RD-LIVES: A Living Evidence Synthesis System for Rare Disease Treatment Efficacy and Safety

Abstract

Although rare diseases (RD) are gaining priority in healthcare worldwide, developing research policies for studying them in public settings remains challenging due to the limited evidence available. Evidence generation is crucial for rare diseases, requiring systematic assessment of study quality across multiple sources. Given the scarcity of patients, literature and clinical trial data for orphan drugs, we developed RD-LIVES-a tool designed to automatically accelerate evidence collection from literature and clinical trials for systematic reviews and meta-analyses. This tool enhances our understanding of treatment outcomes, determines appropriate follow-up durations, and informs the required treatment impact size for new drugs. Using Idiopathic Pulmonary Fibrosis (IPF) as an example, we demonstrate how RD-LIVES automates evidence collection and element extraction. The results indicate that RD-LIVES plays a vital role in designing costly prospective trials and has the potential to increase the likelihood of successful trial outcomes.

Keywords: IPF; Idiopathic Pulmonary Fibrosis; forced vital capacity; meta-analysis; overall survival; pirfenidone; progression-free survival; systematic review.

Figure 1.

RD-LIVES workflow