Module232025

12/05/2025

USA landscape– latest status Dec 24 Key Impact for children Legislation

• Reauthorisation of the award of PRV for rare pediatric diseases • Passed by House of Representatives on a bipartisan, unanimous basis in Sept 2024

Creating Hope Reauthorization Act

• FDA could require a pediatric investigation of a new single novel cancer drug in combination with another active ingredient. • Extend financial penalties for failure to complete pediatric studies under PREA • Passed by House of Representatives on a bipartisan, unanimous basis in Sept 2024 • Collaborative pediatric research effort with the goal to understand the genetic causes and links between childhood cancer and congenital disorders, funded for 10 years in 2014 • V2.0 new source of funding by redirecting penalties collected from industry • Passed by House of Representatives on a bipartisan, unanimous basis in Apr 2024 • Enable pediatric providers to deliver timely, essential care to patients from both within and outside their home states without delays • Passed by House of Representatives on a bipartisan, unanimous basis in Sept 2024

Give Kids a Chance Act

Gabriella Miller Kids First Research Act 2.0

Accelerating Kid’s Access to Care Act

• three critical things to improve BPCA and PREA:

Innovation in Pediatric Drugs Act

• Increase the number of rare disease drugs studied in children, • ensure that required PREA studies actually get completed, and • give the NIH BPCA program its first funding increase in 22 years. • Passed by House of Representatives on a bipartisan, unanimous basis in Sept 2024

The Organisation for Professionals in Regulatory Affairs

7

PREA vs BPCA

PREA • Drugs and biologics • Required studies • Studies may only be required for approved indication(s) • Pediatric Plan • Products with orphan designation are exempt from requirements • Pediatricstudies must be labelled

BPCA • Drugs and biologics • Voluntary studies

• Studies relate to entire moiety and may expand indication(s) • Written Request from FDA • Studies may be requested for products with orphan designation • Pediatricstudies must be labelled

The Organisation for Professionals in Regulatory Affairs

8