Evidence of effectiveness: how much can we extrapolate from existing studies?

AAPS J. 2005 Oct 5;7(2):E467-74. doi: 10.1208/aapsj070247.


Drug development can be a science of extrapolation if the use of a drug exposure-response relationship is embraced and implemented through mechanistically oriented pharmacokinetic (PK)-pharmacodynamic (PD) modeling analysis and clinical trial simulation. The traditional requirement of at least 2 adequate and well-controlled phase III studies by the US Food and Drug Administration for drug approval can be waived in certain situations, substantially reducing the resources and time. In this article, the authors introduce a real drug development case where the chance for this exemption was maximized by actively using PK-PD modeling followed by clinical trial simulation, resulting in faster and more economical introduction of a new dosage regimen to patients.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Clinical Trials as Topic / methods*
  • Clinical Trials as Topic / statistics & numerical data
  • Dose-Response Relationship, Drug
  • Humans
  • Research Design / statistics & numerical data
  • Technology, Pharmaceutical / methods*