Impact of COVID-19 on Rare Disease Research

COVID-19 moved from a ubiquitous virus to a full-on pandemic very quickly. As countries and city centers took steps to stem the tide, businesses, and industries of every ilk felt the impact. Some work was delayed, while other projects were canceled as a result of efforts to flatten the curve and reduce the spread of COVID-19. The world of clinical research was no different, especially within the realm of rare disease, and that fact will influence the ability of new treatments and therapeutics to come to market as well as the opportunity for people with severe conditions to access investigational products that could have provided hope, if not relief.

COVID-19 and Clinical Trials

Healthcare facilities had to triage their resources as the incidence of COVID-19 worsened. While some studies were able to continue with modifications, the pandemic fostered a new reality in which contagion was not the only consideration. The COVID-infected required respirators and specialized care, which meant more resources. In some cases, there simply weren’t enough beds or machines to go around. For those medical entities that were able to continue supporting trials, there was the additional issue of qualified investigators. In many cases, health care workers were too busy attending to COVID-19 patients to provide care for study participants, prompting some clinical research to prohibit or limit site visits. There was also an issue with labs. Facilities that offer blood testing, urinalysis, and imaging were overrun with COVID-19 testing or were closed because of the public health crisis.

Rare Disease Complications

These issues existed in almost every clinical trial in-progress or on-deck during the COVID-19 pandemic, but they were compounded in rare diseases. In one Hong Kong-focused study, 46% of subjects with a rare illness experienced a worsening of health status, while 81% reported a financial impact and 89% reported an impact on their quality of daily life as a result of COVID-19. While none of the participants acquired COVID-19, they were unable to receive the same level of care they had enjoyed before the pandemic began.

Access to Treatment

NORD (the National Organization for Rare Disorders) conducted a survey of rare disease during COVID-19 with over 700 participants. Almost 3 out of 4 study participants had medical appointments canceled because of the pandemic, and some 39% of patients with rare diseases had difficulties accessing care because of COVID-19. In some cases, the diagnosis was delayed because an essential visit with a specialist or a diagnostic procedure was postponed, while in other cases, treatments were interrupted.

Many people with rare diseases had their therapeutic interventions postponed, either because the specialist was unavailable or their risk of infection was too high to risk contagion even with social distancing precautions and personal protective equipment (PPE). The impact on treatment delays was highest amongst those that required an injection or surgical procedure, and those patients tended to be the most severely ill. For some people, home health nurses are a possibility, but many more would be unable to afford that level of dedicated care. There have been some increase in-home infusions, fueled by increased access for Medicare patients. Unfortunately, many people with rare diseases may not qualify. In the NORD study, 40% of respondents lost income from COVID-19, and 11% lost their insurance coverage.

Supply Chain Risks

Rare disease patients were also impacted by supply chain interruptions. Take blood transfusions, for example. Donations are down because of the pandemic. Fewer people can come in for blood draws because of social distancing, and many blood drives were canceled. In addition, donors have to be screened for COVID-19, adding another layer of complexity to blood donation.

There is also a shortage of plasma donations for similar reasons, but the Immunoglobulin (Ig) derived from those donations is an important component of several severe illnesses, including primary immune deficiency and myasthenia gravis. Furthermore, there is some interest in using plasma-based therapies to treat COVID-19, which will impact the plasma supply exponentially. Rare diseases also often have complex supply chains that leverage samples from a patient to manufacture personalized treatments.

Research Delays

The impact of COVID-19 on rare diseases will continue even after supply chain issues are resolved and access to treatment restored. The economic downturn caused by the pandemic means that many resources were shifted toward COVID-19 treatments, therapies, and vaccines. For many pharmaceutical companies, this will mean fewer research dollars that can go towards non-infectious disease-related outlays. In addition, lack of access to sites for clinical trials is still a problem. Site activation can take longer, and disruptions have to be managed.

Rare Disease Research During COVID-19

The influence of COVID-19 on clinical trials came through in several different ways; each had a significant impact on the studies in progress and research efforts planned. The FDA and EMA streamlined their processes to allow existing studies to modify their protocols to accommodate and preserve the safety of participants during the COVID-19 pandemic, modifying data collection and including provisions for virtual assessments where possible. However, it is still vital to approach rare disease research during COVID-19 carefully. Site selection alone can make a big difference in how quickly a study can get started and in the quality of the data collected.


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