15 Years Strong: Rare Disease Week’s Remarkable Journey of Support

In 2009—15 years ago— the National Organization for Rare Disorders (NORD) announced the first U.S. recognition of Rare Disease Day.  NORD’s announcement followed in the footsteps of European rare disease patient organization, Eurodis, who had celebrated the first Rare Disease Day the year before.  Time flies when your goal is to support the thirty million Americans with rare diseases.  The global rare disease community did not just celebrate Rare Disease Day, but Rare Disease Week, the entire last week of February!

In 2009 I served as Chair of NORD and through the hard work of countless colleagues we inaugurated the first Rare Disease Day.  The idea that year was simple and straightforward—every four years there is a rare day, February 29, so what better day to choose to honor those with rare conditions.  In non-leap years we celebrate on February 28 or March 1.

What started as a day has—due to its popularity and the persistent efforts of patients and industry—evolved into a week-long event.  I was honored to be invited to speak at the plenary session of FDA’s inaugural Rare Disease Day gathering several years ago.  And my support and pride continue since we now see a host of programs and events the last week of February playing out around the world.

This year the Everylife Foundation for Rare Diseases held a week of activities in which more than 800 rare disease patients—from all 50 states, DC, and the Cherokee Nation—advocated in Washington DC.  I currently serve as Vice Chair of the Board of the Everylife Foundation.  I’m proud to share with you that Everylife’s “patient army” held more than 330 meetings with members of Congress last week.  Led by Annie Kennedy, Everylife’s VP of Government Affairs and Policy, we worked the halls of Congress to educate lawmakers on the pressing needs of the rare disease community.

On February 28th, the White House hosted its first ever rare disease event.  It’s astounding that it took this long for any administration—Republican or Democrat—to recognize the thirty million strong rare disease community in our country.  Annie Kennedy chaired a panel discussion and the NIH Director and the NCATS Director spoke passionately of the ways in which the federal government is supporting and planning to expand its work on rare diseases.

Among the forty or so individuals invited to this White House event were long-time rare disease advocates like recently retired FDA Principal Deputy Commissioner Janet Woodcock, Christina Hochul of Alexion and Paul Melmeyer of Muscular Dystrophy Association.  It was a highlight of my career to also be invited to this historic event.

Given the energy and progress accomplished each year at this special time, anyone want to second a motion that the entire month of February become dedicated to the support of rare diseases?  Onward!

Photo from February 28, 2024, White House Rare Disease event (from left to right: Frank Sasinowski, Christina Hochul, Paul Melmeyer)