Orphan drugs are called “orphans” because the biopharma industry is said to have little interest in developing and marketing drugs intended for only a small number of patients suffering from very rare conditions. And yet, in the United States, according to a Food and Drug Administration report released Dec. 5, 2012, orphan drug approvals represented 40 percent of the agency’s total drug approvals in 2012. Much of this is due to the legislative and regulatory attention that has been paid to orphan drugs.  

The Orphan Drug Act of 1983 (ODA) has provisions for Prescription Drug User Fee Act (PDUFA) fee exemptions, tax credits, and exclusivity to serve as incentives to orphan drug developers. PDUFA V, which was signed into law last July, offers new incentives, including breakthrough exclusivity and vouchers for pediatric rare diseases.  PDUFA V also increases the role of patients in developing orphan drugs by requiring FDA to implement ways to incorporate patients' views into drug development and FDA’s approval process.  

In Canada, John Norman and Livia Aumand of Gowlings, Ottawa, Canada, told me last month that in October 2012, Health Canada, the country’s federal health department, announced that it would be creating a new regulatory framework for the development, evaluation, and approval of orphan drugs. 

“Both the United States and the European Union already have orphan drug legislationin place.  Health Canada has indicated that the new framework will simplify the approval process for orphan drugs and incentivize research and innovation in this area.  It is expected that 2013 will bring the publication of the regulations for public consultation,” Norman and Aumand said.