Rare Disease Day 2011

Millions Around World To Observe Rare Disease Day

The last day of February has been designated as World Rare Disease Day to call attention to rare diseases, which affect nearly 30 million Americans and countless others around the world, as an important global public health concern.

“People with rare diseases remain a medically underserved population in every country,” said Peter L. Saltonstall, president and CEO of the National Organization for Rare Disorders (NORD), which is sponsoring Rare Disease Day in the U.S. “This day will bring together patients and families around the globe who are dealing with some very challenging issues.”

The coalition, being coordinated by NORD, includes patient organizations, professional medical societies, government agencies, medical researchers, and pharmaceutical and biotechnology companies.

Rare Disease Day 2011 activities in the U.S. will include creating a “video encyclopedia” of two-minute videos about many of the nearly 7,000 rare diseases. Also, patients and others will help NORD create a database of physician experts on various rare diseases. In addition, state and municipal proclamations in honor of the day will be sought, and there will be a drive to enlist support for a new Rare and Neglected Diseases Caucus in the U.S. Congress.

A rare disease is one that affects fewer than 200,000 Americans. According to the National Institutes of Health (NIH), there are nearly 7,000 such diseases affecting nearly 30 million Americans.

“More than half of the people who have rare diseases are children,” Saltonstall said. “Challenges faced by patients and their families include delay in getting an accurate 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 350 “orphan” (for rare diseases) drugs and biologics have been approved by the U.S. Food and Drug Administration (FDA). FDA estimates that from 11 to 14 million Americans benefit from these products, but that still leaves more than 15 million Americans with diseases for which there is no approved treatment.

This will be the fourth annual Rare Disease Day. The concept was launched in 2008 by EURORDIS, NORD’s counterpart in Europe. Last year, EURORDIS asked NORD to host Rare Disease Day in the U.S. In 2010, Rare Disease Day was observed in 46 nations around the world. The observance is always on the last day of February.

For more information about Rare Disease Day go to www.rarediseaseday.org