OPEN Act introduced in USA to repurpose drugs for rare diseases

Last week, US Representatives Gus Bilirakis (Republican, Florida) and G K Butterfield (Democrat, North Carolina) took a bipartisan step on behalf of patients with rare diseases by introducing the Orphan Product Extensions Now Accelerating Cures and Treatments Act of 2014 (OPEN ACT).

The Act will incentivize drug makers and innovators to "repurpose" major market drugs for life-threatening rare diseases and pediatric cancers, which opens the door to the development of hundreds of safe, effective, and affordable treatments for rare disease patients. Original co-sponsors of the legislation include Representatives McCaul (Rep, Texas) and Hastings (Dem, Florida).

"The OPEN Act has the potential to result in hundreds of new drugs and treatments for individuals with rare diseases, as well as a new surge in biotechnology jobs and investments. This bill can help millions of people by ensuring medications are safe and effective for rare conditions, and can be reimbursed through insurance coverage," said Rep Bilirakis.