Module 1 2021

Other Considerations: Pediatrics (1)

• Office of Pediatric Therapeutics • Drugs marketed in US are used in patients who are less than 18 years old however, less than 25% have an indication for use in children • Best Pharmaceuticals for Children Act – 2002 − Reauthorized the pediatric exclusivity provisions of FDAMA (+ 6 months) − All pediatric supplements to receive Priority review • Pediatric Research Equity Act – 2003 − Reinstates the main provisions of the Pediatric Rule − Requires pediatric studies of certain drugs and biological products unless waived or deferred − Establishes pediatric advisory committee • http://www.fda.gov/cder/pediatric/

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Other Considerations: Pediatrics (2) • Exemptions for PREA requirements − Waiver can be requested − Orphan Drugs ( c.f . EU where a PIP is required for orphan product) • Rare Pediatric Disease Priority Review Voucher Program − Sponsor who receives an approval for a drug or biologic for a "rare pediatric disease" may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product − Note: voucher can be transferred − Rare pediatric disease defined as a disease that meets each of the following criteria: − The disease is a serious or life-threatening disease in which the serious or life- threatening manifestations primarily affect individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents − The disease is rare disease or condition

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