Single submission of orphan drug designation annual report for both FDA and EMA

Voluntary move can save time, simplify development process

In recognition of World Rare Disease Day, the U.S. Food and Drug Administration and the European Medicines Agency (EMA) today announced 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. 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 United States and the European Union.

Currently, if an orphan product was granted designation on the exact same day in both the United States and European Union, sponsors must 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 United States and the European Union for orphan designated products.

"This process provides benefits for both agencies," said Timothy Cote, M.D., M.P.H., 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, much like separate 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 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 their 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. Starting Feb. 28, 2010 – World Rare Disease Day – sponsors may send the single Orphan Drug Designation Annual Report tomailto: both agencies. If they choose to do so, a sponsor may submit the report on their normal annual reporting date.

"We are very pleased with this collaboration on regulatory requirements and about sharing data that will help us understand the viability of the products," said Jordi Llinares, M.D., M.Sc., head of Orphan Medicines at the EMA.

Rare diseases are often chronic, progressive, degenerative, and often life-threatening. A disease or disorder is defined as rare in Europe when it affects less than 1 in 2,000 people (<250,000 people presently).  A disease or disorder is defined as rare in the United States if it affects fewer than 200,000 Americans at any given time. It is estimated that there are 6,000 to 8,000 rare diseases in the world today.

"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," added Kerstin Westermark, chair of the EMA's Committee for Orphan Medicinal Products.

For more information

Developing Products for Rare Diseases & Conditions

http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/default.htm

FDA International Programs – Europe

http://www.fda.gov/InternationalPrograms/FDABeyondOurBordersForeignOffices/EuropeanUnion/default.htm

World Rare Disease Day 2010

SOURCE U.S. Food and Drug Administration