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Model-informed drug development (MIDD) has long been an approach to guide pediatric drug development both from the standpoint of challenging critical assumptions regarding the underlying disease biology and clinical pharmacology as well as de-risking critical decisions related to the progression of the clinical development plan. MIDD performance is often based on multidisciplinary engagement of subject matter experts across several institutions. Digital Research Environments (DRE) provide a secure collaborative research environment for digital analysis of data. Essential companions to the DRE are dynamically-updated and searchable metadata catalogs, in situ analysis tools with code versioning, as well as data provenance, and audit trails. This features facilitate the collaboration but also make it compatible with regulatory requirements. The Aridhia DRE specifically is the backbone of several highly successful pediatric multi-institutional collaborations including the RDCA-DAP and neonatal DAP managed by the Critical Path Institute (CPATH) and the International Neonatal Consortium (INC) respectively. Collaborative efforts to support expanded workspace functionality include the addition of nlmixr2 with the nimixr team and Great Ormand Street Hospital (GOSH) for nonlinear mixed effect modeling and r-based system pharmacology capabilities in collaboration with ESQ Labs. Additional proprietary and open-source tools are being added in collaboration with EMA and other stakeholders. The status and functionality of these solutions for pediatric R&D will be showcased and demonstrated at the meeting.
Barrett et al. (Wed,) studied this question.