Rare Disease Patients Celebrate 20 Years of Progress; In 20 Years, 241 Orphan Drugs Have Been Approved by the FDA

5/19/2003

From: Mary Dunkle, 203-744-0100 Diane Dorman, 202-258-6457 both of the National Organization for Rare Disorders

WASHINGTON, May 19 -- Two decades of progress in the search for treatments for rare diseases were highlighted in a gala celebration of the 20th anniversary of the Orphan Drug Act and the 20th birthday of the National Organization for Rare Disorders (NORD).

"In 20 years, 241 orphan drugs have been approved by the U.S. Food and Drug Administration for marketing," Abbey S. Meyers, NORD's president, said proudly, "and more than 1,100 new drugs and biologics have entered the research pipeline. This represents tremendous progress. An average of over 10 new treatments for rare diseases are being approved each year, compared to just one per year in the decade before the Orphan Drug Act was passed by Congress."

"The Orphan Drug Act has unarguably improved, and in many cases, saved the lives of millions of individuals with rare diseases, for whom drugs and drug breakthroughs were the only hope," said Susan Dentzer, Health Correspondent for the NewsHour with Jim Lehrer and emcee for the evening. Dentzer gave credit to Meyers for leading the patient movement that pushed the Act through Congress, and praised NORD for its continuing advocacy for rare-disease patients.

To celebrate the 20th anniversary, NORD paid special tribute to the government institutions that have made this dramatic progress possible: the United States Congress, the Food and Drug Administration and the National Institutes of Health.

In addition, NORD presented special anniversary awards to 20 pharmaceutical manufacturers for leadership in conducting research on new therapies for rare diseases and bringing new treatments to market.

NORD was founded in 1983 by members of patient organizations who had worked together to get the Orphan Drug Act passed. That legislation provides financial incentives to companies that develop new treatments for rare diseases, called "orphan" drugs. Prior to the Act, few pharmaceutical companies were willing to invest in research on treatments for diseases where the markets were too small to justify the expense.

NORD, which was named one of the 100 best charities in America by Worth magazine in 2002 and 2003, provides information to patients and medical professionals, administers programs to help patients obtain medications they couldn't otherwise afford, oversees research grants and fellowships, and conducts advocacy to promote increase support for health programs. Its Web site (http://www.rarediseases.org) receives approximately 100,000 visits a month from around the world. Its new book for physicians (The NORD Guide to Rare Disorders) was reviewed in the May 10 issue of the medical journal, The Lancet.

For a complete list of awardees go to NORD's Web site, http://www.rarediseases.org.