Bridging the Translational Gap: IVIVC in Personalized Medicine for Rare Diseases

Abstract

The development of effective therapies for rare diseases remains a significant challenge due to limited patient populations and complex disease mechanisms. Personalized medicine offers promising avenues to tailor treatments to individual patients, yet translating preclinical findings into clinical success often encounters a substantial translational gap. In vitro–in vivo correlation (IVIVC) serves as a critical tool in bridging this gap by enabling the prediction of clinical drug behavior from laboratory data. This article explores the pivotal role of IVIVC in advancing personalized medicine for rare diseases, highlighting its capacity to optimize dosing, enhance drug efficacy, and reduce adverse effects. By examining recent case studies and addressing current challenges, this review underscores the potential of IVIVC to transform therapeutic strategies, ultimately improving patient outcomes and accelerating drug development in the realm of rare diseases.