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Rare Diseases: US Pediatric Biomedical Research Legislation

November 18, 2013

United States bipartisan legislation, known as the National Pediatric Research Network Act (NPRNA) of 2013, H.R. 225, passes the Senate unanimously (11/14) as part of a three-bill health package (S. 252 – PREEMIE Reauthorization Act), 2 days after passing the House of Representatives (11/12). NPRNA is now awaiting President Obama’s signature for it to become law. The legislation is introduced into Congress by Representative Lois Capps (California) and Senator Sherrod Brown (Ohio). The purpose of the bill is to accelerate research to develop new treatments and therapies for pediatric diseases, with a special emphasis on rare and genetic diseases.

NPRNA allows the National Institutes of Health (NIH) to create up to 20 National Pediatric Research Consortia throughout the United States. Each consortium will operate in a “hub and spoke” structure – one central academic hub coordinating research and clinical work of satellite sites. The intent of this type of structure is to encourage collaboration and exchanging of information between researchers, clinicians, and academic institutions with the goal of success.  Other features of NPRNA, according to Senator Brown’s summary of the legislation :

•   “5-year grants will be made, with potential 5-year renewals based on evaluations by a peer review panel and Director

•   Consortia will be selected through a competitive peer-review process, and private or public nonprofit agencies or institution will all be eligible to apply

•   Some consortium locations will focus primarily on rare pediatric diseases, ensuring much needed attention to rare pediatric diseases.”

By using the network and partnership concept, the new legislation will maximize pediatric research resources, encourage data-sharing, and provide the capability to coordinate multi-site clinical trials among participating educational and research institutions.

FDASIA (Food and Drug Administration Safety and Innovation Act) is signed into law by President Obama in July 2012. According to Section 510 (“Pediatric Rare Diseases”) of FDASIA, the following requirements must be met :

•    Public meeting to be held no later than 18 months after the enactment of this law, to “discuss ways to encourage and accelerate the development of new therapies for pediatric rare diseases” (January 2014)

•   No later than 180 days after the date of the public meeting, a Report is to be issued that “includes a strategic plan for encouraging and accelerating the development of new therapies for treating pediatric rare diseases” (July 2014).

To satisfy FDASIA Section 510’s first requirement above, a 2-day FDA sponsored public workshop on “Complex Issues in Rare Disease Drug Development & Accelerating Development of New Therapies for Pediatric Rare Diseases” is scheduled for January 6 – 7, 2013.  The second day of the public workshop will be devoted to the topic of accelerating the development of new therapies for pediatric rare diseases.


Rare Diseases: Pediatric Priority Review Voucher Incentive Program” Post

Rare Diseases: Comparison of Pediatric Policies in US & EU” Post.

Please Note: “Newborn Screening” courtesy of the National Human Genome Research Institute (NHGRI)/National Institutes of Health (NIH) – Public Domain.

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

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