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Agencies Streamline Orphan Drug Submission Process

April 1, 2010

The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have announced that both agencies have agreed to accept the submission of a single annual report from sponsors of orphan drugs and biologics. Orphan drugs are developed for patients with rare diseases that are difficult to treat and life-threatening. The FDA announcement was made in anticipation of World Rare Disease Day, an annual awareness event, held this year on Sunday, February 28.

The FDA’s Office of Orphan Products Development grants orphan drug status, which means the drugs go through an expedited review process. The designation allows for accelerated approval, so that much-needed drugs or biologics become available to patients with rare diseases. By European standards a disease/disorder is defined as rare when it affects less than 1 in 2,000 people (<250,000 people presently). In the U.S. a disease/disorder is considered rare if it affects less than 200,000 Americans “at any given time.” There are approximately 6,000 to 8,000 rare diseases globally.

Previously, sponsors of products under consideration for orphan drug status in the U.S. and Europe submitted separate reports to the respective regulatory agencies, the FDA and EMA. The new arrangement allows sponsors to submit one report. Each agency will conduct its own review on the report to assure the information meets all of its legal and scientific requirements. The agencies will then exchange reports via a secure portal.

“This process provides benefits for both agencies,” said Timothy Coté, MD, MPH, 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.

“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 in identifying and acting on bottlenecks,” added Kerstin Westermark, chair of the EMA’s Committee for Orphan Medicinal Products.

The single annual report submission process is voluntary and will apply only to sponsors that have obtained an orphan designation status for their product from both the FDA and EMA.

Source: FDA news release dated February 26, 2010