Rare Disease Report features the OPEN ACT (Orphan Product Extensions Now Accelerating Cures and Treatments)

The June 3rd edition of The Rare Disease Report has an article detailing the goals of the OPEN ACT, which aims to help rare disease patients through “rare purposing”. The OPEN ACT  (Orphan Product Extensions Now Accelerating Cures & Treatments) and was originally introduced by EveryLife Foundation President, Dr Emil Kakkis, at the World Orphan Drug Congress in April.

The idea behind the OPEN ACT is that many drugs currently on the market could be re-purposed for rare disease indications but as Ms Jenkins noted, companies are reluctant to add rare disease indications to their currently approved drugs since the financial benefits (small population) do not outweigh the risks (e.g., fear that potential adverse effects in clinical trials on very sick patients would risk the product’s market). In the meantime, 95% of rare disease have no approved treatments and at the current rate of 10 new approved drugs per year, it will take 500 years to treat all rare diseases. The OPEN ACT is designed to improve those numbers by changing the risk/benefit ratios that restrain companies from adding a rare disease indication to their already approved treatments.