Pediatric exclusivity and other contemporary regulatory changes: impact on pediatric drug study, labeling and safety

Author(s): Kevin D Hill, P Brian Smith, Michael Cohen-Wolkowiez, Daniel K Benjamin Jr, Jennifer S Li

Historically, most drugs have been used ‘off-label’ in children due to a lack of specific information on dosing, safety and efficacy. This practice risks adverse events, leading to serious public health consequences. Regulatory changes have attempted to address such issues by mandating and incentivizing the study of drugs in children. By all accounts, these regulatory changes have been enormously successful in stimulating >400 pediatric clinical trials enrolling >170,000 children over the last 5 years. These trials and others conducted over the preceding 15 years have resulted in >400 pediatric-specific labeling changes. While these labeling changes have improved pediatric drug safety, critics voice continuing concerns about the ‘financial windfall’ for industry, relative lack of study of off-patent agents and continued neglect of several important pediatric subpopulations (e.g., neonates).