Rare diseases affect more than just health — they are often a source of significant financial hardship.
Individuals with rare conditions face high, often catastrophic, out-of-pocket costs for medical care, rehabilitation, and specialized equipment. Experts on rare disease care are often few and far between, and patients must cover travel and accommodation costs. Many Persons Living With a Rare Disease (PLWRD) and their caregivers — commonly family members — face loss of income. All in all, these costs present a massive financial burden for many households.
The cost of caring for a rare disease
Over 40% of PLWRD say that they spend over 2 hours a day on tasks related to their rare condition, and 38% miss more than 30 days of work per year for medical reasons. 30% of family caregivers spend more than 6 hours a day on care. The responsibility of caring for a rare condition makes it difficult to maintain employment outside the home, impacting the family’s finances. As over 70% of rare diseases begin in childhood, care requirements can extend over several years, even decades.
In high-income countries, family caregivers provide more than 80% of the care required by individuals with chronic conditions. This percentage is likely to be even higher in low- to middle-income countries. Women are disproportionately affected, representing 70-80% of family caregivers globally.
Life-saving treatments are out of reach for many
The cost burden of rare diseases is not always due to expensive medicines. For example, Persons with Albinism (PWAs) are vulnerable to skin cancer due to the absence of melanin in their skin. Yet, high-SPF sunscreen, an essential tool in preventing skin cancer, is beyond the reach of most people with albinism in low- and middle-income countries. The photosensitivity of PWAs is also painful to the eyes and can make being outdoors very difficult, yet sunglasses are often unavailable or unaffordable to PWAs in regions where the sun is strongest.
Antoine Gliksohn, executive director of the Global Albinism Alliance, says, “It is particularly sad that so much of the suffering from this rare condition of albinism could be prevented were it not for the cost of items that those of us in wealthier countries would consider generally affordable: sunglasses, sun hats, and, most importantly, sunscreen.“
A consistent commitment
Global and national advocacy has contributed towards progress in reducing the financial burden of rare diseases. For example, Colombia has made significant strides in improving access to medications, technologies, and healthcare services for patients with rare diseases. Legislative measures have been put in place to eliminate administrative and economic barriers to care for PLWRDs. Additionally, Colombia has maintained one of the lowest out-of-pocket health expenditures in Latin America, with households contributing approximately 15% of total health spending, compared to the regional average of 30%.
However, recent government decisions have caused a concerning regression in these advancements, creating new barriers to access and deepening the financial and emotional burden on families.
Now more than ever, patient organizations play a critical role in advocating for this community and working collaboratively with other stakeholders to promote progressive policies that protect and advance the rights of rare disease patients. Diego Gil Cardozo, president of the Federación Colombiana de Enfermadedes Raras (FEOCER) says, “By building alliances and fostering a unified approach, we can drive sustainable solutions that restore hope and dignity to thousands of families living with rare diseases in Colombia.”
The importance of investing in rare diseases
To address the debilitating costs shouldered by PLWRD and their families, we need a solution at the global level. That is why Rare Diseases International, together with a coalition of allied civil society organizations, is advocating for a World Health Assembly Resolution calling for a Global Action Plan on Rare Diseases to ensure long-term, sustainable financial protection for PLWRD and their families.
A Global Action Plan on Rare Diseases will create a pivotal platform for collective action and cement rare diseases as a priority on the global health agenda. It will provide governments with a tangible framework for action with clear targets and accountability measures to reach the global health 2035 goals and to make universal healthcare a reality for all, including PLWRD.
