As a part of the ROADMAP project participation, we asked the rare disease nonprofits to forward the survey to their US-based researcher network and invite them to take the survey. Wee asked questions regarding the researchers’ involvement in research projects to advance drug repurposing for any rare diseases, what their motivations are and what challenges they’ve encountered.

Although the sample size of participating researchers was small (43), we present the data as an initial reference for understanding their involvement in different aspects of drug repurposing initiatives. Making broad generalizations as to researchers whose research relates to rare disease drug repurposing based on this data may be challenging based on the small sample size.

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

Current title at research institution

Among the researchers that participated in our survey, 23 currently professor-level appointments at academic institutions, most of which reported being associate professors. This illustrates that most participants are likely in the mid-career stage of their academic careers.

n (titles): 41
n (researchers): 40

n (researchers): 40

Primary Affiliation

The majority (33, 83%) of the participating researchers consider their primary affiliation an academic institution, while just 3 work primarily at a rare disease research organization.

n (researchers): 40

Years at current position

The researchers in our dataset vary greatly in the time they have been in their current position - ranging from 41 years to less than a year at their current position. Though this is not a perfect measure, it does suggest some variance in overall years of experience.

n (researchers): 40

Drug repurposing

For purposes of this project, we utilized a broad definition of drug repurposing, as a process of research to identify potential treatments that are already FDA-approved or in development for one disease, for use in another disease by gathering data and analyzing efficacy in order to improve treatment guidelines and access.

Drug repurposing: level of familiarity


n (researchers): 40

Drug repurposing: level of interest

The majority of researchers in our dataset indicated that they were highly interested in drug repurposing research, with 20 expressing an extremely high level of interest and the rest indicating a very high level of interest (13).

n (researchers): 40

Drug repurposing: assessment of how common it is

While 25 (62.5%) researchers stated that drug repurposing research is a fairly common area of focus, 12 indicated that they consider it to be not very common.

n (researchers): 40

Drug repurposing: level of importance for rare diseases

Overall, most researchers (22, 55%) feel that drug repurposing research is extremely important for the rare disease space.

n (researchers): 40

Supporting different types of research

Among the researchers in our dataset, we found overlap between those that focus on novel drug development and those that focus on drug repurposing research (19/26, 73% focus on both). This illustrates that researchers are able to pursue both, and maximize their impact on the lives of rare disease patients.

novel drug development research

n (researchers): 40

drug repurposing research

n (researchers): 40

Personally involved in supporting drug repurposing projects

Most researchers in our data set (32, 80%) are either currently or have previously been involved in drug repurposing research.

n (researchers): 40

Personally involved in supporting drug repurposing projects for a rare disease

Among the 32 researchers that have prior drug repurposing research experience, all but one reported that this research was in benefit of a rare disease.

n (researchers): 32

Drugs of focus for repurposing research

Among the 47 drugs reported to have been pursued for repurposing, again sirolimus is most common, with 5 researchers reporting it as their promising drug of focus. Also, among the top 5 are ruxolitinib, sodium phenylbutyrate and various statins not otherwise specified.

*Note: this section merges data from 1-5 drug(s) listed per researcher. Where applicable, both researcher and drug totals are provided. These numbers vary by how many organizations answered each question and how many drugs they listed. The unit which is visualized in the chart is provided in bold*

n (drugs): 47
n (researchers): 26

Drug repurposing: motivation for pursuit

Among the motivations reported among the researchers experienced in drug repurposing work, the top choices were impact to patients (27 researchers), interest in the research subject area (20), opportunities for collaboration (15) and publication (12). Though potentially a big factor, IP or patents did not play a major role for the researchers who participated in our survey, with only 6 researchers listing it as a factor.

Note: respondents were able to select multiple answer choices.

n (researchers): 28

Drug repurposing: techniques and resources utilized

For these drug repurposing projects, the most common research techniques and resources reported were cell lines (16), clinical trials (15), and mouse models (11).

Note: respondents were able to select multiple answer choices.

n (researchers): 28

Drug repurposing: roadblocks for research

Among the many difficulties researchers might face when pursuing drug repurposing, the top by far in our data is the lack of financial resources, reported by 20 (71%) researchers. Though, also significant are a lack of support from the pharmaceutical company (13), a lack of bio-specimens (8) and patients (7).

Note: respondents were able to select multiple answer choices.

n (researchers): 28

Current stage of repurposing work

Out of the 29 researchers, only 3 consider the repurposing project to be completed while the majority reported the research to be ongoing.

n (researchers): 29

Drug repurposing: completed work contributions

Among the researchers who have completed their repurposing projects, all of them reported to have published the results of their research and made significant contributions to the rare disease space by identifying the primary mechanisms involved.

Note: respondents were able to select multiple answer choices.

n (researchers): 3

Drug repurposing: ongoing work, current stage(s)

Among the researchers who reported that their research is ongoing, 14 reported to be working on understanding disease mechanisms, 12 are searching for drug targets. 25 researchers reported that there have been drugs identified; among these 10 are in the process of being considered which patients they are most likely to help, 8 have some preclinical data generated, 7 have not had validated in preclinical data.

Note: respondents were able to select multiple answer choices.

n (researchers): 25

Drug repurposing: access to drugs for research

Most researchers (21, 72.4%) reported to not have issues accessing the drugs they have identified as promising for repurposing work.

n (researchers): 29

Drug repurposing: publishing work

Overall, regardless of status of the research, 15 (51.7%) researchers reported to have published the results of their repurposing work.

n (researchers): 29

Dissemination of information regarding drug repurposing research to rare disease community

Out of those that said they have published the results of their repurposing work, most (10, 66.6%) said they are directly involved in the dissemination of this information to the various rare disease communities the research could impact.

n (researchers): 15

Methods of dissemination of information regarding drug repurposing research to rare disease community

Among those researchers that reported to be directly involved in the dissemination of this information to the rare disease community, most (8, 80%) reported accomplishing this task by working with a rare disease nonprofit organization, though social media and traditional media outlets are also utilized to a lesser extent.

Note: respondents were able to select multiple answer choices.

n (researchers): 10

Engagement of rare disease patient population during research process

A promising finding is that most (30, 83.3%) of researchers in our data reported engaging the rare disease patient population at least in some stage of their research process. Patients bring unique perspectives and insights into their conditions, which can inform research priorities and improve the relevance and quality of research outcomes.

n (researchers): 36