Today is the day when people around the world will be posting stories, photos and videos about what it means to live with a rare disease. From Kalamazoo to Kiev, the last day of February is observed as Rare Disease Day.
The buzz about this has been building for the past several weeks with a rising crescendo of blogs, news stories and social media posts. And, while every Rare Disease Day is important, this one has special significance in the U.S.
That’s because this year is the 30th anniversary of NORD and the Orphan Drug Act. It’s the perfect time to take stock of progress made since this game-changing legislation was enacted in 1983 … and to assess how best to accelerate the development of treatments and assure patient access to them.
The science of rare diseases is advancing faster than ever before. And the FDA Safety and Innovation Act (FDASIA), enacted with bipartisan support last summer, is potentially the most important legislation for the rare disease community since the Orphan Drug Act.
The negotiations leading up to FDASIA, in which NORD played an integral role, resulted in major expansion of the Rare Disease Program at FDA. Also, several important pieces of legislation were rolled into it, including the FAST, TREAT, Breakthrough Therapies, Creating Hope and EXPERRT acts.
If sequestration and related budget problems don’t derail the process, 2013 promises to be the launch of a new era for rare disease research and regulatory science advances.
Rare Disease Day is for Everyone
Established in Europe by EURORDIS in 2008 and brought to the U.S. the following year by NORD, Rare Disease Day is for everyone. While there are national events such as the NIH conference for patients and researchers, most Rare Disease Day activity takes place at the grassroots level in local schools, hospitals and community centers. And the purpose is always greater awareness of the needs and challenges, and increased commitment to collaborative action.
How Can You Get Involved?
About 2/3 of rare disease patients are children. For that reason, many Rare Disease Day activities take place in schools and children’s hospitals. NORD provides resources for teachers on topics ranging from bullying to careers. You can see if anything is planned in your area on the map on the Rare Disease Day US website.
Events are also planned in five State Houses this year (California, Connecticut, Massachusetts, New Jersey and Texas). Important decisions related to issues such as newborn screening and health insurance are made at the state level, so communication with state legislators is very important.
For a 24-hour period on Feb. 28, anyone wishing to voice support for rare disease programs at the National Institutes of Health and Food and Drug Administration may send a special Rare Disease Day message to President Obama, Members of Congress and other elected officials through the “Legislative Action Center” on NORD’s website.
And … perhaps easiest of all … is simply to watch for social media posts on Rare Disease Day and throughout the year with heightened awareness that we all know someone with a rare disease and we all can work together to ease the isolation and address the issues.
RareDisease Dialog is the official blog for the National Organization for Rare Disorders (NORD). NORD’s staff and friends will share information of interest to the entire rare disease community.
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