A Pediatrician’s Plea: Pass the Give Kids a Chance Act

I’ve cared for children and families for decades, and there is nothing more heartbreaking than telling parents that no treatment exists for their child’s illness—and worse, that no one is even working on one.

This is the reality for thousands of children with rare diseases. Half of all rare disease patients are kids, and 95% of rare diseases lack an FDA-approved therapy. That must change.

Congress can help right now by passing the Give Kids a Chance Act, a bipartisan, no-cost reform that will accelerate innovation and deliver hope to families. This act strengthens the Pediatric Rare Disease Priority Review Voucher program, which has already led to dozens of lifesaving therapies.

For example, treatments for Rett Syndrome and pediatric neuroblastoma—conditions once viewed as hopeless—are available today thanks in part to this program. Yet the program has a glaring gap: if a therapy is being developed for an adult version of a disease, it’s often blocked from receiving pediatric rare disease incentives, even when it could help a child. The Give Kids a Chance Act closes that loophole.

This is about more than policy—it’s about doing right by our most vulnerable children. Congress must include the Give Kids a Chance Act in September’s funding package. Our kids deserve nothing less.

By Dr. Barry Ricks