Global Pediatric Policy Update: Are you Ready to Implement FDARA Section 504?

Key Takeaways

FDARA Section 504 eliminated the orphan exemption for pediatric studies for cancer drugs and instead empowers the FDA to require pediatric assessments of new therapies when the molecular targets are substantially relevant to children’s cancers.
As shown in drug development programs in other therapeutic areas, public-private partnerships may increase efficiencies and reduce costs and may offer advantages for companies engaged in pediatric cancer research.
There's a growing acceptance of master protocols that use a single infrastructure, design, and protocol to simultaneously evaluate multiple drugs and/or disease populations in multiple sub-studies.
In addition to organizational and cost efficiencies, master protocols provide biopharma companies with a mechanism to prioritize their development portfolios.