For decades, patient advocates have helped shape PKU care, from newborn screening and access to treatment to better standards of care. Yet one aspect of advocacy has received far too little attention: its impact on the mental wellbeing of those who dedicate themselves to supporting others. It is time to start that conversation.
By Małgorzata Henek, Secretary, ESPKU and Kevin Alexander, Patient Advocate (USA)
On the occasion of this year’s International PKU Day, E.S.PKU during its Traces of PKU campaign is focusing on the mental health of people with Phenylketonuria and their families. One aspect that has not been sufficiently highlighted or discussed is the mental health of patient advocates.
The World Health Organization defines advocacy as a process aimed at influencing individuals, communities, and decision-makers in order to achieve change. According to the organisation, health advocacy encompasses both individual and collective actions undertaken to secure political commitment, policy support, social acceptance, and system-wide support for a specific health goal or program. Patient advocates include parents of children with Phenylketonuria, individuals living with Phenylketonuria, and partners of people affected by the condition. Within the global PKU community, advocacy is carried out both by individual patient advocates and by advocates actively involved in patient organisations.
We asked representatives from patient organizations around the world about how advocacy has affected their mental health. PKU advocacy has evolved over the years, requiring patient organizations to adapt their strategies and the result is increased pressure on individual advocates. These quotations are anonymous to ensure that the individuals felt like they could speak freely.
One person said, “Patient organizations are no longer only community shelters where we can feel safe, eat safely, exchange experiences, concerns and day-to-day advice, bond with one another and form lasting friendships.” Of course, those activities remain central to what a patient organization provides for the community. But organizations face new challenges that require volunteers to serve in new ways. This volunteer continued, “We are increasingly involved in health policymaking, and we increasingly do so as active participants, not just observers or ‘nice-to-haves’ to increase policymakers’ public accountability and legitimacy.”
Another advocate described how demanding advocacy has been for them. This person said, “I am used to working in fast-paced, high-pressure environments in the corporate sector and in the media, but I can honestly say that working for a patient organization has been the most challenging experience of my career.” The people who shared their experiences with us all describe a sense of duty they feel to the community, and so they continue with the work even when they want to let someone else serve in their place. This advocate also said, “Overall, I cannot say that this work has had a positive impact on my mental health, nevertheless, because I feel a strong pressure and sense of duty to stand up for our small group of rare-disease patients, I have to continue for now, as these people simply cannot be left without support.”
“Responsibility” is a theme we encountered repeatedly in these comments from those serving in patient organizations. One advocate said, “The work is exhausting. On one hand, you’re trying to manage your own experience with PKU. But at the same time, you’re trying to care for others. And since non-profits always struggle to get dedicated volunteers who show up, the same people keep doing the same work, over and over, until they are burned out and exhausted.” This sense of responsibility has caused this person to struggle with balancing their career and service to patient organizations. They said, “I feel a deep sense of responsibility to care for those who follow me. As a result, I have often prioritized advocacy over my career. I can be at work and need to focus on a certain task, but if I take a break and look at my social media messages, my mind will wander for the rest of the day, and I won’t be as productive. People share deeply personal experiences with me, and so my mind is often filled with the struggles of those who can’t access their PKU treatment, are facing imminent danger depending on where they live in the world, or who have endured unspeakable personal tragedies.”
That advocate wasn’t the only person who discussed missing out on other life experiences apart from the PKU community. Another person said, “Much of the time, it felt like carrying a constant and crushing weight of worry, responsibility, and repeated heartbreak when progress stalled or hopes were raised. I did have interesting experiences along the way, but to be honest, I also missed out on lots of opportunities too – in my career and in my family and personal life, because I had to solve this problem for my family.” And they, too, described the burden of carrying the burdens of others. “You inevitably have to spend a lot of time dealing with your own worry and concern, and then taking on the experience, pain, and worry of others,” they said.
We must also consider the challenge of accepting the burdens of advocacy while also managing one’s personal experience with PKU. One volunteer said, “Being president means never truly disconnecting from the disease. It invites itself to the dinner table, into my evenings, and into my couple’s life. The guilt of taking time for oneself when ‘everything rests on us’ is a toxic mental health burden that prevents true recovery.” This person also described how PKU affects mental health from a biological perspective. “We often forget that PKU is a metabolic disease where fatigue and concentration issues linked to phenylalanine levels create a vicious cycle that leads to more exhaustion,” they said. “When the associative mental load becomes overwhelming, it accentuates this cycle. Stress and exhaustion disrupt diet management, and in return, fluctuating levels make thinking and decision-making much more arduous. It is a permanent cognitive battle.”
That advocate also describes how advocacy helped them manage their PKU. “It was by taking care of others that I began to manage my own condition better,” they said. “Understanding the science behind PKU to explain it to others pushed me to be more rigorous and aligned with my own treatment.”
PKU advocacy is often framed as “inspirational” work. But this testimony from advocates around the world demonstrates that there is another perspective—it can be a burden.
Patient advocates have played a pivotal role in many of the most significant advancements in PKU care. Through their dedication and perseverance, they have helped establish newborn screening programs, expand access to specialized care for people with PKU, secure reimbursement for medical formulas, low-protein foods and pharmaceutical treatment, and ensure that the perspectives of patients and families are represented in discussions with clinicians, researchers, and policymakers.
While considerable attention is appropriately focused on the wellbeing of patients and caregivers, the mental health of patient advocates doesn’t receive recognition.
Patient advocates are often the first people families contact after diagnosis. They answer calls, messages, and emails at moments of fear, uncertainty, and despair. They listen to stories of trauma, loss, frustration, and isolation. They help families navigate complex healthcare systems and carry the emotional weight of those conversations long after they end. Alongside the pain, they also share moments of hope, relief, achievement, and joy. But carrying the stories of an entire community, year after year, leaves a mark.
The burden is not borne by advocates alone. Their families, partners, and loved ones are often affected as well. Advocacy rarely ends at the office door or after a meeting. It becomes part of daily life, shaping relationships, consuming time and energy, and sometimes placing emotional demands on those closest to the advocate.
For too long, the mental health of patient advocates has been overlooked. We celebrate their achievements, rely on their dedication, and turn to them in moments of need, yet we rarely ask who supports them when they are struggling.
A burned-out advocate cannot sustainably support a community. An exhausted advocate cannot continue carrying the weight of hundreds of stories alone. If we value advocacy, we must value the wellbeing of advocates.
If we want advocates to continue changing lives, we must ensure that they are not left to carry the burden alone. Because those who spend their lives supporting others deserve support themselves.