Working in rare diseases demands a constant push-pull. We must rally against limitations in the medical field, and ask that scientists push to be creative and relentless in their pursuit of treatments and cures. Advocates must push lawmakers to recognize the importance of prioritizing the needs and rights of affected individuals. Patient organizations, like Cure CMD, must pull together to create avenues for children and adults to build community and speak their voices.
And still, each aspect of working to prevent, combat, treat, and/or cure diseases such as congenital muscular dystrophy (for which Cure CMD works) has one thing in common during this push-pull:
Every bit of work we do requires funds. Research, space and equipment to conduct that research, staff salaries (when staff is paid; often, patient advocacy organizations like Cure CMD are part or all volunteer-run), and community and/or scientific events all require funds in order to operate. It’s not that these organizations are making money, like most companies; they’re called “not-for-profit” for a reason—any and all revenues go directly back into the organization. Cure CMD, for example, in the most recently completed fiscal year, spent 94 percent of its revenues on program services such as research. The other six percent went to overhead costs like office costs, information technology and infrastructure, staff costs, insurance, etc.
Every day, we push to fulfill our mission to find treatments and a cure for CMD, and to support the families who are part of our community; however, we are continually pulled back in the direction of needing to fundraise. After all, the work depends on it. So how does an organization mitigate the push-pull? How can we balance the work to fulfill our mission with the need to fund it?
1. Gathering Data.
A recent article in the Orphanet Journal of Rare Diseases
states that one of the most important steps along the path toward treatments and cures for rare diseases is to establish a disease-specific registry. Because rare diseases by nature have fewer cases worldwide, a central repository of natural history data over several years is crucial to developing effective drugs and ideally exists before viable drug options. Cure CMD is a leading partner in the Congenital Muscle Disease International Registry, established in 2009, and continually encourages its community to keep their information up-to-date in the CMDIR.
Cure CMD is the number one online source of news, information, and research about CMD for our community members. Our goal is to remain relevant and connected with affected individuals, families, and CMD experts, and regular interactions via social media and email extend Cure CMD’s conversation with the community. By providing a service, we earn value in the hearts and minds of those who can contribute financially to the organization.
Of course, when dollars are scarce, creativity is key. We must think beyond the NIH to explore who may be interested in supporting our activities. The best method is to keep an open mind while seeking support, and listen to donors—both individuals and institutions—to hear what they care about.
With seemingly hundreds of things to do before the end of the day, every day, acknowledgement is one thing that can easily fall to the wayside. However, people are the only reason Cure CMD can exist. From the staff who manage day-to-day operations and plan events and conferences, to the researchers, scientists, and physicians who conduct CMD research, to the individuals and institutions who fund Cure CMD’s work—we would be nothing without our community. Cure CMD aims to ensure that the people we engage with feel valued and supported.
Of course, the challenges of fundraising may unfortunately always exist. Even in broad nonprofits, say, that provide social services, or education, face fundraising challenges. Working within a niche like rare disease creates even further limitations. However, our passion for this work keeps us committed, and the strength of the community keeps us motivated to work tirelessly toward our mission and push toward a CMD cure.