I know firsthand the uphill battle parents of children with rare diseases face. Finding knowledgeable, compassionate care can feel like navigating an endless maze. Families are often left exhausted and overwhelmed as they search for specialists who understand their child’s condition and can offer real solutions.
Each year on the last day of February, Rare Disease Day shines a spotlight on the 400 million people worldwide living with a rare disease. Awareness matters, but awareness alone does not pay for lifesaving medications or remove the financial barriers that stand between patients and care.
That is why Congress’ recent passage of pharmacy benefit manager reform is so important. These bipartisan reforms represent a meaningful step toward fixing a system that has failed patients with rare diseases for too long.
PBMs were originally intended to help negotiate lower drug prices and manage prescription benefits. Over time, they became powerful middlemen whose opaque practices often drove prices higher, particularly for specialty and orphan drugs. Too often, rebates and fees benefited PBMs rather than patients at the pharmacy counter.
For individuals with rare diseases, treatment options are already limited. While more than 10,000 rare diseases have been identified, only a fraction have FDA- approved therapies. When treatments do exist, PBM practices have too often made them unaffordable.
Rare Disease Day is about action. With these reforms, Congress took a critical step toward a fairer, more transparent prescription drug system. For the 1.2 million Pennsylvanians living with rare diseases, this moment matters.
Jennifer Riley
Malvern