Phenotypic vs. target-based drug discovery for first-in-class medicines

Clin Pharmacol Ther. 2013 Apr;93(4):299-301. doi: 10.1038/clpt.2012.236.


Current drug discovery strategies include both molecular and empirical approaches. The molecular approaches are predominantly hypothesis-driven and are referred to as target-based. The empirical approaches are referred to as phenotypic because they rely on phenotypic measures of response. A recent analysis revealed the phenotypic approaches to be the more successful strategy for small-molecule, first-in-class medicines. The rationalization for this success was the unbiased identification of the molecular mechanism of action (MMOA).

MeSH terms

  • Animals
  • Disease Models, Animal*
  • Drug Discovery / methods*
  • Drug Discovery / statistics & numerical data
  • Humans
  • Molecular Targeted Therapy / methods*