Bill will speed up the process for approving new drugs and treatment options for Americans affected by rare diseases.
WASHINGTON, D.C. – Today, U.S. Senators John Barrasso (R-Wyo.) and Martin Heinrich (D-N.M.) introduced bipartisan legislation to make it easier to develop new life-saving cures for Americans affected by rare diseases. The Optimizing Research Progress Hope and New (ORPHAN) Cures Act supports the development of breakthrough treatments and cures through the continued investment in American innovation.
“Helping patients with rare diseases find treatment has been a priority of mine since my time as a doctor in Wyoming,” said Barrasso. “We should be making it as easy as possible to develop and to access new cures and treatments, not harder. Our bipartisan ORPHAN Cures Act ensures America continues to lead in drug development and can continue bringing cures and hope to the millions living with rare diseases.”
“Rare disease drug development is uniquely challenging, and a one-size-fits-all approach to policy can stymie innovation for the 30 million Americans living with a rare disease,” said Stacey Frisk, Executive Director of the Rare Disease Company Coalition. “The ORPHAN Cures Act removes harmful barriers to innovation and opens the door for promising research, providing much-needed hope to the rare disease community. We thank Senators Barrasso and Heinrich for recognizing the needs of rare disease patients and championing this crucial bill.”
Click HERE for more statements of support for the ORPHAN Cures Act.
The Optimizing Research Progress Hope and New (ORPHAN) Cures Act is supported by: Arya Singh, MPH/BA, Public Health Researcher & Rare Disease Advocate, AiArthritis, Alliance to Cure Cavernous Malformation, The Bonnell Foundation: Living with Cystic Fibrosis, CancerCare, Cancer Support Community, Chondrosarcoma Foundation, Cholangiocarcinoma Foundation, Cure CMD, Cure POGZ Disorders Foundation, Cure LGMD2i, Eosinophilic & Rare disease Cooperative, FORCE: Facing Hereditary Cancer EMPOWERED, Firefly Fund: Shining a Light on NPCI, Friedreich’s Ataxia Research Alliance (FARA), Global Liver Institute, Histiocytosis Association, Littlest Tumor Foundation, IgA Nephropathy Foundation of America, Little Hercules Foundation, The Mended Hearts, Inc., Neurofibromatosis Network, PBC Research Foundation, Prevent Blindness, Parkinson & Movement Disorder Alliance, Hope for Stomach Cancer, Coalition of Skin Diseases, Tigerlily Foundation, Undiagnosed Diseases Network Foundation, Agios, AstraZeneca, Alnylam, Alexion AstraZeneca Rare Disease, Alkermes, BIO, California Life Sciences, Daiichi-Sankyo, Incyte, Jazz Pharmaceuticals, National Pharmaceutical Council, Rare Access Action Project, Rare Disease Company Coalition, Takeda and UCB.
Background:
Under current federal law, a drug or treatment that receives approval from the U.S. Food and Drug Administration (FDA) to treat exclusively one rare disease – commonly known as an “orphan drug” – is eligible for certain incentives, including an exemption from Medicare’s drug negotiation program.
These same incentives do not exist if an orphan drug receives FDA approval to treat two or more rare diseases. This in turn discourages American innovators from engaging in the expensive and time-intensive research necessary to determine if an orphan drug could cure or treat additional rare diseases.
The ORPHAN Cures Act fixes this unintended consequence by exempting drugs that treat more than one rare disease from Medicare’s drug negotiation program.
###