TribLive: Congress takes critical step for rare disease families
By Jennifer Riley, Patients Come First Pennsylvania’s Executive Director
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.
Read the full LTE in TribLive here.