By Delman Lebel, head of U.S. Corporate Affairs at Alexion, AstraZeneca Rare Disease and Chair of the Rare Disease Company Coalition
Finding cures for rare diseases is a fundamentally nonpartisan pursuit. That’s why members of the Rare Disease Company Coalition (RDCC) are calling on our elected leaders to seize this unique opportunity for bipartisanship, reach across the aisle and unite on common-sense legislation to offer hope to millions of Americans living with rare diseases.
Right now, over 30 million Americans, many of whom are children, live with a rare disease. These aren’t abstract medical conditions; they’re the daily realities for families facing diagnoses that too often carry a devastating prognosis – and a glaring lack of treatment options.
A staggering 95% of these 10,000 diseases lack FDA-approved therapies. Diagnosis can take years, and during that time the disease can quickly progress. For those in need of treatment the clock is ticking, and the key to progress lies with our lawmakers.
As Chair of the RDCC, an advocacy-focused coalition representing life science companies committed to discovering, developing, and delivering rare disease treatments for patients, I understand the unique challenges that come with advancing rare disease therapies. Developing treatments for rare diseases involves creating animal models from scratch, difficulty in recruiting participants for clinical trials, and navigating high costs and risks. It often takes over a decade of sustained investment to move from bench to bedside. Because each disease affects a small population, traditional market incentives are insufficient. This is why a robust and supportive ecosystem with key incentives is crucial. To foster this kind of ecosystem, we need the support of Congress.
Three bipartisan, common-sense legislative priorities offer a concrete path forward to ensure scientific breakthroughs continue and the roughly 30 million Americans living with a rare disease are not left behind.
First, we need to remove barriers that hinder scientific progress, such as the disincentive within the Inflation Reduction Act (IRA) that hinders rare disease innovation. The IRA’s Orphan Drug Exclusion provides a price negotiation exemption for orphan drugs that treat only one orphan, or rare, condition. However, 1 in 5 orphan drugs are FDA-approved for more than one use, and 60% of those second indications are for another rare disease. This unnecessary hurdle discourages companies from further pursuing promising research that could lead to additional treatment options. The ORPHAN Cures Act would expand the single-orphan exclusion, allowing companies to follow the science and invest in promising treatments using existing research. This means more treatment possibilities would be within reach.
Second, we must preserve incentives to develop therapies for rare diseases. The rare pediatric disease priority review voucher (PRV) program has been a proven success, encouraging drug development for rare pediatric diseases by expediting the regulatory process for qualifying products. Since its inception, the PRV program has led to the approval of dozens of life-changing therapies for children, benefiting over 200,000 patients and addressing high unmet needs across 47 rare pediatric indications. However, as of December 20, 2024, the PRV Program has lapsed and begun to expire. Without urgent Congressional action, this incentive, along with hope for millions of children, may vanish for good.
Finally, we must restore the Orphan Drug Tax Credit (ODTC) to its original value. This credit is essential for many small biotech companies leading rare disease research. It promotes research by lowering development costs for manufacturers, reducing the significant financial cost and risk that comes with creating new therapies for small patient populations.
The ODTC was cut in half in 2017 and we are already seeing a chilling effect, making it harder for these companies to pursue potentially life-saving research. Rare disease companies saw nearly $10 billion less in investment available for research in 2022, stemming from decreases in venture capital investments, the IPO market, and partnership revenues. Restoring the credit isn’t just smart policy; it’s a moral imperative. Without it, many promising projects will simply never get off the ground, leaving patients with little hope for treatment.
These aren’t just legislative checkboxes; they are essential to preserve the delicate ecosystem of rare disease research and development, ensuring that scientific progress can continue despite the challenges. They create hope for families who have spent far too long searching for answers.
Policymakers have a chance to make a real difference, to show the American people that bipartisanship isn’t dead and that progress is still possible. By prioritizing these common-sense measures, our lawmakers can send a powerful message: the rare disease community matters, their struggles are seen, and healthier futures for patients living with a rare disease are worth fighting for.