Designing an In Vivo Efficacy Study of Nanomedicines for Preclinical Tumor Growth Inhibition

Methods Mol Biol. 2018;1682:241-253. doi: 10.1007/978-1-4939-7352-1_20.


Novel nanoformulated chemotherapeutics and diagnostics require demonstration of efficacy and safety in appropriate animal models prior to conducting early-phase clinical trials. In vivo efficacy experiments are tailored to the tumor model type and route of administration as well as several parameters related to the nanoformulation, like drug loading to determine dosing volume. When designing in vivo efficacy studies for nanomedicines, understanding the relationship between tumor biology and the nanoformulation characteristics is critical to achieving meaningful results, along with applying appropriate drug and nanoformulation controls. In particular, nanoparticles can have multifunctional roles such as targeting and imaging capabilities that require additional considerations when designing in vivo efficacy studies and choosing tumor models. In this chapter, we outline a general study design for a subcutaneously implanted tumor model along with an example of tumor growth inhibition and survival analysis.

Keywords: Biology; Nanoformulation; Statistical analysis; Subcutaneous; Tumor model.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Animals
  • Antineoplastic Agents / therapeutic use*
  • Cell Line, Tumor
  • Drug Evaluation, Preclinical / methods*
  • Humans
  • Kaplan-Meier Estimate
  • Mice
  • Nanomedicine / methods*
  • Neoplasms / drug therapy*
  • Neoplasms / pathology
  • Xenograft Model Antitumor Assays / methods


  • Antineoplastic Agents