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Starting and running a patient advocacy organization is a massive undertaking. It often includes fundraising, standing up and operating a board and non-profit structure, educating and connecting patient communities, providing assistance to patients and families, engaging in policy and research, and much more. Many patient advocates starting new patient advocacy organizations are thrown into this space by a family member’s disease journey and quickly find themselves navigating largely uncharted territory.
That was the impetus for the Biotechnology Innovation Organization’s (BIO) May Coffee Chat, “The Secret of Success: How to start—and Thrive—as a Patient Advocacy Organization.”
The goal was to exchange tips, tricks, and best practices and make it a bit easier to leverage some of the many existing tools, resources, and relevant coalitions, explained Karin Hoelzer, Senior Director of Patient Advocacy, who moderated the discussion.
Skin in the game
As the panelists introduced themselves, one unifying theme arose: patient advocacy is personal.
Case in point, Jim Palma, CEO, TargetCancer Foundation, was brought into the world of patient advocacy by his brother-in-law, Paul, who was diagnosed with a rare cancer called cholangiocarcinoma in his late 30s.
“Despite being in Boston at a top medical center, Paul very quickly learned that his cancer had no treatments—which was quite a shock,” said Palma. “And over the course of just under two years that Paul was treated, he never received a treatment that was particularly effective. So he started the foundation himself in an effort to bring greater awareness to this situation and rare cancers, but also to start to drive research as well.”
Sadly, Paul passed away not long after, but the work of his foundation continues. “We’re in our 16th year now, and today we focus on a variety of different initiatives—most notably a clinical trial called TRACK, focusing on precision medicine in rare cancers,” said Palma. “But as an umbrella organization, we look at the challenges that patients with rare cancers face as a whole.”
Similarly, Kelly Brazzo, founder and CEO of Cure LGMD2i, was brought into the patient advocacy world by her daughter, Samantha, who, at two years old, was diagnosed with the ultra rare form of muscular dystrophy called Limb-girdle muscular dystrophy (LGMD).
“We were told, Sorry, there’s really no treatment for your daughter,” Brazzo recalled. “I said, Well, what do I do? What can I do for her? They said, start a foundation and raise money. There’s just not enough research in this space because it’s so rare. So I took that as a prescription and kind of ran with it.”
Today, Brazzo and her organization are still working towards an approved therapy with three clinical trials in their disease under development. “One of them is in phase three,” Brazzo said. “We also have two gene therapy trials, all of which have orphan drug and rare pediatric disease designations. So we’re excited about the future of our families affected by LGMD 2I/R9.”
Ashira Vantrees, of Aimed Alliance, for her part, explained how she got involved with KIF1A.org when her sister was diagnosed with the disease.
“My sister was diagnosed five years ago, and I had this unique opportunity to bring my skills and knowledge from Aimed Alliance and working in this health policy space to our KIF1A organization to help make sure that the treatments that we think are so important are adequately supported within policy,” Vantrees said.
Lesson One: Leverage existing resources and don’t reinvent the wheel
“When you move into this world, and you’re learning on the job about so many factors—and maybe doing so at the same time that you’re caring for a loved one—it can be very easy to set goals and then just try to orient your foundation towards accomplishing those goals. But often what that can lead to is reinventing the wheel,” explained Palma.
Palma pointed out that for any given disease, there may already be tools such as registries or tissue banks, and existing organizations that advocates can leverage, so exploring the advocacy landscape is key.
This sentiment was echoed by Patrick Wildman, Senior Vice President of Advocacy and Government Relations at the Lupus Foundation of America, who leads the Medicare Access for Patients Rx (MAPRx) coalition, a coalition of patient, family caregiver and health professional organizations committed to safeguarding the well-being of patients with chronic diseases and disabilities who rely on Medicare’s prescription drug coverage, Medicare Part D.
Wildman reminded participants of the importance of collaboration and connecting with established groups. “I remember early in my career, I had the good fortune of having a great mentor,” Wildman said. “We used to go to different advocacy days run by different organizations to learn how they do what they do, and what ideas we can take from them and apply to our disease.”
Getting to know people offers connections to networks and leaders that can serve as mentors, explained Wildman. This, in turn, helps advocates succeed. Many patient advocacy organizations share common struggles and learning from each other can be key to realizing their goals, Wildman noted. In this way, coalitions like MAPRX and organizations like the National Health Council (NHC) act as critical conveners—tracking policy issues that impact patient’s daily lives across many therapeutic areas, while also working to connect patient advocacy organizations with each other, as well as with industry partners, provider groups, and more.
Lesson Two: Prioritize community building and grassroots engagement
Building strong ties to the patient community is foundational for serving their needs. And as Wildman noted, there are other stakeholders to consider, including researchers, physicians, as well as the staff and board of the patient advocacy organization itself. Regularly surveying these groups is key to setting goals, priorities, and policy timelines that truly reflect the community’s priorities and needs.
Developing short and long-term goals can also help families and volunteers stay engaged over time. “I think it can be difficult to keep people engaged when that long term goal can feel out of reach because there are so many things that go into it before you get that treatment,” said Vantrees. This input is particularly important when it comes to prioritizing long-term policy priorities. “When we decided to set our policy positions, we surveyed the community to first understand what the issues are,” explained Vantrees
Dedicated tools and resources can make it much easier to build such strong grassroots advocacy. “If and when you can hire somebody, I recommend the first person you hire to be a grassroots person who can cultivate and educate consistently throughout the year,” said Kimberly Beer, the Senior VP of Policy and External Affairs at the NHC.
You also need to be able to group your advocates based on interests and expertise as you continue to develop them, Beer explained, ranging from those who work one-on-one with patients and families, to those who have the interest and skills to talk to policymakers on the Hill. Continuing to provide development opportunities keeps advocates engaged, and over time advocates can become highly effective advocacy ambassadors.
Lesson Three: Delegate! Leverage your volunteers—and your Board
“The recommendation I would give to everyone is to learn how to delegate confidently,” said Brazzo. “That is something that took me a long time to learn.”
As Brazzo explained, many people volunteer because of a personal connection to the disease; they bring their unique set of skills and expertise—such as IT, graphic design, accounting, fundraising and more—and are usually happy to help in small or big ways.
Another key step is the establishment, development, and engagement of a Board, as Palma noted. “Developing a Board can really help organizations function more like a business,” he said. “Having a Board who can support that business and provide it with resources that it otherwise might not be able to take on—like pro bono legal counsel or accounting help—can be transformational.”
The organization’s needs inform how the Board is best structured. “Board members may approach things with a different lens, whether it’s as a caregiver or someone living with the disease—or even as someone who’s really connected to a different disease and may have a different perspective.” Boards often also play a key role in fundraising—an undeniable challenge organizations must face head on.
Driving forward cures
Patient advocates are the MVPs of advancing scientific innovation and dismantling barriers to access. Patient advocacy organizations empower patients and families, and are profoundly shaping today’s complex healthcare landscape, from patient education and care navigation to shaping research and policy. But, nobody can do it alone. Coalitions like MAPRx and groups like NHC bring together patient organizations to join forces, learn from each other, and speak with one voice.
As the BIO May Coffee Chat showed, dedication and determination, together with ingenuity, collaboration and the sharing of information among patient advocacy organizations—no matter what the disease—is the secret to patient advocacy success.
