New Advancements in Exposure-Response Analysis to Inform Regulatory Decision Making

  • Liang Zhao
  • Li Hongshan
  • Anshu Marathe
  • Jingyu (Jerry) Yu
  • Dinko Rekić
  • Nitin Mehrotra
  • Vikram Sinha
  • Yaning Wang
Chapter
First Online:

Abstract

To date, Exposure-Response (E-R) analyses have been recognized and routinely utilized in regulatory reviews, mainly to address key questions such as whether the proposed dosing regimen for a new drug is optimal or is warranted for further adjustment in specific populations in the context of the overall risk/benefit profile. This chapter will start from a summary of E-R methods commonly used in current applications followed with new methodology development and applications of E-R analyses on other aspects of reviews. Reporting on new methodology development is focused on case–control analyses when drug exposure is confounded with baseline disease status for several antibody oncology drugs. Reporting on applications of E-R analysis in new areas of review falls into using E-R analysis to derive the effect size for the noninferiority trial and subgroup analyses to identify favorable risk/benefit profile in specific population(s). The chapter also mentioned the potential role of E-R analysis in precision medicine by leveraging individual drug exposure to achieve balanced risk/benefit at the individual level.

Keywords

Oncology Pharmacokinetics Pharmacodynamics Modeling Simulation Case control 
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Notes

Acknowledgments

Disclaimer: Opinions expressed in this chapter are those of the authors and do not necessarily reflect the views or policies of the FDA.

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Copyright information

© Springer International Publishing Switzerland 2016

Authors and Affiliations

  • Liang Zhao
    • 1
  • Li Hongshan
    • 1
  • Anshu Marathe
    • 1
  • Jingyu (Jerry) Yu
    • 1
  • Dinko Rekić
    • 1
  • Nitin Mehrotra
    • 1
  • Vikram Sinha
    • 1
  • Yaning Wang
    • 1
  1. 1.Division of PharmacometricsOffice of Clinical Pharmacology, Center for Drug Evaluation and Research, U.S. Food and Drug AdministrationSilver SpringUSA

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