Rare Disease Families Are on the Frontlines of Climate Change
- The Rare360 Editorial Team
- Aug 29
- 12 min read
From the parched lands of southern Europe to flood-ravaged regions in Asia, climate change is no longer a distant threat - it’s a daily reality. In July 2025, Earth experienced its third-hottest month on record, capping a 12-month streak where global temperatures averaged 1.53 °C (34.75 °F) above pre-industrial levels. More than 4 billion people endured at least one extra month of extreme heat from May 2024 to May 2025, an alarming trend that directly endangers over 300 million people worldwide living with a rare disease.
For the global rare disease community, these escalating climate extremes present a unique and growing threat. Most of them already navigate fragmented care, limited treatment options, and systemic barriers to medical access. Now, climate change is compounding their challenges by disrupting essential therapies, worsening symptom severity, and undermining already-precarious health systems.
What’s worse, the rare disease community’s needs are often invisible in emergency response planning. The world’s attention is rightly focused on the broader public health impacts of climate change, which is estimated to cause 250,000 additional deaths per year between 2030 and 2050 due to malnutrition, malaria, and heat-related illness. But rare disease patients, despite being medically vulnerable, are rarely included in these conversations.
As the climate crisis accelerates, the rare disease community faces not just physical threats—but emotional and psychological burdens as well. Climate anxiety is growing among patients and caregivers who must now worry not only about their disease, but also about whether their homes, treatments, and healthcare access will withstand the next storm, heatwave, or wildfire.
This article explores how climate change is deepening the crisis for rare disease patients worldwide, and why the path forward must include policies and protections tailored to this uniquely at-risk population.
Climate Change as a Public Health Emergency
Climate change has been recognized by the World Health Organization (WHO) as the single biggest health threat facing humanity in the 21st century. Rising global temperatures, worsening air quality, and an increase in extreme weather events are already reshaping how we experience health and disease.
For the global rare disease community, climate change isn’t just an environmental concern; it’s a health crisis amplifier. It intensifies existing vulnerabilities and exacerbates the everyday challenges of managing complex, resource-sensitive health conditions.
Heatwaves and temperature spikes put individuals with neuromuscular and metabolic conditions at higher risk of heat stress and organ complications.
Air pollution, which now contributes to an estimated 7 million premature deaths annually worldwide (WHO, 2023), disproportionately impacts those with rare respiratory or immunological conditions.
Extreme weather events such as floods, hurricanes, and wildfires are becoming more frequent and severe, often cutting off essential healthcare access, medications, and medical devices that rare disease patients rely on.
The United Nations Intergovernmental Panel on Climate Change (IPCC) has warned that no region in the world is immune to these risks, with vulnerable populations—including those with chronic or rare health conditions bearing the heaviest burden. This makes climate change not only an environmental issue but also a profound public health emergency.
For the rare disease community, this means climate change is not just about rising sea levels or shifting weather; it’s about survival, resilience, and the urgent need for healthcare systems to adapt to an increasingly unstable world.
Environmental Triggers & Worsening Symptoms
Climate change isn't just a faraway threat it directly influences the day-to-day health of rare disease patients by activating environmental triggers that worsen symptoms, destabilize health, and disrupt fragile care routines.
Extreme Weather Threatens Continuity of Care and Infrastructure
Extreme weather events, air pollution, rising temperatures, and ecological disruption act as stressors that destabilize health systems, strain infrastructure, and reduce the availability of essential resources. These conditions are difficult for any population, but for individuals with rare diseases, many of whom rely on uninterrupted medical care, power-dependent equipment, temperature-sensitive medications, and clean air or water, the consequences can be devastating.
For example, flooding can incapacitate hospitals and contaminate water supplies, leading to spikes in infectious diseases and putting treatment continuity at risk. Patients with chronic lung conditions face significantly increased mortality risk when exposed to excessive heat, estimated up to a 43% higher daily mortality risk during temperature spikes.
Air Pollution, Heatwaves, and Wildfires Inflate Disease Burdens
Heatwaves can intensify symptoms in patients with metabolic, mitochondrial, or cardiovascular rare diseases. Certain disorders impair the body’s ability to regulate temperature, putting patients at risk of dehydration, heat exhaustion, or even fatal complications.
Air pollution and wildfires exacerbate rare pulmonary diseases like pulmonary arterial hypertension (PAH) or cystic fibrosis (CF), leading to increased hospitalizations and long-term health deterioration. Increased allergens and pollutants from pollen to wildfire smoke are altering the immune system’s balance, contributing to rising rates of asthma, autoimmune diseases, allergies, and even cancers.
Vector and Water‑borne Diseases Spread to New Regions
Climate shifts expand the range of disease vectors, exposing immunocompromised or multi-system rare disease patients to new infectious threats. Alarmingly, 58% of known infectious diseases have been aggravated by climate change.
Waterborne outbreaks are also increasing, driven by disrupted ecosystems and extreme weather, posing a threat to those reliant on clean water and stable immune function. Roughly 58% of infectious diseases (218 out of 375 studied) are worsened by climate-linked hazards such as heatwaves, flooding, and changing precipitation patterns.
These climate-related health stressors don’t occur in isolation. They intersect with social determinants of health, such as poverty, rural residency, and systemic inequality, that already impact many in the rare disease community. For example, patients living in low-resource settings are less likely to have access to climate-resilient infrastructure or telehealth services, which can be life-saving during extreme events.
The World Health Organization warns that the indirect health impacts of climate change, from food insecurity and displacement to water scarcity and mental health strain, will grow substantially in the coming decades. For rare disease patients, whose care is already medically, logistically, and emotionally complex, these indirect impacts may pose an existential risk.
Barriers to Healthcare Access During Climate Disasters
When climate disasters strike, whether through floods, hurricanes, wildfires, or prolonged heatwaves, one of the first casualties is access to healthcare. For most people, delays in treatment may be an inconvenience. But for those living with rare diseases, where every infusion, every prescription, and every diagnostic test is a lifeline, disruptions can have devastating and sometimes irreversible consequences.
Extreme weather events often damage roads, bridges, and public transport systems, cutting patients off from hospitals, specialty clinics and even mandatory refrigeration for temperature-sensitive treatments. Rare disease patients frequently travel long distances to access specialized care, as local facilities may lack the expertise or treatments they require. A flood that blocks a highway or a hurricane that shuts down an airport doesn’t just delay travel—it can mean a missed infusion, a postponed transplant evaluation, or the loss of access to a clinical trial that might offer the only treatment hope.
Even when patients manage to reach healthcare facilities, hospitals and clinics themselves may be strained or inaccessible. During Hurricane Katrina in 2005, more than 20 hospitals in New Orleans were forced to close or evacuate due to flooding, leaving thousands of chronically ill patients displaced and without continuity of care. In such emergencies, healthcare systems typically prioritize acute trauma and infectious disease cases, meaning rare disease patients whose conditions often require complex attention are pushed aside.
The supply of medications presents another critical challenge. Many rare disease treatments, such as enzyme replacement therapies or biologic infusions, are highly specialized and temperature-sensitive. They require a continuous cold chain for safe storage and transport. Power outages caused by storms, heatwaves, or wildfires can compromise these medications, rendering them ineffective or unsafe. Pharmacies and distributors may also struggle with shortages when supply chains are disrupted, leaving patients without essential prescriptions for weeks or months.
For patients who rely on medical devices, the risks are even more immediate. Ventilators, oxygen concentrators, feeding pumps, and dialysis machines depend on stable electricity. In Puerto Rico, after Hurricane Maria in 2017, widespread power outages lasted for months, creating life-threatening conditions for people reliant on at-home medical devices. For rare disease patients, even a few hours without access to powered devices can result in organ damage or death.
Finally, climate disasters can derail diagnostic processes and clinical research. Many rare disease patients already endure years-long diagnostic odysseys, and interruptions caused by weather events further extend these delays. Laboratories may shut down, imaging centers may close, and specialists may become unavailable during crises. Similarly, rare disease clinical trials, which are already limited, can be postponed or cancelled when sites are damaged, travel is unsafe, or resources are diverted. For patients depending on trial participation as their only chance at an effective therapy, this can be devastating.
In short, climate disasters expose just how fragile the healthcare system is for rare disease patients. What for others might be a temporary inconvenience can, for this community, mean progression of disease, organ failure, or even loss of life. The intersection of climate vulnerability and rare disease care highlights the urgent need for climate-resilient healthcare systems designed with the most medically fragile populations in mind.
The Mental Health Toll of Climate Anxiety
Climate change is not only a physical health crisis but also an emotional and psychological one. For rare disease patients and their caregivers, the mental health burden of living with a chronic, poorly understood condition is already immense. Climate change adds another layer of distress, creating what experts call “climate anxiety”: a chronic fear of environmental catastrophe and its impact on health, safety, and survival. For rare disease patients and their caregivers, this anxiety is not abstract; it is deeply personal and grounded in lived reality.
Rare disease patients often require complex care routines that depend on stability; regular access to clinics, refrigeration for medications, a reliable power supply for medical devices, and predictable schedules for treatments. When climate disasters threaten these lifelines, the resulting anxiety can be overwhelming. The uncertainty of “what if the power goes out?” or “what if the next flood blocks access to the hospital?” weighs heavily, especially for families who have already fought tirelessly for proper care.
Studies show that climate change is already contributing to a rise in mental health disorders worldwide. A systematic review published in Nature Climate Change found that climate-related events are strongly linked to increased anxiety, depression, and post-traumatic stress disorder (PTSD). After Hurricane Katrina, for example, rates of PTSD among survivors were as high as 49%, with chronic health patients disproportionately affected.
For caregivers in the rare disease community, climate anxiety takes on a unique form. They often bear the dual responsibility of managing their loved one’s medical needs while preparing for unpredictable disasters. This can result in heightened stress, caregiver burnout, and feelings of helplessness. The emotional toll is compounded when children are involved, as parents worry not just about immediate survival but also about their child’s long-term future.
Isolation also amplifies these struggles. Many rare disease families already feel disconnected from mainstream healthcare systems due to the rarity of their conditions. When disasters cut off communication or transportation, these feelings of being “forgotten” intensify, worsening mental health outcomes.
What makes this particularly concerning is that mental health resources remain limited for rare disease patients. Many are already underserved by mental health professionals who lack knowledge of their unique medical challenges. The looming threat of climate change adds another psychological weight to a community that is already fighting to be seen, heard, and supported. Without proactive investment in mental health services, the intersection of rare disease and climate anxiety risks becoming a silent crisis.
The Economic Strain on Families
The financial burden of living with a rare disease is already immense. Studies show that families affected by rare diseases in the United States spend nearly 10 times more on healthcare annually than the average household, with costs spanning from specialized treatments to frequent hospital visits and supportive care. Climate change adds yet another layer of economic strain, pushing already fragile families into deeper financial insecurity.
One of the most immediate costs comes from energy use. Heatwaves, which are becoming longer and more intense, force families to rely heavily on air conditioning to prevent dangerous overheating, particularly for patients with neuromuscular or metabolic conditions who struggle to regulate body temperature. At the same time, many rare disease patients depend on electricity for medical devices such as ventilators, feeding pumps, or oxygen concentrators. Rising utility bills, combined with the risk of power outages, create both financial and safety pressures that are difficult to manage.
Another hidden cost is the storage and preservation of medications. Many rare disease treatments are biologics or enzyme replacement therapies that must be kept under strict refrigeration. Power outages during storms or extreme heat events can render these medications useless, forcing families to replace them—often at astronomical prices, sometimes amounting to tens of thousands of dollars for a single treatment cycle. For uninsured or underinsured families, this financial loss can be catastrophic.
Climate disasters also create indirect financial burdens. Families may be forced to evacuate, leading to expenses for temporary housing, transportation, and emergency supplies. Caregivers may lose income if workplaces shut down or if they are unable to work while managing disaster-related care responsibilities. After Hurricane Maria in Puerto Rico, studies reported that individuals with chronic or rare conditions not only faced months-long healthcare disruptions but also devastating economic hardship, as recovery efforts left them without access to employment and increased household costs.
Finally, insurance gaps magnify this strain. While some emergency assistance programs exist, most health insurance policies are not designed to cover the unique costs rare disease families incur during climate disasters. This leaves families caught in a cycle of medical and financial vulnerability, where survival requires not only resilience but also significant financial resources.
For the rare disease community, the economic impact of climate change is more than just a household budget issue—it is a matter of survival. Rising costs, combined with systemic gaps in insurance and disaster preparedness, highlight the urgent need for policy solutions that address the intersection of climate resilience and rare disease care.
The Need for Climate-Resilient Healthcare for Rare Diseases
As climate change accelerates, it has become clear that healthcare systems are not adequately equipped to protect those living with rare diseases. While climate-related disruptions affect everyone, rare disease patients experience consequences that are both immediate and life-threatening. Building climate-resilient healthcare is no longer optional; it is a necessity if we are to safeguard these medically fragile communities.
One of the most urgent priorities is emergency preparedness tailored to rare disease needs. Current disaster response frameworks rarely account for the complexities of rare conditions. For example, evacuation centers may not have refrigeration for temperature-sensitive medications, backup power for ventilators, or staff trained to handle rare metabolic or genetic disorders. Advocacy groups have emphasized the importance of integrating rare disease considerations into emergency planning, including designated medical shelters, priority access to backup power, and stockpiles of critical medications.
Telehealth and digital care solutions also represent lifelines during climate disruptions. The COVID-19 pandemic demonstrated how telemedicine can bridge gaps in access for patients who cannot travel to specialists. Expanding telehealth infrastructure, including secure digital platforms and insurance coverage, can help ensure continuity of care when physical access to clinics or hospitals is cut off by disasters. For rare disease patients, telehealth can mean the difference between stability and crisis during climate emergencies.
At the policy level, governments and healthcare systems must adopt reforms that anticipate climate threats. This includes strengthening cold-chain systems for medications, ensuring hospitals have reliable backup power, and developing patient registries that can help locate and support rare disease patients during emergencies. The World Health Organization (WHO) has already called for climate action as a public health priority, noting that protecting vulnerable populations must be central to national adaptation strategies. Rare disease patients, however, are often overlooked in these frameworks.
Equity is another critical factor. Families living with rare diseases already spend significantly more on healthcare than the general population, and they cannot bear the additional costs of climate adaptation alone. Policies that provide financial protections such as subsidies for backup power, expanded insurance coverage for equipment loss, and disaster relief funds specifically for medically vulnerable populations can help reduce the disproportionate burden on rare disease households.
Advocacy and Community Voices
When it comes to addressing the intersection of climate change and rare diseases, advocacy is not only helpful but also essential. Patients with rare diseases and their caregivers are often experts in resilience, navigating fragmented healthcare systems and constant uncertainty. Their voices bring a human dimension to climate discussions that might otherwise remain abstract. By sharing their lived experiences, they highlight the urgent need for climate action that protects the most medically fragile populations.
Patient advocacy organizations are playing an increasingly critical role in bridging this gap. Expanding their missions to include climate resilience ensures that policymakers and healthcare leaders recognize the unique vulnerabilities of rare disease families. For instance, advocating for emergency preparedness protocols that specifically address rare conditions, such as stockpiling medications, safeguarding cold-chain storage, and prioritizing power access for life-supporting medical devices, can transform how communities respond to disasters.
Partnerships between researchers, advocacy groups, and public health organizations can further amplify these voices. Including rare disease communities in climate and health research ensures that their needs are reflected in adaptation strategies and policy reforms. This kind of collaboration also opens the door to innovative solutions, such as digital health registries to locate patients during emergencies, or specialized support networks that connect families across disaster-prone regions.
Most importantly, advocacy creates visibility. Rare disease patients are often overlooked in broader health policies because they represent a small percentage of the population. But by joining climate action conversations, they can ensure their community is not left behind in adaptation planning. Climate change is a collective crisis, and solutions must be inclusive—designed not just for the many, but also for those who are most at risk.
Conclusion
Climate change is more than an environmental challenge; it is a pressing health equity issue that deeply affects the lives of rare disease patients and their families. From disrupted medication supply chains to the mental strain of navigating disasters with fragile health needs, the consequences are profound and far-reaching. While these challenges are daunting, they also highlight the resilience of the rare disease community and the urgent need for inclusive solutions.
By amplifying patient voices, investing in climate-resilient healthcare systems, and integrating rare disease perspectives into policy planning, we can build a future where no patient is left behind during times of crisis. Advocacy, preparedness, and collaboration between healthcare providers, policymakers, and patient organizations will be critical to ensuring that rare disease communities are protected against the growing threats of a changing climate.
Ultimately, climate resilience is not just about protecting infrastructure; it is about protecting people. For rare disease patients, this means guaranteeing continuous access to care, medications, and support, even under the most unpredictable circumstances. As we face a rapidly shifting climate, we must ensure that the rare disease community is seen, heard, and included in every step of the journey toward a healthier, more equitable future.
Sources
Comments