When Mikaela Naylon was diagnosed with osteosarcoma—a rare and aggressive form of bone cancer—her parents struggled to find hope in a maze of few options. Osteosarcoma is a challenging cancer to cure or treat, and despite major gains decades ago, survival rates have largely plateaued.
Mikaela understood this better than most and wanted to help others. Before she died at age 16, she became an advocate for pediatric research, asking a simple, devastating question: why were there so few therapies for children like her?
For families confronting rare pediatric cancers, this question comes up again and again. Promising science exists. Innovative ideas exist. Yet too often, the pipeline that turns research into results runs dry before it reaches children with the rarest conditions.
Economics and market failure, instead of indifference, are the true culprits here.
That is why the Department of Health and Human Services recently encouraged Congress to advance the Mikaela Naylon Give Kids a Chance Act. The legislation restores and strengthens key incentives that drive investment into pediatric cancer and rare disease research, including reauthorizing the priority review voucher program.
Incentivizing the development of new cures and therapies for rare diseases is among the most stubborn challenges in modern health care. It is, at its core, an economic riddle with few easy answers. How do you persuade investors, researchers, and companies to commit time, talent, and capital to develop treatments that may serve only a small number of patients?
When development costs are high, failure rates are steep, and potential returns are limited, investment dries up, it creates a drought in innovation precisely where it is needed most. These misaligned incentives do more than distort markets; they leave serious medical needs unmet and prolong human suffering, especially for children with rare and life-threatening diseases.
Lawmakers from both parties have long recognized that markets alone do not adequately provide incentives for small patient populations.
One of the most effective tools has been the Rare Pediatric Disease Priority Review Voucher program (PRV), a market-based incentive created to unlock private investment where traditional economics fall short.
The concept is straightforward. When a company successfully develops and secures FDA approval for a treatment for a rare pediatric disease, it earns a priority review voucher. That voucher can be used to accelerate FDA review of another drug or sold to another company—often for a substantial sum. For smaller biotech firms, that value can mean the difference between advancing the next therapy or abandoning it altogether.
Importantly, the voucher does not relax safety or efficacy standards. FDA review remains rigorous. What changes is the timeline. The reward comes only after a company delivers an approved therapy for children with rare diseases. No success, no voucher. It is a pay-for-performance model that aligns incentives with outcomes.
The impact has been real. Since the program’s creation, dozens of treatments for rare pediatric diseases have reached patients, many representing first-ever therapies for conditions that previously had none. Before these incentives existed, only a handful of rare pediatric diseases had FDA-approved treatments. Today, the research landscape is still challenging, but measurably stronger due to the PRV program.
Yet this progress is fragile and potentially fleeting. The PRV has expired, and without congressional action, a proven incentive for pediatric innovation will disappear. The result would not be theoretical. Investment would retreat. Research pipelines would shrink. Families would face the same maze of limited options Mikaela Naylon knew too well.
The bill reflects a principle that has historically drawn bipartisan agreement: when the market fails to meet critical needs, smart, targeted incentives can help close the gap. The Kids First Research Act does not mandate outcomes or pick winners. Instead, it creates conditions that make it possible for innovation to occur. It aligns scientific promise with economic reality.
Critics sometimes frame incentives like vouchers as favors to industry. But that critique misses the structure of the program, and the stakes involved. These incentives are earned only after companies deliver tangible results for children with rare diseases. They channel private capital toward public good. And they do so efficiently.
The Mikaela Naylon Give Kids a Chance Act will not solve every challenge in pediatric cancer research. But it will preserve a proven tool that has already translated scientific breakthroughs into real treatments. In health care, when incentives lapse outcomes slow or stop all together. However, when aligned wisely, innovation follows.
Congress has an opportunity to choose progress and move cures for childhood cancer from impossible to inevitable. For children like Mikaela, it’s time to turn uncertainty into hope and hope into action.
The writer is Assistant Secretary of Legislation at the U.S. Department of Health and Human Services
Gary Andres is Assistant Secretary of Legislation at the U.S. Department of Health and Human Services
