Members of the rare disease community—patients, families and caregivers—are accustomed to managing life in crisis mode, cancelling plans at the first sign of illness and feeling isolated, but the pandemic has introduced a new level of concern. With so much focus on COVID-19, rare disease patients are left with fear and uncertainty over healthcare systems’ bandwidth to accommodate their needs. It’s possible that they could experience critical gaps in care like product shortages, fewer healthcare workers available to administer drugs and difficulty getting to infusion appointments. Unfortunately, the response to many of today’s questions about what the novel coronavirus means for rare disease patients is, “We don’t know, exactly.” But we need to ensure that we’re exploring new alternatives and thinking creatively to develop solutions that either ease these tensions for patients or create bridges that are meaningful, empathetic, supportive and, of course, compliant.


In the healthcare industry, providers and pharmaceutical companies have shifted their focus to caring for and addressing the needs of patients with COVID-19. While this needs to remain a top priority, we believe that pharma also can help to identify and troubleshoot some of the critical issues that rare patients are now facing. In doing so, pharma can show the rare disease patient community that the well-being, both emotionally and physically, of all patients is at the center of the industry’s mission.


The emotional and treatment impact on rare disease patients and families are important pillars to guide pharma’s action planning. But in order to truly understand what’s necessary “on the ground” for our patients, pharma companies need to first and foremost reach out to and engage patients. We can’t solely rely on our industry’s leaders—even the most compassionate and educated among us—to develop the right solutions around a conference room table, or rather via today’s virtual conference room, without including the voice of the patient.

Patients and families regularly interact with others in the rare disease community to share experiences and lift each other up. While patients and advocacy groups already accomplish a lot of this on social media platforms, the pandemic has shifted all social interactions to virtual technologies like tele- and video-conferencing. That means that patient communities are faced with indefinite postponements and cancellations of family meet-ups, conferences and other in-person events. Patients count on these events to feel connected (let alone their importance for critical fundraising and general awareness). Parents of children with rare diseases often rely on a large team of educational professionals and therapists to provide daily services. These in-person interactions also have disappeared.


There are a few ways that pharma companies can help patients who are feeling more isolated than ever due to the present circumstances. For one, many companies with rare disease assets or pipelines have patient case managers or similar roles that regularly engage with patients and can help them feel more connected virtually. Several companies have shared that they’re evaluating partnerships with advocacy organizations to help rare disease communities create virtual hangouts and meetings. We applaud these efforts as reaching out to patients, and communicating honestly and transparently, is critical for their emotional (and likely physical) well-being.


A few pharma patient services leaders who work in the rare disease space already have shared a few positive examples of this approach in action. Specifically, one company created an online FAQ offering that quickly answers the most critical questions, which are evolving every day. One client is planning to support patients who are out of work, including looking at what loss of employment might mean for their ability to afford co-pays. Another approach is to leverage expanded access and patient assistance programs to alleviate patients’ financial fears. It’s important, of course, that companies review these new approaches to ensure that they’re compliant. But this crisis is a new and unique time, so resting on preconceived notions of what is possible and what is not will often over-index on the “not.”

Providing treatment support to rare disease patients is a challenging and complex endeavor, as the needs greatly vary among disease states and patients. And that’s true even under normal circumstances. Now the pandemic is further limiting healthcare systems’ resources, and projections suggest that the situation will get worse before it improves. The needs of small and underserved patient communities often get overlooked during normal times, so it’s important that we acknowledge their needs now. That means that we must explore new ways to bring the care to them, determine solutions that maintain therapeutic consistency and create alternative solutions until regular care starts or resumes.


A recent Fierce Healthcare article highlighted Biogen’s concerns that some spinal muscular atrophy patients might need to postpone treatments to avoid exposure to COVID-19 in treatment centers. While a delay in the treatment cycle for some patients may be feasible, we need to ensure that we’re effectively communicating with our patients so that they can make the right decisions and address trade-offs in their decision-making.


In addition to workarounds to delaying treatments (which is not always an option), rare disease patients need creative solutions to the continuity of care challenges that they face. These solutions may include home healthcare (which has its own drawbacks given the criticality of social distancing), scheduling labs and diagnostic tests, and making sure that basic supplies are available. Some patient populations are struggling to get Gatorade (a regular part of their therapy) or distilled water (required to operate a medical device) due to limited supplies on supermarket shelves. Can pharma companies supply patients with distilled water? This may be unlikely to be approved by compliance, but it’s important that we continue to brainstorm creative solutions. Perhaps pharma companies can collaborate with advocacy organizations to help manage the flow of critical goods and services in a compliant yet supportive way. The answers may not always be clear, but it’s important to challenge everyday thinking and everyday norms—particularly during the pandemic.


Pharma companies should start by aspiring to create an informed and more highly supported community of patients. And perhaps along the way we will create an even greater symbiotic relationship between patients and pharma. Regardless, patient-centricity is no longer a “nice to have” or a buzzword. It’s time to prove that it’s at the core of the work we do—during a crisis or not.