Entering our third year of coping with a pandemic that continues to disrupt lives brings into ever sharper focus the multiple difficulties involved for patients with rare and complex diseases. COVID-19 piles on detours, delays, and additional obstacles to an already challenging journey. The following are five ways the COVID-19 pandemic has impacted immunocompromised and rare disease patients, including statistics and expert opinions on this increasingly important subject.

1) For the immunocompromised, significant concerns with no clear-cut solutions: Patients with weakened immune systems are at a higher risk of severe complications from COVID-19. At the same time, these patients still encounter a high degree of uncertainty regarding the vaccines' safety and efficacy due to their immunocompromised condition.

A true Cornelian dilemma is defined as a situation in which one is obliged to choose between two courses of action, either of which will have a detrimental effect on themselves or someone near them. The dangers of getting COVID-19 are highlighted continuously for those with weakened immune systems, including the 3% of the population taking immunosuppressants for various rare and complex conditions. However, as Frontiers in Public Health reports, few patients with rare diseases present with COVID-19, perhaps because of their awareness of risks and preventive measures. As a result, only a few small cohort studies and case reports on the effects of COVID-19 on people with rare diseases are available.

The case for COVID-19 vaccines faces similar barriers. As the need for a third or even fourth vaccine shot emerges, only minimal reassurance is provided, even in the CDC's most recent guidance, dated 1.7.22: "The safety, efficacy, and benefit of the additional primary dose (of mRNA COVID-19 vaccine) in immunocompromised people continue to be evaluated. So far, reactions reported after the additional primary shot are similar to that of the two-dose primary series. Serious side effects are rare but may occur." 

The European Rare Neurological Diseases (RND) network concurs. They state that it is difficult to provide a well-grounded answer to RND patients who wonder if their disease brings specific risk factors to a COVID-19 vaccination or if they are especially vulnerable to the condition and should get vaccinated on a high priority. "As clinical trials have not been conducted in RND patients, there is no data that indicates specific risks from COVID vaccination for patients with RND including chorea, dystonia, frontotemporal dementia, atypical Parkinsonism and others." 

For physicians and their patients all too familiar with battling conditions experienced by just 4% of the world's population, the rarity of potentially serious side effects from a vaccine intended for protection continues to pose a difficult decision.

2) Confusion surrounding boosters for the immunocompromised: Multiple changes in guidelines surrounding eligibility for third and fourth doses among the immunocompromised have resulted in a reported lack of vaccine access for the population that needs them most.

Vaccine recommendations for immunocompromised people have evolved rapidly since last summer but haven't always been communicated clearly to physicians, health departments, hospitals, or pharmacists. The result has been a rising number of people in this vulnerable population who cannot receive the additional doses essential to protecting them from severe COVID, long-haul COVID, and breakthrough infections. 

3) Decreased giving to non-profits: Organizations providing support to rare disease patients are experiencing a significant decline in donations due to the pandemic. Non-profits are still reeling from the combination of an economic downfall, continued restrictions, and social distancing protocols. According to National Council of Nonprofits CEO Rick Cohen, "Non-profits having to cancel events that they normally hold each year means losses of tens of thousands of dollars resulting in staff layoffs and cutting back on programs that help people in their communities."

For example, one of the country's largest and best-known non-profits, the Muscular Dystrophy Association, reports a 46% decrease in event fundraising in 2020 due to stay-at-home orders, restrictions on in-person gatherings, and impacts to sponsors and donor levels of giving.

4) A slowdown in essential research: Many rare disease clinical trials and research are suspended or abandoned entirely due to the pandemic. As pharmaceutical researchers understandably focus on COVID-19, resources and attention for other disease therapies are lessened. Currently, less than 10% of rare diseases have an FDA-approved treatment, leaving the vast majority of these patients with minimal options and an urgent need to develop additional treatments.

The research landscape for rare diseases is not easy, often requiring collaboration among institutions to gather a significant number of patients to enroll in trials. In the first year of the pandemic, an estimated 80% of non-COVID trials were stopped or interrupted, sparking the establishment of new guidelines from the FDA. Even as some companies proudly announce restarts of trials this year, the Omicron variant may force additional postponements to ensure the safety of its often fragile patient cohorts. Delays in producing treatments for rare or complex conditions can cause patients to backslide to a non-recoverable or unmanageable state.  

5) Uncertainty around a return to normal: For people coping with rare diseases and complex conditions, resuming activities without fear may not be possible for some time. While recommendations reducing isolation and quarantine times for individuals with COVID can help vaccinated people return to work, school, and everyday activities more quickly, the environment continues to pose an unacceptable level of risk for those with compromised immune systems.

As recently reported by NPR, it can mean limiting all but essential social contact and stepped-up vigilance with every new variant. Children with rare neurological diseases, unable to access their often complex network of outside support, experience ongoing delays in growth and development. An adult fighting rare cancer explains, "There's an element of risk no matter where I go. I can't step out into public and not assume that there's somebody unvaccinated nearby." There is a constant, largely publicly unacknowledged fear that even if the pandemic becomes endemic, the immunocompromised and those with rare diseases may experience disproportionate levels of severe complications.
Dr. Dorry Segev, Johns Hopkins University, who studies transplant recipients' response to the COVID vaccine, says, "Right now the best thing we can do for immune-compromised people is for everybody else to get vaccinated so that we protect our vulnerable friends and neighbors."

Providing patients with comprehensive and compassionate care since 1987, Orsini Specialty Pharmacy is the leading independent specialty pharmacy focused on rare diseases, gene therapies, and complex conditions. Orsini's high-touch care model centers around experienced, therapy-specific teams that provide personalized care to patients. Learn more about Orsini's essential services for rare and complex disease therapies and our dedicated support for physicians, manufacturers, payors, and patients. You can also reach out to us to learn how we're supporting our patients during this ever-changing and challenging time.