This week, Rare Disease Day 2017 was celebrated in over 90 countries around the world, to help raise awareness of the 350 million people currently living with a rare disease. In honor of this day, in the US, the Orphan Product Extensions Now Accelerating Cures and Treatments Act, or the OPEN Act (H.R. 1223), was reintroduced by Congressman Gus Bilirakis.
This bill focuses on improving treatment access for rare disease patients. There are around 7,000 rare diseases, affecting almost 30 million Americans. However, the problem remains that, for 95% those conditions, there is no approved treatment, let alone a cure.
There are some incentives provided to companies in the US to develop new drugs for these diseases. But, because each disease affects such a small patient population, there exists little opportunity for pharma companies to offset their R&D and marketing costs without charging patients extortionate amounts. Furthermore, small patient populations make it hard for companies to recruit patients for clinical trials and prove the effectiveness of their products when seeking regulatory approval for market use. Because of this it can take up to 14 years to bring a new drug to market.
The OPEN Act is intended to remove these barriers to providing safe, effective, and reasonably priced medications in a timely manner. The bill proposes providing incentives for pharmaceutical companies to repurpose their already approved drugs, meaning discovering new uses for drugs that are already being used on the market. Because such an approach builds on previous R&D efforts, less time and money has to be spent on getting a drug approved for a rare disease.
This bill was initially included in the 21st Century Cures Act, but was removed prior to the approval of the Cures Act in December of 2016. Since then, more than 150 rare disease patient advocacy groups have been pressuring government to reconsider the OPEN Act.