FDA and EMA accept single orphan drug annual report
US and Europe in co-operative move to speed annual reports
The US FDA and the European Medicines Agency (EMA) have agreed on a more streamlined process to help regulators better identify and share information throughout the development process of orphan drug and biologic products, which are developed specifically to treat rare medical conditions. With effect from 28 February 2010, both agencies have agreed to accept the submission of a single annual report from sponsors of orphan drug and biologic products designated by both the US and the EU.
Until now, if an orphan product were granted designation on the exact same day in both locations, sponsors would have to submit separate reports to their respective regulatory agency. The use of one annual report will also benefit sponsors by eliminating the duplication of efforts and by simplifying the process that meets the annual reporting requirements of both the US and the EU for orphan designated products.
"This process provides benefits for both agencies," said Dr Timothy Cote, director of FDA's Office of Orphan Products Development. "Additionally, it reduces the duplication involved for sponsors in reporting to two separate regulatory agencies."
The single annual report will provide information to both agencies on the development of orphan medical products, including a review and status of ongoing clinical studies, a description of the investigation plan for the coming year and anticipated or current problems in the process that may affect their designation as an orphan product. The single annual report submission to both regulatory agencies is voluntary and will apply only to sponsors who have obtained an orphan designation status for their product from both the FDA and EMA.
The optional new process for submission will not introduce any additional regulatory requirements. Each regulatory body will conduct its own review and assessment of the annual report to assure the information meets all the legal and scientific requirements of each agency. The FDA and EMA will exchange the annual reports electronically through a secure portal.
"This new step in our collaboration provides each of our agencies with information in real-time on any challenges arising during the development of products for rare diseases and will help identifying and acting on bottlenecks," said Kerstin Westermark, chair of the EMA's Committee for Orphan Medicinal Products.