Ask Your Legislators to Co-Sponsor the OPEN ACT to Repurpose Drugs for Rare Disease Patients

Take action to support the OPEN ACT - Orphan Product Extensions Now, Accelerating Cures & Treatments (HR 971/S 1421).  The OPEN ACT could bring hundreds of safe, effective, and affordable medicines to rare disease patients within the next several years by incentivizing drug makers to repurpose therapies for the treatment of life-threatening rare diseases and pediatric cancers. EveryLife Foundation, the National Organization for Rare Disorders (NORD), Global Genes, Genetic Alliance and an additional 155 patient organizations support this bipartisan legislation. Please take a minute to phone your legislators and share this action alert on social media! All you have to do is edit the letter below to advocate for your disease community, enter your name, address, and click submit. In order to address your message to the appropriate recipient, we need to identify where you are. Please look up and use your full nine-digit zip for the best results. (If zip code link does not work, please use this link    

Take action to support the OPEN ACT - Orphan Product Extensions Now, Accelerating Cures & Treatments (HR 971/S 1421).  The OPEN ACT could bring hundreds of safe, effective, and affordable medicines to rare disease patients within the next several years by incentivizing drug makers to repurpose therapies for the treatment of life-threatening rare diseases and pediatric cancers. EveryLife Foundation, the National Organization for Rare Disorders (NORD), Global Genes, Genetic Alliance and an additional 155 patient organizations support this bipartisan legislation.

Please take a minute to phone your legislators and share this action alert on social media! All you have to do is edit the letter below to advocate for your disease community, enter your name, address, and click submit.

In order to address your message to the appropriate recipient, we need to identify where you are.
Please look up and use your full nine-digit zip for the best results.

(If zip code link does not work, please use this link
 

 

  Legislative Status The OPEN ACT passed the House in July of 2015, but was not signed into law. The bill was reintroduced in the House on February 27th, 2017.  The bill will be reintroduced in the Senate in early 2017. Click here to download an informational OPEN ACT one-sheet. Issue:  Despite advances made by the Orphan Drug Act, 95 percent of the 7,000 rare diseases still have no FDA-approved treatment. Biopharmaceutical companies seldom consider repurposing already approved therapies to treat rare diseases because there is little incentive for them to do so. Solution:  Modeled on the incentive programs in the Best Pharmaceuticals for Children Act (BPCA), the OPEN ACT establishes an exclusivity extension, which would provide an additional six months of market exclusivity for the drug being repurposed for rare disease treatment. The sponsor company must demonstrate that the repurposed therapy is designated to treat a rare disease and obtains an approved rare disease indication from the FDA on the drug label.  Repurposing drugs is faster, cheaper, and presents fewer risks than traditional drug development. Outcomes: Double the number of treatments for rare disease patients. Many of these drugs would be priced at major market drug prices, thus bringing down the average cost of rare disease drugs. A surge in biotech investment, new jobs, and grants to research universities to conduct clinical trials. Fewer rare disease patients using untested and potentially ineffective drugs off–label.   http://everylifefoundation.org/open-act/

 

Legislative Status

The OPEN ACT passed the House in July of 2015, but was not signed into law. The bill was reintroduced in the House on February 27th, 2017.  The bill will be reintroduced in the Senate in early 2017. Click here to download an informational OPEN ACT one-sheet.

Issue:  Despite advances made by the Orphan Drug Act, 95 percent of the 7,000 rare diseases still have no FDA-approved treatment. Biopharmaceutical companies seldom consider repurposing already approved therapies to treat rare diseases because there is little incentive for them to do so.

Solution:  Modeled on the incentive programs in the Best Pharmaceuticals for Children Act (BPCA), the OPEN ACT establishes an exclusivity extension, which would provide an additional six months of market exclusivity for the drug being repurposed for rare disease treatment. The sponsor company must demonstrate that the repurposed therapy is designated to treat a rare disease and obtains an approved rare disease indication from the FDA on the drug label.  Repurposing drugs is faster, cheaper, and presents fewer risks than traditional drug development.

Outcomes:

Double the number of treatments for rare disease patients. Many of these drugs would be priced at major market drug prices, thus bringing down the average cost of rare disease drugs.

A surge in biotech investment, new jobs, and grants to research universities to conduct clinical trials.

Fewer rare disease patients using untested and potentially ineffective drugs off–label.

 

http://everylifefoundation.org/open-act/