P4.018: From Bench to Bedside: Review of Bioscience Research Contributions to Pediatric Therapeutics

Background: The translation of fundamental bioscience discoveries into clinically meaningful pediatric therapeutics has historically lagged behind adult medical advances, leaving children with limited treatment options for many conditions. Recent breakthroughs in genomics, molecular biology, and bioengineering have created unprecedented opportunities to develop targeted pediatric therapies, yet significant gaps remain in understanding how these scientific advances translate to clinical benefit. This comprehensive review examines the contributions of basic bioscience research to pediatric drug development over the past decade, identifying successful translation pathways, persistent barriers, and emerging technologies with high potential to address unmet therapeutic needs in children.

Methods: We conducted a systematic analysis of preclinical and clinical studies published between 2013-2023 using PubMed, Web of Science, and clinical trial registries. Search terms combined pediatric focus ("child*," "pediatric*," "neonat*") with research domains ("translational research," "drug development," "precision medicine"). Included studies demonstrated a clear pathway from basic science discovery to clinical application in pediatric populations. Data extraction focused on four translational phases: target identification, preclinical validation, clinical trial design, and therapeutic implementation. Success metrics included time-to-translation, clinical efficacy, and FDA approval status.

Results: From 8,421 screened publications, 217 studies met inclusion criteria, representing 19 therapeutic areas. Oncology (32%) and rare genetic diseases (28%) accounted for most successful translations, with median development timelines decreasing from 14.2 to 9.8 years for biologics versus small molecules. CRISPR-based therapies showed the fastest bench-to-bedside progression (5.4 years for sickle cell disease). Organoid models improved preclinical prediction accuracy by 40% compared to animal models, while multi-omics approaches identified 112 novel pediatric drug targets. However, only 18% of translated therapies included neonatal formulations, and 63% of gene therapy trials excluded children under 5 years. Thematic analysis revealed three key translation accelerators: (1) NIH-funded pediatric research networks (OR=3.2 for faster translation), (2) adaptive trial designs (35% shorter recruitment phases), and (3) biomarker-guided patient selection (2.1-fold higher response rates).

Conclusion: Bioscience innovations are transforming pediatric therapeutics, particularly in molecularly-defined diseases, though significant age-based disparities persist in therapeutic access. Strategic investments in pediatric-specific translational infrastructure—including developmental pharmacogenomics, age-appropriate formulation science, and inclusive trial designs—could accelerate progress across all pediatric subspecialties. We identify three critical priorities: (1) Expansion of pediatric preclinical model systems, (2) Development of neonatal-specific therapeutic platforms, and (3) Creation of cross-disciplinary translation teams combining basic scientists with clinical trialists. These measures, coupled with regulatory incentives for pediatric drug development, will help realize the full potential of modern bioscience to address the unique therapeutic needs of children across the developmental spectrum.