Championing rare disease innovation, ending foreign dependence Image By Gus Bilirakis Every patient deserves a chance at a healthy life — especially those facing the most devastating, life-threatening illnesses. Yet, of the more than 10,000 known rare diseases, only 5% have an FDA-approved treatment. For children, the stakes are even higher: approximately 30% of those diagnosed with a rare disease will not live to see their fifth birthday. These numbers are not just tragic — they’re unacceptable. Fortunately, one of the most effective tools created to combat this crisis has been the Rare Pediatric Disease Priority Review Voucher (RPD PRV) program. This market-based solution has helped bring more than 50 treatments to children suffering from conditions like spinal muscular atrophy, cystic fibrosis, and Friedreich’s ataxia — 36 of which had no previous FDA-approved treatments. However, this lifesaving program expired on Dec. 20, 2024. Without immediate reauthorization, the RPD PRV program’s proven incentives for pharmaceutical companies to invest in rare pediatric disease research will vanish. Critical program must continue As co-chair of the Congressional Rare Disease Caucus, I lead the bipartisan charge to reauthorize this critical program. Clinical trials for these conditions are already among the most complex and costly in medicine. Without the potential for priority review vouchers — which can fast-track FDA approval or be sold to fund additional R&D — many promising therapies may never reach the children who desperately need them. This isn’t just about science. It’s about values. It's about whether we are willing to support a program that has delivered life-changing results at zero cost to taxpayers, simply by harnessing the efficiency of the free market to reward innovation that saves lives. This moment in history also calls for a broader reckoning. While we reignite rare disease innovation, we must also confront a second crisis: America’s over-reliance on adversarial nations — particularly China — for essential pharmaceutical ingredients and finished drugs. Drug supply chain poses risk Today, nearly 80% of the active pharmaceutical ingredients (APIs) used in U.S. medications come from overseas. This includes everything from high-tech prescription drugs to basic over-the-counter medications like Tylenol. This isn’t just a supply chain vulnerability — it’s a national security risk. We learned this the hard way during the COVID-19 pandemic, when medicine shortages exposed just how fragile our pharmaceutical pipeline truly is. If geopolitical tensions escalate, the U.S. could find itself without access to critical medications. That’s a terrifying prospect, especially for patients with chronic or life-threatening conditions. As we navigate a rapidly evolving international landscape, we simply must address this strategic need. We cannot allow China and other foreign adversaries to interrupt our supply chains. Instead, we must re-focus our efforts to ensure more products are made in the USA. Through my work on the Select Committee on the CCP and as co-founder of the Domestic Pharmaceutical Manufacturing Caucus, I will continue to work on this priority policy area. Bold action to help the most vulnerable We need to act now. Reauthorizing the RPD PRV program should be an easy, bipartisan win. It has already helped hundreds of thousands of children and embodies the very best of American values: compassion, innovation, and smart policy that rewards private-sector solutions to public health challenges. But we must also go further. Congress and the administration must make domestic pharmaceutical manufacturing a national priority. We must invest in U.S.-based production capacity, incentivizing the reshoring of essential drug manufacturing, and ensuring that Americans are never dependent on the goodwill of geopolitical adversaries for their medicine. Our rarest diseases and our most vulnerable citizens deserve our boldest action. The path forward is clear: reauthorize the RPD PRV program, invest in domestic drug manufacturing, and put American health security first. Together, we can make America Healthy Again! U.S. Rep. Gus Bilirakis, a Republican, represents Florida's 12th Congressional District and is a senior member on the House Health Subcommittee. *The opinions expressed in this column are those of the author and do not necessarily reflect the views of HealthPlatform.News.
