Rare disease patients now face yet another health threat in the global coronavirus pandemic. Many of the families with children diagnosed with rare diseases have turned to various social media platforms to share their worries and frustrations.
These parents now find themselves, perhaps for the first time, in a position where parents of healthy children are better equipped to empathise with and understand their struggles.
These parents, however, also recognise that COVID-19 hasn’t just disrupted lives by requiring self-isolation, but has also interrupted important rare disease research, impacted patient care, and led to possible new serious diseases in children.
Self-Isolation for everyone
Parents of ill children may find themselves garnering more sympathy from parents with healthy children who must also isolate their families to protect them from the COVID-19 virus.
But whilst families that include children with rare diseases may have coped with this type of isolation for years, the shut-down of schools, medical facilities, and other services has added new mental and physical challenges.
These parents have become solely responsible for not only their children’s educations, but also the therapies they need. And many caregivers fear that they are poor substitutes for that support.
Responses from healthcare and patient advocacy groups
The pandemic has forced healthcare practitioners, research organisations, and biotechnology companies to shift gears to focus on developing treatments and vaccines to control the virus.
Many of these organisations have halted enrolment in rare disease clinical trials and other rare disease research. They’ve had to close, making it difficult for patients already enrolled in clinical trials to access the care and regimen they need. Critical fundraising has slowed or stopped.
Social media channels feature many frustrated parents sharing their stories and concerns. Patient advocacy groups have shared helpful content within their respective communities.
Access to patient care
The global pandemic has forced the closures of healthcare facilities worldwide, seriously decreasing access to necessary care and negatively impacting rare disease patients’ quality of life.
A Eurodis survey learnt that:
- 60% of patients have experienced interrupted care
- 50%+ of patents have experienced the postponement or cancellation of planned surgeries/transplants
- 80%+ of patients have experienced postponed/cancelled rehabilitation appointments
Telehealth and telemedicine have seen a significant increase in use since the start of the pandemic, helping to offset the significant decrease in in-person care. Of the 70% of patients whose in-person medical appointments were cancelled, 65% were offered alternative appointments via video or telephone.
However, an inability to access care makes diagnosing potentially rare diseases and disorders that much more difficult. Some of the most challenging diseases can take years to accurately identify, leaving patients vulnerable to degenerative conditions and requiring more complex treatments.
PMIS: The “new” Kawasaki disease?
A disturbing new illness has appeared in some children affected by COVID-19: Pediatric Multisystem Inflammatory Disease (PMIS). The UK confirmed its first victim in April, 10 Italian children were with PMIS in March and April, and New York doctors identified 102 school-aged children with PMIS symptoms.
The coronavirus pandemic has infiltrated everywhere, shifting focuses away from rare disease research and clinical trials and complicating access to necessary care. It’s also led to potentially new diseases, like PMIS, which presents similarly to Kawasaki disease, another rare disease.
Additional challenges, frustrations of parents whose children suffer from rare diseases, the response of medical and patient advocacy groups, and the impact of COVID-19 on patient care are outlined in our white paper, “The Impact of COVID-19 on Rare Diseases”.