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Cited by 119

The DOI matches its Crossref record; 8 of the 8 references it cites were found in a registry or an index.

Nonrandomized real-world evidence to support regulatory decision making: process for a randomized trial replication project

Franklin, Jessica M., Pawar, Ajinkya, Martin, David, Glynn, Robert J., Levenson, Mark, Temple, Robert, Schneeweiss, Sebastian·2020·Clinical Pharmacology & Therapeutics
DOI 10.1002/cpt.1633
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Abstract

Recent legislation mandates that the US Food and Drug Administration issue guidance regarding when real‐world evidence (RWE) could be used to support regulatory decision making. Although RWE could come from randomized or nonrandomized designs, there are significant concerns about the validity of RWE assessing medication effectiveness based on nonrandomized designs. We propose an initiative using healthcare claims data to assess the ability of nonrandomized RWE to provide results that are comparable with those from randomized controlled trials (RCT s). We selected 40 RCT s, and we estimate that approximately 30 attempted replications will be completed after feasibility analyses. We designed an implementation process to ensure that each attempted replication is consistent, transparent, and reproducible. This initiative is the first to systematically evaluate the ability of nonrandomized RWE to replicate multiple RCT s using a structured process. Results from this study should provide insight on the strengths and limitations of using nonrandomized RWE from claims for regulatory decision making.

Overview

Integrity signals

Registry

Crossref · 10.1002/cpt.1633

Cites

8 of 8 references verified0 citation errors · 0 not checkable

Statistics

Recomputing p-values needs the paper’s full text.

Verification

Claims the paper makes

This paper’s full text isn’t openly available, so its claims and self-check weren’t run. The registry and citation checks still apply.

Claims other papers make about this one

Each is checked against this paper’s own text: does it say what the citing paper says it does?

  • An advanced methodological approach was applied using the RCT-DUPLICATE design, data, and analytics framework to emulate key aspects of randomized studies.

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    From Semaglutide and Tirzepatide in Patients With Heart Failure With Preserved Ejection Fraction (2025) · cites this paper as [33]

  • The database studies were on average 19-fold larger than the trials and showed strong concordance in prespecified agreement metrics.

    “The database studies, which were on average 19-fold larger than the trials, showed strong concordance in prespecified agreement metrices.33”

    From Semaglutide and Tirzepatide in Patients With Heart Failure With Preserved Ejection Fraction (2025) · cites this paper as [33]

Citations

Cites

Flagged first, then by citations

Research works printed but not linked: 35

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Metadata

Authors
Franklin, J. M., Pawar, A., Martin, D., Glynn, R. J., Levenson, M., Temple, R., & Schneeweiss, S.
Affiliations
  • Division of Pharmacoepidemiology and Pharmacoeconomics Brigham and Women's Hospital Harvard Medical School Boston Massachusetts USA
  • Office of Medical Policy Center for Drug Evaluation and Research US Food and Drug Administration Silver Spring Maryland USA
  • Division of Biometrics VII Office of Biostatistics Center for Drug Evaluation and Research US Food and Drug Administration Silver Spring Maryland USA
  • Office of Drug Evaluation I Office of New Drugs Center for Drug Evaluation and Research US Food and Drug Administration Silver Spring Maryland USA
ORCID
Venue
Clinical Pharmacology & Therapeutics
Publisher
Wiley
Published
2019-10-25
Volume
107
Issue
4
Pages
817-826
ISSN
  • 0009-9236
  • 1532-6535
DOI
10.1002/cpt.1633
Cited by
119
References
8
Language
en
Licence
onlinelibrary.wiley.com/termsAndConditions#vor (vor)

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