On this day, millions of patients and their families will share their stories to focus a spotlight on rare diseases as an important global public health concern.
“There are nearly 30 million Americans—and millions more around the world—affected by rare diseases,” said Peter L. Saltonstall, president and CEO of NORD. “Everyone knows someone with a rare disease. But, while many of these diseases are serious and lifelong, most have no treatment and many are not even being studied by researchers. This leaves patients and families without hope for a better future.”
On Rare Disease Day, people with rare diseases around the world promote awareness of the challenges of living with a rare disease. The global theme for 2013 is “Rare Disorders Without Borders.”
World Rare Disease Day was launched in Europe in 2008 and last year was observed in more than 60 nations. It is always observed on the last day of February. On that day, patients and patient organizations will post stories, videos and blogs online and host events to raise awareness of these diseases, which are often called “orphans”.
This year, the observance has special significance in the U.S. because 1983 is also the 30th anniversary of the Orphan Drug Act, which provides incentives to encourage companies to develop treatments for rare diseases, and of NORD, which was established by patient advocates in 1983.
“More than half of the people who have rare diseases are children,” Saltonstall said. “Challenges faced by patients and their families include delayed diagnosis, few treatment options, and difficulty finding medical experts. Many rare diseases have no approved treatment. Insurance may not cover treatments that aren’t approved. Also, treatments for rare diseases tend to be more expensive than those for common diseases.”
In 1983, the Orphan Drug Act was passed by Congress to create financial incentives for companies to develop treatments for rare diseases. Since then, more than 400 orphan drugs and biologics have been approved by the Food and Drug Administration (FDA). It is estimated that approximately 15 million Americans benefit from these products, but that still leaves millions more with diseases for which there is no approved treatment.
For more information about Rare Disease Day activities in the U.S., go to www.rarediseaseday.us. For information about global activities, go to www.rarediseaseday.org).
*******
In conjunction with Rare Disease Day, Pandora Poikilos' books will be free from 28th February 2013 till 15th March 2013. To download your free copy, please click on the links below.
Excuse Me, My Brains Have Stepped Out is free via KDP Select (Kindle) from now till 3rd March.
Dora's Essentials - Examining Anxiety is free via Smashwords from now till 15th March.
Genetically Modified Foods vs. Sustainability is free via Smashwords from now till 15th March.
Pandora's Reading Room (Short Story compilation) is free via Smashwords from now till 15th March.
