U.S. – Measure Aims to Speed Rare Pediatric Disease Treatments and Tighten FDA Oversight 

Washington, D.C. — A new bipartisan House bill aims to give children with cancer better access to cutting-edge treatments by tightening pediatric research requirements for drugmakers.  

H.R. 1262, the “Mikaela Naylon Give Kids a Chance Act,” would require more “molecularly targeted” pediatric cancer studies when a company seeks approval for a cancer drug that hits targets also relevant in children’s tumors. It also clarifies how those studies must be designed so families and doctors get meaningful information on dosing, safety and early effectiveness.  

The bill strengthens enforcement of existing pediatric study rules, allowing FDA to pursue penalties if a company fails to complete required child studies and hasn’t shown “due diligence.” It would also extend the federal priority review voucher program for rare pediatric diseases through September 30, 2029, preserving a key financial incentive for companies to develop treatments for very rare childhood conditions.  

For families, the changes would not create new treatments overnight. But over time, they could mean more clinical trials built for children rather than relying on adult data alone, especially in hard-to-treat cancers. Parents of children with cancer can ask their care teams whether a drug is being studied in pediatric trials and how to find reputable trial listings.

The measure has been introduced in the U.S. House and still must clear both chambers of Congress and be signed by the president before taking effect. Families can follow FDA and major childhood cancer organizations for updates as the bill moves forward.  

This article was produced by a journalist with the assistance of Ai. This is not legal advice. All content is reviewed for accuracy and fairness.