Decentralized clinical trials (DCTs), and hybrid iterations of them, redefine clinical research by shifting critical trial components – recruitment, informed consent, data collection, and monitoring – beyond the confines of traditional clinical trial sites like hospitals and research centers. This paradigm shift holds immense promise for transforming the landscape of pediatric rare disease research, offering hope to countless families facing these challenging diagnoses.
To help navigate this intricate terrain, we have created a comprehensive checklist that serves as a roadmap for the implementation of decentralized clinical trials in the specialized area of pediatric rare diseases.