Biomarkers Used as Outcomes – Drug and Device Digest


Biomarkers In Development of FDA-Approved Therapeutics

October 2007- December 2015


  • used as clinical trial outcome
  • accepted as basis for drug and biologic approvals
  • consult with FDA review division as early as possible in drug development regarding the use of a particular biomarker in clinical trials
  • examples of approved biomarkers below


Anesthesiology T1*; magnitude of T4/T1* ratio by acceleromyography
Cardiology Blood pressure
Serum low-density lipoprotein (LDL-C)
Hematology Hemoglobin
Platelet count
Ecarin clotting time; activated partial thromboplastin time; thrombin time; activated clotting time; plasma diluted thrombin time
Serum ferritin
Infectious Disease Hepatitis C virus (HCV) RNA*
Human immunodeficiency virus (HIV)-1 RNA
Sputum culture conversion to negative
Parasite count resolution
Inborn Errors of Metabolism White blood cell count; neutrophil count; red blood cell count; mean corpuscular volume
Growth in height or weight
Serum LDL-C
Blood phenylalanine
Forced vital capacity (FVC)
Plasma ammonia; plasma glutamine; and plasma citrulline
Splenic volume by magnetic resonance imaging (MRI)
Metabolism and Endocrinology Body weight
Bone mineral density by DEXA* scan
Hemoglobin A1c*
Serum calcium; oral calcium supplements; oral vitamin D supplements
Serum LDL-C
Urinary free cortisol (UFC)
Vertebral fractures by X-ray
Visceral adipose tissue (VAT) by computed tomography (CT) scan
Nephrology Hemoglobin
Serum sodium
Oncology CD34 positive cell count
Complete blood count (e.g., absolute neutrophil count)
Tumor burden by Bcr-Abl* (Philadelphia chromosome)
Tumor burden by Philadelphia chromosome positive cells
Plasma methotrexate
Splenic volume
Serum asparaginase
Serum testosterone
Tumor burden by imaging (using criteria such as RECIST* or EBMT*)
Ophthalmology Anterior chamber cells
Intraocular pressure (IOP)
Vitreomacular adhesion (VMA) by optical coherency tomography
Pulmonology Forced expiratory volume in one second (FEV1)
Respiratory distress syndrome (RDS) by chest X-ray and fraction of inspired oxygen (FiO2)
Rheumatology Joint angle
Uric acid
Transplant Biopsy-proven acute rejection (BPAR)

Medical Imaging Qualitative assessment of cerebral distribution of radioactive signal
Qualitative assessment of radioactive uptake (such as lymph node or tumor detection)
Qualitative regional assessment of localized radiographic signal intensity
Quantitation of arterial narrowing
Radioactive uptake in myocardial segments
Semi-quantitative lesion characteristics (such as internal morphology; contrast enhancement; border delineation)
Ultrasonographic signal intensity


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