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news.US FDA and the European Medicines Agency (EMA) have come out with a more simpler process to help regulators better recognize and share information throughout the development process of orphan drug and biologic products, which are developed specifically to treat rare medical conditions.
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Both agencies have consented 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. At present, if an orphan product was awarded designation on exactly the same day in both the US and EU, sponsors must submit individual reports to their corresponding regulatory agency.
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 agencies. The use of one annual report will also be advantageous to sponsors by avoiding the duplication of efforts and by streamlining the process that meets the annual reporting requirements of both the US and the EU for orphan designated products.
Timothy Cote, director of FDA’s Office of orphan products development, said: “This process provides benefits for both agencies. Additionally, it reduces the duplication involved for sponsors in reporting to two separate regulatory agencies.”
The single annual report, much like individual agency reports, 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 impact their designation as an orphan product.
The optional new process for submission will not introduce any further 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 interchange the annual reports electronically through a secure portal.
Jordi Llinares, head of orphan medicines at the EMA, said: “We are very pleased with this collaboration on regulatory requirements and about sharing data that will help us understand the viability of the products.”
Kerstin Westermark, chairman of the committee for orphan medicinal products of EMA, said: “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.”