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COVID-19 Lengthens the Journey to Health Equity for Rare Disease Patients

This past year has shone a harsh light on some of our healthcare system’s most glaring flaws, illuminating a divide too deep to be ignored. Limited access to care for COVID-19 for huge swaths of the population at the beginning of the pandemic were later replaced by limited access to the vaccine, sparking a nascent movement centered on identifying and resolving health inequities to unfold at lightning speed. For the 25 million people living with a rare disease, the pandemic exacerbated their status as an underserved patient population facing unique barriers to health equity.

At Orsini, a specialty pharmacy provider of therapies to thousands of people with rare diseases, we recognize the impact of COVID-19 on our patients, further affecting their health outcomes. Without equal access to medical expertise, treatment, and care in more normal times, the challenges faced by rare disease patients during the pandemic continue to resonate. We share these here to raise awareness and raise hope that the hard work and advocacy inspired by this challenging period will result in a stronger and more enduring safety net for our vulnerable patients.

First, it is important to define terms. While ‘health inequity’ and ‘health disparity’ are often used interchangeably, the situation is more nuanced. Health equity refers to the opportunity for everyone to live their healthiest life with equal access to diagnosis and care. Inequities are often viewed as the cause of health disparities, which describe societal barriers to care, including racism, poverty, limited education and career opportunities, sexual orientation, and disabilities.

The path to health equity for rare disease patients is beset with obstacles, including:

  • Difficulty in finding appropriate medical resources to aid in a diagnosis, such as access to specialists and genetic testing
  • Ineffective or unproductive treatment due to unfamiliarity with their disease state and progression
  • Underrepresentation in clinical trials, which are pivotal to a better understanding of their disease and availability of new and promising treatments

Health disparities become inexorably intertwined with health inequity for rare disease patients and are subsequently magnified. For example, living in poverty means a lack of healthcare insurance to defray the cost of preventive care or costly treatments and limits a patient’s access to major medical centers and specialists. Underserved black and Hispanic communities also report the prevalence of a number of rare diseases, including sickle cell disease, Factor XII deficiency, Apert syndrome, Behcet’s syndrome, Kawaski disease, IgA nephropathy, and Hermansky–Pudlak syndrome. Additionally, people of color are frequently left out of clinical trials in which most participants are of European descent, severely limiting the ability to tailor care and create better outcomes.

COVID-19’s Impact

For the entire rare disease population, the impact of COVID-19 was significant and extensive. Limited or no access to hospital care throughout the pandemic caused the cancellation of all but the most critical laboratory tests, corrective surgeries, and transplant procedures. Similarly, the prolonged pause in physical, occupational, and rehabilitation therapies frequently required in the long-term management of rare diseases resulted in hard-earned gains lost and some degree of regression. The need to quarantine was critical for patients in fragile health but resulted in enhanced feelings of isolation and loneliness.

For minorities, the scale was tipped even further. As Linda Goler Blount, president of the Black Women’s Health Imperative noted, The COVID-19 pandemic, which is devastating communities of color, is a painfully fresh reminder of the disparities in our healthcare system. For those living with a rare disease, these disparities are no different: communities of color face unacceptable barriers to accessing a diagnosis and proper treatment.”

As the medical world turned its focus almost solely on COVID-19 treatment and vaccine research, rare disease studies and clinical trials were paused or suspended, potentially causing delayed access to new drugs and management strategies. Fundraising efforts needed to sustain expensive, long-term treatments for rare diseases were paused or stopped. This prompted the National Organization of Rare Diseases (NORD) to launch a program aimed specifically at arming non-profits with tools to successfully pivot to virtual events.

The initial turbulence of the COVID-19 vaccine rollout in winter 2021 created more confusion and concern for rare disease patients, who were frequently not eligible for early vaccination despite many having a higher risk of severe disease due to their underlying condition. As a result, a consortium of rare disease organizations wrote to the Centers for Disease Control (CDC), stating in no uncertain terms the need to provide better guidance to states as to vaccine priority for those with a rare disease and their caregivers.

Today, with vaccines more readily available and the very real promise of restoring normalcy, it is essential to keep working with the same fervor toward the goal of health equity for rare disease patients. At Orsini, we are privileged to do our part by working with payors, pharmaceutical manufacturers, and non-profit organizations to ensure patients receive expert support with financial assistance, insurance eligibility, and coverage for needed therapies. We are also committed to educating our employees about the challenges of health disparities faced by patients they are assisting, along with ways to respond helpfully, sensitively, and with genuine compassion. And we will continue to use these blogs as a platform to advocate for and champion our rare disease patients and their dedicated healthcare providers. Please forward this on and help us share the message.

If you want to learn how Orsini Specialty Pharmacy can support your rare disease patients, we invite you to connect with our team.