Single orphan document will simplify process for industry
pharmafile | March 1, 2010 | News story | Research and Development | EMA, FDA, Orphan
The FDA and European Medicines Agency are to simplify the regulatory process for orphan medicines by agreeing to accept a single document.
Pharma companies have had to produce separate documents for the two regulators to meet their specific requirements, but now the agencies have agreed on a common document which they will both accept.
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 a product’s designation as an orphan product.
The single annual report submission 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 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.
The new system came into operation on February 28 to coincide with World Rare Disease Day, [http://www.rarediseaseday.org/] but sponsors do not have to alter their existing 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, head of Orphan Medicines at the EMA.
Rare diseases are 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.
• Orphan medicines information from the EMA can be found here
Related Content
GSK’s Jemperli accepted for FDA review for endometrial cancer treatment
GSK has announced that the US Food and Drug Administration (FDA) has accepted its supplemental …
FDA approves ImmunityBio’s Anktiva bladder cancer treatment
ImmunityBio has announced that the US Food and Drug Administration (FDA) has approved Anktiva (N-803, …
Roche’s Alecensa approved by FDA as lung cancer treatment
Roche has announced that the US Food and Drug Administration (FDA) has approved Alecensa (alectinib) …
