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COVID-19 Puts Hope on Hold for Rare Disease Patients

The coronavirus pandemic has disrupted nearly every facet of health care.  

Patients and providers alike now don personal protective equipment.  Telemedicine has replaced most in-person visits.  Patients wait in their cars, instead of the waiting room, for the rare in-office appointment.  And the majority of prescriptions are mailed or delivered instead of picked up in person.  

Meanwhile, the pandemic has shaken clinical research, prompting facilities to pause existing clinical trials and delay enrollment in new ones.  The move could be devastating for people living with any condition.  But the frustration is heightened in the rare disease community.  

For some rare diseases, ongoing clinical trials may be  patients’ only source of treatment.  In other cases, approved medications exist but access barriers block patients from treatment.  Clinical trials provide an important alternative.  

This can be true for people living with amyloidosis, for example.  The rare, sometimes hereditary, condition leads to a build-up of amyloid protein in the body’s organs.  It can cause an array of debilitating symptoms and can take years to diagnose.  Three FDA-approved treatments for one type of amyloidosis exist, but patients often face insurance barriers.  The obstacles can be so onerous that clinicians advise patients to join a clinical trial just to get treated.

With clinical trials enrollment at a standstill, however, that avenue to treatment is not available right now.

In addition to cutting off treatment access, postponing clinical trials puts hope on hold for patients and their loved ones.  Even for patients not actively enrolled, a successful clinical trial conveys the promise of progress and new treatments down the road.  That future, and the innovative possibilities it holds, recedes further into the horizon while the world waits for solutions to COVID-19.

This disappointment is featured in a recent survey from the National Organization for Rare Disorders.  One respondent noted that the “newest trial just rolled out and now no one can enroll.”

Patients with rare diseases wait a lot.  They wait for appointments, wait for referrals, wait for insurance approvals or denials.  Now, amid COVID-19, they wait for researchers to identify vaccines and treatments for the novel coronavirus.  Because successful COVID-19 clinical trials won’t just aid the countless people put at risk by the current pandemic.  They will also free up rare disease research to begin in earnest one again. 

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