The problem: Backlogs and outdated requirements
Another reason why the FDA is proposing changes is because in recent years, the proliferation of diagnostic tools and better understanding of genetic markers have afforded the ability to segment many common diseases into smaller, more precise groups that qualify for orphan designation, Dombal says.
In addition, some of the FDA’s proposed changes address process improvements, such as how to clear a backlog of more than 200 requests for orphan status. The number of requests received by the FDA under the Orphan Drug Designation (ODD) Program has more than doubled from 2012 to 2016.
“Not only has the number of requests increased dramatically, but the products that are the subject of requests have become more scientifically complex and may require specialized reviews from various FDA centers,” says Nancy Pire-Smerkanich, DRSc, MS, assistant professor, Department of Regulatory and Quality Sciences, University of Southern California, School of Pharmacy, Los Angeles, California.
How these changes will affect you
The FDA’s efforts will affect the healthcare industry in several ways. First, it will likely result in more clinical trials in pediatric populations. PREA requires that the FDA mandate clinical studies in a pediatric population for drugs approved for adult populations where they are likely to be used in a younger patient group. “Sponsors will no longer be able to gain exemption from pediatric studies via an orphan designation,” Dombal says.
The logical offshoot of increased studies in children will be significant; it could reveal new information regarding safety, efficacy, dose schedules, and other information for many drugs in the pediatric space, Dombal continues.
This could lead to a significant change (and perhaps an improvement) in drug labeling. This would increase the complexity of managing formularies, pharmacy operations, and clinical administration. On the plus side, clinical studies in the actual population will lead to significantly greater consistency in dosing, decreasing dosing errors and increasing safety for pediatric patients.
Karen Appold is a medical writer in Lehigh Valley, Pennsylvania.