ERDC submits a letter for the record to the US Senate Finance Committee about the importance of correcting the Orphan Cures Act to ensure rare disease research continues.
September 16, 2024
Statement for the Record for Senate Finance Committee, September 17, 2024, Full Committee Hearing “Lower Health Care Costs for Americans: Understanding the Benefits of the Inflation Reduction Act”.
We, Eosinophilic and Rare Disease Cooperative, ERDC, write in support of the Orphan Cures Act Legislation.
We are a national non-profit organization providing support and resources for individuals effected by rare disease to more effectively access quality care and treatments in 48 states. This year we have served people with 37 rare diseases, with the majority of our work helping those with multiple forms of Vasculitis and Myasthenia Gravis.
Over 30 million Americans have a rare disease and 95 percent of them do not have an FDA approved treatment for their condition. The ORPHAN Cures Act legislation would correct the narrow orphan drug exclusions. The MDPNP Orphan Drug Exemption creates financial disincentives to pursue orphan drug development.
In our work we see the need for this legislation every day. People with vasculitis have an expected lifespan at diagnosis of 5- 8 years without treatment. People with myasthenia gravis often experience a life threatening complication – myasthenia crisis – in which the muscles that control breathing are too weak to do their job resulting in respiratory failure. As with many rare diseases, the treatments are not a necessity, they are literally life and death. Only a few of the twenty forms of vasculitis have FDA approved treatments. This legislation will assist in incentivizing investors to continue to pursue treatment options.
The changes in this legislation are critical to ensuring that individuals with rare disease, including ultra rare, can continue to benefit from scientific research in their disease states. Not only does this legislation provide hope for the millions effected by rare disease, but will ultimately have a cascading effect on the pressures in the health care system. Those with a rare disease have double the costs when hospitalized, not to mention that rare diseases predominantly effect women.
Because we at Eosinophilic and Rare Disease, ERDC, support individuals with rare diseases, we whole heartedly support S. 3131, the Orphan Cures Act.
