Organizational characteristics

147 rare disease nonprofit organizations (RDNPs) participated in the ROADMAP survey. In this section we share information about the general characteristics of these organizations.​ These characteristics not only provide insight into the kinds of organizations who participated in the project, but they also help to explore precursors for success in both pushing rare disease research forward and the pursuit of drug repurposing.

*Note that most of the survey questions were not required, so the total number of responses is noted for each graph, as they vary.

Leadership Titles

Only members of the rare disease nonprofit’s leadership team were invited to provide data for their organization. Most (101) of our responses were from the leadership team category (President, Founder) and the Director-level category (Executive Director).

n (job titles): 200
n (organizations): 146

Organization year of founding​

The participating organizations are "young"! 83 organizations (56%) were founded in 2010 or later, with 9 founded as recently as 2020 during the COVID pandemic.

n (organizations): 147

Number of full-time staff

Impressively, most organizations in our survey (62%) have no or just one full-time staff member. This speaks to the incredibly valuable role that volunteers play in both leadership and support roles.

n (organizations): 146

Average annual funding (past 3 years)

Though some of the organizations in our survey reported notably higher or lower funding levels, generally most of our 147 organizations lean towards middle ground funding, from $50k to $1,000k on average.

n (organizations): 144

Organizations' activities of focus

Our organizations focus on a wide range of activities. Patient education, disease awareness and community development are clear top choices for both general focus as well as top 3 prioritization.

Note: respondents were able to select multiple answer choices, and then were asked to select their top 3 out of the full list of previously selected choices.

To exclude certain answer choices and view a subset of the data, please click the answer choices in the chart legend.

n (organizations): 147

Organizations supporting research

From the organizations we surveyed, the majority (127, 87.5%) support research and 28 organizations plan to do so in the future. Only 6 organizations do not support research and do not plan to do so in the future.

n (organizations): 145

Those organizations who support research do so by finding and funding researchers that study a specific disease of interest, providing funding through request for proposal (RFP) programs, as well as providing non-financial support for research. Other organizations reported that they support research by creating research partnerships, patient registries, natural history studies and/or conducting research themselves.

n (organizations): 145

Percentage of funding that supports research

For those organizations that support research in one way or another, we asked what percentage of their funding is targeted towards supporting research. 71 organizations stated that 50% or more of their funding goes to research support, while 11 organizations reported that 100% of their funding goes towards research.

n (organizations): 100

Fundraising activities the organization is involved in to be able to fund research

The top three fundraising activities organizations used to raise money for supporting research were patient-led initiatives, organization-led events and grants. Other funding for many organizations comes from private donors, industry donors and employer matching donations.

n (organizations): 138

Organization’s resources for research support

Most organizations reported that they already have multiple resources developed, or that they are in process of developing resources that are able to support research. The top 3 resources which organizations already have developed are community discussion spaces, scientific/medical advisory boards (SABs/MABs) and contact registries. Among the resources organizations are not focused on having, the top 3 are biobanks, a patient community navigator, and, interestingly, patient community gatherings.

To exclude certain answer choices and view a subset of the data, please click the answer choices in the chart legend.

n (organizations): 144

Reasons for not developing certain resources

To better understand why certain resources are not of interest to the organizations, we broke down the survey with specific questions. Please use the dropdown menu below to select a given resource.

Organizations reported not prioritizing the development of their own biobanks as they already have access to biobanks from academic or industry partners. A lack of staff is the main reason listed for not focusing on research and strategy development. For natural history studies, reasons cited include the presence of ongoing studies with academic and industry partners and a lack of funding.

n (organizations): = 130