Medications that treat rare medical conditions have historically proven difficult to both develop and market. As a result, they haven’t historically been a priority for large drug manufacturers.
“Orphan drugs”, medication defined in the 1983 Orphan Drug Act as treating diseases affecting fewer than 200,000 people, are expensive to develop, expensive to buy and often unappealing to commercial developers because of low returns on investment.
Nonetheless, developing and commercializing medications for the few people who do need them is certainly worthwhile. There are approximately 7,000 rare, often life-threatening disorders.
As Canada’s federal health department, Health Canada, readies itself to boost orphan drug research, development and access, University of Minnesota researchers are looking for ways to do the same in the United States. Whereas Canada will be building an entirely new policy, U of M researchers are building on one that already exists.
“As a nation, we spend most of our drug development dollars developing medications for conditions such as asthma, erectile dysfunction, and high blood pressure, which affect a large number of people,” said James Cloyd, Pharm.D., Lawrence C. Weaver Endowed-Chair for Orphan Drug Development and Director of the Center for Orphan Drug Research (CODR) within the University of Minnesota’s College of Pharmacy. “But as we learn more about the underlying causes of rare diseases, it’s becoming easier to develop drugs that treat these conditions.”
It has been nearly thirty years since the passage of the Orphan Drug Act, which created a mechanism for developing and marketing orphan drugs for use by a relatively small number of patients. The Act provided several incentives including federal funding for orphan drug research including exemption from Food and Drug Administration (FDA) filing fees, and seven years of market exclusivity.
Over the past thirty years the Orphan Drug Act has proven remarkably successful: it has resulted in FDA approval of 410 medications for treatment of rare disorders.
However, according to Cloyd, today almost 2,500 compounds designated as orphan products remain in development or have been abandoned.
In his current project “Assessing and Improving Orphan Product Development” at the CODR, Cloyd is looking to change that.
“Our project, once finished, will offer insights into the steps needed to capitalize on this opportunity. As a result, legislation or changes in regulations may be enacted to allow the further development of this pool of potential medications for rare conditions,” he said.
For more on Dr. Cloyd, visit the CODR website.