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The CDCN’s ROADMAP project – supporting the pivotal role of rare disease non-profit organisations in accelerating drug repurposing

The CDCN’s ROADMAP project aims to create a roadmap of routes taken in drug repurposing by aggregating the experiences of rare disease stakeholders. Once the roadmap is completed, it will be made available to guide rare disease non-profit organisations in repurposing drugs for their patient communities. This work is supported by a Chan Zuckerberg Initiative grant.

RARE Revolution met with Ania Korsunska, biomedical leadership fellow at the Castleman Disease Collaborative Network (CDCN) and ROADMAP project lead, to find out more

By Geoff Case

Drug repurposing: ‘off-label’ use

In the United States, each of the approximately 2,500 unique drugs approved by the FDA has a safety profile based on existing clinical trial data, meaning that it can potentially be used “off-label” for any other disease, including for rare diseases. Drugs are commonly prescribed off-label by individual doctors for individual patients, but data on their widespread use and effectiveness is seldom captured systematically. This hinders research and slows down the search for effective treatments for rare diseases. The Castleman Disease Collaborative Network (CDCN) is working to address this problem through its ROADMAP (Repurposing Of All Drugs, Mapping All Paths) project.

The CDCN believes rare disease non-profit organisations are ideally positioned to capture data that may otherwise be lost when drugs are used off-label to treat rare diseases: “Non-profit organisations are well placed as intermediaries between pharma companies, academics doing research, rare disease patients, and other stakeholders to accelerate drug repurposing,” says Ania Korsunska.

The strength of non-profit organisations is their inherent drive, she explains. “Often, they’re founded by parents of children that have the condition, or people who have the condition themselves. Consequently, they are very committed to the cause, and often run these organisations on a volunteer basis.”

The challenge, however, is that non-profit organisations are generally small and have limited support and resources, making it harder for them to engage with the complexities of drug repurposing. Compounding these difficulties, it is not clear what the steps are in a successful drug repurposing project and how a rare disease organisation can best support this process.

The goal of the ROADMAP project

The CDCN has extensive experience in drug repurposing, as the co-founder of CDCN Dr David Fajgenbaum saved his own life with a repurposed drug (read more about his “Chasing My Cure” story here). Unfortunately, drug repurposing scenarios are complex — there is no one approach that all rare disease organisations can or should take in their pursuit of drug repurposing, and no shared resource exists for this community to learn from the experience of others, except on a case-by-case basis from talks, conferences and publications.

“Each scenario is complex and any advice depends on the context: the state of disease research, the population affected, and the organisational characteristics (size, age, funding availability, etc). Simple guidelines on how to approach drug repurposing would not be reflective of the nuances that need to be taken into account, the different paths that exist, and the problems that organisations might encounter.”

This is why the ROADMAP project is aggregating data about all the branching paths that organisations might follow when they are pursuing drug repurposing. The CDCN’s goal is to create an accessible tool for rare disease non-profits to use, which would enable organisations to explore how similar organisations in similar situations have tackled drug repurposing, maybe even for the same drug, and be able to make decisions accordingly. The tool would also signpost existing resources and give recommendations about suitable pathways: which steps to take and perhaps even who to contact for more information.

The ROADMAP project: first steps

At the outset of the project, Ania says the team quickly realised that the fundamental data needed for the project wasn’t readily available. “Foundational datasets don’t seem to exist to answer basic questions about what is going on in the rare disease space: how many rare disease non-profits are there in the US? What do they focus on? Do they support research or mostly focus on advocacy? ”

In the United States there is no single list of rare disease non-profit organisations, Ania says. Filling this foundational gap was an essential first step before the project could begin to answer the questions: how many rare disease non-profits are successfully doing drug repurposing and how are they doing it? Ania and a team of volunteers collated different lists and then filtered them to see which organisations are active and are in the US. This resulted in a list of about 800 rare disease non-profit organisations. The next step was to search for high-level data about each of them, e.g. when each organisation was founded, which diseases it works on, whether or not it mentions any drug repurposing experience on their website, etc.

With this high-level data in place, the CDCN team sent out a survey to all US-based rare disease non-profits that they could find contact information for, and they received data from 147 organisations. The CDCN also asked these non-profits to pass the survey on to their patients, patient’s loved ones (parents, spouses, guardians, etc.), as well as their physicians and their researcher networks. There were separate questions for these stakeholders about their experience with drug repurposing, which provided a substantial amount of additional data – over 1,900 survey responses – that is now being processed.

Next, the CDCN team is working on sorting the 147 organisations into categories that describe their organisational characteristics, rare disease state of research, involvement in drug repurposing and levels of collaboration with other organisations. Have they been involved in drug repurposing, or are they interested in pursuing it in the future? And if they’re not, why not? If they have, what steps have they taken? Where are they stuck? What are the problems? How have they achieved success?

“We are trying to capture the entire pathway. Some organisations have been successful, and in those cases, we explore what success looks like and what actions they have taken to get there. But it’s also very important to learn from those organisations who are either in the process and are actively trying to work through issues, or organisations whose drug repurposing project had failed or been abandoned for various reasons. We are also looking into why some organisations aren’t interested in or decided not to pursue drug repurposing at all, just to have the full picture.”

Once Ania and her team have completed the initial processing of the data, they will interview around 30 of these non-profits “to really get into the details of their experience with drug repurposing. The CDCN has its own experience and understanding of drug repurposing, but we know that the way we have done it isn’t necessarily the best way or the approach that will work for everyone. Drug repurposing isn’t one thing: different approaches are required, and we need to be open to the data and what the organisations themselves are telling us. The ROADMAP needs to be useful for everyone.”

The ROADMAP project is able to provide the first aggregate view of how many drug repurposing projects are currently being spearheaded by non-profit organisations in the US and what stage these projects are at. This is a vital first step in understanding what the paths to successful drug repurposing are and how to guide other organisations through the various roadblocks to achieve success. The CDCN is next looking into the details of all the projects, those that have achieved FDA approval, been abandoned or are still in progress, to understand what differentiates them.

Hope for the future

The CDCN (as the second C in the name suggests) is committed to collaboration. Once cleaned and de-identified, the CDCN will make the data from this project open to others: “It seems there could be so much more research which can be done which is currently out of scope for the current project, for example into how these [rare disease non-profit] organisations function. They’re extremely interesting—they’re volunteer-led and quite small, but they have very large goals that they somehow achieve.” That fascinating paradox indicates the scope for investigating the ROADMAP data beyond the current focus on drug repurposing. The CDCN hopes not only to make the data available for each of the participating organisations, but also to any researcher who wants to dive into this fascinating and under-researched space.

RARE Revolution wishes Ania and her team every success with this exciting project. If you’re interested in being a part of this project or are interested in volunteering to help, contact Ania Korsunska:


Castleman Disease Collaborative Network:

ROADMAP Project:

Castleman Disease Drug Repurposing

Dr David Fajgenbaum’s book Chasing My Cure:

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