Gene Therapy Drug Development for First-In-Human Study of Pediatric Diseases: Facilitating Change to Current Paradigms

Drug development in pediatrics is mandated under US and European Union legislation, delays pediatric studies can impact the appropriate labeling use of therapeutics for children. Developing medical products challenging, as there are several critical issues factors to consider when initiating a drug program. The International Conference Harmonisation guideline E11(R1) criteria 21 CFR part 50 subpart D define regulatory standards developers ensure safety participants clinical studies. Adequate adult data typically required before finalizing study designs by virtue Pediatric Research Equity Act. It evolving that Food Administration (FDA) including adolescents Phase 3 trials. In pediatrics, lack coordinated extrapolation efficacy amongst global agencies impacts timelines trial designs. First-in-human trials may be justified if aspects 50, specifically 50.52 addressed. For example, first-in-human gene therapy have been allowed spinal muscular atrophy (SMA) using benefit-risk assessment justify conduct statutory requirement children influenced nature disease currently needs personalized. These addressed this perspective on treatment