Operation and Other Considerations

Advancement in science and technology in the past few decades has not only enabled molecular understanding of the pediatric and rare diseases, but also enriched the possible data collection methods via digital health technologies (DHTs) and integration of siloed data generated through various sources. In the chapter, we first discuss the requirement and validations for use of the DHTs in the regulatory setting. Various emerging practical considerations are illustrated, including extrapolations approaches based on same mechanism of actions (MOAs), artificial intelligence (AI) in clinical trial setting, and decentralized clinical trials. Several use cases in AI are illustrated: success drug repurposing applied during the COVID-19 pandemic, disease classification, and patient monitoring. Additionally, translational relevance of preclinical models to pediatric and rare diseases and potential pharmacodynamic endpoints are included in the considerations.