CF Advocacy Campaign

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Time is critical! Please urge your Legislators to Approve the OPEN ACT Bill!

Urgent Call to Action! We need your help to pass HR 1223, the OPEN ACT (Orphan Product Extensions Now ~ Accelerating Cures & Treatments)!

The next few days are critical for this legislation, which could help double the number of affordable treatments available to rare disease patients. The OPEN ACT has broad bipartisan support and is supported by 224 patient organizations, including Cystic Fibrosis Research, Inc., Genetic Alliance, Global Genes, National MPS Society, the National Organization for Rare Disorders (NORD), and the Pediatric Cancer Foundation. The OPEN ACT passed the House in July of 2015, but was not signed into law. Due to legislative procedures, the bill was reintroduced in the House in February 2017.

Sadly, 95% of rare diseases still have no treatments approved by the Food and Drug Administration (FDA). The OPEN ACT would create incentives for pharmaceutical companies to develop medications for diseases that impact very small numbers of people in the United States, by providing an additional six months of market exclusivity for drugs being repurposed to treat a rare disease. These repurposed drugs must receive FDA approval for use. It is well established that repurposing drugs is the most efficient and effective way to save time and money to get needed drugs to the rare disease community, while presenting fewer risks to patients.

Passage of the OPEN ACT could double the number of drugs available to those impacted by a rare disease, including CF. It is important to note that these are drugs that have already been approved for use. The OPEN ACT could also allow for insurance reimbursement of otherwise off-label treatments or procedures, and reduce the cost of orphan therapies.

Please write to your representatives today, urging them to bring HR 1223 to a vote now!

CFRI’s Many Voices ~ One Voice Advocacy and Awareness Campaign needs your voice. Please share this message with your friends and families. You can make a difference!


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Approve the OPEN ACT Bill

Dear [Decision Maker],

HR 1223, the OPEN ACT (Orphan Product Extensions Now ~ Accelerating Cures & Treatments) legislation is vitally important to the rare disease community, including the cystic fibrosis (CF) community. The bill, which has wide bipartisan support and is endorsed by 224 patient advocacy groups, will help bring critical therapies to those living with rare diseases. Currently, 95% of rare diseases have no treatments approved by the Food and Drug Administration. Our cystic fibrosis community needs this legislation so that new treatments are made available for this disease, the most common fatal genetic disease in North America for which there is currently no cure.



Please act now and approve the OPEN ACT legislation, which would create incentives for pharmaceutical companies to develop medications for diseases that impact very small numbers of people in the United States, by providing an additional six months of "market exclusivity" for drugs being repurposed to treat a rare disease. These repurposed drugs must receive FDA approval for use. It is well established that repurposing drugs is the most efficient and effective way to save time and money to get needed drugs to the rare disease community, while presenting fewer risks to patients. With the new Congress, we cannot afford to allow the Act to languish. A delay in legislation will jeopardize the development of life-saving treatments. The OPEN ACT bill is patient-focused legislation that will assist those battling cystic fibrosis - the most common fatal genetic disease in North America - as well as other rare diseases. It is critically important that potentially life-saving drugs be repurposed to ease the cystic fibrosis and other rare disease communities' pain and suffering.

Please approve HR 1223, the OPEN ACT legislation!

The CF community is often overlooked since this complex disease impacts approximately 30,000 people in the U.S. and is considered a "rare disease" by the federal government, i.e., one that impacts less than 200,000 people in the nation. One in 29 Americans is believed to be a silent carrier of the mutated CF gene that leads to a defective exchange of fluids across cells, causing secretions to clog ducts and passageways in the lungs, pancreas, sinuses, and reproductive systems. The primary cause of death is respiratory failure.

It is heartbreaking that the median age of death for an individual diagnosed with cystic fibrosis is only 30 years.

I am proud to be part of Cystic Fibrosis Research, Incorporated (CFRI) and its efforts to increase federal funding for CF research and federal drug review processes. CFRI was founded in 1975, when children with CF rarely survived childhood. Based in Palo Alto, CA but with a global reach, CFRI provides vital education and support, promotes awareness, and funds CF research. Please vote to approve the OPEN ACT bill. You can make a difference in the lives of 30 million people affected by rare diseases in America, including 30,ooo with cystic fibrosis. Thank you for recognizing the overwhelming challenges of these life-shortening diseases and supporting funding to find new therapeutic treatments and a cure.

Sincerely,
[Your Name]
[Your Address]
[City, State ZIP]

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