Biomarkers In Development of FDA-Approved Therapeutics
October 2007- December 2015
BIOMARKERS
- 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) | |
| Hemoglobin | |
| 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) |
| FVC | |
| 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 |