As we saw with COVID-19 vaccines, regulatory review processes must balance the desire for speed with the non-negotiables of safety and effectiveness.
But even prior to the global pandemic, the U.S. Food and Drug Administration had a process for giving priority review to potential treatments that “if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.”
Last month, the FDA accepted CSL Behring’s Biologics License Application for priority review of a potential gene therapy treatment for hemophilia B. The designation means the FDA, which determines whether new treatments are approved for use in the United States, will set a goal to take action on an application within six months. Standard review is 10 months.
The FDA’s priority review designation does not affect the length of clinical trials or change the scientific standards or evidence necessary, the FDA says. The agency also has three additional approaches for reviewing potential new treatments: fast track; breakthrough therapy; and accelerated approval.
CSL Behring’s potential treatment for hemophilia B, a rare bleeding disorder, previously received a similar designation called accelerated assessment from the European Medicines Agency (EMA). The EMA asks applicants to show the potential product is of “major public health interest, particularly from the point of view of therapeutic innovation.”