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We Treat Rare Disease. We Don’t Always Treat What It Does to People.


I think we do not talk enough about the mental health impacts of rare disease on both rare kids and their parents.


For kids, that can mean missing school, feeling different from their peers, enduring repeated appointments, and trying to make sense of a body that does not always cooperate. For parents, it can mean living in a near-constant state of worry, making decisions no one should have to make alone, and carrying the emotional weight of a diagnosis that may still not have a clear path forward.


For us, Rona's diagnosis felt like death by a thousand paper cuts.


Every week, it seemed like there was another restriction. Another food taken away. Another activity she could no longer do. Piece by piece, the world she loved got smaller, until so much of what made her feel like a kid had been stripped away.


And once we were in the hospital, I asked for a therapist to visit Rona for weeks. She did not get seen until after she had been in protective isolation for so long that she had already reached a breaking point. That was not because the hospital did not care. It was because there were not enough resources. But the impact was still real.


To put what it is like to live in protective isolation in context: Last year, I had a panic attack during a "fun" outing to Alcatraz after seeing the solitary confinement room, because I was suddenly right back in the hospital. That is what trauma does. It follows you, even into moments that are supposed to be light.


That reaction makes sense in the context of what we know about pediatric critical illness. Long-term psychological effects after PICU admission are common, and PTSD is among the outcomes documented in follow-up studies (Colville & Pierce, n.d.; Mikkelsen et al., 2022). Earlier review data found that children after PICU admission can experience PTSD symptoms and that parents are also frequently affected by post-traumatic stress, anxiety, and depression (Colville & Pierce, n.d.; Mikkelsen et al., 2022).


And that is the part people do not always understand: the emotional toll does not wait for a perfect system. It builds. It builds in the child who is losing control over their own life, and it builds in the caregiver who is trying to hold everything together (Colville & Pierce, n.d.; Mikkelsen et al., 2022).


Rare disease is not actually rare in the way people often assume. One in 10 Americans lives with a rare disease, and about half of those affected are children (NORD, n.d.). NAMI reports that 23.4% of U.S. adults experienced mental illness in 2024, 5.6% experienced serious mental illness, and 16.5% of U.S. youth ages 6 to 17 experienced a mental health disorder (NAMI, n.d.). Put simply: a lot of families are carrying both medical complexity and mental health strain at the same time (NORD, n.d.; NAMI, n.d.).


What Kids Feel


Kids may not always have the words for anxiety, grief, or fear, but they feel it all the same.

They may worry about pain, procedures, hospital stays, or what their future will look like. They may notice when adults around them are stressed, even when no one says it out loud. They may feel isolated because their condition makes them different, or because they have to miss things other kids take for granted.


A child with a rare disease may also carry the burden of chronic uncertainty. There may be questions without answers, treatments without guarantees, and symptoms that change from one day to the next. That kind of unpredictability can be hard for any adult to manage, let alone a child (Bull et al., 2025; Mikkelsen et al., 2022).



This is why therapeutic play matters so much in pediatric care. A systematic review of hospitalized children found that therapeutic play decreased postoperative pain, improved behavior and attitude, and reduced anxiety during hospitalization (He et al., 2020). Child Life's evidence-based guidance also frames therapeutic play as an essential part of pediatric health care, not an optional extra (Child Life Council, n.d.).


In a rare disease setting, play does more than distract. It gives children a way to practice control, rehearse hard moments, and express feelings they may not yet be able to explain. When a child's world has become filled with restrictions, play helps return a sense of agency (He et al., 2020; Child Life Council, n.d.).


What Parents Carry


Parents of children with rare diseases often become experts overnight.


They learn medical language, coordinate specialists, advocate for services, and try to stay calm in front of their children even when they are scared themselves. Along the way, many carry chronic stress that can turn into anxiety, depression, or burnout if they do not get support (Bull et al., 2025; Mikkelsen et al., 2022).



I think about the mental health impact on caregivers, too. Not just the exhaustion of caring around the clock, but the pain of watching your child suffer and feeling helpless to stop it. That kind of helplessness changes you. It is heavy, it is lonely, and it is a burden too many parents are carrying without enough support (Bull et al., 2025; Colville & Pierce, n.d.).


The research backs this up. Studies of parents and primary caregivers of children living with rare diseases consistently show elevated anxiety, stress, caregiver burden, and reduced quality of life (Bull et al., 2025). In one systematic review, anxiety and stress were the most frequent and significant psychological harms reported by caregivers, and many studies found that mothers carried especially high levels of distress (Bull et al., 2025).


Parents of children in the PICU face similar trauma exposure. Critical care can leave families with persistent post-traumatic stress symptoms long after discharge, which is one reason family-centered mental health support is so important (Colville & Pierce, n.d.; Mikkelsen et al., 2022).


Why Both Matter


Rare disease and mental health are often treated like separate issues, but in real life they are deeply connected.


A child's physical symptoms can shape their emotional health. A parent's mental health can shape how well the family can navigate care. And when support is fragmented, the whole family feels it (Bull et al., 2025; Mikkelsen et al., 2022; Colville & Pierce, n.d.).


That is why support should be built around the whole family, not only the diagnosis. Kids need child-friendly explanations, opportunities to play and process, and caregivers who help them feel safe. Parents need respite, mental health care, community, and honest acknowledgment that this is hard (Bull et al., 2025; Child Life Council, n.d.).


Mental health should be treated with the same seriousness as physical health. That principle matters in general, but it becomes even more urgent when a family is already navigating a rare disease (NORD, n.d.; NAMI, n.d.).


What Helps


Families do not need perfection. They need support that is steady, practical, and compassionate.


That can look like:

  • Clear explanations that match a child's age and understanding.

  • Mental health screening for both children and parents.

  • Support groups where families can hear "me too."

  • Access to counselors who understand chronic illness, ICU trauma, and medical grief.

  • Therapeutic play as part of routine pediatric care.

  • Music therapy in hospital settings, especially for children facing pain, isolation, or repeated procedures.

  • Schools that make room for absences, fatigue, and brain fog.

  • Care teams that treat emotional wellbeing as part of treatment, not separate from it (He et al., 2020; Child Life Council, n.d.; Lim et al., 2022; Ullán et al., 2022; Hsu & Chen, 2022).


When kids feel understood, they cope better. When parents feel supported, they can keep supporting their children. And when both are cared for, the whole family has a better chance of getting through the hardest parts with dignity intact (Bull et al., 2025; Mikkelsen et al., 2022).


Rare disease can make life feel smaller in some ways, but it should never make a family feel invisible.


If we want to truly care for children with rare diseases, we have to care for their mental health too. If we want to support parents, we have to stop expecting them to hold everything alone. And if we want pediatric care to be truly healing, we have to make room for play, music, and trauma-informed support that helps children feel like children again (He et al., 2020; Child Life Council, n.d.; Lim et al., 2022).



The diagnosis may be rare. The need for compassion is not (NORD, n.d.; NAMI, n.d.).


References





 
 
 

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