As Rare Disease Day Approaches, Patient Advocates Celebrate 30 Years of Collaboration But Worry About Looming Budget Cuts

Feb 26, 2013, 13:16 ET from National Organization for Rare Disorders (NORD)

On 30th Anniversary of Orphan Drug Act and NORD, Rare Disease Patient Advocates Fear That Budget Cuts for NIH and FDA Will Slow Momentum

WASHINGTON, Feb. 26, 2013 /PRNewswire-USNewswire/ -- On Thursday (Feb. 28), millions of people around the world will be observing Rare Disease Day and this annual observance has special significance for Americans this year for two reasons.

The first is that 2013 is the 30th anniversary of the Orphan Drug Act – landmark legislation made possible, in part, by a coalition of patient advocates who also established the National Organization for Rare Disorders (NORD) at the same time.   

The second reason is that across-the-board budget cuts for federal agencies including the National Institutes of Health (NIH) and Food and Drug Administration (FDA) are scheduled to take place on March 1, with the potential for major impact on rare disease research and the development of treatments.

"NORD feels that the rare disease community is on the cusp of a major new era. But we're worried that the looming budget cuts may derail that process," says Peter L. Saltonstall, NORD's president and CEO.

"Enormous progress has been made in recent years on both the research and the regulatory fronts," he adds.  "But budget cuts at NIH and FDA could have a very harmful effect on the current momentum."

In particular, NORD is concerned that initiatives such as "translational research" programs at NIH to bridge the gap between promising early research and clinical trials, as well as implementation of the FDA Safety and Innovation Act (FDA SIA) of 2012, could be delayed.

"FDA SIA includes many provisions of great importance to the rare disease community," Saltonstall says. "It would be a serious loss for FDA not to be able to implement it as planned."

NORD sees Rare Disease Day as an important reminder of the need to address the challenges people living with rare diseases endure. These include diagnosis delay, too little research, too few treatments, and limited access to treatments.

Many people with rare diseases go for years without getting an accurate diagnosis. Only a few hundred of the nearly 7,000 diseases considered rare have FDA-approved treatments. And people affected by rare diseases often feel isolated and abandoned. 

About two-thirds of the 30 million Americans with rare diseases are children, and the diseases tend to be serious and lifelong. In the U.S., any disease affecting fewer than 200,000 Americans is considered rare. 

Rare Disease Day is observed around the world on the last day of February each year. As the national sponsor in the U.S., NORD works with organizations in other countries to plan activities each year. To view activities in the U.S. this year, go to www.RareDiseaseDay.US. To see what is planned globally, go to

Visit NORD's interactive timeline for the 30th anniversary of NORD and the Orphan Drug Act. 

SOURCE National Organization for Rare Disorders (NORD)