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Rare Diseases: Comparison Of Pediatric Policies In US And EU

August 18, 2013

Special Note: This is the first of a series of Blog Posts on rare diseases and pediatrics.

Rare Diseases is an online, peer-reviewed, open access journal from Landes Bioscience, that publishes research addressing the underlying mechanisms of rare diseases. The goals of the journal is to “publish original research articles and to raise awareness about rare diseases.” Special issues in the future are planned to be dedicated to a particular rare disease or a related group of diseases. The first issue of this journal is available online.

An article in Rare Diseases compares the present policies and future opportunities for pediatric rare diseases in the United States (US) and the European Union (EU). The following is the citation for the article along with the link for the free PDF download of the article :

Bavisetty S, Grody W, Yazdani S. Emergence of pediatric rare diseases: Review of present policies and opportunities for improvement. Rare Diseases 2013; 1:e23579.

The article reviews and discusses steps taken by both the US and the EU to meet the needs of pediatric rare diseases, and also identifies future steps for further improvement. Since approximately 50-75% of rare diseases begin in childhood, it is important to take a look at the present policies specific to pediatric rare diseases. An important fact to keep in mind is the difference in the definition of a rare disease :

•    US: affects < 200,000    people
•    EU: affects < 1 in 2,000 people.

The authors review 23 reports and 9 legislative documents related to pediatric rare diseases and public policy. The analysis of existing policies and surveys indicate differences between the two regions :

•    US: policies aim at disease diagnosis and neonatal screening
•    EU: policies focus on access to existing specialized care.

The article says US policies focus on the diagnosis of rare diseases by concentrating on research, improving access to credible laboratories, new-born screening, and facilitating a coordinated research effort. Whereas in the EU, policy makers concentrate on providing access to facilities and qualified centers that provide specialized care to the pediatric rare disease patient. The article concludes that both approaches to pediatric rare diseases are not perfect and identifies future improvements.

The FDA Safety & Innovation Act (S. 3187), or FDASIA, is signed by President Obama in July 2012. FDASIA’s Section 908, “Rare Pediatric Disease Priority Review Voucher Incentive Program”, extends the existing Priority Review Voucher Program to rare pediatric diseases.

A Blog Post is scheduled for Tuesday, August 20, 2013, on the topic of the new FDASIA rare disease pediatric priority review voucher incentive program.

Please Note: “Stick Figure Standing On Grass” by Weedwacker (Own work) [CC-BY-SA-3.0] | Wikimedia Commons

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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