Date: 10/04/2021

Being diagnosed with a rare disorder can be frightening and lonely. Often, newly diagnosed individuals will turn to the internet to find information about treatments, prognosis, and to connect other individuals dealing with the same condition. But, in many cases, the information that is available is unreliable–or it may not exist at all. There are more than 7,000 identified rare disorders. Many do not have existing advocacy groups or medical research networks, leaving affected individuals with nowhere to turn for information or support. This frustrating experience often leads to ‘kitchen table’ conversations between parents and loved ones who decide that they will build their own advocacy group to fill the void.

Creating a rare disease Patient Advocacy Group (PAG) can have a life-changing impact on the entire patient community. Most PAGs are nonprofit organizations with a mission to engage patients, families, researchers, and medical professionals impacted by a specific medical condition through advocacy, education and community support. These organizations serve as a centralized resource hub to help families accelerate access to better therapies and cures.

Running PAG can be a full-time commitment that requires every ounce of creativity and commitment its founders can muster. But for those passionate individuals who are willing to do what it takes to build a community, and to generate the data and resources needed to capture the research industry’s attention, it can lead to life-saving innovations that benefit families across the globe.  

Where to Begin

Getting started is often the most difficult part. To ease that process, Prometheus asked Michele Manion, founder of the Primary Ciliary Dyskinesia (PCD) Foundation, and Carey Kauffman, patient registry director for PCD Foundation, to share some advice on how to start a patient advocacy foundation, and the pitfalls to avoid. Here’s what they shared.

1. Define your mission, write a mission statement and commit to it

It’s easy to assume your mission is clear, but as your organization grows you may find yourself fielding requests from patients, caregivers and researchers who want your help (and data) to support their projects. They may all have good intentions, but if you are not careful, you can get pulled in so many directions that you can’t accomplish anything.

If your primary goal is to find a cure (or better diagnostics, or new treatments) It is important to focus on projects that support that goal. Most rare disorder PAGs have very limited resources—both financial and human—so need to ensure that their limited resources are used in ways that support their mission. A clear, well written mission statement can help you with this. 

When writing your mission statement, be brief and be specific about your goals and the problems you intend to solve. Being able to point to a clear mission statement will help you stay focused and convince your team members to choose the right path forward.

This does not mean that your mission should remain static and inflexible. Missions and mission statements will often grow and evolve as the organization grows and evolves and that’s okay. In fact, it is good practice to review your mission statement with your stakeholders annually to ensure it still accurately reflects your needs.

2. Make research a core part of your mission

Clinical research is the foundation for understanding a disease, developing therapies, and ultimately finding a cure. And research is built on data. One of the most valuable things an advocacy organization can do is capture patient data from members about their diagnostic journey, symptoms, and outcomes.  Information about early symptoms, time to diagnosis, common misdiagnoses, treatment paths, and how the disease progresses in different subpopulations can help researchers define trial protocols, prioritize drug candidates, and establish future recruiting criteria.

Making patient data collection a key pillar of your organization will help win the attention of researchers interested in your disease and can accelerate the drug/diagnostic development process.

3. To support research acceleration, collect data that is clinically relevant

PAGs collect data in many forms, including basic contact data, specific surveys about symptoms or quality of life, etc. All these data collection efforts may have value for patient communities. In order to be of value to the research community, however, patient data has to be clean and meaningful. This means data captured for research generally must meet certain criteria for use in this highly regulated industry. Because of this, it is important to match your data collection efforts with intended use. For instance, information you capture via social media conversations or online surveys may have value for your patient community but may not be as useful for clinical trials or formal research studies.

Most PAGs whose mission supports research create patient registries, which are technology platforms that use approved observational study methods to collect clinical, demographic, geographic and other data, from patients and/or through their physicians. When vetting registry options, make sure the tool is scalable, meets data privacy requirements in all countries where patients may reside. Legal requirements in most countries dictate that if the data collected contains private and personal information about individual health, it must have approval and ongoing oversight from a recognized regulatory entity. In the US, these entities are known as Institutional Review Boards (IRBs) which reside at most universities involved in biomedical research or are free-standing commercial companies. Most countries have similar regulatory requirements. 

These extra steps in setting up data collection efforts take more time and resources, but ultimately you will have data that is valuable for researchers and for industry partners interested in therapeutic development. This data will become one of your most valuable organizational assets.

4. Retain ownership of your most valuable assets, including data

Patients and families have entrusted your PAG with their most precious and private health information. This data, in the aggregate, is what will inform therapeutic interventions, direct future research decisions, and ultimately hopefully improve quality of life for your patient community. It is one of the most valuable assets your patient community/PAG has, so don’t give it away. When choosing a registry partner, read the fine print – most “free” platforms offer advocates use of their technology in exchange for ownership of the data they collect. That means they determine who uses the data, who they sell that data to, and who profits from those transactions. This means your data may be used to support priorities that may not match those of your PAG. Instead look for a platform that provides the data collection methods you need and leaves ownership of that data to your PAG.

It is important to create an internal governance structure so that requests for access to your data can be reviewed in a systematic fashion and honored in a way that supports the mission of your PAG. In general, outright selling of data from your registry/data collection is not ideal for any of the parties involved. Data collections are ongoing and evolve over time. Selling a discrete set of data means the PAG loses control over how the data will be used, and the buyer acquires data that may be out-of-date by the time it is used. Instead, look for opportunities to license the data. This means that you give permission to use it in exchange for a fee, but you continue to maintain ownership of it. Licensing your data allows you to generate revenue, share data with multiple organizations pursuing treatments or cures, and ensure the use of data conforms with the mission of your PAG.

5. Celebrate every win, no matter how small

Small victories will sustain your momentum and give you reasons to stay connected with the community who helped make it happen. Their passion and determination will help you get over the rough patches so you can continue your journey to find a cure.