Familiarity with drug repurposing

Most organizations (48) responded that they were moderately familiar with drug repurposing concepts for their disease(s) of interest, while only 13 were totally unfamiliar with the concept.

n (organizations): 138

Interest in pursuing drug repurposing

98 organizations reported being extremely or very interested in pursuing drug repurposing for their rare disease(s) prior to this survey, while only 6 organizations were not at all interested.

n (organizations): 138

Involvement in drug repurposing initiatives

Drug repurposing initiatives with external partners

Future drug repurposing interest

Reasons for non pursuit

Among the reasons for why the inexperienced organizations have not yet pursued drug repurposing projects, the top three in our data were a lack of funds, a lack of information for how to best pursue drug repurposing and a lack of staff to help support this project. Notably, 5 organizations stated that they have never heard of drug repurposing before.

Note: respondents were able to select multiple answer choices.

n (organizations): 77

Drugs being repurposed

*Note: In this section, organizations had the option of listing - and answering questions about - up to 10 drugs. Where applicable, we include how many organizations answered the question, and how many drugs (i.e., response selections) are included in the visualization. The number of response selections is bolded.

Though 58 organizations said they were involved in drug repurposing initiatives, only 40 were able to provide a specific drug or drugs that are being pursued. To view a list of rare diseases and what drugs are being repurposed for them, click HERE.

Since many (19) organizations are focused on more than one drug, from these 40 organizations we received a list of 94 (76 unique) drugs that are currently being repurposed. From this data, we can clearly see that sirolimus (Rapamyacin) is a very commonly repurposed drug. Please refer to the repurposing sirolimus page for more details.

n (drugs): 94
n (organizations): 40

Organizations pursuing multiple drugs for repurposing

Drug repurposing: Identification Methods

Traditional preclinical/translational research approaches led to the identification of 65 drug(s) for repurposing, while 28 drugs were identified by looking at similar diseases/conditions. High throughput drug screens, though often thought of as the most common repurposing method, was only utilized to identify 21 drugs in our dataset.

Note: respondents were able to select multiple answer choices.

n (drugs): 91
n (organizations): 38

Drug repurposing: Current Stage

Out of our 94 drugs currently in the repurposing process, most are in early stage clinical trial stages (patients being recruited, securing funding, running Phase II clinical trials), as well as using the drug off label. 20 drugs are still being tested in animal models, and 12 have been abandoned for various reasons. 5 drugs have already been given FDA approval for their new rare disease indication.

Note: respondents were able to select multiple answer choices.

n (drugs): 94
n (organizations): 40

Drug repurposing: Organizational Involvement

Most of the organizations that reported to support drug repurposing initiatives did so by funding preclinical research (for 52 drugs), facilitating international cooperation (48 drugs) and supporting recruitment of patients into a clinical trial (42 drugs).

Note: respondents were able to select multiple answer choices.

n (drugs): 92
n (organizations): 39

Drug repurposing: Roadblocks

Lack of funds is a major roadblock for many organizations in drug repurposing initiatives - they reported financial roadblocks in regards to 38 drug repurposing projects. Lack of pharmaceutical company support was slowing down the repurposing process for 24 drugs, and small patient populations were causing issues for 17 drugs. Interestingly, 14 drug projects are reported to be going forward without hitting any roadblocks.

Note: respondents were able to select multiple answer choices.

n (drugs): 90
n (organizations): 38

Drug repurposing: Success Endpoints

52 drugs are being repurposed with the goal of providing significant improvement in quality of life and 50 - for the reduction of symptoms. FDA approval was the targeted success outcome for 40 drugs.

Note: respondents were able to select multiple answer choices, and then respondents were asked whether their selected endpoints have been met (below).

n (endpoints): 235
n (drugs): 92
n (organizations): 40

Drug Repurposing: Success Endpoints Met

Among all the success endpoints selected for the ongoing drug repurposing projects in our dataset, most of them have not been met, or not been met yet. Among the top ones that have been met, however, is making the drug available off-label with at least some safety/efficacy data as to its use (17 drugs), as well as providing significant improvement in QOL (11 drugs).

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

n (endpoints): 219
n (organizations): 40

Drug repurposing: Additional Support

We asked participating organizations to select where they feel they need additional support based on three main categories: more collaboration, more data/information, and more resources. For collaboration, the top choices were to connect with organizations focused on the same or similar rare disease(s), partnering for the development of clinical trials, and developing outcome measures. For those that selected that they needed more data/information, the top choices were also in developing robust outcome measures, as well as developing a drug target and understanding the natural history of the disease. As for more resources, they are needed most in developing research infrastructure and models, developing clinical trials and improving patient access to off-label drugs.

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

n (organizations): 124