Connected Support in Rare Disease Care

Key Points

• Rare disease care is often complex, lengthy and difficult to navigate for patients and caregivers.
• Providers may need added support with specialty workflows such as prior authorizations, benefit verification and hub enrollment.
• Coordination across providers, hub services, biopharma and specialty pharmacy can help create a clearer, more consistent care experience.

Rare disease care is rarely straightforward. For patients and caregivers, the journey to understanding unexplained symptoms is filled with questions, challenges and moments of vulnerability. The path can be complex and emotionally exhausting, and for many, a diagnosis does not come with a clear treatment option. Even when therapies are available, navigating specialist and insurance requirements can make the care experience feel overwhelming.  

But out of complexity emerges the power of connection. When care is coordinated across providers, hub services, biopharma and specialty pharmacy, patients and providers can experience a transformation: confusion gives way to clarity, isolation becomes support and uncertainty is met with coordinated care across the treatment journey.  

At Biologics by McKesson, we work with patients and providers to weave together the threads of care to create a powerful, connected network.  

“When treatment becomes possible, it can introduce a new kind of stress — one patients and caregivers should feel supported in managing,” said Stephanie Patten, vice president of Rare Disease and Advanced Therapies, Biologics by McKesson. “Patients and caregivers need a care experience that feels connected, clear and supported. Collaboration across the care team can transform what often feels fragmented into coordinated rare disease care – the kind of experience families deserve.” 

What is a rare disease?

According to the National Institutes of Health, a rare disease is defined in the U.S. as a condition that affects fewer than 200,000 people. Collectively, however, an estimated 25-30 million Americans are affected by rare diseases, living with one of the approximately 7,000 identified conditions with a known underlying biological cause.¹

What makes rare disease care complex? 

For many, the challenge begins long before treatment. It can take five or more years on average to receive a diagnosis, and patients may move between specialists while also managing travel demands, insurance questions and financial stress. Additionally, rare diseases are usually serious, progressive or life-limiting with only 5% having an FDA-approved treatment.²  

Why do providers need added support? 

Rare diseases appear across many specialties, including pediatrics, neurology, nephrology, pulmonology and gastroenterology. Because many clinicians may see only a small number of patients with a specific rare condition in their careers, the administrative steps tied to treatment such as prior authorizations, documentation, benefit verification and hub enrollment may be unfamiliar or time-consuming. 

That is why providers often benefit from partners who can help clarify what is needed, when it is needed and how to keep patients moving forward without unnecessary delays. Specialty pharmacy can help in that role by translating complex therapy requirements into more manageable next steps for both the practice and the patient. 

How does specialty pharmacy support rare disease providers?

As part of the rare disease provider team, specialty pharmacy plays a key role by acting as an extension of the practice and helping manage the steps involved in starting and maintaining therapy. This support includes: 

• Setting clear expectations on what information is needed and when. 

• Supporting provider-office workflow and documentation, including prior authorization support, benefit verification and hub enrollment. 

• Keeping practices informed so teams know where a patient is in the treatment process. 

• Providing clinical support services that promote adherence, with rare disease-trained nurses and clinicians who help patients understand and manage their therapy, sharing relevant updates with the provider. 

How does connected care benefit patients and caregivers? 

For patients and caregivers, connected support can make a difficult process feel more manageable. Patients deserve a care experience that feels clear, coordinated and supported, especially when they are already navigating uncertainty, evolving medical information and the emotional strain of a rare diagnosis. 

That is why collaboration matters in rare disease care. When providers, hub services, biopharma and specialty pharmacy work together, patients are better supported as they move from diagnosis to treatment and beyond. 

“At Biologics by McKesson, we’re honored to stand alongside patients, caregivers and the providers who support them,” Patten says. “And we remain committed to building the collaborative relationships that make rare disease care possible.” 

References: 

1. Rare Diseases | National Institutes of Health (NIH) 
2. A comprehensive study of the rare diseases