Table 2

Biomarkers in drug development

  • Determine whether a drug hits the target and has impact on the biological pathway

  • Evaluate MOA

  • PK/PD correlations and determine dose and schedule

  • Determine biologically effective dose

  • Research test used during drug development

  • Exploratory biomarkers that may help stratify patient populations for late stage trials

  • Drug dose

  • Drug-drug interactions

  • Early assessment of toxicity

  • Identify patients most likely to respond, or are least likely to suffer an adverse event when treated with a drug

  • Complementary/Companion diagnostic test (eg, HER2/neu, anaplastic lymphoma kinase (ALK) translocation, PD-L1 IHC)

  • Stratify patients into study arms

  • Biomarker of efficacy and/or safety

  • Identify mechanisms driving acquired or innate drug resistance

  • Therapy escape mechanisms

  • Clonal evolution of tumor

  • Mutation analyses (eg, KRAS mutation for EGFR antibodies)

  • Predicts course of disease independent of any specific treatment modality

  • Patient stratification into study arms

  • Surrogate end points

  • Circulating tumor cells (Cell Search), Gene expression profiling (PAM50, Mammaprint)

Surrogate end point
  • Approved registrational end points

  • Standard of care diagnostic tests (eg, LDL, HbA1c, viral load, blood pressure)

  • HbA1c, hemaglobin A1c; IHC, immunohistochemistry; LDL, low-density lipoprotein; MOA, mechanism of action; PD, pharmacodynamics; PD-L1, programmed death-ligand 1; PK, pharmacokinetics.