By Clinical Informatics News Staff

December 7, 2015 | Last year was the first time that all of the orphan drugs approved by FDA—17 total—took advantage of the Priority Review option. But Priority Review is only one of four options available to developers of orphan compounds for expedited FDA review. In a paper published in September in The Journal of Rare Disorders, authors posit that this indicates that the options are neither fully understood by orphan drug companies nor are not being used optimally by them.

In addition to Priority Review, drug developers can apply for Fast Track status, Accelerated Approval, or designation as a Breakthrough Therapy. The 17 approved drugs in 2014 used a mix of those four options, with Priority Review being the only option used in common across all 17.

“It appears to us, on examining the data presented, that some companies might have benefited if they had applied for more of the four options,” the authors say in the paper. They suggest several changes that could be made to the process. “We consider that there is now a case for waiving the Priority Review assessment process for all drugs granted orphan status and suggest that they should be granted Priority Review status automatically.” They also suggest combining the Accelerated Approval and Breakthrough Therapies processes to further simplify the process.

“A more unified system that reduces the current duplication of assessment processes would reduce resources required by the FDA, which is important as use of the four expedited approval methods is increasing,” the authors suggest. “It should at the same time be advantageous to companies making submissions, particularly those developing orphan drugs, whose resources—personnel, time, and financial—are often particularly limited.”

Read the paper.