By Elie Dolgin
NORTH BETHESDA, MARYLAND—Rare diseases, defined in the US as those occurring in fewer than 200,000 people in the country, collectively affect around 10% of individuals worldwide. Yet the majority of the public can hardly name a single rare disease. As a result, most orphan disorders fall under the radar and remain poorly funded.
Patient advocacy groups are one of the primary backers of research into rare diseases. But the hundreds of diseasespecific foundations and organizations out there rarely work together to raise funds, and the rare disease landscape has remained fractured and siloed. To remedy the situation, the R.A.R.E. Project, an initiative launched in 2008 to raise awareness and accelerate the development of therapies for rare diseases, is rolling out a new platform to serve as a one-stop shop for innovative research into all 6,000-plus rare diseases.
“We’re trying to bring new people in to care about rare disease,” says Nicole Boice, founder and president of the Children’s Rare Disease Network, part of the R.A.R.E. Project. “The idea in fact is that we will stimulate foundations to think differently about funding and research,” adds R.A.R.E. Project CEO Jonathan Jacoby.
Modeled after services such as Kiva and Save the Children, where donors can precisely match their contributions to the specific project of their choice, R.A.R.E. is launching a website, called the Global Genes Fund, intended as a clearinghouse for rare disease philanthropy, where people can select projects to fund. Jacoby hopes that by bringing hundreds of research projects under one umbrella, individuals, foundations and corporations will be more likely to donate to multiple causes.
Last month, R.A.R.E. secured $50,000 for a beta version of the site, which the organization plans to make public later this year, Boice and Jacoby announced here at the Genetic Alliance annual conference on 16 July.
For projects listed on the page—which will be vetted through some as yet undefined criteria—supporters will be able to read an affected child’s personal story, the details of the study and why the research is important, among other details.
“The challenge with rare diseases is that they’re rare, and there aren’t that many families that can raise money,” says Geraldine Bliss, research chair of the Phelan-McDermid Syndrome Foundation. “A concept like [the Global Genes Fund] is really great because it allows you to reach beyond your immediate circle of support.”
“The rare disease community is large enough and deserving enough to have an effort like this and to succeed at it,” Boice says. “It’s time, it’s really time.”