EveryLife Issues Statement on Rare Disease Community’s Drug Pricing Considerations

Context: Senate Finance Committee Hearing, Prescription Drug Price Inflation: An Urgent Need to Lower Drug Prices in Medicare

On behalf of the EveryLife Foundation for Rare Diseases and the more than 30 million Americans living with a rare disease or disorder, we are grateful for the leadership and commitment to making sure all Americans can access the lifesaving treatments they need.

As Congressional leaders consider different policy options for lowering the cost of prescription medications, we urge that the needs of our community are central, including the unique complexities of rare disease drug development and the high unmet need faced by the more than 30 million Americans living with rare diseases. While exciting advances in science and medicine have propelled new rare disease therapies into reality, between 93-95% of the over 7,000 rare diseases still have no FDA approved treatment. For those living without approved treatments, policies intended to lower drug costs without consideration of the impact on incentives for rare disease research and development infrastructure will mean lives are lost and unnecessary suffering will be extended.

The National Economic Burden of Rare Disease Study in the United States estimated in 2019 the overall annual economic burden of rare disease exceeded $966 billion. Of the total economic burden, the largest costs were indirect costs from productivity losses at $437 billion, direct medical costs at $418 billion and non-medical and uncovered healthcare costs of $111 billion absorbed directly by families living with rare diseases. Aside from absenteeism, inpatient care was the biggest expense, accounting for nearly 15% of the overall economic burden while prescription medication and administration costs accounted for about 10% and outpatient care for about 6%. Meaningful policy engagements around affordability must focus on more than just one aspect of the overall health care system¹.

To inform ongoing discussions, the EveryLife Foundation developed the enclosed Drug Pricing Statement in collaboration with the rare disease community. The statement conveys goals that any proposed drug pricing policies should be evaluated against. These goals fall into the areas of; incentives for rare disease therapeutic development, upholding the role of the FDA, innovations in payment models, putting patients at the center of defining value and decreasing the time and cost related access hurdles faced by patients. Read the statement.

We are grateful for the commitment to ensuring that all Americans can access the lifesaving treatments they need and stand ready to engage the community in further, patient-community-centered discussions.

Sources

1. The EveryLife Foundation. The National Economic Burden of Rare Disease. www.burdenstudy.org. Accessed May 4, 2021.