Under PREA, if the FDA declares a drug possibly useful to ill children, what may the pharmaceutical company be required to do?

• A. Provide labeling stating that the safety and efficacy of the drug is not established for children.
• B. Survey existing data about the drug to determine potential use in the pediatric population.
• C. Receive a patent extension for conducting pediatric studies to determine use in children.
• D. Conduct pediatric drug studies to determine whether the drug is safe and effective in children.

Answer: (D)

Under PREA, the aim is to ensure medicines used in children have proven safety and effectiveness based on studies in the pediatric population. When the FDA identifies that a drug being developed could benefit children, the sponsor is required to conduct pediatric studies to determine safety and efficacy in kids. This directly builds pediatric labeling and informs appropriate dosing and risk assessment for young patients, rather than simply labeling that safety or efficacy in children is unestablished or relying on data from adults. While there are related concepts like waivers, deferrals, or separate incentives, the core requirement in this scenario is to conduct pediatric drug studies to determine whether the drug is safe and effective in children.