Our role as patient advocates is vital, but funding remains an issue

I recently celebrated the fifth anniversary of my work as a patient advocate for people diagnosed with amyloidosis in New Zealand. This journey began because of my late husband, Aubrey, who had hereditary ATTR amyloidosis. Out of desperation, we set out to access treatment for him. But without representation in New Zealand, patients like him are often overlooked by pharmaceutical companies, and many people don’t even know that there are people in this part of the world with this debilitating and terminal condition.

In 2020, we launched the New Zealand Amyloidosis Patients Association (NZAPA). Since then, our organization has continued to grow stronger. Despite Aubrey’s passing that same year, his legacy continues to inspire our work. I use the term “our” because NZAPA has evolved beyond just me and includes a collective of clinicians, patients, and families, including my own.

However, as November rolls around, I always worry about whether we can sustain our efforts for another year. Much of our work depends on the funding we receive. For diseases like amyloidosis, public awareness and clinical expertise are still limited. I never want to face the possibility of having to shut down our organization, because we often serve as vital lifelines, helping patients navigate diagnosis, treatment, and access to care.

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Competing for resources

While our mission is crucial, the funding landscape is particularly fragile. We face ongoing challenges due to small patient populations, limited public recognition, and complex ethical issues, making financial sustainability a continuous struggle for both the board and me. Unlike larger disease organizations, such as cancer or heart foundations, we face a fundamental barrier: the number of individuals affected by amyloidosis is very small. This translates into fewer potential members, donors, and volunteers.

This scarcity directly impacts our fundraising capacity. Public appeals often struggle to gain traction because the disease name is unfamiliar and the symptoms are complex. Even well-designed campaigns reach only a niche audience, making it difficult to generate a steady funding base. I hesitate to ask our patient community for support, as many of its members are already stretched thin due to their circumstances.

To survive, we have relied on the help of pharmaceutical and biotech companies that develop treatments for amyloidosis. While this support has been essential for our charity’s operations, I am acutely aware that funding from these sources can raise concerns about a conflict of interest. Nonetheless, we cannot function without their help.

With amyloidosis, where treatment options are limited and drug prices are high, advocacy groups like ours must tread carefully. Accepting corporate sponsorship carries the risk of a perception of bias, especially if the group advocates for access to specific therapies. To uphold public trust, it is essential that we are transparent about our funding sources and that patients’ voices remain at the center of our mission.

I’ve been encouraged to explore government funding opportunities, but these often prioritize more common conditions or broader public health issues. As a result, we rarely receive sustained public grants. Philanthropic foundations may also focus on research or clinical care rather than advocacy and patient support, leaving a funding gap for organizations like NZAPA, which mainly concentrate on education, peer support, and system navigation.

When grants are available, the application process is often time-consuming and burdensome. Detailed reporting requirements, compliance with nonprofit standards, and annual audits take away time and energy that could be spent supporting patients. This is not to say I won’t pursue grants again, but it is a significant challenge.

Another obstacle in advocating for patients with a rare condition like amyloidosis is competing for attention, not only with other health causes but also specifically within the rare disease community. Large umbrella groups can amplify our collective voice, but they may also draw limited public and corporate resources toward broader agendas, leaving disease-specific organizations like ours underfunded.

For NZAPA and similar groups, maintaining visibility requires strategic partnerships and creative communication. We leverage patient stories, social media, and medical collaborations to highlight the human impact of this disease.

After five years, I have much to share on this topic. Despite the challenges, we have demonstrated that dedicated, patient-driven advocacy can indeed make a real difference, even with limited funding.

Note: FAP News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of FAP News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to familial amyloid polyneuropathy.