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Ascertaining Nonfatal Endpoints in Clinical Trials: Central Adjudication Versus Patient Insurance Claims.

Publication ,  Journal Article
Eisenstein, EL; Zozus, MN; Terry, SF; Davidson-Ray, L; Anstrom, KJ
Published in: Ther Innov Regul Sci
November 2021

BACKGROUND: The 21st Century Cures Act allows the US Food and Drug Administration (FDA) to utilize real-world data (RWD) to create real-world evidence (RWE) for new indications or post approval study requirements. We compared central adjudication with two insurance claims data sources to understand how endpoint accuracy differences impact RWE results. METHODS: We developed a decision analytic model to compare differences in efficacy (all-cause death, stroke and myocardial infarction) and safety (bleeding requiring transfusion) results for a simulated acute coronary syndrome antiplatelet therapy clinical trial. Endpoint accuracy metrics were derived from previous studies that compared centrally-adjudicated and insurance claims-based clinical trial endpoints. RESULTS: Efficacy endpoint results per 100 patients were similar for the central adjudication model (intervention event rate, 11.3; control, 13.7; difference, 2.4) and the prospective claims data collection model (intervention event rate, 11.2; control 13.6; difference, 2.3). However, the retrospective claims linking model's efficacy results were larger (intervention event rate, 14.6; control, 18.0; difference, 3.4). True positive event rate results (intervention, control and difference) for both insurance claims-based models were less than the central adjudication model due to false negative events. Differences in false positive event rates were responsible for differences in efficacy results for the two insurance claims-based models. CONCLUSION: Efficacy endpoint results differed by data source. Investigators need guidance to determine which data sources produce regulatory-grade RWE.

Duke Scholars

Published In

Ther Innov Regul Sci

DOI

EISSN

2168-4804

Publication Date

November 2021

Volume

55

Issue

6

Start / End Page

1250 / 1257

Location

Switzerland

Related Subject Headings

  • Stroke
  • Statistics & Probability
  • Retrospective Studies
  • Prospective Studies
  • Myocardial Infarction
  • Insurance
  • Humans
  • 4905 Statistics
  • 3214 Pharmacology and pharmaceutical sciences
  • 1117 Public Health and Health Services
 

Citation

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Eisenstein, E. L., Zozus, M. N., Terry, S. F., Davidson-Ray, L., & Anstrom, K. J. (2021). Ascertaining Nonfatal Endpoints in Clinical Trials: Central Adjudication Versus Patient Insurance Claims. Ther Innov Regul Sci, 55(6), 1250–1257. https://doi.org/10.1007/s43441-021-00321-9
Eisenstein, Eric L., Meredith N. Zozus, Sharon F. Terry, Linda Davidson-Ray, and Kevin J. Anstrom. “Ascertaining Nonfatal Endpoints in Clinical Trials: Central Adjudication Versus Patient Insurance Claims.Ther Innov Regul Sci 55, no. 6 (November 2021): 1250–57. https://doi.org/10.1007/s43441-021-00321-9.
Eisenstein EL, Zozus MN, Terry SF, Davidson-Ray L, Anstrom KJ. Ascertaining Nonfatal Endpoints in Clinical Trials: Central Adjudication Versus Patient Insurance Claims. Ther Innov Regul Sci. 2021 Nov;55(6):1250–7.
Eisenstein, Eric L., et al. “Ascertaining Nonfatal Endpoints in Clinical Trials: Central Adjudication Versus Patient Insurance Claims.Ther Innov Regul Sci, vol. 55, no. 6, Nov. 2021, pp. 1250–57. Pubmed, doi:10.1007/s43441-021-00321-9.
Eisenstein EL, Zozus MN, Terry SF, Davidson-Ray L, Anstrom KJ. Ascertaining Nonfatal Endpoints in Clinical Trials: Central Adjudication Versus Patient Insurance Claims. Ther Innov Regul Sci. 2021 Nov;55(6):1250–1257.
Journal cover image

Published In

Ther Innov Regul Sci

DOI

EISSN

2168-4804

Publication Date

November 2021

Volume

55

Issue

6

Start / End Page

1250 / 1257

Location

Switzerland

Related Subject Headings

  • Stroke
  • Statistics & Probability
  • Retrospective Studies
  • Prospective Studies
  • Myocardial Infarction
  • Insurance
  • Humans
  • 4905 Statistics
  • 3214 Pharmacology and pharmaceutical sciences
  • 1117 Public Health and Health Services