A rare disease is just that — specifically, a malady that afflicts fewer than 200,000 people in the United States. But while many individual diseases may be rare, many individuals have rare diseases. Hence the slogan of The National Organization for Rare Disorders: “Alone we are rare. Together we are strong.”
It’s also the strategy employed by several healthcare organizations, led by BioNJ and the HealthCare Institute of New Jersey (HINJ), in organizing a local event around the global Rare Disease Day 2016.
About two dozen participants gathered at NJBIA’s headquarters in Trenton for an hour-long presentation followed by a group trip to the Capitol to meet with Senators who were in town for committee meetings. Members of rare-disease organizations and several individuals with family members who have such rare conditions spent the afternoon educating lawmakers about the importance of and challenges to making drugs and treatments available.
Patients with rare diseases have few or no drugs to treat their conditions, according to BioNJ President Debbie Hart, a circumstance that Rare Disease Day supporters are committed to changing.
“We pledge to work with you to further our collective vision for a New Jersey where science is supported, companies are created, jobs are developed, and patients are paramount,” Hart said.
HINJ President Dean Paranicas emphasized the importance of a collaborative effort.
“Even though any one of you may represent a fairly small community, think of the power, from an advocacy perspective, you will represent when you can say one in nine New Jerseyans has one kind of rare disease or another,” Paranicus said.
BIO-NJ and HINJ represent companies that work to develop the drugs and therapies that are critically important to the treatment of rare diseases. Rare disease day is about making policy-makers aware of the challenges of developing such treatments.Related Articles: