Lupus Biomarker Discovery, Validation, Approval, and Impact on Clinical Trials

Abstract

Current estimates suggest that bringing a new therapeutic from the laboratory to the patient requires 16–17 years and US$4–5 billion. One of the challenges in lupus clinical trials is the dearth of available biomarkers to identify those most likely to respond, to demonstrate response, and to predict adverse events. The lupus biomarker discovery pipeline, much like the lupus pharmaceutical discovery pipeline, has seen many potential advances but enjoyed all too few ultimate successes. New lupus biomarkers are essential and certain to accelerate lupus drug discovery and to move “lupology” from being less of an art to being more of a science. There is no precise path to lupus biomarker validation, FDA approval is generally not required, a multidisciplinary team is essential to optimize success, and a commercial partner is almost required to move advances in academia through the translational process. Despite the relatively few successes to date, the trajectory for lupus biomarker discovery and validation is steep and more promising than ever. Tandem and synergic advances in lupus biomarker validation and approval of new lupus therapeutics are hopefully on the horizon.