How to Keep the Momentum Going in Orphan Drugs

The passage of the 21^st Century Cures Act by the US House of Representatives means that there’s potential for faster drug development in the future. That’s good news for everyone.

By every measure, the world of orphan drugs is growing rapidly. According to the Wall Street Journal, this was apparent in 2014. There was a 35% increase in requests for orphan designations (467), a 13% increase in drugs granted orphan status (293) and an impressive 53% increase in orphan drugs approved (49) as compared to 2013.

Orphan drugs have the potential to reduce the cost of complex medical care for rare disease patients, more than 50% of whom are children suffering from chronic and debilitating conditions. According to an estimate done by Pacific Research, the cost of rare diseases in terms of healthcare expenditures, lost wages and lost productivity is currently around $474.5 billion per year. Effective treatment could lower these costs, while providing better quality of life for patients.

But is the orphan drug sector sustainable? “It has to be,” Henri Termeer, former president of Genzyme, said in his address to the 5^th annual World Orphan Drug Congress USA in April.

President Obama’s Precision Medicine Initiative recognizes the importance of orphan drugs not just to rare disease patients, but also to healthcare in general. A tweet posted by NIH Director Francis Collins during the Precision Medicine Initiative Working Group Meeting in April noted that “Identifying molecular causes of rare genetic disorders in #PrecisionMedicine informs the diagnosis and treatment of common disorders.”

But at the same time, the government is asking businesses, healthcare institutions and consumers to work together to help keep costs under control. Is there a way to keep the momentum going in orphan drugs and also curb the rise in health costs?

Siren and Dohmen are working to provide innovative solutions to that challenge. We believe that the future of orphan drugs must include new models of integrated care and data capture that will enable strides in diagnosis, adherence and matching treatment to patients. We’re looking forward to partnering with you to bring that future to life.