Navigation Links
International Collaboration: FDA and European Medicines Agency Agree to Accept a Single Orphan Drug Designation Annual Report
Date:2/26/2010

Voluntary move can save time, simplify development process

WHITE OAK, Md., Feb. 26 /PRNewswire-USNewswire/ -- 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.

(Logo:  http://www.newscom.com/cgi-bin/prnh/20090824/FDALOGO )

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

http://www.rarediseaseday.org/

U.S. inquiries: Jeff.Fritsch@fda.hhs.gov

EMA inquiries: orphandrugs@ema.europa.eu

U.S. Media Inquiries: Rita Chappelle, 301-796-4672, rita.chappelle@fda.hhs.gov

Trade Press Inquiries: Crystal Rice, 301-796-3111, crystal.rice@fda.hhs.gov

U.S. Consumer Inquiries:  1-888-INFO-FDA

EU Media Inquiries: Martin Havey Allchurch or Monika Benstetter, 011-44-(0)20-7418-8427, press@ema.europa.eu

SOURCE U.S. Food and Drug Administration

Back to top

RELATED LINKS
http://www.fda.gov

'/>"/>

SOURCE U.S. Food and Drug Administration
Copyright©2010 PR Newswire.
All rights reserved


Related medicine news :

1. International study strengthens case for daily calcium pill
2. Mettler-Toledo International Inc. Announces Webcast of Presentation at Thomas Weisel Partners 2007 Healthcare Conference
3. Australian-led international study shows blood pressure drugs cut death rate in type 2 diabetes
4. LCA Hails International Investigators Studying Lung Cancer
5. Arrow International Urges Shareholders To Vote for Approval of the $45.50 Teleflex Inc. Merger and for the Current Board of Directors
6. PRA International Adds Dr. Susan Stansfield as Executive Vice President
7. Medical Services International Maintains Pace in Second Quarter
8. Clarity Imaging International, Inc. Rolls Out Mobile DEXA Program
9. International Gateway Insurance Brokers Partners With MEDILINQ
10. Tulane University to receive $14M for international HIV/AIDS program
11. International Database Seeks to Boost Treatment of Altitude Sickness
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:1/17/2017)... ... January 17, 2017 , ... ... assistance to families and business professionals throughout central Colorado, is joining local nonprofit ... , The Aurora Warms The Night organization is committed to breaking the cycle ...
(Date:1/17/2017)... ... January 17, 2017 , ... Physicians Education ... Lee as keynote speaker for the 34th Annual Miami Breast Cancer Conference®, on ... announcement, PER® president, Phil Talamo, said, “We are delighted to have Sandra Lee ...
(Date:1/17/2017)... ... , ... Today, FloSports , a global leader in live digital sports ... of a long-term extension of their media partnership. The partnership, which began in 2013, ... sport’s premier events exclusively on FloWrestling.com as well as usage of the FloArena meet ...
(Date:1/16/2017)... ... January 16, 2017 , ... California Southern University has named Dr. Steven ... Board of Trustees and as a core faculty member, teaching master’s and doctoral courses ... of psychology at Pepperdine University, where he earned his Doctor of Psychology degree in ...
(Date:1/16/2017)... ... ... Anybody who may be looking for a substance abuse rehabilitation program with ... a holistic treatment center for addiction located in Marne, MI. This video, which can ... and staff that visited the 2016 Recovery Palooza support event in nearby Grand Rapids. ...
Breaking Medicine News(10 mins):
(Date:1/17/2017)... -- Interpace Diagnostics Group, Inc., (NASDAQ: IDXG ), ... and pathology services for evaluating risks of cancer, announced ... 13, 2017 from the NASDAQ Listing Qualifications notifying the ... of the NASDAQ Stock Market to maintain a minimum ... The letter noted that for the last 10 days ...
(Date:1/17/2017)... Research and Markets has announced the addition ... their offering. ... The global laparotomy sponges market to grow at a CAGR of ... Sponges Market 2016-2020, has been prepared based on an in-depth market ... landscape and its growth prospects over the coming years. The report ...
(Date:1/17/2017)... , Jan. 17, 2017 Tempus, ... cancer care, and University Hospitals Seidman Cancer Center ... by making genomic data, advanced molecular analysis and ... researchers, physicians and healthcare providers. ... provide genomic and transcriptomic sequencing and analysis for ...
Breaking Medicine Technology: