Rare Disease Day 2015

In honor of Rare Disease Day, World Orphan Drug Congress USA is giving back

In Advocacy by Freya Smale

Rare Disease Day 2015We are constantly inspired by the work of the rare disease advocacy groups. Their tireless work to fight for Rare Disease recognition has laid the foundation for the Orphan Drug industry to thrive. In honor of Rare Disease Day and the work of all rare disease advocates, the World Orphan Drug Congress USA is giving back. For the last 4 months, we’ve been raising money for Kids V Cancer.

Kids V Cancer promotes pediatric cancer research by identifying structural impediments at key junctures in the research process – new drugs, tissue donation and access to funding – and developing strategies to address them.

Since Kids v Cancer was founded in 2009:

  • They have started an autopsy tissue donation program for pediatric brain cancers which has already had significant impact on scientific discoveries.
  • They have raised the profile of pediatric cancer issues by having September declared to be National Childhood Cancer Awareness Month for each of the past three years.
  • They have worked with the FDA to pursue a new interpretation of the Best Pharmaceuticals for Children’s Act, a framework law governing pediatric cancer research, which will allow the FDA to accelerate the development of drugs potentially useful in treating pediatric cancer.
  • They are working to identify areas of potential convergence between FDA and foreign regulatory practices that can help strengthen pediatric drug development.
  • And most significantly, they were successful in authoring and championing for the Creating Hope Act, Section 908 of the FDA Safety and Innovation Act, which created a market-based incentive to spur pediatric cancer drug development. The Creating Hope Act when into effect on October 1st, and we are now working with the FDA, researchers and drug companies to ensure its full and effective implementation

This Friday, February 27th, we will be presenting Kids V Cancer with the money we have raised. Keep an eye on Total Orphan Drugs to find out exactly how much we’ve raised!

If you have any Rare Disease Day stories to share, then let us know. Send your stories to freya.smale@terrapinn.com and get them posted on Total Orphan Drugs!

Rare Disease Day