Today’s guest blog was written by Peter L. Saltonstall, who is the President and CEO of the National Organization for Rare Disorders (NORD). NORD is a nonprofit organization representing the 30 million Americans who have rare diseases and those seeking to improve their lives. NORD provides advocacy, educational programs, research and patient assistance programs for the rare disease community.
On the last day of February each year, millions of rare disease patients and those seeking to help them, join in a worldwide observance of Rare Disease Day. The purpose is to focus attention on rare diseases as a public health concern.
The National Organization for Rare Disorders (NORD) sponsors Rare Disease Day in the U.S., working with our partner in Europe, EURORDIS, and others around the world.
Since it was established in 2008, Rare Disease Day has been growing each year, but this year has special significance in the U.S. That’s because 2013 is the 30th anniversary of the Orphan Drug Act and of NORD. It’s an important time to reflect upon progress to date, future challenges and how to accelerate addressing the needs of the 30 million Americans with rare diseases.
At NORD, we feel that the rare disease community is on the cusp of a promising new era. 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.
On Rare Disease Day, patients and their supporters post stories, videos and photos online to raise awareness of the challenges of living with rare diseases. These include diagnosis delay, too little research, too few treatments and limited patient access to treatments.
Events are planned across the U.S. at schools, hospitals, universities and community centers. NIH is hosting a conference for patients and researchers. NORD and its partners are hosting “State House Events”, since many of the issues such as newborn screening have relevance at the state level.