Congressional Rare Disease Caucus Chairs Mark National Rare Disease Day

Feb 29, 2016 Issues: Health Care

Crowley, Lance Urge Colleagues to Add Their Voice and Join Caucus

(Washington, D.C.) – Today, Rep. Joe Crowley (D-Queens, the Bronx), Vice Chair of the Democratic Caucus, and Rep. Leonard Lance (R-NJ07), co-chairs of the Congressional Rare Disease Caucus, issued the following statement in recognition of National Rare Disease Day, which is held on the last day of February each year. This year’s theme is “Patient Voice”, which recognizes the important role patients play in voicing their needs and inspiring change. 

“Rare Disease Day provides an opportunity to draw attention to the 30 million Americans who are afflicted by rare or orphan diseases.  With one out of every 10 Americans suffering from a rare disease, it is absolutely critical that we do all we can to identify research opportunities, therapy options, and treatment.

“Despite the numbers, those living with rare diseases face an uphill climb in getting treatments. Much progress has been made in improving access to research funding and opportunities, but there is more that must be done to help those struggling with a rare disease -- approximately 50 percent of whom are children -- obtain needed therapies.

“Recognizing this public health issue, the Rare Disease Caucus will continue its efforts to build awareness and identify solutions to help the millions of Americans living with a rare disease. In the spirit of this year’s theme, we urge our colleagues to join our caucus and make the voices of rare diseases heard!”

Established in 2010, the Rare Disease Caucus is over 100-members-strong. The Caucus is focused on bringing Congressional attention to the nearly 7,000 known rare diseases; highlighting the need to ensure sufficient funding for research and orphan product development‎; exploring ways to incentivize companies to create new drugs, biologics and humanitarian use devices; and providing an opportunity for Members of Congress, families, and advocacy groups to exchange ideas and policy concerns. Rare and neglected diseases afflict nearly 30 million Americans, approximately half of whom are children. Only 289 of the 7,000 rare diseases, or less than 5%, have Federal Drug Administration (FDA) approved treatments.

Each year Rare Disease Day is observed on the last day of February. The goal is to draw attention to rare diseases as an important public health issue. Launched in Europe in 2008, the first Rare Disease Day was first observed in the United States in 2009. Organizations in over 80 countries and regions are participating in Rare Disease Day 2016, and 32 states in the U.S. are holding events to mark the day.