Work continues on the part of 237 patient advocacy organizations to try to pass the OPEN Act (Orphan Product Extensions Now, Accelerating Cures and Treatments; HR 1223 / S 1509). The HPS Network is one of the patient organizations supporting this bill.
The OPEN Act offers incentives to pharmaceutical companies to repurpose existing drugs for use to treat rare diseases. Currently, only 5 percent of the more than 7,000 rare diseases have an FDA approved treatment. Many symptoms for rare diseases, like HPS, are often treated with medications that are off label, and thus can sometimes be difficult to pay for. The bill would offer pharma companies an extended six months of patent exclusivity if they work to repurpose medications for use in rare diseases. 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.
The legislation has the potential to double the number of treatments available for rare disorders.
Asking your House and Senate representatives to support, or co-sponsor, this legislation is easy. Go to this link and it will automatically locate your representatives as well as provide a ready-made letter you can send them. Just enter your information in the provided fields. http://action.everylifefoundation.org/p/dia/action4/common/public/?action_KEY=22781