Can FDA require a sponsor to conduct preclinical (animal pharmacology/toxicology) studies in young animals?
A27. Yes. The rule requires each application to contain data adequate to assess the safety and effectiveness of a drug product in pediatric patients for its claimed use(s) (314.55(a)). If animal studies are necessary to provide “adequate data” they are required under the rule. However, each case must be considered on a case by case basis. There is no standard number or type of “pediatric preclinical studies” that would be requested. Divisions should consult with pharmacology/toxicology reviewing staff regarding the need for any juvenile animal data. Q28. FDA receives an application containing a new adult indication that does not occur in the pediatric population. The agency knows that the active moiety in the application is also used off-label for a pediatric indication. Can FDA require the applicant to conduct pediatric studies on the off-label indication? A28. No. FDA cannot require an applicant to conduct a pediatric assessment for off-label indications. However, FDA can issue a Wri
