The Rare Disease Company Coalition today issued the following statement from Executive Director Amanda Malakoff in response to the three payment models released by Health and Human Services for testing by the Centers for Medicare & Medicaid Services. The proposal, which aims to reduce Medicare spending for some accelerated approval drugs, undermines the intent of the pathway and mischaracterizes the extent of sponsors’ clinical research. Any attempt at indication-based pricing by the US government would have enormous impacts on rare disease patients, as well as on the companies investing in critical research.
“The Rare Disease Company Coalition stands against policies that put safe and effective treatments further away from the 1 in 10 Americans that live with a rare disease. A recent Health and Human Services (HHS) proposal on payment models for testing by CMS (Centers for Medicare & Medicaid Services) does just that by limiting coverage for drugs developed under the proven accelerated approval pathway. While intended to cut costs, it would harm patients with serious, life-threatening rare diseases and make it more difficult for companies to invest in critical research. The RDCC supports policies that preserve the use of accelerated approval as mandated by Congress, and give all patients who have debilitating rare diseases a chance to be treated with cutting edge, and often life-saving science. We urge members of Congress to recognize the harmful consequences of undercutting reimbursement, coverage and patient access to therapies developed under the accelerated approval pathway.”
Amanda Malakoff, Executive Director, Rare Disease Company Coalition
The FDA’s accelerated approval pathway recognizes that a “one-size-fits-all” traditional model for the approval of new medicines cannot always work – especially for rare diseases. This well-established, proven, scientifically rigorous path forward enables rare disease companies to make promising new and improved treatments available to patients at the earliest possible point in time for use in life-threatening and serious rare diseases.