Module232025

12/05/2025

USA Legislation and regulations with important paediatric drug regulation Impact Legislation Year

• Made BPCA and PREA permanent • WR must include rationale for neonates • Ensure completion of pediatric studies • Pediatric Study Plans • Stakeholder meeting and report to Congress

2012

FDA Safety and Innovation Act (FDASIA) Title V (Sections 501–511) and Title IX: Section 908

• Hold one public meeting for pediatric rare disease development • Required neonatal expertise in Office of Pediatric Therapeutics

• Established the rare pediatric disease priority review voucher (RPD PRV) program as an additional incentive for the study of rare pediatric diseases • Several provisions pertinent to pediatric drug development for rare diseases including the Regenerative Medicines Advanced Therapies designation program • Cancer Moonshot – though not specific in pediatrics, supports the study of some rare cancers that disproportionately affect children • Updates PREA so FDA may now require pediatric assessments for oncology drugs when the molecular targets is substantially relevant to children’s cancers • Removed exemption under PREA for orphan drugs in oncology • Requires evaluation of oncology products based on molecular targets

2012

Creating Hope Act

21 st Century Cures Act

2016

2017 2017

FDA Reauthorization Act

Research to Accelerate Cures and Equity for Children Act (RACE)

The Organisation for Professionals in Regulatory Affairs

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Select FDASIA Sections

• Section 502 Written Requests • Must include neonatal studies or rational for not including; template available and includes section for discussion of neonatal studies • Section 505 Compliance with PREA • Allow extension for deferred studies • Authority for FDA to issue and publicly post non-compliance letters for overdue PREA studies – may result in a misbranded product • Section 506 Pediatric Study Plans • Encourage sponsors to identify studies as early as possible and conduct pediatric studies prior to marketing application submission • Encourage sponsors to include all pediatric plan (including those under WR) in PSP – but FDA cannot compel sponsors to do so

The Organisation for Professionals in Regulatory Affairs

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