How Decentralized Clinical Trials Can Shift The Pediatric Rare Disease Research Landscape

Despite the global prevalence of rare diseases in children, pediatric research lags significantly behind adult medicine, with fewer than 10% of conditions having approved therapies. To improve clinical outcomes for children with rare diseases, research models must shift from traditional settings to decentralized and hybrid approaches. By integrating trials into daily life through in-home visits and remote monitoring, the industry prioritizes the family's needs over these logistical hurdles. Ultimately, designing trials around flexibility and partnership increases participation, transforming abstract hope into tangible medical evidence.