Regulatory forum opinion piece: differences between protein-based biologic products (biotherapeutics) and chemical entities (small molecules) of relevance to the toxicologic pathologist

Toxicol Pathol. 2013 Jan;41(1):128-36. doi: 10.1177/0192623312451371. Epub 2012 Jun 28.

Abstract

With the advances in cell culture methodologies and molecular biology that have occurred over the past several decades, biologics have become as common as small molecules within the portfolios of the pharmaceutical industry. Toxicologic pathologists should be aware of some of the fundamental differences between small molecules and biologics. Effects are not always observed in studies following administration of biologics. When findings are observed, the toxicologic pathologist should initially determine whether the effect(s) are mediated (directly or indirectly) via the intended pharmacology, exaggerated pharmacology, an immune response, and/or off target effects. Following this determination, the toxicologic pathologist should provide an assessment regarding the relevance of the findings to the intended clinical population, usually humans. The toxicologic pathologist may also be asked to assess unusual species and models. Given their broad background in physiology and immunology, toxicologic pathologists are uniquely positioned to provide this input to drug development teams.

MeSH terms

  • Animals
  • Biological Assay
  • Biomedical Research
  • Biosimilar Pharmaceuticals / toxicity*
  • Biotechnology / methods*
  • Dose-Response Relationship, Drug
  • Drug Discovery / methods*
  • Humans
  • Toxicology*

Substances

  • Biosimilar Pharmaceuticals