More than 400 million people around the world, including almost 1 in 10 Americans, live with a rare disease. Last week, advocates for rare disease patients came together at the World Orphan Drug Congress to unite in support of the development of rare disease medicines, also known as "orphan drugs."
In the U.S., by definition, a rare disease affects fewer than 200,000 people. Such small patient populations and the complexity of many rare diseases often hinder the process of creating orphan drugs, lengthening the time it takes to develop and test them. In fact, less than 5% of the 6,800 rare diseases have treatments.
The MODDERN Cures Act can help. If passed, it would create a new class of medicines known as dormant therapies. This new class would align intellectual property protection with the scientific reality of creating a new treatment, providing medicines for rare diseases a concrete amount of patent protection. This would incentivize scientists to develop the best medicines, not just the medicines with the best patents.
By bringing together rare disease stakeholders from all paths, the 2014 World Orphan Drug Congress helped amplify all voices to promote advances in orphan drug development and improve patient access to lifesaving treatments.