How Health Systems, Pharmacy Teams Tackle Rare Disease Therapy Barriers: David Mitchell, PharmD, MBA

In part 1 of this 2-part Q&A, David Mitchell, PharmD, MBA, senior pharmacist manager and assistant clinical professor at UC Davis, discusses how health systems and specialty pharmacy teams are stepping up to ensure patients with rare diseases receive the care they need in a health care landscape where rare disease therapies are becoming increasingly complex.

From navigating insurance authorizations to identifying eligible patients and managing therapy coordination, these teams are at the forefront of breaking down barriers and advancing innovative treatments for rare diseases.

This transcript was lightly edited for clarity.

The American Journal of Managed Care® (AJMC®): Why is a strategy targeting rare diseases important for health?

Mitchell: I think a strategy for targeting rare disease is important for health systems because health systems are where care for patients with rare disease mainly happens. Health systems have specialists who focus on these disease states that patients are afflicted with. At health systems, that’s where these patients are receiving care. It’s important to have a strategy for the patients we care for.

I also think we’re very well aligned to provide the best care because we have those specialists, and we also have the infrastructure to support clinical trials that are important for these patients in bringing therapies to market. Many of us have health system specialty pharmacies that can help navigate the patient journey and overcome barriers, such as financial assistance and authorizations required for these therapies and for patients to be able to access them.

I also think rare disease aligns with health systems’ innovative leadership and mission. Many health systems are focused on bringing innovative research and therapies not only to their patients but also to the market and to care in general. I think the mission and vision of most health systems, including us at UC Davis Health, are very well aligned with rare disease.

AJMC: What issues does the pharmacy team resolve when they're involved with these therapies?

Mitchell: I think there are a number of issues that health system specialty pharmacies can help address for patients on innovative therapies, particularly in rare diseases. Many of these barriers also exist for therapies that aren’t for rare diseases, but they can sometimes be even greater for rare disease patients.

One example is the initial authorization for therapies. Helping patients navigate that authorization process is something we specialize in. Another big piece, once authorization is obtained, is ensuring the patient can actually afford the medication.

Backing up to the broader picture—health system specialty pharmacies can help resolve different issues for patients on rare disease therapies. One area where we’re making a big impact is with our pharmacy informatics teams, who help identify patients that could benefit from these therapies. Because these are rare diseases, identifying patients just from their clinic visits can be challenging. But with access to the EMR [electronic medical record] and our data capabilities, we can work with providers to find those patients. Patient identification is 1 barrier we can help overcome.

Once a patient is identified, we also support clinical and therapy coordination throughout the patient journey. Our clinical pharmacists help screen medications to make sure they are safe and effective for each patient, and they provide education. We also have specialists who help with authorization barriers, which can be significant with these therapies. Then, once a therapy is authorized, one of the biggest challenges is overcoming financial barriers and patient cost sharing. We have specialists who work with foundations and manufacturers to find innovative strategies to ensure patients can afford their medications.

AJMC: What are the barriers that the health system faces with rare diseases and cell and therapy programs?

Mitchell: I think there are a number of barriers that patients with rare disease face. One of the biggest, especially with cell and gene therapy programs, is financial risk. Providers and health systems themselves can sometimes be on the hook for these therapies. Having a team that understands both the therapies and the payer landscape helps ensure that neither the patient, the provider, nor the health system ends up bearing that burden and that the therapies are properly covered. I think that’s a big piece.

There are also operational complexities around the preparation and administration of these medications, and health systems—especially those that utilize pharmacy in these processes—are well positioned to help manage that. Another key area where pharmacy plays a role is with the limited distribution nature of some of these medications. Working with manufacturers to gain access so that therapies can be administered in our care settings, and documenting everything in the EHR [electronic health record], is essential.

As I mentioned before, tracking and trending that data in the EHR for safety and efficacy is very important for transparency. For example, when a therapy is dispensed through our specialty pharmacy, providers can see that it was dispensed and also view any interactions pharmacy staff had with the patient. That transparency across the process is really valuable.

I also think skills and training play a big role. Because of our experience in health systems and the fact that we’ve evolved alongside these therapies, we have teams who understand them, have hands-on experience, and are well trained to prescribe, monitor, and administer them. That makes health systems particularly well suited to care for patients with rare diseases.

AJMC: What is the role of the pharmacy team in overcoming these barriers?

Mitchell: I think the role of the pharmacy team in overcoming barriers within rare disease—both in onboarding and maintaining these therapies—is vast. It really spans all levels, from evaluating whether a patient is a good candidate for therapy to ensuring the financial, billing, and coverage aspects are in place. We have pharmacy personnel who are deeply involved in all of those steps.

Advocating for access is also a critical part of the role. That means working with manufacturers to obtain access to medications but also advocating for patients by working with payers—providing evidence that a patient meets criteria and is an appropriate candidate for therapy.

A major component of access is the financial piece. Many of our staff have become experts in the financial landscape of these therapies. That could mean working with foundations, coordinating with manufacturers in free-drug programs, or navigating other avenues to address financial barriers.

As I mentioned before, data is also a big piece. These patient populations are small, which makes identifying and tracking them especially challenging. Our pharmacy personnel have developed strong expertise in health care informatics. Having everything documented in the EMR and being able to use that data to identify, monitor, and track patients is critical. We have specialists in pharmacy and health care informatics who bring that expertise, ensuring that patients are identified, monitored, and supported throughout their treatment journey.