How Does Rare Disease Research Actually Get Funded?

According to a story from Inside Philanthropy, a recent case study was conducted in order to determine the main sources of funding for rare disease research. This broad ranging study looked at fundraising for rare diseases as a whole, using the definition widely accepted in the US as any disease that affects less than 200,000 people in the country.

Big Money is Scarce

The research serves to highlight some realities that many people in the rare disease world already know: that large sources of funding are scarce. Many big players in the drug development industry do not see rare diseases as a worthwhile avenue of research or development because of the inherently limited pool of potential customers. In addition, funding from the government, while present, is also limited in comparison to many other health or disease related initiatives. 

Small Donors, Big Impact

With these factors in mind, it may come as little surprise that the main source of funding for rare disease comes from private donations. However, donations are usually small and often come directly from people that are affected by rare diseases as well as their friends and families. Big wig donors and their foundations are less inclined to donate to rare causes. After all, they may prefer to donate to a cause that is more likely to grab headlines.

Despite the fact that most donations are small, all of these can add up to serious amounts of cash that have been essential rare disease research and drug development that probably would never have gotten off of the ground otherwise. Such research has led to essential advancements in treatment.

The Cystinosis Research Foundation

An example of an effective rare disease fundraiser is the Cystinosis Research Foundation. Cystinosis is an incredibly rare illness that only impacts about 500 people in the US, but the organization has nevertheless managed to find great success in its fundraising. The foundation has funded grants for cystinosis research and drug development all over the world, and has given over $2 million in grant funding this last year alone. The group has mastered appealing to potential donors and has helped fund every recent breakthrough in cystinosis treatment in the last few years.

This research serves to remind the rare disease community that while there are few people who can donate large chunks of money, a lot of donations from passionate people can make a serious impact.


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