodney Hayward, John P. A. Ioannidis, et al. “The Predictive Approaches to Treatment effect Heterogeneity (PATH) Statement: Explanation and Elaboration.Ann Intern Med 172, no. 1 (January 7, 2020): W1–25. https://doi.org/10.7326/M18-3668.
Kent DM, van Klaveren D, Paulus JK, D’Agostino R, Goodman S, Hayward R, et al. The Predictive Approaches to Treatment effect Heterogeneity (PATH) Statement: Explanation and Elaboration. Ann Intern Med. 2020 Jan 7;172(1):W1–25.
Kent, David M., et al. “The Predictive Approaches to Treatment effect Heterogeneity (PATH) Statement: Explanation and Elaboration.Ann Intern Med, vol. 172, no. 1, Jan. 2020, pp. W1–25. Pubmed, doi:10.7326/M18-3668.
Kent DM, van Klaveren D, Paulus JK, D’Agostino R, Goodman S, Hayward R, Ioannidis JPA, Patrick-Lake B, Morton S, Pencina M, Raman G, Ross JS, Selker HP, Varadhan R, Vickers A, Wong JB, Steyerberg EW. The Predictive Approaches to Treatment effect Heterogeneity (PATH) Statement: Explanation and Elaboration. Ann Intern Med. 2020 Jan 7;172(1):W1–W25.

Published In

Ann Intern Med

DOI

EISSN

1539-3704

Publication Date

January 7, 2020

Volume

172

Issue

1

Start / End Page

W1 / W25

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Risk Assessment
  • Randomized Controlled Trials as Topic
  • Models, Statistical
  • Individuality
  • Humans
  • General & Internal Medicine
  • Evidence-Based Medicine
  • Clinical Decision-Making
  • Clinical Decision Rules