Mariannette Miller-Meeks

Medicare Physician Data-driven Performance Payment System Act of 2026

in committee

Rural and Municipal Utility Cybersecurity Act

introduced

Fisher House Availability Act of 2026

introduced

“Mrs. MILLER-MEEKS. Mr. Speaker, I rise today to recognize the importance of the Orphan Drug Act in driving rare disease therapy clinical research in my district at the University [[Page E487]] of Iowa. As a physician, a legislator, and a member of the Rare Disease Congressional Caucus, I have seen firsthand the life-changing impact that medical innovation can have on patients battling rare diseases. Iowa's first congressional district is home to clinical trials for several rare diseases that lack a Food and Drug Administration (FDA)- approved therapy, including Becker muscular dystrophy, Gaucher disease type 3, and limb girdle muscular dystrophy. For these patients, and the millions of Americans suffering from rare diseases, scientific advancements represent more than just medical progress--they offer hope where none previously existed. That hope would not be possible without the Orphan Drug Act, which has served as the foundation for rare disease drug development for over four decades. The Orphan Drug Act's most critical incentive is orphan drug exclusivity, which is a seven-year period of market exclusivity. This incentive ensures that innovators willing to take the extraordinary risk of developing a first-in-class treatment for a complex condition affecting a small patient population are appropriately rewarded. For many rare diseases, the first FDA-approved treatment is a lifeline. These therapies often require years of research, billions in investment, and considerable perseverance to bring these medicines to patients. Without a strong and reliable exclusivity framework, these treatments simply would not exist. Congress intended orphan exclusivity to serve as ``a marketplace-oriented solution that provide incentives for private enterprise to develop orphan drugs.'' FDA established regulations that aimed to protect this critical economic incentive while also encouraging continued innovation for a rare disease by allowing a follow-on product that is the same drug for the same use to break orphan exclusivity if it demonstrates greater efficacy, greater safety, or a ``major contribution to patient care'' relative to the original orphan drug. Congress codified these regulations in 2017. Congress has long recognized that the ``major contribution care'' prong of the ``clinical superiority'' standard is meant to be a high bar. We did not intend for minor modifications such as changes in dosing frequency or formulation to erode the exclusivity protections of the first innovator drug unless such modifications result in a significant clinical benefit in treating the underlying conditions. If we allow marginal improvements rather than true clinical advancements to justify breaking orphan exclusivity, we undermine the very incentive that encourages companies to develop first-in-class therapies in the first place. The FDA has a clear precedent for maintaining a high bar for clinical superiority determinations. If we lower the bar for breaking orphan exclusivity, especially for ``major contribution to patient care,'' we will undermine the future of rare disease drug development. Lowering the bar will deter investment in first-in-class therapies by reducing the certainty that companies will be able to benefit from the exclusivity protections Congress intended. Lowering the bar will create regulatory uncertainty, leading to litigation, market instability, and hesitation from investors who fund rare disease innovation. Lowering the bar will set a dangerous precedent for copycat products with few modifications, encouraging incremental changes rather than bold scientific advancements. Lowering the bar will hurt patients most of all, delaying or discouraging the development of a first-ever therapy for the nearly 10,000 rare diseases that still lack an FDA-approved treatment. As a physician, I know that true medical breakthroughs require bold investment, sustained commitment, and regulatory certainty. As a legislator, I am committed to preserving the integrity of the Orphan Drug Act and ensuring that it continues to drive life-changing treatments for patients. FDA must apply the law a Congress intended and not allow minor modifications to erode exclusivity protections that serve as the foundation for rare disease innovation. Today, I am calling on my fellow members of the Rare Disease Congressional Caucus to stand together to protect the value of the incentives established in the Orphan Drug Act. ____________________”

2025-05-29 · Healthcare