The European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) have agreed to accept a single annual report for orphan drugs for use in both the EU and the US.
The announcement coincides with World Rare Disease Day (28th February), which seeks to call attention to rare diseases as a public health issue, and to reinforce the increased need for access to information, research and treatment.
The use of just one report will simplify the process and reduce the workload for sponsor companies, but also make it easier for the EMA and FDA to share information.
Previously both regulatory agents required the submission of an annual report for orphan designated products. These would include a review of ongoing clinical studies, an investigation plan for the coming year and any potential changes that may impact the product’s orphan status.
The single annual report is voluntary, and only applies to sponsors who have obtained orphan designation status in both the EU and the US. Each regulatory body will conduct its own assessment of the report to assure the information meets all of its legal and scientific requirements.
“This process provides benefits for both agencies,” said Timothy Coté, Director of FDA’s Office of Orphan Products Development. “Additionally, it reduces the duplication involved for sponsors in reporting to two separate regulatory agencies.”
Professor Kerstin Westermark, Chair of the EMA Committee for Orphan Medicinal
Products, commented: “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.”
Under previous guidelines, the FDA required orphan product sponsors to submit an annual report within 14 months of submission and annually thereafter, whereas the EMA requested annual updates from designation onwards.